User login
Genomic assay changes minds on HER2+ BC treatment
The prospective pilot study is small, and the researchers didn’t report on how the patients fared, according to a poster presented at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress. Plus, the test itself hasn’t been analyzed prospectively. But the study’s lead author, Olga Martínez-Sáez, MD, PhD, said in an interview that the 56% number is significant.
“We consider this percentage to be clinically very relevant,” said Dr. Martínez-Sáez, an oncologist at Hospital Clinic of Barcelona and the University of Barcelona. “HER2DX can change practice.”
Also in an interview, Kent Hoskins, MD, associate chief of hematology/oncology at University of Illinois at Chicago, described HER2DX as a next-generation genomic test that builds on assays developed 2 decades ago to help identify patients who would benefit – or not – from adjuvant chemotherapy.
Dr. Hoskins, who isn’t connected to the new study but has studied genomic tests for breast cancer, said the HER2DX test seeks to provide guidance to oncologists about which of several treatments are most effective in treating patients with HER2+ breast cancer.
“The overall trend in the HER2+ space is escalating therapy, and the cure rates have improved quite substantially,” he said. “But do they all need that much therapy? That’s the clinical question that this assay is addressing.”
The assay examines clinical features and the expression of 4 gene signatures, Dr. Martínez-Sáez said. It provides a risk score estimating the likelihood of recurrence plus a score that estimates the likelihood of achieving pathological complete response (pCR) with trastuzumab-based neoadjuvant therapy and an ERBB2 mRNA score.
In a retrospective 2022 study published in eBioMedicine, researchers reported that the assay “predicts response following neoadjuvant letrozole in combination with dual HER2 blockade with trastuzumab and pertuzumab in early-stage HER2-positive/hormone receptor–positive breast cancer.”
In the 2022 study, researchers wrote that assay results and other scores “might help better tailor systemic therapy in this context and identify candidates for avoiding chemotherapy, a therapy associated with short- and long-term toxicities and impact in quality of life.”
For the new study, a decision-impact analysis, researchers tracked 89 patients with HER2+ breast cancer (median age = 53 years, range 30-79, and 52% postmenopausal), the poster says. Most had T1-2 tumors (87%), negative nodes (64%), grade 2 (56%) or 3 (41%) tumors, and ductal histology (87%). And most were hormone receptor positive (65%). Seventy-eight percent of patients received neoadjuvant therapy (NAT), and 22% underwent upfront surgery.
In 56% of cases, oncologists changed their treatment decisions after getting the results of the HER2DX assays. In 59% of these cases, oncologists de-escalated therapy; in 41%, they escalated therapy, opting for more intense chemotherapy 65% of the time, according to the poster.
Clinician confidence in their decisions improved in 67% of cases, the researchers reported in their poster. Among 56 patients treated with neoadjuvant therapy who could be evaluated, “HER2DX pCR score was significantly associated with pCR (81% in pCR-medium/high and 32% in pCR-low; odds ratio=9.3, P = 0.001) independently of the rest of variables.”
Dr. Hoskins said the new report suggests that the assay can change treatment decisions, although he cautioned that “this study does not in itself establish its place in standard of care.” Large, prospective, randomized research is still needed, he said.
Dr. Martínez-Sáez said, in an interview, that the HER2DX assay should cost about as much as genomic assays for other breast cancer subtypes. These kinds of tests have cost several thousand dollars each in recent years.
What’s next? The decision impact study is ongoing. As for research into the assay itself, “prospective clinical trials are planned to demonstrate its clinical utility to de-escalate and guide therapy,” Dr. Martínez-Sáez said.
No funding is reported. Reveal Genomics is the developer of the HER2DX assay. Dr. Martinez- Saez reports financial relationships with Novartis, Eisai, Roche, and Reveal Genomics. Other study authors report multiple disclosures. Dr. Hoskins discloses non-financial research support from Agendia, which makes the MammaPrint early-breast-cancer assay.
The prospective pilot study is small, and the researchers didn’t report on how the patients fared, according to a poster presented at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress. Plus, the test itself hasn’t been analyzed prospectively. But the study’s lead author, Olga Martínez-Sáez, MD, PhD, said in an interview that the 56% number is significant.
“We consider this percentage to be clinically very relevant,” said Dr. Martínez-Sáez, an oncologist at Hospital Clinic of Barcelona and the University of Barcelona. “HER2DX can change practice.”
Also in an interview, Kent Hoskins, MD, associate chief of hematology/oncology at University of Illinois at Chicago, described HER2DX as a next-generation genomic test that builds on assays developed 2 decades ago to help identify patients who would benefit – or not – from adjuvant chemotherapy.
Dr. Hoskins, who isn’t connected to the new study but has studied genomic tests for breast cancer, said the HER2DX test seeks to provide guidance to oncologists about which of several treatments are most effective in treating patients with HER2+ breast cancer.
“The overall trend in the HER2+ space is escalating therapy, and the cure rates have improved quite substantially,” he said. “But do they all need that much therapy? That’s the clinical question that this assay is addressing.”
The assay examines clinical features and the expression of 4 gene signatures, Dr. Martínez-Sáez said. It provides a risk score estimating the likelihood of recurrence plus a score that estimates the likelihood of achieving pathological complete response (pCR) with trastuzumab-based neoadjuvant therapy and an ERBB2 mRNA score.
In a retrospective 2022 study published in eBioMedicine, researchers reported that the assay “predicts response following neoadjuvant letrozole in combination with dual HER2 blockade with trastuzumab and pertuzumab in early-stage HER2-positive/hormone receptor–positive breast cancer.”
In the 2022 study, researchers wrote that assay results and other scores “might help better tailor systemic therapy in this context and identify candidates for avoiding chemotherapy, a therapy associated with short- and long-term toxicities and impact in quality of life.”
For the new study, a decision-impact analysis, researchers tracked 89 patients with HER2+ breast cancer (median age = 53 years, range 30-79, and 52% postmenopausal), the poster says. Most had T1-2 tumors (87%), negative nodes (64%), grade 2 (56%) or 3 (41%) tumors, and ductal histology (87%). And most were hormone receptor positive (65%). Seventy-eight percent of patients received neoadjuvant therapy (NAT), and 22% underwent upfront surgery.
In 56% of cases, oncologists changed their treatment decisions after getting the results of the HER2DX assays. In 59% of these cases, oncologists de-escalated therapy; in 41%, they escalated therapy, opting for more intense chemotherapy 65% of the time, according to the poster.
Clinician confidence in their decisions improved in 67% of cases, the researchers reported in their poster. Among 56 patients treated with neoadjuvant therapy who could be evaluated, “HER2DX pCR score was significantly associated with pCR (81% in pCR-medium/high and 32% in pCR-low; odds ratio=9.3, P = 0.001) independently of the rest of variables.”
Dr. Hoskins said the new report suggests that the assay can change treatment decisions, although he cautioned that “this study does not in itself establish its place in standard of care.” Large, prospective, randomized research is still needed, he said.
Dr. Martínez-Sáez said, in an interview, that the HER2DX assay should cost about as much as genomic assays for other breast cancer subtypes. These kinds of tests have cost several thousand dollars each in recent years.
What’s next? The decision impact study is ongoing. As for research into the assay itself, “prospective clinical trials are planned to demonstrate its clinical utility to de-escalate and guide therapy,” Dr. Martínez-Sáez said.
No funding is reported. Reveal Genomics is the developer of the HER2DX assay. Dr. Martinez- Saez reports financial relationships with Novartis, Eisai, Roche, and Reveal Genomics. Other study authors report multiple disclosures. Dr. Hoskins discloses non-financial research support from Agendia, which makes the MammaPrint early-breast-cancer assay.
The prospective pilot study is small, and the researchers didn’t report on how the patients fared, according to a poster presented at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress. Plus, the test itself hasn’t been analyzed prospectively. But the study’s lead author, Olga Martínez-Sáez, MD, PhD, said in an interview that the 56% number is significant.
“We consider this percentage to be clinically very relevant,” said Dr. Martínez-Sáez, an oncologist at Hospital Clinic of Barcelona and the University of Barcelona. “HER2DX can change practice.”
Also in an interview, Kent Hoskins, MD, associate chief of hematology/oncology at University of Illinois at Chicago, described HER2DX as a next-generation genomic test that builds on assays developed 2 decades ago to help identify patients who would benefit – or not – from adjuvant chemotherapy.
Dr. Hoskins, who isn’t connected to the new study but has studied genomic tests for breast cancer, said the HER2DX test seeks to provide guidance to oncologists about which of several treatments are most effective in treating patients with HER2+ breast cancer.
“The overall trend in the HER2+ space is escalating therapy, and the cure rates have improved quite substantially,” he said. “But do they all need that much therapy? That’s the clinical question that this assay is addressing.”
The assay examines clinical features and the expression of 4 gene signatures, Dr. Martínez-Sáez said. It provides a risk score estimating the likelihood of recurrence plus a score that estimates the likelihood of achieving pathological complete response (pCR) with trastuzumab-based neoadjuvant therapy and an ERBB2 mRNA score.
In a retrospective 2022 study published in eBioMedicine, researchers reported that the assay “predicts response following neoadjuvant letrozole in combination with dual HER2 blockade with trastuzumab and pertuzumab in early-stage HER2-positive/hormone receptor–positive breast cancer.”
In the 2022 study, researchers wrote that assay results and other scores “might help better tailor systemic therapy in this context and identify candidates for avoiding chemotherapy, a therapy associated with short- and long-term toxicities and impact in quality of life.”
For the new study, a decision-impact analysis, researchers tracked 89 patients with HER2+ breast cancer (median age = 53 years, range 30-79, and 52% postmenopausal), the poster says. Most had T1-2 tumors (87%), negative nodes (64%), grade 2 (56%) or 3 (41%) tumors, and ductal histology (87%). And most were hormone receptor positive (65%). Seventy-eight percent of patients received neoadjuvant therapy (NAT), and 22% underwent upfront surgery.
In 56% of cases, oncologists changed their treatment decisions after getting the results of the HER2DX assays. In 59% of these cases, oncologists de-escalated therapy; in 41%, they escalated therapy, opting for more intense chemotherapy 65% of the time, according to the poster.
Clinician confidence in their decisions improved in 67% of cases, the researchers reported in their poster. Among 56 patients treated with neoadjuvant therapy who could be evaluated, “HER2DX pCR score was significantly associated with pCR (81% in pCR-medium/high and 32% in pCR-low; odds ratio=9.3, P = 0.001) independently of the rest of variables.”
Dr. Hoskins said the new report suggests that the assay can change treatment decisions, although he cautioned that “this study does not in itself establish its place in standard of care.” Large, prospective, randomized research is still needed, he said.
Dr. Martínez-Sáez said, in an interview, that the HER2DX assay should cost about as much as genomic assays for other breast cancer subtypes. These kinds of tests have cost several thousand dollars each in recent years.
What’s next? The decision impact study is ongoing. As for research into the assay itself, “prospective clinical trials are planned to demonstrate its clinical utility to de-escalate and guide therapy,” Dr. Martínez-Sáez said.
No funding is reported. Reveal Genomics is the developer of the HER2DX assay. Dr. Martinez- Saez reports financial relationships with Novartis, Eisai, Roche, and Reveal Genomics. Other study authors report multiple disclosures. Dr. Hoskins discloses non-financial research support from Agendia, which makes the MammaPrint early-breast-cancer assay.
FROM ESMO BREAST CANCER 2023
T-DXd for HER2-low BC: Analysis confirms adverse effects
Interstitial lung disease (ILD) also remains a concern, and it’s not clear if retreatment after resolution is warranted.
In general, however, “T-DXd demonstrates a manageable safety profile consistent with prior reports. Results from this safety analysis continued to support its use as a new standard of care in patients with HER2-low metastatic breast cancer,” said report lead author Hope Rugo, MD, of the University of California, San Francisco, during a presentation at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
T-DXd, an antibody-drug conjugate, received FDA approval in August 2022 for patients with HER2-low disease. The drug has been touted as “practice changing” and a “new standard of care.”
However, physicians have noted that the benefits of the drug come at the cost of significant adverse effects, including some that can cause hospitalization. There’s special concern about high-grade interstitial lung disease/pneumonitis, and an FDA boxed warning cautions clinicians about this possible side effect.
For the new analysis, researchers presented additional safety data from the industry-funded DESTINY-Breast04 trial, whose results was published in July 2022 in the New England Journal of Medicine. That study randomized 373 patients to T-DXd and 184 to physician’s choice of treatment. It found that, “among all patients, the median progression-free survival was 9.9 months in the trastuzumab deruxtecan group and 5.1 months in the physician’s choice group (hazard ratio for disease progression or death, 0.50; P < .001), and overall survival was 23.4 months and 16.8 months, respectively (hazard ratio for death, 0.64; P = .001).”
Exposure-adjusted incidence rates for any-grade treatment-emergent adverse events were lower for T-DXd versus physician’s choice of treatment (1.30 vs. 2.66). However, nausea and vomiting events were more than twice as common in patients who took T-DXd versus the physician’s choice (79.5% vs. 35.5%).
A total of 50.9% of patients in the T-DXd arm received antiemetic prophylaxis versus 37.2% in the physician’s choice arm. A single patient discontinued T-DXd treatment because of vomiting, and a single patient discontinued treatment because of nausea. No patients in the physician choice group discontinued treatment because of nausea or vomiting.
Neutropenia and febrile neutropenia were less frequent in the T-DXd arm versus physician’s choice (12.9% vs. 18.0% and 0.3% vs. 2.9%, respectively.)
ILD occurred in 45 patients (12.1%) of those in the T-DXd arm versus 1 (0.6%) in the physician choice arm. Ten patients of the patients in the T-DXd arm had not recovered by the data cutoff point.
Six patients with ILD were retreated following resolution; one discontinued because of an adverse event, two discontinued because of progressive disease, and three remained on the drug. “Given that there was only a small number of patients who were retreated with T-DXd, it’s difficult to make clinically meaningful conclusions on the effect of retreatment following grade IDL events that have resolved,” Dr. Rugo said.
In the big picture, “ILD pneumonitis remains an important identified risk and an adverse event of interest associated with T-DXd,” Dr. Rugo said. “It’s important that we adhere to management guidelines and updated toxicity management guidelines.”
In a discussion, Dr. Rugo said she prescribes three antiemetic drugs to help patients tolerate T-DXd therapy: “It makes a big difference. Anecdotally, it really has improved management of nausea. Start more and back down [as symptoms fade].”
