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CHMP recommends products for thrombosis, hemostasis
Credit: Piotr Bodzek
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending approval for the antiplatelet agent cangrelor (Kengrexal) and a hemostatic sealant powder to be marketed as Raplixa (formerly known as Fibrocaps).
Cangrelor is an intravenous antiplatelet agent that provides immediate and reversible P2Y12 inhibition. It is intended to prevent thrombosis in the acute care setting.
The sealant powder is a ready-to-use, biologically active, powdered fibrin sealant that provides hemostasis in a range of bleeding settings. It is intended for use in patients undergoing surgery.
Neither product has been approved for commercial use in any market, and both are under review by the US Food and Drug Administration as well as the European Medicines Agency/European Commission.
The European Commission generally follows CHMP recommendations for marketing authorizations and delivers its final decision within 3 months of the CHMP recommendation. Decisions are applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About cangrelor
Cangrelor is intended to prevent platelet activation and aggregation that leads to thrombosis in the acute care setting, particularly in patients undergoing percutaneous coronary intervention (PCI).
The CHMP is recommending cangrelor for European marketing authorization based on results from the CHAMPION PHOENIX trial, which was funded by the drug’s developer, The Medicines Company.
This phase 3, randomized, double-blind trial was a comparison of cangrelor and oral clopidogrel in 11,145 patients undergoing PCI.
In the initial analysis of the trial data, researchers found that cangrelor reduced the overall odds of complications from stenting procedures—including death, myocardial infarction, ischemia-driven revascularization, and stent thrombosis—compared to clopidogrel.
However, cangrelor also increased the risk of major and minor bleeding, as well as transient dyspnea.
Results of a second analysis suggested cangrelor can reduce the risk of stent thrombosis alone in patients undergoing PCI, when compared to clopidogrel. Cangrelor was an independent predictor of freedom from stent thrombosis at 30 days after PCI.
About Raplixa
This sealant powder is a mixture of 2 essential blood clotting proteins, fibrinogen and thrombin, formulated as a dry powder for topical use. It is intended to aid hemostasis during surgery.
The CHMP is recommending Raplixa (formerly Fibrocaps) for European marketing authorization based on results of the phase 3 FINISH-3 trial, which was funded by ProFibrix, Inc., the company that was developing the product at the time. (The Medicines Company recently purchased all the outstanding equity of ProFibrix.)
In this randomized, single-blind, controlled trial, researchers compared Raplixa administered with a gelatin sponge to the gelatin sponge alone as a hemostat for surgical bleeding in 4 indications: spinal, hepatic, vascular, and soft tissue dissection.
Raplixa significantly reduced the median time to hemostasis and the restricted mean time to hemostasis, compared with the gelatin sponge alone, for all 4 indications. Raplixa also significantly increased the probability of hemostasis at 3- and 5-minute time points.
The incidence of adverse events was generally similar between the treatment arms. However, non-neutralizing, anti-thrombin antibodies were slightly more common in the sponge-alone arm. 
Credit: Piotr Bodzek
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending approval for the antiplatelet agent cangrelor (Kengrexal) and a hemostatic sealant powder to be marketed as Raplixa (formerly known as Fibrocaps).
Cangrelor is an intravenous antiplatelet agent that provides immediate and reversible P2Y12 inhibition. It is intended to prevent thrombosis in the acute care setting.
The sealant powder is a ready-to-use, biologically active, powdered fibrin sealant that provides hemostasis in a range of bleeding settings. It is intended for use in patients undergoing surgery.
Neither product has been approved for commercial use in any market, and both are under review by the US Food and Drug Administration as well as the European Medicines Agency/European Commission.
The European Commission generally follows CHMP recommendations for marketing authorizations and delivers its final decision within 3 months of the CHMP recommendation. Decisions are applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About cangrelor
Cangrelor is intended to prevent platelet activation and aggregation that leads to thrombosis in the acute care setting, particularly in patients undergoing percutaneous coronary intervention (PCI).
The CHMP is recommending cangrelor for European marketing authorization based on results from the CHAMPION PHOENIX trial, which was funded by the drug’s developer, The Medicines Company.
This phase 3, randomized, double-blind trial was a comparison of cangrelor and oral clopidogrel in 11,145 patients undergoing PCI.
In the initial analysis of the trial data, researchers found that cangrelor reduced the overall odds of complications from stenting procedures—including death, myocardial infarction, ischemia-driven revascularization, and stent thrombosis—compared to clopidogrel.
However, cangrelor also increased the risk of major and minor bleeding, as well as transient dyspnea.
Results of a second analysis suggested cangrelor can reduce the risk of stent thrombosis alone in patients undergoing PCI, when compared to clopidogrel. Cangrelor was an independent predictor of freedom from stent thrombosis at 30 days after PCI.
About Raplixa
This sealant powder is a mixture of 2 essential blood clotting proteins, fibrinogen and thrombin, formulated as a dry powder for topical use. It is intended to aid hemostasis during surgery.
The CHMP is recommending Raplixa (formerly Fibrocaps) for European marketing authorization based on results of the phase 3 FINISH-3 trial, which was funded by ProFibrix, Inc., the company that was developing the product at the time. (The Medicines Company recently purchased all the outstanding equity of ProFibrix.)
In this randomized, single-blind, controlled trial, researchers compared Raplixa administered with a gelatin sponge to the gelatin sponge alone as a hemostat for surgical bleeding in 4 indications: spinal, hepatic, vascular, and soft tissue dissection.
Raplixa significantly reduced the median time to hemostasis and the restricted mean time to hemostasis, compared with the gelatin sponge alone, for all 4 indications. Raplixa also significantly increased the probability of hemostasis at 3- and 5-minute time points.
The incidence of adverse events was generally similar between the treatment arms. However, non-neutralizing, anti-thrombin antibodies were slightly more common in the sponge-alone arm.
Credit: Piotr Bodzek
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending approval for the antiplatelet agent cangrelor (Kengrexal) and a hemostatic sealant powder to be marketed as Raplixa (formerly known as Fibrocaps).
Cangrelor is an intravenous antiplatelet agent that provides immediate and reversible P2Y12 inhibition. It is intended to prevent thrombosis in the acute care setting.
The sealant powder is a ready-to-use, biologically active, powdered fibrin sealant that provides hemostasis in a range of bleeding settings. It is intended for use in patients undergoing surgery.
Neither product has been approved for commercial use in any market, and both are under review by the US Food and Drug Administration as well as the European Medicines Agency/European Commission.
The European Commission generally follows CHMP recommendations for marketing authorizations and delivers its final decision within 3 months of the CHMP recommendation. Decisions are applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About cangrelor
Cangrelor is intended to prevent platelet activation and aggregation that leads to thrombosis in the acute care setting, particularly in patients undergoing percutaneous coronary intervention (PCI).
The CHMP is recommending cangrelor for European marketing authorization based on results from the CHAMPION PHOENIX trial, which was funded by the drug’s developer, The Medicines Company.
This phase 3, randomized, double-blind trial was a comparison of cangrelor and oral clopidogrel in 11,145 patients undergoing PCI.
In the initial analysis of the trial data, researchers found that cangrelor reduced the overall odds of complications from stenting procedures—including death, myocardial infarction, ischemia-driven revascularization, and stent thrombosis—compared to clopidogrel.
However, cangrelor also increased the risk of major and minor bleeding, as well as transient dyspnea.
Results of a second analysis suggested cangrelor can reduce the risk of stent thrombosis alone in patients undergoing PCI, when compared to clopidogrel. Cangrelor was an independent predictor of freedom from stent thrombosis at 30 days after PCI.
About Raplixa
This sealant powder is a mixture of 2 essential blood clotting proteins, fibrinogen and thrombin, formulated as a dry powder for topical use. It is intended to aid hemostasis during surgery.
The CHMP is recommending Raplixa (formerly Fibrocaps) for European marketing authorization based on results of the phase 3 FINISH-3 trial, which was funded by ProFibrix, Inc., the company that was developing the product at the time. (The Medicines Company recently purchased all the outstanding equity of ProFibrix.)
In this randomized, single-blind, controlled trial, researchers compared Raplixa administered with a gelatin sponge to the gelatin sponge alone as a hemostat for surgical bleeding in 4 indications: spinal, hepatic, vascular, and soft tissue dissection.
Raplixa significantly reduced the median time to hemostasis and the restricted mean time to hemostasis, compared with the gelatin sponge alone, for all 4 indications. Raplixa also significantly increased the probability of hemostasis at 3- and 5-minute time points.
The incidence of adverse events was generally similar between the treatment arms. However, non-neutralizing, anti-thrombin antibodies were slightly more common in the sponge-alone arm.
CHMP recommends ruxolitinib for PV
Credit: Zak Hubbard
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending the approval of ruxolitinib (Jakavi) to
treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
The European Commission generally follows the CHMP’s advice and delivers its final decision within 3 months of the CHMP recommendation.
If approved, ruxolitinib would be the first targeted treatment option for PV patients in Europe.
The European Commission’s decision will be applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About ruxolitinib
Ruxolitinib is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. It was approved by the European Commission in August 2012 to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF. The drug is approved to treat MF in more than 70 countries.
Novartis licensed ruxolitinib from Incyte Corporation (which markets the drug as Jakafi in the US) for development and commercialization outside the US. Ruxolitinib is approved in the US to treat patients with MF and those with PV.
Ruxolitinib in PV: The RESPONSE trial
The CHMP’s recommendation to approve ruxolitinib for PV was based on results from the phase 3 RESPONSE trial, which was funded by Incyte.
The trial included 222 patients who had PV for at least 24 weeks. All patients had an inadequate response to or could not tolerate hydroxyurea, had undergone a phlebotomy, and had an enlarged spleen.
They were randomized to receive ruxolitinib starting at 10 mg twice daily or best available therapy (BAT) as determined by the investigator. The ruxolitinib dose was adjusted as needed.
The study was designed to measure the reduced need for phlebotomy beginning at week 8 and continuing through week 32, in addition to at least a 35% reduction in spleen volume at week 32.
Twenty-one percent of ruxolitinib-treated patients met this endpoint, compared to 1% of patients who received BAT.
Ruxolitinib was generally well-tolerated, but 3.6% of patients discontinued treatment due to adverse events, compared to 1.8% of patients on BAT.
The most common hematologic adverse events associated with ruxolitinib were anemia and thrombocytopenia. The most common non-hematologic events were headache, diarrhea, fatigue, dizziness, constipation, and shingles.
Credit: Zak Hubbard
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending the approval of ruxolitinib (Jakavi) to
treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
The European Commission generally follows the CHMP’s advice and delivers its final decision within 3 months of the CHMP recommendation.
If approved, ruxolitinib would be the first targeted treatment option for PV patients in Europe.
The European Commission’s decision will be applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About ruxolitinib
Ruxolitinib is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. It was approved by the European Commission in August 2012 to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF. The drug is approved to treat MF in more than 70 countries.
Novartis licensed ruxolitinib from Incyte Corporation (which markets the drug as Jakafi in the US) for development and commercialization outside the US. Ruxolitinib is approved in the US to treat patients with MF and those with PV.
Ruxolitinib in PV: The RESPONSE trial
The CHMP’s recommendation to approve ruxolitinib for PV was based on results from the phase 3 RESPONSE trial, which was funded by Incyte.
The trial included 222 patients who had PV for at least 24 weeks. All patients had an inadequate response to or could not tolerate hydroxyurea, had undergone a phlebotomy, and had an enlarged spleen.
They were randomized to receive ruxolitinib starting at 10 mg twice daily or best available therapy (BAT) as determined by the investigator. The ruxolitinib dose was adjusted as needed.
The study was designed to measure the reduced need for phlebotomy beginning at week 8 and continuing through week 32, in addition to at least a 35% reduction in spleen volume at week 32.
Twenty-one percent of ruxolitinib-treated patients met this endpoint, compared to 1% of patients who received BAT.
Ruxolitinib was generally well-tolerated, but 3.6% of patients discontinued treatment due to adverse events, compared to 1.8% of patients on BAT.
The most common hematologic adverse events associated with ruxolitinib were anemia and thrombocytopenia. The most common non-hematologic events were headache, diarrhea, fatigue, dizziness, constipation, and shingles.
Credit: Zak Hubbard
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is recommending the approval of ruxolitinib (Jakavi) to
treat adults with polycythemia vera (PV) who are resistant to or cannot tolerate hydroxyurea.
The European Commission generally follows the CHMP’s advice and delivers its final decision within 3 months of the CHMP recommendation.
If approved, ruxolitinib would be the first targeted treatment option for PV patients in Europe.
The European Commission’s decision will be applicable to all 28 member states of the European Union, plus Iceland, Norway, and Liechtenstein.
About ruxolitinib
Ruxolitinib is an oral inhibitor of the JAK 1 and JAK 2 tyrosine kinases. It was approved by the European Commission in August 2012 to treat adults with primary myelofibrosis (MF), post-PV MF, or post-essential thrombocythemia MF. The drug is approved to treat MF in more than 70 countries.
Novartis licensed ruxolitinib from Incyte Corporation (which markets the drug as Jakafi in the US) for development and commercialization outside the US. Ruxolitinib is approved in the US to treat patients with MF and those with PV.
Ruxolitinib in PV: The RESPONSE trial
The CHMP’s recommendation to approve ruxolitinib for PV was based on results from the phase 3 RESPONSE trial, which was funded by Incyte.
The trial included 222 patients who had PV for at least 24 weeks. All patients had an inadequate response to or could not tolerate hydroxyurea, had undergone a phlebotomy, and had an enlarged spleen.
They were randomized to receive ruxolitinib starting at 10 mg twice daily or best available therapy (BAT) as determined by the investigator. The ruxolitinib dose was adjusted as needed.
The study was designed to measure the reduced need for phlebotomy beginning at week 8 and continuing through week 32, in addition to at least a 35% reduction in spleen volume at week 32.
Twenty-one percent of ruxolitinib-treated patients met this endpoint, compared to 1% of patients who received BAT.
Ruxolitinib was generally well-tolerated, but 3.6% of patients discontinued treatment due to adverse events, compared to 1.8% of patients on BAT.
The most common hematologic adverse events associated with ruxolitinib were anemia and thrombocytopenia. The most common non-hematologic events were headache, diarrhea, fatigue, dizziness, constipation, and shingles.
From the Washington Office
Shortly after passage of the $1.1 trillion “Cromnibus,” the House and Senate concluded their legislative business for the 113th Congress and adjourned. Many issues of importance to surgeons and our patients remain unresolved and are expected to be high on the legislative agenda of the 114th Congress in the first quarter of 2015.
Returning as Members of Congress in January will be two physicians whose campaigns were assisted by the active participation of SurgeonsPAC. They are Dr. Ami Bera, an emergency physician from the 7th District of California (Sacramento area) and Dr. Dan Benishek, a general surgeon and fellow of the American College of Surgeons from the 1st District of Michigan (Upper Peninsula). Both Dr. Bera and Dr. Benishek have served as champions for the cause of the legislative agenda supported by the College.
One of the ways by which SurgeonsPAC participated in the re-election campaign of both Members is known as an independent expenditure (IE). The Code of Federal Regulations defines and independent expenditure as an expenditure for a communication that expressly advocates for “the election or defeat of a clearly identified candidate that is not made in cooperation, consultation, or concert with, or at the request or suggestion of, a candidate, a candidate’s authorized committee, or their agents, or a political party committee or its agents.” [11 CFR 100.16(a)]
In August, the Board of Directors of SurgeonsPAC unanimously voted to direct staff of the Division of Advocacy and Health Policy to develop recommendations for independent expenditures for a bipartisan slate of candidates who had a record of being supportive of the College’s legislative agenda and whose election was enough at risk that an independent expenditure by the College would potentially be of significant benefit for their race. In October, the PAC Board considered those recommendations and voted to support the expenditure of $100,000 each for IEs for Dr. Bera, a Democrat, and Dr. Benishek, a Republican. Both have been champions on issues such as repeal and replacement of the Sustainable Growth Rate (SGR), medical liability reform, and repeal of the 96-hour rule.
For Dr. Bera, the SurgeonsPAC dollars were utilized for a radio and direct mail campaign that was part of a larger effort in which other physician political action committees participated similarly. For Dr. Benishek, a television ad was produced and run through local cable providers.
On election night, Dr. Benishek was declared the winner, receiving 52.1% of the vote compared with his opponent’s 45.3%. As one of four fellows of the American College of Surgeons in Congress, we look forward to continuing to work with “Dr. Dan” and his excellent staff in his upcoming third term.
Dr. Bera’s race was much closer, as he actually trailed his opponent when election night closed with 49.8% of the vote. Subsequently, with the counting and inclusion of the mail-in ballots specifically targeted by the physician community’s IE effort, Dr. Bera overtook his opponent’s slim margin. Two weeks later, on 19 Nov. 2014, the Associated Press called the election for Dr. Bera, whose 1,400-vote lead at that time was felt to be substantial enough to preclude his opponent making up the difference with the remaining 4,300 provisional ballots that had yet to be counted. Dr. Bera’s CA-7 district race proved to be the most expensive in the nation with an estimated $19.6 million in total expenditures. Despite representing only 0.51% of that total, SurgeonsPAC’s contribution, in the collective with that of other physician organizations, no doubt played a significant role in returning a physician to Congress to continue to champion our causes.
