SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

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.

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

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.

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

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.

SAN DIEGO — CD19-directed chimeric antigen receptor (CAR) T-cell therapy, which has transformed the treatment landscape for B-cell malignancies, is now showing great promise in at least three distinct autoantibody-dependent 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.

SAN DIEGO — CD19-directed chimeric antigen receptor (CAR) T-cell therapy, which has transformed the treatment landscape for B-cell malignancies, is now showing great promise in at least three distinct autoantibody-dependent 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.

SAN DIEGO — CD19-directed chimeric antigen receptor (CAR) T-cell therapy, which has transformed the treatment landscape for B-cell malignancies, is now showing great promise in at least three distinct autoantibody-dependent 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.

SAN DIEGO — In the real world, outcomes for patients with multiple myeloma who received standard regimens were dramatically worse than those reported in clinical trials, a new study found. 

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.

SAN DIEGO — In the real world, outcomes for patients with multiple myeloma who received standard regimens were dramatically worse than those reported in clinical trials, a new study found. 

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.

SAN DIEGO — In the real world, outcomes for patients with multiple myeloma who received standard regimens were dramatically worse than those reported in clinical trials, a new study found. 

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.

SAN DIEGO — A polygenic score can link outcomes in Black pediatric patients with acute myeloid leukemia (AML) to genetic traits that arise more frequently in this population, new data reveal. 

The score, dubbed ACS10 and initially highlighted in a 2022 report, predicts how well patients will respond to cytarabine based on their genetic make-up, and has the potential to personalize treatment for Black pediatric patients, a group that often has worse outcomes than White patients.

In the current study, presented at the annual meeting of the American Society of Hematology (ASH) , Black patients with low ACS10 scores had significantly worse outcomes compared with those with high scores when initially treated with low-dose cytarabine, daunorubicin, and etoposide. 

The difference in outcomes disappeared, however, for patients who received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine. 

The genetic traits revealed by the test likely help explain why Black patients with AML typically fare worse on certain regimens, Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute, commented in an ASH press preview briefing.

This study also suggests that clinicians should perform testing for genetic variants and biomarkers that impact outcomes “instead of assuming that a certain dose should be given simply based on perceived or reported race or ethnicity,” said Dr. Dunbar, also secretary of ASH. 

The ACS10 test, derived from a combination of 10 single nucleotide polymorphisms, is not yet available, but one could be developed to help guide treatment decisions for clinicians, especially those in developing countries where AML treatment can be very expensive, said study lead author Jatinder Lamba, PhD, MSc, of the University of Florida College of Pharmacy, Gainesville, at an ASH press briefing on Thursday. 

Prior research shows that Black pediatric patients with AML often have worse outcomes than White patients. A recent study , for instance, found Black patients with AML, especially those aged 18 to 29 years, had a higher early death rate compared with White patients (16% vs 3%) and significantly lower 5-year overall survival rates (22% vs 51%). The authors of this study suggested that genetic differences between young Black and White patients could help explain the disparity. 

In the new analysis, Dr. Lamba and colleagues explored how outcomes by race and cytarabine pharmacogenomics varied in pediatric patients with AML. 

The study included 86 Black patients and 359 White patients with newly diagnosed AML treated on two multi-institutional clinical trials. The patients received one of three initial treatments that included cytarabine: high-dose or low-dose cytarabine, daunorubicin, and etoposide, or clofarabine and cytarabine. 

Most Black patients in the analysis (73%) had low ACS10 scores compared with 30% of White patients.

Unlike other recent reports, this study found that Black and White patients had similar complete remission rates following two courses of induction therapy (92.6% vs 95%) as well as similar rates of minimal residual disease negativity after one course (55.8% vs 55.4%). 

Event-free survival (EFS) and overall survival rates were also similar, with 5-year EFS estimates at 58.3% for Black patients and 58.2% for White patients and overall survival rates at 63.8% vs 69.4%, respectively (P = .24). 

However, when separating outcomes by ACS10 scores, Black patients with low scores had significantly worse EFS following low-dose cytarabine, daunorubicin, and etoposide compared with those with high ACS10 scores. And when these patients received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine induction therapy instead, the differences went away. 

Overall, Black patients demonstrated significantly better EFS following treatment with clofarabine and cytarabine compared with the low-dose cytarabine triple therapy (hazard ratio, 0.17; P = .01). After adjusting for cofounders, clofarabine and cytarabine induction was the best treatment for Black patients with low ACS10 scores (HR for EFS, 0.2).

“Our results suggest that pharmacogenomics differences between Black and White patients should be considered when tailoring induction regimens to improve outcomes of Black patients and bridge the racial disparity gap in AML treatment,” the researchers concluded.

In developing countries, especially in Africa, starting patients on high-dose cytarabine, daunorubicin, and etoposide can lead to better results “without increasing much of the economic burden” since this treatment is the cheapest, Dr. Lamba said. “At the same time, if the patients have high ACS10 score, you can reduce their economic burden by giving them standard dose” cytarabine, daunorubicin, and etoposide and achieve similar results.

No study funding was reported. Dr. Lamba reported no relevant financial relationships, and three other authors reported various disclosures. Disclosures for Dr. Dunbar were unavailable.. 
 

A version of this article appeared on Medscape.com.

SAN DIEGO — A polygenic score can link outcomes in Black pediatric patients with acute myeloid leukemia (AML) to genetic traits that arise more frequently in this population, new data reveal. 

The score, dubbed ACS10 and initially highlighted in a 2022 report, predicts how well patients will respond to cytarabine based on their genetic make-up, and has the potential to personalize treatment for Black pediatric patients, a group that often has worse outcomes than White patients.

In the current study, presented at the annual meeting of the American Society of Hematology (ASH) , Black patients with low ACS10 scores had significantly worse outcomes compared with those with high scores when initially treated with low-dose cytarabine, daunorubicin, and etoposide. 

The difference in outcomes disappeared, however, for patients who received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine. 

The genetic traits revealed by the test likely help explain why Black patients with AML typically fare worse on certain regimens, Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute, commented in an ASH press preview briefing.

This study also suggests that clinicians should perform testing for genetic variants and biomarkers that impact outcomes “instead of assuming that a certain dose should be given simply based on perceived or reported race or ethnicity,” said Dr. Dunbar, also secretary of ASH. 

The ACS10 test, derived from a combination of 10 single nucleotide polymorphisms, is not yet available, but one could be developed to help guide treatment decisions for clinicians, especially those in developing countries where AML treatment can be very expensive, said study lead author Jatinder Lamba, PhD, MSc, of the University of Florida College of Pharmacy, Gainesville, at an ASH press briefing on Thursday. 

Prior research shows that Black pediatric patients with AML often have worse outcomes than White patients. A recent study , for instance, found Black patients with AML, especially those aged 18 to 29 years, had a higher early death rate compared with White patients (16% vs 3%) and significantly lower 5-year overall survival rates (22% vs 51%). The authors of this study suggested that genetic differences between young Black and White patients could help explain the disparity. 

In the new analysis, Dr. Lamba and colleagues explored how outcomes by race and cytarabine pharmacogenomics varied in pediatric patients with AML. 

The study included 86 Black patients and 359 White patients with newly diagnosed AML treated on two multi-institutional clinical trials. The patients received one of three initial treatments that included cytarabine: high-dose or low-dose cytarabine, daunorubicin, and etoposide, or clofarabine and cytarabine. 

Most Black patients in the analysis (73%) had low ACS10 scores compared with 30% of White patients.

Unlike other recent reports, this study found that Black and White patients had similar complete remission rates following two courses of induction therapy (92.6% vs 95%) as well as similar rates of minimal residual disease negativity after one course (55.8% vs 55.4%). 

Event-free survival (EFS) and overall survival rates were also similar, with 5-year EFS estimates at 58.3% for Black patients and 58.2% for White patients and overall survival rates at 63.8% vs 69.4%, respectively (P = .24). 

However, when separating outcomes by ACS10 scores, Black patients with low scores had significantly worse EFS following low-dose cytarabine, daunorubicin, and etoposide compared with those with high ACS10 scores. And when these patients received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine induction therapy instead, the differences went away. 

Overall, Black patients demonstrated significantly better EFS following treatment with clofarabine and cytarabine compared with the low-dose cytarabine triple therapy (hazard ratio, 0.17; P = .01). After adjusting for cofounders, clofarabine and cytarabine induction was the best treatment for Black patients with low ACS10 scores (HR for EFS, 0.2).

“Our results suggest that pharmacogenomics differences between Black and White patients should be considered when tailoring induction regimens to improve outcomes of Black patients and bridge the racial disparity gap in AML treatment,” the researchers concluded.

In developing countries, especially in Africa, starting patients on high-dose cytarabine, daunorubicin, and etoposide can lead to better results “without increasing much of the economic burden” since this treatment is the cheapest, Dr. Lamba said. “At the same time, if the patients have high ACS10 score, you can reduce their economic burden by giving them standard dose” cytarabine, daunorubicin, and etoposide and achieve similar results.

No study funding was reported. Dr. Lamba reported no relevant financial relationships, and three other authors reported various disclosures. Disclosures for Dr. Dunbar were unavailable.. 
 

A version of this article appeared on Medscape.com.

SAN DIEGO — A polygenic score can link outcomes in Black pediatric patients with acute myeloid leukemia (AML) to genetic traits that arise more frequently in this population, new data reveal. 

The score, dubbed ACS10 and initially highlighted in a 2022 report, predicts how well patients will respond to cytarabine based on their genetic make-up, and has the potential to personalize treatment for Black pediatric patients, a group that often has worse outcomes than White patients.

In the current study, presented at the annual meeting of the American Society of Hematology (ASH) , Black patients with low ACS10 scores had significantly worse outcomes compared with those with high scores when initially treated with low-dose cytarabine, daunorubicin, and etoposide. 

The difference in outcomes disappeared, however, for patients who received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine. 

The genetic traits revealed by the test likely help explain why Black patients with AML typically fare worse on certain regimens, Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute, commented in an ASH press preview briefing.

This study also suggests that clinicians should perform testing for genetic variants and biomarkers that impact outcomes “instead of assuming that a certain dose should be given simply based on perceived or reported race or ethnicity,” said Dr. Dunbar, also secretary of ASH. 