Gustavo Werutsky, MD, PhD, of Moinhos de Vento Hospital, Porto Alegre, Brazil, the discussant for the presentation, also emphasized the importance of prevention and said he prescribes two or three prophylactic drugs. “In the beginning, we didn’t know these events were so important. A big part of the message is that patients from the beginning need to have a good prophylaxis for the nausea and vomiting.”
The researchers also presented a related report at the conference, an analysis of patient-reported outcomes from DESTINY-Breast02, a randomized phase 3 study of T-DXd (n = 406) versus physician’s choice of treatment (n = 202) in patients with HER2-positive metastatic breast cancer who were resistant/refractory to trastuzumab emtansine.
The analysis, led by Tanja Fehm, MD, of University Hospital Düsseldorf (Germany), found that the median time to definitive deterioration was longer with T-DXd versus the other arm per the EORTC QLQ-C30 global health status/quality of life score (14.1 vs. 5.9 months; HR, 0.56; 95% confidence interval, 0.44-0.71).
The studies were funded by Daiichi Sankyo and AstraZeneca, which make T-DXd. Dr. Hugo discloses relationships with Puma, NAPO, Blueprint, Scorpion Therapeutics, Merck, AstraZeneca, Gilead, Astellas, Daiichi Sankyo, F. Hoffmann–La Roche/Genentech, GlaxoSmithKline, Lilly, Novartis, OBI, Pfizer, Pionyr, Sermonix, Taiho Oncology, and Veru. Multiple other authors of both studies have various disclosures.
Interstitial lung disease (ILD) also remains a concern, and it’s not clear if retreatment after resolution is warranted.
In general, however, “T-DXd demonstrates a manageable safety profile consistent with prior reports. Results from this safety analysis continued to support its use as a new standard of care in patients with HER2-low metastatic breast cancer,” said report lead author Hope Rugo, MD, of the University of California, San Francisco, during a presentation at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
T-DXd, an antibody-drug conjugate, received FDA approval in August 2022 for patients with HER2-low disease. The drug has been touted as “practice changing” and a “new standard of care.”
However, physicians have noted that the benefits of the drug come at the cost of significant adverse effects, including some that can cause hospitalization. There’s special concern about high-grade interstitial lung disease/pneumonitis, and an FDA boxed warning cautions clinicians about this possible side effect.
For the new analysis, researchers presented additional safety data from the industry-funded DESTINY-Breast04 trial, whose results was published in July 2022 in the New England Journal of Medicine. That study randomized 373 patients to T-DXd and 184 to physician’s choice of treatment. It found that, “among all patients, the median progression-free survival was 9.9 months in the trastuzumab deruxtecan group and 5.1 months in the physician’s choice group (hazard ratio for disease progression or death, 0.50; P < .001), and overall survival was 23.4 months and 16.8 months, respectively (hazard ratio for death, 0.64; P = .001).”
Exposure-adjusted incidence rates for any-grade treatment-emergent adverse events were lower for T-DXd versus physician’s choice of treatment (1.30 vs. 2.66). However, nausea and vomiting events were more than twice as common in patients who took T-DXd versus the physician’s choice (79.5% vs. 35.5%).
A total of 50.9% of patients in the T-DXd arm received antiemetic prophylaxis versus 37.2% in the physician’s choice arm. A single patient discontinued T-DXd treatment because of vomiting, and a single patient discontinued treatment because of nausea. No patients in the physician choice group discontinued treatment because of nausea or vomiting.
Neutropenia and febrile neutropenia were less frequent in the T-DXd arm versus physician’s choice (12.9% vs. 18.0% and 0.3% vs. 2.9%, respectively.)
ILD occurred in 45 patients (12.1%) of those in the T-DXd arm versus 1 (0.6%) in the physician choice arm. Ten patients of the patients in the T-DXd arm had not recovered by the data cutoff point.
Six patients with ILD were retreated following resolution; one discontinued because of an adverse event, two discontinued because of progressive disease, and three remained on the drug. “Given that there was only a small number of patients who were retreated with T-DXd, it’s difficult to make clinically meaningful conclusions on the effect of retreatment following grade IDL events that have resolved,” Dr. Rugo said.
In the big picture, “ILD pneumonitis remains an important identified risk and an adverse event of interest associated with T-DXd,” Dr. Rugo said. “It’s important that we adhere to management guidelines and updated toxicity management guidelines.”
In a discussion, Dr. Rugo said she prescribes three antiemetic drugs to help patients tolerate T-DXd therapy: “It makes a big difference. Anecdotally, it really has improved management of nausea. Start more and back down [as symptoms fade].”
Gustavo Werutsky, MD, PhD, of Moinhos de Vento Hospital, Porto Alegre, Brazil, the discussant for the presentation, also emphasized the importance of prevention and said he prescribes two or three prophylactic drugs. “In the beginning, we didn’t know these events were so important. A big part of the message is that patients from the beginning need to have a good prophylaxis for the nausea and vomiting.”
The researchers also presented a related report at the conference, an analysis of patient-reported outcomes from DESTINY-Breast02, a randomized phase 3 study of T-DXd (n = 406) versus physician’s choice of treatment (n = 202) in patients with HER2-positive metastatic breast cancer who were resistant/refractory to trastuzumab emtansine.
The analysis, led by Tanja Fehm, MD, of University Hospital Düsseldorf (Germany), found that the median time to definitive deterioration was longer with T-DXd versus the other arm per the EORTC QLQ-C30 global health status/quality of life score (14.1 vs. 5.9 months; HR, 0.56; 95% confidence interval, 0.44-0.71).
The studies were funded by Daiichi Sankyo and AstraZeneca, which make T-DXd. Dr. Hugo discloses relationships with Puma, NAPO, Blueprint, Scorpion Therapeutics, Merck, AstraZeneca, Gilead, Astellas, Daiichi Sankyo, F. Hoffmann–La Roche/Genentech, GlaxoSmithKline, Lilly, Novartis, OBI, Pfizer, Pionyr, Sermonix, Taiho Oncology, and Veru. Multiple other authors of both studies have various disclosures.
Interstitial lung disease (ILD) also remains a concern, and it’s not clear if retreatment after resolution is warranted.
In general, however, “T-DXd demonstrates a manageable safety profile consistent with prior reports. Results from this safety analysis continued to support its use as a new standard of care in patients with HER2-low metastatic breast cancer,” said report lead author Hope Rugo, MD, of the University of California, San Francisco, during a presentation at the European Society for Medical Oncology (ESMO) Breast Cancer annual congress.
T-DXd, an antibody-drug conjugate, received FDA approval in August 2022 for patients with HER2-low disease. The drug has been touted as “practice changing” and a “new standard of care.”
However, physicians have noted that the benefits of the drug come at the cost of significant adverse effects, including some that can cause hospitalization. There’s special concern about high-grade interstitial lung disease/pneumonitis, and an FDA boxed warning cautions clinicians about this possible side effect.
For the new analysis, researchers presented additional safety data from the industry-funded DESTINY-Breast04 trial, whose results was published in July 2022 in the New England Journal of Medicine. That study randomized 373 patients to T-DXd and 184 to physician’s choice of treatment. It found that, “among all patients, the median progression-free survival was 9.9 months in the trastuzumab deruxtecan group and 5.1 months in the physician’s choice group (hazard ratio for disease progression or death, 0.50; P < .001), and overall survival was 23.4 months and 16.8 months, respectively (hazard ratio for death, 0.64; P = .001).”
Exposure-adjusted incidence rates for any-grade treatment-emergent adverse events were lower for T-DXd versus physician’s choice of treatment (1.30 vs. 2.66). However, nausea and vomiting events were more than twice as common in patients who took T-DXd versus the physician’s choice (79.5% vs. 35.5%).
A total of 50.9% of patients in the T-DXd arm received antiemetic prophylaxis versus 37.2% in the physician’s choice arm. A single patient discontinued T-DXd treatment because of vomiting, and a single patient discontinued treatment because of nausea. No patients in the physician choice group discontinued treatment because of nausea or vomiting.
Neutropenia and febrile neutropenia were less frequent in the T-DXd arm versus physician’s choice (12.9% vs. 18.0% and 0.3% vs. 2.9%, respectively.)
ILD occurred in 45 patients (12.1%) of those in the T-DXd arm versus 1 (0.6%) in the physician choice arm. Ten patients of the patients in the T-DXd arm had not recovered by the data cutoff point.
Six patients with ILD were retreated following resolution; one discontinued because of an adverse event, two discontinued because of progressive disease, and three remained on the drug. “Given that there was only a small number of patients who were retreated with T-DXd, it’s difficult to make clinically meaningful conclusions on the effect of retreatment following grade IDL events that have resolved,” Dr. Rugo said.
In the big picture, “ILD pneumonitis remains an important identified risk and an adverse event of interest associated with T-DXd,” Dr. Rugo said. “It’s important that we adhere to management guidelines and updated toxicity management guidelines.”
In a discussion, Dr. Rugo said she prescribes three antiemetic drugs to help patients tolerate T-DXd therapy: “It makes a big difference. Anecdotally, it really has improved management of nausea. Start more and back down [as symptoms fade].”
Gustavo Werutsky, MD, PhD, of Moinhos de Vento Hospital, Porto Alegre, Brazil, the discussant for the presentation, also emphasized the importance of prevention and said he prescribes two or three prophylactic drugs. “In the beginning, we didn’t know these events were so important. A big part of the message is that patients from the beginning need to have a good prophylaxis for the nausea and vomiting.”
The researchers also presented a related report at the conference, an analysis of patient-reported outcomes from DESTINY-Breast02, a randomized phase 3 study of T-DXd (n = 406) versus physician’s choice of treatment (n = 202) in patients with HER2-positive metastatic breast cancer who were resistant/refractory to trastuzumab emtansine.
The analysis, led by Tanja Fehm, MD, of University Hospital Düsseldorf (Germany), found that the median time to definitive deterioration was longer with T-DXd versus the other arm per the EORTC QLQ-C30 global health status/quality of life score (14.1 vs. 5.9 months; HR, 0.56; 95% confidence interval, 0.44-0.71).
The studies were funded by Daiichi Sankyo and AstraZeneca, which make T-DXd. Dr. Hugo discloses relationships with Puma, NAPO, Blueprint, Scorpion Therapeutics, Merck, AstraZeneca, Gilead, Astellas, Daiichi Sankyo, F. Hoffmann–La Roche/Genentech, GlaxoSmithKline, Lilly, Novartis, OBI, Pfizer, Pionyr, Sermonix, Taiho Oncology, and Veru. Multiple other authors of both studies have various disclosures.
FROM ESMO BREAST CANCER 2023
One in five brain injury trials shows errors, signs of spin
LOS ANGELES –
“This is concerning result,” said general physician Lucas Piason F. Martins, MD, of the Bahiana School of Medicine and Public Health, Salvador, Brazil. “Many of these trials have been included in clinical guidelines and cited extensively in systematic reviews and meta-analyses, especially those related to hypothermia therapy.”
Dr. Martins presented the findings at the annual meeting of the American Association of Neurological Surgeons.
Defining spin
In recent years, medical researchers have sought to define and identify spin in medical literature. According to a 2017 report in PLOS Biology, “spin refers to reporting practices that distort the interpretation of results and mislead readers so that results are viewed in a more favorable light.”
Any spin can be dangerous, Dr. Martins said, because it “can potentially mislead readers and affect the interpretation of study results, which in turn can impact clinical decision-making.”
For the new report, a systematic review, Dr. Martins and colleagues examined 150 studies published in 18 top-ranked journals including the Journal of Neurotrauma (26%), the Journal of Neurosurgery (15%), Critical Care Medicine (9%), and the New England Journal of Medicine (8%).
Studies were published between 1960 and 2020. The review protocol was previously published in BMJ Open.
According to the report, most of the 32 studies with spin (75%) had a “focus on statistically significant results not based on primary outcome.”
For example, Dr. Martins said in an interview that the abstract for a study about drug treatment of brain contusions highlighted a secondary result instead of the main finding that the medication had no effect. Another study of treatment for severe closed head injuries focused on a subgroup outcome.
As Dr. Martins noted, it’s potentially problematic for studies to have several outcomes, measure outcomes in different ways, and have multiple time points without a predefined primary outcome. “A positive finding based on such strategies could potentially be explained by chance alone,” he said.
The researchers also reported that 65% of the studies with spin highlighted “the beneficial effect of the treatment despite statistically nonsignificant results” and that 9% had incorrect statistical analysis.
The findings are especially noteworthy because “the trials we analyzed were deemed to have the highest quality of methodology,” Dr. Martins said.
The researchers didn’t identify specific studies that they deemed to have spin, and they won’t do so, Dr. Martins said. The authors do plan to reveal which journals were most spin-heavy but only when these findings are published.
Were the study authors trying to mislead readers? Not necessarily. Researchers “may search for positive results to confirm their beliefs, although with good intentions,” Dr. Martins said, adding that the researchers found that “positive research tends to be more cited.”
They also reported that studies with smaller sample sizes were more likely to have spin (P = .04).
At 21%, the percentage of studies with spin was lower than that found in some previous reports that analyzed medical literature in other specialties.
A 2019 study of 93 randomized clinical studies in cardiology, for example, found spin in 57% of abstracts and 67% of full texts. The lower number in the new study may be due to its especially conservative definition of spin, Dr. Martins said.
Appropriate methodology
Cardiologist Richard Krasuski, MD, of Duke University Medical Center, Durham, N.C., who coauthored the 2019 study into spin in cardiology studies, told this news organization that the new analysis follows appropriate methodology and appears to be valid.
It makes sense, he said, that smaller studies had more spin: “It is much harder to show statistical significance in small studies and softer endpoints can be harder to predict. Small neutral trials are also much harder to publish in high-level journals. This all increases the tendency to spin the results so the reviewer and eventually the reader is more captivated.”
Why is there so much spin in medical research? “As an investigator, you always hope to positively impact patient health and outcomes, so there is a tendency to look at secondary analyses to have something good to emphasize,” he said. “This is an inherent trait in most of us, to find something good we can focus on. I do believe that much of this is subconscious and perhaps with noble intent.”
Dr. Krasuski said that he advises trainees to look at the methodology of studies, not just the abstract or discussion sections. “You don’t have to be a trained statistician to identify how well the findings match the author’s interpretation.
“Always try to identify what the primary outcome of the study was at the time of the design and whether the investigators achieved their objective. As a reviewer, my own personal experience in research into spin makes me more cognizant of its existence, and I generally require authors to reword and tone down their message if it is not supported by the data.”
