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MDedge conference coverage features onsite reporting of the latest study results and expert perspectives from leading researchers.
AI Improves Lesion Detection in IBD Over Standard Methods
BERLIN — in a first-of-its-kind, multicenter study.
In addition to the model’s superior diagnostic performance than standard of care, it also achieved a significant reduction in the mean reading time per exam.
Furthermore, the study clinically validated an AI model in real time for small-bowel CE.
The AI model addresses long-standing limitations of CE interpretation, including time-consuming readings and interobserver variability.
“It’s a huge improvement on the technology readiness level of the AI model,” said senior study investigator Miguel Mascarenhas, MD, PhD, head of the precision medicine unit at the Hospital São João, Faculty of Medicine, University of Porto, Portugal.
Until now, there has been no AI system using a CE platform that has proven so effective in so many real-life clinical settings, he explained. “This technology is set to transform endoscopic practice and clinical management in inflammatory bowel disease.”
The findings were presented at European Crohn’s and Colitis Organisation 2025 Congress by Francisco Mendes, MD, a resident in gastroenterology, also at the Hospital São João.
More Lesions, Less Time
Researchers conducted the prospective study involving centers in Portugal, Spain, and the United States between January 2021 and April 2024. Two CE devices (PillCamSB3 and Olympus EC-10) were analyzed for their performance across 137 CE exams in 137 patients, 49 of whom had Crohn’s disease. AI-assisted readings were compared with standard-of-care readings, with expert board consensus considered to be the gold standard. Key performance metrics included sensitivity, specificity, positive predictive value (PPV), and negative PV (NPV).
During expert board review, ulcers and erosions were identified in 56 patients (40.9%), with a sensitivity of 60.7%, specificity of 98.8%, a PPV of 97.1%, and an NPV of 78.4%, leading to an overall accuracy for the detection of ulcers and erosions of 83.2%.
In comparison, the AI-assisted readings outperformed conventional readings with a sensitivity of 94.6%, specificity of 80.2%, a PPV of 76.8%, an NPV of 95.6%, leading to an overall accuracy of 86.1%.
The AI-assisted model diagnosis was noninferior (P < .001) and superior (P < .001) to conventional diagnosis for detection of ulcers and erosions. The AI model demonstrated consistent performance across different CE devices and centers.
In addition, the mean time taken per reading was under 4 minutes (239 seconds) per exam for AI, compared with around 1.0-1.5 hours for standard-of-care readings.
The increased diagnostic accuracy of this AI model done in far less time allows us to engage more with the patient and attend to other care-related tasks, Mascarenhas said.
CE has great potential not only in IBD but also in other gastrointestinal-related screening, including colorectal cancer screening, he added. Once the bottleneck of reading time with CE is solved, it will become the first-line tool for screening.
Reading time is “one of several barriers” to integration of CE into clinical practice, Shomron Ben-Horin, MD, director, Sheba Medical Center, Tel-Aviv University, Israel, said in an interview. But it “is the most accurate modality for detection of inflammatory activity along the entire small bowel.”
Based on these study results, AI is the way to go, said Ben-Horin, who was not involved in the study. “There was even a signal for better accuracy, which is intriguing,” he added. This study points toward AI being more accurate than the physicians in reading, and that is important.
Also commenting was Miles Parkes, MD, consultant gastroenterologist at Addenbrooke’s Hospital in Cambridge, England.
“Both the sensitivity and the specificity of the output are reassuring, but there might be some devil in the detail,” he said. “However, as a general principle the performance of this model is impressive.”
Mascarenhas and Mendes declared no financial disclosures. Ben Horin received fees from Medtronic to attend the conference. Parkes declared no financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — in a first-of-its-kind, multicenter study.
In addition to the model’s superior diagnostic performance than standard of care, it also achieved a significant reduction in the mean reading time per exam.
Furthermore, the study clinically validated an AI model in real time for small-bowel CE.
The AI model addresses long-standing limitations of CE interpretation, including time-consuming readings and interobserver variability.
“It’s a huge improvement on the technology readiness level of the AI model,” said senior study investigator Miguel Mascarenhas, MD, PhD, head of the precision medicine unit at the Hospital São João, Faculty of Medicine, University of Porto, Portugal.
Until now, there has been no AI system using a CE platform that has proven so effective in so many real-life clinical settings, he explained. “This technology is set to transform endoscopic practice and clinical management in inflammatory bowel disease.”
The findings were presented at European Crohn’s and Colitis Organisation 2025 Congress by Francisco Mendes, MD, a resident in gastroenterology, also at the Hospital São João.
More Lesions, Less Time
Researchers conducted the prospective study involving centers in Portugal, Spain, and the United States between January 2021 and April 2024. Two CE devices (PillCamSB3 and Olympus EC-10) were analyzed for their performance across 137 CE exams in 137 patients, 49 of whom had Crohn’s disease. AI-assisted readings were compared with standard-of-care readings, with expert board consensus considered to be the gold standard. Key performance metrics included sensitivity, specificity, positive predictive value (PPV), and negative PV (NPV).
During expert board review, ulcers and erosions were identified in 56 patients (40.9%), with a sensitivity of 60.7%, specificity of 98.8%, a PPV of 97.1%, and an NPV of 78.4%, leading to an overall accuracy for the detection of ulcers and erosions of 83.2%.
In comparison, the AI-assisted readings outperformed conventional readings with a sensitivity of 94.6%, specificity of 80.2%, a PPV of 76.8%, an NPV of 95.6%, leading to an overall accuracy of 86.1%.
The AI-assisted model diagnosis was noninferior (P < .001) and superior (P < .001) to conventional diagnosis for detection of ulcers and erosions. The AI model demonstrated consistent performance across different CE devices and centers.
In addition, the mean time taken per reading was under 4 minutes (239 seconds) per exam for AI, compared with around 1.0-1.5 hours for standard-of-care readings.
The increased diagnostic accuracy of this AI model done in far less time allows us to engage more with the patient and attend to other care-related tasks, Mascarenhas said.
CE has great potential not only in IBD but also in other gastrointestinal-related screening, including colorectal cancer screening, he added. Once the bottleneck of reading time with CE is solved, it will become the first-line tool for screening.
Reading time is “one of several barriers” to integration of CE into clinical practice, Shomron Ben-Horin, MD, director, Sheba Medical Center, Tel-Aviv University, Israel, said in an interview. But it “is the most accurate modality for detection of inflammatory activity along the entire small bowel.”
Based on these study results, AI is the way to go, said Ben-Horin, who was not involved in the study. “There was even a signal for better accuracy, which is intriguing,” he added. This study points toward AI being more accurate than the physicians in reading, and that is important.
Also commenting was Miles Parkes, MD, consultant gastroenterologist at Addenbrooke’s Hospital in Cambridge, England.
“Both the sensitivity and the specificity of the output are reassuring, but there might be some devil in the detail,” he said. “However, as a general principle the performance of this model is impressive.”
Mascarenhas and Mendes declared no financial disclosures. Ben Horin received fees from Medtronic to attend the conference. Parkes declared no financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — in a first-of-its-kind, multicenter study.
In addition to the model’s superior diagnostic performance than standard of care, it also achieved a significant reduction in the mean reading time per exam.
Furthermore, the study clinically validated an AI model in real time for small-bowel CE.
The AI model addresses long-standing limitations of CE interpretation, including time-consuming readings and interobserver variability.
“It’s a huge improvement on the technology readiness level of the AI model,” said senior study investigator Miguel Mascarenhas, MD, PhD, head of the precision medicine unit at the Hospital São João, Faculty of Medicine, University of Porto, Portugal.
Until now, there has been no AI system using a CE platform that has proven so effective in so many real-life clinical settings, he explained. “This technology is set to transform endoscopic practice and clinical management in inflammatory bowel disease.”
The findings were presented at European Crohn’s and Colitis Organisation 2025 Congress by Francisco Mendes, MD, a resident in gastroenterology, also at the Hospital São João.
More Lesions, Less Time
Researchers conducted the prospective study involving centers in Portugal, Spain, and the United States between January 2021 and April 2024. Two CE devices (PillCamSB3 and Olympus EC-10) were analyzed for their performance across 137 CE exams in 137 patients, 49 of whom had Crohn’s disease. AI-assisted readings were compared with standard-of-care readings, with expert board consensus considered to be the gold standard. Key performance metrics included sensitivity, specificity, positive predictive value (PPV), and negative PV (NPV).
During expert board review, ulcers and erosions were identified in 56 patients (40.9%), with a sensitivity of 60.7%, specificity of 98.8%, a PPV of 97.1%, and an NPV of 78.4%, leading to an overall accuracy for the detection of ulcers and erosions of 83.2%.
In comparison, the AI-assisted readings outperformed conventional readings with a sensitivity of 94.6%, specificity of 80.2%, a PPV of 76.8%, an NPV of 95.6%, leading to an overall accuracy of 86.1%.
The AI-assisted model diagnosis was noninferior (P < .001) and superior (P < .001) to conventional diagnosis for detection of ulcers and erosions. The AI model demonstrated consistent performance across different CE devices and centers.
In addition, the mean time taken per reading was under 4 minutes (239 seconds) per exam for AI, compared with around 1.0-1.5 hours for standard-of-care readings.
The increased diagnostic accuracy of this AI model done in far less time allows us to engage more with the patient and attend to other care-related tasks, Mascarenhas said.
CE has great potential not only in IBD but also in other gastrointestinal-related screening, including colorectal cancer screening, he added. Once the bottleneck of reading time with CE is solved, it will become the first-line tool for screening.
Reading time is “one of several barriers” to integration of CE into clinical practice, Shomron Ben-Horin, MD, director, Sheba Medical Center, Tel-Aviv University, Israel, said in an interview. But it “is the most accurate modality for detection of inflammatory activity along the entire small bowel.”
Based on these study results, AI is the way to go, said Ben-Horin, who was not involved in the study. “There was even a signal for better accuracy, which is intriguing,” he added. This study points toward AI being more accurate than the physicians in reading, and that is important.
Also commenting was Miles Parkes, MD, consultant gastroenterologist at Addenbrooke’s Hospital in Cambridge, England.
“Both the sensitivity and the specificity of the output are reassuring, but there might be some devil in the detail,” he said. “However, as a general principle the performance of this model is impressive.”
Mascarenhas and Mendes declared no financial disclosures. Ben Horin received fees from Medtronic to attend the conference. Parkes declared no financial disclosures.
A version of this article appeared on Medscape.com.
FROM ECCO 2025
Antibody Profiles Predict IBD Up To 10 Years Before Onset
BERLIN — a new study suggested.
The research was presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
“High-throughput and high-resolution antibody profiling delineates a previously underappreciated landscape of selective serological responses in inflammatory bowel disease,” said study presenter Arno R. Bourgonje, MD, PhD, of the Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York City.