The ACS10 test, derived from a combination of 10 single nucleotide polymorphisms, is not yet available, but one could be developed to help guide treatment decisions for clinicians, especially those in developing countries where AML treatment can be very expensive, said study lead author Jatinder Lamba, PhD, MSc, of the University of Florida College of Pharmacy, Gainesville, at an ASH press briefing on Thursday. 

Prior research shows that Black pediatric patients with AML often have worse outcomes than White patients. A recent study , for instance, found Black patients with AML, especially those aged 18 to 29 years, had a higher early death rate compared with White patients (16% vs 3%) and significantly lower 5-year overall survival rates (22% vs 51%). The authors of this study suggested that genetic differences between young Black and White patients could help explain the disparity. 

In the new analysis, Dr. Lamba and colleagues explored how outcomes by race and cytarabine pharmacogenomics varied in pediatric patients with AML. 

The study included 86 Black patients and 359 White patients with newly diagnosed AML treated on two multi-institutional clinical trials. The patients received one of three initial treatments that included cytarabine: high-dose or low-dose cytarabine, daunorubicin, and etoposide, or clofarabine and cytarabine. 

Most Black patients in the analysis (73%) had low ACS10 scores compared with 30% of White patients.

Unlike other recent reports, this study found that Black and White patients had similar complete remission rates following two courses of induction therapy (92.6% vs 95%) as well as similar rates of minimal residual disease negativity after one course (55.8% vs 55.4%). 

Event-free survival (EFS) and overall survival rates were also similar, with 5-year EFS estimates at 58.3% for Black patients and 58.2% for White patients and overall survival rates at 63.8% vs 69.4%, respectively (P = .24). 

However, when separating outcomes by ACS10 scores, Black patients with low scores had significantly worse EFS following low-dose cytarabine, daunorubicin, and etoposide compared with those with high ACS10 scores. And when these patients received high-dose cytarabine, daunorubicin, and etoposide or clofarabine and cytarabine induction therapy instead, the differences went away. 

Overall, Black patients demonstrated significantly better EFS following treatment with clofarabine and cytarabine compared with the low-dose cytarabine triple therapy (hazard ratio, 0.17; P = .01). After adjusting for cofounders, clofarabine and cytarabine induction was the best treatment for Black patients with low ACS10 scores (HR for EFS, 0.2).

“Our results suggest that pharmacogenomics differences between Black and White patients should be considered when tailoring induction regimens to improve outcomes of Black patients and bridge the racial disparity gap in AML treatment,” the researchers concluded.

In developing countries, especially in Africa, starting patients on high-dose cytarabine, daunorubicin, and etoposide can lead to better results “without increasing much of the economic burden” since this treatment is the cheapest, Dr. Lamba said. “At the same time, if the patients have high ACS10 score, you can reduce their economic burden by giving them standard dose” cytarabine, daunorubicin, and etoposide and achieve similar results.

No study funding was reported. Dr. Lamba reported no relevant financial relationships, and three other authors reported various disclosures. Disclosures for Dr. Dunbar were unavailable.. 
 

A version of this article appeared on Medscape.com.

PARIS — Although metabolic liver diseases are mainly seen in patients with obesity or type 2 diabetes, studies have shown that non-alcoholic fatty liver disease, recently renamed metabolic dysfunction-associated steatotic liver disease (MASLD), also affects slim patients. Moreover, the condition could be particularly severe in this population. 

A recent study carried out using data from the French Constance cohort showed that of the 25,753 patients with MASLD, 16.3% were lean (BMI of less than 25 kg/m²). In addition, 50% of these patients had no metabolic risk factors. 

These slim patients with MASLD were most often young patients, for the most part female, and less likely to present with symptoms of metabolic syndrome. Asian patients were overrepresented in this group. 

“These patients probably have genetic and/or environmental risk factors,” commented senior author Lawrence Serfaty, MD, PhD, head of the metabolic liver unit at the new Strasbourg public hospital, during a press conference at the Paris NASH meeting. 

The disease was more severe in slim subjects. Overall, 3.6% of the slim subjects had advanced fibrosis (Forns index > 6.9) vs 1.7% of patients with overweight or obesity (P < .001), regardless of demographic variables, metabolic risk factors, and lifestyle. They also had higher alanine aminotransferase levels. 

In addition, over the course of a mean follow-up of 3.8 years, liver events (eg, cirrhosis, decompensated cirrhosis, and liver cancer), chronic kidney diseases, and all-cause mortality were much more common in these patients than in patients with overweight or obesity (adjusted hazard ratios of 5.84, 2.49, and 3.01, respectively). It should be noted that these clinical results were linked to fibrosis severity in both slim and overweight subjects with MASLD. 

Nonetheless, cardiovascular events remained more common in patients with overweight or obesity, suggesting that obesity itself is a major risk factor for cardiovascular diseases, regardless of MASLD. 

“Armed with these results, which confirm those obtained from other studies, we must seek to understand the pathogenesis of the disease in slim patients and study the role of the microbiota, genetics, and diet, as well as determining the effects of alcohol and tobacco, consumption of which was slightly more common in this subpopulation,” said Dr. Serfaty. 

According to the study authors, sarcopenia and bile acids could also be involved in the pathogenesis of MASLD in slim patients. The researchers concluded that “due to the relatively low rate of MASLD in slim subjects, screening should target patients presenting with metabolic anomalies and/or unexplained cytolysis.”
 

This article was translated from the Medscape French edition.

PARIS — Although metabolic liver diseases are mainly seen in patients with obesity or type 2 diabetes, studies have shown that non-alcoholic fatty liver disease, recently renamed metabolic dysfunction-associated steatotic liver disease (MASLD), also affects slim patients. Moreover, the condition could be particularly severe in this population. 

A recent study carried out using data from the French Constance cohort showed that of the 25,753 patients with MASLD, 16.3% were lean (BMI of less than 25 kg/m²). In addition, 50% of these patients had no metabolic risk factors. 

These slim patients with MASLD were most often young patients, for the most part female, and less likely to present with symptoms of metabolic syndrome. Asian patients were overrepresented in this group. 

“These patients probably have genetic and/or environmental risk factors,” commented senior author Lawrence Serfaty, MD, PhD, head of the metabolic liver unit at the new Strasbourg public hospital, during a press conference at the Paris NASH meeting. 

The disease was more severe in slim subjects. Overall, 3.6% of the slim subjects had advanced fibrosis (Forns index > 6.9) vs 1.7% of patients with overweight or obesity (P < .001), regardless of demographic variables, metabolic risk factors, and lifestyle. They also had higher alanine aminotransferase levels. 

In addition, over the course of a mean follow-up of 3.8 years, liver events (eg, cirrhosis, decompensated cirrhosis, and liver cancer), chronic kidney diseases, and all-cause mortality were much more common in these patients than in patients with overweight or obesity (adjusted hazard ratios of 5.84, 2.49, and 3.01, respectively). It should be noted that these clinical results were linked to fibrosis severity in both slim and overweight subjects with MASLD. 

Nonetheless, cardiovascular events remained more common in patients with overweight or obesity, suggesting that obesity itself is a major risk factor for cardiovascular diseases, regardless of MASLD. 

“Armed with these results, which confirm those obtained from other studies, we must seek to understand the pathogenesis of the disease in slim patients and study the role of the microbiota, genetics, and diet, as well as determining the effects of alcohol and tobacco, consumption of which was slightly more common in this subpopulation,” said Dr. Serfaty. 

According to the study authors, sarcopenia and bile acids could also be involved in the pathogenesis of MASLD in slim patients. The researchers concluded that “due to the relatively low rate of MASLD in slim subjects, screening should target patients presenting with metabolic anomalies and/or unexplained cytolysis.”
 

This article was translated from the Medscape French edition.

PARIS — Although metabolic liver diseases are mainly seen in patients with obesity or type 2 diabetes, studies have shown that non-alcoholic fatty liver disease, recently renamed metabolic dysfunction-associated steatotic liver disease (MASLD), also affects slim patients. Moreover, the condition could be particularly severe in this population. 

A recent study carried out using data from the French Constance cohort showed that of the 25,753 patients with MASLD, 16.3% were lean (BMI of less than 25 kg/m²). In addition, 50% of these patients had no metabolic risk factors. 

These slim patients with MASLD were most often young patients, for the most part female, and less likely to present with symptoms of metabolic syndrome. Asian patients were overrepresented in this group. 

“These patients probably have genetic and/or environmental risk factors,” commented senior author Lawrence Serfaty, MD, PhD, head of the metabolic liver unit at the new Strasbourg public hospital, during a press conference at the Paris NASH meeting. 

The disease was more severe in slim subjects. Overall, 3.6% of the slim subjects had advanced fibrosis (Forns index > 6.9) vs 1.7% of patients with overweight or obesity (P < .001), regardless of demographic variables, metabolic risk factors, and lifestyle. They also had higher alanine aminotransferase levels. 

In addition, over the course of a mean follow-up of 3.8 years, liver events (eg, cirrhosis, decompensated cirrhosis, and liver cancer), chronic kidney diseases, and all-cause mortality were much more common in these patients than in patients with overweight or obesity (adjusted hazard ratios of 5.84, 2.49, and 3.01, respectively). It should be noted that these clinical results were linked to fibrosis severity in both slim and overweight subjects with MASLD. 

Nonetheless, cardiovascular events remained more common in patients with overweight or obesity, suggesting that obesity itself is a major risk factor for cardiovascular diseases, regardless of MASLD. 

“Armed with these results, which confirm those obtained from other studies, we must seek to understand the pathogenesis of the disease in slim patients and study the role of the microbiota, genetics, and diet, as well as determining the effects of alcohol and tobacco, consumption of which was slightly more common in this subpopulation,” said Dr. Serfaty. 

According to the study authors, sarcopenia and bile acids could also be involved in the pathogenesis of MASLD in slim patients. The researchers concluded that “due to the relatively low rate of MASLD in slim subjects, screening should target patients presenting with metabolic anomalies and/or unexplained cytolysis.”
 

This article was translated from the Medscape French edition.

Blood-based liquid biopsy tests for colorectal cancer (CRC) are in development and may soon hit the market, expanding potential options for patients who refuse traditional colonoscopy. But would they be a cost-effective screening tool?

Not according to an economic analysis by researchers at Columbia University Irving Medical Center in New York.