In my opinion, these examples of careful candidate selection and subsequent support of Drs. Benishek and Bera exemplify the importance of a strong political action committee. The ultimate goal of SurgeonsPAC is the election to Congress and retention in Congress of those who support our policy positions and legislative agenda. Though SurgeonsPAC is one of nine physician PACs that can be accurately labeled as “million dollar” (per election cycle) PACs, our relative size and, thus, the number of candidates like Drs. Bera and Benishek we could support would be much greater if the Fellow participation rate more closely resembled that of our colleagues in other physician organizations. However, year after year, only 3%-4% of Fellows contribute, as compared with participation rates of 10%-25% in those physician PACs larger than SurgeonsPAC.
Recently, all Fellows received an e-mail from Dr. Andrew Warshaw, founding SurgeonsPAC Board Chairman and current President of the ACS, urging Fellows to make a $25 donation to SurgeonsPAC. Those wishing to learn more about the critical role SurgeonsPAC plays in our advocacy efforts can log on to www.surgeonspac.org.
Until next month...
Dr. Bailey is a pediatric surgeon and Medical Director, Advocacy for the Division of Advocacy and Health Policy in the ACS offices in Washington, DC.
Shortly after passage of the $1.1 trillion “Cromnibus,” the House and Senate concluded their legislative business for the 113th Congress and adjourned. Many issues of importance to surgeons and our patients remain unresolved and are expected to be high on the legislative agenda of the 114th Congress in the first quarter of 2015.
Returning as Members of Congress in January will be two physicians whose campaigns were assisted by the active participation of SurgeonsPAC. They are Dr. Ami Bera, an emergency physician from the 7th District of California (Sacramento area) and Dr. Dan Benishek, a general surgeon and fellow of the American College of Surgeons from the 1st District of Michigan (Upper Peninsula). Both Dr. Bera and Dr. Benishek have served as champions for the cause of the legislative agenda supported by the College.
One of the ways by which SurgeonsPAC participated in the re-election campaign of both Members is known as an independent expenditure (IE). The Code of Federal Regulations defines and independent expenditure as an expenditure for a communication that expressly advocates for “the election or defeat of a clearly identified candidate that is not made in cooperation, consultation, or concert with, or at the request or suggestion of, a candidate, a candidate’s authorized committee, or their agents, or a political party committee or its agents.” [11 CFR 100.16(a)]
In August, the Board of Directors of SurgeonsPAC unanimously voted to direct staff of the Division of Advocacy and Health Policy to develop recommendations for independent expenditures for a bipartisan slate of candidates who had a record of being supportive of the College’s legislative agenda and whose election was enough at risk that an independent expenditure by the College would potentially be of significant benefit for their race. In October, the PAC Board considered those recommendations and voted to support the expenditure of $100,000 each for IEs for Dr. Bera, a Democrat, and Dr. Benishek, a Republican. Both have been champions on issues such as repeal and replacement of the Sustainable Growth Rate (SGR), medical liability reform, and repeal of the 96-hour rule.
For Dr. Bera, the SurgeonsPAC dollars were utilized for a radio and direct mail campaign that was part of a larger effort in which other physician political action committees participated similarly. For Dr. Benishek, a television ad was produced and run through local cable providers.
On election night, Dr. Benishek was declared the winner, receiving 52.1% of the vote compared with his opponent’s 45.3%. As one of four fellows of the American College of Surgeons in Congress, we look forward to continuing to work with “Dr. Dan” and his excellent staff in his upcoming third term.
Dr. Bera’s race was much closer, as he actually trailed his opponent when election night closed with 49.8% of the vote. Subsequently, with the counting and inclusion of the mail-in ballots specifically targeted by the physician community’s IE effort, Dr. Bera overtook his opponent’s slim margin. Two weeks later, on 19 Nov. 2014, the Associated Press called the election for Dr. Bera, whose 1,400-vote lead at that time was felt to be substantial enough to preclude his opponent making up the difference with the remaining 4,300 provisional ballots that had yet to be counted. Dr. Bera’s CA-7 district race proved to be the most expensive in the nation with an estimated $19.6 million in total expenditures. Despite representing only 0.51% of that total, SurgeonsPAC’s contribution, in the collective with that of other physician organizations, no doubt played a significant role in returning a physician to Congress to continue to champion our causes.
In my opinion, these examples of careful candidate selection and subsequent support of Drs. Benishek and Bera exemplify the importance of a strong political action committee. The ultimate goal of SurgeonsPAC is the election to Congress and retention in Congress of those who support our policy positions and legislative agenda. Though SurgeonsPAC is one of nine physician PACs that can be accurately labeled as “million dollar” (per election cycle) PACs, our relative size and, thus, the number of candidates like Drs. Bera and Benishek we could support would be much greater if the Fellow participation rate more closely resembled that of our colleagues in other physician organizations. However, year after year, only 3%-4% of Fellows contribute, as compared with participation rates of 10%-25% in those physician PACs larger than SurgeonsPAC.
Recently, all Fellows received an e-mail from Dr. Andrew Warshaw, founding SurgeonsPAC Board Chairman and current President of the ACS, urging Fellows to make a $25 donation to SurgeonsPAC. Those wishing to learn more about the critical role SurgeonsPAC plays in our advocacy efforts can log on to www.surgeonspac.org.
Until next month...
Dr. Bailey is a pediatric surgeon and Medical Director, Advocacy for the Division of Advocacy and Health Policy in the ACS offices in Washington, DC.
Shortly after passage of the $1.1 trillion “Cromnibus,” the House and Senate concluded their legislative business for the 113th Congress and adjourned. Many issues of importance to surgeons and our patients remain unresolved and are expected to be high on the legislative agenda of the 114th Congress in the first quarter of 2015.
Returning as Members of Congress in January will be two physicians whose campaigns were assisted by the active participation of SurgeonsPAC. They are Dr. Ami Bera, an emergency physician from the 7th District of California (Sacramento area) and Dr. Dan Benishek, a general surgeon and fellow of the American College of Surgeons from the 1st District of Michigan (Upper Peninsula). Both Dr. Bera and Dr. Benishek have served as champions for the cause of the legislative agenda supported by the College.
One of the ways by which SurgeonsPAC participated in the re-election campaign of both Members is known as an independent expenditure (IE). The Code of Federal Regulations defines and independent expenditure as an expenditure for a communication that expressly advocates for “the election or defeat of a clearly identified candidate that is not made in cooperation, consultation, or concert with, or at the request or suggestion of, a candidate, a candidate’s authorized committee, or their agents, or a political party committee or its agents.” [11 CFR 100.16(a)]
In August, the Board of Directors of SurgeonsPAC unanimously voted to direct staff of the Division of Advocacy and Health Policy to develop recommendations for independent expenditures for a bipartisan slate of candidates who had a record of being supportive of the College’s legislative agenda and whose election was enough at risk that an independent expenditure by the College would potentially be of significant benefit for their race. In October, the PAC Board considered those recommendations and voted to support the expenditure of $100,000 each for IEs for Dr. Bera, a Democrat, and Dr. Benishek, a Republican. Both have been champions on issues such as repeal and replacement of the Sustainable Growth Rate (SGR), medical liability reform, and repeal of the 96-hour rule.
For Dr. Bera, the SurgeonsPAC dollars were utilized for a radio and direct mail campaign that was part of a larger effort in which other physician political action committees participated similarly. For Dr. Benishek, a television ad was produced and run through local cable providers.
On election night, Dr. Benishek was declared the winner, receiving 52.1% of the vote compared with his opponent’s 45.3%. As one of four fellows of the American College of Surgeons in Congress, we look forward to continuing to work with “Dr. Dan” and his excellent staff in his upcoming third term.
Dr. Bera’s race was much closer, as he actually trailed his opponent when election night closed with 49.8% of the vote. Subsequently, with the counting and inclusion of the mail-in ballots specifically targeted by the physician community’s IE effort, Dr. Bera overtook his opponent’s slim margin. Two weeks later, on 19 Nov. 2014, the Associated Press called the election for Dr. Bera, whose 1,400-vote lead at that time was felt to be substantial enough to preclude his opponent making up the difference with the remaining 4,300 provisional ballots that had yet to be counted. Dr. Bera’s CA-7 district race proved to be the most expensive in the nation with an estimated $19.6 million in total expenditures. Despite representing only 0.51% of that total, SurgeonsPAC’s contribution, in the collective with that of other physician organizations, no doubt played a significant role in returning a physician to Congress to continue to champion our causes.
In my opinion, these examples of careful candidate selection and subsequent support of Drs. Benishek and Bera exemplify the importance of a strong political action committee. The ultimate goal of SurgeonsPAC is the election to Congress and retention in Congress of those who support our policy positions and legislative agenda. Though SurgeonsPAC is one of nine physician PACs that can be accurately labeled as “million dollar” (per election cycle) PACs, our relative size and, thus, the number of candidates like Drs. Bera and Benishek we could support would be much greater if the Fellow participation rate more closely resembled that of our colleagues in other physician organizations. However, year after year, only 3%-4% of Fellows contribute, as compared with participation rates of 10%-25% in those physician PACs larger than SurgeonsPAC.
Recently, all Fellows received an e-mail from Dr. Andrew Warshaw, founding SurgeonsPAC Board Chairman and current President of the ACS, urging Fellows to make a $25 donation to SurgeonsPAC. Those wishing to learn more about the critical role SurgeonsPAC plays in our advocacy efforts can log on to www.surgeonspac.org.
Until next month...
Dr. Bailey is a pediatric surgeon and Medical Director, Advocacy for the Division of Advocacy and Health Policy in the ACS offices in Washington, DC.
Rural cancer care – if you build it (and measure it!), they will come
Rural surgeons who provide cancer care face a particular set of challenges. Rural patients tend to be older, sicker, less educated and economically disadvantaged. Rural areas have a higher prevalence of chronic diseases including heart disease and cancer. Rural patients present with more advanced cancers than their urban counterparts. Specific rural regions (i.e., Appalachia) have documented higher cancer incidences and mortality rates.
In addition, there are several barriers to providing cancer care for rural populations. These include poor access to health care services and specialists; geographic barriers preventing access to providers, services, and technology; minimal transportation options for either cancer screening or treatment; limited knowledge of cancer and low participation in screening and other healthy practices; prohibitive costs of screening and cancer treatment; and, in some cases, suboptimal care provided to cancer patients (J. Am. Coll. Surg. 2014;219:814-8; Gosschalk, A. and Carozza, S., “Cancer in rural areas: A literature review,” Rural Healthy People 2010, Vol. 2 [College Station: The Texas A&M University System Health Science Center, 2003]).
Several examples of suboptimal care for rural cancer patients have appeared in professional journals and meetings and in the lay press. Examples of rural cancer care inadequacies include lower use of needle biopsy and sentinel lymph node biopsy techniques for breast cancer patients (Am. J. Surg. 2014;208:382-90; Am. J. Surg. 2013;206:674-81; ACS Surgery News, “Use of minimally invasive biopsy lags in Texas,” February 2013, p. 15); significantly lower rates of radiation treatment in breast lumpectomy patients (USA Today, Nov. 18, 2012); lower rates of adequate lymph node dissection, appropriate chemotherapy, and higher death rates in colon cancer patients (Chow, C.J., ACS Clinical Congress Presentation 2012); higher mastectomy rates and later-stage cancers in breast cancer patients (Jethwa, K., AACR Annual Conference Presentation 2013); higher likelihood of discharge to a skilled nursing facility instead of home in colon cancer patients; and the list goes on and on. These articles all come from academic centers through database studies. It is rare indeed to see data collected and published by the rural centers and providers actually caring for rural cancer patients.
My personal bias is that rural surgeons provide very competent, compassionate, high quality care that allows the cancer patient to remain close to their homes and support systems. This opinion has been reinforced by my involvement with the ACS Advisory Council on Rural Surgery, the rural listserve /ACS Rural Community and through my interaction with surgeons across the country at ACS Chapter meetings and at the Congress.
A study done by Finlayson (Med. Care 1999;37:204-9) documented that nearly 100% of rural patients preferred to receive their care locally, especially if the quality of care was the same as the larger distant hospital. In fact, nearly half of the patients polled would choose to remain local even if the mortality rate at the local hospital was double that of a hospital requiring the patient to travel for care. More recent papers however suggest that patients may be bypassing their local hospitals for care because of concerns about the quality of care provided locally (J. Rural Health 1999;10:70-9; J. Rural Health 2007;23:17-24).
A 2013 ASCO presentation documented that General Surgeons perform a majority of cancer surgeries in the United States (Stizenberg, K.; SSO 66th Annual Cancer Symposium; Abstract 75, March 8, 2013). Only 303 (< 8%) counties in the United States even have a surgical oncologist. A 2014 ASCO presentation estimated a 43% increase in inpatient oncology procedures and a 25% increase in outpatient procedures between 2002 and 2020. By 2025 the total demand for oncology care will rise by 43% (ASCO, “The state of cancer care 2014,” J. Oncol. Prac. 2014;10:119-42). Another study (ACS Surgery News, “Surgeon supply to drop 18% by 2028,” January 2013, p. 1) has estimated that, with predicted future surgeon shortages, general surgeons will be called upon to perform 25% of cases now done by other surgical specialists. General surgeons, both rural and urban, are clearly providing the bulk of cancer care to patients and this trend is on the rise in coming years.
Rural surgeons have certain barriers that prevent them from measuring, documenting, and publicizing the specifics of the care provided to their patients. These surgeons often are in single or small group practices and manage their own businesses. They have no office or hospital staff dedicated to quality endeavors. Financial and time constraints prevent them from being champions of quality care in their communities. Additionally, small numbers of specific cases can result in high statistical complication and mortality rates even if these events happen infrequently. This inability to collect data and assess the quality of care provided can lead to patient outmigration and even “tiering” by third-party payers that forces patients away from their hometown hospitals and providers using financial disincentives.
Rural surgeons can and must now take the lead in collecting, assessing, and reporting data about the care they provide for their communities. This process can be in the form of standardized programs like ACS Rural National Surgical Quality Improvement Program (NSQIP), Surgical Care Improvement Project (SCIP), or Commission on Cancer (CoC) accreditation involvement. Smaller institutions with a single surgeon may not have the financial and staff resources to formally participate but each surgeon can assess what makes up the majority of his or her caseload, whether that is endoscopy or breast or colon cases, and find national benchmarks that can easily be measured. Every endoscopy department can collect data on cecal intubation rates, withdrawal times, adequacy of preps, adenoma detection rates, and appropriate follow-up intervals. Individual surgeons can pick and chose specific benchmarks from NSQIP, Commission on Cancer, National Accreditation Program for Breast Centers (NAPBC), or ACS Committee on Trauma standards documents and use these to evaluate the care they provide. Easy examples might include number of lymph nodes harvested at colon cancer surgery, percentage of breast cancer patients diagnosed with needle biopsy, and percentage treated with conservative surgery and appropriate postop radiation and or chemotherapy, or the percentage of melanoma patients treated per National Comprehensive Cancer Network guidelines. Other ways for institutions to document quality might be to partner with their tertiary referral center for CoC accreditation or just to participate in cancer conferences by videoconferencing. The Commission on Cancer has designated CoC Liason Program Chairs in each state that are available to aid in setting up these types of programs.
It is time for rural surgeons to partner with their hospitals and communities to carry out needs assessments and think of ways to fill those needs. An example might be to work with primary care providers to develop aggressive colon, breast, or lung cancer screening programs in communities with high numbers of late-stage cases. Transparency of the data collected and the care provided in the form of “Quality Reports to the Community” can improve local referral patterns, improve the financial viability of the local hospital, and prove to the community that quality care is being rendered. Pooling the data from a large base of rural surgeons could serve to disprove the academic community notion that rural patients receive suboptimal cancer care.
Dr. Sarap is a practicing general surgeon in Cambridge, Ohio. He is a member of the Advisory Council on Rural Surgery and has been appointed the Commission on Cancer Liaison Program Chair for Ohio.
Rural surgeons who provide cancer care face a particular set of challenges. Rural patients tend to be older, sicker, less educated and economically disadvantaged. Rural areas have a higher prevalence of chronic diseases including heart disease and cancer. Rural patients present with more advanced cancers than their urban counterparts. Specific rural regions (i.e., Appalachia) have documented higher cancer incidences and mortality rates.
In addition, there are several barriers to providing cancer care for rural populations. These include poor access to health care services and specialists; geographic barriers preventing access to providers, services, and technology; minimal transportation options for either cancer screening or treatment; limited knowledge of cancer and low participation in screening and other healthy practices; prohibitive costs of screening and cancer treatment; and, in some cases, suboptimal care provided to cancer patients (J. Am. Coll. Surg. 2014;219:814-8; Gosschalk, A. and Carozza, S., “Cancer in rural areas: A literature review,” Rural Healthy People 2010, Vol. 2 [College Station: The Texas A&M University System Health Science Center, 2003]).
