User login
Sickle Cell Gene Therapy ‘Truly Transformative’
.
More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.
“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added.
For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
One and Done
Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US.
People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events.
On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.
Both are one-time, single-dose cell-based gene therapies.
With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.
Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel.
All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months.
Most patients achieved a durable globin response through their final follow-up visit.
Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events.
In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.
Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.
Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke.
One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.
To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.
However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”
The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio.
A version of this article appeared on Medscape.com.
.
More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.
“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added.
For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
One and Done
Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US.
People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events.
On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.
Both are one-time, single-dose cell-based gene therapies.
With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.
Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel.
All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months.
Most patients achieved a durable globin response through their final follow-up visit.
Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events.
In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.
Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.
Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke.
One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.
To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.
However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”
The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio.
A version of this article appeared on Medscape.com.
.
More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.
“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added.
For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
One and Done
Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US.
People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events.
On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.
Both are one-time, single-dose cell-based gene therapies.
With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.
Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel.
All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months.
Most patients achieved a durable globin response through their final follow-up visit.
Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events.
In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.
Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.
Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke.
One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.
To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.
However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”
The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio.
A version of this article appeared on Medscape.com.
FROM ASH 2023
CAR T-Cell Therapy: Cure for Systemic Autoimmune Diseases?
A single infusion of autologous CD19-directed CAR T-cell therapy led to persistent, drug-free remission in 15 patients with life-threatening systemic lupus erythematosus, idiopathic inflammatory myositis, or systemic sclerosis, according to research presented at the American Society of Hematology annual meeting.
The responses persisted at 15 months median follow-up, with all patients achieving complete remission, reported Fabian Mueller, MD, of the Bavarian Cancer Research Center and Friedrich-Alexander University of Erlangen-Nuremberg, Bavaria, Germany.
The CAR T-cell treatment appears to provide an “entire reset of B cells,” possibly even a cure, for these 15 patients who had run out of treatment options and had short life expectancies, Dr. Mueller said. “It’s impressive that we have treated these patients.”
Some of the cases have been described previously — including in Annals of the Rheumatic Diseases earlier this year, Nature Medicine in 2022, and the New England Journal of Medicine in 2021.
Now with substantially longer follow-up, the investigators have gained a greater understanding of “the B-cell biology behind our treatment,” Dr. Mueller said. However, “we need longer follow-up to establish how effective the treatment is going to be in the long run.”
All 15 patients included in the analysis were heavily pretreated and had multi-organ involvement. Prior to CAR T-cell therapy, patients had a median disease duration of 3 years, ranging from 1 to as many as 20 years, and had failed a median of five previous treatments. Patients were young — a median age of 36 years — which is much younger than most oncology patients who undergo CAR T-cell therapy, Dr. Mueller said.
The 15 patients underwent typical lymphodepletion and were apheresed and treated with a single infusion of 1 x 106 CD19 CAR T cells per kg of body weight — an established safe dose used in a phase 1 trial of B cell malignancies.
The CAR T cells, manufactured in-house, expanded rapidly, peaking around day 9. B cells disappeared within 7 days and began to reoccur in peripheral blood in all patients between 60 and 180 days. However, no disease flares occurred, Dr. Mueller said.
After 3 months, eight patients with systemic lupus erythematosus showed no sign of disease activity and dramatic improvement in symptoms. Three patients with idiopathic inflammatory myositis experienced major improvements in symptoms and normalization of creatinine kinase levels, the most clinically relevant marker for muscle inflammation. And three of four patients with systemic sclerosis demonstrated major improvements in symptoms and no new disease activity. These responses lasted for a median of 15 months, and all patients stopped taking immunosuppressive drugs.
Patients also tolerated the CAR T-cell treatment well, especially compared with the adverse event profile in oncology patients. Only low-grade inflammatory CAR T-related side effects occurred, and few patients required support for B-cell-derived immune deficiency.
However, infectious complications occurred in 14 patients, including urinary tract and respiratory infections, over the 12-month follow-up. One patient was hospitalized for severe pneumonia a few weeks after CAR T therapy, and two patients experienced herpes zoster reactivations, including one at 6 months and one at 12 months following treatment.
During a press briefing at the ASH conference, Dr. Mueller addressed the “critical question” of patient selection for CAR T-cell therapy, especially in light of the recently announced US Food and Drug Administration investigation exploring whether CAR T cells can cause secondary blood cancers.
Although the T-cell malignancy risk complicates matters, CAR T cells appear to behave differently in patients with autoimmune diseases than those with cancer, he said.
“We don’t understand the biology” related to the malignancy risk yet, Dr. Mueller said, but the benefit for end-of-life patients with no other treatment option likely outweighs the risk. That risk-benefit assessment, however, is more uncertain for those with less severe autoimmune diseases.
For now, it’s important to conduct individual assessments and inform patients about the risk, Dr. Mueller said.
Dr. Mueller disclosed relationships with BMS, AstraZeneca, Gilead, Janssen, Miltenyi Biomedicine, Novartis, Incyte, Abbvie, Sobi, and BeiGene.
A version of this article appeared on Medscape.com.
A single infusion of autologous CD19-directed CAR T-cell therapy led to persistent, drug-free remission in 15 patients with life-threatening systemic lupus erythematosus, idiopathic inflammatory myositis, or systemic sclerosis, according to research presented at the American Society of Hematology annual meeting.
The responses persisted at 15 months median follow-up, with all patients achieving complete remission, reported Fabian Mueller, MD, of the Bavarian Cancer Research Center and Friedrich-Alexander University of Erlangen-Nuremberg, Bavaria, Germany.
The CAR T-cell treatment appears to provide an “entire reset of B cells,” possibly even a cure, for these 15 patients who had run out of treatment options and had short life expectancies, Dr. Mueller said. “It’s impressive that we have treated these patients.”
Some of the cases have been described previously — including in Annals of the Rheumatic Diseases earlier this year, Nature Medicine in 2022, and the New England Journal of Medicine in 2021.
Now with substantially longer follow-up, the investigators have gained a greater understanding of “the B-cell biology behind our treatment,” Dr. Mueller said. However, “we need longer follow-up to establish how effective the treatment is going to be in the long run.”
All 15 patients included in the analysis were heavily pretreated and had multi-organ involvement. Prior to CAR T-cell therapy, patients had a median disease duration of 3 years, ranging from 1 to as many as 20 years, and had failed a median of five previous treatments. Patients were young — a median age of 36 years — which is much younger than most oncology patients who undergo CAR T-cell therapy, Dr. Mueller said.
The 15 patients underwent typical lymphodepletion and were apheresed and treated with a single infusion of 1 x 106 CD19 CAR T cells per kg of body weight — an established safe dose used in a phase 1 trial of B cell malignancies.
The CAR T cells, manufactured in-house, expanded rapidly, peaking around day 9. B cells disappeared within 7 days and began to reoccur in peripheral blood in all patients between 60 and 180 days. However, no disease flares occurred, Dr. Mueller said.
After 3 months, eight patients with systemic lupus erythematosus showed no sign of disease activity and dramatic improvement in symptoms. Three patients with idiopathic inflammatory myositis experienced major improvements in symptoms and normalization of creatinine kinase levels, the most clinically relevant marker for muscle inflammation. And three of four patients with systemic sclerosis demonstrated major improvements in symptoms and no new disease activity. These responses lasted for a median of 15 months, and all patients stopped taking immunosuppressive drugs.
Patients also tolerated the CAR T-cell treatment well, especially compared with the adverse event profile in oncology patients. Only low-grade inflammatory CAR T-related side effects occurred, and few patients required support for B-cell-derived immune deficiency.
However, infectious complications occurred in 14 patients, including urinary tract and respiratory infections, over the 12-month follow-up. One patient was hospitalized for severe pneumonia a few weeks after CAR T therapy, and two patients experienced herpes zoster reactivations, including one at 6 months and one at 12 months following treatment.
During a press briefing at the ASH conference, Dr. Mueller addressed the “critical question” of patient selection for CAR T-cell therapy, especially in light of the recently announced US Food and Drug Administration investigation exploring whether CAR T cells can cause secondary blood cancers.
Although the T-cell malignancy risk complicates matters, CAR T cells appear to behave differently in patients with autoimmune diseases than those with cancer, he said.
“We don’t understand the biology” related to the malignancy risk yet, Dr. Mueller said, but the benefit for end-of-life patients with no other treatment option likely outweighs the risk. That risk-benefit assessment, however, is more uncertain for those with less severe autoimmune diseases.
For now, it’s important to conduct individual assessments and inform patients about the risk, Dr. Mueller said.
Dr. Mueller disclosed relationships with BMS, AstraZeneca, Gilead, Janssen, Miltenyi Biomedicine, Novartis, Incyte, Abbvie, Sobi, and BeiGene.
A version of this article appeared on Medscape.com.
A single infusion of autologous CD19-directed CAR T-cell therapy led to persistent, drug-free remission in 15 patients with life-threatening systemic lupus erythematosus, idiopathic inflammatory myositis, or systemic sclerosis, according to research presented at the American Society of Hematology annual meeting.
The responses persisted at 15 months median follow-up, with all patients achieving complete remission, reported Fabian Mueller, MD, of the Bavarian Cancer Research Center and Friedrich-Alexander University of Erlangen-Nuremberg, Bavaria, Germany.
The CAR T-cell treatment appears to provide an “entire reset of B cells,” possibly even a cure, for these 15 patients who had run out of treatment options and had short life expectancies, Dr. Mueller said. “It’s impressive that we have treated these patients.”
Some of the cases have been described previously — including in Annals of the Rheumatic Diseases earlier this year, Nature Medicine in 2022, and the New England Journal of Medicine in 2021.
Now with substantially longer follow-up, the investigators have gained a greater understanding of “the B-cell biology behind our treatment,” Dr. Mueller said. However, “we need longer follow-up to establish how effective the treatment is going to be in the long run.”
All 15 patients included in the analysis were heavily pretreated and had multi-organ involvement. Prior to CAR T-cell therapy, patients had a median disease duration of 3 years, ranging from 1 to as many as 20 years, and had failed a median of five previous treatments. Patients were young — a median age of 36 years — which is much younger than most oncology patients who undergo CAR T-cell therapy, Dr. Mueller said.
The 15 patients underwent typical lymphodepletion and were apheresed and treated with a single infusion of 1 x 106 CD19 CAR T cells per kg of body weight — an established safe dose used in a phase 1 trial of B cell malignancies.
The CAR T cells, manufactured in-house, expanded rapidly, peaking around day 9. B cells disappeared within 7 days and began to reoccur in peripheral blood in all patients between 60 and 180 days. However, no disease flares occurred, Dr. Mueller said.
After 3 months, eight patients with systemic lupus erythematosus showed no sign of disease activity and dramatic improvement in symptoms. Three patients with idiopathic inflammatory myositis experienced major improvements in symptoms and normalization of creatinine kinase levels, the most clinically relevant marker for muscle inflammation. And three of four patients with systemic sclerosis demonstrated major improvements in symptoms and no new disease activity. These responses lasted for a median of 15 months, and all patients stopped taking immunosuppressive drugs.
Patients also tolerated the CAR T-cell treatment well, especially compared with the adverse event profile in oncology patients. Only low-grade inflammatory CAR T-related side effects occurred, and few patients required support for B-cell-derived immune deficiency.
However, infectious complications occurred in 14 patients, including urinary tract and respiratory infections, over the 12-month follow-up. One patient was hospitalized for severe pneumonia a few weeks after CAR T therapy, and two patients experienced herpes zoster reactivations, including one at 6 months and one at 12 months following treatment.
During a press briefing at the ASH conference, Dr. Mueller addressed the “critical question” of patient selection for CAR T-cell therapy, especially in light of the recently announced US Food and Drug Administration investigation exploring whether CAR T cells can cause secondary blood cancers.
Although the T-cell malignancy risk complicates matters, CAR T cells appear to behave differently in patients with autoimmune diseases than those with cancer, he said.
“We don’t understand the biology” related to the malignancy risk yet, Dr. Mueller said, but the benefit for end-of-life patients with no other treatment option likely outweighs the risk. That risk-benefit assessment, however, is more uncertain for those with less severe autoimmune diseases.
For now, it’s important to conduct individual assessments and inform patients about the risk, Dr. Mueller said.
Dr. Mueller disclosed relationships with BMS, AstraZeneca, Gilead, Janssen, Miltenyi Biomedicine, Novartis, Incyte, Abbvie, Sobi, and BeiGene.
A version of this article appeared on Medscape.com.
FROM ASH 2023
In real world, patients with myeloma have worse outcomes
The analysis, which included nearly 4,000 patients with multiple myeloma, revealed that patients in a real-world setting demonstrated worse progression-free and overall survival on six of seven standard treatments compared with patients evaluated in randomized controlled trials.
Lead author Alissa Visram, MD, MPH, who spoke about the study at the annual meeting of the American Society of Hematology, said the findings will likely change the way she speaks to patients about their potential outcomes.
“I’ll probably present both numbers [from real-life and clinical-trial data] and give them a sense of the best-case scenario,” Dr. Visram said during an ASH media briefing. But she said she will also caution her patients that the real-world numbers reflect how people on these drugs actually fare.
The effectiveness of multiple myeloma drugs remains unclear outside the clinical trial setting, explained Dr. Visram, of the Division of Hematology at the Ottawa Hospital Research Institute, Ottawa, Ontario, Canada. Outcomes from randomized controlled trials form the basis of drug approvals but many patients in the real world do not meet the “stringent” trial inclusion criteria.
Dr. Visram and colleagues launched the current study to better understand the potential differences between real-world and clinical trial outcomes. In the analysis, the researchers compared real-world outcomes among patients receiving seven standard multiple myeloma regimens covered by Ontario’s public health plan with patient outcomes reported in phase 3 randomized controlled trials.
The retrospective study included 3951 patients with newly diagnosed and refractory multiple myeloma treated from 2007 to 2020 in Ontario. Regimens for newly diagnosed transplant ineligible patients included lenalidomide plus dexamethasone and triple therapy with bortezomib, lenalidomide, and dexamethasone. Regimens for patients with relapsed disease included pomalidomide plus dexamethasone or carfilzomib plus dexamethasone as well as triple combinations including carfilzomib, lenalidomide, and dexamethasone.
Overall, Dr. Visram and colleagues found that patients in the real-world setting demonstrated worse overall survival for six of the seven regimens evaluated (pooled hazard ratio [HR], 1.75; P = .010).
The real-world patients also had worse progression-free survival for six of the seven regimens (pooled HR, 1.44; P = .034).
For these regimens, progression-free survival was at least 3-18 months longer in the clinical trial cohort, while median overall survival was at least 19 months longer compared with real-world patients, Dr. Visram explained.