What’s next? The investigators want to look for spin in the wider neurosurgery literature, Dr. Martins said, with an eye toward developing “practical strategies to assess spin and give pragmatic recommendations for good practice in clinical research.”
No study funding is reported. Dr. Martins has no disclosures, and several study authors reported funding from the UK National Institute for Health Research. Dr. Krasuski has no disclosures.
A version of this article first appeared on Medscape.com.
LOS ANGELES –
“This is concerning result,” said general physician Lucas Piason F. Martins, MD, of the Bahiana School of Medicine and Public Health, Salvador, Brazil. “Many of these trials have been included in clinical guidelines and cited extensively in systematic reviews and meta-analyses, especially those related to hypothermia therapy.”
Dr. Martins presented the findings at the annual meeting of the American Association of Neurological Surgeons.
Defining spin
In recent years, medical researchers have sought to define and identify spin in medical literature. According to a 2017 report in PLOS Biology, “spin refers to reporting practices that distort the interpretation of results and mislead readers so that results are viewed in a more favorable light.”
Any spin can be dangerous, Dr. Martins said, because it “can potentially mislead readers and affect the interpretation of study results, which in turn can impact clinical decision-making.”
For the new report, a systematic review, Dr. Martins and colleagues examined 150 studies published in 18 top-ranked journals including the Journal of Neurotrauma (26%), the Journal of Neurosurgery (15%), Critical Care Medicine (9%), and the New England Journal of Medicine (8%).
Studies were published between 1960 and 2020. The review protocol was previously published in BMJ Open.
According to the report, most of the 32 studies with spin (75%) had a “focus on statistically significant results not based on primary outcome.”
For example, Dr. Martins said in an interview that the abstract for a study about drug treatment of brain contusions highlighted a secondary result instead of the main finding that the medication had no effect. Another study of treatment for severe closed head injuries focused on a subgroup outcome.
As Dr. Martins noted, it’s potentially problematic for studies to have several outcomes, measure outcomes in different ways, and have multiple time points without a predefined primary outcome. “A positive finding based on such strategies could potentially be explained by chance alone,” he said.
The researchers also reported that 65% of the studies with spin highlighted “the beneficial effect of the treatment despite statistically nonsignificant results” and that 9% had incorrect statistical analysis.
The findings are especially noteworthy because “the trials we analyzed were deemed to have the highest quality of methodology,” Dr. Martins said.
The researchers didn’t identify specific studies that they deemed to have spin, and they won’t do so, Dr. Martins said. The authors do plan to reveal which journals were most spin-heavy but only when these findings are published.
Were the study authors trying to mislead readers? Not necessarily. Researchers “may search for positive results to confirm their beliefs, although with good intentions,” Dr. Martins said, adding that the researchers found that “positive research tends to be more cited.”
They also reported that studies with smaller sample sizes were more likely to have spin (P = .04).
At 21%, the percentage of studies with spin was lower than that found in some previous reports that analyzed medical literature in other specialties.
A 2019 study of 93 randomized clinical studies in cardiology, for example, found spin in 57% of abstracts and 67% of full texts. The lower number in the new study may be due to its especially conservative definition of spin, Dr. Martins said.
Appropriate methodology
Cardiologist Richard Krasuski, MD, of Duke University Medical Center, Durham, N.C., who coauthored the 2019 study into spin in cardiology studies, told this news organization that the new analysis follows appropriate methodology and appears to be valid.
It makes sense, he said, that smaller studies had more spin: “It is much harder to show statistical significance in small studies and softer endpoints can be harder to predict. Small neutral trials are also much harder to publish in high-level journals. This all increases the tendency to spin the results so the reviewer and eventually the reader is more captivated.”
Why is there so much spin in medical research? “As an investigator, you always hope to positively impact patient health and outcomes, so there is a tendency to look at secondary analyses to have something good to emphasize,” he said. “This is an inherent trait in most of us, to find something good we can focus on. I do believe that much of this is subconscious and perhaps with noble intent.”
Dr. Krasuski said that he advises trainees to look at the methodology of studies, not just the abstract or discussion sections. “You don’t have to be a trained statistician to identify how well the findings match the author’s interpretation.
“Always try to identify what the primary outcome of the study was at the time of the design and whether the investigators achieved their objective. As a reviewer, my own personal experience in research into spin makes me more cognizant of its existence, and I generally require authors to reword and tone down their message if it is not supported by the data.”
What’s next? The investigators want to look for spin in the wider neurosurgery literature, Dr. Martins said, with an eye toward developing “practical strategies to assess spin and give pragmatic recommendations for good practice in clinical research.”
No study funding is reported. Dr. Martins has no disclosures, and several study authors reported funding from the UK National Institute for Health Research. Dr. Krasuski has no disclosures.
A version of this article first appeared on Medscape.com.
LOS ANGELES –
“This is concerning result,” said general physician Lucas Piason F. Martins, MD, of the Bahiana School of Medicine and Public Health, Salvador, Brazil. “Many of these trials have been included in clinical guidelines and cited extensively in systematic reviews and meta-analyses, especially those related to hypothermia therapy.”
Dr. Martins presented the findings at the annual meeting of the American Association of Neurological Surgeons.
Defining spin
In recent years, medical researchers have sought to define and identify spin in medical literature. According to a 2017 report in PLOS Biology, “spin refers to reporting practices that distort the interpretation of results and mislead readers so that results are viewed in a more favorable light.”
Any spin can be dangerous, Dr. Martins said, because it “can potentially mislead readers and affect the interpretation of study results, which in turn can impact clinical decision-making.”
For the new report, a systematic review, Dr. Martins and colleagues examined 150 studies published in 18 top-ranked journals including the Journal of Neurotrauma (26%), the Journal of Neurosurgery (15%), Critical Care Medicine (9%), and the New England Journal of Medicine (8%).
Studies were published between 1960 and 2020. The review protocol was previously published in BMJ Open.
According to the report, most of the 32 studies with spin (75%) had a “focus on statistically significant results not based on primary outcome.”
For example, Dr. Martins said in an interview that the abstract for a study about drug treatment of brain contusions highlighted a secondary result instead of the main finding that the medication had no effect. Another study of treatment for severe closed head injuries focused on a subgroup outcome.
As Dr. Martins noted, it’s potentially problematic for studies to have several outcomes, measure outcomes in different ways, and have multiple time points without a predefined primary outcome. “A positive finding based on such strategies could potentially be explained by chance alone,” he said.
The researchers also reported that 65% of the studies with spin highlighted “the beneficial effect of the treatment despite statistically nonsignificant results” and that 9% had incorrect statistical analysis.
The findings are especially noteworthy because “the trials we analyzed were deemed to have the highest quality of methodology,” Dr. Martins said.
The researchers didn’t identify specific studies that they deemed to have spin, and they won’t do so, Dr. Martins said. The authors do plan to reveal which journals were most spin-heavy but only when these findings are published.
Were the study authors trying to mislead readers? Not necessarily. Researchers “may search for positive results to confirm their beliefs, although with good intentions,” Dr. Martins said, adding that the researchers found that “positive research tends to be more cited.”
They also reported that studies with smaller sample sizes were more likely to have spin (P = .04).
At 21%, the percentage of studies with spin was lower than that found in some previous reports that analyzed medical literature in other specialties.
A 2019 study of 93 randomized clinical studies in cardiology, for example, found spin in 57% of abstracts and 67% of full texts. The lower number in the new study may be due to its especially conservative definition of spin, Dr. Martins said.
Appropriate methodology
Cardiologist Richard Krasuski, MD, of Duke University Medical Center, Durham, N.C., who coauthored the 2019 study into spin in cardiology studies, told this news organization that the new analysis follows appropriate methodology and appears to be valid.
It makes sense, he said, that smaller studies had more spin: “It is much harder to show statistical significance in small studies and softer endpoints can be harder to predict. Small neutral trials are also much harder to publish in high-level journals. This all increases the tendency to spin the results so the reviewer and eventually the reader is more captivated.”
Why is there so much spin in medical research? “As an investigator, you always hope to positively impact patient health and outcomes, so there is a tendency to look at secondary analyses to have something good to emphasize,” he said. “This is an inherent trait in most of us, to find something good we can focus on. I do believe that much of this is subconscious and perhaps with noble intent.”
Dr. Krasuski said that he advises trainees to look at the methodology of studies, not just the abstract or discussion sections. “You don’t have to be a trained statistician to identify how well the findings match the author’s interpretation.
“Always try to identify what the primary outcome of the study was at the time of the design and whether the investigators achieved their objective. As a reviewer, my own personal experience in research into spin makes me more cognizant of its existence, and I generally require authors to reword and tone down their message if it is not supported by the data.”
What’s next? The investigators want to look for spin in the wider neurosurgery literature, Dr. Martins said, with an eye toward developing “practical strategies to assess spin and give pragmatic recommendations for good practice in clinical research.”
No study funding is reported. Dr. Martins has no disclosures, and several study authors reported funding from the UK National Institute for Health Research. Dr. Krasuski has no disclosures.
A version of this article first appeared on Medscape.com.
FROM AANS 2023
CLL: Black patients die sooner than Whites
The findings, published in the American Journal of Hematology, hint that the racial disparity has shrunk over time, especially within the first few years of the targeted-therapy era. Still, “Black patients had a shorter median overall survival of 7 years compared to 9 years for White patients,” study coauthor Deborah Stephens, DO, of the University of Utah Huntsman Cancer Institute, said in an interview. “Clearly, more research is needed to tease out the biologic or economic barriers to achieving prolonged survival.”
As the researchers noted, CLL is far more common among White patients (5.1 cases per 100,000) than other races (Black patients: 3.2 cases per 100,000; Hispanic patients: 2.1 cases per 100,000; Asian American patients: 1.1 per 100,000). In total, non-White patients make up just 11%-13% of CLL cases in the United States.
According to Dr. Stephens, “little is known or published” about Black patients with CLL, “and it is still a mystery why fewer patients that are Black develop CLL and why this group would have shorter survival.”
Dr. Stephens and colleagues launched the new study – the largest of its kind to date – to understand disparities between White and Black patients over most of the past 20 years. The researchers especially wanted to analyze trends during the last decade, when targeted therapies revolutionized treatment of the disease.
The study authors analyzed data in the National Cancer Database for 97,804 patients diagnosed from 2004 to 2018 (90.7% White, 7.6% Black, 0.6% Asian, 1.1% other). Of patients who reported ethnicity (n = 93,555), 2.6% were Hispanic.
Black patients were more likely to have begun CLL therapy at diagnosis (35.9%) than were White patients (23.6%), a sign that Black patients had more advanced disease. Black patients also had shorter overall survival (7.0 years, 95% confidence interval [CI], 6.7–7.3 years) vs. White patients (9.1 years, 95% CI, 9.0–9.3 years, P < .001).
“This finding could be due to underlying biologic differences in the pathology of CLL, when comparing patients across racial groups,” Dr. Stephens said. “Additionally, there could be differences in access to care. Notably, there are fewer racial minorities enrolled in clinical trials, and perhaps we are not individualizing therapy for unique biologic factors seen in CLL affecting racial minorities.”
Other factors also could be at play. Black patients were more likely than were White patients to have one or more comorbidities (27.9% vs. 21.3%, P < .001), lack insurance (6.6% vs. 2.1%, P < .001) and live in lower-income neighborhoods (47.7% vs. 13.1%, P < .001).
What explains the gap in outcomes? In an interview, study lead author Victoria Vardell, MD, of the University of Utah, Salt Lake City, noted that researchers often attribute worse medical outcomes in Black patients to economic and social disparities.
“However, when we adjusted for a number of surrogate markers of health care access, including income, comorbidities, and location, among others, this disparity remained. That indicates that this may be a more complex problem in CLL in particular. Certainly, we cannot adjust for all the socioeconomic strain placed on Black Americans, including those with CLL, but there may be molecular features related to ancestry or environmental exposures that also play a role,” Dr. Vardell said.
She added that “the high cost and difficulty obtaining many novel therapies, particularly in the clinical trial setting, places significantly higher burdens on already disadvantaged populations.”
There is some good news in the new report. “Promisingly, our data suggest that the survival disparity between White and Black patients with CLL may be improving, particularly within the last 5 years, though longer follow-up is needed to confirm significance,” the researchers reported.
Alessandra Ferrajoli, MD, of M.D. Anderson Cancer Center, Houston, who has studied racial disparities in CLL, praised the study in an interview. As she noted, it examines an impressively large population.
The explanations for the disparities are still elusive, she said, although it seems clear there are multiple factors at play. “We don’t know if the disease has the same characteristics in African-Americans as in Whites,” Dr. Ferrajoli said. However, she noted, there’s “no indication that the response to treatment is different according to race.”
Moving forward, she said, the study findings “reinforce the fact that we need to pay attention to this population and be quite attentive to their characteristics.”
No study funding was reported. The authors and Dr. Ferrajoli have no disclosures.
The findings, published in the American Journal of Hematology, hint that the racial disparity has shrunk over time, especially within the first few years of the targeted-therapy era. Still, “Black patients had a shorter median overall survival of 7 years compared to 9 years for White patients,” study coauthor Deborah Stephens, DO, of the University of Utah Huntsman Cancer Institute, said in an interview. “Clearly, more research is needed to tease out the biologic or economic barriers to achieving prolonged survival.”
As the researchers noted, CLL is far more common among White patients (5.1 cases per 100,000) than other races (Black patients: 3.2 cases per 100,000; Hispanic patients: 2.1 cases per 100,000; Asian American patients: 1.1 per 100,000). In total, non-White patients make up just 11%-13% of CLL cases in the United States.
According to Dr. Stephens, “little is known or published” about Black patients with CLL, “and it is still a mystery why fewer patients that are Black develop CLL and why this group would have shorter survival.”
Dr. Stephens and colleagues launched the new study – the largest of its kind to date – to understand disparities between White and Black patients over most of the past 20 years. The researchers especially wanted to analyze trends during the last decade, when targeted therapies revolutionized treatment of the disease.
The study authors analyzed data in the National Cancer Database for 97,804 patients diagnosed from 2004 to 2018 (90.7% White, 7.6% Black, 0.6% Asian, 1.1% other). Of patients who reported ethnicity (n = 93,555), 2.6% were Hispanic.
Black patients were more likely to have begun CLL therapy at diagnosis (35.9%) than were White patients (23.6%), a sign that Black patients had more advanced disease. Black patients also had shorter overall survival (7.0 years, 95% confidence interval [CI], 6.7–7.3 years) vs. White patients (9.1 years, 95% CI, 9.0–9.3 years, P < .001).