The discovery represents just the “tip of the iceberg” in terms of understanding how antibody response could predict IBD onset, he added. Although validation studies are ongoing, the findings “allow for novel insights into disease pathogenesis and also for allowing for disease prediction.”
In IBD, the integrity of the intestinal barrier is compromised and luminal agents, like bacteria, can leak through, which leads to immune activation, Bourgonje said.
However, only a few serological antibody responses are known to occur in IBD, such as antibodies against the yeast Saccharomyces cerevisiae and those against the cytoplasm of neutrophils, he said.
But most antibody responses are directed against bacteria, Bourgonje noted. The gut microbiome represents thousands of different bacterial species, each of which encode for thousands of different genes, representing a tremendous number of potential antigens. But conventional antibody-profiling technologies weren’t powerful enough to identify antibodies in patients with IBD that signal an immune response to potential antigens in the gut.
To get at that problem, the researchers recently leveraged a high-throughput technology called phage-display immunoprecipitation sequencing (PhIP-Seq) to look for specific immune-based biomarker signatures in the blood of individuals with IBD. This effort revealed a distinct repertoire of antibodies not only against bacteria but also against viruses and cell antigens.
The researchers next turned their sights on discovering whether they could find evidence of immunological alterations before IBD onset to enable disease prediction.
Predictive Signatures Found
The team used a longitudinal preclinical IBD cohort called PREDICTS (Proteomic Evaluation and Discovery in an IBD Cohort of Tri-service Subjects) that is housed in the US Department of Defense Serum Repository.
Using PhIP-Seq, the researchers analyzed serum samples from 200 individuals who developed Crohn’s disease, 200 who developed ulcerative colitis, and 100 non-IBD controls matched for age, sex, race, and study time point. The samples were collected approximately 2 years, 4 years, and 10 years prior to diagnosis as well around the time of diagnosis.
The results showed that, compared with healthy controls, the diversity of the antibody repertoire was significantly lower in the sera of individuals with preclinical Crohn’s disease (P < .05) and ulcerative colitis (P < .001), with the lowest similarity seen in people with preclinical Crohn’s disease approximately 4 years prior to their diagnosis (P < .001).
The study also found that, compared with healthy controls, antibody responses in individuals with preclinical Crohn’s disease against herpes viruses such as Epstein-Barr virus (EBV), cytomegalovirus (CMV), and herpes simplex virus (HSV)–1 and HSV-2 were significantly higher approximately 10 years prior to the diagnosis of Crohn’s disease, whereas anti-Streptococcus responses were lower.
In individuals with ulcerative colitis, antibody responses to EBV, CMV, HSV-1, and influenza viruses were significantly higher than that in healthy controls approximately 10 years prior to diagnosis, whereas anti-rhinovirus responses were lower.
Further analysis demonstrated that antibody responses to CMV and EBV proteins increased over the course of the preclinical phase of Crohn’s disease vs healthy controls (P = .008 and P = .011, respectively).
Similarly, autoantibody responses to MAP kinase–activating death domain increased during the preclinical phase of ulcerative colitis vs healthy controls (P = .0025), whereas anti-Streptococcus responses decreased (P = .005).
Interestingly, no one single antibody response difference with healthy controls was able to accurately predict the onset of IBD 10 years prior to diagnosis, but distinct sets of antibody responses were, with area under the receiver operating characteristic curve of 0.90 for Crohn’s disease and 0.84 for ulcerative colitis.
A Promising Start
The study has potential to be useful for identifying people at risk for IBD, Robin Dart, MD, PhD, a consultant gastroenterologist at Guy’s and St Thomas Hospital, London, England, who co-chaired the session, said in an interview.
The difference in antibody responses to viral and bacterial antigens between Crohn’s disease and ulcerative colitis could point toward underlying biological mechanisms, although it is “too early to say,” Dart said.
However, “when you do these kind of big fishing exercises” and identify microbes may be implicated in IBD, “you end up finding more questions than answers,” although that “can only be a good thing,” he added.
Bourgonje noted that the study cohort consisted entirely of men enrolled in the US Army, limiting the applicability of the findings. Another limitation was that researchers were unable to control smoking, antibiotic use, and diet, all of which could have affected the results.
This study was funded by the Leona M. and Harry B. Helmsley Charitable Trust. Bourgonje declared relationships with Janssen Pharmaceuticals, Ferring, AbbVie. Other authors also declared numerous relationships.
A version of this article appeared on Medscape.com.
BERLIN — a new study suggested.
The research was presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
“High-throughput and high-resolution antibody profiling delineates a previously underappreciated landscape of selective serological responses in inflammatory bowel disease,” said study presenter Arno R. Bourgonje, MD, PhD, of the Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York City.
The discovery represents just the “tip of the iceberg” in terms of understanding how antibody response could predict IBD onset, he added. Although validation studies are ongoing, the findings “allow for novel insights into disease pathogenesis and also for allowing for disease prediction.”
In IBD, the integrity of the intestinal barrier is compromised and luminal agents, like bacteria, can leak through, which leads to immune activation, Bourgonje said.
However, only a few serological antibody responses are known to occur in IBD, such as antibodies against the yeast Saccharomyces cerevisiae and those against the cytoplasm of neutrophils, he said.
But most antibody responses are directed against bacteria, Bourgonje noted. The gut microbiome represents thousands of different bacterial species, each of which encode for thousands of different genes, representing a tremendous number of potential antigens. But conventional antibody-profiling technologies weren’t powerful enough to identify antibodies in patients with IBD that signal an immune response to potential antigens in the gut.
To get at that problem, the researchers recently leveraged a high-throughput technology called phage-display immunoprecipitation sequencing (PhIP-Seq) to look for specific immune-based biomarker signatures in the blood of individuals with IBD. This effort revealed a distinct repertoire of antibodies not only against bacteria but also against viruses and cell antigens.
The researchers next turned their sights on discovering whether they could find evidence of immunological alterations before IBD onset to enable disease prediction.
Predictive Signatures Found
The team used a longitudinal preclinical IBD cohort called PREDICTS (Proteomic Evaluation and Discovery in an IBD Cohort of Tri-service Subjects) that is housed in the US Department of Defense Serum Repository.
Using PhIP-Seq, the researchers analyzed serum samples from 200 individuals who developed Crohn’s disease, 200 who developed ulcerative colitis, and 100 non-IBD controls matched for age, sex, race, and study time point. The samples were collected approximately 2 years, 4 years, and 10 years prior to diagnosis as well around the time of diagnosis.
The results showed that, compared with healthy controls, the diversity of the antibody repertoire was significantly lower in the sera of individuals with preclinical Crohn’s disease (P < .05) and ulcerative colitis (P < .001), with the lowest similarity seen in people with preclinical Crohn’s disease approximately 4 years prior to their diagnosis (P < .001).
The study also found that, compared with healthy controls, antibody responses in individuals with preclinical Crohn’s disease against herpes viruses such as Epstein-Barr virus (EBV), cytomegalovirus (CMV), and herpes simplex virus (HSV)–1 and HSV-2 were significantly higher approximately 10 years prior to the diagnosis of Crohn’s disease, whereas anti-Streptococcus responses were lower.
In individuals with ulcerative colitis, antibody responses to EBV, CMV, HSV-1, and influenza viruses were significantly higher than that in healthy controls approximately 10 years prior to diagnosis, whereas anti-rhinovirus responses were lower.
Further analysis demonstrated that antibody responses to CMV and EBV proteins increased over the course of the preclinical phase of Crohn’s disease vs healthy controls (P = .008 and P = .011, respectively).
Similarly, autoantibody responses to MAP kinase–activating death domain increased during the preclinical phase of ulcerative colitis vs healthy controls (P = .0025), whereas anti-Streptococcus responses decreased (P = .005).
Interestingly, no one single antibody response difference with healthy controls was able to accurately predict the onset of IBD 10 years prior to diagnosis, but distinct sets of antibody responses were, with area under the receiver operating characteristic curve of 0.90 for Crohn’s disease and 0.84 for ulcerative colitis.
A Promising Start
The study has potential to be useful for identifying people at risk for IBD, Robin Dart, MD, PhD, a consultant gastroenterologist at Guy’s and St Thomas Hospital, London, England, who co-chaired the session, said in an interview.
The difference in antibody responses to viral and bacterial antigens between Crohn’s disease and ulcerative colitis could point toward underlying biological mechanisms, although it is “too early to say,” Dart said.
However, “when you do these kind of big fishing exercises” and identify microbes may be implicated in IBD, “you end up finding more questions than answers,” although that “can only be a good thing,” he added.
Bourgonje noted that the study cohort consisted entirely of men enrolled in the US Army, limiting the applicability of the findings. Another limitation was that researchers were unable to control smoking, antibiotic use, and diet, all of which could have affected the results.
This study was funded by the Leona M. and Harry B. Helmsley Charitable Trust. Bourgonje declared relationships with Janssen Pharmaceuticals, Ferring, AbbVie. Other authors also declared numerous relationships.
A version of this article appeared on Medscape.com.
BERLIN — a new study suggested.
The research was presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
“High-throughput and high-resolution antibody profiling delineates a previously underappreciated landscape of selective serological responses in inflammatory bowel disease,” said study presenter Arno R. Bourgonje, MD, PhD, of the Henry D. Janowitz Division of Gastroenterology, Icahn School of Medicine at Mount Sinai, New York City.
The discovery represents just the “tip of the iceberg” in terms of understanding how antibody response could predict IBD onset, he added. Although validation studies are ongoing, the findings “allow for novel insights into disease pathogenesis and also for allowing for disease prediction.”
In IBD, the integrity of the intestinal barrier is compromised and luminal agents, like bacteria, can leak through, which leads to immune activation, Bourgonje said.
However, only a few serological antibody responses are known to occur in IBD, such as antibodies against the yeast Saccharomyces cerevisiae and those against the cytoplasm of neutrophils, he said.
But most antibody responses are directed against bacteria, Bourgonje noted. The gut microbiome represents thousands of different bacterial species, each of which encode for thousands of different genes, representing a tremendous number of potential antigens. But conventional antibody-profiling technologies weren’t powerful enough to identify antibodies in patients with IBD that signal an immune response to potential antigens in the gut.
To get at that problem, the researchers recently leveraged a high-throughput technology called phage-display immunoprecipitation sequencing (PhIP-Seq) to look for specific immune-based biomarker signatures in the blood of individuals with IBD. This effort revealed a distinct repertoire of antibodies not only against bacteria but also against viruses and cell antigens.
The researchers next turned their sights on discovering whether they could find evidence of immunological alterations before IBD onset to enable disease prediction.
Predictive Signatures Found
The team used a longitudinal preclinical IBD cohort called PREDICTS (Proteomic Evaluation and Discovery in an IBD Cohort of Tri-service Subjects) that is housed in the US Department of Defense Serum Repository.
