Recently, Federal Practitioner talked with Sanjai Sharma, MD, about how signaling pathways in chronic lymphocytic leukemia (CLL) is critical to the development of therapeutic agents to treat this disease. Ibrutinib and idelalisib are therapeutic agents that block signaling pathways and, therefore, inhibit the growth of CLL cells.

For more information about CLL, read "Signaling Pathways and Novel Inhibitors in Chronic Lymphocytic Leukemia," in our August 2014 issue.

Dr. Sharma is a physician at the West Los Angeles VA Medical Center and associate professor in the Department of Medicine, Hematology/Oncology at UCLA, both in California.

Recently, Federal Practitioner talked with Sanjai Sharma, MD, about how signaling pathways in chronic lymphocytic leukemia (CLL) is critical to the development of therapeutic agents to treat this disease. Ibrutinib and idelalisib are therapeutic agents that block signaling pathways and, therefore, inhibit the growth of CLL cells.

For more information about CLL, read "Signaling Pathways and Novel Inhibitors in Chronic Lymphocytic Leukemia," in our August 2014 issue.

Dr. Sharma is a physician at the West Los Angeles VA Medical Center and associate professor in the Department of Medicine, Hematology/Oncology at UCLA, both in California.

Recently, Federal Practitioner talked with Sanjai Sharma, MD, about how signaling pathways in chronic lymphocytic leukemia (CLL) is critical to the development of therapeutic agents to treat this disease. Ibrutinib and idelalisib are therapeutic agents that block signaling pathways and, therefore, inhibit the growth of CLL cells.

For more information about CLL, read "Signaling Pathways and Novel Inhibitors in Chronic Lymphocytic Leukemia," in our August 2014 issue.

Dr. Sharma is a physician at the West Los Angeles VA Medical Center and associate professor in the Department of Medicine, Hematology/Oncology at UCLA, both in California.

Doctor examines SCD patient

Credit: St Jude Children’s

Research Hospital

Monthly blood transfusions can reduce the risk of silent or overt stroke among children with sickle cell disease (SCD) who previously had a silent stroke, according to a study published in The New England Journal of Medicine.

Children with evidence of silent cerebral infarcts who received monthly blood transfusions for 3 years had a 58% lower risk of suffering repeat silent or overt

strokes than children who did not receive transfusions.

In fact, researchers said the actual benefit of transfusion therapy may be even higher, as 15% of the children who were assigned to receive transfusions either did not receive them or only received them for a brief period.

“The results of our study show that blood transfusions can play a critical role in preventing this insidious and potentially devastating condition,” said study author James F. Casella, MD, of the Johns Hopkins Children’s Center in Baltimore, Maryland.

“They also highlight the importance of intervening early to preclude ongoing or further brain injury among these youngsters. Most importantly, our findings suggest a much-needed treatment option for clinicians and families of children with sickle cell disease who have had silent strokes.”

Previous studies have suggested that blood transfusions may help prevent stroke in patients with SCD by increasing the number of normal red blood cells and decreasing the likelihood of blocked blood vessels.

But Dr Casella and his colleagues wanted to determine if monthly blood transfusions would help prevent stroke in children with SCD who had evidence of a previous silent cerebral infarct, as well as whether the benefits of transfusion outweigh the risks.

The researchers analyzed 196 children, ages 5 to 15 years, who were diagnosed with SCD and had infarct-like lesions on their MRI scans. The children were randomized to an observation arm or to receive blood transfusions every month for 3 years.

Six percent (6/99) of children who received regular transfusions suffered another silent or overt stroke. One of the patients had a stroke, and 5 had new or enlarged silent cerebral infarcts.

In comparison, 14% (14/97) of children in the observation arm experienced a silent or overt stroke. Seven had a stroke, and 7 had new or enlarged silent cerebral infarcts.

So children who did not receive transfusions were more than twice as likely as their peers to have repeat strokes.

Children who did not receive transfusions were also more likely to suffer a range of other SCD-related problems, such as episodes of extreme pain. There were 295 pain episodes among children who did not receive transfusions and 126 episodes among transfused patients.

An unexpected result, according to the researchers, was that intelligence measures were not different between the 2 treatment arms. Previous studies suggested that silent strokes are associated with a 5-point reduction in IQ. The researchers said they plan to explore this finding further.

Nevertheless, this study provides “clear evidence” that transfusions can decrease the progression of silent strokes in children with SCD, said study author Michael R. DeBaun, MD, of  Vanderbilt University in Nashville, Tennessee.

“These results suggest that children who have this disease should be screened early for silent strokes, at least by the time they begin elementary school, to help them manage the disease and to ensure minimal impact on school performance,” he added.

Dr DeBaun and his colleagues said children with SCD should have a surveillance MRI, preferably without sedation, at a young age. Most children with SCD who are at risk for a silent stroke will have one by age 6 years.

The researchers also noted that healthcare providers should discuss treatment options with families to determine if transfusion therapy is appropriate, as there is a risk of transfusion reactions and iron overload.

The decision to transfuse should be made by factoring in each child’s overall health, medical history, and the ability to take time from school for monthly procedures.

The researchers said further study is needed to identify which children with a history of silent strokes are at greatest risk for recurrence so transfusion therapy can be targeted to them.

An editorial related to this study also calls for additional research to determine if the findings can be translated to clinical practice.

Doctor examines SCD patient

Credit: St Jude Children’s

Research Hospital

Monthly blood transfusions can reduce the risk of silent or overt stroke among children with sickle cell disease (SCD) who previously had a silent stroke, according to a study published in The New England Journal of Medicine.

Children with evidence of silent cerebral infarcts who received monthly blood transfusions for 3 years had a 58% lower risk of suffering repeat silent or overt

strokes than children who did not receive transfusions.

In fact, researchers said the actual benefit of transfusion therapy may be even higher, as 15% of the children who were assigned to receive transfusions either did not receive them or only received them for a brief period.

“The results of our study show that blood transfusions can play a critical role in preventing this insidious and potentially devastating condition,” said study author James F. Casella, MD, of the Johns Hopkins Children’s Center in Baltimore, Maryland.

“They also highlight the importance of intervening early to preclude ongoing or further brain injury among these youngsters. Most importantly, our findings suggest a much-needed treatment option for clinicians and families of children with sickle cell disease who have had silent strokes.”

Previous studies have suggested that blood transfusions may help prevent stroke in patients with SCD by increasing the number of normal red blood cells and decreasing the likelihood of blocked blood vessels.

But Dr Casella and his colleagues wanted to determine if monthly blood transfusions would help prevent stroke in children with SCD who had evidence of a previous silent cerebral infarct, as well as whether the benefits of transfusion outweigh the risks.

The researchers analyzed 196 children, ages 5 to 15 years, who were diagnosed with SCD and had infarct-like lesions on their MRI scans. The children were randomized to an observation arm or to receive blood transfusions every month for 3 years.

Six percent (6/99) of children who received regular transfusions suffered another silent or overt stroke. One of the patients had a stroke, and 5 had new or enlarged silent cerebral infarcts.

In comparison, 14% (14/97) of children in the observation arm experienced a silent or overt stroke. Seven had a stroke, and 7 had new or enlarged silent cerebral infarcts.

So children who did not receive transfusions were more than twice as likely as their peers to have repeat strokes.

Children who did not receive transfusions were also more likely to suffer a range of other SCD-related problems, such as episodes of extreme pain. There were 295 pain episodes among children who did not receive transfusions and 126 episodes among transfused patients.

An unexpected result, according to the researchers, was that intelligence measures were not different between the 2 treatment arms. Previous studies suggested that silent strokes are associated with a 5-point reduction in IQ. The researchers said they plan to explore this finding further.

Nevertheless, this study provides “clear evidence” that transfusions can decrease the progression of silent strokes in children with SCD, said study author Michael R. DeBaun, MD, of  Vanderbilt University in Nashville, Tennessee.

“These results suggest that children who have this disease should be screened early for silent strokes, at least by the time they begin elementary school, to help them manage the disease and to ensure minimal impact on school performance,” he added.

Dr DeBaun and his colleagues said children with SCD should have a surveillance MRI, preferably without sedation, at a young age. Most children with SCD who are at risk for a silent stroke will have one by age 6 years.

The researchers also noted that healthcare providers should discuss treatment options with families to determine if transfusion therapy is appropriate, as there is a risk of transfusion reactions and iron overload.

The decision to transfuse should be made by factoring in each child’s overall health, medical history, and the ability to take time from school for monthly procedures.

The researchers said further study is needed to identify which children with a history of silent strokes are at greatest risk for recurrence so transfusion therapy can be targeted to them.

An editorial related to this study also calls for additional research to determine if the findings can be translated to clinical practice.

Doctor examines SCD patient

Credit: St Jude Children’s

Research Hospital

Monthly blood transfusions can reduce the risk of silent or overt stroke among children with sickle cell disease (SCD) who previously had a silent stroke, according to a study published in The New England Journal of Medicine.

Children with evidence of silent cerebral infarcts who received monthly blood transfusions for 3 years had a 58% lower risk of suffering repeat silent or overt

strokes than children who did not receive transfusions.

In fact, researchers said the actual benefit of transfusion therapy may be even higher, as 15% of the children who were assigned to receive transfusions either did not receive them or only received them for a brief period.

“The results of our study show that blood transfusions can play a critical role in preventing this insidious and potentially devastating condition,” said study author James F. Casella, MD, of the Johns Hopkins Children’s Center in Baltimore, Maryland.

“They also highlight the importance of intervening early to preclude ongoing or further brain injury among these youngsters. Most importantly, our findings suggest a much-needed treatment option for clinicians and families of children with sickle cell disease who have had silent strokes.”

Previous studies have suggested that blood transfusions may help prevent stroke in patients with SCD by increasing the number of normal red blood cells and decreasing the likelihood of blocked blood vessels.

But Dr Casella and his colleagues wanted to determine if monthly blood transfusions would help prevent stroke in children with SCD who had evidence of a previous silent cerebral infarct, as well as whether the benefits of transfusion outweigh the risks.

The researchers analyzed 196 children, ages 5 to 15 years, who were diagnosed with SCD and had infarct-like lesions on their MRI scans. The children were randomized to an observation arm or to receive blood transfusions every month for 3 years.

Six percent (6/99) of children who received regular transfusions suffered another silent or overt stroke. One of the patients had a stroke, and 5 had new or enlarged silent cerebral infarcts.

In comparison, 14% (14/97) of children in the observation arm experienced a silent or overt stroke. Seven had a stroke, and 7 had new or enlarged silent cerebral infarcts.

So children who did not receive transfusions were more than twice as likely as their peers to have repeat strokes.

Children who did not receive transfusions were also more likely to suffer a range of other SCD-related problems, such as episodes of extreme pain. There were 295 pain episodes among children who did not receive transfusions and 126 episodes among transfused patients.