Several examples of suboptimal care for rural cancer patients have appeared in professional journals and meetings and in the lay press. Examples of rural cancer care inadequacies include lower use of needle biopsy and sentinel lymph node biopsy techniques for breast cancer patients (Am. J. Surg. 2014;208:382-90; Am. J. Surg. 2013;206:674-81; ACS Surgery News, “Use of minimally invasive biopsy lags in Texas,” February 2013, p. 15); significantly lower rates of radiation treatment in breast lumpectomy patients (USA Today, Nov. 18, 2012); lower rates of adequate lymph node dissection, appropriate chemotherapy, and higher death rates in colon cancer patients (Chow, C.J., ACS Clinical Congress Presentation 2012); higher mastectomy rates and later-stage cancers in breast cancer patients (Jethwa, K., AACR Annual Conference Presentation 2013); higher likelihood of discharge to a skilled nursing facility instead of home in colon cancer patients; and the list goes on and on. These articles all come from academic centers through database studies. It is rare indeed to see data collected and published by the rural centers and providers actually caring for rural cancer patients.
My personal bias is that rural surgeons provide very competent, compassionate, high quality care that allows the cancer patient to remain close to their homes and support systems. This opinion has been reinforced by my involvement with the ACS Advisory Council on Rural Surgery, the rural listserve /ACS Rural Community and through my interaction with surgeons across the country at ACS Chapter meetings and at the Congress.
A study done by Finlayson (Med. Care 1999;37:204-9) documented that nearly 100% of rural patients preferred to receive their care locally, especially if the quality of care was the same as the larger distant hospital. In fact, nearly half of the patients polled would choose to remain local even if the mortality rate at the local hospital was double that of a hospital requiring the patient to travel for care. More recent papers however suggest that patients may be bypassing their local hospitals for care because of concerns about the quality of care provided locally (J. Rural Health 1999;10:70-9; J. Rural Health 2007;23:17-24).
A 2013 ASCO presentation documented that General Surgeons perform a majority of cancer surgeries in the United States (Stizenberg, K.; SSO 66th Annual Cancer Symposium; Abstract 75, March 8, 2013). Only 303 (< 8%) counties in the United States even have a surgical oncologist. A 2014 ASCO presentation estimated a 43% increase in inpatient oncology procedures and a 25% increase in outpatient procedures between 2002 and 2020. By 2025 the total demand for oncology care will rise by 43% (ASCO, “The state of cancer care 2014,” J. Oncol. Prac. 2014;10:119-42). Another study (ACS Surgery News, “Surgeon supply to drop 18% by 2028,” January 2013, p. 1) has estimated that, with predicted future surgeon shortages, general surgeons will be called upon to perform 25% of cases now done by other surgical specialists. General surgeons, both rural and urban, are clearly providing the bulk of cancer care to patients and this trend is on the rise in coming years.
Rural surgeons have certain barriers that prevent them from measuring, documenting, and publicizing the specifics of the care provided to their patients. These surgeons often are in single or small group practices and manage their own businesses. They have no office or hospital staff dedicated to quality endeavors. Financial and time constraints prevent them from being champions of quality care in their communities. Additionally, small numbers of specific cases can result in high statistical complication and mortality rates even if these events happen infrequently. This inability to collect data and assess the quality of care provided can lead to patient outmigration and even “tiering” by third-party payers that forces patients away from their hometown hospitals and providers using financial disincentives.
Rural surgeons can and must now take the lead in collecting, assessing, and reporting data about the care they provide for their communities. This process can be in the form of standardized programs like ACS Rural National Surgical Quality Improvement Program (NSQIP), Surgical Care Improvement Project (SCIP), or Commission on Cancer (CoC) accreditation involvement. Smaller institutions with a single surgeon may not have the financial and staff resources to formally participate but each surgeon can assess what makes up the majority of his or her caseload, whether that is endoscopy or breast or colon cases, and find national benchmarks that can easily be measured. Every endoscopy department can collect data on cecal intubation rates, withdrawal times, adequacy of preps, adenoma detection rates, and appropriate follow-up intervals. Individual surgeons can pick and chose specific benchmarks from NSQIP, Commission on Cancer, National Accreditation Program for Breast Centers (NAPBC), or ACS Committee on Trauma standards documents and use these to evaluate the care they provide. Easy examples might include number of lymph nodes harvested at colon cancer surgery, percentage of breast cancer patients diagnosed with needle biopsy, and percentage treated with conservative surgery and appropriate postop radiation and or chemotherapy, or the percentage of melanoma patients treated per National Comprehensive Cancer Network guidelines. Other ways for institutions to document quality might be to partner with their tertiary referral center for CoC accreditation or just to participate in cancer conferences by videoconferencing. The Commission on Cancer has designated CoC Liason Program Chairs in each state that are available to aid in setting up these types of programs.
It is time for rural surgeons to partner with their hospitals and communities to carry out needs assessments and think of ways to fill those needs. An example might be to work with primary care providers to develop aggressive colon, breast, or lung cancer screening programs in communities with high numbers of late-stage cases. Transparency of the data collected and the care provided in the form of “Quality Reports to the Community” can improve local referral patterns, improve the financial viability of the local hospital, and prove to the community that quality care is being rendered. Pooling the data from a large base of rural surgeons could serve to disprove the academic community notion that rural patients receive suboptimal cancer care.
Dr. Sarap is a practicing general surgeon in Cambridge, Ohio. He is a member of the Advisory Council on Rural Surgery and has been appointed the Commission on Cancer Liaison Program Chair for Ohio.
Rural surgeons who provide cancer care face a particular set of challenges. Rural patients tend to be older, sicker, less educated and economically disadvantaged. Rural areas have a higher prevalence of chronic diseases including heart disease and cancer. Rural patients present with more advanced cancers than their urban counterparts. Specific rural regions (i.e., Appalachia) have documented higher cancer incidences and mortality rates.
In addition, there are several barriers to providing cancer care for rural populations. These include poor access to health care services and specialists; geographic barriers preventing access to providers, services, and technology; minimal transportation options for either cancer screening or treatment; limited knowledge of cancer and low participation in screening and other healthy practices; prohibitive costs of screening and cancer treatment; and, in some cases, suboptimal care provided to cancer patients (J. Am. Coll. Surg. 2014;219:814-8; Gosschalk, A. and Carozza, S., “Cancer in rural areas: A literature review,” Rural Healthy People 2010, Vol. 2 [College Station: The Texas A&M University System Health Science Center, 2003]).
Several examples of suboptimal care for rural cancer patients have appeared in professional journals and meetings and in the lay press. Examples of rural cancer care inadequacies include lower use of needle biopsy and sentinel lymph node biopsy techniques for breast cancer patients (Am. J. Surg. 2014;208:382-90; Am. J. Surg. 2013;206:674-81; ACS Surgery News, “Use of minimally invasive biopsy lags in Texas,” February 2013, p. 15); significantly lower rates of radiation treatment in breast lumpectomy patients (USA Today, Nov. 18, 2012); lower rates of adequate lymph node dissection, appropriate chemotherapy, and higher death rates in colon cancer patients (Chow, C.J., ACS Clinical Congress Presentation 2012); higher mastectomy rates and later-stage cancers in breast cancer patients (Jethwa, K., AACR Annual Conference Presentation 2013); higher likelihood of discharge to a skilled nursing facility instead of home in colon cancer patients; and the list goes on and on. These articles all come from academic centers through database studies. It is rare indeed to see data collected and published by the rural centers and providers actually caring for rural cancer patients.
My personal bias is that rural surgeons provide very competent, compassionate, high quality care that allows the cancer patient to remain close to their homes and support systems. This opinion has been reinforced by my involvement with the ACS Advisory Council on Rural Surgery, the rural listserve /ACS Rural Community and through my interaction with surgeons across the country at ACS Chapter meetings and at the Congress.
A study done by Finlayson (Med. Care 1999;37:204-9) documented that nearly 100% of rural patients preferred to receive their care locally, especially if the quality of care was the same as the larger distant hospital. In fact, nearly half of the patients polled would choose to remain local even if the mortality rate at the local hospital was double that of a hospital requiring the patient to travel for care. More recent papers however suggest that patients may be bypassing their local hospitals for care because of concerns about the quality of care provided locally (J. Rural Health 1999;10:70-9; J. Rural Health 2007;23:17-24).
A 2013 ASCO presentation documented that General Surgeons perform a majority of cancer surgeries in the United States (Stizenberg, K.; SSO 66th Annual Cancer Symposium; Abstract 75, March 8, 2013). Only 303 (< 8%) counties in the United States even have a surgical oncologist. A 2014 ASCO presentation estimated a 43% increase in inpatient oncology procedures and a 25% increase in outpatient procedures between 2002 and 2020. By 2025 the total demand for oncology care will rise by 43% (ASCO, “The state of cancer care 2014,” J. Oncol. Prac. 2014;10:119-42). Another study (ACS Surgery News, “Surgeon supply to drop 18% by 2028,” January 2013, p. 1) has estimated that, with predicted future surgeon shortages, general surgeons will be called upon to perform 25% of cases now done by other surgical specialists. General surgeons, both rural and urban, are clearly providing the bulk of cancer care to patients and this trend is on the rise in coming years.
Rural surgeons have certain barriers that prevent them from measuring, documenting, and publicizing the specifics of the care provided to their patients. These surgeons often are in single or small group practices and manage their own businesses. They have no office or hospital staff dedicated to quality endeavors. Financial and time constraints prevent them from being champions of quality care in their communities. Additionally, small numbers of specific cases can result in high statistical complication and mortality rates even if these events happen infrequently. This inability to collect data and assess the quality of care provided can lead to patient outmigration and even “tiering” by third-party payers that forces patients away from their hometown hospitals and providers using financial disincentives.
Rural surgeons can and must now take the lead in collecting, assessing, and reporting data about the care they provide for their communities. This process can be in the form of standardized programs like ACS Rural National Surgical Quality Improvement Program (NSQIP), Surgical Care Improvement Project (SCIP), or Commission on Cancer (CoC) accreditation involvement. Smaller institutions with a single surgeon may not have the financial and staff resources to formally participate but each surgeon can assess what makes up the majority of his or her caseload, whether that is endoscopy or breast or colon cases, and find national benchmarks that can easily be measured. Every endoscopy department can collect data on cecal intubation rates, withdrawal times, adequacy of preps, adenoma detection rates, and appropriate follow-up intervals. Individual surgeons can pick and chose specific benchmarks from NSQIP, Commission on Cancer, National Accreditation Program for Breast Centers (NAPBC), or ACS Committee on Trauma standards documents and use these to evaluate the care they provide. Easy examples might include number of lymph nodes harvested at colon cancer surgery, percentage of breast cancer patients diagnosed with needle biopsy, and percentage treated with conservative surgery and appropriate postop radiation and or chemotherapy, or the percentage of melanoma patients treated per National Comprehensive Cancer Network guidelines. Other ways for institutions to document quality might be to partner with their tertiary referral center for CoC accreditation or just to participate in cancer conferences by videoconferencing. The Commission on Cancer has designated CoC Liason Program Chairs in each state that are available to aid in setting up these types of programs.
It is time for rural surgeons to partner with their hospitals and communities to carry out needs assessments and think of ways to fill those needs. An example might be to work with primary care providers to develop aggressive colon, breast, or lung cancer screening programs in communities with high numbers of late-stage cases. Transparency of the data collected and the care provided in the form of “Quality Reports to the Community” can improve local referral patterns, improve the financial viability of the local hospital, and prove to the community that quality care is being rendered. Pooling the data from a large base of rural surgeons could serve to disprove the academic community notion that rural patients receive suboptimal cancer care.
Dr. Sarap is a practicing general surgeon in Cambridge, Ohio. He is a member of the Advisory Council on Rural Surgery and has been appointed the Commission on Cancer Liaison Program Chair for Ohio.
'The Talk'
The surgery chief resident texted me: “Patient in ED with peritonitis. Incarcerated hernia.”
I came to the ED; should be easy I thought. See the patient, have a short conversation since the chief resident would have done all the preop. Of course, things are rarely that simple.
First, she was a Jehovah’s Witness on warfarin with an INR of 4. More significantly, she had end-stage COPD. I reviewed her CT scan and labs and went to see her and could tell without any conversation she needed an operation. I needed more information, but not about her belly pain or when it started or whether she had peritonitis on exam. I needed to know how far she could walk, how much oxygen was she on at home, how much was she sleeping, how much weight had she lost, and could she leave the house.
It took 30 minutes to determine her functional status, whether she would survive the postoperative ICU care, whether she would come off the ventilator, and would she want a tracheostomy. It took another 15 minutes to discuss blood product use. Her husband was present, and I left them to decide what they wanted within the context of her goals for her life. She accepted some blood products, underwent surgery, and did well. She came off the ventilator quickly postop and returned home from the hospital. Although she did well, we had a plan for the ventilator and her goals of care defined should she have done poorly.
He was 72, had stage IV lung cancer, and was too weak for chemotherapy. Now he had free air and peritonitis. The referring institution called me and said he was too much for their surgeons. I suggested perhaps they needed to have “the talk” prior to transport. They were unwilling to do so and sent him by helicopter because he was hypotensive. He clearly needed an operation by all surgical standards. But I wanted to wait for his family and talk with them before taking him to the OR; he was in no condition for any component of informed consent.
The family arrived 30 minutes later. His wife, the daughters who had been helping to take care of him, and two more relatives that had just arrived in town. His wife was clear: The man was sleeping all the time, losing weight, barely eating a couple of bites, unsteady on his feet, and confused when he was awake. I discussed options: The surgeon part of me wanted to “fix the hole,” the ICU doc in me knew he would die in the ICU on a ventilator, and the palliative medicine part of me knew hospice was his best option. I told the family he needed surgery but would likely not leave the hospital alive. I recommended hospice.
The wife was thoughtful and looked at her daughters and said, “We need to send him to hospice.” The wife and daughters were the easiest family I have spoken with in a long time. She was clear about whet he wanted and how much he had declined in the past few months. We were able to get him to inpatient hospice from the ED.
Surgery has changed, at least in my practice. Oh, what would I give for a healthy 17-year-old with appendicitis who gets better within hours of the operating room and my conversation with the family that takes 10 seconds. Instead, I take care of the very old and the very sick and the very chronically ill. My conversations are not “you need surgery;” that is done by the chief resident. My conversations are “tell me your functional status and what do you want if/when things go poorly.” Much more complicated and much less fun. The surgeon wants to fix the hole. I can fix the hole. But have I really helped the patient and his or her family? Although it takes me longer and involves more of my emotions and skills, it really is better for the patients and their families to have “the talk.”
Next time you see a patient who is chronically ill, please have the conversation about functional status and goals of care, before you operate. Or transfer. Perhaps then we can save the patient a helicopter ride to hospice.
Dr. Toevs is a trauma critical care surgeon at Allegheny General Hospital in Pittsburgh. She has a master’s degree in bioethics and board certification in hospice and palliative medicine.
The surgery chief resident texted me: “Patient in ED with peritonitis. Incarcerated hernia.”
I came to the ED; should be easy I thought. See the patient, have a short conversation since the chief resident would have done all the preop. Of course, things are rarely that simple.
First, she was a Jehovah’s Witness on warfarin with an INR of 4. More significantly, she had end-stage COPD. I reviewed her CT scan and labs and went to see her and could tell without any conversation she needed an operation. I needed more information, but not about her belly pain or when it started or whether she had peritonitis on exam. I needed to know how far she could walk, how much oxygen was she on at home, how much was she sleeping, how much weight had she lost, and could she leave the house.
It took 30 minutes to determine her functional status, whether she would survive the postoperative ICU care, whether she would come off the ventilator, and would she want a tracheostomy. It took another 15 minutes to discuss blood product use. Her husband was present, and I left them to decide what they wanted within the context of her goals for her life. She accepted some blood products, underwent surgery, and did well. She came off the ventilator quickly postop and returned home from the hospital. Although she did well, we had a plan for the ventilator and her goals of care defined should she have done poorly.
He was 72, had stage IV lung cancer, and was too weak for chemotherapy. Now he had free air and peritonitis. The referring institution called me and said he was too much for their surgeons. I suggested perhaps they needed to have “the talk” prior to transport. They were unwilling to do so and sent him by helicopter because he was hypotensive. He clearly needed an operation by all surgical standards. But I wanted to wait for his family and talk with them before taking him to the OR; he was in no condition for any component of informed consent.
The family arrived 30 minutes later. His wife, the daughters who had been helping to take care of him, and two more relatives that had just arrived in town. His wife was clear: The man was sleeping all the time, losing weight, barely eating a couple of bites, unsteady on his feet, and confused when he was awake. I discussed options: The surgeon part of me wanted to “fix the hole,” the ICU doc in me knew he would die in the ICU on a ventilator, and the palliative medicine part of me knew hospice was his best option. I told the family he needed surgery but would likely not leave the hospital alive. I recommended hospice.