Using PhIP-Seq, the researchers analyzed serum samples from 200 individuals who developed Crohn’s disease, 200 who developed ulcerative colitis, and 100 non-IBD controls matched for age, sex, race, and study time point. The samples were collected approximately 2 years, 4 years, and 10 years prior to diagnosis as well around the time of diagnosis.
The results showed that, compared with healthy controls, the diversity of the antibody repertoire was significantly lower in the sera of individuals with preclinical Crohn’s disease (P < .05) and ulcerative colitis (P < .001), with the lowest similarity seen in people with preclinical Crohn’s disease approximately 4 years prior to their diagnosis (P < .001).
The study also found that, compared with healthy controls, antibody responses in individuals with preclinical Crohn’s disease against herpes viruses such as Epstein-Barr virus (EBV), cytomegalovirus (CMV), and herpes simplex virus (HSV)–1 and HSV-2 were significantly higher approximately 10 years prior to the diagnosis of Crohn’s disease, whereas anti-Streptococcus responses were lower.
In individuals with ulcerative colitis, antibody responses to EBV, CMV, HSV-1, and influenza viruses were significantly higher than that in healthy controls approximately 10 years prior to diagnosis, whereas anti-rhinovirus responses were lower.
Further analysis demonstrated that antibody responses to CMV and EBV proteins increased over the course of the preclinical phase of Crohn’s disease vs healthy controls (P = .008 and P = .011, respectively).
Similarly, autoantibody responses to MAP kinase–activating death domain increased during the preclinical phase of ulcerative colitis vs healthy controls (P = .0025), whereas anti-Streptococcus responses decreased (P = .005).
Interestingly, no one single antibody response difference with healthy controls was able to accurately predict the onset of IBD 10 years prior to diagnosis, but distinct sets of antibody responses were, with area under the receiver operating characteristic curve of 0.90 for Crohn’s disease and 0.84 for ulcerative colitis.
A Promising Start
The study has potential to be useful for identifying people at risk for IBD, Robin Dart, MD, PhD, a consultant gastroenterologist at Guy’s and St Thomas Hospital, London, England, who co-chaired the session, said in an interview.
The difference in antibody responses to viral and bacterial antigens between Crohn’s disease and ulcerative colitis could point toward underlying biological mechanisms, although it is “too early to say,” Dart said.
However, “when you do these kind of big fishing exercises” and identify microbes may be implicated in IBD, “you end up finding more questions than answers,” although that “can only be a good thing,” he added.
Bourgonje noted that the study cohort consisted entirely of men enrolled in the US Army, limiting the applicability of the findings. Another limitation was that researchers were unable to control smoking, antibiotic use, and diet, all of which could have affected the results.
This study was funded by the Leona M. and Harry B. Helmsley Charitable Trust. Bourgonje declared relationships with Janssen Pharmaceuticals, Ferring, AbbVie. Other authors also declared numerous relationships.
A version of this article appeared on Medscape.com.
FROM ECCO 2025
New Biomarkers Identified for Treatment Response in IBD
BERLIN — presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Differences uncovered in multiple messenger RNAs, proteins, metabolites, and gut microbiota were associated with responders and nonresponders to biologics and Janus kinase inhibitors, suggesting the potential for predictive biomarkers in IBD, including Crohn’s disease (CD) and ulcerative colitis (UC).
“With further work we hope to confirm these findings and evaluate their clinical relevance in identifying patients most likely to respond to tailored therapeutic interventions,” said Montserrat Baldan-Martin, PhD, a researcher at the University Hospital of the Princess, Madrid, Spain.
The treatment of IBD is challenging because of the heterogeneity across clinical, immunological, molecular, genetic, and microbiologic features, with one third of patients failing to respond to any one treatment.
Baldan-Martin and colleagues wanted to find predictive biomarkers of response by examining the differences across multi-omics profiles relative to different therapies.
The study analyzed 127 patients with IBD (57 with CD and 70 with UC) before and after 14 weeks of treatment with one of the following: anti–tumor necrosis factors (TNFs), ustekinumab, vedolizumab, or tofacitinib. Patient response to treatment was evaluated using endoscopic criteria that categorized them as responders or nonresponders to the different therapies.
In addition, molecular data from various biologic samples — serum, urine, extracellular vesicles, intestinal biopsies, and stool — were tested using transcriptomics, proteomics, metabolomics, and metagenomics.
Clear Differences
“The most significant differences were seen in gene expression within intestinal tissue of responder and nonresponder patients with ulcerative colitis taking vedolizumab,” Baldan-Martin reported.
Proteomic analysis revealed that a total of 1377 proteins were identified across all groups (CD, UC, and the four drug classes/therapies). Responders and nonresponders for each therapy expressed different proteins in serum extracellular vesicles and intestinal tissues.
For example, patients with CD who responded to anti-TNF therapies had 138 different proteins from those of anti-TNF nonresponders, while patients with UC who responded to anti-TNF therapies had 218 different proteins from those of anti-TNF nonresponders, reported Baldan-Martin.
Also, we observed almost no proteins “in common between ulcerative colitis responders versus nonresponders for all treatments,” she noted. And we “saw only three proteins in common with Crohn’s disease patients [on different drugs].”
Metabolomic analysis identified deregulation of 24 serum lipoproteins in CD responders to ustekinumab, compared with nonresponders.
“We observed greater differences in the lipoproteins in serum than metabolites in serum and urine,” Baldan-Martin added.
Analysis of biologic pathways also highlighted enrichment in ketone and butyrate metabolism, mitochondrial electron transport chain activity, carnitine synthesis, and fatty acid oxidation pathways, while metagenomic analysis revealed the greatest microbial differences in UC responders and nonresponders to anti-TNF therapies.
Baldan-Martin said research was ongoing with a new cohort of patients that aims to validate some of the biomarkers and help identify the patients most likely to respond to tailored therapeutic interventions.
“One of the challenges is integrating results from different omics approaches to create a more holistic understanding of the disease,” she said, adding that she hopes the research “will potentially open doors for early detection through multi-panel biomarkers.”
Session moderator Mark Samaan, MD, consultant gastroenterologist at Guy’s and St Thomas’ NHS Foundation Trust, London, England, said that “the findings related to nonresponse to specific drugs in UC and CD were interesting. With longitudinal follow-up, we’d hope this might help us pick out patients less likely to respond and who show early nonresponse to specific drugs based on serum, urine, and fecal sampling.”
“It’s very helpful to know if someone is a nonresponder within 14 weeks because we can then move the patient on to something else relatively quickly,” he added.
Baldan-Martin and Samaan declared no relevant financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Differences uncovered in multiple messenger RNAs, proteins, metabolites, and gut microbiota were associated with responders and nonresponders to biologics and Janus kinase inhibitors, suggesting the potential for predictive biomarkers in IBD, including Crohn’s disease (CD) and ulcerative colitis (UC).
“With further work we hope to confirm these findings and evaluate their clinical relevance in identifying patients most likely to respond to tailored therapeutic interventions,” said Montserrat Baldan-Martin, PhD, a researcher at the University Hospital of the Princess, Madrid, Spain.
The treatment of IBD is challenging because of the heterogeneity across clinical, immunological, molecular, genetic, and microbiologic features, with one third of patients failing to respond to any one treatment.
Baldan-Martin and colleagues wanted to find predictive biomarkers of response by examining the differences across multi-omics profiles relative to different therapies.
The study analyzed 127 patients with IBD (57 with CD and 70 with UC) before and after 14 weeks of treatment with one of the following: anti–tumor necrosis factors (TNFs), ustekinumab, vedolizumab, or tofacitinib. Patient response to treatment was evaluated using endoscopic criteria that categorized them as responders or nonresponders to the different therapies.
In addition, molecular data from various biologic samples — serum, urine, extracellular vesicles, intestinal biopsies, and stool — were tested using transcriptomics, proteomics, metabolomics, and metagenomics.
Clear Differences
“The most significant differences were seen in gene expression within intestinal tissue of responder and nonresponder patients with ulcerative colitis taking vedolizumab,” Baldan-Martin reported.
Proteomic analysis revealed that a total of 1377 proteins were identified across all groups (CD, UC, and the four drug classes/therapies). Responders and nonresponders for each therapy expressed different proteins in serum extracellular vesicles and intestinal tissues.
For example, patients with CD who responded to anti-TNF therapies had 138 different proteins from those of anti-TNF nonresponders, while patients with UC who responded to anti-TNF therapies had 218 different proteins from those of anti-TNF nonresponders, reported Baldan-Martin.
Also, we observed almost no proteins “in common between ulcerative colitis responders versus nonresponders for all treatments,” she noted. And we “saw only three proteins in common with Crohn’s disease patients [on different drugs].”
Metabolomic analysis identified deregulation of 24 serum lipoproteins in CD responders to ustekinumab, compared with nonresponders.
“We observed greater differences in the lipoproteins in serum than metabolites in serum and urine,” Baldan-Martin added.
Analysis of biologic pathways also highlighted enrichment in ketone and butyrate metabolism, mitochondrial electron transport chain activity, carnitine synthesis, and fatty acid oxidation pathways, while metagenomic analysis revealed the greatest microbial differences in UC responders and nonresponders to anti-TNF therapies.
Baldan-Martin said research was ongoing with a new cohort of patients that aims to validate some of the biomarkers and help identify the patients most likely to respond to tailored therapeutic interventions.
“One of the challenges is integrating results from different omics approaches to create a more holistic understanding of the disease,” she said, adding that she hopes the research “will potentially open doors for early detection through multi-panel biomarkers.”
Session moderator Mark Samaan, MD, consultant gastroenterologist at Guy’s and St Thomas’ NHS Foundation Trust, London, England, said that “the findings related to nonresponse to specific drugs in UC and CD were interesting. With longitudinal follow-up, we’d hope this might help us pick out patients less likely to respond and who show early nonresponse to specific drugs based on serum, urine, and fecal sampling.”
“It’s very helpful to know if someone is a nonresponder within 14 weeks because we can then move the patient on to something else relatively quickly,” he added.
Baldan-Martin and Samaan declared no relevant financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — presented at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Differences uncovered in multiple messenger RNAs, proteins, metabolites, and gut microbiota were associated with responders and nonresponders to biologics and Janus kinase inhibitors, suggesting the potential for predictive biomarkers in IBD, including Crohn’s disease (CD) and ulcerative colitis (UC).
“With further work we hope to confirm these findings and evaluate their clinical relevance in identifying patients most likely to respond to tailored therapeutic interventions,” said Montserrat Baldan-Martin, PhD, a researcher at the University Hospital of the Princess, Madrid, Spain.
The treatment of IBD is challenging because of the heterogeneity across clinical, immunological, molecular, genetic, and microbiologic features, with one third of patients failing to respond to any one treatment.
Baldan-Martin and colleagues wanted to find predictive biomarkers of response by examining the differences across multi-omics profiles relative to different therapies.