The only regimen with comparable outcomes in the clinical trial and real-world settings was pomalidomide and dexamethasone, she said. One reason could be that patients receiving pomalidomide plus dexamethasone in the clinical trial setting had similar or more advanced disease than those in the real-world setting.
The study also found that adverse effects were similar between the clinical and real-world groups.
The next step, Dr. Visram said, would be to explore what’s driving the differences in outcomes.
Are patients in the real-world setting older or frailer? Do they have more advanced disease? Are providers using these regimens differently?
Mikkael A. Sekeres, MD, MS, explained that the difference likely comes down to the health of the patient.
Patients in these types of clinical trials “are just these pristine specimens of human beings except for the cancer that’s being treated,” Dr. Sekeres, of the Sylvester Comprehensive Cancer Center at the University of Miami, Florida, said in an earlier ASH press briefing.
Cynthia E. Dunbar, MD, noted that patients in clinical trials have other advantages as well.
“Patients who are able to enroll in clinical trials are more likely to be able to show up at the treatment center at the right time and for every dose, have transportation, and afford drugs to prevent side effects,” said Dr. Dunbar, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute and secretary of ASH. These patients also “might stay on the drug for longer, or they have nurses who are always encouraging them on how to make it through a toxicity.”
Dr. Dunbar said hematologists and patients should consider randomized controlled trials to be “the best possible outcome, and perhaps adjust their thinking if an individual patient is older, sicker, or less able to follow a regimen exactly.”
No study funding was reported. Dr. Visram reported consulting and honoraria relationships with Apotex, Janssen, and Sanofi. Other study authors reported multiple relationships with industry. Disclosures for Dr. Dunbar and Dr. Sekeres were unavailable.
A version of this article appeared on Medscape.com.
The analysis, which included nearly 4,000 patients with multiple myeloma, revealed that patients in a real-world setting demonstrated worse progression-free and overall survival on six of seven standard treatments compared with patients evaluated in randomized controlled trials.
Lead author Alissa Visram, MD, MPH, who spoke about the study at the annual meeting of the American Society of Hematology, said the findings will likely change the way she speaks to patients about their potential outcomes.
“I’ll probably present both numbers [from real-life and clinical-trial data] and give them a sense of the best-case scenario,” Dr. Visram said during an ASH media briefing. But she said she will also caution her patients that the real-world numbers reflect how people on these drugs actually fare.
The effectiveness of multiple myeloma drugs remains unclear outside the clinical trial setting, explained Dr. Visram, of the Division of Hematology at the Ottawa Hospital Research Institute, Ottawa, Ontario, Canada. Outcomes from randomized controlled trials form the basis of drug approvals but many patients in the real world do not meet the “stringent” trial inclusion criteria.
Dr. Visram and colleagues launched the current study to better understand the potential differences between real-world and clinical trial outcomes. In the analysis, the researchers compared real-world outcomes among patients receiving seven standard multiple myeloma regimens covered by Ontario’s public health plan with patient outcomes reported in phase 3 randomized controlled trials.
The retrospective study included 3951 patients with newly diagnosed and refractory multiple myeloma treated from 2007 to 2020 in Ontario. Regimens for newly diagnosed transplant ineligible patients included lenalidomide plus dexamethasone and triple therapy with bortezomib, lenalidomide, and dexamethasone. Regimens for patients with relapsed disease included pomalidomide plus dexamethasone or carfilzomib plus dexamethasone as well as triple combinations including carfilzomib, lenalidomide, and dexamethasone.
Overall, Dr. Visram and colleagues found that patients in the real-world setting demonstrated worse overall survival for six of the seven regimens evaluated (pooled hazard ratio [HR], 1.75; P = .010).
The real-world patients also had worse progression-free survival for six of the seven regimens (pooled HR, 1.44; P = .034).
For these regimens, progression-free survival was at least 3-18 months longer in the clinical trial cohort, while median overall survival was at least 19 months longer compared with real-world patients, Dr. Visram explained.
The only regimen with comparable outcomes in the clinical trial and real-world settings was pomalidomide and dexamethasone, she said. One reason could be that patients receiving pomalidomide plus dexamethasone in the clinical trial setting had similar or more advanced disease than those in the real-world setting.
The study also found that adverse effects were similar between the clinical and real-world groups.
The next step, Dr. Visram said, would be to explore what’s driving the differences in outcomes.
Are patients in the real-world setting older or frailer? Do they have more advanced disease? Are providers using these regimens differently?
Mikkael A. Sekeres, MD, MS, explained that the difference likely comes down to the health of the patient.
Patients in these types of clinical trials “are just these pristine specimens of human beings except for the cancer that’s being treated,” Dr. Sekeres, of the Sylvester Comprehensive Cancer Center at the University of Miami, Florida, said in an earlier ASH press briefing.
Cynthia E. Dunbar, MD, noted that patients in clinical trials have other advantages as well.
“Patients who are able to enroll in clinical trials are more likely to be able to show up at the treatment center at the right time and for every dose, have transportation, and afford drugs to prevent side effects,” said Dr. Dunbar, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute and secretary of ASH. These patients also “might stay on the drug for longer, or they have nurses who are always encouraging them on how to make it through a toxicity.”
Dr. Dunbar said hematologists and patients should consider randomized controlled trials to be “the best possible outcome, and perhaps adjust their thinking if an individual patient is older, sicker, or less able to follow a regimen exactly.”
No study funding was reported. Dr. Visram reported consulting and honoraria relationships with Apotex, Janssen, and Sanofi. Other study authors reported multiple relationships with industry. Disclosures for Dr. Dunbar and Dr. Sekeres were unavailable.
A version of this article appeared on Medscape.com.
The analysis, which included nearly 4,000 patients with multiple myeloma, revealed that patients in a real-world setting demonstrated worse progression-free and overall survival on six of seven standard treatments compared with patients evaluated in randomized controlled trials.
Lead author Alissa Visram, MD, MPH, who spoke about the study at the annual meeting of the American Society of Hematology, said the findings will likely change the way she speaks to patients about their potential outcomes.
“I’ll probably present both numbers [from real-life and clinical-trial data] and give them a sense of the best-case scenario,” Dr. Visram said during an ASH media briefing. But she said she will also caution her patients that the real-world numbers reflect how people on these drugs actually fare.
The effectiveness of multiple myeloma drugs remains unclear outside the clinical trial setting, explained Dr. Visram, of the Division of Hematology at the Ottawa Hospital Research Institute, Ottawa, Ontario, Canada. Outcomes from randomized controlled trials form the basis of drug approvals but many patients in the real world do not meet the “stringent” trial inclusion criteria.
Dr. Visram and colleagues launched the current study to better understand the potential differences between real-world and clinical trial outcomes. In the analysis, the researchers compared real-world outcomes among patients receiving seven standard multiple myeloma regimens covered by Ontario’s public health plan with patient outcomes reported in phase 3 randomized controlled trials.
The retrospective study included 3951 patients with newly diagnosed and refractory multiple myeloma treated from 2007 to 2020 in Ontario. Regimens for newly diagnosed transplant ineligible patients included lenalidomide plus dexamethasone and triple therapy with bortezomib, lenalidomide, and dexamethasone. Regimens for patients with relapsed disease included pomalidomide plus dexamethasone or carfilzomib plus dexamethasone as well as triple combinations including carfilzomib, lenalidomide, and dexamethasone.
Overall, Dr. Visram and colleagues found that patients in the real-world setting demonstrated worse overall survival for six of the seven regimens evaluated (pooled hazard ratio [HR], 1.75; P = .010).
The real-world patients also had worse progression-free survival for six of the seven regimens (pooled HR, 1.44; P = .034).
For these regimens, progression-free survival was at least 3-18 months longer in the clinical trial cohort, while median overall survival was at least 19 months longer compared with real-world patients, Dr. Visram explained.
The only regimen with comparable outcomes in the clinical trial and real-world settings was pomalidomide and dexamethasone, she said. One reason could be that patients receiving pomalidomide plus dexamethasone in the clinical trial setting had similar or more advanced disease than those in the real-world setting.
The study also found that adverse effects were similar between the clinical and real-world groups.
The next step, Dr. Visram said, would be to explore what’s driving the differences in outcomes.
Are patients in the real-world setting older or frailer? Do they have more advanced disease? Are providers using these regimens differently?
Mikkael A. Sekeres, MD, MS, explained that the difference likely comes down to the health of the patient.
Patients in these types of clinical trials “are just these pristine specimens of human beings except for the cancer that’s being treated,” Dr. Sekeres, of the Sylvester Comprehensive Cancer Center at the University of Miami, Florida, said in an earlier ASH press briefing.
Cynthia E. Dunbar, MD, noted that patients in clinical trials have other advantages as well.
“Patients who are able to enroll in clinical trials are more likely to be able to show up at the treatment center at the right time and for every dose, have transportation, and afford drugs to prevent side effects,” said Dr. Dunbar, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute and secretary of ASH. These patients also “might stay on the drug for longer, or they have nurses who are always encouraging them on how to make it through a toxicity.”
Dr. Dunbar said hematologists and patients should consider randomized controlled trials to be “the best possible outcome, and perhaps adjust their thinking if an individual patient is older, sicker, or less able to follow a regimen exactly.”
No study funding was reported. Dr. Visram reported consulting and honoraria relationships with Apotex, Janssen, and Sanofi. Other study authors reported multiple relationships with industry. Disclosures for Dr. Dunbar and Dr. Sekeres were unavailable.
A version of this article appeared on Medscape.com.
FROM ASH 2023
Which migraine medications are most effective?
TOPLINE:
new results from large, real-world analysis of self-reported patient data show.
METHODOLOGY:
- Researchers analyzed nearly 11 million migraine attack records extracted from Migraine Buddy, an e-diary smartphone app, over a 6-year period.
- They evaluated self-reported treatment effectiveness for 25 acute migraine medications among seven classes: acetaminophen, NSAIDs, triptans, combination analgesics, ergots, antiemetics, and opioids.
- A two-level nested multivariate logistic regression model adjusted for within-subject dependency and for concomitant medications taken within each analyzed migraine attack.
- The final analysis included nearly 5 million medication-outcome pairs from 3.1 million migraine attacks in 278,000 medication users.
TAKEAWAY:
- Using ibuprofen as the reference, triptans, ergots, and antiemetics were the top three medication classes with the highest effectiveness (mean odds ratios [OR] 4.80, 3.02, and 2.67, respectively).
- The next most effective medication classes were opioids (OR, 2.49), NSAIDs other than ibuprofen (OR, 1.94), combination analgesics acetaminophen/acetylsalicylic acid/caffeine (OR, 1.69), and others (OR, 1.49).
- Acetaminophen (OR, 0.83) was considered to be the least effective.
- The most effective individual medications were eletriptan (Relpax) (OR, 6.1); zolmitriptan (Zomig) (OR, 5.7); and sumatriptan (Imitrex) (OR, 5.2).
IN PRACTICE:
“Our findings that triptans, ergots, and antiemetics are the most effective classes of medications align with the guideline recommendations and offer generalizable insights to complement clinical practice,” the authors wrote.
SOURCE:
The study, with first author Chia-Chun Chiang, MD, Department of Neurology, Mayo Clinic, Rochester, Minnesota, was published online November 29 in Neurology.
LIMITATIONS:
The findings are based on subjective user-reported ratings of effectiveness and information on side effects, dosages, and formulations were not available. The newer migraine medication classes, gepants and ditans, were not included due to the relatively lower number of treated attacks. The regression model did not include age, gender, pain intensity, and other migraine-associated symptoms, which could potentially affect treatment effectiveness.
DISCLOSURES:
Funding for the study was provided by the Kanagawa University of Human Service research fund. A full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
TOPLINE:
new results from large, real-world analysis of self-reported patient data show.
METHODOLOGY:
- Researchers analyzed nearly 11 million migraine attack records extracted from Migraine Buddy, an e-diary smartphone app, over a 6-year period.
- They evaluated self-reported treatment effectiveness for 25 acute migraine medications among seven classes: acetaminophen, NSAIDs, triptans, combination analgesics, ergots, antiemetics, and opioids.
- A two-level nested multivariate logistic regression model adjusted for within-subject dependency and for concomitant medications taken within each analyzed migraine attack.
- The final analysis included nearly 5 million medication-outcome pairs from 3.1 million migraine attacks in 278,000 medication users.
TAKEAWAY:
- Using ibuprofen as the reference, triptans, ergots, and antiemetics were the top three medication classes with the highest effectiveness (mean odds ratios [OR] 4.80, 3.02, and 2.67, respectively).
- The next most effective medication classes were opioids (OR, 2.49), NSAIDs other than ibuprofen (OR, 1.94), combination analgesics acetaminophen/acetylsalicylic acid/caffeine (OR, 1.69), and others (OR, 1.49).
- Acetaminophen (OR, 0.83) was considered to be the least effective.
- The most effective individual medications were eletriptan (Relpax) (OR, 6.1); zolmitriptan (Zomig) (OR, 5.7); and sumatriptan (Imitrex) (OR, 5.2).
IN PRACTICE:
“Our findings that triptans, ergots, and antiemetics are the most effective classes of medications align with the guideline recommendations and offer generalizable insights to complement clinical practice,” the authors wrote.
SOURCE:
The study, with first author Chia-Chun Chiang, MD, Department of Neurology, Mayo Clinic, Rochester, Minnesota, was published online November 29 in Neurology.
LIMITATIONS:
The findings are based on subjective user-reported ratings of effectiveness and information on side effects, dosages, and formulations were not available. The newer migraine medication classes, gepants and ditans, were not included due to the relatively lower number of treated attacks. The regression model did not include age, gender, pain intensity, and other migraine-associated symptoms, which could potentially affect treatment effectiveness.
DISCLOSURES:
Funding for the study was provided by the Kanagawa University of Human Service research fund. A full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
TOPLINE:
new results from large, real-world analysis of self-reported patient data show.
METHODOLOGY:
- Researchers analyzed nearly 11 million migraine attack records extracted from Migraine Buddy, an e-diary smartphone app, over a 6-year period.
- They evaluated self-reported treatment effectiveness for 25 acute migraine medications among seven classes: acetaminophen, NSAIDs, triptans, combination analgesics, ergots, antiemetics, and opioids.
- A two-level nested multivariate logistic regression model adjusted for within-subject dependency and for concomitant medications taken within each analyzed migraine attack.
- The final analysis included nearly 5 million medication-outcome pairs from 3.1 million migraine attacks in 278,000 medication users.
TAKEAWAY:
- Using ibuprofen as the reference, triptans, ergots, and antiemetics were the top three medication classes with the highest effectiveness (mean odds ratios [OR] 4.80, 3.02, and 2.67, respectively).