An unexpected result, according to the researchers, was that intelligence measures were not different between the 2 treatment arms. Previous studies suggested that silent strokes are associated with a 5-point reduction in IQ. The researchers said they plan to explore this finding further.

Nevertheless, this study provides “clear evidence” that transfusions can decrease the progression of silent strokes in children with SCD, said study author Michael R. DeBaun, MD, of  Vanderbilt University in Nashville, Tennessee.

“These results suggest that children who have this disease should be screened early for silent strokes, at least by the time they begin elementary school, to help them manage the disease and to ensure minimal impact on school performance,” he added.

Dr DeBaun and his colleagues said children with SCD should have a surveillance MRI, preferably without sedation, at a young age. Most children with SCD who are at risk for a silent stroke will have one by age 6 years.

The researchers also noted that healthcare providers should discuss treatment options with families to determine if transfusion therapy is appropriate, as there is a risk of transfusion reactions and iron overload.

The decision to transfuse should be made by factoring in each child’s overall health, medical history, and the ability to take time from school for monthly procedures.

The researchers said further study is needed to identify which children with a history of silent strokes are at greatest risk for recurrence so transfusion therapy can be targeted to them.

An editorial related to this study also calls for additional research to determine if the findings can be translated to clinical practice.

Blood smear showing MDS

The UK’s National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending lenalidomide (Revlimid) as an option for treating myelodysplastic syndromes (MDS) characterized by 5q deletion.

Lenalidomide is approved in the European Union to treat transfusion-dependent anemia caused by low- or intermediate-1 risk MDS characterized by 5q deletion when other therapeutic options are insufficient or inadequate.

However, the main treatment option for this patient population in the UK is best supportive care, which involves regular red blood cell transfusions.

In earlier draft guidances, NICE did not support lenalidomide use in MDS patients with 5q deletion. Although data suggested the drug is effective for these patients, a NICE advisory committee was not convinced the drug provided a survival benefit.

But now, the committee has concluded that lenalidomide is a clinically effective treatment for these patients because it is associated with a statistically significant improvement in transfusion independence and health-related quality of life compared with placebo.

Furthermore, the committee said it is plausible that lenalidomide can indirectly improve overall survival by improving transfusion independence.

“The committee heard from clinical experts that lenalidomide is an effective therapy,” said Sir Andrew Dillon, NICE chief executive.

“Celgene–who market lenalidomide–worked with us to provide enough evidence to make it possible for us to recommend it for this group of people. Celgene provided a revised analysis and further information on their proposal for a reduction in the cost of the drug to the NHS [National Health Service].”

This patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. And Celgene will provide the drug free of charge for those people who receive more than 26 monthly cycles.

Lenalidomide is available in 21-day packs of 10 mg and 5 mg capsules at net prices of £3780 and £3570, respectively. The cost of a 28-day cycle of treatment with 10 mg of lenalidomide (excluding value-added tax) is £3780.

The committee noted that the incremental cost-effectiveness ratio for lenalidomide compared with best supportive care is uncertain because the proportion of people who might need treatment beyond 26 cycles is uncertain.

However, the committee accepted that a commitment from Celgene to publish data on the proportion of people receiving treatment beyond 26 cycles would provide reassurance that lenalidomide is a cost-effective use of NHS resources in MDS patients with 5q deletion.

NICE’s final draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues a final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Blood smear showing MDS

The UK’s National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending lenalidomide (Revlimid) as an option for treating myelodysplastic syndromes (MDS) characterized by 5q deletion.

Lenalidomide is approved in the European Union to treat transfusion-dependent anemia caused by low- or intermediate-1 risk MDS characterized by 5q deletion when other therapeutic options are insufficient or inadequate.

However, the main treatment option for this patient population in the UK is best supportive care, which involves regular red blood cell transfusions.

In earlier draft guidances, NICE did not support lenalidomide use in MDS patients with 5q deletion. Although data suggested the drug is effective for these patients, a NICE advisory committee was not convinced the drug provided a survival benefit.

But now, the committee has concluded that lenalidomide is a clinically effective treatment for these patients because it is associated with a statistically significant improvement in transfusion independence and health-related quality of life compared with placebo.

Furthermore, the committee said it is plausible that lenalidomide can indirectly improve overall survival by improving transfusion independence.

“The committee heard from clinical experts that lenalidomide is an effective therapy,” said Sir Andrew Dillon, NICE chief executive.

“Celgene–who market lenalidomide–worked with us to provide enough evidence to make it possible for us to recommend it for this group of people. Celgene provided a revised analysis and further information on their proposal for a reduction in the cost of the drug to the NHS [National Health Service].”

This patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. And Celgene will provide the drug free of charge for those people who receive more than 26 monthly cycles.

Lenalidomide is available in 21-day packs of 10 mg and 5 mg capsules at net prices of £3780 and £3570, respectively. The cost of a 28-day cycle of treatment with 10 mg of lenalidomide (excluding value-added tax) is £3780.

The committee noted that the incremental cost-effectiveness ratio for lenalidomide compared with best supportive care is uncertain because the proportion of people who might need treatment beyond 26 cycles is uncertain.

However, the committee accepted that a commitment from Celgene to publish data on the proportion of people receiving treatment beyond 26 cycles would provide reassurance that lenalidomide is a cost-effective use of NHS resources in MDS patients with 5q deletion.

NICE’s final draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues a final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Blood smear showing MDS

The UK’s National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending lenalidomide (Revlimid) as an option for treating myelodysplastic syndromes (MDS) characterized by 5q deletion.

Lenalidomide is approved in the European Union to treat transfusion-dependent anemia caused by low- or intermediate-1 risk MDS characterized by 5q deletion when other therapeutic options are insufficient or inadequate.

However, the main treatment option for this patient population in the UK is best supportive care, which involves regular red blood cell transfusions.

In earlier draft guidances, NICE did not support lenalidomide use in MDS patients with 5q deletion. Although data suggested the drug is effective for these patients, a NICE advisory committee was not convinced the drug provided a survival benefit.

But now, the committee has concluded that lenalidomide is a clinically effective treatment for these patients because it is associated with a statistically significant improvement in transfusion independence and health-related quality of life compared with placebo.

Furthermore, the committee said it is plausible that lenalidomide can indirectly improve overall survival by improving transfusion independence.

“The committee heard from clinical experts that lenalidomide is an effective therapy,” said Sir Andrew Dillon, NICE chief executive.

“Celgene–who market lenalidomide–worked with us to provide enough evidence to make it possible for us to recommend it for this group of people. Celgene provided a revised analysis and further information on their proposal for a reduction in the cost of the drug to the NHS [National Health Service].”

This patient access scheme involves the NHS paying for lenalidomide treatment for up to 26 monthly cycles. And Celgene will provide the drug free of charge for those people who receive more than 26 monthly cycles.

Lenalidomide is available in 21-day packs of 10 mg and 5 mg capsules at net prices of £3780 and £3570, respectively. The cost of a 28-day cycle of treatment with 10 mg of lenalidomide (excluding value-added tax) is £3780.

The committee noted that the incremental cost-effectiveness ratio for lenalidomide compared with best supportive care is uncertain because the proportion of people who might need treatment beyond 26 cycles is uncertain.

However, the committee accepted that a commitment from Celgene to publish data on the proportion of people receiving treatment beyond 26 cycles would provide reassurance that lenalidomide is a cost-effective use of NHS resources in MDS patients with 5q deletion.

NICE’s final draft guidance is now with consultees, who have the opportunity to appeal against it. Until NICE issues a final guidance, NHS bodies should make decisions locally on the funding of specific treatments.

Sleeping newborn

Credit: Vera Kratochvil

Severe combined immunodeficiency (SCID) may be nearly twice as common as we thought, new research suggests.

The study is the first combined analysis of more than 3 million infants screened for SCID in 10 US states and the Navajo Nation.

The results suggest SCID may affect as many as 1 in 58,000 infants. Previous estimates had indicated that 1 in 100,000 infants may be born with SCID.

This study and a related editorial were published in JAMA.

Jennifer Puck, MD, of the University of California, San Francisco, and her colleagues analyzed infants from 10 states and the Navajo Nation who were born from January 2008 through July 2013 and were screened for SCID.

The screening detected 52 cases of SCID among the 3,030,083 newborns, suggesting the disorder may affect roughly 1 in 58,000 infants.

The incidence of SCID was not significantly different in any state program, but it was higher in the Navajo Nation, where an ancestral trait is known to confer a higher risk of the disease.

Of the 52 infants who had SCID, 49 received therapies such as hematopoietic stem cell transplants, enzyme replacement therapy, and/or gene therapy. Three infants died before receiving treatment, and 4 children died after transplant, but the other 45 treated infants survived.

Of those 52 cases of SCID identified by newborn screening, 9 were considered “leaky SCID.” These infants had an incomplete mutation in a typical SCID gene, retaining small amounts of immune function that can actually be detrimental because the poorly regulated cells can attack the child’s own tissues.

“We’re finding that leaky SCID is more common than previously thought,” Dr Puck said. “Before screening, we’d typically not make the diagnosis for several months or even years, but because of newborn screening, they are being treated before they get into any trouble.”

The screening test detects more than a dozen genetic causes of SCID, in addition to other conditions with significantly low T cells. The researchers discovered that population-based testing uncovers a broader range of the underlying genetic causes for SCID than previously known.

For example, X-linked SCID, a form of the disorder caused by mutations in a gene on the X chromosome and affecting only males, was previously thought to account for half of SCID cases. But this study showed that only 19% of SCID infants had X-linked disease with a corresponding increase in other gene defects.

Moreover, the proportion of SCID infants without a known genetic defect (15%) was higher than anticipated, indicating that widespread screening presents opportunities to discover previously unknown genes implicated in SCID.

“The whole point of newborn screening is to identify conditions that are treatable, and for which early treatment saves lives,” Dr Puck said. “The excellent outcomes of SCID infants across the country reported in this study prove that SCID is such a condition.”

Sleeping newborn

Credit: Vera Kratochvil

Severe combined immunodeficiency (SCID) may be nearly twice as common as we thought, new research suggests.

The study is the first combined analysis of more than 3 million infants screened for SCID in 10 US states and the Navajo Nation.

The results suggest SCID may affect as many as 1 in 58,000 infants. Previous estimates had indicated that 1 in 100,000 infants may be born with SCID.

This study and a related editorial were published in JAMA.

Jennifer Puck, MD, of the University of California, San Francisco, and her colleagues analyzed infants from 10 states and the Navajo Nation who were born from January 2008 through July 2013 and were screened for SCID.

The screening detected 52 cases of SCID among the 3,030,083 newborns, suggesting the disorder may affect roughly 1 in 58,000 infants.

The incidence of SCID was not significantly different in any state program, but it was higher in the Navajo Nation, where an ancestral trait is known to confer a higher risk of the disease.