The study analyzed 127 patients with IBD (57 with CD and 70 with UC) before and after 14 weeks of treatment with one of the following: anti–tumor necrosis factors (TNFs), ustekinumab, vedolizumab, or tofacitinib. Patient response to treatment was evaluated using endoscopic criteria that categorized them as responders or nonresponders to the different therapies.
In addition, molecular data from various biologic samples — serum, urine, extracellular vesicles, intestinal biopsies, and stool — were tested using transcriptomics, proteomics, metabolomics, and metagenomics.
Clear Differences
“The most significant differences were seen in gene expression within intestinal tissue of responder and nonresponder patients with ulcerative colitis taking vedolizumab,” Baldan-Martin reported.
Proteomic analysis revealed that a total of 1377 proteins were identified across all groups (CD, UC, and the four drug classes/therapies). Responders and nonresponders for each therapy expressed different proteins in serum extracellular vesicles and intestinal tissues.
For example, patients with CD who responded to anti-TNF therapies had 138 different proteins from those of anti-TNF nonresponders, while patients with UC who responded to anti-TNF therapies had 218 different proteins from those of anti-TNF nonresponders, reported Baldan-Martin.
Also, we observed almost no proteins “in common between ulcerative colitis responders versus nonresponders for all treatments,” she noted. And we “saw only three proteins in common with Crohn’s disease patients [on different drugs].”
Metabolomic analysis identified deregulation of 24 serum lipoproteins in CD responders to ustekinumab, compared with nonresponders.
“We observed greater differences in the lipoproteins in serum than metabolites in serum and urine,” Baldan-Martin added.
Analysis of biologic pathways also highlighted enrichment in ketone and butyrate metabolism, mitochondrial electron transport chain activity, carnitine synthesis, and fatty acid oxidation pathways, while metagenomic analysis revealed the greatest microbial differences in UC responders and nonresponders to anti-TNF therapies.
Baldan-Martin said research was ongoing with a new cohort of patients that aims to validate some of the biomarkers and help identify the patients most likely to respond to tailored therapeutic interventions.
“One of the challenges is integrating results from different omics approaches to create a more holistic understanding of the disease,” she said, adding that she hopes the research “will potentially open doors for early detection through multi-panel biomarkers.”
Session moderator Mark Samaan, MD, consultant gastroenterologist at Guy’s and St Thomas’ NHS Foundation Trust, London, England, said that “the findings related to nonresponse to specific drugs in UC and CD were interesting. With longitudinal follow-up, we’d hope this might help us pick out patients less likely to respond and who show early nonresponse to specific drugs based on serum, urine, and fecal sampling.”
“It’s very helpful to know if someone is a nonresponder within 14 weeks because we can then move the patient on to something else relatively quickly,” he added.
Baldan-Martin and Samaan declared no relevant financial disclosures.
A version of this article appeared on Medscape.com.
FROM ECCO 2025
Not All Plant-based Diets Are Equal in IBD Risk Mitigation
BERLIN — according to the results of a large cohort study.
The study, which included both Crohn’s disease (CD) and ulcerative colitis (UC), also showed that diet quality may affect disease progression and surgery risk for individuals already diagnosed with IBD.
“Not all plant-based foods are equal — they don’t all have the same effect on health outcomes,” said study researcher, Judith Wellens, MD, PhD, gastroenterology resident at Leuven University Hospital in Belgium.
“We need to look at what people are eating more carefully because it isn’t black and white, with all plant-based food being good and animal-based food being bad,” said Wellens, who presented the data at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Although she advocates for plant-based diets, Wellens stressed that “they need to be individualized to ensure the overall dietary quality is good. Just cutting out meat products is not very helpful. We think it is the unhealthy additions to some plant-based diets that drive the IBD risk.”
Is It the Plants or the Processed Ingredients?
“Preclinical studies have already taught us that plant-based diets alter the gut microbiota in a beneficial way. However, many diets promoted for IBD — for example the Crohn’s disease exclusion diet — contain ingredients that are animal based. This is confusing for patients and for clinicians,” said Wellens.
To look more closely at the question, she and her colleagues analyzed data for 187,888 participants from the UK Biobank and 341,539 participants from across eight European countries from the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort. None of the participants had IBD at baseline.
Based on participant 24-hour dietary recalls, the researchers constructed plant-based diet indices (PDIs) with diets categorized as healthy (eg, whole grains, fruits, vegetables, legumes, and vegetarian protein alternatives) or unhealthy (eg, emulsifiers, refined grains, fries, fruit juices, sweets, desserts, sugar-sweetened beverages, and processed foods).
The primary outcome was the incidence of IBD (either CD or UC), whereas the secondary outcome was IBD-related surgery, thereby marking disease progression. Cox regression analysis estimated IBD risk and progression. Incidences of IBD were similar between the two cohorts.
In the UK Biobank cohort, 925 participants developed IBD over a median follow-up of 11.6 years. Participants who followed a healthy PDI had a 25% reduced IBD risk, whereas those who followed an unhealthy PDI had a 48% increased risk for disease development. Both CD and UC showed similar outcomes.
The EPIC cohort had a longer median follow-up time of 14.5 years, during which 548 people developed IBD. Healthy PDIs were linked to a 29% reduced risk for IBD, whereas unhealthy PDIs were associated with a 54% increased risk.
A healthy PDI halved the risk for surgery in participants from the UK Biobank, whereas an unhealthy PDI was associated with a twofold higher risk for surgery.
There were no significant associations between PDIs and other outcomes, such as cardiovascular disease, diabetes, or all-cause mortality.
The researchers also looked at the interactions between genetics and plant-based diets, but those results were not presented at the meeting.
However, Wellens said in an interview that people with a moderate to high risk for IBD based on their polygenetic risk score showed increased odds for IBD risk.
“We don’t test people for their genetic risk of IBD, but if people have close relatives with IBD, then there is probably an increased genetic risk of its development,” she added.
Commenting on the findings, James Lindsay, PhD, professor of inflammatory bowel disease, Queen Mary University of London in England, said that several recent epidemiological studies have highlighted “the negative impact of ultra-processed foods on increasing the risk of developing Crohn’s disease.”
Based on these studies, “one might assume that plant-based diets would be protective,” he said, however, the current study shows us “that plant-based diets are not all equal and there are unhealthy aspects to some.”
“Of course, showing that a diet is associated with an outcome is not the same as knowing that changing a diet will reduce the risk,” Lindsay added. “That requires a well-designed, carefully controlled trial.”
Wellens and Lindsay reported no relevant financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — according to the results of a large cohort study.
The study, which included both Crohn’s disease (CD) and ulcerative colitis (UC), also showed that diet quality may affect disease progression and surgery risk for individuals already diagnosed with IBD.
“Not all plant-based foods are equal — they don’t all have the same effect on health outcomes,” said study researcher, Judith Wellens, MD, PhD, gastroenterology resident at Leuven University Hospital in Belgium.
“We need to look at what people are eating more carefully because it isn’t black and white, with all plant-based food being good and animal-based food being bad,” said Wellens, who presented the data at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Although she advocates for plant-based diets, Wellens stressed that “they need to be individualized to ensure the overall dietary quality is good. Just cutting out meat products is not very helpful. We think it is the unhealthy additions to some plant-based diets that drive the IBD risk.”
Is It the Plants or the Processed Ingredients?
“Preclinical studies have already taught us that plant-based diets alter the gut microbiota in a beneficial way. However, many diets promoted for IBD — for example the Crohn’s disease exclusion diet — contain ingredients that are animal based. This is confusing for patients and for clinicians,” said Wellens.
To look more closely at the question, she and her colleagues analyzed data for 187,888 participants from the UK Biobank and 341,539 participants from across eight European countries from the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort. None of the participants had IBD at baseline.
Based on participant 24-hour dietary recalls, the researchers constructed plant-based diet indices (PDIs) with diets categorized as healthy (eg, whole grains, fruits, vegetables, legumes, and vegetarian protein alternatives) or unhealthy (eg, emulsifiers, refined grains, fries, fruit juices, sweets, desserts, sugar-sweetened beverages, and processed foods).
The primary outcome was the incidence of IBD (either CD or UC), whereas the secondary outcome was IBD-related surgery, thereby marking disease progression. Cox regression analysis estimated IBD risk and progression. Incidences of IBD were similar between the two cohorts.
In the UK Biobank cohort, 925 participants developed IBD over a median follow-up of 11.6 years. Participants who followed a healthy PDI had a 25% reduced IBD risk, whereas those who followed an unhealthy PDI had a 48% increased risk for disease development. Both CD and UC showed similar outcomes.
The EPIC cohort had a longer median follow-up time of 14.5 years, during which 548 people developed IBD. Healthy PDIs were linked to a 29% reduced risk for IBD, whereas unhealthy PDIs were associated with a 54% increased risk.
A healthy PDI halved the risk for surgery in participants from the UK Biobank, whereas an unhealthy PDI was associated with a twofold higher risk for surgery.
There were no significant associations between PDIs and other outcomes, such as cardiovascular disease, diabetes, or all-cause mortality.
The researchers also looked at the interactions between genetics and plant-based diets, but those results were not presented at the meeting.
However, Wellens said in an interview that people with a moderate to high risk for IBD based on their polygenetic risk score showed increased odds for IBD risk.
“We don’t test people for their genetic risk of IBD, but if people have close relatives with IBD, then there is probably an increased genetic risk of its development,” she added.
Commenting on the findings, James Lindsay, PhD, professor of inflammatory bowel disease, Queen Mary University of London in England, said that several recent epidemiological studies have highlighted “the negative impact of ultra-processed foods on increasing the risk of developing Crohn’s disease.”
Based on these studies, “one might assume that plant-based diets would be protective,” he said, however, the current study shows us “that plant-based diets are not all equal and there are unhealthy aspects to some.”
“Of course, showing that a diet is associated with an outcome is not the same as knowing that changing a diet will reduce the risk,” Lindsay added. “That requires a well-designed, carefully controlled trial.”
Wellens and Lindsay reported no relevant financial disclosures.
A version of this article appeared on Medscape.com.
BERLIN — according to the results of a large cohort study.
The study, which included both Crohn’s disease (CD) and ulcerative colitis (UC), also showed that diet quality may affect disease progression and surgery risk for individuals already diagnosed with IBD.
“Not all plant-based foods are equal — they don’t all have the same effect on health outcomes,” said study researcher, Judith Wellens, MD, PhD, gastroenterology resident at Leuven University Hospital in Belgium.
“We need to look at what people are eating more carefully because it isn’t black and white, with all plant-based food being good and animal-based food being bad,” said Wellens, who presented the data at the European Crohn’s and Colitis Organisation (ECCO) 2025 Congress.
Although she advocates for plant-based diets, Wellens stressed that “they need to be individualized to ensure the overall dietary quality is good. Just cutting out meat products is not very helpful. We think it is the unhealthy additions to some plant-based diets that drive the IBD risk.”
Is It the Plants or the Processed Ingredients?