There is “intense research and patient and public interest” in blood-based cancer tests.

“However, liquid biopsy tests may not have sufficient performance and cost too much for them to be a viable strategy at this time,” corresponding author Chin Hur, MD, MPH, told this news organization.

The study was published online on November 16, 2023 in JAMA Network Open..
 

Better, Cheaper Liquid Biopsies Needed

The researchers developed a Markov model to compare the cost effectiveness of no screening and five CRC screening strategies: colonoscopy, liquid biopsy, liquid biopsy after nonadherence to colonoscopy, stool DNA, and fecal immunochemical test (FIT).

The model simulated a hypothetical cohort of unscreened adults at average risk for CRC with screening starting at age 45 years, in line with current US Preventive Services Task Force advice.

A strategy was considered cost-effective if it had an incremental cost-effectiveness ratio (ICER) below the US willingness-to-pay threshold of $100,000 per life-year gained.

According to their model, colonoscopy was the preferred (most cost-effective) strategy with an ICER of $28,071 per life-year gained.

Offering liquid biopsy screening to adults who refuse colonoscopy was the most effective strategy in terms of number of life-years gained, but it greatly exceeded the accepted threshold of $100,000 per life-year gained coming in at $377,538 per life-year gained. The cost of liquid biopsy would have to drop by 66% for this approach to become a cost-effective option, the researchers write.

Compared with no screening, the cost of liquid biopsy would have to fall by 94% for its ICER to drop below the willingness-to-pay threshold of $100,000 per life-year gained. When compared with stool-based tests, the cost of liquid biopsy would have to drop by 43%-80% to be cost-effective.

Liquid biopsy and the liquid biopsy after refusal of colonoscopy strategies had more life-years gained when polyp detection was introduced, but they did not achieve cost-effectiveness at liquid biopsy’s current price even with perfect performance.

“With current estimate of performance and cost,” liquid biopsy for CRC screening is not cost-effective, Dr. Hur told this news organization.

Liquid biopsy tests for CRC screening may become cost-effective in the future if they are significantly less expensive or if polyp detection is introduced along with a decrease in cost, Dr. Hur said.

Making blood-based CRC screening more effective and cost-effective “is more likely to depend on the ability to detect precancerous polyps than on the detection of CRC itself,” writes John Inadomi, MD, with University of Utah School of Medicine, Salt Lake City, in an invited commentary, also published online in JAMA Network Open. 

The sensitivity of FIT for detecting advanced polyps is roughly 20%, whereas stool multitarget tests for blood and DNA or RNA detect 40%-45% of advanced polyps, Dr. Inadomi notes.

Blood-based tests, on the other hand, have been reported to detect 12%-16% of advanced polyps, “which is close to probability of a false-positive test,” he writes. “Because of their high cost, blood-based CRC screening will not be cost-effective unless they detect a greater proportion of advanced polyps than FIT.”

The need for follow-up colonoscopy in the case of a positive noncolonoscopy CRC screening test result is “an important concept that is not emphasized enough in clinical practice,” Dr. Inadomi adds. “Unfortunately, only 40% to 80% of people with positive noncolonoscopy screening test results follow-up with the requisite colonoscopy. Clinicians need to emphasize the necessity of the follow-up colonoscopy when discussing CRC screening options and adherence.”

Dr. Hur reported receiving consulting fees from Value Analytics outside the submitted work. Dr. Hur and co-authors Fay Kastrinos, MD, MPH, and Sheila Rustgi, MD, are supported by a grant from the National Cancer Institute. Co-author William Grady, MD, reported receiving personal fees from SEngine, Guardant Health, Freenome, Diacarta, Natera, Helio, Guidepoint, and GLG and nonfinancial support from LucidDx outside the submitted work. Grady also disclosed a patent pending for methylated gene biomarker for esophageal cancer. Co-author Yoanna Pumpalova, MD, reported receiving stock from Pfizer outside the submitted work. Inadomi reported receiving grants from Exact Sciences. 

A version of this article appeared on Medscape.com.

Blood-based liquid biopsy tests for colorectal cancer (CRC) are in development and may soon hit the market, expanding potential options for patients who refuse traditional colonoscopy. But would they be a cost-effective screening tool?

Not according to an economic analysis by researchers at Columbia University Irving Medical Center in New York.

There is “intense research and patient and public interest” in blood-based cancer tests.

“However, liquid biopsy tests may not have sufficient performance and cost too much for them to be a viable strategy at this time,” corresponding author Chin Hur, MD, MPH, told this news organization.

The study was published online on November 16, 2023 in JAMA Network Open..
 

Better, Cheaper Liquid Biopsies Needed

The researchers developed a Markov model to compare the cost effectiveness of no screening and five CRC screening strategies: colonoscopy, liquid biopsy, liquid biopsy after nonadherence to colonoscopy, stool DNA, and fecal immunochemical test (FIT).

The model simulated a hypothetical cohort of unscreened adults at average risk for CRC with screening starting at age 45 years, in line with current US Preventive Services Task Force advice.

A strategy was considered cost-effective if it had an incremental cost-effectiveness ratio (ICER) below the US willingness-to-pay threshold of $100,000 per life-year gained.

According to their model, colonoscopy was the preferred (most cost-effective) strategy with an ICER of $28,071 per life-year gained.

Offering liquid biopsy screening to adults who refuse colonoscopy was the most effective strategy in terms of number of life-years gained, but it greatly exceeded the accepted threshold of $100,000 per life-year gained coming in at $377,538 per life-year gained. The cost of liquid biopsy would have to drop by 66% for this approach to become a cost-effective option, the researchers write.

Compared with no screening, the cost of liquid biopsy would have to fall by 94% for its ICER to drop below the willingness-to-pay threshold of $100,000 per life-year gained. When compared with stool-based tests, the cost of liquid biopsy would have to drop by 43%-80% to be cost-effective.

Liquid biopsy and the liquid biopsy after refusal of colonoscopy strategies had more life-years gained when polyp detection was introduced, but they did not achieve cost-effectiveness at liquid biopsy’s current price even with perfect performance.

“With current estimate of performance and cost,” liquid biopsy for CRC screening is not cost-effective, Dr. Hur told this news organization.

Liquid biopsy tests for CRC screening may become cost-effective in the future if they are significantly less expensive or if polyp detection is introduced along with a decrease in cost, Dr. Hur said.

Making blood-based CRC screening more effective and cost-effective “is more likely to depend on the ability to detect precancerous polyps than on the detection of CRC itself,” writes John Inadomi, MD, with University of Utah School of Medicine, Salt Lake City, in an invited commentary, also published online in JAMA Network Open. 

The sensitivity of FIT for detecting advanced polyps is roughly 20%, whereas stool multitarget tests for blood and DNA or RNA detect 40%-45% of advanced polyps, Dr. Inadomi notes.

Blood-based tests, on the other hand, have been reported to detect 12%-16% of advanced polyps, “which is close to probability of a false-positive test,” he writes. “Because of their high cost, blood-based CRC screening will not be cost-effective unless they detect a greater proportion of advanced polyps than FIT.”

The need for follow-up colonoscopy in the case of a positive noncolonoscopy CRC screening test result is “an important concept that is not emphasized enough in clinical practice,” Dr. Inadomi adds. “Unfortunately, only 40% to 80% of people with positive noncolonoscopy screening test results follow-up with the requisite colonoscopy. Clinicians need to emphasize the necessity of the follow-up colonoscopy when discussing CRC screening options and adherence.”

Dr. Hur reported receiving consulting fees from Value Analytics outside the submitted work. Dr. Hur and co-authors Fay Kastrinos, MD, MPH, and Sheila Rustgi, MD, are supported by a grant from the National Cancer Institute. Co-author William Grady, MD, reported receiving personal fees from SEngine, Guardant Health, Freenome, Diacarta, Natera, Helio, Guidepoint, and GLG and nonfinancial support from LucidDx outside the submitted work. Grady also disclosed a patent pending for methylated gene biomarker for esophageal cancer. Co-author Yoanna Pumpalova, MD, reported receiving stock from Pfizer outside the submitted work. Inadomi reported receiving grants from Exact Sciences. 

A version of this article appeared on Medscape.com.

Blood-based liquid biopsy tests for colorectal cancer (CRC) are in development and may soon hit the market, expanding potential options for patients who refuse traditional colonoscopy. But would they be a cost-effective screening tool?

Not according to an economic analysis by researchers at Columbia University Irving Medical Center in New York.

There is “intense research and patient and public interest” in blood-based cancer tests.

“However, liquid biopsy tests may not have sufficient performance and cost too much for them to be a viable strategy at this time,” corresponding author Chin Hur, MD, MPH, told this news organization.

The study was published online on November 16, 2023 in JAMA Network Open..
 

Better, Cheaper Liquid Biopsies Needed

The researchers developed a Markov model to compare the cost effectiveness of no screening and five CRC screening strategies: colonoscopy, liquid biopsy, liquid biopsy after nonadherence to colonoscopy, stool DNA, and fecal immunochemical test (FIT).

The model simulated a hypothetical cohort of unscreened adults at average risk for CRC with screening starting at age 45 years, in line with current US Preventive Services Task Force advice.

A strategy was considered cost-effective if it had an incremental cost-effectiveness ratio (ICER) below the US willingness-to-pay threshold of $100,000 per life-year gained.

According to their model, colonoscopy was the preferred (most cost-effective) strategy with an ICER of $28,071 per life-year gained.

Offering liquid biopsy screening to adults who refuse colonoscopy was the most effective strategy in terms of number of life-years gained, but it greatly exceeded the accepted threshold of $100,000 per life-year gained coming in at $377,538 per life-year gained. The cost of liquid biopsy would have to drop by 66% for this approach to become a cost-effective option, the researchers write.

Compared with no screening, the cost of liquid biopsy would have to fall by 94% for its ICER to drop below the willingness-to-pay threshold of $100,000 per life-year gained. When compared with stool-based tests, the cost of liquid biopsy would have to drop by 43%-80% to be cost-effective.

Liquid biopsy and the liquid biopsy after refusal of colonoscopy strategies had more life-years gained when polyp detection was introduced, but they did not achieve cost-effectiveness at liquid biopsy’s current price even with perfect performance.

“With current estimate of performance and cost,” liquid biopsy for CRC screening is not cost-effective, Dr. Hur told this news organization.