Of the 52 infants who had SCID, 49 received therapies such as hematopoietic stem cell transplants, enzyme replacement therapy, and/or gene therapy. Three infants died before receiving treatment, and 4 children died after transplant, but the other 45 treated infants survived.

Of those 52 cases of SCID identified by newborn screening, 9 were considered “leaky SCID.” These infants had an incomplete mutation in a typical SCID gene, retaining small amounts of immune function that can actually be detrimental because the poorly regulated cells can attack the child’s own tissues.

“We’re finding that leaky SCID is more common than previously thought,” Dr Puck said. “Before screening, we’d typically not make the diagnosis for several months or even years, but because of newborn screening, they are being treated before they get into any trouble.”

The screening test detects more than a dozen genetic causes of SCID, in addition to other conditions with significantly low T cells. The researchers discovered that population-based testing uncovers a broader range of the underlying genetic causes for SCID than previously known.

For example, X-linked SCID, a form of the disorder caused by mutations in a gene on the X chromosome and affecting only males, was previously thought to account for half of SCID cases. But this study showed that only 19% of SCID infants had X-linked disease with a corresponding increase in other gene defects.

Moreover, the proportion of SCID infants without a known genetic defect (15%) was higher than anticipated, indicating that widespread screening presents opportunities to discover previously unknown genes implicated in SCID.

“The whole point of newborn screening is to identify conditions that are treatable, and for which early treatment saves lives,” Dr Puck said. “The excellent outcomes of SCID infants across the country reported in this study prove that SCID is such a condition.”

Sleeping newborn

Credit: Vera Kratochvil

Severe combined immunodeficiency (SCID) may be nearly twice as common as we thought, new research suggests.

The study is the first combined analysis of more than 3 million infants screened for SCID in 10 US states and the Navajo Nation.

The results suggest SCID may affect as many as 1 in 58,000 infants. Previous estimates had indicated that 1 in 100,000 infants may be born with SCID.

This study and a related editorial were published in JAMA.

Jennifer Puck, MD, of the University of California, San Francisco, and her colleagues analyzed infants from 10 states and the Navajo Nation who were born from January 2008 through July 2013 and were screened for SCID.

The screening detected 52 cases of SCID among the 3,030,083 newborns, suggesting the disorder may affect roughly 1 in 58,000 infants.

The incidence of SCID was not significantly different in any state program, but it was higher in the Navajo Nation, where an ancestral trait is known to confer a higher risk of the disease.

Of the 52 infants who had SCID, 49 received therapies such as hematopoietic stem cell transplants, enzyme replacement therapy, and/or gene therapy. Three infants died before receiving treatment, and 4 children died after transplant, but the other 45 treated infants survived.

Of those 52 cases of SCID identified by newborn screening, 9 were considered “leaky SCID.” These infants had an incomplete mutation in a typical SCID gene, retaining small amounts of immune function that can actually be detrimental because the poorly regulated cells can attack the child’s own tissues.

“We’re finding that leaky SCID is more common than previously thought,” Dr Puck said. “Before screening, we’d typically not make the diagnosis for several months or even years, but because of newborn screening, they are being treated before they get into any trouble.”

The screening test detects more than a dozen genetic causes of SCID, in addition to other conditions with significantly low T cells. The researchers discovered that population-based testing uncovers a broader range of the underlying genetic causes for SCID than previously known.

For example, X-linked SCID, a form of the disorder caused by mutations in a gene on the X chromosome and affecting only males, was previously thought to account for half of SCID cases. But this study showed that only 19% of SCID infants had X-linked disease with a corresponding increase in other gene defects.

Moreover, the proportion of SCID infants without a known genetic defect (15%) was higher than anticipated, indicating that widespread screening presents opportunities to discover previously unknown genes implicated in SCID.

“The whole point of newborn screening is to identify conditions that are treatable, and for which early treatment saves lives,” Dr Puck said. “The excellent outcomes of SCID infants across the country reported in this study prove that SCID is such a condition.”

Prescriptions

Credit: CDC

A new study suggests the use of novel oral anticoagulants (NOACs) has surpassed warfarin use in the last few years, but the cost burden for NOACs is much higher than that of warfarin.

By mid-2013, the NOACs dabigatran, rivaroxaban, and apixaban accounted for 62% of all new anticoagulant prescriptions included in the study, but this represented 98% of the total anticoagulant costs.

These findings and a related commentary appear in The American Journal of Medicine.

NOAC use on the rise

To better understand NOAC use, researchers analyzed medical and prescription claims data from the insurance company Aetna. They identified 6893 patients with atrial fibrillation (AF) who were prescribed an oral anticoagulant from 2010 to 2013.

During that time, 45,472 anticoagulant prescriptions were filled—26,253 (57.7%) for warfarin, 14,922 (32.8%) for dabigatran, 4241 (9.3%) for rivaroxaban, and 56 (0.1%) for apixaban.

The researchers noted that the US Food and Drug Administration (FDA) approved dabigatran for use in AF patients in October 2010. And by October 2011, patients were as likely to receive dabigatran as warfarin.

Rivaroxaban was FDA-approved for AF in November 2011, and, by June 2013, the drug had overtaken both warfarin and dabigatran. Apixaban use increased the least, as the drug gained FDA approval for AF in December 2012.

Though NOACs appeared to be on the rise overall, the researchers found NOAC use was significantly less likely for women, patients living in lower income areas, and patients with higher CHADS₂, CHA₂DS₂-VASC, and HAS-BLED scores (P<0.001 for each variable).

The team said this finding is significant because, in the clinical trials supporting NOAC approvals, most patients had CHADS₂/CHA₂DS₂-VASC scores in the higher ranges.

“The greatest absolute benefit from novel anticoagulants has been shown in clinical trials to be among patients at highest baseline risk for stroke or systemic embolization,” said lead investigator Niteesh K. Choudhry, MD, PhD, of Brigham and Women’s Hospital in Boston.

“[This] is at odds with our observation of [physicians selecting] seemingly lower-risk patients for these drugs. Such a finding may reflect provider conservatism for new drug adoption, particularly given longitudinal experience with warfarin.”

Higher costs

Dr Choudhry and his colleagues also found that NOACs confer high healthcare cost consequences. The data revealed that NOACs represented 98% of the total dollars spent on anticoagulants from 2010 to 2013.

Over the first 6 months, the average combined patient and insurer cost associated with starting a NOAC was $900 greater than the cost of starting warfarin.

“Average patient out-of-pocket and insurance spending was more than 5-fold and 15-fold higher, respectively, for novel anticoagulants as compared with warfarin,” Dr Choudhry said. “A 6-month difference in total costs of $900 in our cohort translates into billions of dollars at a national level.”

Taking their findings together, the researchers said this study suggests more information may be needed to fully understand the implications of rising NOAC prescriptions.

“These findings point to the need to conduct ongoing surveillance of the adoption of new agents into clinical practice,” Dr Choudhry said, “as well as the need for robust, real-world comparative-effectiveness analyses of these medications, to enable patients and providers to make informed decisions about their relative benefit, safety, and cost-effectiveness.”

Prescriptions

Credit: CDC

A new study suggests the use of novel oral anticoagulants (NOACs) has surpassed warfarin use in the last few years, but the cost burden for NOACs is much higher than that of warfarin.

By mid-2013, the NOACs dabigatran, rivaroxaban, and apixaban accounted for 62% of all new anticoagulant prescriptions included in the study, but this represented 98% of the total anticoagulant costs.

These findings and a related commentary appear in The American Journal of Medicine.

NOAC use on the rise

To better understand NOAC use, researchers analyzed medical and prescription claims data from the insurance company Aetna. They identified 6893 patients with atrial fibrillation (AF) who were prescribed an oral anticoagulant from 2010 to 2013.

During that time, 45,472 anticoagulant prescriptions were filled—26,253 (57.7%) for warfarin, 14,922 (32.8%) for dabigatran, 4241 (9.3%) for rivaroxaban, and 56 (0.1%) for apixaban.

The researchers noted that the US Food and Drug Administration (FDA) approved dabigatran for use in AF patients in October 2010. And by October 2011, patients were as likely to receive dabigatran as warfarin.

Rivaroxaban was FDA-approved for AF in November 2011, and, by June 2013, the drug had overtaken both warfarin and dabigatran. Apixaban use increased the least, as the drug gained FDA approval for AF in December 2012.

Though NOACs appeared to be on the rise overall, the researchers found NOAC use was significantly less likely for women, patients living in lower income areas, and patients with higher CHADS₂, CHA₂DS₂-VASC, and HAS-BLED scores (P<0.001 for each variable).

The team said this finding is significant because, in the clinical trials supporting NOAC approvals, most patients had CHADS₂/CHA₂DS₂-VASC scores in the higher ranges.

“The greatest absolute benefit from novel anticoagulants has been shown in clinical trials to be among patients at highest baseline risk for stroke or systemic embolization,” said lead investigator Niteesh K. Choudhry, MD, PhD, of Brigham and Women’s Hospital in Boston.

“[This] is at odds with our observation of [physicians selecting] seemingly lower-risk patients for these drugs. Such a finding may reflect provider conservatism for new drug adoption, particularly given longitudinal experience with warfarin.”

Higher costs

Dr Choudhry and his colleagues also found that NOACs confer high healthcare cost consequences. The data revealed that NOACs represented 98% of the total dollars spent on anticoagulants from 2010 to 2013.

Over the first 6 months, the average combined patient and insurer cost associated with starting a NOAC was $900 greater than the cost of starting warfarin.

“Average patient out-of-pocket and insurance spending was more than 5-fold and 15-fold higher, respectively, for novel anticoagulants as compared with warfarin,” Dr Choudhry said. “A 6-month difference in total costs of $900 in our cohort translates into billions of dollars at a national level.”

Taking their findings together, the researchers said this study suggests more information may be needed to fully understand the implications of rising NOAC prescriptions.

“These findings point to the need to conduct ongoing surveillance of the adoption of new agents into clinical practice,” Dr Choudhry said, “as well as the need for robust, real-world comparative-effectiveness analyses of these medications, to enable patients and providers to make informed decisions about their relative benefit, safety, and cost-effectiveness.”

Prescriptions

Credit: CDC

A new study suggests the use of novel oral anticoagulants (NOACs) has surpassed warfarin use in the last few years, but the cost burden for NOACs is much higher than that of warfarin.

By mid-2013, the NOACs dabigatran, rivaroxaban, and apixaban accounted for 62% of all new anticoagulant prescriptions included in the study, but this represented 98% of the total anticoagulant costs.

These findings and a related commentary appear in The American Journal of Medicine.

NOAC use on the rise

To better understand NOAC use, researchers analyzed medical and prescription claims data from the insurance company Aetna. They identified 6893 patients with atrial fibrillation (AF) who were prescribed an oral anticoagulant from 2010 to 2013.