“Preclinical studies have already taught us that plant-based diets alter the gut microbiota in a beneficial way. However, many diets promoted for IBD — for example the Crohn’s disease exclusion diet — contain ingredients that are animal based. This is confusing for patients and for clinicians,” said Wellens.
To look more closely at the question, she and her colleagues analyzed data for 187,888 participants from the UK Biobank and 341,539 participants from across eight European countries from the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort. None of the participants had IBD at baseline.
Based on participant 24-hour dietary recalls, the researchers constructed plant-based diet indices (PDIs) with diets categorized as healthy (eg, whole grains, fruits, vegetables, legumes, and vegetarian protein alternatives) or unhealthy (eg, emulsifiers, refined grains, fries, fruit juices, sweets, desserts, sugar-sweetened beverages, and processed foods).
The primary outcome was the incidence of IBD (either CD or UC), whereas the secondary outcome was IBD-related surgery, thereby marking disease progression. Cox regression analysis estimated IBD risk and progression. Incidences of IBD were similar between the two cohorts.
In the UK Biobank cohort, 925 participants developed IBD over a median follow-up of 11.6 years. Participants who followed a healthy PDI had a 25% reduced IBD risk, whereas those who followed an unhealthy PDI had a 48% increased risk for disease development. Both CD and UC showed similar outcomes.
The EPIC cohort had a longer median follow-up time of 14.5 years, during which 548 people developed IBD. Healthy PDIs were linked to a 29% reduced risk for IBD, whereas unhealthy PDIs were associated with a 54% increased risk.
A healthy PDI halved the risk for surgery in participants from the UK Biobank, whereas an unhealthy PDI was associated with a twofold higher risk for surgery.
There were no significant associations between PDIs and other outcomes, such as cardiovascular disease, diabetes, or all-cause mortality.
The researchers also looked at the interactions between genetics and plant-based diets, but those results were not presented at the meeting.
However, Wellens said in an interview that people with a moderate to high risk for IBD based on their polygenetic risk score showed increased odds for IBD risk.
“We don’t test people for their genetic risk of IBD, but if people have close relatives with IBD, then there is probably an increased genetic risk of its development,” she added.
Commenting on the findings, James Lindsay, PhD, professor of inflammatory bowel disease, Queen Mary University of London in England, said that several recent epidemiological studies have highlighted “the negative impact of ultra-processed foods on increasing the risk of developing Crohn’s disease.”
Based on these studies, “one might assume that plant-based diets would be protective,” he said, however, the current study shows us “that plant-based diets are not all equal and there are unhealthy aspects to some.”
“Of course, showing that a diet is associated with an outcome is not the same as knowing that changing a diet will reduce the risk,” Lindsay added. “That requires a well-designed, carefully controlled trial.”
Wellens and Lindsay reported no relevant financial disclosures.
A version of this article appeared on Medscape.com.
FROM ECCO 2025
Promising New Blood Test for Colorectal Cancer Screening
, the largest study of any blood-based CRC screening test.
With continued optimization, this blood test “may provide a convenient, effective option for colorectal cancer screening in the intended use population,” said Aasma Shaukat, MD, MPH, AGAF, with New York University Grossman School of Medicine in New York City.
CRC screening rates remain suboptimal, with nearly 40% of eligible adults in the United States not up to date with screening, noted Shaukat, who presented the study results at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
Blood-based testing offers a promising complementary approach that may boost patient adherence among unscreened individuals, she added.
The study evaluated the clinical performance of the investigational blood-based screening test in 27,010 adults aged ≥ 45 years at average-risk for CRC. Patients with no personal history of cancer, colorectal adenoma, or inflammatory bowel disease, as well as no family history of CRC or hereditary gastrointestinal cancer syndromes were eligible for the study.
Participants had blood drawn before bowel preparation for colonoscopy, and the blood test results were measured against colonoscopy findings.
The primary endpoints included sensitivity for CRC, specificity for advanced colorectal neoplasia, and negative and positive predictive value for advanced colorectal neoplasia. A secondary endpoint was sensitivity for advanced precancerous lesions.
The blood-based screening test met all primary endpoints, with a sensitivity for CRC of 79.2% and a specificity for advanced colorectal neoplasia of 91.5%, Shaukat reported at a conference briefing. Negative predictive value for advanced colorectal neoplasia was 90.8%, though the positive predictive value was only 15.5% and sensitivity for advanced precancerous lesions, a secondary endpoint, was 12.5%.
Similar results were achieved in a prespecified analysis in which performance of the blood test was weighted to match US census data for sex and age distributions. Sensitivity for CRC was 81.1%, specificity was 90.4%, and negative predictive value for advanced colorectal neoplasia was 90.5%; but the positive predictive value was 15.5%, and the sensitivity for advanced precancerous lesions was 13.7%.
This type of analysis provides “a snapshot of how [the test] would perform in the US population,” Shaukat explained, adding that the sensitivity for CRC and advanced precursor lesions was “lower than expected and will continue to be optimized in future research and development.”
It will also be important to determine when the test should be repeated and how often and to look at the determinants around cost and comparative effectiveness, she said. Modeling and other outcome studies — which will be forthcoming in the future years — could help shed some light on these questions.
Briefing moderator Julie Gralow, MD, ASCO chief medical officer, said it will be important to compare how this new blood test compares with Guardant Health’s Shield CRC blood test that was approved in 2024. Although there’s no study directly comparing the new blood test to Shield, data from the ECLIPSE study reported that Shield had 83% sensitivity for CRC and 90% specificity for advanced neoplasia, but only 13% sensitivity for advanced precancerous lesions — which appears similar, so far, to findings reported for the new blood test.
But any screening is better than no screening, and with further study, this blood test may “add another tool to our toolkit,” said Pamela Kunz, MD, director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut, who spoke at the briefing. Kunz said she is eager to see some of the future work optimizing the sensitivity and subsequent analyses that look at differences by race and ethnicity.
The study had no specific funding. Shaukat consults for Freenome Holdings and Iterative Health. Kunz declared ties with Ipsen, Novartis, Genentech/Roche, Amgen, Crinetics Pharmaceuticals, Natera, HUTCHMED, and ITM Isotope Technologies Munich. Gralow declared consulting or advisory roles with Genentech/Roche.
A version of this article appeared on Medscape.com .
, the largest study of any blood-based CRC screening test.
With continued optimization, this blood test “may provide a convenient, effective option for colorectal cancer screening in the intended use population,” said Aasma Shaukat, MD, MPH, AGAF, with New York University Grossman School of Medicine in New York City.
CRC screening rates remain suboptimal, with nearly 40% of eligible adults in the United States not up to date with screening, noted Shaukat, who presented the study results at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
Blood-based testing offers a promising complementary approach that may boost patient adherence among unscreened individuals, she added.
The study evaluated the clinical performance of the investigational blood-based screening test in 27,010 adults aged ≥ 45 years at average-risk for CRC. Patients with no personal history of cancer, colorectal adenoma, or inflammatory bowel disease, as well as no family history of CRC or hereditary gastrointestinal cancer syndromes were eligible for the study.
Participants had blood drawn before bowel preparation for colonoscopy, and the blood test results were measured against colonoscopy findings.
The primary endpoints included sensitivity for CRC, specificity for advanced colorectal neoplasia, and negative and positive predictive value for advanced colorectal neoplasia. A secondary endpoint was sensitivity for advanced precancerous lesions.
The blood-based screening test met all primary endpoints, with a sensitivity for CRC of 79.2% and a specificity for advanced colorectal neoplasia of 91.5%, Shaukat reported at a conference briefing. Negative predictive value for advanced colorectal neoplasia was 90.8%, though the positive predictive value was only 15.5% and sensitivity for advanced precancerous lesions, a secondary endpoint, was 12.5%.
Similar results were achieved in a prespecified analysis in which performance of the blood test was weighted to match US census data for sex and age distributions. Sensitivity for CRC was 81.1%, specificity was 90.4%, and negative predictive value for advanced colorectal neoplasia was 90.5%; but the positive predictive value was 15.5%, and the sensitivity for advanced precancerous lesions was 13.7%.
This type of analysis provides “a snapshot of how [the test] would perform in the US population,” Shaukat explained, adding that the sensitivity for CRC and advanced precursor lesions was “lower than expected and will continue to be optimized in future research and development.”
It will also be important to determine when the test should be repeated and how often and to look at the determinants around cost and comparative effectiveness, she said. Modeling and other outcome studies — which will be forthcoming in the future years — could help shed some light on these questions.
Briefing moderator Julie Gralow, MD, ASCO chief medical officer, said it will be important to compare how this new blood test compares with Guardant Health’s Shield CRC blood test that was approved in 2024. Although there’s no study directly comparing the new blood test to Shield, data from the ECLIPSE study reported that Shield had 83% sensitivity for CRC and 90% specificity for advanced neoplasia, but only 13% sensitivity for advanced precancerous lesions — which appears similar, so far, to findings reported for the new blood test.
But any screening is better than no screening, and with further study, this blood test may “add another tool to our toolkit,” said Pamela Kunz, MD, director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut, who spoke at the briefing. Kunz said she is eager to see some of the future work optimizing the sensitivity and subsequent analyses that look at differences by race and ethnicity.
The study had no specific funding. Shaukat consults for Freenome Holdings and Iterative Health. Kunz declared ties with Ipsen, Novartis, Genentech/Roche, Amgen, Crinetics Pharmaceuticals, Natera, HUTCHMED, and ITM Isotope Technologies Munich. Gralow declared consulting or advisory roles with Genentech/Roche.
A version of this article appeared on Medscape.com .
, the largest study of any blood-based CRC screening test.
With continued optimization, this blood test “may provide a convenient, effective option for colorectal cancer screening in the intended use population,” said Aasma Shaukat, MD, MPH, AGAF, with New York University Grossman School of Medicine in New York City.
CRC screening rates remain suboptimal, with nearly 40% of eligible adults in the United States not up to date with screening, noted Shaukat, who presented the study results at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
Blood-based testing offers a promising complementary approach that may boost patient adherence among unscreened individuals, she added.
The study evaluated the clinical performance of the investigational blood-based screening test in 27,010 adults aged ≥ 45 years at average-risk for CRC. Patients with no personal history of cancer, colorectal adenoma, or inflammatory bowel disease, as well as no family history of CRC or hereditary gastrointestinal cancer syndromes were eligible for the study.
Participants had blood drawn before bowel preparation for colonoscopy, and the blood test results were measured against colonoscopy findings.
The primary endpoints included sensitivity for CRC, specificity for advanced colorectal neoplasia, and negative and positive predictive value for advanced colorectal neoplasia. A secondary endpoint was sensitivity for advanced precancerous lesions.
The blood-based screening test met all primary endpoints, with a sensitivity for CRC of 79.2% and a specificity for advanced colorectal neoplasia of 91.5%, Shaukat reported at a conference briefing. Negative predictive value for advanced colorectal neoplasia was 90.8%, though the positive predictive value was only 15.5% and sensitivity for advanced precancerous lesions, a secondary endpoint, was 12.5%.