Liquid biopsy tests for CRC screening may become cost-effective in the future if they are significantly less expensive or if polyp detection is introduced along with a decrease in cost, Dr. Hur said.

Making blood-based CRC screening more effective and cost-effective “is more likely to depend on the ability to detect precancerous polyps than on the detection of CRC itself,” writes John Inadomi, MD, with University of Utah School of Medicine, Salt Lake City, in an invited commentary, also published online in JAMA Network Open. 

The sensitivity of FIT for detecting advanced polyps is roughly 20%, whereas stool multitarget tests for blood and DNA or RNA detect 40%-45% of advanced polyps, Dr. Inadomi notes.

Blood-based tests, on the other hand, have been reported to detect 12%-16% of advanced polyps, “which is close to probability of a false-positive test,” he writes. “Because of their high cost, blood-based CRC screening will not be cost-effective unless they detect a greater proportion of advanced polyps than FIT.”

The need for follow-up colonoscopy in the case of a positive noncolonoscopy CRC screening test result is “an important concept that is not emphasized enough in clinical practice,” Dr. Inadomi adds. “Unfortunately, only 40% to 80% of people with positive noncolonoscopy screening test results follow-up with the requisite colonoscopy. Clinicians need to emphasize the necessity of the follow-up colonoscopy when discussing CRC screening options and adherence.”

Dr. Hur reported receiving consulting fees from Value Analytics outside the submitted work. Dr. Hur and co-authors Fay Kastrinos, MD, MPH, and Sheila Rustgi, MD, are supported by a grant from the National Cancer Institute. Co-author William Grady, MD, reported receiving personal fees from SEngine, Guardant Health, Freenome, Diacarta, Natera, Helio, Guidepoint, and GLG and nonfinancial support from LucidDx outside the submitted work. Grady also disclosed a patent pending for methylated gene biomarker for esophageal cancer. Co-author Yoanna Pumpalova, MD, reported receiving stock from Pfizer outside the submitted work. Inadomi reported receiving grants from Exact Sciences. 

A version of this article appeared on Medscape.com.

LEIPZIG, GERMANY — For patients newly diagnosed with type 2 diabetes, nutrition therapy is highly effective at achieving remission. “The greater the reduction in body weight, the higher the chances that blood sugar levels will normalize,” Diana Rubin, MD, said at the fall press conference of the German Diabetes Society (DDG). Dr. Rubin is conference president and chief physician of the Center for Nutritional Medicine and Diabetology at Vivantes Humboldt Hospital and the Spandau Hospital, Berlin, Germany. 

Because of the development of modern medicines, nutrition therapy has increasingly been pushed into the background over the past 50 years. However, nutrition therapy and weight reduction can effectively delay diabetes for years, said Dr. Rubin. The patients are healthy, without being healed. 

Nevertheless, the remission is rarely permanent. Most of the patients develop type 2 diabetes again after 5 years. 

Personalized Nutrition Therapy

It is not just developments in medicine that have pushed nutrition therapy into the background. Another contributing factor is that statutory health insurance companies do not cover personalized nutrition counseling as standard, said Dr. Rubin. 

Modern research in nutrition therapy has shown that patients with diabetes should receive personalized treatment. However, this idea is not taken into consideration in current diabetes training programs, which are the only forms of nutrition therapy covered by statutory health insurance companies. 

Instead, nutrition information is mostly conveyed through group training sessions. Individuals do not necessarily find each other again. What’s more, these sessions are seldom led by nutrition experts. “It is rarely helpful to use a ‘one size fits all’ approach, as is often the case with these group training sessions,” said Dr. Rubin. 

The DiRECT study, in which patients reduced their weight by 15 kg and achieved remission rates of almost 90%, is an example of how nutrition therapy can be highly effective. This is especially true if the aims and methods are determined on an individual basis and if there is frequent contact with a therapist. German and international guidelines, including the DDG’s best practice guides from 2022, highlight the importance of personalized nutrition therapy. 

Telemedicine Encourages Adherence

“It is very important to consider the current living situation of the person concerned,” said Dr. Rubin. “It is important to set small objectives that can also be implemented in everyday life.” This can only succeed with a professional face-to-face consultation. “Achieving this objective then also becomes realistic — i.e., losing 10% to 15% body weight and maintaining this loss,” she said. “Long-term monitoring is needed to maintain this weight.” 

Weight reduction methods should generally be determined according to the preferences of the person concerned, since dietary habits and environments are personal. For example, reducing the intake of carbohydrates and fats, intermittent fasting, or using meal replacement drinks can all be considered. 

New data also show that digital apps available on prescription (DiGA) can be helpful for support; this idea is reflected in the DDG’s nutrition best practice guides for patients with type 2 diabetes. 

“Studies show that adherence is highly dependent on the amount of contact with therapists and the long-term nature of the treatment,” said Dr. Rubin. She referred to the need for long-term monitoring, during which the patient can be repeatedly reminded of the therapeutic objective. “In this respect, I see a lot of potential in digital apps, and also in telemedicine, to cater to the short-term contact with the person concerned.” 

A 2015 meta-analysis of 92 studies revealed a significant reduction in A1c for patients with type 1 or type 2 diabetes when using telemedicine nutrition therapy. Dr. Rubin frequently prescribes DiGAs, which are approved for obesity, “simply because I can recognize it makes it easier for many patients to stick to their goals.” 

Dr. Rubin also recommends connecting with sport groups and self-help groups. “Maintaining the weight is a long-term project.” 

Abdominal Fat Decisive

Prediabetes is a precursor to type 2 diabetes and entails an increased risk of heart attack, kidney and eye diseases, and various kinds of cancer. To date, physicians have tried to delay the onset of type 2 diabetes by aiming to reduce the weight of patients with prediabetes. However, scientists at the German Center for Diabetes Research showed with the Prediabetes Lifestyle Intervention Study that abdominal fat plays an important role in the remission of prediabetes. 

The 1-year program with a healthy diet and increased physical activity was followed by 1105 patients with prediabetes. When every subject lost at least 5% of their weight, it turned out that some achieved remission, and others did not. 

People who achieved remission exhibited better insulin sensitivity and had lost more visceral abdominal fat. Visceral abdominal fat can influence insulin sensitivity, not least by an inflammatory reaction in the fatty tissue. 

Reducing visceral abdominal fat is clearly crucially important in achieving prediabetes remission. Subjects who achieved remission in the study had a strongly reduced risk for type 2 diabetes for up to 2 years after the end of the program. They had improved kidney function, and their blood vessels were in better condition. 

Waist Circumference 

According to the new results, the chances of remission increase if body weight is reduced by 5% and waist circumference is reduced by around 4 cm in women and 7 cm in men. 

“Based on the new data, remission should be the new therapeutic objective in people with prediabetes. This could potentially change clinical practice and minimize the complication rate for our patients, both male and female,” said author Reiner Jumpertz-von Schwartzenberg, MD, a researcher at the Tübingen University Hospital in Germany. 

Prediabetes remission can be assumed if the fasting blood glucose falls below 100 mg/dL (5.6 mmol/L), the 2-hour glucose below 140 mg/dL (7.8 mmol/L), and the A1c value below 5.7%. From the new findings, it can be seen that the chances of remission increase the more the body weight decreases.  

Dr. Jumpertz-von Schwartzenberg and his colleagues want to investigate whether this strategy is cost-effective so that the support of payers can also be ensured. 
 

This article was translated from the Medscape German edition.

A version of this article appeared on Medscape.com.

LEIPZIG, GERMANY — For patients newly diagnosed with type 2 diabetes, nutrition therapy is highly effective at achieving remission. “The greater the reduction in body weight, the higher the chances that blood sugar levels will normalize,” Diana Rubin, MD, said at the fall press conference of the German Diabetes Society (DDG). Dr. Rubin is conference president and chief physician of the Center for Nutritional Medicine and Diabetology at Vivantes Humboldt Hospital and the Spandau Hospital, Berlin, Germany. 

Because of the development of modern medicines, nutrition therapy has increasingly been pushed into the background over the past 50 years. However, nutrition therapy and weight reduction can effectively delay diabetes for years, said Dr. Rubin. The patients are healthy, without being healed. 

Nevertheless, the remission is rarely permanent. Most of the patients develop type 2 diabetes again after 5 years. 

Personalized Nutrition Therapy

It is not just developments in medicine that have pushed nutrition therapy into the background. Another contributing factor is that statutory health insurance companies do not cover personalized nutrition counseling as standard, said Dr. Rubin. 

Modern research in nutrition therapy has shown that patients with diabetes should receive personalized treatment. However, this idea is not taken into consideration in current diabetes training programs, which are the only forms of nutrition therapy covered by statutory health insurance companies. 

Instead, nutrition information is mostly conveyed through group training sessions. Individuals do not necessarily find each other again. What’s more, these sessions are seldom led by nutrition experts. “It is rarely helpful to use a ‘one size fits all’ approach, as is often the case with these group training sessions,” said Dr. Rubin. 

The DiRECT study, in which patients reduced their weight by 15 kg and achieved remission rates of almost 90%, is an example of how nutrition therapy can be highly effective. This is especially true if the aims and methods are determined on an individual basis and if there is frequent contact with a therapist. German and international guidelines, including the DDG’s best practice guides from 2022, highlight the importance of personalized nutrition therapy. 

Telemedicine Encourages Adherence

“It is very important to consider the current living situation of the person concerned,” said Dr. Rubin. “It is important to set small objectives that can also be implemented in everyday life.” This can only succeed with a professional face-to-face consultation. “Achieving this objective then also becomes realistic — i.e., losing 10% to 15% body weight and maintaining this loss,” she said. “Long-term monitoring is needed to maintain this weight.” 

Weight reduction methods should generally be determined according to the preferences of the person concerned, since dietary habits and environments are personal. For example, reducing the intake of carbohydrates and fats, intermittent fasting, or using meal replacement drinks can all be considered. 

New data also show that digital apps available on prescription (DiGA) can be helpful for support; this idea is reflected in the DDG’s nutrition best practice guides for patients with type 2 diabetes. 

“Studies show that adherence is highly dependent on the amount of contact with therapists and the long-term nature of the treatment,” said Dr. Rubin. She referred to the need for long-term monitoring, during which the patient can be repeatedly reminded of the therapeutic objective. “In this respect, I see a lot of potential in digital apps, and also in telemedicine, to cater to the short-term contact with the person concerned.” 