During that time, 45,472 anticoagulant prescriptions were filled—26,253 (57.7%) for warfarin, 14,922 (32.8%) for dabigatran, 4241 (9.3%) for rivaroxaban, and 56 (0.1%) for apixaban.

The researchers noted that the US Food and Drug Administration (FDA) approved dabigatran for use in AF patients in October 2010. And by October 2011, patients were as likely to receive dabigatran as warfarin.

Rivaroxaban was FDA-approved for AF in November 2011, and, by June 2013, the drug had overtaken both warfarin and dabigatran. Apixaban use increased the least, as the drug gained FDA approval for AF in December 2012.

Though NOACs appeared to be on the rise overall, the researchers found NOAC use was significantly less likely for women, patients living in lower income areas, and patients with higher CHADS₂, CHA₂DS₂-VASC, and HAS-BLED scores (P<0.001 for each variable).

The team said this finding is significant because, in the clinical trials supporting NOAC approvals, most patients had CHADS₂/CHA₂DS₂-VASC scores in the higher ranges.

“The greatest absolute benefit from novel anticoagulants has been shown in clinical trials to be among patients at highest baseline risk for stroke or systemic embolization,” said lead investigator Niteesh K. Choudhry, MD, PhD, of Brigham and Women’s Hospital in Boston.

“[This] is at odds with our observation of [physicians selecting] seemingly lower-risk patients for these drugs. Such a finding may reflect provider conservatism for new drug adoption, particularly given longitudinal experience with warfarin.”

Higher costs

Dr Choudhry and his colleagues also found that NOACs confer high healthcare cost consequences. The data revealed that NOACs represented 98% of the total dollars spent on anticoagulants from 2010 to 2013.

Over the first 6 months, the average combined patient and insurer cost associated with starting a NOAC was $900 greater than the cost of starting warfarin.

“Average patient out-of-pocket and insurance spending was more than 5-fold and 15-fold higher, respectively, for novel anticoagulants as compared with warfarin,” Dr Choudhry said. “A 6-month difference in total costs of $900 in our cohort translates into billions of dollars at a national level.”

Taking their findings together, the researchers said this study suggests more information may be needed to fully understand the implications of rising NOAC prescriptions.

“These findings point to the need to conduct ongoing surveillance of the adoption of new agents into clinical practice,” Dr Choudhry said, “as well as the need for robust, real-world comparative-effectiveness analyses of these medications, to enable patients and providers to make informed decisions about their relative benefit, safety, and cost-effectiveness.”

PARIS – The usual rule that the larger an aortic aneurysm grows the greater the risk it will undergo dissection or rupture doesn’t work in patients with giant-cell arteritis. Their aortic aneurysms appear liable to dissect or rupture at any size after the diagnosis of giant-cell arteritis occurs, based on a retrospective study of 195 patients followed at a single U.S. center.

"Aortic size at diagnosis or last follow-up did not predict aortic dissection or rupture," Dr. Ashima Makol reported at the annual European Congress of Rheumatology, nor were linear, serial measurements of aortic size able to reliably predict risk for these complications in patients with GCA. Without a reliable way to identify patients with GCA at risk for dissection or rupture, the only management advice remaining is to follow GCA patients annually with imaging, said Dr. Makol, a rheumatologist at the Mayo Clinic in Rochester, Minn.

Positron emission tomography, CT angiography, or MR angiography seem to be the best ways to follow these patients, but if those are too costly to do annually, then transesophageal echocardiography or a chest x-ray are other options, Dr. Makol said in an interview.

Although 30% of patients with GCA have a vasculitis that involves the aorta and its branches and an increased risk for developing aortic aneurysms, the way these aneurysms change over time and the relationship between aneurysm size and the risk for dissection or rupture in GCA patients were not previously reported. To address this, Dr. Makol and her associates reviewed 195 patients with GCA and an aortic aneurysm seen at the Mayo Clinic during 2000-2012.

The aneurysms occurred in the ascending thoracic aorta in 161 patients (83%), the descending thoracic aorta in 21 (11%), and the abdominal aorta in the remaining 13 patients (7%). (Percentages total 101% because of rounding.) The patients averaged 74 years old, 62% were women, and 49% had a history of smoking.

During follow-up, 14 patients (7%) had an aneurysm dissection, and 1 patient (1%) had an aneurysm rupture, the investigators reported. All of the dissections and the rupture occurred in thoracic aorta aneurysms.

At the time of GCA diagnosis, the average aneurysm size in the 15 patients who developed an aneurysm complication was 51 mm, which was very similar to the average size of 49 mm in the 180 patients who did not have an aneurysm dissection or rupture during follow-up.

Patients also showed no clear link between aneurysm size at the time of dissection or rupture and the aneurysm size during follow-up of patients without these complications. The average maximum aneurysm diameter among the 15 patients with a complication at the time of their event was 54 mm, while the average aneurysm size at last follow-up among those without a dissection or rupture during follow-up was 50 mm, a difference that was not statistically significant, Dr. Makol said.

The average rate of aneurysm growth during 3 years of follow-up for all the GCA patients in the analysis was 1.59 mm/year, a rate "somewhat higher" than the average annual growth rate of 1 mm/year reported for aortic aneurysms in patients without inflammatory disease. The 54-mm average aneurysm diameter at the time of dissection or rupture in the CGA patients was "somewhat lower" than the 65-mm average aneurysm diameter seen at the time of dissection or rupture in patients without inflammatory disease, she noted.

The study is the first reported to look at the pattern of aneurysm growth and complications in GCA patients, although it is limited to the retrospective experience at one tertiary referral center and so may reflect a referral bias, Dr. Makol said. But the inability of the analysis to identify aneurysm characteristics in GCA patients that can telegraph an increased risk for complications means that all GCA patients with an aortic aneurysm need careful surveillance by annual imaging, she advised.

Dr. Makol said that she had no disclosures.

[email protected]

PARIS – The usual rule that the larger an aortic aneurysm grows the greater the risk it will undergo dissection or rupture doesn’t work in patients with giant-cell arteritis. Their aortic aneurysms appear liable to dissect or rupture at any size after the diagnosis of giant-cell arteritis occurs, based on a retrospective study of 195 patients followed at a single U.S. center.

"Aortic size at diagnosis or last follow-up did not predict aortic dissection or rupture," Dr. Ashima Makol reported at the annual European Congress of Rheumatology, nor were linear, serial measurements of aortic size able to reliably predict risk for these complications in patients with GCA. Without a reliable way to identify patients with GCA at risk for dissection or rupture, the only management advice remaining is to follow GCA patients annually with imaging, said Dr. Makol, a rheumatologist at the Mayo Clinic in Rochester, Minn.

Positron emission tomography, CT angiography, or MR angiography seem to be the best ways to follow these patients, but if those are too costly to do annually, then transesophageal echocardiography or a chest x-ray are other options, Dr. Makol said in an interview.

Although 30% of patients with GCA have a vasculitis that involves the aorta and its branches and an increased risk for developing aortic aneurysms, the way these aneurysms change over time and the relationship between aneurysm size and the risk for dissection or rupture in GCA patients were not previously reported. To address this, Dr. Makol and her associates reviewed 195 patients with GCA and an aortic aneurysm seen at the Mayo Clinic during 2000-2012.

The aneurysms occurred in the ascending thoracic aorta in 161 patients (83%), the descending thoracic aorta in 21 (11%), and the abdominal aorta in the remaining 13 patients (7%). (Percentages total 101% because of rounding.) The patients averaged 74 years old, 62% were women, and 49% had a history of smoking.

During follow-up, 14 patients (7%) had an aneurysm dissection, and 1 patient (1%) had an aneurysm rupture, the investigators reported. All of the dissections and the rupture occurred in thoracic aorta aneurysms.

At the time of GCA diagnosis, the average aneurysm size in the 15 patients who developed an aneurysm complication was 51 mm, which was very similar to the average size of 49 mm in the 180 patients who did not have an aneurysm dissection or rupture during follow-up.

Patients also showed no clear link between aneurysm size at the time of dissection or rupture and the aneurysm size during follow-up of patients without these complications. The average maximum aneurysm diameter among the 15 patients with a complication at the time of their event was 54 mm, while the average aneurysm size at last follow-up among those without a dissection or rupture during follow-up was 50 mm, a difference that was not statistically significant, Dr. Makol said.

The average rate of aneurysm growth during 3 years of follow-up for all the GCA patients in the analysis was 1.59 mm/year, a rate "somewhat higher" than the average annual growth rate of 1 mm/year reported for aortic aneurysms in patients without inflammatory disease. The 54-mm average aneurysm diameter at the time of dissection or rupture in the CGA patients was "somewhat lower" than the 65-mm average aneurysm diameter seen at the time of dissection or rupture in patients without inflammatory disease, she noted.

The study is the first reported to look at the pattern of aneurysm growth and complications in GCA patients, although it is limited to the retrospective experience at one tertiary referral center and so may reflect a referral bias, Dr. Makol said. But the inability of the analysis to identify aneurysm characteristics in GCA patients that can telegraph an increased risk for complications means that all GCA patients with an aortic aneurysm need careful surveillance by annual imaging, she advised.

Dr. Makol said that she had no disclosures.

[email protected]

PARIS – The usual rule that the larger an aortic aneurysm grows the greater the risk it will undergo dissection or rupture doesn’t work in patients with giant-cell arteritis. Their aortic aneurysms appear liable to dissect or rupture at any size after the diagnosis of giant-cell arteritis occurs, based on a retrospective study of 195 patients followed at a single U.S. center.

"Aortic size at diagnosis or last follow-up did not predict aortic dissection or rupture," Dr. Ashima Makol reported at the annual European Congress of Rheumatology, nor were linear, serial measurements of aortic size able to reliably predict risk for these complications in patients with GCA. Without a reliable way to identify patients with GCA at risk for dissection or rupture, the only management advice remaining is to follow GCA patients annually with imaging, said Dr. Makol, a rheumatologist at the Mayo Clinic in Rochester, Minn.

Positron emission tomography, CT angiography, or MR angiography seem to be the best ways to follow these patients, but if those are too costly to do annually, then transesophageal echocardiography or a chest x-ray are other options, Dr. Makol said in an interview.

Although 30% of patients with GCA have a vasculitis that involves the aorta and its branches and an increased risk for developing aortic aneurysms, the way these aneurysms change over time and the relationship between aneurysm size and the risk for dissection or rupture in GCA patients were not previously reported. To address this, Dr. Makol and her associates reviewed 195 patients with GCA and an aortic aneurysm seen at the Mayo Clinic during 2000-2012.

The aneurysms occurred in the ascending thoracic aorta in 161 patients (83%), the descending thoracic aorta in 21 (11%), and the abdominal aorta in the remaining 13 patients (7%). (Percentages total 101% because of rounding.) The patients averaged 74 years old, 62% were women, and 49% had a history of smoking.