“This finding could be due to underlying biologic differences in the pathology of CLL, when comparing patients across racial groups,” Dr. Stephens said. “Additionally, there could be differences in access to care. Notably, there are fewer racial minorities enrolled in clinical trials, and perhaps we are not individualizing therapy for unique biologic factors seen in CLL affecting racial minorities.”
Other factors also could be at play. Black patients were more likely than were White patients to have one or more comorbidities (27.9% vs. 21.3%, P < .001), lack insurance (6.6% vs. 2.1%, P < .001) and live in lower-income neighborhoods (47.7% vs. 13.1%, P < .001).
What explains the gap in outcomes? In an interview, study lead author Victoria Vardell, MD, of the University of Utah, Salt Lake City, noted that researchers often attribute worse medical outcomes in Black patients to economic and social disparities.
“However, when we adjusted for a number of surrogate markers of health care access, including income, comorbidities, and location, among others, this disparity remained. That indicates that this may be a more complex problem in CLL in particular. Certainly, we cannot adjust for all the socioeconomic strain placed on Black Americans, including those with CLL, but there may be molecular features related to ancestry or environmental exposures that also play a role,” Dr. Vardell said.
She added that “the high cost and difficulty obtaining many novel therapies, particularly in the clinical trial setting, places significantly higher burdens on already disadvantaged populations.”
There is some good news in the new report. “Promisingly, our data suggest that the survival disparity between White and Black patients with CLL may be improving, particularly within the last 5 years, though longer follow-up is needed to confirm significance,” the researchers reported.
Alessandra Ferrajoli, MD, of M.D. Anderson Cancer Center, Houston, who has studied racial disparities in CLL, praised the study in an interview. As she noted, it examines an impressively large population.
The explanations for the disparities are still elusive, she said, although it seems clear there are multiple factors at play. “We don’t know if the disease has the same characteristics in African-Americans as in Whites,” Dr. Ferrajoli said. However, she noted, there’s “no indication that the response to treatment is different according to race.”
Moving forward, she said, the study findings “reinforce the fact that we need to pay attention to this population and be quite attentive to their characteristics.”
No study funding was reported. The authors and Dr. Ferrajoli have no disclosures.
The findings, published in the American Journal of Hematology, hint that the racial disparity has shrunk over time, especially within the first few years of the targeted-therapy era. Still, “Black patients had a shorter median overall survival of 7 years compared to 9 years for White patients,” study coauthor Deborah Stephens, DO, of the University of Utah Huntsman Cancer Institute, said in an interview. “Clearly, more research is needed to tease out the biologic or economic barriers to achieving prolonged survival.”
As the researchers noted, CLL is far more common among White patients (5.1 cases per 100,000) than other races (Black patients: 3.2 cases per 100,000; Hispanic patients: 2.1 cases per 100,000; Asian American patients: 1.1 per 100,000). In total, non-White patients make up just 11%-13% of CLL cases in the United States.
According to Dr. Stephens, “little is known or published” about Black patients with CLL, “and it is still a mystery why fewer patients that are Black develop CLL and why this group would have shorter survival.”
Dr. Stephens and colleagues launched the new study – the largest of its kind to date – to understand disparities between White and Black patients over most of the past 20 years. The researchers especially wanted to analyze trends during the last decade, when targeted therapies revolutionized treatment of the disease.
The study authors analyzed data in the National Cancer Database for 97,804 patients diagnosed from 2004 to 2018 (90.7% White, 7.6% Black, 0.6% Asian, 1.1% other). Of patients who reported ethnicity (n = 93,555), 2.6% were Hispanic.
Black patients were more likely to have begun CLL therapy at diagnosis (35.9%) than were White patients (23.6%), a sign that Black patients had more advanced disease. Black patients also had shorter overall survival (7.0 years, 95% confidence interval [CI], 6.7–7.3 years) vs. White patients (9.1 years, 95% CI, 9.0–9.3 years, P < .001).
“This finding could be due to underlying biologic differences in the pathology of CLL, when comparing patients across racial groups,” Dr. Stephens said. “Additionally, there could be differences in access to care. Notably, there are fewer racial minorities enrolled in clinical trials, and perhaps we are not individualizing therapy for unique biologic factors seen in CLL affecting racial minorities.”
Other factors also could be at play. Black patients were more likely than were White patients to have one or more comorbidities (27.9% vs. 21.3%, P < .001), lack insurance (6.6% vs. 2.1%, P < .001) and live in lower-income neighborhoods (47.7% vs. 13.1%, P < .001).
What explains the gap in outcomes? In an interview, study lead author Victoria Vardell, MD, of the University of Utah, Salt Lake City, noted that researchers often attribute worse medical outcomes in Black patients to economic and social disparities.
“However, when we adjusted for a number of surrogate markers of health care access, including income, comorbidities, and location, among others, this disparity remained. That indicates that this may be a more complex problem in CLL in particular. Certainly, we cannot adjust for all the socioeconomic strain placed on Black Americans, including those with CLL, but there may be molecular features related to ancestry or environmental exposures that also play a role,” Dr. Vardell said.
She added that “the high cost and difficulty obtaining many novel therapies, particularly in the clinical trial setting, places significantly higher burdens on already disadvantaged populations.”
There is some good news in the new report. “Promisingly, our data suggest that the survival disparity between White and Black patients with CLL may be improving, particularly within the last 5 years, though longer follow-up is needed to confirm significance,” the researchers reported.
Alessandra Ferrajoli, MD, of M.D. Anderson Cancer Center, Houston, who has studied racial disparities in CLL, praised the study in an interview. As she noted, it examines an impressively large population.
The explanations for the disparities are still elusive, she said, although it seems clear there are multiple factors at play. “We don’t know if the disease has the same characteristics in African-Americans as in Whites,” Dr. Ferrajoli said. However, she noted, there’s “no indication that the response to treatment is different according to race.”
Moving forward, she said, the study findings “reinforce the fact that we need to pay attention to this population and be quite attentive to their characteristics.”
No study funding was reported. The authors and Dr. Ferrajoli have no disclosures.
FROM AMERICAN JOURNAL OF HEMATOLOGY
Surgeons, intensivists earn more than do colleagues from private insurance
General and orthopedic surgeons and intensivists earn the highest net reimbursements from private U.S. insurers, a new report estimates.
On average in 2021, they were paid $5.8 million, $4.9 million, and $3.3 million, respectively, according to figures compiled by AMN Healthcare, a Dallas-based health staffing company.
None of 15 other physician specialties topped $3 million in net reimbursement on average, and three – dermatology, pediatrics, and family medicine – didn’t reach $1 million.
The report doesn’t include data about reimbursement from Medicare and Medicaid, and its numbers assume that 50% of insurance claims are denied. Denial rates differ from practice to practice.
Still, the findings offer a “benchmark tool” to help clinicians understand how they rank against their peers, Linda Murphy, president of AMN Healthcare’s Revenue Cycle Solutions division, said in an interview.
This is the first year that the company has calculated physician reimbursement levels by using claim and clearinghouse data, Ms. Murphy said. Previously, a division of the firm compiled data by surveying chief financial officers from hospitals.
The report’s estimate that insurers deny 50% of claims is “conservative,” Ms. Murphy said. Miscoding is a significant factor behind that number.
The estimated 2021 net private insurance reimbursements by specialty for direct services, assuming a 50% denial rate:
- Anesthesiology: $1,665,510
- Cardiology: $1,703,013
- Critical Care (intensivist): $3,338,656
- Dermatology: $729,107
- Family medicine: $697,094
- Gastroenterology: $2,765,110
- Internal medicine: $1,297,200
- Neurology: $1,390,181
- Obstetrician/gynecology: $1,880,888
- Otolaryngology: $2,095,277
- Pediatrics: $661,552
- Psychiatry: $1,348,730
- Pulmonology: $1,561,617
- Radiology: $1,015,750
- Rheumatology: $1,705,140
- General surgery: $5,834,508
- Orthopedic surgery: $4,904,757
- Urology: $2,943,381
Among 18 physician specialties overall, the report estimated that the average net reimbursement in 2021 was $1.9 million.
The report also estimated that the net reimbursement amounts at $875,140 for certified registered nurse anesthetists and $388,696 for nurse practitioners.
Surprisingly, Ms. Murphy said, there’s “a really large swing” among reimbursement levels for individual specialties. The quartile of cardiologists with the lowest level of reimbursement, for example, submitted $2.1 million in claims in 2021, netting about $1 million at a 50% denial rate versus the $7.3 million made by those in the highest quartile, netting about $3.6 million.
The gap seems to be due to regional variations, she said, adding that a rural cardiologist will have different billing practices than does one practicing in New York City.
The quartile of general surgeons with the highest reimbursement levels billed for $21.1 million on average in 2021, making about $10.5 million at a 50% denial rate. The lowest quartile billed for $5.5 million, making about $2.7 million at a 50% denial rate.
The report noted that primary care physicians – that is, family medicine, internal medicine, and pediatrics specialists – have much lower levels of reimbursement, compared with most other specialties. But the work of primary care physicians “may lead to considerable ‘downstream revenue’ through the hospital admissions, tests and treatment they order.”
A previous analysis by a division of AMN Healthcare found that primary care physicians, on average, generate $2,113,273 a year in net annual revenue for their affiliated hospitals, nearing the $2,446,429 in net annual hospital revenue generated by specialists.
AMN Healthcare is preparing another report that will examine Medicare reimbursements, Ms. Murphy said. According to the new report, payments by nonprivate insurers amount to about one-third of the total amount of reimbursement by commercial insurers.
A version of this article originally appeared on Medscape.com.
General and orthopedic surgeons and intensivists earn the highest net reimbursements from private U.S. insurers, a new report estimates.
On average in 2021, they were paid $5.8 million, $4.9 million, and $3.3 million, respectively, according to figures compiled by AMN Healthcare, a Dallas-based health staffing company.
None of 15 other physician specialties topped $3 million in net reimbursement on average, and three – dermatology, pediatrics, and family medicine – didn’t reach $1 million.
The report doesn’t include data about reimbursement from Medicare and Medicaid, and its numbers assume that 50% of insurance claims are denied. Denial rates differ from practice to practice.
Still, the findings offer a “benchmark tool” to help clinicians understand how they rank against their peers, Linda Murphy, president of AMN Healthcare’s Revenue Cycle Solutions division, said in an interview.
This is the first year that the company has calculated physician reimbursement levels by using claim and clearinghouse data, Ms. Murphy said. Previously, a division of the firm compiled data by surveying chief financial officers from hospitals.
The report’s estimate that insurers deny 50% of claims is “conservative,” Ms. Murphy said. Miscoding is a significant factor behind that number.
The estimated 2021 net private insurance reimbursements by specialty for direct services, assuming a 50% denial rate:
- Anesthesiology: $1,665,510
- Cardiology: $1,703,013
- Critical Care (intensivist): $3,338,656
- Dermatology: $729,107
- Family medicine: $697,094
- Gastroenterology: $2,765,110
- Internal medicine: $1,297,200
- Neurology: $1,390,181
- Obstetrician/gynecology: $1,880,888
- Otolaryngology: $2,095,277
- Pediatrics: $661,552
- Psychiatry: $1,348,730
- Pulmonology: $1,561,617
- Radiology: $1,015,750
- Rheumatology: $1,705,140
- General surgery: $5,834,508
- Orthopedic surgery: $4,904,757
- Urology: $2,943,381
Among 18 physician specialties overall, the report estimated that the average net reimbursement in 2021 was $1.9 million.
The report also estimated that the net reimbursement amounts at $875,140 for certified registered nurse anesthetists and $388,696 for nurse practitioners.
Surprisingly, Ms. Murphy said, there’s “a really large swing” among reimbursement levels for individual specialties. The quartile of cardiologists with the lowest level of reimbursement, for example, submitted $2.1 million in claims in 2021, netting about $1 million at a 50% denial rate versus the $7.3 million made by those in the highest quartile, netting about $3.6 million.
The gap seems to be due to regional variations, she said, adding that a rural cardiologist will have different billing practices than does one practicing in New York City.
The quartile of general surgeons with the highest reimbursement levels billed for $21.1 million on average in 2021, making about $10.5 million at a 50% denial rate. The lowest quartile billed for $5.5 million, making about $2.7 million at a 50% denial rate.
The report noted that primary care physicians – that is, family medicine, internal medicine, and pediatrics specialists – have much lower levels of reimbursement, compared with most other specialties. But the work of primary care physicians “may lead to considerable ‘downstream revenue’ through the hospital admissions, tests and treatment they order.”
A previous analysis by a division of AMN Healthcare found that primary care physicians, on average, generate $2,113,273 a year in net annual revenue for their affiliated hospitals, nearing the $2,446,429 in net annual hospital revenue generated by specialists.
AMN Healthcare is preparing another report that will examine Medicare reimbursements, Ms. Murphy said. According to the new report, payments by nonprivate insurers amount to about one-third of the total amount of reimbursement by commercial insurers.
A version of this article originally appeared on Medscape.com.
General and orthopedic surgeons and intensivists earn the highest net reimbursements from private U.S. insurers, a new report estimates.
On average in 2021, they were paid $5.8 million, $4.9 million, and $3.3 million, respectively, according to figures compiled by AMN Healthcare, a Dallas-based health staffing company.
None of 15 other physician specialties topped $3 million in net reimbursement on average, and three – dermatology, pediatrics, and family medicine – didn’t reach $1 million.
The report doesn’t include data about reimbursement from Medicare and Medicaid, and its numbers assume that 50% of insurance claims are denied. Denial rates differ from practice to practice.
Still, the findings offer a “benchmark tool” to help clinicians understand how they rank against their peers, Linda Murphy, president of AMN Healthcare’s Revenue Cycle Solutions division, said in an interview.
This is the first year that the company has calculated physician reimbursement levels by using claim and clearinghouse data, Ms. Murphy said. Previously, a division of the firm compiled data by surveying chief financial officers from hospitals.
The report’s estimate that insurers deny 50% of claims is “conservative,” Ms. Murphy said. Miscoding is a significant factor behind that number.