Similar results were achieved in a prespecified analysis in which performance of the blood test was weighted to match US census data for sex and age distributions. Sensitivity for CRC was 81.1%, specificity was 90.4%, and negative predictive value for advanced colorectal neoplasia was 90.5%; but the positive predictive value was 15.5%, and the sensitivity for advanced precancerous lesions was 13.7%.
This type of analysis provides “a snapshot of how [the test] would perform in the US population,” Shaukat explained, adding that the sensitivity for CRC and advanced precursor lesions was “lower than expected and will continue to be optimized in future research and development.”
It will also be important to determine when the test should be repeated and how often and to look at the determinants around cost and comparative effectiveness, she said. Modeling and other outcome studies — which will be forthcoming in the future years — could help shed some light on these questions.
Briefing moderator Julie Gralow, MD, ASCO chief medical officer, said it will be important to compare how this new blood test compares with Guardant Health’s Shield CRC blood test that was approved in 2024. Although there’s no study directly comparing the new blood test to Shield, data from the ECLIPSE study reported that Shield had 83% sensitivity for CRC and 90% specificity for advanced neoplasia, but only 13% sensitivity for advanced precancerous lesions — which appears similar, so far, to findings reported for the new blood test.
But any screening is better than no screening, and with further study, this blood test may “add another tool to our toolkit,” said Pamela Kunz, MD, director of the Center for Gastrointestinal Cancers at Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut, who spoke at the briefing. Kunz said she is eager to see some of the future work optimizing the sensitivity and subsequent analyses that look at differences by race and ethnicity.
The study had no specific funding. Shaukat consults for Freenome Holdings and Iterative Health. Kunz declared ties with Ipsen, Novartis, Genentech/Roche, Amgen, Crinetics Pharmaceuticals, Natera, HUTCHMED, and ITM Isotope Technologies Munich. Gralow declared consulting or advisory roles with Genentech/Roche.
A version of this article appeared on Medscape.com .
FROM ASCO GI 2025
Low-Dose Aspirin Cuts CRC Recurrence
according to findings from the phase 3 ALASCCA trial.
These results stress “the importance of upfront genomic testing” in patients with CRC, said Anna Martling, MD, PhD, from Karolinska Institutet, Stockholm, Sweden, who reported the findings at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
This is the first trial to show that mutations in the PI3K signaling pathway, beyond PIK3CA alterations, predict aspirin response, “expanding the targetable patient population substantially,” Martling added. Genetic mutations along the PI3K signaling pathway are found in about 30% of CRCs.
While aspirin as chemoprevention in CRC has been studied, data confirming its effectiveness as well as uptake of this approach in practice have been lacking, explained ASCO expert commenter Pamela Kunz, MD, with Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut.
“It’s really clear that this is a practice-changing study,” said Kunz. The findings indicate that this approach “checks all of the boxes: It’s effective, it’s low risk, it’s inexpensive, and it’s easy to administer.”
The trial included 626 patients (median age, 66 years; 52% women) with stages II-III colon cancer (67%) or stages I-III rectal cancer (33%) across 33 hospitals in Sweden, Denmark, Finland, and Norway.
Patients were stratified into two groups based on specific PI3K pathway alterations Group A (n = 314) included patients with PIK3CA mutations in exon 9 and/or 20, and group B (n = 312) included those with other PI3K pathway mutations, including PIK3CA mutations outside exon 9/20, or mutations in PIK3R1 or PTEN genes.
Participants in both groups were randomly allocated 1:1 to 160 mg/d of aspirin or placebo for 3 years. The primary outcome was CRC recurrence; disease-free survival was a secondary outcome.
Compared with placebo, aspirin reduced the risk for recurrence by 51% (hazard ratio [HR], 0.49) in patients with PIK3CA mutations, with a 3-year recurrence rate of 7.7% in those taking aspirin vs 14.1% in the placebo group.
“Interestingly,” Martling noted, in the exploratory arm that included other mutations along the PIK3 pathway beyond PIK3CA (Group B), the effect was even stronger. Patients in this group had a 58% (HR, 0.42) lower risk for recurrence than those in the placebo group, with a 3-year recurrence rate of 7.7% in the aspirin group vs 16.8% recurrence rate in the placebo group.
Aspirin also had a disease-free survival benefit in both groups, but it was significant only in group B.
While the study was not specifically designed for subgroup analysis, the benefit of aspirin was observed in all subgroups examined, including men and women with colon or rectal cancer, those who did and did not receive neoadjuvant or adjuvant treatment, and those with stages I-III disease.
The incidence of adverse events was as expected and severe side effects associated with 160 mg/d aspirin were rare, Martling said.
Both Martling and Kunz predicted that these findings will change clinical practice. “I anticipate that we’ll be seeing adoption of this [strategy],” Kunz said.
This study received funding from the Swedish Research Council, Swedish Cancer Society, ALF (a regional agreement on medical training and clinical research between the Stockholm County Council and Karolinska Institutet), and the Stockholm Cancer Society. Martling disclosed various relationships with Bactiguard, Smartcella, CarpoNovum and Pfizer. Kunz disclosed relationships with Bristol-Myers Squibb, Novartis and TayzeBio.
A version of this article appeared on Medscape.com .
according to findings from the phase 3 ALASCCA trial.
These results stress “the importance of upfront genomic testing” in patients with CRC, said Anna Martling, MD, PhD, from Karolinska Institutet, Stockholm, Sweden, who reported the findings at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
This is the first trial to show that mutations in the PI3K signaling pathway, beyond PIK3CA alterations, predict aspirin response, “expanding the targetable patient population substantially,” Martling added. Genetic mutations along the PI3K signaling pathway are found in about 30% of CRCs.
While aspirin as chemoprevention in CRC has been studied, data confirming its effectiveness as well as uptake of this approach in practice have been lacking, explained ASCO expert commenter Pamela Kunz, MD, with Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut.
“It’s really clear that this is a practice-changing study,” said Kunz. The findings indicate that this approach “checks all of the boxes: It’s effective, it’s low risk, it’s inexpensive, and it’s easy to administer.”
The trial included 626 patients (median age, 66 years; 52% women) with stages II-III colon cancer (67%) or stages I-III rectal cancer (33%) across 33 hospitals in Sweden, Denmark, Finland, and Norway.
Patients were stratified into two groups based on specific PI3K pathway alterations Group A (n = 314) included patients with PIK3CA mutations in exon 9 and/or 20, and group B (n = 312) included those with other PI3K pathway mutations, including PIK3CA mutations outside exon 9/20, or mutations in PIK3R1 or PTEN genes.
Participants in both groups were randomly allocated 1:1 to 160 mg/d of aspirin or placebo for 3 years. The primary outcome was CRC recurrence; disease-free survival was a secondary outcome.
Compared with placebo, aspirin reduced the risk for recurrence by 51% (hazard ratio [HR], 0.49) in patients with PIK3CA mutations, with a 3-year recurrence rate of 7.7% in those taking aspirin vs 14.1% in the placebo group.
“Interestingly,” Martling noted, in the exploratory arm that included other mutations along the PIK3 pathway beyond PIK3CA (Group B), the effect was even stronger. Patients in this group had a 58% (HR, 0.42) lower risk for recurrence than those in the placebo group, with a 3-year recurrence rate of 7.7% in the aspirin group vs 16.8% recurrence rate in the placebo group.
Aspirin also had a disease-free survival benefit in both groups, but it was significant only in group B.
While the study was not specifically designed for subgroup analysis, the benefit of aspirin was observed in all subgroups examined, including men and women with colon or rectal cancer, those who did and did not receive neoadjuvant or adjuvant treatment, and those with stages I-III disease.
The incidence of adverse events was as expected and severe side effects associated with 160 mg/d aspirin were rare, Martling said.
Both Martling and Kunz predicted that these findings will change clinical practice. “I anticipate that we’ll be seeing adoption of this [strategy],” Kunz said.
This study received funding from the Swedish Research Council, Swedish Cancer Society, ALF (a regional agreement on medical training and clinical research between the Stockholm County Council and Karolinska Institutet), and the Stockholm Cancer Society. Martling disclosed various relationships with Bactiguard, Smartcella, CarpoNovum and Pfizer. Kunz disclosed relationships with Bristol-Myers Squibb, Novartis and TayzeBio.
A version of this article appeared on Medscape.com .
according to findings from the phase 3 ALASCCA trial.
These results stress “the importance of upfront genomic testing” in patients with CRC, said Anna Martling, MD, PhD, from Karolinska Institutet, Stockholm, Sweden, who reported the findings at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium 2025 in San Francisco.
This is the first trial to show that mutations in the PI3K signaling pathway, beyond PIK3CA alterations, predict aspirin response, “expanding the targetable patient population substantially,” Martling added. Genetic mutations along the PI3K signaling pathway are found in about 30% of CRCs.
While aspirin as chemoprevention in CRC has been studied, data confirming its effectiveness as well as uptake of this approach in practice have been lacking, explained ASCO expert commenter Pamela Kunz, MD, with Smilow Cancer Hospital and Yale Cancer Center, New Haven, Connecticut.
“It’s really clear that this is a practice-changing study,” said Kunz. The findings indicate that this approach “checks all of the boxes: It’s effective, it’s low risk, it’s inexpensive, and it’s easy to administer.”
The trial included 626 patients (median age, 66 years; 52% women) with stages II-III colon cancer (67%) or stages I-III rectal cancer (33%) across 33 hospitals in Sweden, Denmark, Finland, and Norway.
Patients were stratified into two groups based on specific PI3K pathway alterations Group A (n = 314) included patients with PIK3CA mutations in exon 9 and/or 20, and group B (n = 312) included those with other PI3K pathway mutations, including PIK3CA mutations outside exon 9/20, or mutations in PIK3R1 or PTEN genes.
Participants in both groups were randomly allocated 1:1 to 160 mg/d of aspirin or placebo for 3 years. The primary outcome was CRC recurrence; disease-free survival was a secondary outcome.
Compared with placebo, aspirin reduced the risk for recurrence by 51% (hazard ratio [HR], 0.49) in patients with PIK3CA mutations, with a 3-year recurrence rate of 7.7% in those taking aspirin vs 14.1% in the placebo group.
“Interestingly,” Martling noted, in the exploratory arm that included other mutations along the PIK3 pathway beyond PIK3CA (Group B), the effect was even stronger. Patients in this group had a 58% (HR, 0.42) lower risk for recurrence than those in the placebo group, with a 3-year recurrence rate of 7.7% in the aspirin group vs 16.8% recurrence rate in the placebo group.
Aspirin also had a disease-free survival benefit in both groups, but it was significant only in group B.
While the study was not specifically designed for subgroup analysis, the benefit of aspirin was observed in all subgroups examined, including men and women with colon or rectal cancer, those who did and did not receive neoadjuvant or adjuvant treatment, and those with stages I-III disease.