- The next most effective medication classes were opioids (OR, 2.49), NSAIDs other than ibuprofen (OR, 1.94), combination analgesics acetaminophen/acetylsalicylic acid/caffeine (OR, 1.69), and others (OR, 1.49).
- Acetaminophen (OR, 0.83) was considered to be the least effective.
- The most effective individual medications were eletriptan (Relpax) (OR, 6.1); zolmitriptan (Zomig) (OR, 5.7); and sumatriptan (Imitrex) (OR, 5.2).
IN PRACTICE:
“Our findings that triptans, ergots, and antiemetics are the most effective classes of medications align with the guideline recommendations and offer generalizable insights to complement clinical practice,” the authors wrote.
SOURCE:
The study, with first author Chia-Chun Chiang, MD, Department of Neurology, Mayo Clinic, Rochester, Minnesota, was published online November 29 in Neurology.
LIMITATIONS:
The findings are based on subjective user-reported ratings of effectiveness and information on side effects, dosages, and formulations were not available. The newer migraine medication classes, gepants and ditans, were not included due to the relatively lower number of treated attacks. The regression model did not include age, gender, pain intensity, and other migraine-associated symptoms, which could potentially affect treatment effectiveness.
DISCLOSURES:
Funding for the study was provided by the Kanagawa University of Human Service research fund. A full list of author disclosures can be found with the original article.
A version of this article first appeared on Medscape.com.
Just gas? New study on colic suggests some longer-term implications
Pediatricians commonly are asked to see infants presenting with symptoms of colic. The frequent and intense crying associated with colic is understandably quite distressing to parents, who often worry about a serious underlying medical cause. There also is the stress of trying to soothe an irritable infant who often does not seem to respond to the typical interventions.
Conventional wisdom about colic has been that the behaviors are the result of some gastrointestinal problem that, while not perfectly understood, tends to be mercifully self-limited and not predictive of future medical or mental health problems. This perspective then leads to pediatricians typically offering mainly sympathy and reassurance at these visits.
A new study,1 however, challenges some of this traditional thinking. The data come from a remarkable longitudinal study called the Generation R Study (R being Rotterdam in the Netherlands) that has prospectively studied a group of nearly 5,000 children from before birth into adolescence. Colic symptoms were briefly assessed when infants were about 3 months old and emotional-behavioral problems have been prospectively measured at multiple time points subsequently using well-validated rating scales.
The main finding of the study was and, to a lesser extent, in adolescence. This held for both internalizing problems (like anxiety and depressive symptoms) and externalizing problems (like defiance and aggressive behavior). At age 10, participants also underwent an MRI scan and those who were excessive criers as infants were found to have a smaller amygdala, a region known for being important in regulating emotions.
The authors concluded that colicky behavior in infancy may reflect some underlying temperamental vulnerabilities and may have more predictive value than previously thought. The connection between excessive crying and a measurable brain region difference later in life is also interesting, although these kinds of brain imaging findings have been notoriously difficult to interpret clinically.
Overall, this is a solid study that deserves to be considered. Colic may reflect a bit more than most of us have been taught and shouldn’t necessarily be “shrugged off,” as the authors state in their discussion.
At the same time, however, it is important not to overinterpret the findings. The magnitude of the effects were on the small side (about 0.2 of a standard deviation) and most children with excessive crying in early infancy did not manifest high levels of mental health problems later in life. The mothers of high crying infants also had slightly higher levels of mental health problems themselves so there could be other mechanisms at work here, such as genetic differences between the two groups.
So how could a pediatrician best use this new information without taking things too far? Regardless of the question of whether the excessive crying infancy is a true risk factor for later behavior problems (in the causal sense) or whether it represents more of a marker for something else, its presence so early in life offers an opportunity. Primary care clinicians would still likely want to provide the reassurance that has typically been given in these visits but perhaps with the caveat that some of these kids go on to struggle a bit more with mental health and that they might benefit from some additional support. We are not talking about prophylactic medications here, but something like additional parenting skills. Especially if you, as the pediatrician, suspect that the parents might benefit from expanding their parenting toolkit already, here is a nice opportunity to invite them to learn some new approaches and skills — framed in a way that focuses on the temperament of the child rather than any “deficits” you perceive in the parents. Some parents may be more receptive and less defensive to the idea of participating in parent training under the framework that they are doing this because they have a temperamentally more challenging child (rather than feeling that they are deficient in basic parenting skills).
It’s always a good idea to know about what resources are available in the community when it comes to teaching parenting skills. In addition to scientifically supported books and podcasts, there has been a steady increase in reliable websites, apps, and other digital platforms related to parenting, as well as standard in-person groups and classes. This could also be a great use of an integrated behavioral health professional for practices fortunate enough to have one.
In summary, there is some new evidence that colic can represent a little more than “just gas,” and while we shouldn’t take this one study to the extreme, there may be some good opportunities here to discuss and support good parenting practices in general.
Dr. Rettew is a child and adolescent psychiatrist with Lane County Behavioral Health in Eugene, Ore., and Oregon Health & Science University, Portland. His latest book is “Parenting Made Complicated: What Science Really Knows about the Greatest Debates of Early Childhood.” You can follow him on Twitter and Facebook @PediPsych.
Reference
1. Sammallahti S et al. Excessive crying, behavior problems, and amygdala volume: A study from infancy to adolescence. J Am Acad Child Adolesc Psychiatry. 2023 Jun;62(6):675-83. doi: 10.1016/j.jaac.2023.01.014.
Pediatricians commonly are asked to see infants presenting with symptoms of colic. The frequent and intense crying associated with colic is understandably quite distressing to parents, who often worry about a serious underlying medical cause. There also is the stress of trying to soothe an irritable infant who often does not seem to respond to the typical interventions.
Conventional wisdom about colic has been that the behaviors are the result of some gastrointestinal problem that, while not perfectly understood, tends to be mercifully self-limited and not predictive of future medical or mental health problems. This perspective then leads to pediatricians typically offering mainly sympathy and reassurance at these visits.
A new study,1 however, challenges some of this traditional thinking. The data come from a remarkable longitudinal study called the Generation R Study (R being Rotterdam in the Netherlands) that has prospectively studied a group of nearly 5,000 children from before birth into adolescence. Colic symptoms were briefly assessed when infants were about 3 months old and emotional-behavioral problems have been prospectively measured at multiple time points subsequently using well-validated rating scales.
The main finding of the study was and, to a lesser extent, in adolescence. This held for both internalizing problems (like anxiety and depressive symptoms) and externalizing problems (like defiance and aggressive behavior). At age 10, participants also underwent an MRI scan and those who were excessive criers as infants were found to have a smaller amygdala, a region known for being important in regulating emotions.
The authors concluded that colicky behavior in infancy may reflect some underlying temperamental vulnerabilities and may have more predictive value than previously thought. The connection between excessive crying and a measurable brain region difference later in life is also interesting, although these kinds of brain imaging findings have been notoriously difficult to interpret clinically.
Overall, this is a solid study that deserves to be considered. Colic may reflect a bit more than most of us have been taught and shouldn’t necessarily be “shrugged off,” as the authors state in their discussion.
At the same time, however, it is important not to overinterpret the findings. The magnitude of the effects were on the small side (about 0.2 of a standard deviation) and most children with excessive crying in early infancy did not manifest high levels of mental health problems later in life. The mothers of high crying infants also had slightly higher levels of mental health problems themselves so there could be other mechanisms at work here, such as genetic differences between the two groups.
So how could a pediatrician best use this new information without taking things too far? Regardless of the question of whether the excessive crying infancy is a true risk factor for later behavior problems (in the causal sense) or whether it represents more of a marker for something else, its presence so early in life offers an opportunity. Primary care clinicians would still likely want to provide the reassurance that has typically been given in these visits but perhaps with the caveat that some of these kids go on to struggle a bit more with mental health and that they might benefit from some additional support. We are not talking about prophylactic medications here, but something like additional parenting skills. Especially if you, as the pediatrician, suspect that the parents might benefit from expanding their parenting toolkit already, here is a nice opportunity to invite them to learn some new approaches and skills — framed in a way that focuses on the temperament of the child rather than any “deficits” you perceive in the parents. Some parents may be more receptive and less defensive to the idea of participating in parent training under the framework that they are doing this because they have a temperamentally more challenging child (rather than feeling that they are deficient in basic parenting skills).
It’s always a good idea to know about what resources are available in the community when it comes to teaching parenting skills. In addition to scientifically supported books and podcasts, there has been a steady increase in reliable websites, apps, and other digital platforms related to parenting, as well as standard in-person groups and classes. This could also be a great use of an integrated behavioral health professional for practices fortunate enough to have one.
In summary, there is some new evidence that colic can represent a little more than “just gas,” and while we shouldn’t take this one study to the extreme, there may be some good opportunities here to discuss and support good parenting practices in general.
Dr. Rettew is a child and adolescent psychiatrist with Lane County Behavioral Health in Eugene, Ore., and Oregon Health & Science University, Portland. His latest book is “Parenting Made Complicated: What Science Really Knows about the Greatest Debates of Early Childhood.” You can follow him on Twitter and Facebook @PediPsych.
Reference
1. Sammallahti S et al. Excessive crying, behavior problems, and amygdala volume: A study from infancy to adolescence. J Am Acad Child Adolesc Psychiatry. 2023 Jun;62(6):675-83. doi: 10.1016/j.jaac.2023.01.014.
Pediatricians commonly are asked to see infants presenting with symptoms of colic. The frequent and intense crying associated with colic is understandably quite distressing to parents, who often worry about a serious underlying medical cause. There also is the stress of trying to soothe an irritable infant who often does not seem to respond to the typical interventions.
Conventional wisdom about colic has been that the behaviors are the result of some gastrointestinal problem that, while not perfectly understood, tends to be mercifully self-limited and not predictive of future medical or mental health problems. This perspective then leads to pediatricians typically offering mainly sympathy and reassurance at these visits.
A new study,1 however, challenges some of this traditional thinking. The data come from a remarkable longitudinal study called the Generation R Study (R being Rotterdam in the Netherlands) that has prospectively studied a group of nearly 5,000 children from before birth into adolescence. Colic symptoms were briefly assessed when infants were about 3 months old and emotional-behavioral problems have been prospectively measured at multiple time points subsequently using well-validated rating scales.
The main finding of the study was and, to a lesser extent, in adolescence. This held for both internalizing problems (like anxiety and depressive symptoms) and externalizing problems (like defiance and aggressive behavior). At age 10, participants also underwent an MRI scan and those who were excessive criers as infants were found to have a smaller amygdala, a region known for being important in regulating emotions.
The authors concluded that colicky behavior in infancy may reflect some underlying temperamental vulnerabilities and may have more predictive value than previously thought. The connection between excessive crying and a measurable brain region difference later in life is also interesting, although these kinds of brain imaging findings have been notoriously difficult to interpret clinically.
Overall, this is a solid study that deserves to be considered. Colic may reflect a bit more than most of us have been taught and shouldn’t necessarily be “shrugged off,” as the authors state in their discussion.
At the same time, however, it is important not to overinterpret the findings. The magnitude of the effects were on the small side (about 0.2 of a standard deviation) and most children with excessive crying in early infancy did not manifest high levels of mental health problems later in life. The mothers of high crying infants also had slightly higher levels of mental health problems themselves so there could be other mechanisms at work here, such as genetic differences between the two groups.
So how could a pediatrician best use this new information without taking things too far? Regardless of the question of whether the excessive crying infancy is a true risk factor for later behavior problems (in the causal sense) or whether it represents more of a marker for something else, its presence so early in life offers an opportunity. Primary care clinicians would still likely want to provide the reassurance that has typically been given in these visits but perhaps with the caveat that some of these kids go on to struggle a bit more with mental health and that they might benefit from some additional support. We are not talking about prophylactic medications here, but something like additional parenting skills. Especially if you, as the pediatrician, suspect that the parents might benefit from expanding their parenting toolkit already, here is a nice opportunity to invite them to learn some new approaches and skills — framed in a way that focuses on the temperament of the child rather than any “deficits” you perceive in the parents. Some parents may be more receptive and less defensive to the idea of participating in parent training under the framework that they are doing this because they have a temperamentally more challenging child (rather than feeling that they are deficient in basic parenting skills).
It’s always a good idea to know about what resources are available in the community when it comes to teaching parenting skills. In addition to scientifically supported books and podcasts, there has been a steady increase in reliable websites, apps, and other digital platforms related to parenting, as well as standard in-person groups and classes. This could also be a great use of an integrated behavioral health professional for practices fortunate enough to have one.
In summary, there is some new evidence that colic can represent a little more than “just gas,” and while we shouldn’t take this one study to the extreme, there may be some good opportunities here to discuss and support good parenting practices in general.
Dr. Rettew is a child and adolescent psychiatrist with Lane County Behavioral Health in Eugene, Ore., and Oregon Health & Science University, Portland. His latest book is “Parenting Made Complicated: What Science Really Knows about the Greatest Debates of Early Childhood.” You can follow him on Twitter and Facebook @PediPsych.
Reference
1. Sammallahti S et al. Excessive crying, behavior problems, and amygdala volume: A study from infancy to adolescence. J Am Acad Child Adolesc Psychiatry. 2023 Jun;62(6):675-83. doi: 10.1016/j.jaac.2023.01.014.
New cardiology certification board: What’s the plan?
The proposal by the major cardiovascular societies in the US to form a new board of cardiovascular medicine to manage initial and ongoing certification of cardiologists represents something of a revolution in the field of continuing medical education and assessment of competency.
Five US cardiovascular societies — the American College of Cardiology (ACC), the American Heart Association (AHA), the Heart Failure Society of America (HFSA), the Heart Rhythm Society (HRS), and the Society for Cardiovascular Angiography & Interventions (SCAI) — have now joined forces to propose a new professional certification board for cardiovascular medicine, to be known as the American Board of Cardiovascular Medicine (ABCVM).
The ABCVM would be independent of the American Board of Internal Medicine (ABIM), the current organization providing maintenance of certification for cardiologists as well as many other internal medicine subspecialties. The ABIM’s maintenance of certification process has been widely criticized for many years and has been described as “needlessly burdensome and expensive.”
, according to Jeffrey Kuvin, MD, a trustee of the ACC, who has been heading up the working group to develop this plan.
Dr. Kuvin, who is chair of the cardiology at Northwell Health, Manhasset, New York, a large academic healthcare system, explained that maintenance of certification has been a topic of discussion across the cardiovascular community for many years, and the ACC has a working group focused on the next steps for evaluation of competency, which he chairs.