The estimated 2021 net private insurance reimbursements by specialty for direct services, assuming a 50% denial rate:
- Anesthesiology: $1,665,510
- Cardiology: $1,703,013
- Critical Care (intensivist): $3,338,656
- Dermatology: $729,107
- Family medicine: $697,094
- Gastroenterology: $2,765,110
- Internal medicine: $1,297,200
- Neurology: $1,390,181
- Obstetrician/gynecology: $1,880,888
- Otolaryngology: $2,095,277
- Pediatrics: $661,552
- Psychiatry: $1,348,730
- Pulmonology: $1,561,617
- Radiology: $1,015,750
- Rheumatology: $1,705,140
- General surgery: $5,834,508
- Orthopedic surgery: $4,904,757
- Urology: $2,943,381
Among 18 physician specialties overall, the report estimated that the average net reimbursement in 2021 was $1.9 million.
The report also estimated that the net reimbursement amounts at $875,140 for certified registered nurse anesthetists and $388,696 for nurse practitioners.
Surprisingly, Ms. Murphy said, there’s “a really large swing” among reimbursement levels for individual specialties. The quartile of cardiologists with the lowest level of reimbursement, for example, submitted $2.1 million in claims in 2021, netting about $1 million at a 50% denial rate versus the $7.3 million made by those in the highest quartile, netting about $3.6 million.
The gap seems to be due to regional variations, she said, adding that a rural cardiologist will have different billing practices than does one practicing in New York City.
The quartile of general surgeons with the highest reimbursement levels billed for $21.1 million on average in 2021, making about $10.5 million at a 50% denial rate. The lowest quartile billed for $5.5 million, making about $2.7 million at a 50% denial rate.
The report noted that primary care physicians – that is, family medicine, internal medicine, and pediatrics specialists – have much lower levels of reimbursement, compared with most other specialties. But the work of primary care physicians “may lead to considerable ‘downstream revenue’ through the hospital admissions, tests and treatment they order.”
A previous analysis by a division of AMN Healthcare found that primary care physicians, on average, generate $2,113,273 a year in net annual revenue for their affiliated hospitals, nearing the $2,446,429 in net annual hospital revenue generated by specialists.
AMN Healthcare is preparing another report that will examine Medicare reimbursements, Ms. Murphy said. According to the new report, payments by nonprivate insurers amount to about one-third of the total amount of reimbursement by commercial insurers.
A version of this article originally appeared on Medscape.com.
From Beirut to frontline hematology research
“If we start treatment earlier, in the smoldering phase, maybe there is a chance of actually curing the disease and completely getting rid of it,” said Dr. Mouhieddine, 31, a research fellow at the Icahn School of Medicine at Mount Sinai, in New York. “We haven’t proven that yet, and it’s going to take years before we’re able to prove it. I’m hoping to be one of those spearheading the initiative.”
As he develops clinical trials, the young physician scientist has another focus: A deeply personal connection to the very disease he’s trying to cure. Last year Dr. Mouhieddine diagnosed his aunt back in Lebanon with multiple myeloma. “I have always been close to her, and I’m like her son,” he said, and her situation is especially scary because she lives in a country where treatment options are limited.
Dr. Mouhieddine was born and raised in Beirut, the son of a sports journalist father and a mother who worked in a bank. Lebanon’s civil war ended in 1990, shortly before his birth, but political instability returned when he was a child.
“Everything was a disaster,” he recalled. “There was a period of time when there were bombs throughout the city because certain politicians were being targeted. I remember when groups of people would have gunfights in the street.”
Dr. Mouhieddine attended the American University of Beirut, then after college and medical school there, he headed to the United States.
“I wanted to make a difference in medicine. And I knew that if I stayed back home, I wouldn’t be able to,” he said. Fortunately, “everybody has made me feel that I really belong here, and I’ve never felt like I’m an outsider.”
Early on, as he went through fellowships and residency, he developed an interest in multiple myeloma.
Ajai Chari, MD, a colleague of Dr. Mouhieddine’s at Icahn School of Medicine, said in an interview, “I remember meeting him at a conference before he had even started an internal medicine residency, let alone a hematology oncology fellowship. He was already certain he wanted to work in multiple myeloma, due to his work at Dana-Farber Cancer Institute.”
Myeloma was especially intriguing to Dr. Mouhieddine because of the rapid rate of progress in treating the disease. “Over the past 10 years, the myeloma field has advanced at such an extremely fast pace, more than any other cancer,” he said. “Maybe 15 years ago, you would tell someone with newly diagnosed myeloma that they had a chance for an average of another 2 years. Now, we tell patients they have 10 years to live on average, which means you could live 15 or 20 years. That alone was astounding to me and piqued my interest in myeloma.”
At the same time, smoldering myeloma – which can be discovered during routine blood work – remains little understood. As the National Cancer Institute explains, “smoldering myeloma is a precancerous condition that alters certain proteins in blood and/or increases plasma cells in bone marrow, but it does not cause symptoms of disease. About half of those diagnosed with the condition, however, will develop multiple myeloma within 5 years.”
“If we understand what drives smoldering myeloma, we may be able to prevent it from progressing to its active form,” said hematologist oncologist Samir Parekh, MD, who works with Dr. Mouhieddine at Icahn School of Medicine. “Or at the minimum, we could better predict who will progress so we can tailor therapy for high-risk patients and minimize toxicity by not overtreating patients who may not need therapy.”
Dr. Mouhieddine’s current work is focusing on developing clinical trials to test whether immunotherapy can snuff out myeloma when it’s at the smoldering stage, “before anything bad happens.
“If a myeloma patient comes in with renal failure, and we treat the myeloma at that stage, it doesn’t mean that the patient’s kidneys are gonna go back to normal. A lot of the damage can be permanent,” he said. “Even when you treat multiple myeloma, and it goes into remission, it ends up coming back. And you just have to go from one therapy to the other.”
In contrast, a successful treatment for smoldering myeloma would prevent progression to the full disease. In other words, it would be a cure – which is now elusive.
Specifically, Dr. Mouhieddine hopes to test whether bispecific antibodies, a type of immunotherapy that enlists the body’s T cells to kill myeloma cells, will be effective in the smoldering phase. Bispecific antibodies are now being explored as treatments for full multiple myeloma when the immune system is weaker, he said, and they may be even more effective earlier, when the body is better equipped to fight off the disease.
Dr. Mouhieddine hopes better treatments for multiple myeloma itself will help save his 64-year-old aunt Hassana, back in Beirut. He diagnosed her in 2022 after she told him that she felt tired all the time and underwent various tests. The woman he calls his “second mom” is doing well, despite struggles to buy medication due to the lack of access to bank funds in Lebanon.
“I’m always going to be afraid that the disease is going to progress or come back at some point,” he said. “Lebanon doesn’t have as many options as people in the U.S. do. Once you exhaust your first option, and maybe your second option, then you don’t have any other options. Here, we have outpatients who exhaust option number 15 and go to option number 16. That’s definitely not the case over there.”
For now, Dr. Mouhieddine is treating patients and working to launch clinical trials into smoldering myeloma. “His work ethic is incredible,” said his colleague, Dr. Chari. “He has seen multiple projects to publication, and he develops deep connections with his patients and follows up on their care whether or not he is in clinic on a particular day.”
Dr. Parekh, another colleague, said Dr. Mouhieddine can even be a role model. “Other trainees may benefit from thinking about their career early on and exploring both lab and clinical research projects, so that they can develop the necessary experience to be competitive in academia later on.”
His workload can a burden for Dr. Mouhieddine, who is Muslim. He expressed regret that his busy schedule does not always permit him to fast during Ramadan. On a nonmedical front, his recent efforts have paid off. In March 2023 Dr. Mouhieddine became a U.S. citizen.
“It’s surreal,” he said, “but also a dream come true. I feel very grateful, like it’s like an appreciation of who I am, what I’ve done, and what I can do for this country.”
“If we start treatment earlier, in the smoldering phase, maybe there is a chance of actually curing the disease and completely getting rid of it,” said Dr. Mouhieddine, 31, a research fellow at the Icahn School of Medicine at Mount Sinai, in New York. “We haven’t proven that yet, and it’s going to take years before we’re able to prove it. I’m hoping to be one of those spearheading the initiative.”
As he develops clinical trials, the young physician scientist has another focus: A deeply personal connection to the very disease he’s trying to cure. Last year Dr. Mouhieddine diagnosed his aunt back in Lebanon with multiple myeloma. “I have always been close to her, and I’m like her son,” he said, and her situation is especially scary because she lives in a country where treatment options are limited.
Dr. Mouhieddine was born and raised in Beirut, the son of a sports journalist father and a mother who worked in a bank. Lebanon’s civil war ended in 1990, shortly before his birth, but political instability returned when he was a child.
“Everything was a disaster,” he recalled. “There was a period of time when there were bombs throughout the city because certain politicians were being targeted. I remember when groups of people would have gunfights in the street.”
Dr. Mouhieddine attended the American University of Beirut, then after college and medical school there, he headed to the United States.
“I wanted to make a difference in medicine. And I knew that if I stayed back home, I wouldn’t be able to,” he said. Fortunately, “everybody has made me feel that I really belong here, and I’ve never felt like I’m an outsider.”
Early on, as he went through fellowships and residency, he developed an interest in multiple myeloma.
Ajai Chari, MD, a colleague of Dr. Mouhieddine’s at Icahn School of Medicine, said in an interview, “I remember meeting him at a conference before he had even started an internal medicine residency, let alone a hematology oncology fellowship. He was already certain he wanted to work in multiple myeloma, due to his work at Dana-Farber Cancer Institute.”
Myeloma was especially intriguing to Dr. Mouhieddine because of the rapid rate of progress in treating the disease. “Over the past 10 years, the myeloma field has advanced at such an extremely fast pace, more than any other cancer,” he said. “Maybe 15 years ago, you would tell someone with newly diagnosed myeloma that they had a chance for an average of another 2 years. Now, we tell patients they have 10 years to live on average, which means you could live 15 or 20 years. That alone was astounding to me and piqued my interest in myeloma.”
At the same time, smoldering myeloma – which can be discovered during routine blood work – remains little understood. As the National Cancer Institute explains, “smoldering myeloma is a precancerous condition that alters certain proteins in blood and/or increases plasma cells in bone marrow, but it does not cause symptoms of disease. About half of those diagnosed with the condition, however, will develop multiple myeloma within 5 years.”
“If we understand what drives smoldering myeloma, we may be able to prevent it from progressing to its active form,” said hematologist oncologist Samir Parekh, MD, who works with Dr. Mouhieddine at Icahn School of Medicine. “Or at the minimum, we could better predict who will progress so we can tailor therapy for high-risk patients and minimize toxicity by not overtreating patients who may not need therapy.”
Dr. Mouhieddine’s current work is focusing on developing clinical trials to test whether immunotherapy can snuff out myeloma when it’s at the smoldering stage, “before anything bad happens.
“If a myeloma patient comes in with renal failure, and we treat the myeloma at that stage, it doesn’t mean that the patient’s kidneys are gonna go back to normal. A lot of the damage can be permanent,” he said. “Even when you treat multiple myeloma, and it goes into remission, it ends up coming back. And you just have to go from one therapy to the other.”
In contrast, a successful treatment for smoldering myeloma would prevent progression to the full disease. In other words, it would be a cure – which is now elusive.
Specifically, Dr. Mouhieddine hopes to test whether bispecific antibodies, a type of immunotherapy that enlists the body’s T cells to kill myeloma cells, will be effective in the smoldering phase. Bispecific antibodies are now being explored as treatments for full multiple myeloma when the immune system is weaker, he said, and they may be even more effective earlier, when the body is better equipped to fight off the disease.
Dr. Mouhieddine hopes better treatments for multiple myeloma itself will help save his 64-year-old aunt Hassana, back in Beirut. He diagnosed her in 2022 after she told him that she felt tired all the time and underwent various tests. The woman he calls his “second mom” is doing well, despite struggles to buy medication due to the lack of access to bank funds in Lebanon.
“I’m always going to be afraid that the disease is going to progress or come back at some point,” he said. “Lebanon doesn’t have as many options as people in the U.S. do. Once you exhaust your first option, and maybe your second option, then you don’t have any other options. Here, we have outpatients who exhaust option number 15 and go to option number 16. That’s definitely not the case over there.”
For now, Dr. Mouhieddine is treating patients and working to launch clinical trials into smoldering myeloma. “His work ethic is incredible,” said his colleague, Dr. Chari. “He has seen multiple projects to publication, and he develops deep connections with his patients and follows up on their care whether or not he is in clinic on a particular day.”
Dr. Parekh, another colleague, said Dr. Mouhieddine can even be a role model. “Other trainees may benefit from thinking about their career early on and exploring both lab and clinical research projects, so that they can develop the necessary experience to be competitive in academia later on.”
His workload can a burden for Dr. Mouhieddine, who is Muslim. He expressed regret that his busy schedule does not always permit him to fast during Ramadan. On a nonmedical front, his recent efforts have paid off. In March 2023 Dr. Mouhieddine became a U.S. citizen.
“It’s surreal,” he said, “but also a dream come true. I feel very grateful, like it’s like an appreciation of who I am, what I’ve done, and what I can do for this country.”
“If we start treatment earlier, in the smoldering phase, maybe there is a chance of actually curing the disease and completely getting rid of it,” said Dr. Mouhieddine, 31, a research fellow at the Icahn School of Medicine at Mount Sinai, in New York. “We haven’t proven that yet, and it’s going to take years before we’re able to prove it. I’m hoping to be one of those spearheading the initiative.”
As he develops clinical trials, the young physician scientist has another focus: A deeply personal connection to the very disease he’s trying to cure. Last year Dr. Mouhieddine diagnosed his aunt back in Lebanon with multiple myeloma. “I have always been close to her, and I’m like her son,” he said, and her situation is especially scary because she lives in a country where treatment options are limited.
Dr. Mouhieddine was born and raised in Beirut, the son of a sports journalist father and a mother who worked in a bank. Lebanon’s civil war ended in 1990, shortly before his birth, but political instability returned when he was a child.
“Everything was a disaster,” he recalled. “There was a period of time when there were bombs throughout the city because certain politicians were being targeted. I remember when groups of people would have gunfights in the street.”
Dr. Mouhieddine attended the American University of Beirut, then after college and medical school there, he headed to the United States.
“I wanted to make a difference in medicine. And I knew that if I stayed back home, I wouldn’t be able to,” he said. Fortunately, “everybody has made me feel that I really belong here, and I’ve never felt like I’m an outsider.”
Early on, as he went through fellowships and residency, he developed an interest in multiple myeloma.