The incidence of adverse events was as expected and severe side effects associated with 160 mg/d aspirin were rare, Martling said.
Both Martling and Kunz predicted that these findings will change clinical practice. “I anticipate that we’ll be seeing adoption of this [strategy],” Kunz said.
This study received funding from the Swedish Research Council, Swedish Cancer Society, ALF (a regional agreement on medical training and clinical research between the Stockholm County Council and Karolinska Institutet), and the Stockholm Cancer Society. Martling disclosed various relationships with Bactiguard, Smartcella, CarpoNovum and Pfizer. Kunz disclosed relationships with Bristol-Myers Squibb, Novartis and TayzeBio.
A version of this article appeared on Medscape.com .
FROM ASCO GI 2025
Vitiligo: Updated Guidelines, New Treatments Reviewed
of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.
A Distinct Disease
An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation.
“This is a message we must change,” he said.
The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.
As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.
Updated Guidelines
Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.
“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.
For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.
However, when a decision is made to pursue treatment, its goals should be:
- Halting disease progression and melanocyte loss
- Achieving repigmentation (a process that can take 6-24 months)
- Preventing relapse after treatment discontinuation
For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.
In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.
Targeted Therapies
Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.
Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.
Key Findings
The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.
“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.
Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.
“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.
He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.
New Therapies
Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.
In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.
On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.
“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.
This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.
A Distinct Disease
An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation.
“This is a message we must change,” he said.
The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.
As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.
Updated Guidelines
Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.
“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.
For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.
However, when a decision is made to pursue treatment, its goals should be:
- Halting disease progression and melanocyte loss
- Achieving repigmentation (a process that can take 6-24 months)
- Preventing relapse after treatment discontinuation
For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.
In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.
Targeted Therapies
Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.
Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.
Key Findings
The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.
“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.
Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.
“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.
He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.
New Therapies
Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.
In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.
On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.
“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.
This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
of the disease, delegates heard at a recent conference, the Dermatology Days of Paris 2024, organized by the French Society of Dermatology.
A Distinct Disease
An estimated 65% of patients with vitiligo in Europe have been told that their disease is untreatable, according to a recent international study, and this figure rises to 75% in France, Julien Seneschal, MD, PhD, professor of dermatology at Bordeaux University Hospital in Bordeaux, France, told the audience during his presentation.
“This is a message we must change,” he said.
The survey also revealed that in France, even when treatment is offered, 80% of patients do not receive appropriate care. However, treatments do exist, and novel approaches are revolutionizing the management of patients, whatever the degree of severity, he explained.
As a specialist in inflammatory and autoimmune skin diseases, he stressed that these advances are important because vitiligo is a distinct disease and not merely a cosmetic issue. When widespread, it has a significant impact on quality of life and can lead to depression, anxiety, and even suicidal thoughts, even though it does not affect life expectancy.
Updated Guidelines
Since October 2023, new international guidelines for vitiligo management have defined a therapeutic algorithm.
“Nowadays, we place the patient at the center of therapeutic decision-making,” Seneschal said. It is essential to educate patients about the disease and take the time to understand their treatment goals.
For patients with mild vitiligo that does not affect quality of life, simple monitoring may suffice.
However, when a decision is made to pursue treatment, its goals should be:
- Halting disease progression and melanocyte loss
- Achieving repigmentation (a process that can take 6-24 months)
- Preventing relapse after treatment discontinuation
For moderate cases affecting less than 10% of the skin surface, localized treatment is recommended. Previously, topical corticosteroids were used for body lesions, while tacrolimus 0.1% (off-label) was often prescribed for the face and neck. However, as of March 2024, tacrolimus has been officially approved for use in patients aged ≥ 2 years.
In more severe, generalized, and/or active cases, oral treatments such as corticosteroids taken twice weekly for 12-24 weeks can stabilize the disease in 80% of cases (off-label use). Other off-label options include methotrexate, cyclosporine, and tetracyclines.
Targeted Therapies
Recent targeted therapies have significantly advanced the treatment of moderate to severe vitiligo. Since January 2024, the Janus kinase 1 (JAK1)/JAK2 inhibitor ruxolitinib cream has been available in community pharmacies after previously being restricted to hospital use, Seneschal said, and can have spectacular results if previous treatments have failed.
Ruxolitinib is approved for patients aged > 12 years with nonsegmental vitiligo and facial involvement, covering up to 10% of the body surface area. Treatment typically lasts 6 months to 1 year.
Key Findings
The cream-formulated drug has been demonstrated effective in reducing inflammation in two phase 3 clinical trials published in the New England Journal of Medicine that demonstrated its efficacy and safety in patients aged ≥ 12 years. The treatment was well tolerated despite some mild acne-like reactions in 8% of patients. It was shown to be very effective on the face, with a reduction of over 75% in facial lesions in more than 50% of patients, and had good effectiveness on the body, with a 50% decrease in lesions in more than 50% of patients on the body, trunk, arms, and legs, excluding hands and feet.
“Areas like the underarms, hands, and feet are more resistant to treatment,” Seneschal noted.
Although some improvement continues after 1 year, disease recurrence is common if treatment is stopped: Only 40% of patients maintain therapeutic benefits in the year following discontinuation.
“It is therefore important to consider the value of continuing treatment in order to achieve better efficacy or to maintain the repigmentation obtained,” Seneschal said.
He stressed that all treatments should be paired with phototherapy, typically narrowband UVB, to accelerate repigmentation. “There is no increased skin cancer risk in vitiligo patients treated with narrowband UVB,” Seneschal said.
New Therapies
Emerging treatments under development, including injectable biologics alone or in combination with phototherapy, show great promise, he said. Oral JAK inhibitors such as ritlecitinib, upadacitinib, and povorcitinib are also under investigation.
In particular, ritlecitinib, a JAK3/TEC pathway inhibitor, has shown significant reductions in affected skin area in severely affected patients in a phase 2b trial. Phase 3 trials are now underway.
On the safety profile of JAK inhibitors, Seneschal said that studies are reassuring but highlighted the need to monitor cardiovascular, thromboembolic, and infectious risks.
“The question of safety is important because vitiligo is a visible but nonsevere condition, and we do not want to expose patients to unnecessary risks,” added Gaëlle Quéreux, MD, PhD, president of the French Society of Dermatology.
This story was translated from Medscape’s French edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Pruritus: Diagnosing and Treating Older Adults
Chronic pruritus is a common problem among older individuals. During a session at the Dermatology Days of Paris 2024 conference dedicated to general practitioners, Juliette Delaunay, MD, a dermatologist and venereologist at Angers University Hospital Center in Angers, France, and Gabrielle Lisembard, MD, a general practitioner in the French town Grand-Fort-Philippe, discussed diagnostic approaches and key principles for the therapeutic management of pruritus.
Identifying Causes
“Pruritus in older people is most often linked to physiological changes in the skin caused by aging, leading to significant xerosis. However, before attributing it to aging, we need to rule out several causes,” Delaunay noted.
Beyond simple aging, one must consider autoimmune bullous dermatoses (bullous pemphigoid), drug-related causes, metabolic disorders (can occur at any age), cutaneous T-cell lymphomas, scabies, lice, and HIV infection.
Senile Pruritus
Aging-related xerosis can cause senile pruritus, often presenting as itching with scratch marks and dry skin. “This is a diagnosis of exclusion,” Delaunay insisted.
In older individuals with pruritus, initial examinations should include complete blood cell count (CBC), liver function tests, and thyroid-stimulating hormone levels. Syphilis serology, HIV testing, and beta-2 microglobulin levels are secondary evaluations. Renal function analysis may also be performed, and imaging may be required to investigate neoplasia.
“Annual etiological reassessment is essential if the initial evaluation is negative, as patients may later develop or report a neoplasia or hematological disorder,” Delaunay emphasized.
Paraneoplastic pruritus can occur, particularly those linked to hematological disorders (lymphomas, polycythemia, or myeloma).
Bullous Pemphigoid
Bullous pemphigoid often begins with pruritus, which can be severe and lead to insomnia. General practitioners should consider bullous pemphigoids when there is a bullous rash (tense blisters with citrine content) or an urticarial or chronic eczematous rash that does not heal spontaneously within a few days. The first-line biologic test to confirm the diagnosis is the CBC, which may reveal significant hypereosinophilia.
The diagnosis is confirmed by a skin biopsy showing a subepidermal blister with a preserved roof, unlike intraepidermal dermatoses, where the roof ruptures.
Direct immunofluorescence revealed deposits of immunoglobulin G antibodies along the dermoepidermal junction.
Approximately 40% of cases of bullous pemphigoid are associated with neurodegenerative diseases, such as stroke, parkinsonism, or dementia syndromes — occurring at a rate two to three times higher than in the general population.
It’s important to identify drugs that induce bullous pemphigoid, such as gliptins, anti-programmed cell death protein 1-programmed death-ligand 1 agents, loop diuretics (furosemide and bumetanide), anti-aldosterones (spironolactone), antiarrhythmics (amiodarone), and neuroleptics (phenothiazines).
“Stopping the medication is not mandatory if the bullous pemphigoid is well controlled by local or systemic treatments and the medication is essential. The decision to stop should be made on a case-by-case basis in consultation with the treating specialist,” Delaunay emphasized.
Treatment consists of very strong local corticosteroid therapy as the first-line treatment. If ineffective, systemic treatments based on methotrexate, oral corticosteroids, or immunomodulatory agents may be considered. Hospitalization is sometimes required.
Drug-Induced Pruritus
Drug-induced pruritus is common because older individuals often take multiple medications (antihypertensives, statins, oral hypoglycemics, psychotropic drugs, antiarrhythmics, etc.). “Sometimes, drug-induced pruritus can occur even if the medication was started several months or years ago,” Delaunay emphasized.
The lesions are generally nonspecific and scratching.
“This is a diagnosis of exclusion for other causes of pruritus. In the absence of specific lesions pointing to a dermatosis, eviction/reintroduction tests with treatments should be conducted one by one, which can be quite lengthy,” she explained.
Awareness for Scabies
Delaunay reminded attendees to consider scabies in older individuals when classic signs of pruritus flare up at night, with a rash affecting the face, scabs, or vesicles in the interdigital spaces of the hands, wrists, scrotal area, or the peri-mammary region.
“The incidence is increasing, particularly in nursing homes, where outbreaks pose a significant risk of rapid spread. Treatment involves three courses of topical and oral treatments administered on days 0, 7, and 14. All contact cases must also be treated. Sometimes, these thick lesions are stripped with 10% salicylated petroleum jelly. Environmental treatment with acaricides is essential, along with strict isolation measures,” Delaunay emphasized.
Adherent nits on the scalp or other hairy areas should raise suspicion of pediculosis.
Neurogenic and Psychogenic Origins
Neurogenic pruritus can occur during a stroke, presenting as contralateral pruritus, or in the presence of a brain tumor or following neurosurgery. Opioid-containing medications may also induce neurogenic pruritus.