“The topic of evaluation of competence has been on the mind of the ACC for many years and hence a work group was developed to focus on this,” Dr. Kuvin noted. “A lot of evolution of the concepts and next steps have been drawn out of this working group. And now other cardiovascular societies have joined to show unification across the house of cardiology and that this is indeed the way that the cardiovascular profession should move.”
“Time to Separate from Internal Medicine”
The general concept behind the new cardiology board is to separate cardiology from the ABIM.
“This is rooted from the concept that cardiology has evolved so much over the last few decades into such a large multidimensional specialty that it really does demarcate itself from internal medicine, and as such, it deserves a separate board governed by cardiologists with collaboration across the entirely of cardiology,” Dr. Kuvin said.
Cardiology has had significant growth and expansion of technology, tools, medications, and the approach to patients in many specialities and subspecialties, he added. “We have defined training programs in many different areas within cardiology; we have our own guidelines, our own competency statements, and in many cases, cardiology exists as its own department outside of medicine in many institutions. It’s just time to separate cardiology from the umbrella of internal medicine.”
The new cardiology board would be separate from, and not report to, the ABIM; rather, it would report directly to the American Board of Medical Specialties (ABMS), the only recognized medical certification body in the US.
What Are the Proposed Changes
Under the present system, managed by the ABIM, clinicians must undergo two stages of certification to be a cardiologist. First, they have to pass the initial certification exam in general cardiology, and then exams in one of four subspecialties if they plan to enter one of these, including interventional cardiology, electrophysiology, advanced heart failure or adult congenital heart disease.
Next, clinicians enter the maintenance of certification phase, which can take three different forms: 1) taking another recertification exam every 10 years; 2) the collaborative maintenance pathway — a collaboration between ACC and ABIM, which includes evaluation, learning and a certified exam each year; or 3) longitudinal knowledge and assessment — in which the program interacts with the clinician on an ongoing basis, sending secured questions regularly.
All three of these pathways for maintenance of certification involve high stakes questions and a set bar for passing or failing.
Under the proposed new cardiology board, an initial certification exam would still be required after fellowship training, but the maintenance of certification process would be completely restructured, with the new approach taking the form of continuous learning and assessment of competency.
“This is an iterative process, but we envision with a new American Board of Cardiovascular Medicine, we will pick up where the ABIM left off,” Dr. Kuvin notes. “That includes an initial certifying examination for the five areas that already exist under the ABIM system but with the opportunities to expand that to further specialties as well.”
He points out that there are several areas in cardiology that are currently not represented by these five areas that warrant some discussion, including multimodality imaging, vascular heart disease, and cardio-oncology.
“At present, everybody has to pass the general cardiology exam and then some may wish to further train and get certified in one of the other four other specific areas. But one topic that has been discussed over many years is how do we maintain competency in the areas in which clinicians practice over their lifetime as a cardiologist,” Dr. Kuvin commented.
He said the proposed cardiology board would like to adhere to some basic principles that are fundamental to the practice of medicine.
“We want to make sure that we are practicing medicine so that our patients derive the most benefit from seeing a cardiologist,” he said. “We also want to make sure, however, that this is a supportive process, supporting cardiologists to learn what they know and more importantly what they don’t know; to identify knowledge gaps in specific area; to help the cardiologist fill those knowledge gaps; to acknowledge those gaps have been filled; and then move on to another area of interest. This will be the focus of this new and improved model of continuous competency.”
The proposed new board also says it wants to make sure this is appropriate to the area in which the clinician is practicing.
“To take a closed book certified exam every 10 years on the world of cardiology as happens at the current time – or the assessments conducted in the other two pathways – is often meaningless to the cardiologist,” Dr. Kuvin says. “All three current pathways involve high stakes questions that are often irrelevant to one’s clinical practice.”
Lifelong Learning
“The crux of the changes we are proposing will be away from the focus of passing a test towards a model of helping the individual with their competency, with continuous learning and evaluation of competency to help the clinician fill in their knowledge gaps,” he explains.
He described the new approach as “lifelong learning,” adding that, instead of it being “a punitive pass/fail environment with no feedback, which causes a lot of discontent among clinicians,” it will be a supportive process, where a clinician will be helped in filling their knowledge gaps.
“I think this would be a welcome change not just for cardiology but across medical specialties,” Dr. Kuvin said.
He also pointed out the ABMS itself is considering a continuous competency approach, and the proposed new cardiology board aims to work with the ABMS to make sure that their goals of continuous competency assessment are matched.
“The world has changed. The ability to access information has changed. It is no longer imperative for a clinician to have every piece of knowledge in their brain, but rather to know how to get knowledge and to incorporate that knowledge into clinical practice,” Dr. Kuvin noted. “Competency should not involve knowledge alone as in a closed book exam. It is more about understanding the world that we live in, how to synthesize information, where we need to improve knowledge and how to do that.”
Dr. Kuvin acknowledged that asking clinicians questions is a very helpful tool to identify their knowledge base and their knowledge gaps. “But we believe the clinician needs to be given resources — that could be a conference, an article, a simulation — to fill that knowledge gap. Then we could ask clinicians some different questions and if they get those right then we have provided a service.”
Tactile skills for cardiologists needing to perform procedures – such as interventionalists or electrophysiologists may be incorporated by simulation in a technology-based scenario.
On how often these assessments would take place, Dr. Kuvin said that hadn’t been decided for sure.
“We certainly do not think an assessment every 10 years is appropriate. We envision, instead of an episodic model, it will be rather a lifelong journey of education and competency. This will involve frequent contact and making sure knowledge gaps are being filled. There are criteria being set out by the ABMS that there should be a certain number of touch points with individuals on an annual as well as a 5-year basis to make sure cardiologists are staying within specific guardrails. The exact nature of these is yet to be determined,” he said.
Dr. Kuvin added that it was not known yet what sort of hours would be required but added that “this will not be a significant time burden.”
What is the Timeframe?
The application to the ABMS for a separate cardiology board is still ongoing and has not yet received formal acceptance. Representatives from the five US cardiovascular societies are in the initial stages of formulating a transition board.
“The submission to the ABMS will take time for them to review. This could take up to a year or so,” Dr. Kuvin estimates.
This is the first time the ABMS has entertained the concept of a new board in many years, he noted. “It will be a paradigm shift for the whole country. I think that cardiology is really at the forefront and in a position where we can actually do this. If cardiovascular medicine is granted a new board, I think this will help change the approach of how physicians are assessed in terms of continuous competency not just in cardiology but across all specialties of medicine.”
He added: “We are confident that we can work within the construct of the ABMS guidelines that have been revised to be much more holistic in the approach of continuous competence across the board. This includes thinking beyond rote medical knowledge and thinking about the clinician as a whole and their abilities to communicate, act professionally, work within a complex medical system, utilize medical resources effectively. These all have to be part of continuous competence.”
How Much Will This Cost?
Noting that the ABIM has received criticism over the costs of the certification process, Dr. Kuvin said they intend to make this “as lean a machine as possible with the focus on reducing the financial [burden] as well as the time burden for cardiologists. It is very important that this is not cumbersome, that it is woven into clinical practice, and that it is not costly.”
But he pointed out that building a new board will have significant costs.
“We have to think about developing initial board certification examinations as well as changing the paradigm on continuous certification,” he said. “This will take some up-front costs, and our society partners have decided that they are willing to provide some start-up funds for this. We anticipate the initial certification will remain somewhat similar in price, but the cost of ongoing continuous competency assessment will be significantly reduced compared to today’s models.”
Dr. Kuvin said the collaboration of the five participating US cardiovascular societies was unprecedented. But he noted that while the transition board is beginning with representatives of these individual societies, it will ultimately be independent from these societies and have its own board of directors.
He suggested that other societies representing other parts of cardiology are also interested. “Cardiology has recognized how important this is,” he said. “Everybody is excited about this.”
A version of this article appeared on Medscape.com.
The proposal by the major cardiovascular societies in the US to form a new board of cardiovascular medicine to manage initial and ongoing certification of cardiologists represents something of a revolution in the field of continuing medical education and assessment of competency.
Five US cardiovascular societies — the American College of Cardiology (ACC), the American Heart Association (AHA), the Heart Failure Society of America (HFSA), the Heart Rhythm Society (HRS), and the Society for Cardiovascular Angiography & Interventions (SCAI) — have now joined forces to propose a new professional certification board for cardiovascular medicine, to be known as the American Board of Cardiovascular Medicine (ABCVM).
The ABCVM would be independent of the American Board of Internal Medicine (ABIM), the current organization providing maintenance of certification for cardiologists as well as many other internal medicine subspecialties. The ABIM’s maintenance of certification process has been widely criticized for many years and has been described as “needlessly burdensome and expensive.”
, according to Jeffrey Kuvin, MD, a trustee of the ACC, who has been heading up the working group to develop this plan.
Dr. Kuvin, who is chair of the cardiology at Northwell Health, Manhasset, New York, a large academic healthcare system, explained that maintenance of certification has been a topic of discussion across the cardiovascular community for many years, and the ACC has a working group focused on the next steps for evaluation of competency, which he chairs.
“The topic of evaluation of competence has been on the mind of the ACC for many years and hence a work group was developed to focus on this,” Dr. Kuvin noted. “A lot of evolution of the concepts and next steps have been drawn out of this working group. And now other cardiovascular societies have joined to show unification across the house of cardiology and that this is indeed the way that the cardiovascular profession should move.”
“Time to Separate from Internal Medicine”
The general concept behind the new cardiology board is to separate cardiology from the ABIM.
“This is rooted from the concept that cardiology has evolved so much over the last few decades into such a large multidimensional specialty that it really does demarcate itself from internal medicine, and as such, it deserves a separate board governed by cardiologists with collaboration across the entirely of cardiology,” Dr. Kuvin said.
Cardiology has had significant growth and expansion of technology, tools, medications, and the approach to patients in many specialities and subspecialties, he added. “We have defined training programs in many different areas within cardiology; we have our own guidelines, our own competency statements, and in many cases, cardiology exists as its own department outside of medicine in many institutions. It’s just time to separate cardiology from the umbrella of internal medicine.”
The new cardiology board would be separate from, and not report to, the ABIM; rather, it would report directly to the American Board of Medical Specialties (ABMS), the only recognized medical certification body in the US.
What Are the Proposed Changes
Under the present system, managed by the ABIM, clinicians must undergo two stages of certification to be a cardiologist. First, they have to pass the initial certification exam in general cardiology, and then exams in one of four subspecialties if they plan to enter one of these, including interventional cardiology, electrophysiology, advanced heart failure or adult congenital heart disease.
Next, clinicians enter the maintenance of certification phase, which can take three different forms: 1) taking another recertification exam every 10 years; 2) the collaborative maintenance pathway — a collaboration between ACC and ABIM, which includes evaluation, learning and a certified exam each year; or 3) longitudinal knowledge and assessment — in which the program interacts with the clinician on an ongoing basis, sending secured questions regularly.
All three of these pathways for maintenance of certification involve high stakes questions and a set bar for passing or failing.
Under the proposed new cardiology board, an initial certification exam would still be required after fellowship training, but the maintenance of certification process would be completely restructured, with the new approach taking the form of continuous learning and assessment of competency.
“This is an iterative process, but we envision with a new American Board of Cardiovascular Medicine, we will pick up where the ABIM left off,” Dr. Kuvin notes. “That includes an initial certifying examination for the five areas that already exist under the ABIM system but with the opportunities to expand that to further specialties as well.”
He points out that there are several areas in cardiology that are currently not represented by these five areas that warrant some discussion, including multimodality imaging, vascular heart disease, and cardio-oncology.
“At present, everybody has to pass the general cardiology exam and then some may wish to further train and get certified in one of the other four other specific areas. But one topic that has been discussed over many years is how do we maintain competency in the areas in which clinicians practice over their lifetime as a cardiologist,” Dr. Kuvin commented.
He said the proposed cardiology board would like to adhere to some basic principles that are fundamental to the practice of medicine.
“We want to make sure that we are practicing medicine so that our patients derive the most benefit from seeing a cardiologist,” he said. “We also want to make sure, however, that this is a supportive process, supporting cardiologists to learn what they know and more importantly what they don’t know; to identify knowledge gaps in specific area; to help the cardiologist fill those knowledge gaps; to acknowledge those gaps have been filled; and then move on to another area of interest. This will be the focus of this new and improved model of continuous competency.”
The proposed new board also says it wants to make sure this is appropriate to the area in which the clinician is practicing.
“To take a closed book certified exam every 10 years on the world of cardiology as happens at the current time – or the assessments conducted in the other two pathways – is often meaningless to the cardiologist,” Dr. Kuvin says. “All three current pathways involve high stakes questions that are often irrelevant to one’s clinical practice.”
Lifelong Learning
“The crux of the changes we are proposing will be away from the focus of passing a test towards a model of helping the individual with their competency, with continuous learning and evaluation of competency to help the clinician fill in their knowledge gaps,” he explains.
He described the new approach as “lifelong learning,” adding that, instead of it being “a punitive pass/fail environment with no feedback, which causes a lot of discontent among clinicians,” it will be a supportive process, where a clinician will be helped in filling their knowledge gaps.
“I think this would be a welcome change not just for cardiology but across medical specialties,” Dr. Kuvin said.
He also pointed out the ABMS itself is considering a continuous competency approach, and the proposed new cardiology board aims to work with the ABMS to make sure that their goals of continuous competency assessment are matched.
“The world has changed. The ability to access information has changed. It is no longer imperative for a clinician to have every piece of knowledge in their brain, but rather to know how to get knowledge and to incorporate that knowledge into clinical practice,” Dr. Kuvin noted. “Competency should not involve knowledge alone as in a closed book exam. It is more about understanding the world that we live in, how to synthesize information, where we need to improve knowledge and how to do that.”
Dr. Kuvin acknowledged that asking clinicians questions is a very helpful tool to identify their knowledge base and their knowledge gaps. “But we believe the clinician needs to be given resources — that could be a conference, an article, a simulation — to fill that knowledge gap. Then we could ask clinicians some different questions and if they get those right then we have provided a service.”
Tactile skills for cardiologists needing to perform procedures – such as interventionalists or electrophysiologists may be incorporated by simulation in a technology-based scenario.
On how often these assessments would take place, Dr. Kuvin said that hadn’t been decided for sure.
“We certainly do not think an assessment every 10 years is appropriate. We envision, instead of an episodic model, it will be rather a lifelong journey of education and competency. This will involve frequent contact and making sure knowledge gaps are being filled. There are criteria being set out by the ABMS that there should be a certain number of touch points with individuals on an annual as well as a 5-year basis to make sure cardiologists are staying within specific guardrails. The exact nature of these is yet to be determined,” he said.
Dr. Kuvin added that it was not known yet what sort of hours would be required but added that “this will not be a significant time burden.”
What is the Timeframe?