During follow-up, 14 patients (7%) had an aneurysm dissection, and 1 patient (1%) had an aneurysm rupture, the investigators reported. All of the dissections and the rupture occurred in thoracic aorta aneurysms.

At the time of GCA diagnosis, the average aneurysm size in the 15 patients who developed an aneurysm complication was 51 mm, which was very similar to the average size of 49 mm in the 180 patients who did not have an aneurysm dissection or rupture during follow-up.

Patients also showed no clear link between aneurysm size at the time of dissection or rupture and the aneurysm size during follow-up of patients without these complications. The average maximum aneurysm diameter among the 15 patients with a complication at the time of their event was 54 mm, while the average aneurysm size at last follow-up among those without a dissection or rupture during follow-up was 50 mm, a difference that was not statistically significant, Dr. Makol said.

The average rate of aneurysm growth during 3 years of follow-up for all the GCA patients in the analysis was 1.59 mm/year, a rate "somewhat higher" than the average annual growth rate of 1 mm/year reported for aortic aneurysms in patients without inflammatory disease. The 54-mm average aneurysm diameter at the time of dissection or rupture in the CGA patients was "somewhat lower" than the 65-mm average aneurysm diameter seen at the time of dissection or rupture in patients without inflammatory disease, she noted.

The study is the first reported to look at the pattern of aneurysm growth and complications in GCA patients, although it is limited to the retrospective experience at one tertiary referral center and so may reflect a referral bias, Dr. Makol said. But the inability of the analysis to identify aneurysm characteristics in GCA patients that can telegraph an increased risk for complications means that all GCA patients with an aortic aneurysm need careful surveillance by annual imaging, she advised.

Dr. Makol said that she had no disclosures.

[email protected]

SAN FRANCISCO – Infection with hepatitis C virus is a risk factor for poor outcomes after kidney transplantation, but infection with human immunodeficiency virus is not, finds a cohort study reported at the 2014 World Transplant Congress.

"Centers should be more selective in transplanting HCV-positive kidney transplant candidates. But controlled HIV infection should no longer be perceived as a barrier to kidney transplantation," said first author Dr. Deirdre L. Sawinski of the University of Pennsylvania in Philadelphia.

Further, control of hepatitis C with treatment should be a priority in hopes of improving outcomes, she added.

Researchers studied 111,990 patients from the United Network for Organ Sharing (UNOS) database who had a known serostatus and underwent kidney transplantation in 1996 or later when highly active antiretroviral therapy became widespread. Overall, 4.6% were infected with HCV alone, 0.4% were infected with HIV alone, and 0.1% were coinfected.

Multivariate analyses adjusted for a variety of donor and recipient characteristics, and included a variable for transplant after 2001. This time point "reflects both the year in which more than half of patients were discharged on tacrolimus maintenance therapy as well as the year in which interferon therapy was approved for treatment for hepatitis C," Dr. Sawinski said.

HCV-infected patients and especially HCV and HIV-coinfected patients had significantly higher risks of death (hazard ratio, 1.52 and 3.83, respectively) and of graft loss (HR, 1.48 and 3.40, respectively), compared with uninfected patients. In contrast, patients infected with HIV alone were not at higher risk of death or graft loss.

"The main cause of death for the reference [uninfected] group and the hep C–positive patients was listed as cardiovascular disease, whereas the HIV-positive and coinfected patients most often had infection as their causes of death," Dr. Sawinski said. "However, you have to take that with a grain of salt as 40% of patients across all four groups had missing data [regarding] cause of death."

The risk persisted even after researchers corrected for the impact of antibody-depleting therapy during transplantation on graft survival. However, the UNOS database does not provide information about treatment with interferon before and after direct-acting antiretroviral drugs became available. Additionally, Dr. Sawinski noted, "the UNOS data set does not identify which patients are actually viremic vs. antibody positive." Data are similarly nonspecific for deceased donors but are detailed (antibody positive vs. viremic) for living donors.

A secondary analysis considered a cohort of 180,177 patients with unknown serostatus. In this analysis, the risks of death and graft loss were elevated among patients with dual unknown status relative to those known to be dually uninfected (HR, 1.06 and 1.02), according to data reported at the 2014 World Transplant Congress, which was sponsored by the American Society of Transplant Surgeons. However, these small elevations of risk are "probably not clinically meaningful," commented Dr. Sawinski, who disclosed no relevant conflicts of interest. Risks were not significantly elevated for HCV-positive patients vs. HCV-positive, HIV-unknown patients, or for HIV-positive patients vs. HIV-positive, HCV-unknown patients.

Additionally, main study findings were essentially the same when the cohort with known serostatus and the cohort with an unknown serostatus were combined and the researchers assumed the unknown status patients were uninfected.

SAN FRANCISCO – Infection with hepatitis C virus is a risk factor for poor outcomes after kidney transplantation, but infection with human immunodeficiency virus is not, finds a cohort study reported at the 2014 World Transplant Congress.

"Centers should be more selective in transplanting HCV-positive kidney transplant candidates. But controlled HIV infection should no longer be perceived as a barrier to kidney transplantation," said first author Dr. Deirdre L. Sawinski of the University of Pennsylvania in Philadelphia.

Further, control of hepatitis C with treatment should be a priority in hopes of improving outcomes, she added.

Researchers studied 111,990 patients from the United Network for Organ Sharing (UNOS) database who had a known serostatus and underwent kidney transplantation in 1996 or later when highly active antiretroviral therapy became widespread. Overall, 4.6% were infected with HCV alone, 0.4% were infected with HIV alone, and 0.1% were coinfected.

Multivariate analyses adjusted for a variety of donor and recipient characteristics, and included a variable for transplant after 2001. This time point "reflects both the year in which more than half of patients were discharged on tacrolimus maintenance therapy as well as the year in which interferon therapy was approved for treatment for hepatitis C," Dr. Sawinski said.

HCV-infected patients and especially HCV and HIV-coinfected patients had significantly higher risks of death (hazard ratio, 1.52 and 3.83, respectively) and of graft loss (HR, 1.48 and 3.40, respectively), compared with uninfected patients. In contrast, patients infected with HIV alone were not at higher risk of death or graft loss.

"The main cause of death for the reference [uninfected] group and the hep C–positive patients was listed as cardiovascular disease, whereas the HIV-positive and coinfected patients most often had infection as their causes of death," Dr. Sawinski said. "However, you have to take that with a grain of salt as 40% of patients across all four groups had missing data [regarding] cause of death."

The risk persisted even after researchers corrected for the impact of antibody-depleting therapy during transplantation on graft survival. However, the UNOS database does not provide information about treatment with interferon before and after direct-acting antiretroviral drugs became available. Additionally, Dr. Sawinski noted, "the UNOS data set does not identify which patients are actually viremic vs. antibody positive." Data are similarly nonspecific for deceased donors but are detailed (antibody positive vs. viremic) for living donors.

A secondary analysis considered a cohort of 180,177 patients with unknown serostatus. In this analysis, the risks of death and graft loss were elevated among patients with dual unknown status relative to those known to be dually uninfected (HR, 1.06 and 1.02), according to data reported at the 2014 World Transplant Congress, which was sponsored by the American Society of Transplant Surgeons. However, these small elevations of risk are "probably not clinically meaningful," commented Dr. Sawinski, who disclosed no relevant conflicts of interest. Risks were not significantly elevated for HCV-positive patients vs. HCV-positive, HIV-unknown patients, or for HIV-positive patients vs. HIV-positive, HCV-unknown patients.

Additionally, main study findings were essentially the same when the cohort with known serostatus and the cohort with an unknown serostatus were combined and the researchers assumed the unknown status patients were uninfected.

SAN FRANCISCO – Infection with hepatitis C virus is a risk factor for poor outcomes after kidney transplantation, but infection with human immunodeficiency virus is not, finds a cohort study reported at the 2014 World Transplant Congress.

"Centers should be more selective in transplanting HCV-positive kidney transplant candidates. But controlled HIV infection should no longer be perceived as a barrier to kidney transplantation," said first author Dr. Deirdre L. Sawinski of the University of Pennsylvania in Philadelphia.

Further, control of hepatitis C with treatment should be a priority in hopes of improving outcomes, she added.

Researchers studied 111,990 patients from the United Network for Organ Sharing (UNOS) database who had a known serostatus and underwent kidney transplantation in 1996 or later when highly active antiretroviral therapy became widespread. Overall, 4.6% were infected with HCV alone, 0.4% were infected with HIV alone, and 0.1% were coinfected.

Multivariate analyses adjusted for a variety of donor and recipient characteristics, and included a variable for transplant after 2001. This time point "reflects both the year in which more than half of patients were discharged on tacrolimus maintenance therapy as well as the year in which interferon therapy was approved for treatment for hepatitis C," Dr. Sawinski said.

HCV-infected patients and especially HCV and HIV-coinfected patients had significantly higher risks of death (hazard ratio, 1.52 and 3.83, respectively) and of graft loss (HR, 1.48 and 3.40, respectively), compared with uninfected patients. In contrast, patients infected with HIV alone were not at higher risk of death or graft loss.

"The main cause of death for the reference [uninfected] group and the hep C–positive patients was listed as cardiovascular disease, whereas the HIV-positive and coinfected patients most often had infection as their causes of death," Dr. Sawinski said. "However, you have to take that with a grain of salt as 40% of patients across all four groups had missing data [regarding] cause of death."

The risk persisted even after researchers corrected for the impact of antibody-depleting therapy during transplantation on graft survival. However, the UNOS database does not provide information about treatment with interferon before and after direct-acting antiretroviral drugs became available. Additionally, Dr. Sawinski noted, "the UNOS data set does not identify which patients are actually viremic vs. antibody positive." Data are similarly nonspecific for deceased donors but are detailed (antibody positive vs. viremic) for living donors.

A secondary analysis considered a cohort of 180,177 patients with unknown serostatus. In this analysis, the risks of death and graft loss were elevated among patients with dual unknown status relative to those known to be dually uninfected (HR, 1.06 and 1.02), according to data reported at the 2014 World Transplant Congress, which was sponsored by the American Society of Transplant Surgeons. However, these small elevations of risk are "probably not clinically meaningful," commented Dr. Sawinski, who disclosed no relevant conflicts of interest. Risks were not significantly elevated for HCV-positive patients vs. HCV-positive, HIV-unknown patients, or for HIV-positive patients vs. HIV-positive, HCV-unknown patients.

Additionally, main study findings were essentially the same when the cohort with known serostatus and the cohort with an unknown serostatus were combined and the researchers assumed the unknown status patients were uninfected.

Hoarding disorder (HD), catego­rized in DSM-5 under obsessive-compulsive and related disorders, is defined as the “persistent difficulty discarding or parting with possessions, regardless of their actual value.”¹ Hoarders feel that they need to save items, and expe­rience distress when discarding them. Prevalence of HD among the general pop­ulation is 2% to 5%.