A 2015 meta-analysis of 92 studies revealed a significant reduction in A1c for patients with type 1 or type 2 diabetes when using telemedicine nutrition therapy. Dr. Rubin frequently prescribes DiGAs, which are approved for obesity, “simply because I can recognize it makes it easier for many patients to stick to their goals.” 

Dr. Rubin also recommends connecting with sport groups and self-help groups. “Maintaining the weight is a long-term project.” 

Abdominal Fat Decisive

Prediabetes is a precursor to type 2 diabetes and entails an increased risk of heart attack, kidney and eye diseases, and various kinds of cancer. To date, physicians have tried to delay the onset of type 2 diabetes by aiming to reduce the weight of patients with prediabetes. However, scientists at the German Center for Diabetes Research showed with the Prediabetes Lifestyle Intervention Study that abdominal fat plays an important role in the remission of prediabetes. 

The 1-year program with a healthy diet and increased physical activity was followed by 1105 patients with prediabetes. When every subject lost at least 5% of their weight, it turned out that some achieved remission, and others did not. 

People who achieved remission exhibited better insulin sensitivity and had lost more visceral abdominal fat. Visceral abdominal fat can influence insulin sensitivity, not least by an inflammatory reaction in the fatty tissue. 

Reducing visceral abdominal fat is clearly crucially important in achieving prediabetes remission. Subjects who achieved remission in the study had a strongly reduced risk for type 2 diabetes for up to 2 years after the end of the program. They had improved kidney function, and their blood vessels were in better condition. 

Waist Circumference 

According to the new results, the chances of remission increase if body weight is reduced by 5% and waist circumference is reduced by around 4 cm in women and 7 cm in men. 

“Based on the new data, remission should be the new therapeutic objective in people with prediabetes. This could potentially change clinical practice and minimize the complication rate for our patients, both male and female,” said author Reiner Jumpertz-von Schwartzenberg, MD, a researcher at the Tübingen University Hospital in Germany. 

Prediabetes remission can be assumed if the fasting blood glucose falls below 100 mg/dL (5.6 mmol/L), the 2-hour glucose below 140 mg/dL (7.8 mmol/L), and the A1c value below 5.7%. From the new findings, it can be seen that the chances of remission increase the more the body weight decreases.  

Dr. Jumpertz-von Schwartzenberg and his colleagues want to investigate whether this strategy is cost-effective so that the support of payers can also be ensured. 
 

This article was translated from the Medscape German edition.

A version of this article appeared on Medscape.com.

LEIPZIG, GERMANY — For patients newly diagnosed with type 2 diabetes, nutrition therapy is highly effective at achieving remission. “The greater the reduction in body weight, the higher the chances that blood sugar levels will normalize,” Diana Rubin, MD, said at the fall press conference of the German Diabetes Society (DDG). Dr. Rubin is conference president and chief physician of the Center for Nutritional Medicine and Diabetology at Vivantes Humboldt Hospital and the Spandau Hospital, Berlin, Germany. 

Because of the development of modern medicines, nutrition therapy has increasingly been pushed into the background over the past 50 years. However, nutrition therapy and weight reduction can effectively delay diabetes for years, said Dr. Rubin. The patients are healthy, without being healed. 

Nevertheless, the remission is rarely permanent. Most of the patients develop type 2 diabetes again after 5 years. 

Personalized Nutrition Therapy

It is not just developments in medicine that have pushed nutrition therapy into the background. Another contributing factor is that statutory health insurance companies do not cover personalized nutrition counseling as standard, said Dr. Rubin. 

Modern research in nutrition therapy has shown that patients with diabetes should receive personalized treatment. However, this idea is not taken into consideration in current diabetes training programs, which are the only forms of nutrition therapy covered by statutory health insurance companies. 

Instead, nutrition information is mostly conveyed through group training sessions. Individuals do not necessarily find each other again. What’s more, these sessions are seldom led by nutrition experts. “It is rarely helpful to use a ‘one size fits all’ approach, as is often the case with these group training sessions,” said Dr. Rubin. 

The DiRECT study, in which patients reduced their weight by 15 kg and achieved remission rates of almost 90%, is an example of how nutrition therapy can be highly effective. This is especially true if the aims and methods are determined on an individual basis and if there is frequent contact with a therapist. German and international guidelines, including the DDG’s best practice guides from 2022, highlight the importance of personalized nutrition therapy. 

Telemedicine Encourages Adherence

“It is very important to consider the current living situation of the person concerned,” said Dr. Rubin. “It is important to set small objectives that can also be implemented in everyday life.” This can only succeed with a professional face-to-face consultation. “Achieving this objective then also becomes realistic — i.e., losing 10% to 15% body weight and maintaining this loss,” she said. “Long-term monitoring is needed to maintain this weight.” 

Weight reduction methods should generally be determined according to the preferences of the person concerned, since dietary habits and environments are personal. For example, reducing the intake of carbohydrates and fats, intermittent fasting, or using meal replacement drinks can all be considered. 

New data also show that digital apps available on prescription (DiGA) can be helpful for support; this idea is reflected in the DDG’s nutrition best practice guides for patients with type 2 diabetes. 

“Studies show that adherence is highly dependent on the amount of contact with therapists and the long-term nature of the treatment,” said Dr. Rubin. She referred to the need for long-term monitoring, during which the patient can be repeatedly reminded of the therapeutic objective. “In this respect, I see a lot of potential in digital apps, and also in telemedicine, to cater to the short-term contact with the person concerned.” 

A 2015 meta-analysis of 92 studies revealed a significant reduction in A1c for patients with type 1 or type 2 diabetes when using telemedicine nutrition therapy. Dr. Rubin frequently prescribes DiGAs, which are approved for obesity, “simply because I can recognize it makes it easier for many patients to stick to their goals.” 

Dr. Rubin also recommends connecting with sport groups and self-help groups. “Maintaining the weight is a long-term project.” 

Abdominal Fat Decisive

Prediabetes is a precursor to type 2 diabetes and entails an increased risk of heart attack, kidney and eye diseases, and various kinds of cancer. To date, physicians have tried to delay the onset of type 2 diabetes by aiming to reduce the weight of patients with prediabetes. However, scientists at the German Center for Diabetes Research showed with the Prediabetes Lifestyle Intervention Study that abdominal fat plays an important role in the remission of prediabetes. 

The 1-year program with a healthy diet and increased physical activity was followed by 1105 patients with prediabetes. When every subject lost at least 5% of their weight, it turned out that some achieved remission, and others did not. 

People who achieved remission exhibited better insulin sensitivity and had lost more visceral abdominal fat. Visceral abdominal fat can influence insulin sensitivity, not least by an inflammatory reaction in the fatty tissue. 

Reducing visceral abdominal fat is clearly crucially important in achieving prediabetes remission. Subjects who achieved remission in the study had a strongly reduced risk for type 2 diabetes for up to 2 years after the end of the program. They had improved kidney function, and their blood vessels were in better condition. 

Waist Circumference 

According to the new results, the chances of remission increase if body weight is reduced by 5% and waist circumference is reduced by around 4 cm in women and 7 cm in men. 

“Based on the new data, remission should be the new therapeutic objective in people with prediabetes. This could potentially change clinical practice and minimize the complication rate for our patients, both male and female,” said author Reiner Jumpertz-von Schwartzenberg, MD, a researcher at the Tübingen University Hospital in Germany. 

Prediabetes remission can be assumed if the fasting blood glucose falls below 100 mg/dL (5.6 mmol/L), the 2-hour glucose below 140 mg/dL (7.8 mmol/L), and the A1c value below 5.7%. From the new findings, it can be seen that the chances of remission increase the more the body weight decreases.  

Dr. Jumpertz-von Schwartzenberg and his colleagues want to investigate whether this strategy is cost-effective so that the support of payers can also be ensured. 
 

This article was translated from the Medscape German edition.

A version of this article appeared on Medscape.com.

Artificial intelligence (AI) is already impacting the mental health care space, with several new tools available to both clinicians and patients. While this technology could be a game-changer amid a mental health crisis and clinician shortage, there are important ethical and efficacy concerns clinicians should be aware of.

Lifestance Health

Current use cases illustrate both the potential and risks of AI. On one hand, AI has the potential to improve patient care with tools that can support diagnoses and inform treatment decisions at scale. The UK’s National Health Service is using an AI-powered diagnostic tool to help clinicians diagnose mental health disorders and determine the severity of a patient’s needs. Other tools leverage AI to analyze a patient’s voice for signs of depression or anxiety.

On the other hand, there are serious potential risks involving privacy, bias, and misinformation. One chatbot tool designed to counsel patients through disordered eating was shut down after giving problematic weight-loss advice.

The number of AI tools in the healthcare space is expected to increase fivefold by 2035. Keeping up with these advances is just as important for clinicians as keeping up with the latest medication and treatment options. That means being aware of both the limitations and the potential of AI. Here are three questions clinicians can ask as they explore ways to integrate these tools into their practice while navigating the risks.
 

• How can AI augment, not replace, the work of my staff?

AI’s biggest potential lies in its ability to augment the work of clinicians, rather than replacing it. Mental health clinicians should evaluate emerging AI tools through this lens.

For example, documentation and the use of electronic health records have consistently been linked to clinician burnout. Using AI to cut down on documentation would leave clinicians with more time and energy to focus on patient care.

One study from the National Library of Medicine found that physicians who did not have enough time to complete documentation were nearly three times more likely to report burnout. In some cases, clinic schedules were deliberately shortened to allow time for documentation.

New tools are emerging that use audio recording, transcription services, and large language models to generate clinical summaries and other documentation support. Amazon and 3M have partnered to solve documentation challenges using AI. This is an area I’ll definitely be keeping an eye on as it develops.
 

• Do I have patient consent to use this tool?

Since most AI tools remain relatively new, there is a gap in the legal and regulatory framework needed to ensure patient privacy and data protection. Clinicians should draw on existing guardrails and best practices to protect patient privacy and prioritize informed consent. The bottom line: Patients need to know how their data will be used and agree to it.

In the example above regarding documentation, a clinician should obtain patient consent before using technology that records or transcribes sessions. This extends to disclosing the use of AI chat tools and other touch points that occur between sessions. One mental health nonprofit has come under fire for using ChatGPT to provide mental health counseling to thousands of patients who weren’t aware the responses were generated by AI.