The wife was thoughtful and looked at her daughters and said, “We need to send him to hospice.” The wife and daughters were the easiest family I have spoken with in a long time. She was clear about whet he wanted and how much he had declined in the past few months. We were able to get him to inpatient hospice from the ED.
Surgery has changed, at least in my practice. Oh, what would I give for a healthy 17-year-old with appendicitis who gets better within hours of the operating room and my conversation with the family that takes 10 seconds. Instead, I take care of the very old and the very sick and the very chronically ill. My conversations are not “you need surgery;” that is done by the chief resident. My conversations are “tell me your functional status and what do you want if/when things go poorly.” Much more complicated and much less fun. The surgeon wants to fix the hole. I can fix the hole. But have I really helped the patient and his or her family? Although it takes me longer and involves more of my emotions and skills, it really is better for the patients and their families to have “the talk.”
Next time you see a patient who is chronically ill, please have the conversation about functional status and goals of care, before you operate. Or transfer. Perhaps then we can save the patient a helicopter ride to hospice.
Dr. Toevs is a trauma critical care surgeon at Allegheny General Hospital in Pittsburgh. She has a master’s degree in bioethics and board certification in hospice and palliative medicine.
The surgery chief resident texted me: “Patient in ED with peritonitis. Incarcerated hernia.”
I came to the ED; should be easy I thought. See the patient, have a short conversation since the chief resident would have done all the preop. Of course, things are rarely that simple.
First, she was a Jehovah’s Witness on warfarin with an INR of 4. More significantly, she had end-stage COPD. I reviewed her CT scan and labs and went to see her and could tell without any conversation she needed an operation. I needed more information, but not about her belly pain or when it started or whether she had peritonitis on exam. I needed to know how far she could walk, how much oxygen was she on at home, how much was she sleeping, how much weight had she lost, and could she leave the house.
It took 30 minutes to determine her functional status, whether she would survive the postoperative ICU care, whether she would come off the ventilator, and would she want a tracheostomy. It took another 15 minutes to discuss blood product use. Her husband was present, and I left them to decide what they wanted within the context of her goals for her life. She accepted some blood products, underwent surgery, and did well. She came off the ventilator quickly postop and returned home from the hospital. Although she did well, we had a plan for the ventilator and her goals of care defined should she have done poorly.
He was 72, had stage IV lung cancer, and was too weak for chemotherapy. Now he had free air and peritonitis. The referring institution called me and said he was too much for their surgeons. I suggested perhaps they needed to have “the talk” prior to transport. They were unwilling to do so and sent him by helicopter because he was hypotensive. He clearly needed an operation by all surgical standards. But I wanted to wait for his family and talk with them before taking him to the OR; he was in no condition for any component of informed consent.
The family arrived 30 minutes later. His wife, the daughters who had been helping to take care of him, and two more relatives that had just arrived in town. His wife was clear: The man was sleeping all the time, losing weight, barely eating a couple of bites, unsteady on his feet, and confused when he was awake. I discussed options: The surgeon part of me wanted to “fix the hole,” the ICU doc in me knew he would die in the ICU on a ventilator, and the palliative medicine part of me knew hospice was his best option. I told the family he needed surgery but would likely not leave the hospital alive. I recommended hospice.
The wife was thoughtful and looked at her daughters and said, “We need to send him to hospice.” The wife and daughters were the easiest family I have spoken with in a long time. She was clear about whet he wanted and how much he had declined in the past few months. We were able to get him to inpatient hospice from the ED.
Surgery has changed, at least in my practice. Oh, what would I give for a healthy 17-year-old with appendicitis who gets better within hours of the operating room and my conversation with the family that takes 10 seconds. Instead, I take care of the very old and the very sick and the very chronically ill. My conversations are not “you need surgery;” that is done by the chief resident. My conversations are “tell me your functional status and what do you want if/when things go poorly.” Much more complicated and much less fun. The surgeon wants to fix the hole. I can fix the hole. But have I really helped the patient and his or her family? Although it takes me longer and involves more of my emotions and skills, it really is better for the patients and their families to have “the talk.”
Next time you see a patient who is chronically ill, please have the conversation about functional status and goals of care, before you operate. Or transfer. Perhaps then we can save the patient a helicopter ride to hospice.
Dr. Toevs is a trauma critical care surgeon at Allegheny General Hospital in Pittsburgh. She has a master’s degree in bioethics and board certification in hospice and palliative medicine.
Use of NIPT reduces invasive testing at one medical center
Following the introduction of noninvasive prenatal testing (NIPT) at the Maine Medical Center in Portland, the number of invasive prenatal diagnostic procedures decreased.
In a retrospective study, Joseph R. Wax, MD, and colleagues gathered statistics on the rates of genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection among women who were at increased risk for aneuploidy. These rates were compared before and after the availability of NIPT.
The study included 1,046 women who underwent NIPT between June 1, 2012, and February 1, 2013, as well as 1,464 women who would have been eligible for NIPT if it had been available between December 1, 2010, and November 30, 2011. All women were aged 35 years or older and had ultrasound findings suggestive of increased risk of aneuploidy, a positive aneuploidy screen, prior trisomic fetus, or parental balanced translocation with increased risk for trisomy 13 or 21. One laboratory performed NIPT after patients received genetic counseling. The two groups were compared by maternal demographics, aneuploidy risk factors, rates of genetic counseling, invasive diagnostic procedures, and trisomy 21 detection.1
Results of the study
A total of 33 fetuses with trisomy 21 were identified by positive aneuploidy screening. After the introduction of NIPT, genetic counseling for aneuploidy risk increased (adjusted odds ratio [aOR], 1.77; P <.0001). However, the overall use of invasive diagnostic testing (aOR, 0.42; P <.0001), including amniocentesis (aOR, 0.37, P <.0001), decreased, although the prenatal diagnosis of trisomy 21 remained similar (88% vs 100%; P = .86).1
Dr. Wax and colleagues concluded that, “NIPT in clinical practice uses more genetic counseling resources but requires significantly fewer invasive procedures to maintain the detection rates of trisomy 21.”1
Reference
1. Wax JR, Cartin A, Chard R, Lucas FL, Pinette MG. Noninvasive prenatal testing: Impact on genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection. J Clinic Ultrasound. 2015;43(1):1–6.
Following the introduction of noninvasive prenatal testing (NIPT) at the Maine Medical Center in Portland, the number of invasive prenatal diagnostic procedures decreased.
In a retrospective study, Joseph R. Wax, MD, and colleagues gathered statistics on the rates of genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection among women who were at increased risk for aneuploidy. These rates were compared before and after the availability of NIPT.
The study included 1,046 women who underwent NIPT between June 1, 2012, and February 1, 2013, as well as 1,464 women who would have been eligible for NIPT if it had been available between December 1, 2010, and November 30, 2011. All women were aged 35 years or older and had ultrasound findings suggestive of increased risk of aneuploidy, a positive aneuploidy screen, prior trisomic fetus, or parental balanced translocation with increased risk for trisomy 13 or 21. One laboratory performed NIPT after patients received genetic counseling. The two groups were compared by maternal demographics, aneuploidy risk factors, rates of genetic counseling, invasive diagnostic procedures, and trisomy 21 detection.1
Results of the study
A total of 33 fetuses with trisomy 21 were identified by positive aneuploidy screening. After the introduction of NIPT, genetic counseling for aneuploidy risk increased (adjusted odds ratio [aOR], 1.77; P <.0001). However, the overall use of invasive diagnostic testing (aOR, 0.42; P <.0001), including amniocentesis (aOR, 0.37, P <.0001), decreased, although the prenatal diagnosis of trisomy 21 remained similar (88% vs 100%; P = .86).1
Dr. Wax and colleagues concluded that, “NIPT in clinical practice uses more genetic counseling resources but requires significantly fewer invasive procedures to maintain the detection rates of trisomy 21.”1
Following the introduction of noninvasive prenatal testing (NIPT) at the Maine Medical Center in Portland, the number of invasive prenatal diagnostic procedures decreased.
In a retrospective study, Joseph R. Wax, MD, and colleagues gathered statistics on the rates of genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection among women who were at increased risk for aneuploidy. These rates were compared before and after the availability of NIPT.
The study included 1,046 women who underwent NIPT between June 1, 2012, and February 1, 2013, as well as 1,464 women who would have been eligible for NIPT if it had been available between December 1, 2010, and November 30, 2011. All women were aged 35 years or older and had ultrasound findings suggestive of increased risk of aneuploidy, a positive aneuploidy screen, prior trisomic fetus, or parental balanced translocation with increased risk for trisomy 13 or 21. One laboratory performed NIPT after patients received genetic counseling. The two groups were compared by maternal demographics, aneuploidy risk factors, rates of genetic counseling, invasive diagnostic procedures, and trisomy 21 detection.1
Results of the study
A total of 33 fetuses with trisomy 21 were identified by positive aneuploidy screening. After the introduction of NIPT, genetic counseling for aneuploidy risk increased (adjusted odds ratio [aOR], 1.77; P <.0001). However, the overall use of invasive diagnostic testing (aOR, 0.42; P <.0001), including amniocentesis (aOR, 0.37, P <.0001), decreased, although the prenatal diagnosis of trisomy 21 remained similar (88% vs 100%; P = .86).1
Dr. Wax and colleagues concluded that, “NIPT in clinical practice uses more genetic counseling resources but requires significantly fewer invasive procedures to maintain the detection rates of trisomy 21.”1
Reference
1. Wax JR, Cartin A, Chard R, Lucas FL, Pinette MG. Noninvasive prenatal testing: Impact on genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection. J Clinic Ultrasound. 2015;43(1):1–6.
Reference
1. Wax JR, Cartin A, Chard R, Lucas FL, Pinette MG. Noninvasive prenatal testing: Impact on genetic counseling, invasive prenatal diagnosis, and trisomy 21 detection. J Clinic Ultrasound. 2015;43(1):1–6.
Helmets for Positional Skull Deformities: A Good Idea or Not?
PRACTICE CHANGER
Do not recommend helmet therapy for positional skull deformity in infants and children. Wearing a helmet causes adverse effects but does not alter the natural course of head growth.1
STRENGTH OF RECOMMENDATION
B: Based on a single-blind, randomized controlled trial (RCT).1
ILLUSTRATIVE CASE
The parents of a 6-month-old girl with moderate plagiocephaly bring their daughter in for a well-child visit. Previously, you had recommended that the parents increase “tummy time” when the baby is awake, change her position in bed, and monitor the progression of the condition. They do not feel these interventions have made a difference in the shape of their daughter’s skull and ask about using a helmet to help correct the deformity. How would you counsel them?
Approximately 45% of infants ages 7 to 12 weeks are estimated to have positional skull deformity (PSD), although three-quarters of them have mild cases.2 The incidence of PSD began to increase in 1992 after the American Academy of Pediatrics (AAP) introduced its “Back to Sleep” campaign, which encouraged parents to place their infants on their back at bedtime to reduce sudden infant death syndrome.3
There are two common forms of PSD: plagiocephaly and brachycephaly.1 Plagiocephaly is unilateral occipital flattening, which may be accompanied by ipsilateral forehead prominence and asymmetrical ears. Brachycephaly is symmetric flattening of the back of the head, which can lead to prominence of the temporal areas, making the head appear wide. The cranial sutures remain open in both kinds of PSD.
Evaluating infants for PSD is part of the routine physical exam, and when the condition is noted, the exam should also differentiate PSD from other causes of skull deformity (eg, craniosynostosis). Infants and preschool-aged children with PSD may score lower on developmental testing than children without skull deformity.4 However, these differences are small and inconsistent (2-3 points on a 100-point scale).4 Skull deformity persists into adolescence in only 1% to 2% of patients.5
Neither the AAP nor the American Academy of Family Physicians has a guideline or consensus statement on PSD. Helmets are intended to correct PSD by fitting closely to an infant’s head but allowing room for the skull to grow at the flattened area.1 A 2011 clinical report by Laughlin et al6 recommended against use of helmets for infants with mild to moderate deformities but stated that there was little evidence of harm. Earlier studies have suggested that physical therapy might be effective for plagiocephaly identified early (ie, when the child is 7 to 8 weeks of age).7,8 Biggs9 suggested considering helmet therapy for infants whose cranial sutures remain open and who do not respond to four to eight weeks of physical therapy for PSD. van Wijk et al1 conducted an RCT to explore the risks and benefits of helmet therapy for children with PSD.
Continue for study summary >>
STUDY SUMMARY
Helmets: No help, some harm
In this single-blind RCT of 84 infants (ages 5 or 6 months) with moderate or severe PSD, helmet therapy (n = 42) was compared to no intervention (allowing natural growth; n = 42). Infants were excluded if they had very severe PSD or skull deformity from another cause, such as torticollis or craniosynostosis.
Infants in the helmet therapy group received a custom-made helmet that they wore 23 hours a day until age 1 year, with regular evaluation by an orthotist and modification of the helmet as necessary to allow skull growth. The control group received usual care and no helmet.
The primary outcome was improvement in skull shape at age 24 months, as measured by the oblique diameter difference index (ODDI; a unitless measurement of plagiocephaly calculated as the ratio of measures of two dimensions of cranial diameter) and the cranioproportional index (CPI; a similar measurement of brachycephaly). Infants were considered fully recovered if they achieved an ODDI score < 104% and a CPI score < 90%. These scores indicate a normal head shape; higher scores indicate worse PSD.
At study’s end, the reduction in ODDI and CPI scores was almost the same in both groups. Ten children in the helmet group (26%) and nine in the control group (23%) experienced complete resolution of their PSD.
Both groups were similar in secondary outcomes of infant motor development, infant quality of life, and parental satisfaction. Parental anxiety was assessed using the Spielberger State-Trait Anxiety Inventory (scores range from 20 to 80; a higher score indicates greater anxiety). There was less parental anxiety in the helmet therapy group (–3.9).
All parents of infants in the helmet therapy group reported at least one adverse effect from the intervention. These effects included skin irritation (96%), bad helmet odor (76%), pain associated with the helmet (33%), and feeling hindered from cuddling their child (77%).
Continue for new evidence on helmets for PSD >>
WHAT’S NEW
Stronger evidence that helmets are not effective
Previously, the evidence on helmets for PSD had been obtained mainly from observational or poorly designed studies with significant flaws.6 This study by van Wijk et al1 included objective measurement of skull deformity, along with clinically meaningful outcomes of parental satisfaction, motor development, and parental anxiety.
It also found that helmet therapy was significantly more expensive than care that focused on waiting for PSD to resolve on its own ($1,935 vs $196, respectively).1
CAVEATS
Results may not apply to all infants with skull deformity
These findings do not apply to infants with very severe PSD or those with skull deformity due to secondary causes.1 In addition, this is the only RCT to date that has assessed helmet use in PSD, so it is possible that future studies will find helmets are effective.
Continue for challenges to implementation >>
CHALLENGES TO IMPLEMENTATION
Parents may find this evidence hard to accept
To appropriately implement this recommendation, a clinician must be comfortable making the assessment of mild, moderate, severe, or very severe PSD. Referral to physical therapy might be appropriate for infants with very severe PSD.
If another clinician or physical therapist recommends helmet therapy—or if a parent requests it—explaining the findings of this study may be challenging. We believe that the reduction in parental anxiety in the helmet group likely occurred because the parents believed that the helmet would accelerate the normal reshaping of the skull shape that occurs spontaneously in almost all infants with PSD. Since this study shows that helmets don’t help correct skull deformities, parents can be assured that a helmet is unnecessary and costly and causes adverse effects.
REFERENCES
1. van Wijk RM, van Vlimmeren LA, Groothuis-Oudshoorn CG, et al. Helmet therapy in infants with positional skull deformation: randomised controlled trial. BMJ. 2014;348:g2741.
2. Mawji A, Vollman AR, Hatfield J, et al. The incidence of positional plagiocephaly: a cohort study. Pediatrics. 2013;132:298-304.
3. Peitsch WK, Keefer CH, LaBrie RA, et al. Incidence of cranial asymmetry in healthy newborns. Pediatrics. 2002;110:e72.
4. Collett BR, Gray KE, Starr JR, et al. Development at age 36 months in children with deformational plagiocephaly. Pediatrics. 2013;131: e109-e115.
5. Roby BB, Finkelstein M, Tibesar RJ, et al. Prevalence of positional plagiocephaly in teens born after the “Back to Sleep” campaign. Otolaryngol Head Neck Surg. 2012;146:823-828.
6. Laughlin J, Luerssen TG, Dias MS; Committee on Practice and Ambulatory Medicine, Section on Neurological Surgery. Prevention and management of positional skull deformities in infants. Pediatrics. 2011;128:1236-1241.
7. van Vlimmeren LA, van der Graaf Y, Boere-Boonekamp MM, et al. Effect of pediatric physical therapy on deformational plagiocephaly in children with positional preference: a randomized controlled trial. Arch Pediatr Adolesc Med. 2008;162:712-718.