Compulsive hoarders usually keep old items in their home that they do not intend to use. In severe cases, the clutter is so great that areas of the home cannot be used or entered. Hoarders tend to iso­late themselves and usually do not invite people home, perhaps because they are embarrassed about the clutter or anxious that someone might try to clean the house. Hoarders may travel long distances to col­lect items others have discarded.

Hoarding can lead to psychiatric disor­ders and social problems. Hoarders tend to not develop attachment with people because they are more attached to their possessions. They may avoid social inter­actions; in turn, others avoid them. This isolation can lead to depression, anxi­ety, and substance abuse. Hoarders may be evicted from their home if the clutter makes the house dangerous or unfit to live in it. Compulsive hoarding is detrimental to the hoarder and the health and well-being of family members. Hoarding can coexist or can be result of other psychiatric disorders (Table).

Neural mechanism in hoarding
Hoarders may start to accumulate and store large quantities of items because of a cognitive deficit, such as trouble making decisions or poor recognition or acknowl­edgement of the situation, or maladaptive thoughts. Tolin et al¹ found the anterior cingulate cortex and insula was stimulus-dependent in patients with HD. Functional MRI showed when patients with HD were shown an item that was their possession, they exhibited an abnormal brain activ­ity, compared with low activity when the items shown were not theirs.

Interventions
Choice of treatment depends on the age of the patient and severity of illness: behav­ioral, medical, or a combination of both. For an uncomplicated case, management can begin with behavioral modification.

Behavioral modifications. HD can stem from any of several variables, including greater response latency for decision-making about possessions and maladaptive beliefs about, and emotional attachment to, possessions, which can lead to intense emotional experiences about the prospect of losing those posses­sions.² Cognitive-behavioral therapy has shown promising results for treating HD by addressing the aforementioned fac­tors. A step-by-step approach usually is feasible and convenient for the therapist and patient. It involves gradual mental detachment from items to accommodate the patient’s pace.²

Pharmacotherapy. There is no clear evi­dence for treating HD with any particular drug. Hoarders are less likely to use psy­chotropics, possibly because of poor insight (eg, they do not realize the potentially dangerous living conditions hoarding cre­ates).³ Because HD is related to obsessive-compulsive disorder, it is intuitive to con­sider a selective serotonin reuptake inhibitor.

There is still a need for more research on management of HD.

Disclosure
Dr. Silman reports no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.

Hoarding disorder (HD), catego­rized in DSM-5 under obsessive-compulsive and related disorders, is defined as the “persistent difficulty discarding or parting with possessions, regardless of their actual value.”¹ Hoarders feel that they need to save items, and expe­rience distress when discarding them. Prevalence of HD among the general pop­ulation is 2% to 5%.

Compulsive hoarders usually keep old items in their home that they do not intend to use. In severe cases, the clutter is so great that areas of the home cannot be used or entered. Hoarders tend to iso­late themselves and usually do not invite people home, perhaps because they are embarrassed about the clutter or anxious that someone might try to clean the house. Hoarders may travel long distances to col­lect items others have discarded.

Hoarding can lead to psychiatric disor­ders and social problems. Hoarders tend to not develop attachment with people because they are more attached to their possessions. They may avoid social inter­actions; in turn, others avoid them. This isolation can lead to depression, anxi­ety, and substance abuse. Hoarders may be evicted from their home if the clutter makes the house dangerous or unfit to live in it. Compulsive hoarding is detrimental to the hoarder and the health and well-being of family members. Hoarding can coexist or can be result of other psychiatric disorders (Table).

Neural mechanism in hoarding
Hoarders may start to accumulate and store large quantities of items because of a cognitive deficit, such as trouble making decisions or poor recognition or acknowl­edgement of the situation, or maladaptive thoughts. Tolin et al¹ found the anterior cingulate cortex and insula was stimulus-dependent in patients with HD. Functional MRI showed when patients with HD were shown an item that was their possession, they exhibited an abnormal brain activ­ity, compared with low activity when the items shown were not theirs.

Interventions
Choice of treatment depends on the age of the patient and severity of illness: behav­ioral, medical, or a combination of both. For an uncomplicated case, management can begin with behavioral modification.

Behavioral modifications. HD can stem from any of several variables, including greater response latency for decision-making about possessions and maladaptive beliefs about, and emotional attachment to, possessions, which can lead to intense emotional experiences about the prospect of losing those posses­sions.² Cognitive-behavioral therapy has shown promising results for treating HD by addressing the aforementioned fac­tors. A step-by-step approach usually is feasible and convenient for the therapist and patient. It involves gradual mental detachment from items to accommodate the patient’s pace.²

Pharmacotherapy. There is no clear evi­dence for treating HD with any particular drug. Hoarders are less likely to use psy­chotropics, possibly because of poor insight (eg, they do not realize the potentially dangerous living conditions hoarding cre­ates).³ Because HD is related to obsessive-compulsive disorder, it is intuitive to con­sider a selective serotonin reuptake inhibitor.

There is still a need for more research on management of HD.

Disclosure
Dr. Silman reports no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.

Hoarding disorder (HD), catego­rized in DSM-5 under obsessive-compulsive and related disorders, is defined as the “persistent difficulty discarding or parting with possessions, regardless of their actual value.”¹ Hoarders feel that they need to save items, and expe­rience distress when discarding them. Prevalence of HD among the general pop­ulation is 2% to 5%.

Compulsive hoarders usually keep old items in their home that they do not intend to use. In severe cases, the clutter is so great that areas of the home cannot be used or entered. Hoarders tend to iso­late themselves and usually do not invite people home, perhaps because they are embarrassed about the clutter or anxious that someone might try to clean the house. Hoarders may travel long distances to col­lect items others have discarded.

Hoarding can lead to psychiatric disor­ders and social problems. Hoarders tend to not develop attachment with people because they are more attached to their possessions. They may avoid social inter­actions; in turn, others avoid them. This isolation can lead to depression, anxi­ety, and substance abuse. Hoarders may be evicted from their home if the clutter makes the house dangerous or unfit to live in it. Compulsive hoarding is detrimental to the hoarder and the health and well-being of family members. Hoarding can coexist or can be result of other psychiatric disorders (Table).

Neural mechanism in hoarding
Hoarders may start to accumulate and store large quantities of items because of a cognitive deficit, such as trouble making decisions or poor recognition or acknowl­edgement of the situation, or maladaptive thoughts. Tolin et al¹ found the anterior cingulate cortex and insula was stimulus-dependent in patients with HD. Functional MRI showed when patients with HD were shown an item that was their possession, they exhibited an abnormal brain activ­ity, compared with low activity when the items shown were not theirs.

Interventions
Choice of treatment depends on the age of the patient and severity of illness: behav­ioral, medical, or a combination of both. For an uncomplicated case, management can begin with behavioral modification.

Behavioral modifications. HD can stem from any of several variables, including greater response latency for decision-making about possessions and maladaptive beliefs about, and emotional attachment to, possessions, which can lead to intense emotional experiences about the prospect of losing those posses­sions.² Cognitive-behavioral therapy has shown promising results for treating HD by addressing the aforementioned fac­tors. A step-by-step approach usually is feasible and convenient for the therapist and patient. It involves gradual mental detachment from items to accommodate the patient’s pace.²

Pharmacotherapy. There is no clear evi­dence for treating HD with any particular drug. Hoarders are less likely to use psy­chotropics, possibly because of poor insight (eg, they do not realize the potentially dangerous living conditions hoarding cre­ates).³ Because HD is related to obsessive-compulsive disorder, it is intuitive to con­sider a selective serotonin reuptake inhibitor.

There is still a need for more research on management of HD.

Disclosure
Dr. Silman reports no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.

Once regarded as a curse of the urban poor, heroin now has a stronghold on many suburban, middle class communities.I was stunned to learn that heroin use is so prevalent in the quiet Baltimore suburb where I work that our local police officers carry Narcan kits. Use has become so ubiquitous in our country that U.S. Attorney General Eric Holder has said law enforcement officials should consider carrying heroin’s antidote. From Smalltown U.S.A. to booming metropolises, this inexpensive narcotic is wreaking havoc on individuals and their families.

Sure, I admit an occasional patient with "a history of heroin abuse" who takes methadone. Rarely, I may see physical evidence of heroin use. But in most cases, patients who use heroin present as overdose cases in the ED, where they are discharged home when they are stable or admitted directly to the intensive care unit. Sadly, I recently received from the ICU a transfer of a heroin overdose patient – a handsome young man in his 20s, his entire life ahead of him, loving parents and siblings at his bedside. He was completely oblivious to everything around him – comatose, on hospice, dying before he had a real chance to live.

There was nothing I could do for him or his family other than to provide comfort. But perhaps I can do more for future potential overdose victims. You know, the heroin users admitted for a skin abscess after missing a vein or for DKA because they were too high to remember to take their insulin. Yes, we are busy; but we need to take 5-10 minutes to address the issue, to listen to the user’s story and encourage them, uplift them. Substance abuse counselors are invaluable, but by taking the time to care about our patients as individuals, hospitalists might just be the straw to break the camel’s back of heroin use for someone. Considering that most heroin addicts these days are young adults, a 10 minute investment of our time now may help buy those patients back another 40 or 50 years of their lives.

Dr. Hester is a hospitalist at Baltimore-Washington Medical Center in Glen Burnie, Md. She is the creator of the Patient Whiz, a patient-engagement app for iOS. Reach her at [email protected].

Once regarded as a curse of the urban poor, heroin now has a stronghold on many suburban, middle class communities.I was stunned to learn that heroin use is so prevalent in the quiet Baltimore suburb where I work that our local police officers carry Narcan kits. Use has become so ubiquitous in our country that U.S. Attorney General Eric Holder has said law enforcement officials should consider carrying heroin’s antidote. From Smalltown U.S.A. to booming metropolises, this inexpensive narcotic is wreaking havoc on individuals and their families.

Sure, I admit an occasional patient with "a history of heroin abuse" who takes methadone. Rarely, I may see physical evidence of heroin use. But in most cases, patients who use heroin present as overdose cases in the ED, where they are discharged home when they are stable or admitted directly to the intensive care unit. Sadly, I recently received from the ICU a transfer of a heroin overdose patient – a handsome young man in his 20s, his entire life ahead of him, loving parents and siblings at his bedside. He was completely oblivious to everything around him – comatose, on hospice, dying before he had a real chance to live.

There was nothing I could do for him or his family other than to provide comfort. But perhaps I can do more for future potential overdose victims. You know, the heroin users admitted for a skin abscess after missing a vein or for DKA because they were too high to remember to take their insulin. Yes, we are busy; but we need to take 5-10 minutes to address the issue, to listen to the user’s story and encourage them, uplift them. Substance abuse counselors are invaluable, but by taking the time to care about our patients as individuals, hospitalists might just be the straw to break the camel’s back of heroin use for someone. Considering that most heroin addicts these days are young adults, a 10 minute investment of our time now may help buy those patients back another 40 or 50 years of their lives.