The application to the ABMS for a separate cardiology board is still ongoing and has not yet received formal acceptance. Representatives from the five US cardiovascular societies are in the initial stages of formulating a transition board.
“The submission to the ABMS will take time for them to review. This could take up to a year or so,” Dr. Kuvin estimates.
This is the first time the ABMS has entertained the concept of a new board in many years, he noted. “It will be a paradigm shift for the whole country. I think that cardiology is really at the forefront and in a position where we can actually do this. If cardiovascular medicine is granted a new board, I think this will help change the approach of how physicians are assessed in terms of continuous competency not just in cardiology but across all specialties of medicine.”
He added: “We are confident that we can work within the construct of the ABMS guidelines that have been revised to be much more holistic in the approach of continuous competence across the board. This includes thinking beyond rote medical knowledge and thinking about the clinician as a whole and their abilities to communicate, act professionally, work within a complex medical system, utilize medical resources effectively. These all have to be part of continuous competence.”
How Much Will This Cost?
Noting that the ABIM has received criticism over the costs of the certification process, Dr. Kuvin said they intend to make this “as lean a machine as possible with the focus on reducing the financial [burden] as well as the time burden for cardiologists. It is very important that this is not cumbersome, that it is woven into clinical practice, and that it is not costly.”
But he pointed out that building a new board will have significant costs.
“We have to think about developing initial board certification examinations as well as changing the paradigm on continuous certification,” he said. “This will take some up-front costs, and our society partners have decided that they are willing to provide some start-up funds for this. We anticipate the initial certification will remain somewhat similar in price, but the cost of ongoing continuous competency assessment will be significantly reduced compared to today’s models.”
Dr. Kuvin said the collaboration of the five participating US cardiovascular societies was unprecedented. But he noted that while the transition board is beginning with representatives of these individual societies, it will ultimately be independent from these societies and have its own board of directors.
He suggested that other societies representing other parts of cardiology are also interested. “Cardiology has recognized how important this is,” he said. “Everybody is excited about this.”
A version of this article appeared on Medscape.com.
The proposal by the major cardiovascular societies in the US to form a new board of cardiovascular medicine to manage initial and ongoing certification of cardiologists represents something of a revolution in the field of continuing medical education and assessment of competency.
Five US cardiovascular societies — the American College of Cardiology (ACC), the American Heart Association (AHA), the Heart Failure Society of America (HFSA), the Heart Rhythm Society (HRS), and the Society for Cardiovascular Angiography & Interventions (SCAI) — have now joined forces to propose a new professional certification board for cardiovascular medicine, to be known as the American Board of Cardiovascular Medicine (ABCVM).
The ABCVM would be independent of the American Board of Internal Medicine (ABIM), the current organization providing maintenance of certification for cardiologists as well as many other internal medicine subspecialties. The ABIM’s maintenance of certification process has been widely criticized for many years and has been described as “needlessly burdensome and expensive.”
, according to Jeffrey Kuvin, MD, a trustee of the ACC, who has been heading up the working group to develop this plan.
Dr. Kuvin, who is chair of the cardiology at Northwell Health, Manhasset, New York, a large academic healthcare system, explained that maintenance of certification has been a topic of discussion across the cardiovascular community for many years, and the ACC has a working group focused on the next steps for evaluation of competency, which he chairs.
“The topic of evaluation of competence has been on the mind of the ACC for many years and hence a work group was developed to focus on this,” Dr. Kuvin noted. “A lot of evolution of the concepts and next steps have been drawn out of this working group. And now other cardiovascular societies have joined to show unification across the house of cardiology and that this is indeed the way that the cardiovascular profession should move.”
“Time to Separate from Internal Medicine”
The general concept behind the new cardiology board is to separate cardiology from the ABIM.
“This is rooted from the concept that cardiology has evolved so much over the last few decades into such a large multidimensional specialty that it really does demarcate itself from internal medicine, and as such, it deserves a separate board governed by cardiologists with collaboration across the entirely of cardiology,” Dr. Kuvin said.
Cardiology has had significant growth and expansion of technology, tools, medications, and the approach to patients in many specialities and subspecialties, he added. “We have defined training programs in many different areas within cardiology; we have our own guidelines, our own competency statements, and in many cases, cardiology exists as its own department outside of medicine in many institutions. It’s just time to separate cardiology from the umbrella of internal medicine.”
The new cardiology board would be separate from, and not report to, the ABIM; rather, it would report directly to the American Board of Medical Specialties (ABMS), the only recognized medical certification body in the US.
What Are the Proposed Changes
Under the present system, managed by the ABIM, clinicians must undergo two stages of certification to be a cardiologist. First, they have to pass the initial certification exam in general cardiology, and then exams in one of four subspecialties if they plan to enter one of these, including interventional cardiology, electrophysiology, advanced heart failure or adult congenital heart disease.
Next, clinicians enter the maintenance of certification phase, which can take three different forms: 1) taking another recertification exam every 10 years; 2) the collaborative maintenance pathway — a collaboration between ACC and ABIM, which includes evaluation, learning and a certified exam each year; or 3) longitudinal knowledge and assessment — in which the program interacts with the clinician on an ongoing basis, sending secured questions regularly.
All three of these pathways for maintenance of certification involve high stakes questions and a set bar for passing or failing.
Under the proposed new cardiology board, an initial certification exam would still be required after fellowship training, but the maintenance of certification process would be completely restructured, with the new approach taking the form of continuous learning and assessment of competency.
“This is an iterative process, but we envision with a new American Board of Cardiovascular Medicine, we will pick up where the ABIM left off,” Dr. Kuvin notes. “That includes an initial certifying examination for the five areas that already exist under the ABIM system but with the opportunities to expand that to further specialties as well.”
He points out that there are several areas in cardiology that are currently not represented by these five areas that warrant some discussion, including multimodality imaging, vascular heart disease, and cardio-oncology.
“At present, everybody has to pass the general cardiology exam and then some may wish to further train and get certified in one of the other four other specific areas. But one topic that has been discussed over many years is how do we maintain competency in the areas in which clinicians practice over their lifetime as a cardiologist,” Dr. Kuvin commented.
He said the proposed cardiology board would like to adhere to some basic principles that are fundamental to the practice of medicine.
“We want to make sure that we are practicing medicine so that our patients derive the most benefit from seeing a cardiologist,” he said. “We also want to make sure, however, that this is a supportive process, supporting cardiologists to learn what they know and more importantly what they don’t know; to identify knowledge gaps in specific area; to help the cardiologist fill those knowledge gaps; to acknowledge those gaps have been filled; and then move on to another area of interest. This will be the focus of this new and improved model of continuous competency.”
The proposed new board also says it wants to make sure this is appropriate to the area in which the clinician is practicing.
“To take a closed book certified exam every 10 years on the world of cardiology as happens at the current time – or the assessments conducted in the other two pathways – is often meaningless to the cardiologist,” Dr. Kuvin says. “All three current pathways involve high stakes questions that are often irrelevant to one’s clinical practice.”
Lifelong Learning
“The crux of the changes we are proposing will be away from the focus of passing a test towards a model of helping the individual with their competency, with continuous learning and evaluation of competency to help the clinician fill in their knowledge gaps,” he explains.
He described the new approach as “lifelong learning,” adding that, instead of it being “a punitive pass/fail environment with no feedback, which causes a lot of discontent among clinicians,” it will be a supportive process, where a clinician will be helped in filling their knowledge gaps.
“I think this would be a welcome change not just for cardiology but across medical specialties,” Dr. Kuvin said.
He also pointed out the ABMS itself is considering a continuous competency approach, and the proposed new cardiology board aims to work with the ABMS to make sure that their goals of continuous competency assessment are matched.
“The world has changed. The ability to access information has changed. It is no longer imperative for a clinician to have every piece of knowledge in their brain, but rather to know how to get knowledge and to incorporate that knowledge into clinical practice,” Dr. Kuvin noted. “Competency should not involve knowledge alone as in a closed book exam. It is more about understanding the world that we live in, how to synthesize information, where we need to improve knowledge and how to do that.”
Dr. Kuvin acknowledged that asking clinicians questions is a very helpful tool to identify their knowledge base and their knowledge gaps. “But we believe the clinician needs to be given resources — that could be a conference, an article, a simulation — to fill that knowledge gap. Then we could ask clinicians some different questions and if they get those right then we have provided a service.”
Tactile skills for cardiologists needing to perform procedures – such as interventionalists or electrophysiologists may be incorporated by simulation in a technology-based scenario.
On how often these assessments would take place, Dr. Kuvin said that hadn’t been decided for sure.
“We certainly do not think an assessment every 10 years is appropriate. We envision, instead of an episodic model, it will be rather a lifelong journey of education and competency. This will involve frequent contact and making sure knowledge gaps are being filled. There are criteria being set out by the ABMS that there should be a certain number of touch points with individuals on an annual as well as a 5-year basis to make sure cardiologists are staying within specific guardrails. The exact nature of these is yet to be determined,” he said.
Dr. Kuvin added that it was not known yet what sort of hours would be required but added that “this will not be a significant time burden.”
What is the Timeframe?
The application to the ABMS for a separate cardiology board is still ongoing and has not yet received formal acceptance. Representatives from the five US cardiovascular societies are in the initial stages of formulating a transition board.
“The submission to the ABMS will take time for them to review. This could take up to a year or so,” Dr. Kuvin estimates.
This is the first time the ABMS has entertained the concept of a new board in many years, he noted. “It will be a paradigm shift for the whole country. I think that cardiology is really at the forefront and in a position where we can actually do this. If cardiovascular medicine is granted a new board, I think this will help change the approach of how physicians are assessed in terms of continuous competency not just in cardiology but across all specialties of medicine.”
He added: “We are confident that we can work within the construct of the ABMS guidelines that have been revised to be much more holistic in the approach of continuous competence across the board. This includes thinking beyond rote medical knowledge and thinking about the clinician as a whole and their abilities to communicate, act professionally, work within a complex medical system, utilize medical resources effectively. These all have to be part of continuous competence.”
How Much Will This Cost?
Noting that the ABIM has received criticism over the costs of the certification process, Dr. Kuvin said they intend to make this “as lean a machine as possible with the focus on reducing the financial [burden] as well as the time burden for cardiologists. It is very important that this is not cumbersome, that it is woven into clinical practice, and that it is not costly.”
But he pointed out that building a new board will have significant costs.
“We have to think about developing initial board certification examinations as well as changing the paradigm on continuous certification,” he said. “This will take some up-front costs, and our society partners have decided that they are willing to provide some start-up funds for this. We anticipate the initial certification will remain somewhat similar in price, but the cost of ongoing continuous competency assessment will be significantly reduced compared to today’s models.”
Dr. Kuvin said the collaboration of the five participating US cardiovascular societies was unprecedented. But he noted that while the transition board is beginning with representatives of these individual societies, it will ultimately be independent from these societies and have its own board of directors.
He suggested that other societies representing other parts of cardiology are also interested. “Cardiology has recognized how important this is,” he said. “Everybody is excited about this.”
A version of this article appeared on Medscape.com.
Some reasons to get off the fence about COVID booster
Though many people remain on the fence about getting the latest COVID vaccine booster, new research suggests a strong argument for getting the shot this winter: It sharply reduces the risk for COVID.
The risk reduction was 37% for those who received two doses. Experts say the research provides a strong argument for getting the vaccine, noting that about 10% of people infected with COVID go on to have long COVID, which can be debilitating for one quarter of those with long-lasting symptoms.
The data come from a systematic literature review and meta-analysis published in October in Antimicrobial Stewardship & Epidemiology. Researchers examined 32 studies published between December 2019 and June 2023, involving 775,931 adults. Twenty-four studies, encompassing 620,221 individuals, were included in the meta-analysis.
“The body of evidence from all these different studies converge on one single reality — that vaccines reduce the risk of long COVID, and people who keep up to date on their vaccinations also fared better than people who got it once or twice and didn’t follow up,” said Ziyad Al-Aly, MD, a clinical epidemiologist at Washington University in St Louis.
Researchers have reported similar results for children. The National Institutes of Health RECOVER Initiative team found that vaccines are up to 42% effective in preventing long COVID in children, said Dr. Carlos Oliveira, MD, a pediatric infectious diseases specialist and Yale researcher who contributed to the study, which is in preprint.
Vaccines also protect children from multisystem inflammatory syndrome, a condition that can happen after COVID, as well as protect against other COVID-related problems, such as missed school days, Oliveira said. “Even if the vaccine doesn’t completely stop long COVID, it’s still good for kids to get vaccinated for all these other reasons.”
However, uptake for the latest boosters has been slow: the Centers for Disease Control and Prevention reported that by mid-November, less than 16% of people aged 18 years or older had received a shot. For children, the number was closer to 6%. A recent Kaiser Family Foundation survey found that booster rates for adults are similar to what it was 1 year ago.
The survey results suggest that people are no longer as worried about COVID, which is why there is less concerned about keeping up with boosters. Though the current mutation of the virus is not as debilitating as its predecessors, long COVID continues to be a problem: as of January 2023, 28% of people who had contracted the virus had experienced long-COVID symptoms. And though the mechanisms are still not fully understood, and researchers have yet to agree on a definition of long COVID, they are certain about this much: The best way to avoid it is to avoid getting infected to begin with.
The lack of a diagnostic test for long COVID and the fact that the symptoms mimic those of other diseases lead to inconsistency that can make studies hard to replicate. In the papers reviewed for the Antimicrobial Stewardship & Epidemiology study, long COVID was defined as having symptoms lasting from more than 4 weeks to more than 6 months. Alexandre Marra, MD, the lead author and a researcher at the Hospital Israelita Albert Einstein, in São Paulo, Brazil, and at the University of Iowa, said that a clear standard definition is needed to better understand the actual prevalence and evaluate vaccine effectiveness.
Al-Aly noted that there is a logical explanation for one finding in the paper: The percentage of individuals who had COVID and reported that long-COVID symptoms declined from 19% in June 2022 to 11% in January 2023.
Because a pandemic is a dynamic event, constantly producing different variants with different phenotypes, the prevalence of disease is naturally going to be affected. “People who got infected early in the pandemic may have a different long COVID profile and long COVID risk than people who got infected in the second or third year of the pandemic,” Al-Aly said.
Most of the studies reported data from before the Omicron-variant era. Only eight reported data during that era. Omicron was not as lethal as previous variants, and consequently, fewer patients developed long COVID during that time.
One of those who did is Yeng Chang, age 40 years, a family doctor who lives in Sherwood Park, Alberta, Canada. Chang developed long COVID during fall 2022 after getting the virus in June. By then, she’d been vaccinated three times, but she isn’t surprised that she got sick because each vaccine she had was developed before Omicron.