Ajai Chari, MD, a colleague of Dr. Mouhieddine’s at Icahn School of Medicine, said in an interview, “I remember meeting him at a conference before he had even started an internal medicine residency, let alone a hematology oncology fellowship. He was already certain he wanted to work in multiple myeloma, due to his work at Dana-Farber Cancer Institute.”
Myeloma was especially intriguing to Dr. Mouhieddine because of the rapid rate of progress in treating the disease. “Over the past 10 years, the myeloma field has advanced at such an extremely fast pace, more than any other cancer,” he said. “Maybe 15 years ago, you would tell someone with newly diagnosed myeloma that they had a chance for an average of another 2 years. Now, we tell patients they have 10 years to live on average, which means you could live 15 or 20 years. That alone was astounding to me and piqued my interest in myeloma.”
At the same time, smoldering myeloma – which can be discovered during routine blood work – remains little understood. As the National Cancer Institute explains, “smoldering myeloma is a precancerous condition that alters certain proteins in blood and/or increases plasma cells in bone marrow, but it does not cause symptoms of disease. About half of those diagnosed with the condition, however, will develop multiple myeloma within 5 years.”
“If we understand what drives smoldering myeloma, we may be able to prevent it from progressing to its active form,” said hematologist oncologist Samir Parekh, MD, who works with Dr. Mouhieddine at Icahn School of Medicine. “Or at the minimum, we could better predict who will progress so we can tailor therapy for high-risk patients and minimize toxicity by not overtreating patients who may not need therapy.”
Dr. Mouhieddine’s current work is focusing on developing clinical trials to test whether immunotherapy can snuff out myeloma when it’s at the smoldering stage, “before anything bad happens.
“If a myeloma patient comes in with renal failure, and we treat the myeloma at that stage, it doesn’t mean that the patient’s kidneys are gonna go back to normal. A lot of the damage can be permanent,” he said. “Even when you treat multiple myeloma, and it goes into remission, it ends up coming back. And you just have to go from one therapy to the other.”
In contrast, a successful treatment for smoldering myeloma would prevent progression to the full disease. In other words, it would be a cure – which is now elusive.
Specifically, Dr. Mouhieddine hopes to test whether bispecific antibodies, a type of immunotherapy that enlists the body’s T cells to kill myeloma cells, will be effective in the smoldering phase. Bispecific antibodies are now being explored as treatments for full multiple myeloma when the immune system is weaker, he said, and they may be even more effective earlier, when the body is better equipped to fight off the disease.
Dr. Mouhieddine hopes better treatments for multiple myeloma itself will help save his 64-year-old aunt Hassana, back in Beirut. He diagnosed her in 2022 after she told him that she felt tired all the time and underwent various tests. The woman he calls his “second mom” is doing well, despite struggles to buy medication due to the lack of access to bank funds in Lebanon.
“I’m always going to be afraid that the disease is going to progress or come back at some point,” he said. “Lebanon doesn’t have as many options as people in the U.S. do. Once you exhaust your first option, and maybe your second option, then you don’t have any other options. Here, we have outpatients who exhaust option number 15 and go to option number 16. That’s definitely not the case over there.”
For now, Dr. Mouhieddine is treating patients and working to launch clinical trials into smoldering myeloma. “His work ethic is incredible,” said his colleague, Dr. Chari. “He has seen multiple projects to publication, and he develops deep connections with his patients and follows up on their care whether or not he is in clinic on a particular day.”
Dr. Parekh, another colleague, said Dr. Mouhieddine can even be a role model. “Other trainees may benefit from thinking about their career early on and exploring both lab and clinical research projects, so that they can develop the necessary experience to be competitive in academia later on.”
His workload can a burden for Dr. Mouhieddine, who is Muslim. He expressed regret that his busy schedule does not always permit him to fast during Ramadan. On a nonmedical front, his recent efforts have paid off. In March 2023 Dr. Mouhieddine became a U.S. citizen.
“It’s surreal,” he said, “but also a dream come true. I feel very grateful, like it’s like an appreciation of who I am, what I’ve done, and what I can do for this country.”
Study gives new insight into timing of combo treatment in metastatic NSCLC
However, patients still fared poorly on average since overall survival remained low and didn’t change significantly.
While not conclusive, the new research – released at European Lung Cancer Congress 2023 – offers early insight into the best timing for the experimental combination treatment, study coauthor Yanyan Lou, MD, PhD, an oncologist at Mayo Clinic in Jacksonville, Fla., said in an interview.
The wide availability of radiation therapy could also allow the therapy to be administered even in regions with poor access to sophisticated medical care, she said. “Radiation is a very feasible approach that pretty much everybody in your community can get.”
Radiotherapy is typically not added to immunotherapy in patients with non–small cell lung cancer. But “there has been recent interest in the combination: Would tumor necrosis from radiation enhance the immunogenicity of the tumor and thus enhance the effect of immunotherapy?” oncologist Toby Campbell, MD, of University of Wisconsin–Madison, said in an interview.
Research has indeed suggested that the treatments may have a synergistic effect, he said, and it’s clear that “strategies to try and increase immunogenicity are an important area to investigate.”
But he cautioned that “we have a long way to go to understanding how immunogenicity works and how the gut microbiome, tumor, immunotherapy, and the immune system interact with one another.”
For the new study, researchers retrospectively analyzed cases of 225 patients with metastatic non–small cell lung cancer (male = 56%, median age = 68, 79% adenocarcinoma) who were treated with immunotherapy at Mayo Clinic–Jacksonville from 2011 to 2022. The study excluded those who received targeted therapy or prior concurrent chemoradiotherapy and durvalumab.
The most common metastases were bone and central nervous system types (41% and 25%, respectively). Fifty-six percent of patients received radiotherapy before or during immunotherapy. Another 27% never received radiotherapy, and 17% received it after immunotherapy was discontinued.
Common types of immunotherapy included pembrolizumab (78%), nivolumab (14%), and atezolizumab (12%).
Overall, the researchers found no statistically significant differences in various outcomes between patients who received radiotherapy before or during immunotherapy compared with those who didn’t get radiotherapy (progression-free survival: 5.9 vs. 5.5 months, P = .66; overall survival: 16.9 vs. 13.1 months, P = .84; immune-related adverse events: 26.2% vs. 34.4%, P = .24).
However, the researchers found that progression-free survival was significantly higher in one group: Those who received radiotherapy 1-12 months before immunotherapy vs. those who received it less than 1 month before (12.6 vs. 4.2 months, hazard ratio [HR], 0.46, 95% confidence interval [CI], 0.26-0.83, P = .005,) and those who never received radiotherapy (12.6 vs. 5.5 months, HR, 0.56, 95% CI, 0.36-0.89, P = .0197).
There wasn’t a statistically significant difference in overall survival.
The small number of subjects and the variation in treatment protocols may have prevented the study from revealing a survival benefit, Dr. Lou said.
As for adverse effects, she said a preliminary analysis didn’t turn up any.
It’s not clear why a 1- to 12-month gap between radiotherapy and immunotherapy may be most effective, she said. Moving forward, “we need validate this in a large cohort,” she noted.
In regard to cost, immunotherapy is notoriously expensive. Pembrolizumab, for example, has a list price of $10,897 per 200-mg dose given every 3 weeks, and patients may take the drug for a year or two.
Dr. Campbell, who didn’t take part in the new study, said it suggests that research into radiation-immunotherapy combination treatment may be worthwhile.
No funding was reported. The study authors and Dr. Campbell reported no disclosures.
However, patients still fared poorly on average since overall survival remained low and didn’t change significantly.
While not conclusive, the new research – released at European Lung Cancer Congress 2023 – offers early insight into the best timing for the experimental combination treatment, study coauthor Yanyan Lou, MD, PhD, an oncologist at Mayo Clinic in Jacksonville, Fla., said in an interview.
The wide availability of radiation therapy could also allow the therapy to be administered even in regions with poor access to sophisticated medical care, she said. “Radiation is a very feasible approach that pretty much everybody in your community can get.”
Radiotherapy is typically not added to immunotherapy in patients with non–small cell lung cancer. But “there has been recent interest in the combination: Would tumor necrosis from radiation enhance the immunogenicity of the tumor and thus enhance the effect of immunotherapy?” oncologist Toby Campbell, MD, of University of Wisconsin–Madison, said in an interview.
Research has indeed suggested that the treatments may have a synergistic effect, he said, and it’s clear that “strategies to try and increase immunogenicity are an important area to investigate.”
But he cautioned that “we have a long way to go to understanding how immunogenicity works and how the gut microbiome, tumor, immunotherapy, and the immune system interact with one another.”
For the new study, researchers retrospectively analyzed cases of 225 patients with metastatic non–small cell lung cancer (male = 56%, median age = 68, 79% adenocarcinoma) who were treated with immunotherapy at Mayo Clinic–Jacksonville from 2011 to 2022. The study excluded those who received targeted therapy or prior concurrent chemoradiotherapy and durvalumab.
The most common metastases were bone and central nervous system types (41% and 25%, respectively). Fifty-six percent of patients received radiotherapy before or during immunotherapy. Another 27% never received radiotherapy, and 17% received it after immunotherapy was discontinued.
Common types of immunotherapy included pembrolizumab (78%), nivolumab (14%), and atezolizumab (12%).
Overall, the researchers found no statistically significant differences in various outcomes between patients who received radiotherapy before or during immunotherapy compared with those who didn’t get radiotherapy (progression-free survival: 5.9 vs. 5.5 months, P = .66; overall survival: 16.9 vs. 13.1 months, P = .84; immune-related adverse events: 26.2% vs. 34.4%, P = .24).
However, the researchers found that progression-free survival was significantly higher in one group: Those who received radiotherapy 1-12 months before immunotherapy vs. those who received it less than 1 month before (12.6 vs. 4.2 months, hazard ratio [HR], 0.46, 95% confidence interval [CI], 0.26-0.83, P = .005,) and those who never received radiotherapy (12.6 vs. 5.5 months, HR, 0.56, 95% CI, 0.36-0.89, P = .0197).
There wasn’t a statistically significant difference in overall survival.
The small number of subjects and the variation in treatment protocols may have prevented the study from revealing a survival benefit, Dr. Lou said.
As for adverse effects, she said a preliminary analysis didn’t turn up any.
It’s not clear why a 1- to 12-month gap between radiotherapy and immunotherapy may be most effective, she said. Moving forward, “we need validate this in a large cohort,” she noted.
In regard to cost, immunotherapy is notoriously expensive. Pembrolizumab, for example, has a list price of $10,897 per 200-mg dose given every 3 weeks, and patients may take the drug for a year or two.
Dr. Campbell, who didn’t take part in the new study, said it suggests that research into radiation-immunotherapy combination treatment may be worthwhile.
No funding was reported. The study authors and Dr. Campbell reported no disclosures.
However, patients still fared poorly on average since overall survival remained low and didn’t change significantly.
While not conclusive, the new research – released at European Lung Cancer Congress 2023 – offers early insight into the best timing for the experimental combination treatment, study coauthor Yanyan Lou, MD, PhD, an oncologist at Mayo Clinic in Jacksonville, Fla., said in an interview.
The wide availability of radiation therapy could also allow the therapy to be administered even in regions with poor access to sophisticated medical care, she said. “Radiation is a very feasible approach that pretty much everybody in your community can get.”
Radiotherapy is typically not added to immunotherapy in patients with non–small cell lung cancer. But “there has been recent interest in the combination: Would tumor necrosis from radiation enhance the immunogenicity of the tumor and thus enhance the effect of immunotherapy?” oncologist Toby Campbell, MD, of University of Wisconsin–Madison, said in an interview.
Research has indeed suggested that the treatments may have a synergistic effect, he said, and it’s clear that “strategies to try and increase immunogenicity are an important area to investigate.”
But he cautioned that “we have a long way to go to understanding how immunogenicity works and how the gut microbiome, tumor, immunotherapy, and the immune system interact with one another.”
For the new study, researchers retrospectively analyzed cases of 225 patients with metastatic non–small cell lung cancer (male = 56%, median age = 68, 79% adenocarcinoma) who were treated with immunotherapy at Mayo Clinic–Jacksonville from 2011 to 2022. The study excluded those who received targeted therapy or prior concurrent chemoradiotherapy and durvalumab.
The most common metastases were bone and central nervous system types (41% and 25%, respectively). Fifty-six percent of patients received radiotherapy before or during immunotherapy. Another 27% never received radiotherapy, and 17% received it after immunotherapy was discontinued.
Common types of immunotherapy included pembrolizumab (78%), nivolumab (14%), and atezolizumab (12%).
Overall, the researchers found no statistically significant differences in various outcomes between patients who received radiotherapy before or during immunotherapy compared with those who didn’t get radiotherapy (progression-free survival: 5.9 vs. 5.5 months, P = .66; overall survival: 16.9 vs. 13.1 months, P = .84; immune-related adverse events: 26.2% vs. 34.4%, P = .24).
However, the researchers found that progression-free survival was significantly higher in one group: Those who received radiotherapy 1-12 months before immunotherapy vs. those who received it less than 1 month before (12.6 vs. 4.2 months, hazard ratio [HR], 0.46, 95% confidence interval [CI], 0.26-0.83, P = .005,) and those who never received radiotherapy (12.6 vs. 5.5 months, HR, 0.56, 95% CI, 0.36-0.89, P = .0197).
There wasn’t a statistically significant difference in overall survival.
The small number of subjects and the variation in treatment protocols may have prevented the study from revealing a survival benefit, Dr. Lou said.
As for adverse effects, she said a preliminary analysis didn’t turn up any.
It’s not clear why a 1- to 12-month gap between radiotherapy and immunotherapy may be most effective, she said. Moving forward, “we need validate this in a large cohort,” she noted.
In regard to cost, immunotherapy is notoriously expensive. Pembrolizumab, for example, has a list price of $10,897 per 200-mg dose given every 3 weeks, and patients may take the drug for a year or two.
Dr. Campbell, who didn’t take part in the new study, said it suggests that research into radiation-immunotherapy combination treatment may be worthwhile.
No funding was reported. The study authors and Dr. Campbell reported no disclosures.
FROM ELCC 2023
Long-term heavy smoking quadruples likelihood of lung cancer vs. less heavy smoking
People who have smoked an average of 1 pack a day for 20-39 years tripled their tumor risk versus less-heavy smokers and 30-fold versus those who never smoked. For those who smoked the equivalent of 1 pack for 40-60 years, or 2 packs for 20-30 years, the risk levels grew by fourfold and 40-fold, respectively. For those who’ve smoked even more, the likelihood of developing lung cancer is high, but the risk remains stable and doesn’t grow more over time, according to the analysis.