Beyond disclosing the use of these tools, clinicians should sufficiently explain how they work to ensure patients understand what they’re consenting to. Some technology companies offer guidance on how informed consent applies to their products and even offer template consent forms to support clinicians. Ultimately, accountability for maintaining patient privacy rests with the clinician, not the company behind the AI tool.
 

• Where is there a risk of bias?

There has been much discussion around the issue of bias within large language models in particular, since these programs will inherit any bias from the data points or text used to train them. However, there is often little to no visibility into how these models are trained, the algorithms they rely on, and how efficacy is measured.

This is especially concerning within the mental health care space, where bias can contribute to lower-quality care based on a patient’s race, gender or other characteristics. One systemic review published in JAMA Network Open found that most of the AI models used for psychiatric diagnoses that have been studied had a high overall risk of bias — which can lead to outputs that are misleading or incorrect, which can be dangerous in the healthcare field.

It’s important to keep the risk of bias top-of-mind when exploring AI tools and consider whether a tool would pose any direct harm to patients. Clinicians should have active oversight with any use of AI and, ultimately, consider an AI tool’s outputs alongside their own insights, expertise, and instincts.
 

Clinicians have the power to shape AI’s impact

While there is plenty to be excited about as these new tools develop, clinicians should explore AI with an eye toward the risks as well as the rewards. Practitioners have a significant opportunity to help shape how this technology develops by making informed decisions about which products to invest in and holding tech companies accountable. By educating patients, prioritizing informed consent, and seeking ways to augment their work that ultimately improve quality and scale of care, clinicians can help ensure positive outcomes while minimizing unintended consequences.

Dr. Patel-Dunn is a psychiatrist and chief medical officer at Lifestance Health, Scottsdale, Ariz.

Artificial intelligence (AI) is already impacting the mental health care space, with several new tools available to both clinicians and patients. While this technology could be a game-changer amid a mental health crisis and clinician shortage, there are important ethical and efficacy concerns clinicians should be aware of.

Lifestance Health

Current use cases illustrate both the potential and risks of AI. On one hand, AI has the potential to improve patient care with tools that can support diagnoses and inform treatment decisions at scale. The UK’s National Health Service is using an AI-powered diagnostic tool to help clinicians diagnose mental health disorders and determine the severity of a patient’s needs. Other tools leverage AI to analyze a patient’s voice for signs of depression or anxiety.

On the other hand, there are serious potential risks involving privacy, bias, and misinformation. One chatbot tool designed to counsel patients through disordered eating was shut down after giving problematic weight-loss advice.

The number of AI tools in the healthcare space is expected to increase fivefold by 2035. Keeping up with these advances is just as important for clinicians as keeping up with the latest medication and treatment options. That means being aware of both the limitations and the potential of AI. Here are three questions clinicians can ask as they explore ways to integrate these tools into their practice while navigating the risks.
 

• How can AI augment, not replace, the work of my staff?

AI’s biggest potential lies in its ability to augment the work of clinicians, rather than replacing it. Mental health clinicians should evaluate emerging AI tools through this lens.

For example, documentation and the use of electronic health records have consistently been linked to clinician burnout. Using AI to cut down on documentation would leave clinicians with more time and energy to focus on patient care.

One study from the National Library of Medicine found that physicians who did not have enough time to complete documentation were nearly three times more likely to report burnout. In some cases, clinic schedules were deliberately shortened to allow time for documentation.

New tools are emerging that use audio recording, transcription services, and large language models to generate clinical summaries and other documentation support. Amazon and 3M have partnered to solve documentation challenges using AI. This is an area I’ll definitely be keeping an eye on as it develops.
 

• Do I have patient consent to use this tool?

Since most AI tools remain relatively new, there is a gap in the legal and regulatory framework needed to ensure patient privacy and data protection. Clinicians should draw on existing guardrails and best practices to protect patient privacy and prioritize informed consent. The bottom line: Patients need to know how their data will be used and agree to it.

In the example above regarding documentation, a clinician should obtain patient consent before using technology that records or transcribes sessions. This extends to disclosing the use of AI chat tools and other touch points that occur between sessions. One mental health nonprofit has come under fire for using ChatGPT to provide mental health counseling to thousands of patients who weren’t aware the responses were generated by AI.

Beyond disclosing the use of these tools, clinicians should sufficiently explain how they work to ensure patients understand what they’re consenting to. Some technology companies offer guidance on how informed consent applies to their products and even offer template consent forms to support clinicians. Ultimately, accountability for maintaining patient privacy rests with the clinician, not the company behind the AI tool.
 

• Where is there a risk of bias?

There has been much discussion around the issue of bias within large language models in particular, since these programs will inherit any bias from the data points or text used to train them. However, there is often little to no visibility into how these models are trained, the algorithms they rely on, and how efficacy is measured.

This is especially concerning within the mental health care space, where bias can contribute to lower-quality care based on a patient’s race, gender or other characteristics. One systemic review published in JAMA Network Open found that most of the AI models used for psychiatric diagnoses that have been studied had a high overall risk of bias — which can lead to outputs that are misleading or incorrect, which can be dangerous in the healthcare field.

It’s important to keep the risk of bias top-of-mind when exploring AI tools and consider whether a tool would pose any direct harm to patients. Clinicians should have active oversight with any use of AI and, ultimately, consider an AI tool’s outputs alongside their own insights, expertise, and instincts.
 

Clinicians have the power to shape AI’s impact

While there is plenty to be excited about as these new tools develop, clinicians should explore AI with an eye toward the risks as well as the rewards. Practitioners have a significant opportunity to help shape how this technology develops by making informed decisions about which products to invest in and holding tech companies accountable. By educating patients, prioritizing informed consent, and seeking ways to augment their work that ultimately improve quality and scale of care, clinicians can help ensure positive outcomes while minimizing unintended consequences.

Dr. Patel-Dunn is a psychiatrist and chief medical officer at Lifestance Health, Scottsdale, Ariz.

Artificial intelligence (AI) is already impacting the mental health care space, with several new tools available to both clinicians and patients. While this technology could be a game-changer amid a mental health crisis and clinician shortage, there are important ethical and efficacy concerns clinicians should be aware of.

Lifestance Health

Current use cases illustrate both the potential and risks of AI. On one hand, AI has the potential to improve patient care with tools that can support diagnoses and inform treatment decisions at scale. The UK’s National Health Service is using an AI-powered diagnostic tool to help clinicians diagnose mental health disorders and determine the severity of a patient’s needs. Other tools leverage AI to analyze a patient’s voice for signs of depression or anxiety.

On the other hand, there are serious potential risks involving privacy, bias, and misinformation. One chatbot tool designed to counsel patients through disordered eating was shut down after giving problematic weight-loss advice.

The number of AI tools in the healthcare space is expected to increase fivefold by 2035. Keeping up with these advances is just as important for clinicians as keeping up with the latest medication and treatment options. That means being aware of both the limitations and the potential of AI. Here are three questions clinicians can ask as they explore ways to integrate these tools into their practice while navigating the risks.
 

• How can AI augment, not replace, the work of my staff?

AI’s biggest potential lies in its ability to augment the work of clinicians, rather than replacing it. Mental health clinicians should evaluate emerging AI tools through this lens.

For example, documentation and the use of electronic health records have consistently been linked to clinician burnout. Using AI to cut down on documentation would leave clinicians with more time and energy to focus on patient care.

One study from the National Library of Medicine found that physicians who did not have enough time to complete documentation were nearly three times more likely to report burnout. In some cases, clinic schedules were deliberately shortened to allow time for documentation.

New tools are emerging that use audio recording, transcription services, and large language models to generate clinical summaries and other documentation support. Amazon and 3M have partnered to solve documentation challenges using AI. This is an area I’ll definitely be keeping an eye on as it develops.
 

• Do I have patient consent to use this tool?

Since most AI tools remain relatively new, there is a gap in the legal and regulatory framework needed to ensure patient privacy and data protection. Clinicians should draw on existing guardrails and best practices to protect patient privacy and prioritize informed consent. The bottom line: Patients need to know how their data will be used and agree to it.

In the example above regarding documentation, a clinician should obtain patient consent before using technology that records or transcribes sessions. This extends to disclosing the use of AI chat tools and other touch points that occur between sessions. One mental health nonprofit has come under fire for using ChatGPT to provide mental health counseling to thousands of patients who weren’t aware the responses were generated by AI.

Beyond disclosing the use of these tools, clinicians should sufficiently explain how they work to ensure patients understand what they’re consenting to. Some technology companies offer guidance on how informed consent applies to their products and even offer template consent forms to support clinicians. Ultimately, accountability for maintaining patient privacy rests with the clinician, not the company behind the AI tool.
 

• Where is there a risk of bias?

There has been much discussion around the issue of bias within large language models in particular, since these programs will inherit any bias from the data points or text used to train them. However, there is often little to no visibility into how these models are trained, the algorithms they rely on, and how efficacy is measured.

This is especially concerning within the mental health care space, where bias can contribute to lower-quality care based on a patient’s race, gender or other characteristics. One systemic review published in JAMA Network Open found that most of the AI models used for psychiatric diagnoses that have been studied had a high overall risk of bias — which can lead to outputs that are misleading or incorrect, which can be dangerous in the healthcare field.

It’s important to keep the risk of bias top-of-mind when exploring AI tools and consider whether a tool would pose any direct harm to patients. Clinicians should have active oversight with any use of AI and, ultimately, consider an AI tool’s outputs alongside their own insights, expertise, and instincts.
 

Clinicians have the power to shape AI’s impact

While there is plenty to be excited about as these new tools develop, clinicians should explore AI with an eye toward the risks as well as the rewards. Practitioners have a significant opportunity to help shape how this technology develops by making informed decisions about which products to invest in and holding tech companies accountable. By educating patients, prioritizing informed consent, and seeking ways to augment their work that ultimately improve quality and scale of care, clinicians can help ensure positive outcomes while minimizing unintended consequences.

Dr. Patel-Dunn is a psychiatrist and chief medical officer at Lifestance Health, Scottsdale, Ariz.