“When I had COVID I was really sick, but I was well enough to stay home,” she said. “I think if I didn’t have my immunizations, I might have been hospitalized, and I don’t know what would have happened.”
Long COVID has left Chang with brain fog, fatigue, and a lack of physical stamina that forced her to pause her medical practice. For the past year and a half, she’s spent more time as a patient than a physician.
Chang had her fifth COVID vaccination in the fall and recommends that others do the same. “The booster you got however many years ago was effective for the COVID of that time but there is a new COVID now. You can’t just say, ‘I had one and I’m fine forever.’”
A version of this article appeared on Medscape.com.
Though many people remain on the fence about getting the latest COVID vaccine booster, new research suggests a strong argument for getting the shot this winter: It sharply reduces the risk for COVID.
The risk reduction was 37% for those who received two doses. Experts say the research provides a strong argument for getting the vaccine, noting that about 10% of people infected with COVID go on to have long COVID, which can be debilitating for one quarter of those with long-lasting symptoms.
The data come from a systematic literature review and meta-analysis published in October in Antimicrobial Stewardship & Epidemiology. Researchers examined 32 studies published between December 2019 and June 2023, involving 775,931 adults. Twenty-four studies, encompassing 620,221 individuals, were included in the meta-analysis.
“The body of evidence from all these different studies converge on one single reality — that vaccines reduce the risk of long COVID, and people who keep up to date on their vaccinations also fared better than people who got it once or twice and didn’t follow up,” said Ziyad Al-Aly, MD, a clinical epidemiologist at Washington University in St Louis.
Researchers have reported similar results for children. The National Institutes of Health RECOVER Initiative team found that vaccines are up to 42% effective in preventing long COVID in children, said Dr. Carlos Oliveira, MD, a pediatric infectious diseases specialist and Yale researcher who contributed to the study, which is in preprint.
Vaccines also protect children from multisystem inflammatory syndrome, a condition that can happen after COVID, as well as protect against other COVID-related problems, such as missed school days, Oliveira said. “Even if the vaccine doesn’t completely stop long COVID, it’s still good for kids to get vaccinated for all these other reasons.”
However, uptake for the latest boosters has been slow: the Centers for Disease Control and Prevention reported that by mid-November, less than 16% of people aged 18 years or older had received a shot. For children, the number was closer to 6%. A recent Kaiser Family Foundation survey found that booster rates for adults are similar to what it was 1 year ago.
The survey results suggest that people are no longer as worried about COVID, which is why there is less concerned about keeping up with boosters. Though the current mutation of the virus is not as debilitating as its predecessors, long COVID continues to be a problem: as of January 2023, 28% of people who had contracted the virus had experienced long-COVID symptoms. And though the mechanisms are still not fully understood, and researchers have yet to agree on a definition of long COVID, they are certain about this much: The best way to avoid it is to avoid getting infected to begin with.
The lack of a diagnostic test for long COVID and the fact that the symptoms mimic those of other diseases lead to inconsistency that can make studies hard to replicate. In the papers reviewed for the Antimicrobial Stewardship & Epidemiology study, long COVID was defined as having symptoms lasting from more than 4 weeks to more than 6 months. Alexandre Marra, MD, the lead author and a researcher at the Hospital Israelita Albert Einstein, in São Paulo, Brazil, and at the University of Iowa, said that a clear standard definition is needed to better understand the actual prevalence and evaluate vaccine effectiveness.
Al-Aly noted that there is a logical explanation for one finding in the paper: The percentage of individuals who had COVID and reported that long-COVID symptoms declined from 19% in June 2022 to 11% in January 2023.
Because a pandemic is a dynamic event, constantly producing different variants with different phenotypes, the prevalence of disease is naturally going to be affected. “People who got infected early in the pandemic may have a different long COVID profile and long COVID risk than people who got infected in the second or third year of the pandemic,” Al-Aly said.
Most of the studies reported data from before the Omicron-variant era. Only eight reported data during that era. Omicron was not as lethal as previous variants, and consequently, fewer patients developed long COVID during that time.
One of those who did is Yeng Chang, age 40 years, a family doctor who lives in Sherwood Park, Alberta, Canada. Chang developed long COVID during fall 2022 after getting the virus in June. By then, she’d been vaccinated three times, but she isn’t surprised that she got sick because each vaccine she had was developed before Omicron.
“When I had COVID I was really sick, but I was well enough to stay home,” she said. “I think if I didn’t have my immunizations, I might have been hospitalized, and I don’t know what would have happened.”
Long COVID has left Chang with brain fog, fatigue, and a lack of physical stamina that forced her to pause her medical practice. For the past year and a half, she’s spent more time as a patient than a physician.
Chang had her fifth COVID vaccination in the fall and recommends that others do the same. “The booster you got however many years ago was effective for the COVID of that time but there is a new COVID now. You can’t just say, ‘I had one and I’m fine forever.’”
A version of this article appeared on Medscape.com.
Though many people remain on the fence about getting the latest COVID vaccine booster, new research suggests a strong argument for getting the shot this winter: It sharply reduces the risk for COVID.
The risk reduction was 37% for those who received two doses. Experts say the research provides a strong argument for getting the vaccine, noting that about 10% of people infected with COVID go on to have long COVID, which can be debilitating for one quarter of those with long-lasting symptoms.
The data come from a systematic literature review and meta-analysis published in October in Antimicrobial Stewardship & Epidemiology. Researchers examined 32 studies published between December 2019 and June 2023, involving 775,931 adults. Twenty-four studies, encompassing 620,221 individuals, were included in the meta-analysis.
“The body of evidence from all these different studies converge on one single reality — that vaccines reduce the risk of long COVID, and people who keep up to date on their vaccinations also fared better than people who got it once or twice and didn’t follow up,” said Ziyad Al-Aly, MD, a clinical epidemiologist at Washington University in St Louis.
Researchers have reported similar results for children. The National Institutes of Health RECOVER Initiative team found that vaccines are up to 42% effective in preventing long COVID in children, said Dr. Carlos Oliveira, MD, a pediatric infectious diseases specialist and Yale researcher who contributed to the study, which is in preprint.
Vaccines also protect children from multisystem inflammatory syndrome, a condition that can happen after COVID, as well as protect against other COVID-related problems, such as missed school days, Oliveira said. “Even if the vaccine doesn’t completely stop long COVID, it’s still good for kids to get vaccinated for all these other reasons.”
However, uptake for the latest boosters has been slow: the Centers for Disease Control and Prevention reported that by mid-November, less than 16% of people aged 18 years or older had received a shot. For children, the number was closer to 6%. A recent Kaiser Family Foundation survey found that booster rates for adults are similar to what it was 1 year ago.
The survey results suggest that people are no longer as worried about COVID, which is why there is less concerned about keeping up with boosters. Though the current mutation of the virus is not as debilitating as its predecessors, long COVID continues to be a problem: as of January 2023, 28% of people who had contracted the virus had experienced long-COVID symptoms. And though the mechanisms are still not fully understood, and researchers have yet to agree on a definition of long COVID, they are certain about this much: The best way to avoid it is to avoid getting infected to begin with.
The lack of a diagnostic test for long COVID and the fact that the symptoms mimic those of other diseases lead to inconsistency that can make studies hard to replicate. In the papers reviewed for the Antimicrobial Stewardship & Epidemiology study, long COVID was defined as having symptoms lasting from more than 4 weeks to more than 6 months. Alexandre Marra, MD, the lead author and a researcher at the Hospital Israelita Albert Einstein, in São Paulo, Brazil, and at the University of Iowa, said that a clear standard definition is needed to better understand the actual prevalence and evaluate vaccine effectiveness.
Al-Aly noted that there is a logical explanation for one finding in the paper: The percentage of individuals who had COVID and reported that long-COVID symptoms declined from 19% in June 2022 to 11% in January 2023.
Because a pandemic is a dynamic event, constantly producing different variants with different phenotypes, the prevalence of disease is naturally going to be affected. “People who got infected early in the pandemic may have a different long COVID profile and long COVID risk than people who got infected in the second or third year of the pandemic,” Al-Aly said.
Most of the studies reported data from before the Omicron-variant era. Only eight reported data during that era. Omicron was not as lethal as previous variants, and consequently, fewer patients developed long COVID during that time.
One of those who did is Yeng Chang, age 40 years, a family doctor who lives in Sherwood Park, Alberta, Canada. Chang developed long COVID during fall 2022 after getting the virus in June. By then, she’d been vaccinated three times, but she isn’t surprised that she got sick because each vaccine she had was developed before Omicron.
“When I had COVID I was really sick, but I was well enough to stay home,” she said. “I think if I didn’t have my immunizations, I might have been hospitalized, and I don’t know what would have happened.”
Long COVID has left Chang with brain fog, fatigue, and a lack of physical stamina that forced her to pause her medical practice. For the past year and a half, she’s spent more time as a patient than a physician.
Chang had her fifth COVID vaccination in the fall and recommends that others do the same. “The booster you got however many years ago was effective for the COVID of that time but there is a new COVID now. You can’t just say, ‘I had one and I’m fine forever.’”
A version of this article appeared on Medscape.com.
Compelling case for skipping RT in some early breast cancers
Results of the PROSPECT trial provide compelling evidence that high-quality preoperative MRI in combination with postoperative analysis of pathologic features can identify a substantial subset of women with localized early breast cancer who could safely skip radiation therapy.
(1%) at 5 years, reported lead investigator Gregory Bruce Mann, MBBS, PhD, of The Royal Women’s Hospital, Melbourne, Australia, at the San Antonio Breast Cancer Symposium (abstract PS02-03).
Additionally, women who skipped radiation had superior health-related quality of life relative to peers who underwent the treatment and, quite unexpectedly, their fear of cancer recurrence was “dramatically reduced,” Dr, Mann said in an interview.
“The hypothesis was that less treatment [would] lead to more fear of cancer recurrence” because patients would worry that they hadn’t received standard treatment, “but patients who omitted RT actually had less fear of cancer recurrence,” he said.
This may come down to positive perceptions about tailored care and trust, he explained. “If the patient got the impression that the doctor wasn’t worried about recurrence, then the patient wasn’t worried. If they trusted you and you had that relationship with the patient, they were less likely to experience a fear of recurrence.”
Results of the PROSPECT trial were published online on December 5 in The Lancet.
PROSPECT was a prospective, nonrandomized study that evaluated whether preoperative bilateral contrast-enhanced 3-Tesla breast MRI and postoperative tumor pathology could identify patients with “truly localized” disease who might feasibly skip radiation therapy after breast-conserving surgery.
The researchers hypothesised that radiation therapy reduces local recurrence risk by treating occult synchronous disease that has not been identified by conventional imaging techniques. Exclusion of such occult disease using preoperative MRI, in association with low-risk pathology, could define a group of patients with early breast cancer in whom radiation can be omitted without substantially compromising local recurrence rates.
Women aged 50 years or older with cT1N0 non–triple-negative breast cancer were eligible for the trial. Among 443 patients, preoperative MRI detected 61 malignant occult lesions separate from the index cancer in 48 patients (11%) of the total cohort.
Patients with apparently unifocal cancer had breast-conserving surgery and, if pT1N0 or N1mi, did not undergo radiation therapy (group 1: 201 women). Standard treatment including radiation therapy was offered to the others (group 2: 242 women). All women were recommended for systemic therapy. The primary endpoint was the ipsilateral invasive recurrence rate at 5 years, with follow-up to continue to 10 years.
At a median follow-up of 5.4 years, the ipsilateral invasive recurrence rate in group 1 was exceedingly low — just 1.0% (upper 95% CI, 5.4%) — with one local recurrence at 4.5 years and a second at 7.5 years. In group 2, local recurrence at 5 years was also low, at 1.7% (upper 95% CI, 6.1%).
The only case of distant metastasis in the entire cohort was genetically distinct from the index cancer.
Omitting radiation therapy led to better health-related quality of life and functional and cosmetic outcomes, and the women viewed not having radiation as highly acceptable and appropriate treatment, not undertreatment.
PROSPECT has defined a role for “very high quality” preoperative MRI in identifying patients who can be considered for deintensified treatment, Dr. Mann said.
The findings need to be replicated in multicenter, international trials, “and that’s what we are working on,” he added.
Risk Tolerance and Personal Preferences
Writing in a comment for The Lancet, Lior Z. Braunstein, MD, with Memorial Sloan Kettering Cancer Center in New York, says that overall, PROSPECT and comparable trials of radiation therapy omission, “rather than setting uniform clinical practice, will empower patients to delineate their individual risk tolerance and personal preferences.”
He notes, however, that “the use of preoperative MRI among patients at low risk remains somewhat controversial. Indeed, the MRI intervention in PROSPECT was not entirely benign, prompting nearly 200 biopsies and five of the nine observed mastectomies.”
Dr. Braunstein concludes that with numerous approaches to risk profiling, “informed patients might very reasonably choose differing paths. Indeed, it is precisely this individualized approach to breast cancer management that has long been the promise of personalized medicine — PROSPECT adds laudably to that tradition.”
Funding for the trial was provided by Breast Cancer Trials, National Breast Cancer Foundation, Cancer Council Victoria, the Royal Melbourne Hospital Foundation, and the Breast Cancer Research Foundation. Dr. Mann and Dr. Braunstein have no relevant disclosures.Megan Brooks has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
Results of the PROSPECT trial provide compelling evidence that high-quality preoperative MRI in combination with postoperative analysis of pathologic features can identify a substantial subset of women with localized early breast cancer who could safely skip radiation therapy.
(1%) at 5 years, reported lead investigator Gregory Bruce Mann, MBBS, PhD, of The Royal Women’s Hospital, Melbourne, Australia, at the San Antonio Breast Cancer Symposium (abstract PS02-03).
Additionally, women who skipped radiation had superior health-related quality of life relative to peers who underwent the treatment and, quite unexpectedly, their fear of cancer recurrence was “dramatically reduced,” Dr, Mann said in an interview.
“The hypothesis was that less treatment [would] lead to more fear of cancer recurrence” because patients would worry that they hadn’t received standard treatment, “but patients who omitted RT actually had less fear of cancer recurrence,” he said.
This may come down to positive perceptions about tailored care and trust, he explained. “If the patient got the impression that the doctor wasn’t worried about recurrence, then the patient wasn’t worried. If they trusted you and you had that relationship with the patient, they were less likely to experience a fear of recurrence.”
Results of the PROSPECT trial were published online on December 5 in The Lancet.
PROSPECT was a prospective, nonrandomized study that evaluated whether preoperative bilateral contrast-enhanced 3-Tesla breast MRI and postoperative tumor pathology could identify patients with “truly localized” disease who might feasibly skip radiation therapy after breast-conserving surgery.