The report, released at the annual European Lung Cancer Congress 2023 meeting, and an earlier related study “underscore the importance of smoking abstinence and early smoking cessation,” said study lead author J. Anthony Nations, MD, MBA, in an interview.
The earlier study, published in JAMA Oncology, relied on a “pack-year” analysis to evaluate the risk of lung cancer in smokers. A pack-year refers to the cigarette use of a person who smoked a pack a day for 1 year. It’s the equivalent of smoking half a pack for 2 years or 2 packs for 6 months.
By this measure, a smoker with 20 pack-years of cigarette use smoked the equivalent of a pack a day for 20 years or 2 packs a day for 10 years. U.S. guidelines recommend annual low-dose CT lung cancer screening in adults who are aged 50-80, have more than 20 pack-years of tobacco exposure, and either currently smoke or quit within the last 15 years.
The JAMA Oncology report “showed that, compared with never-smokers, current heavy and nonheavy smokers had [a] 40 and 10 times higher risk of lung cancer, respectively,” said Dr. Nations, who is also a pulmonologist with Washington D.C. Veterans Affairs Medical Center. “A smoking history of greater than 20 pack-years was considered heavy, but current heavy smokers had a median pack-year smoking history of 50 pack-years. This observation prompted us to want to look more closely at pack-year smoking history.”
For the new analysis, researchers tracked 2,505 older adults (mean age, 73 ± 5.7 years; 69% women, 17% African American) in the Cardiovascular Health Study. Of those, 532 were current smokers (18% less than 20 pack-years, 30% 20-39 pack-years, 34% 40–59 pack-years, and 18% greater than 60 pack-years).
Lung cancer occurred in 0.5% of those who never smoked, 5% of those who smoked less than 20 pack-years, 14.6% of those who smoked 20-39 pack-years, 17.7% of those who smoked 40-59 pack-years, and 16.0% for those who smoked more than 60 pack-years. In an analysis adjusted for age, sex, race, and competing risk of death, researchers found that those who smoked less than 20 pack-years were 9.73 times more likely to develop lung cancer than those who never smoked (hazard ratio, 9.73). The HRs of lung cancer versus never-smokers for the other groups were 30.33 (20-39 pack-years), 42.97 (40-59 pack-years), and 46.02 (greater than 60 pack-years.).
“While it was not surprising that the risk of lung cancer in current heavy smokers would be proportionately greater in smokers with higher pack-year smoking history, we were surprised to see that the risk almost plateaued in the heaviest current smokers,” Dr. Nations said.
As for the clinical message from the findings, Dr. Nations said they reveal that quitting smoking makes a difference in lung cancer risk, even after many years of heavy smoking. “Smokers who quit after a 30–pack-year smoking history will not incur the higher risk of those with a 40– or 50–pack-year smoking history.”
The previous JAMA Oncology paper also showed that quitting pays dividends by reducing lung cancer risk. Subjects with at least 20 pack-years of smoking who quit less than 15 years ago nearly halved their excess risk of lung cancer, compared with similar current smokers who didn’t quit.
In an interview, cancer researcher Robert J. Volk, PhD, of the University of Texas MD Anderson Cancer Center, Houston, praised the new analysis but noted that it has limitations: “The sample is fairly small – 532 adults who currently smoke – and the subgroups based on pack-years are even smaller.”
No study funding is reported. The study authors and Dr. Volk reported no disclosures.
*This article was updated on 4/17/23.
People who have smoked an average of 1 pack a day for 20-39 years tripled their tumor risk versus less-heavy smokers and 30-fold versus those who never smoked. For those who smoked the equivalent of 1 pack for 40-60 years, or 2 packs for 20-30 years, the risk levels grew by fourfold and 40-fold, respectively. For those who’ve smoked even more, the likelihood of developing lung cancer is high, but the risk remains stable and doesn’t grow more over time, according to the analysis.
The report, released at the annual European Lung Cancer Congress 2023 meeting, and an earlier related study “underscore the importance of smoking abstinence and early smoking cessation,” said study lead author J. Anthony Nations, MD, MBA, in an interview.
The earlier study, published in JAMA Oncology, relied on a “pack-year” analysis to evaluate the risk of lung cancer in smokers. A pack-year refers to the cigarette use of a person who smoked a pack a day for 1 year. It’s the equivalent of smoking half a pack for 2 years or 2 packs for 6 months.
By this measure, a smoker with 20 pack-years of cigarette use smoked the equivalent of a pack a day for 20 years or 2 packs a day for 10 years. U.S. guidelines recommend annual low-dose CT lung cancer screening in adults who are aged 50-80, have more than 20 pack-years of tobacco exposure, and either currently smoke or quit within the last 15 years.
The JAMA Oncology report “showed that, compared with never-smokers, current heavy and nonheavy smokers had [a] 40 and 10 times higher risk of lung cancer, respectively,” said Dr. Nations, who is also a pulmonologist with Washington D.C. Veterans Affairs Medical Center. “A smoking history of greater than 20 pack-years was considered heavy, but current heavy smokers had a median pack-year smoking history of 50 pack-years. This observation prompted us to want to look more closely at pack-year smoking history.”
For the new analysis, researchers tracked 2,505 older adults (mean age, 73 ± 5.7 years; 69% women, 17% African American) in the Cardiovascular Health Study. Of those, 532 were current smokers (18% less than 20 pack-years, 30% 20-39 pack-years, 34% 40–59 pack-years, and 18% greater than 60 pack-years).
Lung cancer occurred in 0.5% of those who never smoked, 5% of those who smoked less than 20 pack-years, 14.6% of those who smoked 20-39 pack-years, 17.7% of those who smoked 40-59 pack-years, and 16.0% for those who smoked more than 60 pack-years. In an analysis adjusted for age, sex, race, and competing risk of death, researchers found that those who smoked less than 20 pack-years were 9.73 times more likely to develop lung cancer than those who never smoked (hazard ratio, 9.73). The HRs of lung cancer versus never-smokers for the other groups were 30.33 (20-39 pack-years), 42.97 (40-59 pack-years), and 46.02 (greater than 60 pack-years.).
“While it was not surprising that the risk of lung cancer in current heavy smokers would be proportionately greater in smokers with higher pack-year smoking history, we were surprised to see that the risk almost plateaued in the heaviest current smokers,” Dr. Nations said.
As for the clinical message from the findings, Dr. Nations said they reveal that quitting smoking makes a difference in lung cancer risk, even after many years of heavy smoking. “Smokers who quit after a 30–pack-year smoking history will not incur the higher risk of those with a 40– or 50–pack-year smoking history.”
The previous JAMA Oncology paper also showed that quitting pays dividends by reducing lung cancer risk. Subjects with at least 20 pack-years of smoking who quit less than 15 years ago nearly halved their excess risk of lung cancer, compared with similar current smokers who didn’t quit.
In an interview, cancer researcher Robert J. Volk, PhD, of the University of Texas MD Anderson Cancer Center, Houston, praised the new analysis but noted that it has limitations: “The sample is fairly small – 532 adults who currently smoke – and the subgroups based on pack-years are even smaller.”
No study funding is reported. The study authors and Dr. Volk reported no disclosures.
*This article was updated on 4/17/23.
People who have smoked an average of 1 pack a day for 20-39 years tripled their tumor risk versus less-heavy smokers and 30-fold versus those who never smoked. For those who smoked the equivalent of 1 pack for 40-60 years, or 2 packs for 20-30 years, the risk levels grew by fourfold and 40-fold, respectively. For those who’ve smoked even more, the likelihood of developing lung cancer is high, but the risk remains stable and doesn’t grow more over time, according to the analysis.
The report, released at the annual European Lung Cancer Congress 2023 meeting, and an earlier related study “underscore the importance of smoking abstinence and early smoking cessation,” said study lead author J. Anthony Nations, MD, MBA, in an interview.
The earlier study, published in JAMA Oncology, relied on a “pack-year” analysis to evaluate the risk of lung cancer in smokers. A pack-year refers to the cigarette use of a person who smoked a pack a day for 1 year. It’s the equivalent of smoking half a pack for 2 years or 2 packs for 6 months.
By this measure, a smoker with 20 pack-years of cigarette use smoked the equivalent of a pack a day for 20 years or 2 packs a day for 10 years. U.S. guidelines recommend annual low-dose CT lung cancer screening in adults who are aged 50-80, have more than 20 pack-years of tobacco exposure, and either currently smoke or quit within the last 15 years.
The JAMA Oncology report “showed that, compared with never-smokers, current heavy and nonheavy smokers had [a] 40 and 10 times higher risk of lung cancer, respectively,” said Dr. Nations, who is also a pulmonologist with Washington D.C. Veterans Affairs Medical Center. “A smoking history of greater than 20 pack-years was considered heavy, but current heavy smokers had a median pack-year smoking history of 50 pack-years. This observation prompted us to want to look more closely at pack-year smoking history.”
For the new analysis, researchers tracked 2,505 older adults (mean age, 73 ± 5.7 years; 69% women, 17% African American) in the Cardiovascular Health Study. Of those, 532 were current smokers (18% less than 20 pack-years, 30% 20-39 pack-years, 34% 40–59 pack-years, and 18% greater than 60 pack-years).
Lung cancer occurred in 0.5% of those who never smoked, 5% of those who smoked less than 20 pack-years, 14.6% of those who smoked 20-39 pack-years, 17.7% of those who smoked 40-59 pack-years, and 16.0% for those who smoked more than 60 pack-years. In an analysis adjusted for age, sex, race, and competing risk of death, researchers found that those who smoked less than 20 pack-years were 9.73 times more likely to develop lung cancer than those who never smoked (hazard ratio, 9.73). The HRs of lung cancer versus never-smokers for the other groups were 30.33 (20-39 pack-years), 42.97 (40-59 pack-years), and 46.02 (greater than 60 pack-years.).
“While it was not surprising that the risk of lung cancer in current heavy smokers would be proportionately greater in smokers with higher pack-year smoking history, we were surprised to see that the risk almost plateaued in the heaviest current smokers,” Dr. Nations said.
As for the clinical message from the findings, Dr. Nations said they reveal that quitting smoking makes a difference in lung cancer risk, even after many years of heavy smoking. “Smokers who quit after a 30–pack-year smoking history will not incur the higher risk of those with a 40– or 50–pack-year smoking history.”
The previous JAMA Oncology paper also showed that quitting pays dividends by reducing lung cancer risk. Subjects with at least 20 pack-years of smoking who quit less than 15 years ago nearly halved their excess risk of lung cancer, compared with similar current smokers who didn’t quit.
In an interview, cancer researcher Robert J. Volk, PhD, of the University of Texas MD Anderson Cancer Center, Houston, praised the new analysis but noted that it has limitations: “The sample is fairly small – 532 adults who currently smoke – and the subgroups based on pack-years are even smaller.”
No study funding is reported. The study authors and Dr. Volk reported no disclosures.
*This article was updated on 4/17/23.
FROM ELCC 2023
First target doesn’t affect survival in NSCLC with brain metastases
“The findings of our study highlight the importance of adopting a personalized, case-based approach when treating each patient” instead of always treating the brain or lung first, lead author Arvind Kumar, a medical student at Icahn School of Medicine at Mount Sinai, New York, said in an interview.
The study was released at European Lung Cancer Congress 2023.
According to the author, current guidelines recommend treating the brain first in patients with non–small cell lung cancer and a tumor that has spread to the brain.
“Determining whether the brain or body gets treated first depends on where the symptoms are coming from, how severe the symptoms are, how bulky the disease is, and how long the treatment to each is expected to take,” radiation oncologist Henry S. Park, MD, MPH, chief of the thoracic radiotherapy program at Yale University, New Haven, Conn., said in an interview. “Often the brain is treated first since surgery is used for both diagnosis of metastatic disease as well as removal of the brain metastasis, especially if it is causing symptoms. The radiosurgery that follows tends to occur within a day or a few days.”
However, he said, “if the brain disease is small and not causing symptoms, and the lung disease is more problematic, then we will often treat the body first and fit in the brain treatment later.”
For the new study, researchers identified 1,044 patients in the National Cancer Database with non–small cell lung cancer and brain metastases who received systemic therapy plus surgery, brain stereotactic radiosurgery, or lung radiation. All were treated from 2010 to 2019; 79.0% received brain treatment first, and the other 21.0% received lung treatment first.
There was no statistically significant difference in overall survival between those whose brains were treated first and those whose lungs were treated first (hazard ratio, 1.24, 95% confidence interval [CI], 0.91-1.70, P = .17). A propensity score–matched analysis turned up no difference in 5-year survival (38.2% of those whose brains were treated first, 95% CI, 27.5-34.4, vs. 38.0% of those whose lungs were treated first, 95% CI, 29.9-44.7, P = .32.)
“These results were consistent regardless of which combination of treatment modalities the patient received – neurosurgery versus brain stereotactic radiosurgery, thoracic surgery versus thoracic radiation,” the author said.
He cautioned that “our study only included patients who were considered candidates for either surgery or radiation to both the brain and lung. The results of our study should therefore be cautiously interpreted for patients who may have contraindications to such treatment.”
Dr. Park, who didn’t take part in the study, said “the results are consistent with what I would generally expect.”
He added: “The take-home message for clinicians should be that there is no one correct answer in how to manage non–small cell lung cancer with synchronous limited metastatic disease in only the brain. If the brain disease is bulky and/or causes symptoms while the body disease isn’t – or if a biopsy or surgery is required to prove that the patient in fact has metastatic disease – then the brain disease should be treated first. On the other hand, if the body disease is bulky and/or causing symptoms while the brain disease isn’t – and there is no need for surgery but rather only a biopsy of the brain – then the body disease can be treated first.”
No funding was reported. The study authors and Dr. Park reported no financial conflicts or other disclosures.
“The findings of our study highlight the importance of adopting a personalized, case-based approach when treating each patient” instead of always treating the brain or lung first, lead author Arvind Kumar, a medical student at Icahn School of Medicine at Mount Sinai, New York, said in an interview.
The study was released at European Lung Cancer Congress 2023.
According to the author, current guidelines recommend treating the brain first in patients with non–small cell lung cancer and a tumor that has spread to the brain.
“Determining whether the brain or body gets treated first depends on where the symptoms are coming from, how severe the symptoms are, how bulky the disease is, and how long the treatment to each is expected to take,” radiation oncologist Henry S. Park, MD, MPH, chief of the thoracic radiotherapy program at Yale University, New Haven, Conn., said in an interview. “Often the brain is treated first since surgery is used for both diagnosis of metastatic disease as well as removal of the brain metastasis, especially if it is causing symptoms. The radiosurgery that follows tends to occur within a day or a few days.”