8. Vargish, L, Mendoza MD, Ewigman, B. Use physical therapy to head off this deformity in infants. Consider early PT to prevent severe deformational plagiocephaly. J Fam Pract. 2009;58:E1-E3.
9. Biggs WS. Diagnosis and management of positional head deformity. Am Fam Physician. 2003;67:1953-1956.
ACKNOWLEDGEMENT
The PURLs Surveillance System was supported in part by Grant Number UL1RR024999 from the National Center For Research Resources, a Clinical Translational Science Award to the University of Chicago. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center For Research Resources or the National Institutes of Health.
Copyright © 2015. The Family Physicians Inquiries Network. All rights reserved.
Reprinted with permission from the Family Physicians Inquiries Network and The Journal of Family Practice. 2015;64(1):44-46.
PRACTICE CHANGER
Do not recommend helmet therapy for positional skull deformity in infants and children. Wearing a helmet causes adverse effects but does not alter the natural course of head growth.1
STRENGTH OF RECOMMENDATION
B: Based on a single-blind, randomized controlled trial (RCT).1
ILLUSTRATIVE CASE
The parents of a 6-month-old girl with moderate plagiocephaly bring their daughter in for a well-child visit. Previously, you had recommended that the parents increase “tummy time” when the baby is awake, change her position in bed, and monitor the progression of the condition. They do not feel these interventions have made a difference in the shape of their daughter’s skull and ask about using a helmet to help correct the deformity. How would you counsel them?
Approximately 45% of infants ages 7 to 12 weeks are estimated to have positional skull deformity (PSD), although three-quarters of them have mild cases.2 The incidence of PSD began to increase in 1992 after the American Academy of Pediatrics (AAP) introduced its “Back to Sleep” campaign, which encouraged parents to place their infants on their back at bedtime to reduce sudden infant death syndrome.3
There are two common forms of PSD: plagiocephaly and brachycephaly.1 Plagiocephaly is unilateral occipital flattening, which may be accompanied by ipsilateral forehead prominence and asymmetrical ears. Brachycephaly is symmetric flattening of the back of the head, which can lead to prominence of the temporal areas, making the head appear wide. The cranial sutures remain open in both kinds of PSD.
Evaluating infants for PSD is part of the routine physical exam, and when the condition is noted, the exam should also differentiate PSD from other causes of skull deformity (eg, craniosynostosis). Infants and preschool-aged children with PSD may score lower on developmental testing than children without skull deformity.4 However, these differences are small and inconsistent (2-3 points on a 100-point scale).4 Skull deformity persists into adolescence in only 1% to 2% of patients.5
Neither the AAP nor the American Academy of Family Physicians has a guideline or consensus statement on PSD. Helmets are intended to correct PSD by fitting closely to an infant’s head but allowing room for the skull to grow at the flattened area.1 A 2011 clinical report by Laughlin et al6 recommended against use of helmets for infants with mild to moderate deformities but stated that there was little evidence of harm. Earlier studies have suggested that physical therapy might be effective for plagiocephaly identified early (ie, when the child is 7 to 8 weeks of age).7,8 Biggs9 suggested considering helmet therapy for infants whose cranial sutures remain open and who do not respond to four to eight weeks of physical therapy for PSD. van Wijk et al1 conducted an RCT to explore the risks and benefits of helmet therapy for children with PSD.
Continue for study summary >>
STUDY SUMMARY
Helmets: No help, some harm
In this single-blind RCT of 84 infants (ages 5 or 6 months) with moderate or severe PSD, helmet therapy (n = 42) was compared to no intervention (allowing natural growth; n = 42). Infants were excluded if they had very severe PSD or skull deformity from another cause, such as torticollis or craniosynostosis.
Infants in the helmet therapy group received a custom-made helmet that they wore 23 hours a day until age 1 year, with regular evaluation by an orthotist and modification of the helmet as necessary to allow skull growth. The control group received usual care and no helmet.
The primary outcome was improvement in skull shape at age 24 months, as measured by the oblique diameter difference index (ODDI; a unitless measurement of plagiocephaly calculated as the ratio of measures of two dimensions of cranial diameter) and the cranioproportional index (CPI; a similar measurement of brachycephaly). Infants were considered fully recovered if they achieved an ODDI score < 104% and a CPI score < 90%. These scores indicate a normal head shape; higher scores indicate worse PSD.
At study’s end, the reduction in ODDI and CPI scores was almost the same in both groups. Ten children in the helmet group (26%) and nine in the control group (23%) experienced complete resolution of their PSD.
Both groups were similar in secondary outcomes of infant motor development, infant quality of life, and parental satisfaction. Parental anxiety was assessed using the Spielberger State-Trait Anxiety Inventory (scores range from 20 to 80; a higher score indicates greater anxiety). There was less parental anxiety in the helmet therapy group (–3.9).
All parents of infants in the helmet therapy group reported at least one adverse effect from the intervention. These effects included skin irritation (96%), bad helmet odor (76%), pain associated with the helmet (33%), and feeling hindered from cuddling their child (77%).
Continue for new evidence on helmets for PSD >>
WHAT’S NEW
Stronger evidence that helmets are not effective
Previously, the evidence on helmets for PSD had been obtained mainly from observational or poorly designed studies with significant flaws.6 This study by van Wijk et al1 included objective measurement of skull deformity, along with clinically meaningful outcomes of parental satisfaction, motor development, and parental anxiety.
It also found that helmet therapy was significantly more expensive than care that focused on waiting for PSD to resolve on its own ($1,935 vs $196, respectively).1
CAVEATS
Results may not apply to all infants with skull deformity
These findings do not apply to infants with very severe PSD or those with skull deformity due to secondary causes.1 In addition, this is the only RCT to date that has assessed helmet use in PSD, so it is possible that future studies will find helmets are effective.
Continue for challenges to implementation >>
CHALLENGES TO IMPLEMENTATION
Parents may find this evidence hard to accept
To appropriately implement this recommendation, a clinician must be comfortable making the assessment of mild, moderate, severe, or very severe PSD. Referral to physical therapy might be appropriate for infants with very severe PSD.
If another clinician or physical therapist recommends helmet therapy—or if a parent requests it—explaining the findings of this study may be challenging. We believe that the reduction in parental anxiety in the helmet group likely occurred because the parents believed that the helmet would accelerate the normal reshaping of the skull shape that occurs spontaneously in almost all infants with PSD. Since this study shows that helmets don’t help correct skull deformities, parents can be assured that a helmet is unnecessary and costly and causes adverse effects.
REFERENCES
1. van Wijk RM, van Vlimmeren LA, Groothuis-Oudshoorn CG, et al. Helmet therapy in infants with positional skull deformation: randomised controlled trial. BMJ. 2014;348:g2741.
2. Mawji A, Vollman AR, Hatfield J, et al. The incidence of positional plagiocephaly: a cohort study. Pediatrics. 2013;132:298-304.
3. Peitsch WK, Keefer CH, LaBrie RA, et al. Incidence of cranial asymmetry in healthy newborns. Pediatrics. 2002;110:e72.
4. Collett BR, Gray KE, Starr JR, et al. Development at age 36 months in children with deformational plagiocephaly. Pediatrics. 2013;131: e109-e115.
5. Roby BB, Finkelstein M, Tibesar RJ, et al. Prevalence of positional plagiocephaly in teens born after the “Back to Sleep” campaign. Otolaryngol Head Neck Surg. 2012;146:823-828.
6. Laughlin J, Luerssen TG, Dias MS; Committee on Practice and Ambulatory Medicine, Section on Neurological Surgery. Prevention and management of positional skull deformities in infants. Pediatrics. 2011;128:1236-1241.
7. van Vlimmeren LA, van der Graaf Y, Boere-Boonekamp MM, et al. Effect of pediatric physical therapy on deformational plagiocephaly in children with positional preference: a randomized controlled trial. Arch Pediatr Adolesc Med. 2008;162:712-718.
8. Vargish, L, Mendoza MD, Ewigman, B. Use physical therapy to head off this deformity in infants. Consider early PT to prevent severe deformational plagiocephaly. J Fam Pract. 2009;58:E1-E3.
9. Biggs WS. Diagnosis and management of positional head deformity. Am Fam Physician. 2003;67:1953-1956.
ACKNOWLEDGEMENT
The PURLs Surveillance System was supported in part by Grant Number UL1RR024999 from the National Center For Research Resources, a Clinical Translational Science Award to the University of Chicago. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center For Research Resources or the National Institutes of Health.
Copyright © 2015. The Family Physicians Inquiries Network. All rights reserved.
Reprinted with permission from the Family Physicians Inquiries Network and The Journal of Family Practice. 2015;64(1):44-46.
PRACTICE CHANGER
Do not recommend helmet therapy for positional skull deformity in infants and children. Wearing a helmet causes adverse effects but does not alter the natural course of head growth.1
STRENGTH OF RECOMMENDATION
B: Based on a single-blind, randomized controlled trial (RCT).1
ILLUSTRATIVE CASE
The parents of a 6-month-old girl with moderate plagiocephaly bring their daughter in for a well-child visit. Previously, you had recommended that the parents increase “tummy time” when the baby is awake, change her position in bed, and monitor the progression of the condition. They do not feel these interventions have made a difference in the shape of their daughter’s skull and ask about using a helmet to help correct the deformity. How would you counsel them?
Approximately 45% of infants ages 7 to 12 weeks are estimated to have positional skull deformity (PSD), although three-quarters of them have mild cases.2 The incidence of PSD began to increase in 1992 after the American Academy of Pediatrics (AAP) introduced its “Back to Sleep” campaign, which encouraged parents to place their infants on their back at bedtime to reduce sudden infant death syndrome.3
There are two common forms of PSD: plagiocephaly and brachycephaly.1 Plagiocephaly is unilateral occipital flattening, which may be accompanied by ipsilateral forehead prominence and asymmetrical ears. Brachycephaly is symmetric flattening of the back of the head, which can lead to prominence of the temporal areas, making the head appear wide. The cranial sutures remain open in both kinds of PSD.
Evaluating infants for PSD is part of the routine physical exam, and when the condition is noted, the exam should also differentiate PSD from other causes of skull deformity (eg, craniosynostosis). Infants and preschool-aged children with PSD may score lower on developmental testing than children without skull deformity.4 However, these differences are small and inconsistent (2-3 points on a 100-point scale).4 Skull deformity persists into adolescence in only 1% to 2% of patients.5
Neither the AAP nor the American Academy of Family Physicians has a guideline or consensus statement on PSD. Helmets are intended to correct PSD by fitting closely to an infant’s head but allowing room for the skull to grow at the flattened area.1 A 2011 clinical report by Laughlin et al6 recommended against use of helmets for infants with mild to moderate deformities but stated that there was little evidence of harm. Earlier studies have suggested that physical therapy might be effective for plagiocephaly identified early (ie, when the child is 7 to 8 weeks of age).7,8 Biggs9 suggested considering helmet therapy for infants whose cranial sutures remain open and who do not respond to four to eight weeks of physical therapy for PSD. van Wijk et al1 conducted an RCT to explore the risks and benefits of helmet therapy for children with PSD.
Continue for study summary >>
STUDY SUMMARY
Helmets: No help, some harm
In this single-blind RCT of 84 infants (ages 5 or 6 months) with moderate or severe PSD, helmet therapy (n = 42) was compared to no intervention (allowing natural growth; n = 42). Infants were excluded if they had very severe PSD or skull deformity from another cause, such as torticollis or craniosynostosis.
Infants in the helmet therapy group received a custom-made helmet that they wore 23 hours a day until age 1 year, with regular evaluation by an orthotist and modification of the helmet as necessary to allow skull growth. The control group received usual care and no helmet.
The primary outcome was improvement in skull shape at age 24 months, as measured by the oblique diameter difference index (ODDI; a unitless measurement of plagiocephaly calculated as the ratio of measures of two dimensions of cranial diameter) and the cranioproportional index (CPI; a similar measurement of brachycephaly). Infants were considered fully recovered if they achieved an ODDI score < 104% and a CPI score < 90%. These scores indicate a normal head shape; higher scores indicate worse PSD.
At study’s end, the reduction in ODDI and CPI scores was almost the same in both groups. Ten children in the helmet group (26%) and nine in the control group (23%) experienced complete resolution of their PSD.
Both groups were similar in secondary outcomes of infant motor development, infant quality of life, and parental satisfaction. Parental anxiety was assessed using the Spielberger State-Trait Anxiety Inventory (scores range from 20 to 80; a higher score indicates greater anxiety). There was less parental anxiety in the helmet therapy group (–3.9).
All parents of infants in the helmet therapy group reported at least one adverse effect from the intervention. These effects included skin irritation (96%), bad helmet odor (76%), pain associated with the helmet (33%), and feeling hindered from cuddling their child (77%).
Continue for new evidence on helmets for PSD >>
WHAT’S NEW
Stronger evidence that helmets are not effective
Previously, the evidence on helmets for PSD had been obtained mainly from observational or poorly designed studies with significant flaws.6 This study by van Wijk et al1 included objective measurement of skull deformity, along with clinically meaningful outcomes of parental satisfaction, motor development, and parental anxiety.
It also found that helmet therapy was significantly more expensive than care that focused on waiting for PSD to resolve on its own ($1,935 vs $196, respectively).1
CAVEATS
Results may not apply to all infants with skull deformity
These findings do not apply to infants with very severe PSD or those with skull deformity due to secondary causes.1 In addition, this is the only RCT to date that has assessed helmet use in PSD, so it is possible that future studies will find helmets are effective.
Continue for challenges to implementation >>
CHALLENGES TO IMPLEMENTATION
Parents may find this evidence hard to accept
To appropriately implement this recommendation, a clinician must be comfortable making the assessment of mild, moderate, severe, or very severe PSD. Referral to physical therapy might be appropriate for infants with very severe PSD.
If another clinician or physical therapist recommends helmet therapy—or if a parent requests it—explaining the findings of this study may be challenging. We believe that the reduction in parental anxiety in the helmet group likely occurred because the parents believed that the helmet would accelerate the normal reshaping of the skull shape that occurs spontaneously in almost all infants with PSD. Since this study shows that helmets don’t help correct skull deformities, parents can be assured that a helmet is unnecessary and costly and causes adverse effects.
REFERENCES
1. van Wijk RM, van Vlimmeren LA, Groothuis-Oudshoorn CG, et al. Helmet therapy in infants with positional skull deformation: randomised controlled trial. BMJ. 2014;348:g2741.
2. Mawji A, Vollman AR, Hatfield J, et al. The incidence of positional plagiocephaly: a cohort study. Pediatrics. 2013;132:298-304.
3. Peitsch WK, Keefer CH, LaBrie RA, et al. Incidence of cranial asymmetry in healthy newborns. Pediatrics. 2002;110:e72.
4. Collett BR, Gray KE, Starr JR, et al. Development at age 36 months in children with deformational plagiocephaly. Pediatrics. 2013;131: e109-e115.
5. Roby BB, Finkelstein M, Tibesar RJ, et al. Prevalence of positional plagiocephaly in teens born after the “Back to Sleep” campaign. Otolaryngol Head Neck Surg. 2012;146:823-828.
6. Laughlin J, Luerssen TG, Dias MS; Committee on Practice and Ambulatory Medicine, Section on Neurological Surgery. Prevention and management of positional skull deformities in infants. Pediatrics. 2011;128:1236-1241.
7. van Vlimmeren LA, van der Graaf Y, Boere-Boonekamp MM, et al. Effect of pediatric physical therapy on deformational plagiocephaly in children with positional preference: a randomized controlled trial. Arch Pediatr Adolesc Med. 2008;162:712-718.
8. Vargish, L, Mendoza MD, Ewigman, B. Use physical therapy to head off this deformity in infants. Consider early PT to prevent severe deformational plagiocephaly. J Fam Pract. 2009;58:E1-E3.
9. Biggs WS. Diagnosis and management of positional head deformity. Am Fam Physician. 2003;67:1953-1956.
ACKNOWLEDGEMENT
The PURLs Surveillance System was supported in part by Grant Number UL1RR024999 from the National Center For Research Resources, a Clinical Translational Science Award to the University of Chicago. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Center For Research Resources or the National Institutes of Health.
Copyright © 2015. The Family Physicians Inquiries Network. All rights reserved.
Reprinted with permission from the Family Physicians Inquiries Network and The Journal of Family Practice. 2015;64(1):44-46.
In Appendicitis Case, Patient Sues Clinic, Clinic Sues NP
A 17-year-old girl with diminished appetite, abdominal pain, and vomiting presented to a pediatrics clinic in New York, where she was examined by an NP. She was found to have hematuria as well, and the NP diagnosed viral gastroenteritis.
Eight days later, the patient returned to the clinic with worsening pain. The pediatrician who examined her had her transported to a hospital, where a ruptured appendix was diagnosed. The patient underwent immediate surgery, which included resection of portions of her colon and intestines.
Despite a good recovery, the patient claimed that she suffers residual gastrointestinal dysfunction. She further claimed that the NP should have diagnosed appendicitis during her initial visit, which would have allowed for less invasive treatment.