The researchers hypothesised that radiation therapy reduces local recurrence risk by treating occult synchronous disease that has not been identified by conventional imaging techniques. Exclusion of such occult disease using preoperative MRI, in association with low-risk pathology, could define a group of patients with early breast cancer in whom radiation can be omitted without substantially compromising local recurrence rates.
Women aged 50 years or older with cT1N0 non–triple-negative breast cancer were eligible for the trial. Among 443 patients, preoperative MRI detected 61 malignant occult lesions separate from the index cancer in 48 patients (11%) of the total cohort.
Patients with apparently unifocal cancer had breast-conserving surgery and, if pT1N0 or N1mi, did not undergo radiation therapy (group 1: 201 women). Standard treatment including radiation therapy was offered to the others (group 2: 242 women). All women were recommended for systemic therapy. The primary endpoint was the ipsilateral invasive recurrence rate at 5 years, with follow-up to continue to 10 years.
At a median follow-up of 5.4 years, the ipsilateral invasive recurrence rate in group 1 was exceedingly low — just 1.0% (upper 95% CI, 5.4%) — with one local recurrence at 4.5 years and a second at 7.5 years. In group 2, local recurrence at 5 years was also low, at 1.7% (upper 95% CI, 6.1%).
The only case of distant metastasis in the entire cohort was genetically distinct from the index cancer.
Omitting radiation therapy led to better health-related quality of life and functional and cosmetic outcomes, and the women viewed not having radiation as highly acceptable and appropriate treatment, not undertreatment.
PROSPECT has defined a role for “very high quality” preoperative MRI in identifying patients who can be considered for deintensified treatment, Dr. Mann said.
The findings need to be replicated in multicenter, international trials, “and that’s what we are working on,” he added.
Risk Tolerance and Personal Preferences
Writing in a comment for The Lancet, Lior Z. Braunstein, MD, with Memorial Sloan Kettering Cancer Center in New York, says that overall, PROSPECT and comparable trials of radiation therapy omission, “rather than setting uniform clinical practice, will empower patients to delineate their individual risk tolerance and personal preferences.”
He notes, however, that “the use of preoperative MRI among patients at low risk remains somewhat controversial. Indeed, the MRI intervention in PROSPECT was not entirely benign, prompting nearly 200 biopsies and five of the nine observed mastectomies.”
Dr. Braunstein concludes that with numerous approaches to risk profiling, “informed patients might very reasonably choose differing paths. Indeed, it is precisely this individualized approach to breast cancer management that has long been the promise of personalized medicine — PROSPECT adds laudably to that tradition.”
Funding for the trial was provided by Breast Cancer Trials, National Breast Cancer Foundation, Cancer Council Victoria, the Royal Melbourne Hospital Foundation, and the Breast Cancer Research Foundation. Dr. Mann and Dr. Braunstein have no relevant disclosures.Megan Brooks has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
Results of the PROSPECT trial provide compelling evidence that high-quality preoperative MRI in combination with postoperative analysis of pathologic features can identify a substantial subset of women with localized early breast cancer who could safely skip radiation therapy.
(1%) at 5 years, reported lead investigator Gregory Bruce Mann, MBBS, PhD, of The Royal Women’s Hospital, Melbourne, Australia, at the San Antonio Breast Cancer Symposium (abstract PS02-03).
Additionally, women who skipped radiation had superior health-related quality of life relative to peers who underwent the treatment and, quite unexpectedly, their fear of cancer recurrence was “dramatically reduced,” Dr, Mann said in an interview.
“The hypothesis was that less treatment [would] lead to more fear of cancer recurrence” because patients would worry that they hadn’t received standard treatment, “but patients who omitted RT actually had less fear of cancer recurrence,” he said.
This may come down to positive perceptions about tailored care and trust, he explained. “If the patient got the impression that the doctor wasn’t worried about recurrence, then the patient wasn’t worried. If they trusted you and you had that relationship with the patient, they were less likely to experience a fear of recurrence.”
Results of the PROSPECT trial were published online on December 5 in The Lancet.
PROSPECT was a prospective, nonrandomized study that evaluated whether preoperative bilateral contrast-enhanced 3-Tesla breast MRI and postoperative tumor pathology could identify patients with “truly localized” disease who might feasibly skip radiation therapy after breast-conserving surgery.
The researchers hypothesised that radiation therapy reduces local recurrence risk by treating occult synchronous disease that has not been identified by conventional imaging techniques. Exclusion of such occult disease using preoperative MRI, in association with low-risk pathology, could define a group of patients with early breast cancer in whom radiation can be omitted without substantially compromising local recurrence rates.
Women aged 50 years or older with cT1N0 non–triple-negative breast cancer were eligible for the trial. Among 443 patients, preoperative MRI detected 61 malignant occult lesions separate from the index cancer in 48 patients (11%) of the total cohort.
Patients with apparently unifocal cancer had breast-conserving surgery and, if pT1N0 or N1mi, did not undergo radiation therapy (group 1: 201 women). Standard treatment including radiation therapy was offered to the others (group 2: 242 women). All women were recommended for systemic therapy. The primary endpoint was the ipsilateral invasive recurrence rate at 5 years, with follow-up to continue to 10 years.
At a median follow-up of 5.4 years, the ipsilateral invasive recurrence rate in group 1 was exceedingly low — just 1.0% (upper 95% CI, 5.4%) — with one local recurrence at 4.5 years and a second at 7.5 years. In group 2, local recurrence at 5 years was also low, at 1.7% (upper 95% CI, 6.1%).
The only case of distant metastasis in the entire cohort was genetically distinct from the index cancer.
Omitting radiation therapy led to better health-related quality of life and functional and cosmetic outcomes, and the women viewed not having radiation as highly acceptable and appropriate treatment, not undertreatment.
PROSPECT has defined a role for “very high quality” preoperative MRI in identifying patients who can be considered for deintensified treatment, Dr. Mann said.
The findings need to be replicated in multicenter, international trials, “and that’s what we are working on,” he added.
Risk Tolerance and Personal Preferences
Writing in a comment for The Lancet, Lior Z. Braunstein, MD, with Memorial Sloan Kettering Cancer Center in New York, says that overall, PROSPECT and comparable trials of radiation therapy omission, “rather than setting uniform clinical practice, will empower patients to delineate their individual risk tolerance and personal preferences.”
He notes, however, that “the use of preoperative MRI among patients at low risk remains somewhat controversial. Indeed, the MRI intervention in PROSPECT was not entirely benign, prompting nearly 200 biopsies and five of the nine observed mastectomies.”
Dr. Braunstein concludes that with numerous approaches to risk profiling, “informed patients might very reasonably choose differing paths. Indeed, it is precisely this individualized approach to breast cancer management that has long been the promise of personalized medicine — PROSPECT adds laudably to that tradition.”
Funding for the trial was provided by Breast Cancer Trials, National Breast Cancer Foundation, Cancer Council Victoria, the Royal Melbourne Hospital Foundation, and the Breast Cancer Research Foundation. Dr. Mann and Dr. Braunstein have no relevant disclosures.Megan Brooks has disclosed no relevant financial relationships.
A version of this article appeared on Medscape.com.
FROM SABCS 2023
FDA mandates five changes to iPLEDGE program for isotretinoin
In a letter dated Nov. 30, 2023, the .
The development follows a March 2023 joint meeting of the FDA’s Drug Safety and Risk Management Advisory Committee and the Dermatologic and Ophthalmic Drugs Advisory Committee about iPLEDGE REMS requirements, which included feedback from patients and dermatologists and recommendations for changes to the REMS program, aimed at minimizing the burden of the program on patients, pharmacies, and prescribers while continuing to maintain safe use of the highly teratogenic drug for patients.
The five changes include the following:
- Remove the requirement that pregnancy tests must be performed in a specially certified (i.e., Clinical Laboratory Improvement Amendments [CLIA]) laboratory. In the opinion of John S. Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital, Boston, this change “may make it easier to perform pregnancy tests in a clinic setting without needing to send the patient to a separate lab,” he said in an interview.
- Allow prescribers the option of using home pregnancy testing for their patients during and after isotretinoin treatment. Prescribers who rely on the patient to perform a home pregnancy test need to take steps to minimize patients falsifying the results of these tests. According to Dr. Barbieri, this means that two pregnancy tests prior to starting isotretinoin must be done in a lab or office setting. “However, all the pregnancy tests on therapy can be either in a medical setting or using a home pregnancy test,” he told this news organization. “This option facilitates the use of telemedicine so that patients would not need to come in; they can just share a pregnancy test with their name and date with their dermatologist.”
- Remove the waiting period requirement — also known as the “19-day lockout” — for patients if they do not obtain isotretinoin within the first 7-day prescription window. According to Dr. Barbieri, this change helps to ensure that patients can begin isotretinoin in a timely manner. “Insurance and pharmacy delays that are no fault of the patient can commonly cause missed initial window periods,” he said. “Allowing for immediate repeat of a pregnancy test to start a new window period, rather than requiring the patient to wait 19 more days, can ensure patient safety and pregnancy prevention without negatively impacting access.”
- Revise the pregnancy registry requirement to remove the objective to document the pregnancy and fetal outcomes for each pregnancy.
- Revise the requirement for prescribers to document patient counseling in patients who cannot become pregnant from monthly to only at enrollment. Dr. Barbieri characterized this change as “major” and said that it could eliminate the need for monthly visits for persons of non–childbearing potential. “This could substantially reduce logistical burdens for patients and reduce wait times to see a dermatologist,” he said.
Future changes to iPLEDGE that Dr. Barbieri would like to see include allowing for home pregnancy tests prior to starting therapy — particularly the test after the 30-day window period. “In addition, it would be good to be able to reduce the 30-day waiting period prior to therapy to something shorter,” such as 14 days, which would still “reliably exclude pregnancy, particularly for those on stable long-acting reversible contraception,” he said. There are also opportunities to improve the iPLEDGE website functionality and to ensure that the website is accessible to patients with limited English proficiency, he added.
He also recommended greater transparency by the Isotretinoin Products Manufacturers Group and inclusion of input from diverse stakeholders such as dermatologists, patients, and pharmacists.
Dr. Barbieri reported personal fees from Dexcel Pharma.
In a letter dated Nov. 30, 2023, the .
The development follows a March 2023 joint meeting of the FDA’s Drug Safety and Risk Management Advisory Committee and the Dermatologic and Ophthalmic Drugs Advisory Committee about iPLEDGE REMS requirements, which included feedback from patients and dermatologists and recommendations for changes to the REMS program, aimed at minimizing the burden of the program on patients, pharmacies, and prescribers while continuing to maintain safe use of the highly teratogenic drug for patients.
The five changes include the following:
- Remove the requirement that pregnancy tests must be performed in a specially certified (i.e., Clinical Laboratory Improvement Amendments [CLIA]) laboratory. In the opinion of John S. Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital, Boston, this change “may make it easier to perform pregnancy tests in a clinic setting without needing to send the patient to a separate lab,” he said in an interview.
- Allow prescribers the option of using home pregnancy testing for their patients during and after isotretinoin treatment. Prescribers who rely on the patient to perform a home pregnancy test need to take steps to minimize patients falsifying the results of these tests. According to Dr. Barbieri, this means that two pregnancy tests prior to starting isotretinoin must be done in a lab or office setting. “However, all the pregnancy tests on therapy can be either in a medical setting or using a home pregnancy test,” he told this news organization. “This option facilitates the use of telemedicine so that patients would not need to come in; they can just share a pregnancy test with their name and date with their dermatologist.”
- Remove the waiting period requirement — also known as the “19-day lockout” — for patients if they do not obtain isotretinoin within the first 7-day prescription window. According to Dr. Barbieri, this change helps to ensure that patients can begin isotretinoin in a timely manner. “Insurance and pharmacy delays that are no fault of the patient can commonly cause missed initial window periods,” he said. “Allowing for immediate repeat of a pregnancy test to start a new window period, rather than requiring the patient to wait 19 more days, can ensure patient safety and pregnancy prevention without negatively impacting access.”
- Revise the pregnancy registry requirement to remove the objective to document the pregnancy and fetal outcomes for each pregnancy.
- Revise the requirement for prescribers to document patient counseling in patients who cannot become pregnant from monthly to only at enrollment. Dr. Barbieri characterized this change as “major” and said that it could eliminate the need for monthly visits for persons of non–childbearing potential. “This could substantially reduce logistical burdens for patients and reduce wait times to see a dermatologist,” he said.
Future changes to iPLEDGE that Dr. Barbieri would like to see include allowing for home pregnancy tests prior to starting therapy — particularly the test after the 30-day window period. “In addition, it would be good to be able to reduce the 30-day waiting period prior to therapy to something shorter,” such as 14 days, which would still “reliably exclude pregnancy, particularly for those on stable long-acting reversible contraception,” he said. There are also opportunities to improve the iPLEDGE website functionality and to ensure that the website is accessible to patients with limited English proficiency, he added.
He also recommended greater transparency by the Isotretinoin Products Manufacturers Group and inclusion of input from diverse stakeholders such as dermatologists, patients, and pharmacists.
Dr. Barbieri reported personal fees from Dexcel Pharma.
In a letter dated Nov. 30, 2023, the .
The development follows a March 2023 joint meeting of the FDA’s Drug Safety and Risk Management Advisory Committee and the Dermatologic and Ophthalmic Drugs Advisory Committee about iPLEDGE REMS requirements, which included feedback from patients and dermatologists and recommendations for changes to the REMS program, aimed at minimizing the burden of the program on patients, pharmacies, and prescribers while continuing to maintain safe use of the highly teratogenic drug for patients.
The five changes include the following:
- Remove the requirement that pregnancy tests must be performed in a specially certified (i.e., Clinical Laboratory Improvement Amendments [CLIA]) laboratory. In the opinion of John S. Barbieri, MD, MBA, director of the Advanced Acne Therapeutics Clinic at Brigham and Women’s Hospital, Boston, this change “may make it easier to perform pregnancy tests in a clinic setting without needing to send the patient to a separate lab,” he said in an interview.
- Allow prescribers the option of using home pregnancy testing for their patients during and after isotretinoin treatment. Prescribers who rely on the patient to perform a home pregnancy test need to take steps to minimize patients falsifying the results of these tests. According to Dr. Barbieri, this means that two pregnancy tests prior to starting isotretinoin must be done in a lab or office setting. “However, all the pregnancy tests on therapy can be either in a medical setting or using a home pregnancy test,” he told this news organization. “This option facilitates the use of telemedicine so that patients would not need to come in; they can just share a pregnancy test with their name and date with their dermatologist.”