However, he said, “if the brain disease is small and not causing symptoms, and the lung disease is more problematic, then we will often treat the body first and fit in the brain treatment later.”
For the new study, researchers identified 1,044 patients in the National Cancer Database with non–small cell lung cancer and brain metastases who received systemic therapy plus surgery, brain stereotactic radiosurgery, or lung radiation. All were treated from 2010 to 2019; 79.0% received brain treatment first, and the other 21.0% received lung treatment first.
There was no statistically significant difference in overall survival between those whose brains were treated first and those whose lungs were treated first (hazard ratio, 1.24, 95% confidence interval [CI], 0.91-1.70, P = .17). A propensity score–matched analysis turned up no difference in 5-year survival (38.2% of those whose brains were treated first, 95% CI, 27.5-34.4, vs. 38.0% of those whose lungs were treated first, 95% CI, 29.9-44.7, P = .32.)
“These results were consistent regardless of which combination of treatment modalities the patient received – neurosurgery versus brain stereotactic radiosurgery, thoracic surgery versus thoracic radiation,” the author said.
He cautioned that “our study only included patients who were considered candidates for either surgery or radiation to both the brain and lung. The results of our study should therefore be cautiously interpreted for patients who may have contraindications to such treatment.”
Dr. Park, who didn’t take part in the study, said “the results are consistent with what I would generally expect.”
He added: “The take-home message for clinicians should be that there is no one correct answer in how to manage non–small cell lung cancer with synchronous limited metastatic disease in only the brain. If the brain disease is bulky and/or causes symptoms while the body disease isn’t – or if a biopsy or surgery is required to prove that the patient in fact has metastatic disease – then the brain disease should be treated first. On the other hand, if the body disease is bulky and/or causing symptoms while the brain disease isn’t – and there is no need for surgery but rather only a biopsy of the brain – then the body disease can be treated first.”
No funding was reported. The study authors and Dr. Park reported no financial conflicts or other disclosures.
“The findings of our study highlight the importance of adopting a personalized, case-based approach when treating each patient” instead of always treating the brain or lung first, lead author Arvind Kumar, a medical student at Icahn School of Medicine at Mount Sinai, New York, said in an interview.
The study was released at European Lung Cancer Congress 2023.
According to the author, current guidelines recommend treating the brain first in patients with non–small cell lung cancer and a tumor that has spread to the brain.
“Determining whether the brain or body gets treated first depends on where the symptoms are coming from, how severe the symptoms are, how bulky the disease is, and how long the treatment to each is expected to take,” radiation oncologist Henry S. Park, MD, MPH, chief of the thoracic radiotherapy program at Yale University, New Haven, Conn., said in an interview. “Often the brain is treated first since surgery is used for both diagnosis of metastatic disease as well as removal of the brain metastasis, especially if it is causing symptoms. The radiosurgery that follows tends to occur within a day or a few days.”
However, he said, “if the brain disease is small and not causing symptoms, and the lung disease is more problematic, then we will often treat the body first and fit in the brain treatment later.”
For the new study, researchers identified 1,044 patients in the National Cancer Database with non–small cell lung cancer and brain metastases who received systemic therapy plus surgery, brain stereotactic radiosurgery, or lung radiation. All were treated from 2010 to 2019; 79.0% received brain treatment first, and the other 21.0% received lung treatment first.
There was no statistically significant difference in overall survival between those whose brains were treated first and those whose lungs were treated first (hazard ratio, 1.24, 95% confidence interval [CI], 0.91-1.70, P = .17). A propensity score–matched analysis turned up no difference in 5-year survival (38.2% of those whose brains were treated first, 95% CI, 27.5-34.4, vs. 38.0% of those whose lungs were treated first, 95% CI, 29.9-44.7, P = .32.)
“These results were consistent regardless of which combination of treatment modalities the patient received – neurosurgery versus brain stereotactic radiosurgery, thoracic surgery versus thoracic radiation,” the author said.
He cautioned that “our study only included patients who were considered candidates for either surgery or radiation to both the brain and lung. The results of our study should therefore be cautiously interpreted for patients who may have contraindications to such treatment.”
Dr. Park, who didn’t take part in the study, said “the results are consistent with what I would generally expect.”
He added: “The take-home message for clinicians should be that there is no one correct answer in how to manage non–small cell lung cancer with synchronous limited metastatic disease in only the brain. If the brain disease is bulky and/or causes symptoms while the body disease isn’t – or if a biopsy or surgery is required to prove that the patient in fact has metastatic disease – then the brain disease should be treated first. On the other hand, if the body disease is bulky and/or causing symptoms while the brain disease isn’t – and there is no need for surgery but rather only a biopsy of the brain – then the body disease can be treated first.”
No funding was reported. The study authors and Dr. Park reported no financial conflicts or other disclosures.
FROM ELCC 2023
New coalition aims to revolutionize stalled lupus research
Clinical research into lupus has long been hampered by failures of medications that initially seemed promising. Now, a coalition of drugmakers, federal regulators, and activists has come together to forge a path toward better-designed studies and – potentially – groundbreaking new drugs.
“We have an opportunity to work collaboratively in lupus to address the challenges in drug development,” Teodora Staeva, PhD, vice president and chief scientific officer of the Lupus Research Alliance, said in an interview.
The alliance held a press conference on March 29 to announce the formation of the public-private Lupus Accelerating Breakthroughs Consortium. Coalition members include several major drugmakers, lupus organizations such as the LRA, the American College of Rheumatology, the Food and Drug Administration, and other federal agencies. Academic researchers, people living with lupus, caregivers and family members, and other members of the lupus community are also on board.
As Dr. Staeva explained, research into lupus has been marked by a high rate of failure. “Often, phase 2 trial successes have not translated into phase 3 successes,” she said.
But researchers, she said, don’t tend to think this is because the drugs themselves are useless.
Instead, it appears that “trial designs are not adequate to capture meaningful readouts of the drug effects, and that may have contributed to the multiple failures,” she said.
According to her, this may because the trials aren’t yet designed to fully detect whether drugs are useful. This is difficult to accomplish since patients have so many manifestations of the disease and trial participants already take a variety of existing drugs.
“Another major limitation has been the lack of integration of the patient’s voice and needs in the drug development process,” she said. It’s also challenging to recruit patients with the most severe lupus to participate in studies, especially since the trials often last 52 weeks.
The new coalition will not directly develop or favor specific drugs. Instead, it will focus on clinical research priorities. “It’s all open and collaborative,” Dr. Staeva explained, and a patient council will provide input. “We have a unique opportunity to bring the voice of people [living with lupus] to the table for the first time and be able to integrate their needs and priorities into the infrastructure.”
The new coalition was inspired by existing public-private partnerships such as the Kidney Health Initiative, she said. That initiative was founded in 2012 by the FDA and the American Society of Nephrology and has dozens of members, including multiple drugmakers and medical societies.
The leadership of the Lupus ABC coalition will include three nonvoting members from the FDA. They’ll offer guidance, Dr. Staeva said. At the press conference, Albert T. Roy, president and CEO of the LRA, said drug companies will appreciate the opportunity to speak with FDA representatives “in a space that is not competitive with respect to intellectual property or anything like that.”
The coalition will meet later in spring 2023, Dr. Staeva said. She hopes it will launch a couple of projects by the end of 2023 and be able to release preliminary results by the end of 2024.
One challenge will be figuring out how to stratify trial subjects so drug studies will more easily detect medications that may work in smaller populations of patients, Hoang Nguyen, PhD, director of scientific partnerships at the LRA, said in an interview. “Now we lump [patients] all together, and that’s not the optimal way to test drugs on patients who have a lot of differences.”
According to Dr. Staeva, the LRA funded the development of the coalition, and drugmakers will primarily provide financial support going forward. The pharmaceutical company members of the coalition are Biogen, Bristol-Myers Squibb, Eli Lilly, EMD Serono, Genentech, Gilead, GlaxoSmithKline, Merck, and Takeda.
Dr. Staeva, Dr. Nguyen, and Mr. Roy have no disclosures.
Clinical research into lupus has long been hampered by failures of medications that initially seemed promising. Now, a coalition of drugmakers, federal regulators, and activists has come together to forge a path toward better-designed studies and – potentially – groundbreaking new drugs.
“We have an opportunity to work collaboratively in lupus to address the challenges in drug development,” Teodora Staeva, PhD, vice president and chief scientific officer of the Lupus Research Alliance, said in an interview.
The alliance held a press conference on March 29 to announce the formation of the public-private Lupus Accelerating Breakthroughs Consortium. Coalition members include several major drugmakers, lupus organizations such as the LRA, the American College of Rheumatology, the Food and Drug Administration, and other federal agencies. Academic researchers, people living with lupus, caregivers and family members, and other members of the lupus community are also on board.
As Dr. Staeva explained, research into lupus has been marked by a high rate of failure. “Often, phase 2 trial successes have not translated into phase 3 successes,” she said.
But researchers, she said, don’t tend to think this is because the drugs themselves are useless.
Instead, it appears that “trial designs are not adequate to capture meaningful readouts of the drug effects, and that may have contributed to the multiple failures,” she said.
According to her, this may because the trials aren’t yet designed to fully detect whether drugs are useful. This is difficult to accomplish since patients have so many manifestations of the disease and trial participants already take a variety of existing drugs.
“Another major limitation has been the lack of integration of the patient’s voice and needs in the drug development process,” she said. It’s also challenging to recruit patients with the most severe lupus to participate in studies, especially since the trials often last 52 weeks.
The new coalition will not directly develop or favor specific drugs. Instead, it will focus on clinical research priorities. “It’s all open and collaborative,” Dr. Staeva explained, and a patient council will provide input. “We have a unique opportunity to bring the voice of people [living with lupus] to the table for the first time and be able to integrate their needs and priorities into the infrastructure.”
The new coalition was inspired by existing public-private partnerships such as the Kidney Health Initiative, she said. That initiative was founded in 2012 by the FDA and the American Society of Nephrology and has dozens of members, including multiple drugmakers and medical societies.
The leadership of the Lupus ABC coalition will include three nonvoting members from the FDA. They’ll offer guidance, Dr. Staeva said. At the press conference, Albert T. Roy, president and CEO of the LRA, said drug companies will appreciate the opportunity to speak with FDA representatives “in a space that is not competitive with respect to intellectual property or anything like that.”
The coalition will meet later in spring 2023, Dr. Staeva said. She hopes it will launch a couple of projects by the end of 2023 and be able to release preliminary results by the end of 2024.
One challenge will be figuring out how to stratify trial subjects so drug studies will more easily detect medications that may work in smaller populations of patients, Hoang Nguyen, PhD, director of scientific partnerships at the LRA, said in an interview. “Now we lump [patients] all together, and that’s not the optimal way to test drugs on patients who have a lot of differences.”
According to Dr. Staeva, the LRA funded the development of the coalition, and drugmakers will primarily provide financial support going forward. The pharmaceutical company members of the coalition are Biogen, Bristol-Myers Squibb, Eli Lilly, EMD Serono, Genentech, Gilead, GlaxoSmithKline, Merck, and Takeda.
Dr. Staeva, Dr. Nguyen, and Mr. Roy have no disclosures.
Clinical research into lupus has long been hampered by failures of medications that initially seemed promising. Now, a coalition of drugmakers, federal regulators, and activists has come together to forge a path toward better-designed studies and – potentially – groundbreaking new drugs.
“We have an opportunity to work collaboratively in lupus to address the challenges in drug development,” Teodora Staeva, PhD, vice president and chief scientific officer of the Lupus Research Alliance, said in an interview.
The alliance held a press conference on March 29 to announce the formation of the public-private Lupus Accelerating Breakthroughs Consortium. Coalition members include several major drugmakers, lupus organizations such as the LRA, the American College of Rheumatology, the Food and Drug Administration, and other federal agencies. Academic researchers, people living with lupus, caregivers and family members, and other members of the lupus community are also on board.
As Dr. Staeva explained, research into lupus has been marked by a high rate of failure. “Often, phase 2 trial successes have not translated into phase 3 successes,” she said.
But researchers, she said, don’t tend to think this is because the drugs themselves are useless.
Instead, it appears that “trial designs are not adequate to capture meaningful readouts of the drug effects, and that may have contributed to the multiple failures,” she said.
According to her, this may because the trials aren’t yet designed to fully detect whether drugs are useful. This is difficult to accomplish since patients have so many manifestations of the disease and trial participants already take a variety of existing drugs.
“Another major limitation has been the lack of integration of the patient’s voice and needs in the drug development process,” she said. It’s also challenging to recruit patients with the most severe lupus to participate in studies, especially since the trials often last 52 weeks.
The new coalition will not directly develop or favor specific drugs. Instead, it will focus on clinical research priorities. “It’s all open and collaborative,” Dr. Staeva explained, and a patient council will provide input. “We have a unique opportunity to bring the voice of people [living with lupus] to the table for the first time and be able to integrate their needs and priorities into the infrastructure.”
The new coalition was inspired by existing public-private partnerships such as the Kidney Health Initiative, she said. That initiative was founded in 2012 by the FDA and the American Society of Nephrology and has dozens of members, including multiple drugmakers and medical societies.
The leadership of the Lupus ABC coalition will include three nonvoting members from the FDA. They’ll offer guidance, Dr. Staeva said. At the press conference, Albert T. Roy, president and CEO of the LRA, said drug companies will appreciate the opportunity to speak with FDA representatives “in a space that is not competitive with respect to intellectual property or anything like that.”
The coalition will meet later in spring 2023, Dr. Staeva said. She hopes it will launch a couple of projects by the end of 2023 and be able to release preliminary results by the end of 2024.
One challenge will be figuring out how to stratify trial subjects so drug studies will more easily detect medications that may work in smaller populations of patients, Hoang Nguyen, PhD, director of scientific partnerships at the LRA, said in an interview. “Now we lump [patients] all together, and that’s not the optimal way to test drugs on patients who have a lot of differences.”
According to Dr. Staeva, the LRA funded the development of the coalition, and drugmakers will primarily provide financial support going forward. The pharmaceutical company members of the coalition are Biogen, Bristol-Myers Squibb, Eli Lilly, EMD Serono, Genentech, Gilead, GlaxoSmithKline, Merck, and Takeda.
Dr. Staeva, Dr. Nguyen, and Mr. Roy have no disclosures.