Dr. Hester is a hospitalist at Baltimore-Washington Medical Center in Glen Burnie, Md. She is the creator of the Patient Whiz, a patient-engagement app for iOS. Reach her at [email protected].

Once regarded as a curse of the urban poor, heroin now has a stronghold on many suburban, middle class communities.I was stunned to learn that heroin use is so prevalent in the quiet Baltimore suburb where I work that our local police officers carry Narcan kits. Use has become so ubiquitous in our country that U.S. Attorney General Eric Holder has said law enforcement officials should consider carrying heroin’s antidote. From Smalltown U.S.A. to booming metropolises, this inexpensive narcotic is wreaking havoc on individuals and their families.

Sure, I admit an occasional patient with "a history of heroin abuse" who takes methadone. Rarely, I may see physical evidence of heroin use. But in most cases, patients who use heroin present as overdose cases in the ED, where they are discharged home when they are stable or admitted directly to the intensive care unit. Sadly, I recently received from the ICU a transfer of a heroin overdose patient – a handsome young man in his 20s, his entire life ahead of him, loving parents and siblings at his bedside. He was completely oblivious to everything around him – comatose, on hospice, dying before he had a real chance to live.

There was nothing I could do for him or his family other than to provide comfort. But perhaps I can do more for future potential overdose victims. You know, the heroin users admitted for a skin abscess after missing a vein or for DKA because they were too high to remember to take their insulin. Yes, we are busy; but we need to take 5-10 minutes to address the issue, to listen to the user’s story and encourage them, uplift them. Substance abuse counselors are invaluable, but by taking the time to care about our patients as individuals, hospitalists might just be the straw to break the camel’s back of heroin use for someone. Considering that most heroin addicts these days are young adults, a 10 minute investment of our time now may help buy those patients back another 40 or 50 years of their lives.

Dr. Hester is a hospitalist at Baltimore-Washington Medical Center in Glen Burnie, Md. She is the creator of the Patient Whiz, a patient-engagement app for iOS. Reach her at [email protected].

Dear Dr. Mossman,

When I take a call from a treatment-seeker at our outpatient clinic, I ask brief screen­ing questions to determine whether our services would be appropriate. Shortly after I screened one caller, Ms. C, she called back requesting a medication refill and asking about her diagnosis.

What obligation do I have to Ms. C? Is she my patient? Would I be liable if I didn’t help her out and something bad happened to her?

Submitted by “Dr. S”

Office and hospital Web sites, LinkedIn profiles, and Facebook pages are just a few of the ways that people find physicians and learn about their services. But most 21st century doctor-patient relationships still start with 19th century technology: a telephone call.

Talking with prospective patients before setting up an appointment makes sense. A short conversation can clarify whether you offer the services that a caller needs and increases the show-up rate for initial appointments.¹

But if you ask for some personal his­tory and information about symptoms in a screening interview, does that make the caller your patient? Ms. C seemed to have thought so. To find out whether Ms. C was right and to learn how Dr. S should handle initial telephone calls, we’ll look at:
   • the rationale for screening callers before initiating treatment
   • features of screening that can create a doctor-patient relationship
   • how to fulfill duties that result from screening.
 

Why screen prospective patients?
Mental health treatment has become more diversified and specialized over the past 30 years. No psychiatrist nowadays has all the therapeutic skills that all potential patients might need.

Before speaking to you, a treatment-seeker often won’t know whether your practice style will fit his (her) needs. You might prefer not to provide medica­tion management for another clinician’s psychotherapy patient or, if you’re like most psychiatrists, you might not offer psychotherapy.

In the absence of prior obligation (eg, agreeing to provide coverage for an emergency room), physicians may struc­ture their practices and contract for their services as they see fit²—but this leaves you with some obligation to screen poten­tial patients for appropriate mutual fit. In years past, some psychiatrists saw poten­tial patients for an in-office evaluation to decide whether to provide treatment—a practicethat remains acceptable if the per­son is told, when the appointment is made, that the first meeting is “to meet each other and see if you want to establish a treatment relationship.”³ 

Good treatment plans take into account patients’ temperament, emotional state, cognitive capacity, culture, family circum­stances, substance use, and medical his­tory.⁴ Common mental conditions often can be identified in a telephone call.^5,6 Although the diagnostic accuracy of such efforts is uncertain,⁷ such calls can help practitio­ners determine whether they offer the right services for callers. Good decisions about initiating care always take financial pres­sures and constraints into account,⁸ and a pre-appointment telephone call can address those issues, too.

For all these reasons, talking to a prospective patient before he comes to see you makes sense. Screening lets you decide:  
   • whether you’re the right clinician for his needs  
   • who the right clinician is if you are not  
   • whether he should seek emergency evaluation when the situation sounds urgent.

Do phone calls start treatment?
As Dr. S’s questions show, telephone screenings might leave some callers think­ing that treatment has started, even before their first office appointment. Having a treatment relationship is a prerequisite to malpractice liability,⁹ and courts have con­cluded that, under the right circumstances, telephone assessments do create physician-patient relationships.

Creating a physician-patient relationship
How or when might telephone screen­ing make someone your patient? This question doesn’t have a precise answer, but how courts decided similar ques­tions has depended on the questions the physician asked and whether the physician offered what sounded like medical advice.^10,11 A physician-patient relationship forms when the phy­sician takes some implied or affirmative action to treat, see, examine, care for, or offer a diagnosis to the patient,^9,12,13 such as:
   • knowingly accepting someone as a patient¹⁴
   • explicitly agreeing to treat a person
   • “acting in some other way such that the patient might reasonably be led to assume a doctor-patient relationship has been established.”¹⁵

Also, the “fact that a physician does not deal directly with a patient does not neces­sarily preclude the existence of a physician-patient relationship,”¹² so a telephone conversation can create such a relationship if it contains the right elements. Table 1¹⁶ highlights actions that, during the course of screening, might constitute initiation of a physician-patient relationship. Table 2 offers suggestions for managing ini­tial telephone contacts to reduce the chance of inadvertently creating a physician-patient relationship.

Prescribing
Although features of the original screening interview indicated that Ms. C was not yet Dr. S’s patient, prescribing certainly would commence a physician-patient relation­ship.¹⁸ But even if the screening had made Ms. C a patient, refilling her prescription now probably is a bad idea.

Assuming that a physician-patient rela­tionship exists, it is unlikely that a short telephone interview gave Dr. S enough infor­mation about Ms. C’s medical history and present mental status to ensure that his diag­nostic reasoning would not be faulty. It also is unlikely that telephone screening allowed Dr. S to meet the standard of care for pre­scribing—a process that involves choosing medications suitable to the patient’s clini­cal needs, checking the results of any neces­sary lab tests, and obtaining appropriate informed consent.¹⁹

Satisfying duties
Outpatient facilities can instruct telephone screeners to conduct interviews in ways that reduce inadvertent establishment of a treatment relationship, but establishing such a relationship cannot be avoided in all cases. If a caller is distraught or in crisis, for example, compassion dictates helping him, and some callers (eg, Ms. C) may feel they have a firmer treatment relationship than actually exists.

Once you have created a physician-patient relationship, you must continue that relationship until you end it appropriately.³ That does not mean you have to provide definitive treatment; you simply need to exercise “reasonable care according to the standards of the profession.”^16,20 If a caller telephones in an emergency situation, for example, the screening clinician should take appropriate steps to ensure safety, which might include calling law enforcement or facilitating hospitalization.³

One way to fulfill the duties of a physi­cian-patient relationship inadvertently estab­lished during initial screening is through explicit discharge (if medically appropriate) or transfer of care to another physician.¹⁵ A prudent clinic or practitioner will describe other mental health resources in the commu­nity and sometimes assist with referral if the inquiring potential patient needs services that the provider does not offer.

In many communities, finding appro­priate mental health resources is diffi­cult. Creative approaches to this problem include transitional psychiatry or crisis sup­port clinics that serve as a “bridge” to lon­ger-term services,^21,22 preliminary process groups,²³ and telepsychiatry transitional clinics.²⁴ When a clinic does not accept a person as a patient, the clinic should clearly document 1) key features of the contact and 2) the rationale for that decision
 

Bottom Line
You have a right and a responsibility to screen prospective patients for good fit to your treatment services. In doing so, however, you might inadvertently create a physician-patient relationship. If this happens, you should fulfill your clinical responsibilities, as you would for any patient, by helping the patient get appropriate care from you or another provider.
 

Disclosures
The authors report no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.

Dear Dr. Mossman,

When I take a call from a treatment-seeker at our outpatient clinic, I ask brief screen­ing questions to determine whether our services would be appropriate. Shortly after I screened one caller, Ms. C, she called back requesting a medication refill and asking about her diagnosis.

What obligation do I have to Ms. C? Is she my patient? Would I be liable if I didn’t help her out and something bad happened to her?

Submitted by “Dr. S”

Office and hospital Web sites, LinkedIn profiles, and Facebook pages are just a few of the ways that people find physicians and learn about their services. But most 21st century doctor-patient relationships still start with 19th century technology: a telephone call.

Talking with prospective patients before setting up an appointment makes sense. A short conversation can clarify whether you offer the services that a caller needs and increases the show-up rate for initial appointments.¹

But if you ask for some personal his­tory and information about symptoms in a screening interview, does that make the caller your patient? Ms. C seemed to have thought so. To find out whether Ms. C was right and to learn how Dr. S should handle initial telephone calls, we’ll look at:
   • the rationale for screening callers before initiating treatment
   • features of screening that can create a doctor-patient relationship
   • how to fulfill duties that result from screening.
 

Why screen prospective patients?
Mental health treatment has become more diversified and specialized over the past 30 years. No psychiatrist nowadays has all the therapeutic skills that all potential patients might need.

Before speaking to you, a treatment-seeker often won’t know whether your practice style will fit his (her) needs. You might prefer not to provide medica­tion management for another clinician’s psychotherapy patient or, if you’re like most psychiatrists, you might not offer psychotherapy.

In the absence of prior obligation (eg, agreeing to provide coverage for an emergency room), physicians may struc­ture their practices and contract for their services as they see fit²—but this leaves you with some obligation to screen poten­tial patients for appropriate mutual fit. In years past, some psychiatrists saw poten­tial patients for an in-office evaluation to decide whether to provide treatment—a practicethat remains acceptable if the per­son is told, when the appointment is made, that the first meeting is “to meet each other and see if you want to establish a treatment relationship.”³ 

Good treatment plans take into account patients’ temperament, emotional state, cognitive capacity, culture, family circum­stances, substance use, and medical his­tory.⁴ Common mental conditions often can be identified in a telephone call.^5,6 Although the diagnostic accuracy of such efforts is uncertain,⁷ such calls can help practitio­ners determine whether they offer the right services for callers. Good decisions about initiating care always take financial pres­sures and constraints into account,⁸ and a pre-appointment telephone call can address those issues, too.