Initially, the plaintiff brought suit against the clinic and several employees, but not the NP. She later moved to add the NP, but that motion was denied due to the statute of limitations. The clinic then impleaded the NP, arguing that it was her negligence in failing to diagnose the appendicitis.
The matter proceeded to trial against the NP and the clinic. The defendants claimed that the plaintiff’s symptoms did not suggest appendicitis at the time of the NP’s examination.
OUTCOME
A defense verdict was returned.
Continue for David M. Lang's comments >>
COMMENT
I used to tell students, “There are only two things in medicine that you need to know well: the common and the dangerous. For everything else, there is time.” I realize now that I sound like that guy from the Dos Equis commercial.
Consider this, however: If we don’t remember the difference between polymyositis and polymyalgia rheumatica, who cares? In such cases, we have time for review—and the patient will be better served by a clinician who has the intellectual curiosity to review conditions that he or she hasn’t seen in a while.
But the diseases that are both common and dangerous require our full proficiency. Basic competence requires us to be well versed in common diseases. And dangerous conditions, even if relatively rare, must be recognized and managed immediately. Entities that are common and dangerous—such as appendicitis—should enter our thoughts often.
In this case, we have a 17-year-old girl presenting to an outpatient clinic setting with abdominal pain, vomiting, and anorexia. Unfortunately, we are not given some important historical information, including duration and location of the pain and the presence or absence of pain migration. Physical exam findings are not described.
The trouble with appendicitis is that there is no single sign or symptom that can effectively diagnose it or exclude it from the differential. When evaluating a patient in a setting in which real-time laboratory testing is not generally ordered, clinicians must distinguish between self-limiting and dangerous abdominal pain. Where does that leave us in this case? Abdominal pain and vomiting are common, and ill patients frequently report anorexia.
Other clinical features associated with appendicitis may be more helpful. For example, pain migration has been described as “the most discriminating feature of the patient’s history,”1 with a sensitivity and specificity of approximately 80%.2 When present, psoas sign is fairly specific (0.95) but not sensitive (0.16).3
When evaluating patients in an outpatient setting, we have a snapshot of a disease process—a still frame of a movie. We are told what happened up to that point (with varying degrees of accuracy). But like the patient, we don’t know what will happen after he or she leaves the office: The still frame is gone, but the movie continues.
It can be helpful to inform patients of the concerning diagnoses in your differential and alert them to patterns of clinical progression that warrant return or immediate emergency department evaluation. Calling the patient to see how he or she is doing can be very useful for clinicians and generally highly valued and appreciated by patients. Here, if gastroenteritis were suspected, a phone call after a few hours of antiemetic and rehydration therapy may have been helpful to determine if the patient’s symptoms had improved. This, of course, would not be conclusive—but at least it would give the clinician additional information and the patient additional comfort.
In this case, the jury was persuaded that the NP provided good treatment and acted within the standard of care. Diagnosing appendicitis can be tricky, even under the best circumstances. The NP’s defense was probably aided by good documentation showing that appendicitis seemed less likely at the time of her evaluation. Ultimately, she performed well enough that her care withstood scrutiny from the plaintiff, the plaintiff’s expert witness, and eventually, her own practice.
This case was interesting from a legal perspective in that the plaintiff originally failed to file suit against the NP—probably resorting to liability under the theory of respondeat superior (generally, employer liability for employee actions). While the plaintiff was unsuccessful in adding the NP later, due to the statute of limitations, the NP was brought into the case by her own practice, through a procedure known as impleader. An impleader action is brought by a co-defendant. Under typical impleader rules, the defendant becomes a “third-party plaintiff” and brings suit against a “third-party defendant” (in this case, the practice and the NP, respectively).
IN SUM
Always keep important diagnoses in mind, and document well. Anticipate a changing clinical course, and instruct patients on how to respond to potential changes. In certain cases, we are well served to pick up the phone, check on the patient, and make the presentation less of a static picture and more of a dynamic movie. —DML
REFERENCES
1. Craig S, Brenner BE. Appendicitis (updated October 26, 2012). Medscape Reference. http://emedicine.medscape.com/article/773895-overview. Accessed January 16, 2015.
2. Yeh B. Evidence-based emergency medicine/rational clinical examination abstract: does this adult patient have appendicitis? Ann Emerg Med. 2008;52(3):301-303.
3. Wagner J, McKinney WP, Carpenter JL. Does this patient have appendicitis? JAMA. 1996;276(19):1589-1594.
A 17-year-old girl with diminished appetite, abdominal pain, and vomiting presented to a pediatrics clinic in New York, where she was examined by an NP. She was found to have hematuria as well, and the NP diagnosed viral gastroenteritis.
Eight days later, the patient returned to the clinic with worsening pain. The pediatrician who examined her had her transported to a hospital, where a ruptured appendix was diagnosed. The patient underwent immediate surgery, which included resection of portions of her colon and intestines.
Despite a good recovery, the patient claimed that she suffers residual gastrointestinal dysfunction. She further claimed that the NP should have diagnosed appendicitis during her initial visit, which would have allowed for less invasive treatment.
Initially, the plaintiff brought suit against the clinic and several employees, but not the NP. She later moved to add the NP, but that motion was denied due to the statute of limitations. The clinic then impleaded the NP, arguing that it was her negligence in failing to diagnose the appendicitis.
The matter proceeded to trial against the NP and the clinic. The defendants claimed that the plaintiff’s symptoms did not suggest appendicitis at the time of the NP’s examination.
OUTCOME
A defense verdict was returned.
Continue for David M. Lang's comments >>
COMMENT
I used to tell students, “There are only two things in medicine that you need to know well: the common and the dangerous. For everything else, there is time.” I realize now that I sound like that guy from the Dos Equis commercial.
Consider this, however: If we don’t remember the difference between polymyositis and polymyalgia rheumatica, who cares? In such cases, we have time for review—and the patient will be better served by a clinician who has the intellectual curiosity to review conditions that he or she hasn’t seen in a while.
But the diseases that are both common and dangerous require our full proficiency. Basic competence requires us to be well versed in common diseases. And dangerous conditions, even if relatively rare, must be recognized and managed immediately. Entities that are common and dangerous—such as appendicitis—should enter our thoughts often.
In this case, we have a 17-year-old girl presenting to an outpatient clinic setting with abdominal pain, vomiting, and anorexia. Unfortunately, we are not given some important historical information, including duration and location of the pain and the presence or absence of pain migration. Physical exam findings are not described.
The trouble with appendicitis is that there is no single sign or symptom that can effectively diagnose it or exclude it from the differential. When evaluating a patient in a setting in which real-time laboratory testing is not generally ordered, clinicians must distinguish between self-limiting and dangerous abdominal pain. Where does that leave us in this case? Abdominal pain and vomiting are common, and ill patients frequently report anorexia.
Other clinical features associated with appendicitis may be more helpful. For example, pain migration has been described as “the most discriminating feature of the patient’s history,”1 with a sensitivity and specificity of approximately 80%.2 When present, psoas sign is fairly specific (0.95) but not sensitive (0.16).3
When evaluating patients in an outpatient setting, we have a snapshot of a disease process—a still frame of a movie. We are told what happened up to that point (with varying degrees of accuracy). But like the patient, we don’t know what will happen after he or she leaves the office: The still frame is gone, but the movie continues.
It can be helpful to inform patients of the concerning diagnoses in your differential and alert them to patterns of clinical progression that warrant return or immediate emergency department evaluation. Calling the patient to see how he or she is doing can be very useful for clinicians and generally highly valued and appreciated by patients. Here, if gastroenteritis were suspected, a phone call after a few hours of antiemetic and rehydration therapy may have been helpful to determine if the patient’s symptoms had improved. This, of course, would not be conclusive—but at least it would give the clinician additional information and the patient additional comfort.
In this case, the jury was persuaded that the NP provided good treatment and acted within the standard of care. Diagnosing appendicitis can be tricky, even under the best circumstances. The NP’s defense was probably aided by good documentation showing that appendicitis seemed less likely at the time of her evaluation. Ultimately, she performed well enough that her care withstood scrutiny from the plaintiff, the plaintiff’s expert witness, and eventually, her own practice.
This case was interesting from a legal perspective in that the plaintiff originally failed to file suit against the NP—probably resorting to liability under the theory of respondeat superior (generally, employer liability for employee actions). While the plaintiff was unsuccessful in adding the NP later, due to the statute of limitations, the NP was brought into the case by her own practice, through a procedure known as impleader. An impleader action is brought by a co-defendant. Under typical impleader rules, the defendant becomes a “third-party plaintiff” and brings suit against a “third-party defendant” (in this case, the practice and the NP, respectively).
IN SUM
Always keep important diagnoses in mind, and document well. Anticipate a changing clinical course, and instruct patients on how to respond to potential changes. In certain cases, we are well served to pick up the phone, check on the patient, and make the presentation less of a static picture and more of a dynamic movie. —DML
REFERENCES
1. Craig S, Brenner BE. Appendicitis (updated October 26, 2012). Medscape Reference. http://emedicine.medscape.com/article/773895-overview. Accessed January 16, 2015.
2. Yeh B. Evidence-based emergency medicine/rational clinical examination abstract: does this adult patient have appendicitis? Ann Emerg Med. 2008;52(3):301-303.
3. Wagner J, McKinney WP, Carpenter JL. Does this patient have appendicitis? JAMA. 1996;276(19):1589-1594.
A 17-year-old girl with diminished appetite, abdominal pain, and vomiting presented to a pediatrics clinic in New York, where she was examined by an NP. She was found to have hematuria as well, and the NP diagnosed viral gastroenteritis.
Eight days later, the patient returned to the clinic with worsening pain. The pediatrician who examined her had her transported to a hospital, where a ruptured appendix was diagnosed. The patient underwent immediate surgery, which included resection of portions of her colon and intestines.
Despite a good recovery, the patient claimed that she suffers residual gastrointestinal dysfunction. She further claimed that the NP should have diagnosed appendicitis during her initial visit, which would have allowed for less invasive treatment.
Initially, the plaintiff brought suit against the clinic and several employees, but not the NP. She later moved to add the NP, but that motion was denied due to the statute of limitations. The clinic then impleaded the NP, arguing that it was her negligence in failing to diagnose the appendicitis.
The matter proceeded to trial against the NP and the clinic. The defendants claimed that the plaintiff’s symptoms did not suggest appendicitis at the time of the NP’s examination.
OUTCOME
A defense verdict was returned.
Continue for David M. Lang's comments >>
COMMENT
I used to tell students, “There are only two things in medicine that you need to know well: the common and the dangerous. For everything else, there is time.” I realize now that I sound like that guy from the Dos Equis commercial.
Consider this, however: If we don’t remember the difference between polymyositis and polymyalgia rheumatica, who cares? In such cases, we have time for review—and the patient will be better served by a clinician who has the intellectual curiosity to review conditions that he or she hasn’t seen in a while.
But the diseases that are both common and dangerous require our full proficiency. Basic competence requires us to be well versed in common diseases. And dangerous conditions, even if relatively rare, must be recognized and managed immediately. Entities that are common and dangerous—such as appendicitis—should enter our thoughts often.
In this case, we have a 17-year-old girl presenting to an outpatient clinic setting with abdominal pain, vomiting, and anorexia. Unfortunately, we are not given some important historical information, including duration and location of the pain and the presence or absence of pain migration. Physical exam findings are not described.
The trouble with appendicitis is that there is no single sign or symptom that can effectively diagnose it or exclude it from the differential. When evaluating a patient in a setting in which real-time laboratory testing is not generally ordered, clinicians must distinguish between self-limiting and dangerous abdominal pain. Where does that leave us in this case? Abdominal pain and vomiting are common, and ill patients frequently report anorexia.
Other clinical features associated with appendicitis may be more helpful. For example, pain migration has been described as “the most discriminating feature of the patient’s history,”1 with a sensitivity and specificity of approximately 80%.2 When present, psoas sign is fairly specific (0.95) but not sensitive (0.16).3
When evaluating patients in an outpatient setting, we have a snapshot of a disease process—a still frame of a movie. We are told what happened up to that point (with varying degrees of accuracy). But like the patient, we don’t know what will happen after he or she leaves the office: The still frame is gone, but the movie continues.
It can be helpful to inform patients of the concerning diagnoses in your differential and alert them to patterns of clinical progression that warrant return or immediate emergency department evaluation. Calling the patient to see how he or she is doing can be very useful for clinicians and generally highly valued and appreciated by patients. Here, if gastroenteritis were suspected, a phone call after a few hours of antiemetic and rehydration therapy may have been helpful to determine if the patient’s symptoms had improved. This, of course, would not be conclusive—but at least it would give the clinician additional information and the patient additional comfort.
In this case, the jury was persuaded that the NP provided good treatment and acted within the standard of care. Diagnosing appendicitis can be tricky, even under the best circumstances. The NP’s defense was probably aided by good documentation showing that appendicitis seemed less likely at the time of her evaluation. Ultimately, she performed well enough that her care withstood scrutiny from the plaintiff, the plaintiff’s expert witness, and eventually, her own practice.
This case was interesting from a legal perspective in that the plaintiff originally failed to file suit against the NP—probably resorting to liability under the theory of respondeat superior (generally, employer liability for employee actions). While the plaintiff was unsuccessful in adding the NP later, due to the statute of limitations, the NP was brought into the case by her own practice, through a procedure known as impleader. An impleader action is brought by a co-defendant. Under typical impleader rules, the defendant becomes a “third-party plaintiff” and brings suit against a “third-party defendant” (in this case, the practice and the NP, respectively).
IN SUM
Always keep important diagnoses in mind, and document well. Anticipate a changing clinical course, and instruct patients on how to respond to potential changes. In certain cases, we are well served to pick up the phone, check on the patient, and make the presentation less of a static picture and more of a dynamic movie. —DML
REFERENCES
1. Craig S, Brenner BE. Appendicitis (updated October 26, 2012). Medscape Reference. http://emedicine.medscape.com/article/773895-overview. Accessed January 16, 2015.
2. Yeh B. Evidence-based emergency medicine/rational clinical examination abstract: does this adult patient have appendicitis? Ann Emerg Med. 2008;52(3):301-303.
3. Wagner J, McKinney WP, Carpenter JL. Does this patient have appendicitis? JAMA. 1996;276(19):1589-1594.
An oversight
After reading the Child Psychiatry Consult column “Aggression and angry outbursts” by Dr. Robert R. Althoff in the September 2014 issue of Pediatric News, I was disappointed that the differential diagnosis did not include an autism spectrum disorder such as DSM-IV Asperger syndrome.
The complex of symptoms described almost perfectly reflects the history of a child with autism. Typical autism spectrum disorder (ASD) issues of needing to direct the play, playing by their rules, and being adamant that things must be the way they see it are noted in the patient’s history. Aggression and outbursts also are typical of a patient with ASD.
Even though autistic behavior is typically predictable, parents are not always alert to the triggers. Most meltdowns are over transitions and denials. Parents of patients with autism often complain that they “walk on eggshells.”
Edward B. Aull, M.D.
Behavioral Pediatrics
St. Vincent Carmel Hospital
Carmel, Ind.
Dr. Althoff responds: I’d like to thank Dr. Aull for pointing out an oversight in my article. Certainly, children on the autistic spectrum can exhibit aggression, although it is not part of the diagnostic criteria for DSM-5 ASD, which include deficits in social interaction and communication, and restricted, repetitive patterns of behavior, interests, or activities. I was not intending for the case to give the impression that this child had difficulty with social communication and restricted interests, but the diagnosis of ASD should be considered on the differential. Similar to the situation in obsessive compulsive disorder or other anxiety disorders, when either the need for social communication becomes exceptionally high or the restricted behavior or interests are challenged, these children can become aggressive, although most do not. Interestingly enough, children with DSM-IV Asperger syndrome and high-functioning autism have co-occurring disorders up to 74% of the time, with the highest percentages in the disorders on the differential that I listed in the original article: behavior disorders, anxiety disorders, and mood disorders (J. Autism Dev. Disord. 2010;40:1080-93). Given these findings, one might consider that, while the diagnosis of an ASD should be considered in the differential, the aggressive behavior may not be associated with the autism symptoms, per se, but rather may be co-occurring symptoms.
After reading the Child Psychiatry Consult column “Aggression and angry outbursts” by Dr. Robert R. Althoff in the September 2014 issue of Pediatric News, I was disappointed that the differential diagnosis did not include an autism spectrum disorder such as DSM-IV Asperger syndrome.
The complex of symptoms described almost perfectly reflects the history of a child with autism. Typical autism spectrum disorder (ASD) issues of needing to direct the play, playing by their rules, and being adamant that things must be the way they see it are noted in the patient’s history. Aggression and outbursts also are typical of a patient with ASD.
Even though autistic behavior is typically predictable, parents are not always alert to the triggers. Most meltdowns are over transitions and denials. Parents of patients with autism often complain that they “walk on eggshells.”
Edward B. Aull, M.D.
Behavioral Pediatrics
St. Vincent Carmel Hospital
Carmel, Ind.