Patients with relapsed diffuse large B cell lymphoma (DLBCL) who achieve a complete remission from interim chemotherapy while awaiting secondary chimeric antigen receptor (CAR) T cell therapy show significantly better outcomes if they then receive conventional autologous stem cell transplantation (auto-HCT), compared with CAR T therapy.

“In patients with relapsed DLBCL in a complete remission, treatment with auto-HCT is associated with a lower rate of relapse/progression, and a longer progression-free survival [versus CAR T therapy],” said first author Mazyar Shadman, MD, MPH, of the Division of Medical Oncology, University of Washington, Seattle.

“The data support utilization of auto-HCT in patients with relapsed LBCL achieving a complete response,” he said.

The findings were presented at the annual meeting of the American Society of Hematology in San Diego.

While approximately 60% of patients with DLBCL are successfully treated after an initial anthracycline-based and rituximab-containing chemotherapy regimen, those who do not improve have poorer outcomes, and CAR T-cell therapy has emerged as the standard of care for those patients, based on results from the ZUMA-7 and TRANSFORM clinical trials.

But with delays in accessing CAR T quite common, patients will often receive interim chemotherapy while awaiting referral to a CAR T center, and occasionally, usually unexpectedly, some will achieve a partial or complete response.

In previous research involving patients who achieved a partial remission in such interim cases, Dr. Shadman and colleagues demonstrated that auto-HCT had favorable outcomes, compared with those who received CAR T therapy.

For the new retrospective, real-world analysis, the authors compared outcomes with the treatment options among 360 patients between the ages of 18 and 75 who were enrolled in the Center for International Blood & Marrow Transplant Research registry and had received auto-HCT or CAR T therapy after achieving a complete remission following salvage chemotherapy.

Of those receiving CAR-T cell therapy, most (53.2%) received tisagenlecleucel (tisa-cel), followed by axicabtagene ciloleucel (axi-cel, 45.6%) and lisocabtagene maraleucel (liso-cel, 1.3%), between 2018 and 2021, while 281 patients were treated with auto-HCT between 2015 and 2021.

With a median follow-up of 49.7 months (range 3.0-94.4) for auto-HCT and 24.7 months (range 3.3-49.4) for CAR-T, a univariate analysis showed the rate of 2-year progression free survival was 66.2% in the auto-HCT group and 47.8% in the CAR T group (P < .001).

The results also favored auto-HCT for 2-year progression/relapse, with a cumulative incidence of 27.8% with auto-HCT versus 48% with CAR T (P < .001), and the 2-year overall survival was higher with auto-HCT (78.9% vs. 65.6%; P = .037).

After adjustment in multivariable analysis adjusting for relevant clinical variables, auto-HCT versus CAR T remained associated with a lower risk of relapse or progression (HR 2.18; P < .0001) and an improved progression-free survival (HR 1.83; P = .0011), with no significant differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

Deaths occurred among 85 patients in the auto-HCT group and 25 in the CAR T cohort, with lymphoma being the main cause of death in both groups (60% and 68%, respectively).

While 37 (13.2%) of auto-HCT patients later received subsequent CAR-T therapy, no patients receiving CAR-T had subsequent auto-HCT.

There were no differences between the CAR-T and auto-HST groups in rates of 2-year treatment-related mortality (4.1% vs. 5.9%; P = .673).

A subanalysis of those who had treatment failure at 12 months, (CAR-T = 57 and auto-HCT = 163) showed that those receiving CAR-T therapy had a higher 2-year relapse rate (46.3% vs. 25%; P < .001); an inferior 2-year progression-free survival rate (48.4% vs. 68.2%; P = .001) compared with auto-HCT, while there were no significant differences between the groups in terms of 2-year overall survival or treatment-related mortality.

After a multivariable analysis adjusting for relevant clinical factors, CAR-T therapy remained associated with higher risk of relapse (HR 2.18; P < .0001) and an inferior progression-free survival (HR 1.83; P = .0011) compared with auto-HCT, with no differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

“These results are consistent with our previously reported findings, indicating higher efficacy of auto-HCT compared with CAR T in patients with partial remission,” Dr. Shadman said.

In addition to the study’s being a retrospective analysis, limitations include that more than half of patients in the CAR T cohort received tisa-cel, which could have lower efficacy compared with other approved CAR T therapies, Dr. Shadman noted.

“A repeat analysis by including more patients treated with axi-cel or liso-cel may address this issue in the future,” he said.

Discussing the results in a press briefing, Dr. Shadman underscored that “there is no question the choice of therapy for these DLBCL patients with primary refractory disease should be second-line CAR T therapy — we are not suggesting that those patients should be sent for auto-HCT,” he said.

“What we are saying is, in real-world practice ... patients may need chemotherapy treatment in the interim (awaiting CAR T treatment), and we don’t expect these patients to respond to those cycles because they have already shown us that they don’t do well with chemotherapy — however some do respond and can go into complete remission.”

The question then becomes whether patients at that point will fare better with CAR T or auto-HCT, and the results indicate that “auto-HCT gives those patients a pretty solid remission that looks better than [that with] CAR T therapy.”

Dr. Shadman noted that the results serve to inform or confirm key clinical practices, including “in patients with late relapses, after 12 months, auto-HCT should remain the standard of care.

“In patients with primary refractory disease or early relapse, CAR T should be the goal of therapy and improving access to CAR T should remain a priority.

“In the subset of patients who achieve a CR with interim treatment, a discussion about the possibility of utilizing auto-HCT seems reasonable and can provide another curative option for some patients while keeping CAR-T as a backup treatment plan in case of auto-HCT failure.”

Commenting on the study, Jonathan W. Friedberg, MD, the Samuel Durand Professor of Medicine and director of the Wilmot Cancer Institute, University of Rochester, New York, said, “these findings confirm utility of auto-HCT in patients who achieve a CR.”

However, “the problem is that only a small fraction of patients achieve CR in this situation, and we do not know who they are going to be at time of relapse,” he told this news organization.

He agreed that “given robust randomized trials showing overall survival benefit of CAR-T compared to auto-HCT in patients with high risk relapsed DLBCL, CAR-T treatment should remain the current standard.

“However, these current results help to confirm the strategy for management of low- risk (late) relapses and indicate that auto-HCT still has a place for these patients if they achieve CR with salvage therapy.”

Dr. Shadman reported relationships with ADC therapeutics, Bristol Myers Squibb, Genmab, Lilly, Vincerx, Kite (Gilead), Janssen, Fate Therapeutics, MorphoSys/Incyte, AstraZeneca, BeiGene, Pharmacyclics, Mustang Bio, AbbVie, Genentech, MEI Pharma, Regeneron, and TG Therapeutics. Dr. Friedberg had no disclosures to report.

Patients with relapsed diffuse large B cell lymphoma (DLBCL) who achieve a complete remission from interim chemotherapy while awaiting secondary chimeric antigen receptor (CAR) T cell therapy show significantly better outcomes if they then receive conventional autologous stem cell transplantation (auto-HCT), compared with CAR T therapy.

“In patients with relapsed DLBCL in a complete remission, treatment with auto-HCT is associated with a lower rate of relapse/progression, and a longer progression-free survival [versus CAR T therapy],” said first author Mazyar Shadman, MD, MPH, of the Division of Medical Oncology, University of Washington, Seattle.

“The data support utilization of auto-HCT in patients with relapsed LBCL achieving a complete response,” he said.

The findings were presented at the annual meeting of the American Society of Hematology in San Diego.

While approximately 60% of patients with DLBCL are successfully treated after an initial anthracycline-based and rituximab-containing chemotherapy regimen, those who do not improve have poorer outcomes, and CAR T-cell therapy has emerged as the standard of care for those patients, based on results from the ZUMA-7 and TRANSFORM clinical trials.

But with delays in accessing CAR T quite common, patients will often receive interim chemotherapy while awaiting referral to a CAR T center, and occasionally, usually unexpectedly, some will achieve a partial or complete response.

In previous research involving patients who achieved a partial remission in such interim cases, Dr. Shadman and colleagues demonstrated that auto-HCT had favorable outcomes, compared with those who received CAR T therapy.

For the new retrospective, real-world analysis, the authors compared outcomes with the treatment options among 360 patients between the ages of 18 and 75 who were enrolled in the Center for International Blood & Marrow Transplant Research registry and had received auto-HCT or CAR T therapy after achieving a complete remission following salvage chemotherapy.

Of those receiving CAR-T cell therapy, most (53.2%) received tisagenlecleucel (tisa-cel), followed by axicabtagene ciloleucel (axi-cel, 45.6%) and lisocabtagene maraleucel (liso-cel, 1.3%), between 2018 and 2021, while 281 patients were treated with auto-HCT between 2015 and 2021.

With a median follow-up of 49.7 months (range 3.0-94.4) for auto-HCT and 24.7 months (range 3.3-49.4) for CAR-T, a univariate analysis showed the rate of 2-year progression free survival was 66.2% in the auto-HCT group and 47.8% in the CAR T group (P < .001).

The results also favored auto-HCT for 2-year progression/relapse, with a cumulative incidence of 27.8% with auto-HCT versus 48% with CAR T (P < .001), and the 2-year overall survival was higher with auto-HCT (78.9% vs. 65.6%; P = .037).

After adjustment in multivariable analysis adjusting for relevant clinical variables, auto-HCT versus CAR T remained associated with a lower risk of relapse or progression (HR 2.18; P < .0001) and an improved progression-free survival (HR 1.83; P = .0011), with no significant differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

Deaths occurred among 85 patients in the auto-HCT group and 25 in the CAR T cohort, with lymphoma being the main cause of death in both groups (60% and 68%, respectively).

While 37 (13.2%) of auto-HCT patients later received subsequent CAR-T therapy, no patients receiving CAR-T had subsequent auto-HCT.

There were no differences between the CAR-T and auto-HST groups in rates of 2-year treatment-related mortality (4.1% vs. 5.9%; P = .673).

A subanalysis of those who had treatment failure at 12 months, (CAR-T = 57 and auto-HCT = 163) showed that those receiving CAR-T therapy had a higher 2-year relapse rate (46.3% vs. 25%; P < .001); an inferior 2-year progression-free survival rate (48.4% vs. 68.2%; P = .001) compared with auto-HCT, while there were no significant differences between the groups in terms of 2-year overall survival or treatment-related mortality.