For all these reasons, talking to a prospective patient before he comes to see you makes sense. Screening lets you decide:  
   • whether you’re the right clinician for his needs  
   • who the right clinician is if you are not  
   • whether he should seek emergency evaluation when the situation sounds urgent.

Do phone calls start treatment?
As Dr. S’s questions show, telephone screenings might leave some callers think­ing that treatment has started, even before their first office appointment. Having a treatment relationship is a prerequisite to malpractice liability,⁹ and courts have con­cluded that, under the right circumstances, telephone assessments do create physician-patient relationships.

Creating a physician-patient relationship
How or when might telephone screen­ing make someone your patient? This question doesn’t have a precise answer, but how courts decided similar ques­tions has depended on the questions the physician asked and whether the physician offered what sounded like medical advice.^10,11 A physician-patient relationship forms when the phy­sician takes some implied or affirmative action to treat, see, examine, care for, or offer a diagnosis to the patient,^9,12,13 such as:
   • knowingly accepting someone as a patient¹⁴
   • explicitly agreeing to treat a person
   • “acting in some other way such that the patient might reasonably be led to assume a doctor-patient relationship has been established.”¹⁵

Also, the “fact that a physician does not deal directly with a patient does not neces­sarily preclude the existence of a physician-patient relationship,”¹² so a telephone conversation can create such a relationship if it contains the right elements. Table 1¹⁶ highlights actions that, during the course of screening, might constitute initiation of a physician-patient relationship. Table 2 offers suggestions for managing ini­tial telephone contacts to reduce the chance of inadvertently creating a physician-patient relationship.

Prescribing
Although features of the original screening interview indicated that Ms. C was not yet Dr. S’s patient, prescribing certainly would commence a physician-patient relation­ship.¹⁸ But even if the screening had made Ms. C a patient, refilling her prescription now probably is a bad idea.

Assuming that a physician-patient rela­tionship exists, it is unlikely that a short telephone interview gave Dr. S enough infor­mation about Ms. C’s medical history and present mental status to ensure that his diag­nostic reasoning would not be faulty. It also is unlikely that telephone screening allowed Dr. S to meet the standard of care for pre­scribing—a process that involves choosing medications suitable to the patient’s clini­cal needs, checking the results of any neces­sary lab tests, and obtaining appropriate informed consent.¹⁹

Satisfying duties
Outpatient facilities can instruct telephone screeners to conduct interviews in ways that reduce inadvertent establishment of a treatment relationship, but establishing such a relationship cannot be avoided in all cases. If a caller is distraught or in crisis, for example, compassion dictates helping him, and some callers (eg, Ms. C) may feel they have a firmer treatment relationship than actually exists.

Once you have created a physician-patient relationship, you must continue that relationship until you end it appropriately.³ That does not mean you have to provide definitive treatment; you simply need to exercise “reasonable care according to the standards of the profession.”^16,20 If a caller telephones in an emergency situation, for example, the screening clinician should take appropriate steps to ensure safety, which might include calling law enforcement or facilitating hospitalization.³

One way to fulfill the duties of a physi­cian-patient relationship inadvertently estab­lished during initial screening is through explicit discharge (if medically appropriate) or transfer of care to another physician.¹⁵ A prudent clinic or practitioner will describe other mental health resources in the commu­nity and sometimes assist with referral if the inquiring potential patient needs services that the provider does not offer.

In many communities, finding appro­priate mental health resources is diffi­cult. Creative approaches to this problem include transitional psychiatry or crisis sup­port clinics that serve as a “bridge” to lon­ger-term services,^21,22 preliminary process groups,²³ and telepsychiatry transitional clinics.²⁴ When a clinic does not accept a person as a patient, the clinic should clearly document 1) key features of the contact and 2) the rationale for that decision
 

Bottom Line
You have a right and a responsibility to screen prospective patients for good fit to your treatment services. In doing so, however, you might inadvertently create a physician-patient relationship. If this happens, you should fulfill your clinical responsibilities, as you would for any patient, by helping the patient get appropriate care from you or another provider.
 

Disclosures
The authors report no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.

Dear Dr. Mossman,

When I take a call from a treatment-seeker at our outpatient clinic, I ask brief screen­ing questions to determine whether our services would be appropriate. Shortly after I screened one caller, Ms. C, she called back requesting a medication refill and asking about her diagnosis.

What obligation do I have to Ms. C? Is she my patient? Would I be liable if I didn’t help her out and something bad happened to her?

Submitted by “Dr. S”

Office and hospital Web sites, LinkedIn profiles, and Facebook pages are just a few of the ways that people find physicians and learn about their services. But most 21st century doctor-patient relationships still start with 19th century technology: a telephone call.

Talking with prospective patients before setting up an appointment makes sense. A short conversation can clarify whether you offer the services that a caller needs and increases the show-up rate for initial appointments.¹

But if you ask for some personal his­tory and information about symptoms in a screening interview, does that make the caller your patient? Ms. C seemed to have thought so. To find out whether Ms. C was right and to learn how Dr. S should handle initial telephone calls, we’ll look at:
   • the rationale for screening callers before initiating treatment
   • features of screening that can create a doctor-patient relationship
   • how to fulfill duties that result from screening.
 

Why screen prospective patients?
Mental health treatment has become more diversified and specialized over the past 30 years. No psychiatrist nowadays has all the therapeutic skills that all potential patients might need.

Before speaking to you, a treatment-seeker often won’t know whether your practice style will fit his (her) needs. You might prefer not to provide medica­tion management for another clinician’s psychotherapy patient or, if you’re like most psychiatrists, you might not offer psychotherapy.

In the absence of prior obligation (eg, agreeing to provide coverage for an emergency room), physicians may struc­ture their practices and contract for their services as they see fit²—but this leaves you with some obligation to screen poten­tial patients for appropriate mutual fit. In years past, some psychiatrists saw poten­tial patients for an in-office evaluation to decide whether to provide treatment—a practicethat remains acceptable if the per­son is told, when the appointment is made, that the first meeting is “to meet each other and see if you want to establish a treatment relationship.”³ 

Good treatment plans take into account patients’ temperament, emotional state, cognitive capacity, culture, family circum­stances, substance use, and medical his­tory.⁴ Common mental conditions often can be identified in a telephone call.^5,6 Although the diagnostic accuracy of such efforts is uncertain,⁷ such calls can help practitio­ners determine whether they offer the right services for callers. Good decisions about initiating care always take financial pres­sures and constraints into account,⁸ and a pre-appointment telephone call can address those issues, too.

For all these reasons, talking to a prospective patient before he comes to see you makes sense. Screening lets you decide:  
   • whether you’re the right clinician for his needs  
   • who the right clinician is if you are not  
   • whether he should seek emergency evaluation when the situation sounds urgent.

Do phone calls start treatment?
As Dr. S’s questions show, telephone screenings might leave some callers think­ing that treatment has started, even before their first office appointment. Having a treatment relationship is a prerequisite to malpractice liability,⁹ and courts have con­cluded that, under the right circumstances, telephone assessments do create physician-patient relationships.

Creating a physician-patient relationship
How or when might telephone screen­ing make someone your patient? This question doesn’t have a precise answer, but how courts decided similar ques­tions has depended on the questions the physician asked and whether the physician offered what sounded like medical advice.^10,11 A physician-patient relationship forms when the phy­sician takes some implied or affirmative action to treat, see, examine, care for, or offer a diagnosis to the patient,^9,12,13 such as:
   • knowingly accepting someone as a patient¹⁴
   • explicitly agreeing to treat a person
   • “acting in some other way such that the patient might reasonably be led to assume a doctor-patient relationship has been established.”¹⁵

Also, the “fact that a physician does not deal directly with a patient does not neces­sarily preclude the existence of a physician-patient relationship,”¹² so a telephone conversation can create such a relationship if it contains the right elements. Table 1¹⁶ highlights actions that, during the course of screening, might constitute initiation of a physician-patient relationship. Table 2 offers suggestions for managing ini­tial telephone contacts to reduce the chance of inadvertently creating a physician-patient relationship.

Prescribing
Although features of the original screening interview indicated that Ms. C was not yet Dr. S’s patient, prescribing certainly would commence a physician-patient relation­ship.¹⁸ But even if the screening had made Ms. C a patient, refilling her prescription now probably is a bad idea.

Assuming that a physician-patient rela­tionship exists, it is unlikely that a short telephone interview gave Dr. S enough infor­mation about Ms. C’s medical history and present mental status to ensure that his diag­nostic reasoning would not be faulty. It also is unlikely that telephone screening allowed Dr. S to meet the standard of care for pre­scribing—a process that involves choosing medications suitable to the patient’s clini­cal needs, checking the results of any neces­sary lab tests, and obtaining appropriate informed consent.¹⁹

Satisfying duties
Outpatient facilities can instruct telephone screeners to conduct interviews in ways that reduce inadvertent establishment of a treatment relationship, but establishing such a relationship cannot be avoided in all cases. If a caller is distraught or in crisis, for example, compassion dictates helping him, and some callers (eg, Ms. C) may feel they have a firmer treatment relationship than actually exists.

Once you have created a physician-patient relationship, you must continue that relationship until you end it appropriately.³ That does not mean you have to provide definitive treatment; you simply need to exercise “reasonable care according to the standards of the profession.”^16,20 If a caller telephones in an emergency situation, for example, the screening clinician should take appropriate steps to ensure safety, which might include calling law enforcement or facilitating hospitalization.³

One way to fulfill the duties of a physi­cian-patient relationship inadvertently estab­lished during initial screening is through explicit discharge (if medically appropriate) or transfer of care to another physician.¹⁵ A prudent clinic or practitioner will describe other mental health resources in the commu­nity and sometimes assist with referral if the inquiring potential patient needs services that the provider does not offer.

In many communities, finding appro­priate mental health resources is diffi­cult. Creative approaches to this problem include transitional psychiatry or crisis sup­port clinics that serve as a “bridge” to lon­ger-term services,^21,22 preliminary process groups,²³ and telepsychiatry transitional clinics.²⁴ When a clinic does not accept a person as a patient, the clinic should clearly document 1) key features of the contact and 2) the rationale for that decision
 

Bottom Line
You have a right and a responsibility to screen prospective patients for good fit to your treatment services. In doing so, however, you might inadvertently create a physician-patient relationship. If this happens, you should fulfill your clinical responsibilities, as you would for any patient, by helping the patient get appropriate care from you or another provider.
 

Disclosures
The authors report no financial relationship with any company whose products are mentioned in this article or with manufacturers of competing products.