Dr. Althoff responds: I’d like to thank Dr. Aull for pointing out an oversight in my article. Certainly, children on the autistic spectrum can exhibit aggression, although it is not part of the diagnostic criteria for DSM-5 ASD, which include deficits in social interaction and communication, and restricted, repetitive patterns of behavior, interests, or activities. I was not intending for the case to give the impression that this child had difficulty with social communication and restricted interests, but the diagnosis of ASD should be considered on the differential. Similar to the situation in obsessive compulsive disorder or other anxiety disorders, when either the need for social communication becomes exceptionally high or the restricted behavior or interests are challenged, these children can become aggressive, although most do not. Interestingly enough, children with DSM-IV Asperger syndrome and high-functioning autism have co-occurring disorders up to 74% of the time, with the highest percentages in the disorders on the differential that I listed in the original article: behavior disorders, anxiety disorders, and mood disorders (J. Autism Dev. Disord. 2010;40:1080-93). Given these findings, one might consider that, while the diagnosis of an ASD should be considered in the differential, the aggressive behavior may not be associated with the autism symptoms, per se, but rather may be co-occurring symptoms.
After reading the Child Psychiatry Consult column “Aggression and angry outbursts” by Dr. Robert R. Althoff in the September 2014 issue of Pediatric News, I was disappointed that the differential diagnosis did not include an autism spectrum disorder such as DSM-IV Asperger syndrome.
The complex of symptoms described almost perfectly reflects the history of a child with autism. Typical autism spectrum disorder (ASD) issues of needing to direct the play, playing by their rules, and being adamant that things must be the way they see it are noted in the patient’s history. Aggression and outbursts also are typical of a patient with ASD.
Even though autistic behavior is typically predictable, parents are not always alert to the triggers. Most meltdowns are over transitions and denials. Parents of patients with autism often complain that they “walk on eggshells.”
Edward B. Aull, M.D.
Behavioral Pediatrics
St. Vincent Carmel Hospital
Carmel, Ind.
Dr. Althoff responds: I’d like to thank Dr. Aull for pointing out an oversight in my article. Certainly, children on the autistic spectrum can exhibit aggression, although it is not part of the diagnostic criteria for DSM-5 ASD, which include deficits in social interaction and communication, and restricted, repetitive patterns of behavior, interests, or activities. I was not intending for the case to give the impression that this child had difficulty with social communication and restricted interests, but the diagnosis of ASD should be considered on the differential. Similar to the situation in obsessive compulsive disorder or other anxiety disorders, when either the need for social communication becomes exceptionally high or the restricted behavior or interests are challenged, these children can become aggressive, although most do not. Interestingly enough, children with DSM-IV Asperger syndrome and high-functioning autism have co-occurring disorders up to 74% of the time, with the highest percentages in the disorders on the differential that I listed in the original article: behavior disorders, anxiety disorders, and mood disorders (J. Autism Dev. Disord. 2010;40:1080-93). Given these findings, one might consider that, while the diagnosis of an ASD should be considered in the differential, the aggressive behavior may not be associated with the autism symptoms, per se, but rather may be co-occurring symptoms.
Pertussis persists
The Centers for Disease Control and Prevention suggests that recurring pertussis outbreaks may be the “new normal.” Such outbreaks show that some of what we “know” about pertussis is still correct, but some things are evolving. So in this new year, what do we need to know about patient vulnerability post vaccine as well as the clinical course, diagnosis, and treatment of this stubborn persisting disease?
Vulnerability after acellular pertussis vaccine
The recent large 2014 California outbreak surpassed the record numbers for the previously highest incidence year, 2010 (MMWR 2014;63:1129-32). This is scary because more cases had been reported in California in 2010 than in any prior year since the 1940s. The overall 2014 California pertussis rate (26/100,000 population) was approximately 10 times the national average for the first 9 years of this century, Are there clues as to who is most vulnerable and why?
No age group was spared, but certain age groups did appear more vulnerable. Infants had a startling 174.6/100,000 incidence (six times the rate for the overall population). It is not surprising to any clinician that infants less than 1 year of age were hardest hit. Infants have the most evident symptoms with pertussis. Infants also have 5-7 months of their first year in which they are incompletely immunized. Therefore, many are not expected to be protected until about 7-9 months of age. This vulnerability could be partly remedied if all pregnant women got Tdap boosters as recommended during pregnancy.
Of note, 15-year-olds had an incidence similar to that of infants (137.8/100,000). Ethnically, non-Hispanic whites had the highest incidence among adolescents (166.2/100,000), compared with Hispanics (64.2/100,000), Asian/Pacific Islanders (43.9/100,000), and non-Hispanic blacks (23.7/100,000). Disturbingly, 87% of cases among 15-year-olds had received a prior Tdap booster dose (median time since booster Tdap = 3 years, range = 0-7 years). Previous data from the 2010 outbreak suggested that immunity to pertussis wanes 3-4 years after receipt of the last acellular pertussis (aP)–containing vaccine. This is likely part of the explanation in 2014 as well. However, waning immunity after the booster does not explain why non-Hispanic whites had two to six times the incidence of other ethnicities. Non-Hispanic whites are thought to be the demographic with the most vaccine refusal and vaccine delay in California, so this may partially explain excess cases. Racial differences in access to care or genetic differences in disease susceptibility also may play a role.
Why is this biphasic increase in incidence in California a microcosm of the new epidemiology of pertussis in the United States? A kinder, gentler DTaP vaccine replaced the whole-cell DTP in the late 1990s. This occurred in response to the public’s concern about potential central nervous system adverse effects associated with the whole-cell DTP vaccine. Immunogenicity studies seemed to show equivalent immune responses in infants and toddlers receiving DTaP, compared with those who received DTP. It has only been in the last 5 years that we now know that the new DTaP and Tdap are not working as well as we had hoped.
The two aspects to the lesser protection provided by aP vaccines are pertactin-deficient pertussis strains and quicker waning of aP vaccine–induced immunity. Antibody to pertactin appears to be important in protection against clinical pertussis. New circulating clinical strains of pertussis may not have pertactin (N. Engl. J. Med. 2013;368:583-4). The strains used in our current DTaP and Tdap were designed to protect against pertactin-containing strains and were tested for this. This means that a proportion of the antibodies induced by vaccine strains are not useful against pertactin-deficient strains. The aP vaccine still induces antibody to the pertussis toxin and other pertussis components in the vaccines, so they will likely still reduce the severity of disease. But the vaccines are not likely to prevent infections from pertactin-deficient strains. This is similar to the partial vaccine mismatch that we are seeing with the current seasonal H3N2 influenza vaccine strain.
The second aspect is that protection appears to wane approximately 3-5 years after the last dose of aP-containing vaccine. This contrasts sharply with expectations in the past of 7-10 years of protection from whole cell pertussis–containing vaccines. The less reactive aP vaccine produces fewer adverse effects by not producing as much inflammation as DPT. The problem is that part of the reason the DPT has such good protective responses is the amount of inflammation it produces. So with less aP vaccine–induced inflammation comes less robust antibody and T-cell responses.
Nevertheless, the current acellular pertussis vaccines remain the most effective available tools to reduce pertussis disease (Cochrane Database Syst. Rev. 2014;9:CD001478]). But until we have new versions of pertussis vaccines that address these two issues, we clinicians need to remain vigilant for signs and symptoms of pertussis.
Clinical course
Remember that a whoop is rarely seen in young children and often also not seen when older patients present. The many outbreaks over the last 10 years have confirmed that paroxysmal cough with/without apnea in an infant/toddler should raise our index of suspicion. Likewise, older children, adolescents, and adults with persistent cough beyond 2 weeks are potential pertussis cases. Once the diagnosis is made, treatment is not expected to have a major impact on the clinical course, in part because the diagnosis is usually delayed (more than 10 days into symptoms). This delay allows more injury to the respiratory mucosa and cilia so that healing can require 6-12 weeks after bacterial replication ceases. This prolonged healing process is what is mostly responsible for the syndrome known as the “100-day cough.” So the clinical course of pertussis has not changed in the last 10 years. However, there have been changes in the commonly used diagnostic approach.
Pertussis diagnosis and contagion
During the last 5 years, polymerase chain reaction (PCR) testing has become the preferred technology to detect pertussis. This is due to the sensitivity and quick turnaround time of the assay. The gold standard for pertussis diagnosis remains culture, but it is expensive, cumbersome, and slow (up to a week to provide results). An ongoing debate arose about how long PCR testing remains positive after the onset of symptoms or treatment. This was not the problem when culture was the diagnostic tool of choice. Data from the 1970s and 1980s indicated that cultures were rarely positive after the third week of symptoms even without treatment. Furthermore, macrolides eliminated both contagion and positive culture results of infected patients after 5 days of treatment.
So now that we use PCR most often for diagnosis, what is the outer limit of positivity? A recent prospective cohort study from Salt Lake City suggests that PCR may detect pertussis DNA way beyond 3 weeks after symptom onset (J. Ped. Infect. Dis. 2014;3:347-9). Among patients hospitalized with laboratory-confirmed Bordetella pertussis infection, half had persistently positive pertussis PCR testing more than 50 days after symptom onset, despite antibiotic treatment and clinical improvement. The median (range) for the last day for a positive test after symptom onset was 58 days (4-172 days).
This raises the question as to whether there are viable pertussis organisms in the respiratory tract beyond the traditional 3 weeks defined by culture data. It is likely that DNA persists in the thick mucus of the respiratory tract way beyond viability of the last pertussis organisms. Put another way, PCR likely detects bacterial corpses or components way beyond the time that the patient is contagious. Unfortunately, current PCR data do not tell us how long patients remain contagious with the current strains of pertussis as infecting agents. Some institutions appear to be extending the isolation time for patients treated for pertussis beyond the traditional 5 days post initiation of effective treatment. Until more data are available, we are somewhat in the dark. But I would take comfort in the fact that it is unlikely the “new” data will be much different from those derived from the traditional studies that use culture to define infectivity. The American Academy of Pediatrics Committee on Infectious Diseases Red Book appears to agree.
For hospitalized pertussis patients, the AAP Committee on Infectious Diseases Red Book recommends standard and droplet precautions for 5 days after starting effective therapy, or 3 weeks after cough onset if appropriate antimicrobial therapy has not been given.
In addition, the CDC states: “PCR has optimal sensitivity during the first 3 weeks of cough when bacterial DNA is still present in the nasopharynx. After the fourth week of cough, the amount of bacterial DNA rapidly diminishes, which increases the risk of obtaining falsely negative results.” Later in the same document, the CDC says: “PCR testing following antibiotic therapy also can result in falsely negative findings. The exact duration of positivity following antibiotic use is not well understood, but PCR testing after 5 days of antibiotic use is unlikely to be of benefit and is generally not recommended.”
So what do we know? Not all PCR assays use the same primers, so some variance from the usual experience of up to 4 weeks of positive PCR results may be due to differences in the assays. But this raises concern that the PCR that you order may be positive at times when the patient is no longer contagious.
Pertussis treatment
If strains of pertussis have changed their pertactin antigen, are they changing their antibiotic susceptibility patterns? While there have been reports of macrolide resistance in a few pertussis strains, these still remain rare. The most recent comprehensive review of treatment efficacy was a Cochrane review performed in 2005 and published in 2007 (Cochrane Database Syst. Rev. 2007;3:CD004404). They evaluated 10 trials from 1969 to 2004 in which microbiologic eradication was defined by negative results from repeat pertussis culture. While meta-analysis of microbiologic eradication was not possible because of differences in antibiotic use, the investigators did conclude that antibiotic treatment “is effective in eliminating B. pertussis from patients with the disease to render them noninfectious, but does not alter the subsequent clinical course of the illness.”
Further, they state that “the best regimens for microbiologic clearance, with fewer side effects,” are 3 days of azithromycin (a single 10-mg/kg dose on 3 consecutive days) or 7 days of clarithromycin (7.5-mg/kg dose twice daily).
Another effective regimen is 14 days of erythromycin ethylsuccinate (60 mg/kg per day in 3 divided doses) .
CDC treatment recommendations include azithromycin or erythromycin, with trimethoprim-sulfamethoxazole as a possibility for macrolide-intolerant patients, although there are less data and success rates may not be as high.
Conclusion
So what do we know now about pertussis?
• Outbreaks are ongoing and likely will continue until newer more effective vaccines are produced, including those that circumvent the problem of pertactin-deficient strains.
• Pertussis is likely contagious up to 5 days on effective therapy, and for as long as 3 weeks if effective therapy has not been administered.
• PCR is a sensitive test that may remain positive for many weeks beyond contagion.
• Treatment with macrolides appears to be the most effective way to eradicate replicating pertussis pathogens.
• Treatment is not likely to have a major impact on the clinical course of disease because most of the damage to the respiratory tract is done prior to diagnosis and treatment. Treatment does reduce infectivity and subsequent cases.
• Current aP vaccines currently are our best preventative tools – including use in pregnant women to protect young infants.
As clinicians, our best course is to continue to immunize with the current vaccines, and remain vigilant for symptoms and signs of pertussis infection of patients so that early diagnosis and treatment can prevent further spread.
Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospitals and Clinics, Kansas City, Mo. Children’s Mercy Hospitals receives funds from GlaxoSmithKline for Dr. Harrison being principal investigator on a multicenter research study of a hexavalent pertussis-containing infant vaccine. E-mail Dr. Harrison at [email protected].
The Centers for Disease Control and Prevention suggests that recurring pertussis outbreaks may be the “new normal.” Such outbreaks show that some of what we “know” about pertussis is still correct, but some things are evolving. So in this new year, what do we need to know about patient vulnerability post vaccine as well as the clinical course, diagnosis, and treatment of this stubborn persisting disease?
Vulnerability after acellular pertussis vaccine
The recent large 2014 California outbreak surpassed the record numbers for the previously highest incidence year, 2010 (MMWR 2014;63:1129-32). This is scary because more cases had been reported in California in 2010 than in any prior year since the 1940s. The overall 2014 California pertussis rate (26/100,000 population) was approximately 10 times the national average for the first 9 years of this century, Are there clues as to who is most vulnerable and why?
No age group was spared, but certain age groups did appear more vulnerable. Infants had a startling 174.6/100,000 incidence (six times the rate for the overall population). It is not surprising to any clinician that infants less than 1 year of age were hardest hit. Infants have the most evident symptoms with pertussis. Infants also have 5-7 months of their first year in which they are incompletely immunized. Therefore, many are not expected to be protected until about 7-9 months of age. This vulnerability could be partly remedied if all pregnant women got Tdap boosters as recommended during pregnancy.
Of note, 15-year-olds had an incidence similar to that of infants (137.8/100,000). Ethnically, non-Hispanic whites had the highest incidence among adolescents (166.2/100,000), compared with Hispanics (64.2/100,000), Asian/Pacific Islanders (43.9/100,000), and non-Hispanic blacks (23.7/100,000). Disturbingly, 87% of cases among 15-year-olds had received a prior Tdap booster dose (median time since booster Tdap = 3 years, range = 0-7 years). Previous data from the 2010 outbreak suggested that immunity to pertussis wanes 3-4 years after receipt of the last acellular pertussis (aP)–containing vaccine. This is likely part of the explanation in 2014 as well. However, waning immunity after the booster does not explain why non-Hispanic whites had two to six times the incidence of other ethnicities. Non-Hispanic whites are thought to be the demographic with the most vaccine refusal and vaccine delay in California, so this may partially explain excess cases. Racial differences in access to care or genetic differences in disease susceptibility also may play a role.
Why is this biphasic increase in incidence in California a microcosm of the new epidemiology of pertussis in the United States? A kinder, gentler DTaP vaccine replaced the whole-cell DTP in the late 1990s. This occurred in response to the public’s concern about potential central nervous system adverse effects associated with the whole-cell DTP vaccine. Immunogenicity studies seemed to show equivalent immune responses in infants and toddlers receiving DTaP, compared with those who received DTP. It has only been in the last 5 years that we now know that the new DTaP and Tdap are not working as well as we had hoped.
The two aspects to the lesser protection provided by aP vaccines are pertactin-deficient pertussis strains and quicker waning of aP vaccine–induced immunity. Antibody to pertactin appears to be important in protection against clinical pertussis. New circulating clinical strains of pertussis may not have pertactin (N. Engl. J. Med. 2013;368:583-4). The strains used in our current DTaP and Tdap were designed to protect against pertactin-containing strains and were tested for this. This means that a proportion of the antibodies induced by vaccine strains are not useful against pertactin-deficient strains. The aP vaccine still induces antibody to the pertussis toxin and other pertussis components in the vaccines, so they will likely still reduce the severity of disease. But the vaccines are not likely to prevent infections from pertactin-deficient strains. This is similar to the partial vaccine mismatch that we are seeing with the current seasonal H3N2 influenza vaccine strain.