- Remove the waiting period requirement — also known as the “19-day lockout” — for patients if they do not obtain isotretinoin within the first 7-day prescription window. According to Dr. Barbieri, this change helps to ensure that patients can begin isotretinoin in a timely manner. “Insurance and pharmacy delays that are no fault of the patient can commonly cause missed initial window periods,” he said. “Allowing for immediate repeat of a pregnancy test to start a new window period, rather than requiring the patient to wait 19 more days, can ensure patient safety and pregnancy prevention without negatively impacting access.”
- Revise the pregnancy registry requirement to remove the objective to document the pregnancy and fetal outcomes for each pregnancy.
- Revise the requirement for prescribers to document patient counseling in patients who cannot become pregnant from monthly to only at enrollment. Dr. Barbieri characterized this change as “major” and said that it could eliminate the need for monthly visits for persons of non–childbearing potential. “This could substantially reduce logistical burdens for patients and reduce wait times to see a dermatologist,” he said.
Future changes to iPLEDGE that Dr. Barbieri would like to see include allowing for home pregnancy tests prior to starting therapy — particularly the test after the 30-day window period. “In addition, it would be good to be able to reduce the 30-day waiting period prior to therapy to something shorter,” such as 14 days, which would still “reliably exclude pregnancy, particularly for those on stable long-acting reversible contraception,” he said. There are also opportunities to improve the iPLEDGE website functionality and to ensure that the website is accessible to patients with limited English proficiency, he added.
He also recommended greater transparency by the Isotretinoin Products Manufacturers Group and inclusion of input from diverse stakeholders such as dermatologists, patients, and pharmacists.
Dr. Barbieri reported personal fees from Dexcel Pharma.
Meet the newest acronym in primary care: CKM
The advisory, published recently in Circulation introduces the concept of CKM health and reevaluates the relationships between obesity, diabetes, kidney disease, and cardiovascular disease (CVD).
“This approach not only raises awareness, it also empowers PCPs to diagnose and treat these conditions more holistically,” Salim Hayek, MD, associate professor of cardiovascular disease and internal medicine, and medical director of the Frankel Cardiovascular Center Clinics at the University of Michigan in Ann Arbor, said in an interview.
New CKM Staging, Testing, and Care Strategies
The advisory introduces a new scoring system that ranges from stage 0 (patients with no risk factors for CKM) through stage 4 (patients with clinical CVD in CKM syndrome). Each stage requires specific management strategies and may include screening starting at age 30 years for diabetes, hypertension, and heart failure.
“Stage 0 CKM is usually found in young people, and CKM risk factors and scores typically increase as people age,” said Sean M. Drake, MD, a primary care physician at Henry Ford Health in Sterling Heights, Michigan.
Dr. Drake advised PCPs to encourage patients who are at stage 0 to maintain ideal cardiovascular health and to monitor those at risk of progressing through the stages.
While PCPs already perform many of the tests the advisory recommends, the conditions overlap and an abnormality in one system should prompt more testing for other conditions. Additional tests, such as urine albumin-creatinine ratio, and more frequent glomerular filtration rate and lipid profile are advised, according to Dr. Drake.
“There also appears to be a role for additional cardiac testing, including echocardiograms and coronary CT scans, and for liver fibrosis screening,” Dr. Drake said. “Medications such as SGLT2 inhibitors, GLP-1 receptor agonists, and ACE inhibitors, beyond current routine use, are emphasized.”
To better characterize body composition and help diagnose metabolic syndrome, the advisory also recommends measuring waist circumference, which is not routine practice, noted Joshua J. Joseph, MD, MPH, an associate professor of endocrinology, diabetes, and metabolism at The Ohio State University Wexner Medical Center in Columbus, and a co-author of the advisory.
Recognizing the interconnected nature of cardiac, kidney, and metabolic diseases encourages a shift in mindset for clinicians, according to Neha Pagidipati, MD, MPH, a cardiologist at Duke Health in Durham, North Carolina.
“We have often been trained to focus on the specific problem in front of us,” Dr. Pagidipati said. “We need to be hyper-aware that many patients we see are at risk for multiple CKM entities. We need to be proactive about screening for and treating these when appropriate.”
The advisory emphasizes the need for CKM coordinators to support teams of clinicians from primary care, cardiology, endocrinology, nephrology, nursing, and pharmacy, as well as social workers, care navigators, or community health workers, Dr. Joseph said.
“The advisory repositions the PCP at the forefront of CKM care coordination, marking a departure from the traditional model where subspecialists primarily manage complications,” Dr. Hayek added.
Changes to Payment
The new recommendations are consistent with current management guidelines for obesity, hypertriglyceridemia, hypertension, type 2 diabetes, and chronic kidney disease.
“The advisory provides integrated algorithms for cardiovascular prevention and management, with specific therapeutic guidance tied to CKM stages, bringing together the current evidence for best practices from the various guidelines and filling gaps in a unified approach,” Dr. Joseph said.
In addition, the advisory draws attention to the care of younger patients, who may be at increased risk for cardiovascular disease due to lifestyle factors, according to Nishant Shah, MD, assistant professor of medicine at Duke.
“It considers barriers to care that prevent people from optimizing their cardiovascular health,” Dr. Shah said.
Although the advisory does not specify proposed payment changes to support the new care model, the move towards value-based care may require billing practices that accommodate integrated care as well as more frequent and more specialized testing, Dr. Hayek said.
“The advisory is an empowering tool for PCPs, underscoring their critical role in healthcare,” Dr. Hayek said. “It encourages PCPs to advocate for integrated care within their practices and to consider workflow adjustments that enhance the identification and initiation of preventive care for at-risk patients.”
Funding information was not provided.
Dr. Joseph reports no relevant financial involvements; several advisory co-authors report financial involvements with pharmaceutical companies. Dr. Pagidipati reports relevant financial involvement with pharmaceutical companies. Dr. Hayek, Dr. Drake, and Dr. Shah report no relevant financial involvements. Dr. Joseph is an author of the advisory. Dr. Pagidipati, Dr. Hayek, Dr. Drake, and Dr. Shah were not involved in the writing of the advisory.
A version of this article appeared on Medscape.com.
The advisory, published recently in Circulation introduces the concept of CKM health and reevaluates the relationships between obesity, diabetes, kidney disease, and cardiovascular disease (CVD).
“This approach not only raises awareness, it also empowers PCPs to diagnose and treat these conditions more holistically,” Salim Hayek, MD, associate professor of cardiovascular disease and internal medicine, and medical director of the Frankel Cardiovascular Center Clinics at the University of Michigan in Ann Arbor, said in an interview.
New CKM Staging, Testing, and Care Strategies
The advisory introduces a new scoring system that ranges from stage 0 (patients with no risk factors for CKM) through stage 4 (patients with clinical CVD in CKM syndrome). Each stage requires specific management strategies and may include screening starting at age 30 years for diabetes, hypertension, and heart failure.
“Stage 0 CKM is usually found in young people, and CKM risk factors and scores typically increase as people age,” said Sean M. Drake, MD, a primary care physician at Henry Ford Health in Sterling Heights, Michigan.
Dr. Drake advised PCPs to encourage patients who are at stage 0 to maintain ideal cardiovascular health and to monitor those at risk of progressing through the stages.
While PCPs already perform many of the tests the advisory recommends, the conditions overlap and an abnormality in one system should prompt more testing for other conditions. Additional tests, such as urine albumin-creatinine ratio, and more frequent glomerular filtration rate and lipid profile are advised, according to Dr. Drake.
“There also appears to be a role for additional cardiac testing, including echocardiograms and coronary CT scans, and for liver fibrosis screening,” Dr. Drake said. “Medications such as SGLT2 inhibitors, GLP-1 receptor agonists, and ACE inhibitors, beyond current routine use, are emphasized.”
To better characterize body composition and help diagnose metabolic syndrome, the advisory also recommends measuring waist circumference, which is not routine practice, noted Joshua J. Joseph, MD, MPH, an associate professor of endocrinology, diabetes, and metabolism at The Ohio State University Wexner Medical Center in Columbus, and a co-author of the advisory.
Recognizing the interconnected nature of cardiac, kidney, and metabolic diseases encourages a shift in mindset for clinicians, according to Neha Pagidipati, MD, MPH, a cardiologist at Duke Health in Durham, North Carolina.
“We have often been trained to focus on the specific problem in front of us,” Dr. Pagidipati said. “We need to be hyper-aware that many patients we see are at risk for multiple CKM entities. We need to be proactive about screening for and treating these when appropriate.”
The advisory emphasizes the need for CKM coordinators to support teams of clinicians from primary care, cardiology, endocrinology, nephrology, nursing, and pharmacy, as well as social workers, care navigators, or community health workers, Dr. Joseph said.
“The advisory repositions the PCP at the forefront of CKM care coordination, marking a departure from the traditional model where subspecialists primarily manage complications,” Dr. Hayek added.
Changes to Payment
The new recommendations are consistent with current management guidelines for obesity, hypertriglyceridemia, hypertension, type 2 diabetes, and chronic kidney disease.
“The advisory provides integrated algorithms for cardiovascular prevention and management, with specific therapeutic guidance tied to CKM stages, bringing together the current evidence for best practices from the various guidelines and filling gaps in a unified approach,” Dr. Joseph said.
In addition, the advisory draws attention to the care of younger patients, who may be at increased risk for cardiovascular disease due to lifestyle factors, according to Nishant Shah, MD, assistant professor of medicine at Duke.
“It considers barriers to care that prevent people from optimizing their cardiovascular health,” Dr. Shah said.
Although the advisory does not specify proposed payment changes to support the new care model, the move towards value-based care may require billing practices that accommodate integrated care as well as more frequent and more specialized testing, Dr. Hayek said.
“The advisory is an empowering tool for PCPs, underscoring their critical role in healthcare,” Dr. Hayek said. “It encourages PCPs to advocate for integrated care within their practices and to consider workflow adjustments that enhance the identification and initiation of preventive care for at-risk patients.”
Funding information was not provided.
Dr. Joseph reports no relevant financial involvements; several advisory co-authors report financial involvements with pharmaceutical companies. Dr. Pagidipati reports relevant financial involvement with pharmaceutical companies. Dr. Hayek, Dr. Drake, and Dr. Shah report no relevant financial involvements. Dr. Joseph is an author of the advisory. Dr. Pagidipati, Dr. Hayek, Dr. Drake, and Dr. Shah were not involved in the writing of the advisory.
A version of this article appeared on Medscape.com.
The advisory, published recently in Circulation introduces the concept of CKM health and reevaluates the relationships between obesity, diabetes, kidney disease, and cardiovascular disease (CVD).
“This approach not only raises awareness, it also empowers PCPs to diagnose and treat these conditions more holistically,” Salim Hayek, MD, associate professor of cardiovascular disease and internal medicine, and medical director of the Frankel Cardiovascular Center Clinics at the University of Michigan in Ann Arbor, said in an interview.
New CKM Staging, Testing, and Care Strategies
The advisory introduces a new scoring system that ranges from stage 0 (patients with no risk factors for CKM) through stage 4 (patients with clinical CVD in CKM syndrome). Each stage requires specific management strategies and may include screening starting at age 30 years for diabetes, hypertension, and heart failure.
“Stage 0 CKM is usually found in young people, and CKM risk factors and scores typically increase as people age,” said Sean M. Drake, MD, a primary care physician at Henry Ford Health in Sterling Heights, Michigan.
Dr. Drake advised PCPs to encourage patients who are at stage 0 to maintain ideal cardiovascular health and to monitor those at risk of progressing through the stages.
While PCPs already perform many of the tests the advisory recommends, the conditions overlap and an abnormality in one system should prompt more testing for other conditions. Additional tests, such as urine albumin-creatinine ratio, and more frequent glomerular filtration rate and lipid profile are advised, according to Dr. Drake.
“There also appears to be a role for additional cardiac testing, including echocardiograms and coronary CT scans, and for liver fibrosis screening,” Dr. Drake said. “Medications such as SGLT2 inhibitors, GLP-1 receptor agonists, and ACE inhibitors, beyond current routine use, are emphasized.”
To better characterize body composition and help diagnose metabolic syndrome, the advisory also recommends measuring waist circumference, which is not routine practice, noted Joshua J. Joseph, MD, MPH, an associate professor of endocrinology, diabetes, and metabolism at The Ohio State University Wexner Medical Center in Columbus, and a co-author of the advisory.
Recognizing the interconnected nature of cardiac, kidney, and metabolic diseases encourages a shift in mindset for clinicians, according to Neha Pagidipati, MD, MPH, a cardiologist at Duke Health in Durham, North Carolina.
“We have often been trained to focus on the specific problem in front of us,” Dr. Pagidipati said. “We need to be hyper-aware that many patients we see are at risk for multiple CKM entities. We need to be proactive about screening for and treating these when appropriate.”
The advisory emphasizes the need for CKM coordinators to support teams of clinicians from primary care, cardiology, endocrinology, nephrology, nursing, and pharmacy, as well as social workers, care navigators, or community health workers, Dr. Joseph said.
“The advisory repositions the PCP at the forefront of CKM care coordination, marking a departure from the traditional model where subspecialists primarily manage complications,” Dr. Hayek added.
Changes to Payment
The new recommendations are consistent with current management guidelines for obesity, hypertriglyceridemia, hypertension, type 2 diabetes, and chronic kidney disease.
“The advisory provides integrated algorithms for cardiovascular prevention and management, with specific therapeutic guidance tied to CKM stages, bringing together the current evidence for best practices from the various guidelines and filling gaps in a unified approach,” Dr. Joseph said.
In addition, the advisory draws attention to the care of younger patients, who may be at increased risk for cardiovascular disease due to lifestyle factors, according to Nishant Shah, MD, assistant professor of medicine at Duke.
“It considers barriers to care that prevent people from optimizing their cardiovascular health,” Dr. Shah said.
Although the advisory does not specify proposed payment changes to support the new care model, the move towards value-based care may require billing practices that accommodate integrated care as well as more frequent and more specialized testing, Dr. Hayek said.
“The advisory is an empowering tool for PCPs, underscoring their critical role in healthcare,” Dr. Hayek said. “It encourages PCPs to advocate for integrated care within their practices and to consider workflow adjustments that enhance the identification and initiation of preventive care for at-risk patients.”
Funding information was not provided.
Dr. Joseph reports no relevant financial involvements; several advisory co-authors report financial involvements with pharmaceutical companies. Dr. Pagidipati reports relevant financial involvement with pharmaceutical companies. Dr. Hayek, Dr. Drake, and Dr. Shah report no relevant financial involvements. Dr. Joseph is an author of the advisory. Dr. Pagidipati, Dr. Hayek, Dr. Drake, and Dr. Shah were not involved in the writing of the advisory.
A version of this article appeared on Medscape.com.
FROM CIRCULATION