After a multivariable analysis adjusting for relevant clinical factors, CAR-T therapy remained associated with higher risk of relapse (HR 2.18; P < .0001) and an inferior progression-free survival (HR 1.83; P = .0011) compared with auto-HCT, with no differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

“These results are consistent with our previously reported findings, indicating higher efficacy of auto-HCT compared with CAR T in patients with partial remission,” Dr. Shadman said.

In addition to the study’s being a retrospective analysis, limitations include that more than half of patients in the CAR T cohort received tisa-cel, which could have lower efficacy compared with other approved CAR T therapies, Dr. Shadman noted.

“A repeat analysis by including more patients treated with axi-cel or liso-cel may address this issue in the future,” he said.

Discussing the results in a press briefing, Dr. Shadman underscored that “there is no question the choice of therapy for these DLBCL patients with primary refractory disease should be second-line CAR T therapy — we are not suggesting that those patients should be sent for auto-HCT,” he said.

“What we are saying is, in real-world practice ... patients may need chemotherapy treatment in the interim (awaiting CAR T treatment), and we don’t expect these patients to respond to those cycles because they have already shown us that they don’t do well with chemotherapy — however some do respond and can go into complete remission.”

The question then becomes whether patients at that point will fare better with CAR T or auto-HCT, and the results indicate that “auto-HCT gives those patients a pretty solid remission that looks better than [that with] CAR T therapy.”

Dr. Shadman noted that the results serve to inform or confirm key clinical practices, including “in patients with late relapses, after 12 months, auto-HCT should remain the standard of care.

“In patients with primary refractory disease or early relapse, CAR T should be the goal of therapy and improving access to CAR T should remain a priority.

“In the subset of patients who achieve a CR with interim treatment, a discussion about the possibility of utilizing auto-HCT seems reasonable and can provide another curative option for some patients while keeping CAR-T as a backup treatment plan in case of auto-HCT failure.”

Commenting on the study, Jonathan W. Friedberg, MD, the Samuel Durand Professor of Medicine and director of the Wilmot Cancer Institute, University of Rochester, New York, said, “these findings confirm utility of auto-HCT in patients who achieve a CR.”

However, “the problem is that only a small fraction of patients achieve CR in this situation, and we do not know who they are going to be at time of relapse,” he told this news organization.

He agreed that “given robust randomized trials showing overall survival benefit of CAR-T compared to auto-HCT in patients with high risk relapsed DLBCL, CAR-T treatment should remain the current standard.

“However, these current results help to confirm the strategy for management of low- risk (late) relapses and indicate that auto-HCT still has a place for these patients if they achieve CR with salvage therapy.”

Dr. Shadman reported relationships with ADC therapeutics, Bristol Myers Squibb, Genmab, Lilly, Vincerx, Kite (Gilead), Janssen, Fate Therapeutics, MorphoSys/Incyte, AstraZeneca, BeiGene, Pharmacyclics, Mustang Bio, AbbVie, Genentech, MEI Pharma, Regeneron, and TG Therapeutics. Dr. Friedberg had no disclosures to report.

Patients with relapsed diffuse large B cell lymphoma (DLBCL) who achieve a complete remission from interim chemotherapy while awaiting secondary chimeric antigen receptor (CAR) T cell therapy show significantly better outcomes if they then receive conventional autologous stem cell transplantation (auto-HCT), compared with CAR T therapy.

“In patients with relapsed DLBCL in a complete remission, treatment with auto-HCT is associated with a lower rate of relapse/progression, and a longer progression-free survival [versus CAR T therapy],” said first author Mazyar Shadman, MD, MPH, of the Division of Medical Oncology, University of Washington, Seattle.

“The data support utilization of auto-HCT in patients with relapsed LBCL achieving a complete response,” he said.

The findings were presented at the annual meeting of the American Society of Hematology in San Diego.

While approximately 60% of patients with DLBCL are successfully treated after an initial anthracycline-based and rituximab-containing chemotherapy regimen, those who do not improve have poorer outcomes, and CAR T-cell therapy has emerged as the standard of care for those patients, based on results from the ZUMA-7 and TRANSFORM clinical trials.

But with delays in accessing CAR T quite common, patients will often receive interim chemotherapy while awaiting referral to a CAR T center, and occasionally, usually unexpectedly, some will achieve a partial or complete response.

In previous research involving patients who achieved a partial remission in such interim cases, Dr. Shadman and colleagues demonstrated that auto-HCT had favorable outcomes, compared with those who received CAR T therapy.

For the new retrospective, real-world analysis, the authors compared outcomes with the treatment options among 360 patients between the ages of 18 and 75 who were enrolled in the Center for International Blood & Marrow Transplant Research registry and had received auto-HCT or CAR T therapy after achieving a complete remission following salvage chemotherapy.

Of those receiving CAR-T cell therapy, most (53.2%) received tisagenlecleucel (tisa-cel), followed by axicabtagene ciloleucel (axi-cel, 45.6%) and lisocabtagene maraleucel (liso-cel, 1.3%), between 2018 and 2021, while 281 patients were treated with auto-HCT between 2015 and 2021.

With a median follow-up of 49.7 months (range 3.0-94.4) for auto-HCT and 24.7 months (range 3.3-49.4) for CAR-T, a univariate analysis showed the rate of 2-year progression free survival was 66.2% in the auto-HCT group and 47.8% in the CAR T group (P < .001).

The results also favored auto-HCT for 2-year progression/relapse, with a cumulative incidence of 27.8% with auto-HCT versus 48% with CAR T (P < .001), and the 2-year overall survival was higher with auto-HCT (78.9% vs. 65.6%; P = .037).

After adjustment in multivariable analysis adjusting for relevant clinical variables, auto-HCT versus CAR T remained associated with a lower risk of relapse or progression (HR 2.18; P < .0001) and an improved progression-free survival (HR 1.83; P = .0011), with no significant differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

Deaths occurred among 85 patients in the auto-HCT group and 25 in the CAR T cohort, with lymphoma being the main cause of death in both groups (60% and 68%, respectively).

While 37 (13.2%) of auto-HCT patients later received subsequent CAR-T therapy, no patients receiving CAR-T had subsequent auto-HCT.

There were no differences between the CAR-T and auto-HST groups in rates of 2-year treatment-related mortality (4.1% vs. 5.9%; P = .673).

A subanalysis of those who had treatment failure at 12 months, (CAR-T = 57 and auto-HCT = 163) showed that those receiving CAR-T therapy had a higher 2-year relapse rate (46.3% vs. 25%; P < .001); an inferior 2-year progression-free survival rate (48.4% vs. 68.2%; P = .001) compared with auto-HCT, while there were no significant differences between the groups in terms of 2-year overall survival or treatment-related mortality.

After a multivariable analysis adjusting for relevant clinical factors, CAR-T therapy remained associated with higher risk of relapse (HR 2.18; P < .0001) and an inferior progression-free survival (HR 1.83; P = .0011) compared with auto-HCT, with no differences in the risk of treatment-related mortality (HR 0.59; P = .36) or overall survival (HR 1.44; P = .12).

“These results are consistent with our previously reported findings, indicating higher efficacy of auto-HCT compared with CAR T in patients with partial remission,” Dr. Shadman said.

In addition to the study’s being a retrospective analysis, limitations include that more than half of patients in the CAR T cohort received tisa-cel, which could have lower efficacy compared with other approved CAR T therapies, Dr. Shadman noted.

“A repeat analysis by including more patients treated with axi-cel or liso-cel may address this issue in the future,” he said.

Discussing the results in a press briefing, Dr. Shadman underscored that “there is no question the choice of therapy for these DLBCL patients with primary refractory disease should be second-line CAR T therapy — we are not suggesting that those patients should be sent for auto-HCT,” he said.

“What we are saying is, in real-world practice ... patients may need chemotherapy treatment in the interim (awaiting CAR T treatment), and we don’t expect these patients to respond to those cycles because they have already shown us that they don’t do well with chemotherapy — however some do respond and can go into complete remission.”

The question then becomes whether patients at that point will fare better with CAR T or auto-HCT, and the results indicate that “auto-HCT gives those patients a pretty solid remission that looks better than [that with] CAR T therapy.”

Dr. Shadman noted that the results serve to inform or confirm key clinical practices, including “in patients with late relapses, after 12 months, auto-HCT should remain the standard of care.

“In patients with primary refractory disease or early relapse, CAR T should be the goal of therapy and improving access to CAR T should remain a priority.

“In the subset of patients who achieve a CR with interim treatment, a discussion about the possibility of utilizing auto-HCT seems reasonable and can provide another curative option for some patients while keeping CAR-T as a backup treatment plan in case of auto-HCT failure.”

Commenting on the study, Jonathan W. Friedberg, MD, the Samuel Durand Professor of Medicine and director of the Wilmot Cancer Institute, University of Rochester, New York, said, “these findings confirm utility of auto-HCT in patients who achieve a CR.”

However, “the problem is that only a small fraction of patients achieve CR in this situation, and we do not know who they are going to be at time of relapse,” he told this news organization.

He agreed that “given robust randomized trials showing overall survival benefit of CAR-T compared to auto-HCT in patients with high risk relapsed DLBCL, CAR-T treatment should remain the current standard.

“However, these current results help to confirm the strategy for management of low- risk (late) relapses and indicate that auto-HCT still has a place for these patients if they achieve CR with salvage therapy.”

Dr. Shadman reported relationships with ADC therapeutics, Bristol Myers Squibb, Genmab, Lilly, Vincerx, Kite (Gilead), Janssen, Fate Therapeutics, MorphoSys/Incyte, AstraZeneca, BeiGene, Pharmacyclics, Mustang Bio, AbbVie, Genentech, MEI Pharma, Regeneron, and TG Therapeutics. Dr. Friedberg had no disclosures to report.

TOPLINE:

For relief of acute migraine, triptans, ergots, and antiemetics are two to five times more effective than ibuprofen, and acetaminophen is the least effective medication, 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. 

laflor/gettyimages

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:

For relief of acute migraine, triptans, ergots, and antiemetics are two to five times more effective than ibuprofen, and acetaminophen is the least effective medication, 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. 

laflor/gettyimages

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:

For relief of acute migraine, triptans, ergots, and antiemetics are two to five times more effective than ibuprofen, and acetaminophen is the least effective medication, 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. 

laflor/gettyimages

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.