The second aspect is that protection appears to wane approximately 3-5 years after the last dose of aP-containing vaccine. This contrasts sharply with expectations in the past of 7-10 years of protection from whole cell pertussis–containing vaccines. The less reactive aP vaccine produces fewer adverse effects by not producing as much inflammation as DPT. The problem is that part of the reason the DPT has such good protective responses is the amount of inflammation it produces. So with less aP vaccine–induced inflammation comes less robust antibody and T-cell responses.
Nevertheless, the current acellular pertussis vaccines remain the most effective available tools to reduce pertussis disease (Cochrane Database Syst. Rev. 2014;9:CD001478]). But until we have new versions of pertussis vaccines that address these two issues, we clinicians need to remain vigilant for signs and symptoms of pertussis.
Clinical course
Remember that a whoop is rarely seen in young children and often also not seen when older patients present. The many outbreaks over the last 10 years have confirmed that paroxysmal cough with/without apnea in an infant/toddler should raise our index of suspicion. Likewise, older children, adolescents, and adults with persistent cough beyond 2 weeks are potential pertussis cases. Once the diagnosis is made, treatment is not expected to have a major impact on the clinical course, in part because the diagnosis is usually delayed (more than 10 days into symptoms). This delay allows more injury to the respiratory mucosa and cilia so that healing can require 6-12 weeks after bacterial replication ceases. This prolonged healing process is what is mostly responsible for the syndrome known as the “100-day cough.” So the clinical course of pertussis has not changed in the last 10 years. However, there have been changes in the commonly used diagnostic approach.
Pertussis diagnosis and contagion
During the last 5 years, polymerase chain reaction (PCR) testing has become the preferred technology to detect pertussis. This is due to the sensitivity and quick turnaround time of the assay. The gold standard for pertussis diagnosis remains culture, but it is expensive, cumbersome, and slow (up to a week to provide results). An ongoing debate arose about how long PCR testing remains positive after the onset of symptoms or treatment. This was not the problem when culture was the diagnostic tool of choice. Data from the 1970s and 1980s indicated that cultures were rarely positive after the third week of symptoms even without treatment. Furthermore, macrolides eliminated both contagion and positive culture results of infected patients after 5 days of treatment.
So now that we use PCR most often for diagnosis, what is the outer limit of positivity? A recent prospective cohort study from Salt Lake City suggests that PCR may detect pertussis DNA way beyond 3 weeks after symptom onset (J. Ped. Infect. Dis. 2014;3:347-9). Among patients hospitalized with laboratory-confirmed Bordetella pertussis infection, half had persistently positive pertussis PCR testing more than 50 days after symptom onset, despite antibiotic treatment and clinical improvement. The median (range) for the last day for a positive test after symptom onset was 58 days (4-172 days).
This raises the question as to whether there are viable pertussis organisms in the respiratory tract beyond the traditional 3 weeks defined by culture data. It is likely that DNA persists in the thick mucus of the respiratory tract way beyond viability of the last pertussis organisms. Put another way, PCR likely detects bacterial corpses or components way beyond the time that the patient is contagious. Unfortunately, current PCR data do not tell us how long patients remain contagious with the current strains of pertussis as infecting agents. Some institutions appear to be extending the isolation time for patients treated for pertussis beyond the traditional 5 days post initiation of effective treatment. Until more data are available, we are somewhat in the dark. But I would take comfort in the fact that it is unlikely the “new” data will be much different from those derived from the traditional studies that use culture to define infectivity. The American Academy of Pediatrics Committee on Infectious Diseases Red Book appears to agree.
For hospitalized pertussis patients, the AAP Committee on Infectious Diseases Red Book recommends standard and droplet precautions for 5 days after starting effective therapy, or 3 weeks after cough onset if appropriate antimicrobial therapy has not been given.
In addition, the CDC states: “PCR has optimal sensitivity during the first 3 weeks of cough when bacterial DNA is still present in the nasopharynx. After the fourth week of cough, the amount of bacterial DNA rapidly diminishes, which increases the risk of obtaining falsely negative results.” Later in the same document, the CDC says: “PCR testing following antibiotic therapy also can result in falsely negative findings. The exact duration of positivity following antibiotic use is not well understood, but PCR testing after 5 days of antibiotic use is unlikely to be of benefit and is generally not recommended.”
So what do we know? Not all PCR assays use the same primers, so some variance from the usual experience of up to 4 weeks of positive PCR results may be due to differences in the assays. But this raises concern that the PCR that you order may be positive at times when the patient is no longer contagious.
Pertussis treatment
If strains of pertussis have changed their pertactin antigen, are they changing their antibiotic susceptibility patterns? While there have been reports of macrolide resistance in a few pertussis strains, these still remain rare. The most recent comprehensive review of treatment efficacy was a Cochrane review performed in 2005 and published in 2007 (Cochrane Database Syst. Rev. 2007;3:CD004404). They evaluated 10 trials from 1969 to 2004 in which microbiologic eradication was defined by negative results from repeat pertussis culture. While meta-analysis of microbiologic eradication was not possible because of differences in antibiotic use, the investigators did conclude that antibiotic treatment “is effective in eliminating B. pertussis from patients with the disease to render them noninfectious, but does not alter the subsequent clinical course of the illness.”
Further, they state that “the best regimens for microbiologic clearance, with fewer side effects,” are 3 days of azithromycin (a single 10-mg/kg dose on 3 consecutive days) or 7 days of clarithromycin (7.5-mg/kg dose twice daily).
Another effective regimen is 14 days of erythromycin ethylsuccinate (60 mg/kg per day in 3 divided doses) .
CDC treatment recommendations include azithromycin or erythromycin, with trimethoprim-sulfamethoxazole as a possibility for macrolide-intolerant patients, although there are less data and success rates may not be as high.
Conclusion
So what do we know now about pertussis?
• Outbreaks are ongoing and likely will continue until newer more effective vaccines are produced, including those that circumvent the problem of pertactin-deficient strains.
• Pertussis is likely contagious up to 5 days on effective therapy, and for as long as 3 weeks if effective therapy has not been administered.
• PCR is a sensitive test that may remain positive for many weeks beyond contagion.
• Treatment with macrolides appears to be the most effective way to eradicate replicating pertussis pathogens.
• Treatment is not likely to have a major impact on the clinical course of disease because most of the damage to the respiratory tract is done prior to diagnosis and treatment. Treatment does reduce infectivity and subsequent cases.
• Current aP vaccines currently are our best preventative tools – including use in pregnant women to protect young infants.
As clinicians, our best course is to continue to immunize with the current vaccines, and remain vigilant for symptoms and signs of pertussis infection of patients so that early diagnosis and treatment can prevent further spread.
Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospitals and Clinics, Kansas City, Mo. Children’s Mercy Hospitals receives funds from GlaxoSmithKline for Dr. Harrison being principal investigator on a multicenter research study of a hexavalent pertussis-containing infant vaccine. E-mail Dr. Harrison at [email protected].
The Centers for Disease Control and Prevention suggests that recurring pertussis outbreaks may be the “new normal.” Such outbreaks show that some of what we “know” about pertussis is still correct, but some things are evolving. So in this new year, what do we need to know about patient vulnerability post vaccine as well as the clinical course, diagnosis, and treatment of this stubborn persisting disease?
Vulnerability after acellular pertussis vaccine
The recent large 2014 California outbreak surpassed the record numbers for the previously highest incidence year, 2010 (MMWR 2014;63:1129-32). This is scary because more cases had been reported in California in 2010 than in any prior year since the 1940s. The overall 2014 California pertussis rate (26/100,000 population) was approximately 10 times the national average for the first 9 years of this century, Are there clues as to who is most vulnerable and why?
No age group was spared, but certain age groups did appear more vulnerable. Infants had a startling 174.6/100,000 incidence (six times the rate for the overall population). It is not surprising to any clinician that infants less than 1 year of age were hardest hit. Infants have the most evident symptoms with pertussis. Infants also have 5-7 months of their first year in which they are incompletely immunized. Therefore, many are not expected to be protected until about 7-9 months of age. This vulnerability could be partly remedied if all pregnant women got Tdap boosters as recommended during pregnancy.
Of note, 15-year-olds had an incidence similar to that of infants (137.8/100,000). Ethnically, non-Hispanic whites had the highest incidence among adolescents (166.2/100,000), compared with Hispanics (64.2/100,000), Asian/Pacific Islanders (43.9/100,000), and non-Hispanic blacks (23.7/100,000). Disturbingly, 87% of cases among 15-year-olds had received a prior Tdap booster dose (median time since booster Tdap = 3 years, range = 0-7 years). Previous data from the 2010 outbreak suggested that immunity to pertussis wanes 3-4 years after receipt of the last acellular pertussis (aP)–containing vaccine. This is likely part of the explanation in 2014 as well. However, waning immunity after the booster does not explain why non-Hispanic whites had two to six times the incidence of other ethnicities. Non-Hispanic whites are thought to be the demographic with the most vaccine refusal and vaccine delay in California, so this may partially explain excess cases. Racial differences in access to care or genetic differences in disease susceptibility also may play a role.
Why is this biphasic increase in incidence in California a microcosm of the new epidemiology of pertussis in the United States? A kinder, gentler DTaP vaccine replaced the whole-cell DTP in the late 1990s. This occurred in response to the public’s concern about potential central nervous system adverse effects associated with the whole-cell DTP vaccine. Immunogenicity studies seemed to show equivalent immune responses in infants and toddlers receiving DTaP, compared with those who received DTP. It has only been in the last 5 years that we now know that the new DTaP and Tdap are not working as well as we had hoped.
The two aspects to the lesser protection provided by aP vaccines are pertactin-deficient pertussis strains and quicker waning of aP vaccine–induced immunity. Antibody to pertactin appears to be important in protection against clinical pertussis. New circulating clinical strains of pertussis may not have pertactin (N. Engl. J. Med. 2013;368:583-4). The strains used in our current DTaP and Tdap were designed to protect against pertactin-containing strains and were tested for this. This means that a proportion of the antibodies induced by vaccine strains are not useful against pertactin-deficient strains. The aP vaccine still induces antibody to the pertussis toxin and other pertussis components in the vaccines, so they will likely still reduce the severity of disease. But the vaccines are not likely to prevent infections from pertactin-deficient strains. This is similar to the partial vaccine mismatch that we are seeing with the current seasonal H3N2 influenza vaccine strain.
The second aspect is that protection appears to wane approximately 3-5 years after the last dose of aP-containing vaccine. This contrasts sharply with expectations in the past of 7-10 years of protection from whole cell pertussis–containing vaccines. The less reactive aP vaccine produces fewer adverse effects by not producing as much inflammation as DPT. The problem is that part of the reason the DPT has such good protective responses is the amount of inflammation it produces. So with less aP vaccine–induced inflammation comes less robust antibody and T-cell responses.
Nevertheless, the current acellular pertussis vaccines remain the most effective available tools to reduce pertussis disease (Cochrane Database Syst. Rev. 2014;9:CD001478]). But until we have new versions of pertussis vaccines that address these two issues, we clinicians need to remain vigilant for signs and symptoms of pertussis.
Clinical course
Remember that a whoop is rarely seen in young children and often also not seen when older patients present. The many outbreaks over the last 10 years have confirmed that paroxysmal cough with/without apnea in an infant/toddler should raise our index of suspicion. Likewise, older children, adolescents, and adults with persistent cough beyond 2 weeks are potential pertussis cases. Once the diagnosis is made, treatment is not expected to have a major impact on the clinical course, in part because the diagnosis is usually delayed (more than 10 days into symptoms). This delay allows more injury to the respiratory mucosa and cilia so that healing can require 6-12 weeks after bacterial replication ceases. This prolonged healing process is what is mostly responsible for the syndrome known as the “100-day cough.” So the clinical course of pertussis has not changed in the last 10 years. However, there have been changes in the commonly used diagnostic approach.
Pertussis diagnosis and contagion
During the last 5 years, polymerase chain reaction (PCR) testing has become the preferred technology to detect pertussis. This is due to the sensitivity and quick turnaround time of the assay. The gold standard for pertussis diagnosis remains culture, but it is expensive, cumbersome, and slow (up to a week to provide results). An ongoing debate arose about how long PCR testing remains positive after the onset of symptoms or treatment. This was not the problem when culture was the diagnostic tool of choice. Data from the 1970s and 1980s indicated that cultures were rarely positive after the third week of symptoms even without treatment. Furthermore, macrolides eliminated both contagion and positive culture results of infected patients after 5 days of treatment.
So now that we use PCR most often for diagnosis, what is the outer limit of positivity? A recent prospective cohort study from Salt Lake City suggests that PCR may detect pertussis DNA way beyond 3 weeks after symptom onset (J. Ped. Infect. Dis. 2014;3:347-9). Among patients hospitalized with laboratory-confirmed Bordetella pertussis infection, half had persistently positive pertussis PCR testing more than 50 days after symptom onset, despite antibiotic treatment and clinical improvement. The median (range) for the last day for a positive test after symptom onset was 58 days (4-172 days).
This raises the question as to whether there are viable pertussis organisms in the respiratory tract beyond the traditional 3 weeks defined by culture data. It is likely that DNA persists in the thick mucus of the respiratory tract way beyond viability of the last pertussis organisms. Put another way, PCR likely detects bacterial corpses or components way beyond the time that the patient is contagious. Unfortunately, current PCR data do not tell us how long patients remain contagious with the current strains of pertussis as infecting agents. Some institutions appear to be extending the isolation time for patients treated for pertussis beyond the traditional 5 days post initiation of effective treatment. Until more data are available, we are somewhat in the dark. But I would take comfort in the fact that it is unlikely the “new” data will be much different from those derived from the traditional studies that use culture to define infectivity. The American Academy of Pediatrics Committee on Infectious Diseases Red Book appears to agree.
For hospitalized pertussis patients, the AAP Committee on Infectious Diseases Red Book recommends standard and droplet precautions for 5 days after starting effective therapy, or 3 weeks after cough onset if appropriate antimicrobial therapy has not been given.
In addition, the CDC states: “PCR has optimal sensitivity during the first 3 weeks of cough when bacterial DNA is still present in the nasopharynx. After the fourth week of cough, the amount of bacterial DNA rapidly diminishes, which increases the risk of obtaining falsely negative results.” Later in the same document, the CDC says: “PCR testing following antibiotic therapy also can result in falsely negative findings. The exact duration of positivity following antibiotic use is not well understood, but PCR testing after 5 days of antibiotic use is unlikely to be of benefit and is generally not recommended.”
So what do we know? Not all PCR assays use the same primers, so some variance from the usual experience of up to 4 weeks of positive PCR results may be due to differences in the assays. But this raises concern that the PCR that you order may be positive at times when the patient is no longer contagious.
Pertussis treatment
If strains of pertussis have changed their pertactin antigen, are they changing their antibiotic susceptibility patterns? While there have been reports of macrolide resistance in a few pertussis strains, these still remain rare. The most recent comprehensive review of treatment efficacy was a Cochrane review performed in 2005 and published in 2007 (Cochrane Database Syst. Rev. 2007;3:CD004404). They evaluated 10 trials from 1969 to 2004 in which microbiologic eradication was defined by negative results from repeat pertussis culture. While meta-analysis of microbiologic eradication was not possible because of differences in antibiotic use, the investigators did conclude that antibiotic treatment “is effective in eliminating B. pertussis from patients with the disease to render them noninfectious, but does not alter the subsequent clinical course of the illness.”
Further, they state that “the best regimens for microbiologic clearance, with fewer side effects,” are 3 days of azithromycin (a single 10-mg/kg dose on 3 consecutive days) or 7 days of clarithromycin (7.5-mg/kg dose twice daily).
Another effective regimen is 14 days of erythromycin ethylsuccinate (60 mg/kg per day in 3 divided doses) .
CDC treatment recommendations include azithromycin or erythromycin, with trimethoprim-sulfamethoxazole as a possibility for macrolide-intolerant patients, although there are less data and success rates may not be as high.
Conclusion
So what do we know now about pertussis?
• Outbreaks are ongoing and likely will continue until newer more effective vaccines are produced, including those that circumvent the problem of pertactin-deficient strains.
• Pertussis is likely contagious up to 5 days on effective therapy, and for as long as 3 weeks if effective therapy has not been administered.
• PCR is a sensitive test that may remain positive for many weeks beyond contagion.
• Treatment with macrolides appears to be the most effective way to eradicate replicating pertussis pathogens.
• Treatment is not likely to have a major impact on the clinical course of disease because most of the damage to the respiratory tract is done prior to diagnosis and treatment. Treatment does reduce infectivity and subsequent cases.
• Current aP vaccines currently are our best preventative tools – including use in pregnant women to protect young infants.
As clinicians, our best course is to continue to immunize with the current vaccines, and remain vigilant for symptoms and signs of pertussis infection of patients so that early diagnosis and treatment can prevent further spread.
Dr. Harrison is professor of pediatrics and pediatric infectious diseases at Children’s Mercy Hospitals and Clinics, Kansas City, Mo. Children’s Mercy Hospitals receives funds from GlaxoSmithKline for Dr. Harrison being principal investigator on a multicenter research study of a hexavalent pertussis-containing infant vaccine. E-mail Dr. Harrison at [email protected].