The presence of unilateral painful or itchy sensations should prompt the investigation of shingles in older individuals.
Psychogenic pruritus is also common and can arise in the context of psychosis with parasitophobia or as part of anxiety-depression syndromes.
Supportive Measures
For managing pruritus, it is essential to:
- Keep nails trimmed short
- Wash with cold or lukewarm water
- Use lipid-rice soaps and syndets
- Avoid irritants, including antiseptics, cologne, no-rinse cleansers, and steroidal or nonsteroidal anti-inflammatory drugs
- Limit bathing frequency
- Avoid wearing nylon, wool, or tight clothing
- Minimize exposure to heat and excessive heating
“Alternatives to scratching, such as applying a moisturizing emollient, can be beneficial and may have a placebo effect,” explained the dermatologist. She further emphasized that local corticosteroids are effective only in the presence of inflammatory dermatosis and should not be applied to healthy skin. Similarly, antihistamines should only be prescribed if the pruritus is histamine-mediated.
Capsaicin may be useful in the treatment of localized neuropathic pruritus.
In cases of neurogenic pruritus, gabapentin and pregabalin may be prescribed, but tolerance can be problematic at this age. Other measures include acupuncture, cryotherapy, relaxation, hypnosis, psychotherapy, and music therapy. In cases of repeated therapeutic failure, patients may be treated with biotherapy (dupilumab) by a dermatologist.
This story was translated from Medscape’s French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Chronic pruritus is a common problem among older individuals. During a session at the Dermatology Days of Paris 2024 conference dedicated to general practitioners, Juliette Delaunay, MD, a dermatologist and venereologist at Angers University Hospital Center in Angers, France, and Gabrielle Lisembard, MD, a general practitioner in the French town Grand-Fort-Philippe, discussed diagnostic approaches and key principles for the therapeutic management of pruritus.
Identifying Causes
“Pruritus in older people is most often linked to physiological changes in the skin caused by aging, leading to significant xerosis. However, before attributing it to aging, we need to rule out several causes,” Delaunay noted.
Beyond simple aging, one must consider autoimmune bullous dermatoses (bullous pemphigoid), drug-related causes, metabolic disorders (can occur at any age), cutaneous T-cell lymphomas, scabies, lice, and HIV infection.
Senile Pruritus
Aging-related xerosis can cause senile pruritus, often presenting as itching with scratch marks and dry skin. “This is a diagnosis of exclusion,” Delaunay insisted.
In older individuals with pruritus, initial examinations should include complete blood cell count (CBC), liver function tests, and thyroid-stimulating hormone levels. Syphilis serology, HIV testing, and beta-2 microglobulin levels are secondary evaluations. Renal function analysis may also be performed, and imaging may be required to investigate neoplasia.
“Annual etiological reassessment is essential if the initial evaluation is negative, as patients may later develop or report a neoplasia or hematological disorder,” Delaunay emphasized.
Paraneoplastic pruritus can occur, particularly those linked to hematological disorders (lymphomas, polycythemia, or myeloma).
Bullous Pemphigoid
Bullous pemphigoid often begins with pruritus, which can be severe and lead to insomnia. General practitioners should consider bullous pemphigoids when there is a bullous rash (tense blisters with citrine content) or an urticarial or chronic eczematous rash that does not heal spontaneously within a few days. The first-line biologic test to confirm the diagnosis is the CBC, which may reveal significant hypereosinophilia.
The diagnosis is confirmed by a skin biopsy showing a subepidermal blister with a preserved roof, unlike intraepidermal dermatoses, where the roof ruptures.
Direct immunofluorescence revealed deposits of immunoglobulin G antibodies along the dermoepidermal junction.
Approximately 40% of cases of bullous pemphigoid are associated with neurodegenerative diseases, such as stroke, parkinsonism, or dementia syndromes — occurring at a rate two to three times higher than in the general population.
It’s important to identify drugs that induce bullous pemphigoid, such as gliptins, anti-programmed cell death protein 1-programmed death-ligand 1 agents, loop diuretics (furosemide and bumetanide), anti-aldosterones (spironolactone), antiarrhythmics (amiodarone), and neuroleptics (phenothiazines).
“Stopping the medication is not mandatory if the bullous pemphigoid is well controlled by local or systemic treatments and the medication is essential. The decision to stop should be made on a case-by-case basis in consultation with the treating specialist,” Delaunay emphasized.
Treatment consists of very strong local corticosteroid therapy as the first-line treatment. If ineffective, systemic treatments based on methotrexate, oral corticosteroids, or immunomodulatory agents may be considered. Hospitalization is sometimes required.
Drug-Induced Pruritus
Drug-induced pruritus is common because older individuals often take multiple medications (antihypertensives, statins, oral hypoglycemics, psychotropic drugs, antiarrhythmics, etc.). “Sometimes, drug-induced pruritus can occur even if the medication was started several months or years ago,” Delaunay emphasized.
The lesions are generally nonspecific and scratching.
“This is a diagnosis of exclusion for other causes of pruritus. In the absence of specific lesions pointing to a dermatosis, eviction/reintroduction tests with treatments should be conducted one by one, which can be quite lengthy,” she explained.
Awareness for Scabies
Delaunay reminded attendees to consider scabies in older individuals when classic signs of pruritus flare up at night, with a rash affecting the face, scabs, or vesicles in the interdigital spaces of the hands, wrists, scrotal area, or the peri-mammary region.
“The incidence is increasing, particularly in nursing homes, where outbreaks pose a significant risk of rapid spread. Treatment involves three courses of topical and oral treatments administered on days 0, 7, and 14. All contact cases must also be treated. Sometimes, these thick lesions are stripped with 10% salicylated petroleum jelly. Environmental treatment with acaricides is essential, along with strict isolation measures,” Delaunay emphasized.
Adherent nits on the scalp or other hairy areas should raise suspicion of pediculosis.
Neurogenic and Psychogenic Origins
Neurogenic pruritus can occur during a stroke, presenting as contralateral pruritus, or in the presence of a brain tumor or following neurosurgery. Opioid-containing medications may also induce neurogenic pruritus.
The presence of unilateral painful or itchy sensations should prompt the investigation of shingles in older individuals.
Psychogenic pruritus is also common and can arise in the context of psychosis with parasitophobia or as part of anxiety-depression syndromes.
Supportive Measures
For managing pruritus, it is essential to:
- Keep nails trimmed short
- Wash with cold or lukewarm water
- Use lipid-rice soaps and syndets
- Avoid irritants, including antiseptics, cologne, no-rinse cleansers, and steroidal or nonsteroidal anti-inflammatory drugs
- Limit bathing frequency
- Avoid wearing nylon, wool, or tight clothing
- Minimize exposure to heat and excessive heating
“Alternatives to scratching, such as applying a moisturizing emollient, can be beneficial and may have a placebo effect,” explained the dermatologist. She further emphasized that local corticosteroids are effective only in the presence of inflammatory dermatosis and should not be applied to healthy skin. Similarly, antihistamines should only be prescribed if the pruritus is histamine-mediated.
Capsaicin may be useful in the treatment of localized neuropathic pruritus.
In cases of neurogenic pruritus, gabapentin and pregabalin may be prescribed, but tolerance can be problematic at this age. Other measures include acupuncture, cryotherapy, relaxation, hypnosis, psychotherapy, and music therapy. In cases of repeated therapeutic failure, patients may be treated with biotherapy (dupilumab) by a dermatologist.
This story was translated from Medscape’s French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Chronic pruritus is a common problem among older individuals. During a session at the Dermatology Days of Paris 2024 conference dedicated to general practitioners, Juliette Delaunay, MD, a dermatologist and venereologist at Angers University Hospital Center in Angers, France, and Gabrielle Lisembard, MD, a general practitioner in the French town Grand-Fort-Philippe, discussed diagnostic approaches and key principles for the therapeutic management of pruritus.
Identifying Causes
“Pruritus in older people is most often linked to physiological changes in the skin caused by aging, leading to significant xerosis. However, before attributing it to aging, we need to rule out several causes,” Delaunay noted.
Beyond simple aging, one must consider autoimmune bullous dermatoses (bullous pemphigoid), drug-related causes, metabolic disorders (can occur at any age), cutaneous T-cell lymphomas, scabies, lice, and HIV infection.
Senile Pruritus
Aging-related xerosis can cause senile pruritus, often presenting as itching with scratch marks and dry skin. “This is a diagnosis of exclusion,” Delaunay insisted.
In older individuals with pruritus, initial examinations should include complete blood cell count (CBC), liver function tests, and thyroid-stimulating hormone levels. Syphilis serology, HIV testing, and beta-2 microglobulin levels are secondary evaluations. Renal function analysis may also be performed, and imaging may be required to investigate neoplasia.
“Annual etiological reassessment is essential if the initial evaluation is negative, as patients may later develop or report a neoplasia or hematological disorder,” Delaunay emphasized.
Paraneoplastic pruritus can occur, particularly those linked to hematological disorders (lymphomas, polycythemia, or myeloma).
Bullous Pemphigoid
Bullous pemphigoid often begins with pruritus, which can be severe and lead to insomnia. General practitioners should consider bullous pemphigoids when there is a bullous rash (tense blisters with citrine content) or an urticarial or chronic eczematous rash that does not heal spontaneously within a few days. The first-line biologic test to confirm the diagnosis is the CBC, which may reveal significant hypereosinophilia.
The diagnosis is confirmed by a skin biopsy showing a subepidermal blister with a preserved roof, unlike intraepidermal dermatoses, where the roof ruptures.
Direct immunofluorescence revealed deposits of immunoglobulin G antibodies along the dermoepidermal junction.
Approximately 40% of cases of bullous pemphigoid are associated with neurodegenerative diseases, such as stroke, parkinsonism, or dementia syndromes — occurring at a rate two to three times higher than in the general population.
It’s important to identify drugs that induce bullous pemphigoid, such as gliptins, anti-programmed cell death protein 1-programmed death-ligand 1 agents, loop diuretics (furosemide and bumetanide), anti-aldosterones (spironolactone), antiarrhythmics (amiodarone), and neuroleptics (phenothiazines).
“Stopping the medication is not mandatory if the bullous pemphigoid is well controlled by local or systemic treatments and the medication is essential. The decision to stop should be made on a case-by-case basis in consultation with the treating specialist,” Delaunay emphasized.
Treatment consists of very strong local corticosteroid therapy as the first-line treatment. If ineffective, systemic treatments based on methotrexate, oral corticosteroids, or immunomodulatory agents may be considered. Hospitalization is sometimes required.
Drug-Induced Pruritus
Drug-induced pruritus is common because older individuals often take multiple medications (antihypertensives, statins, oral hypoglycemics, psychotropic drugs, antiarrhythmics, etc.). “Sometimes, drug-induced pruritus can occur even if the medication was started several months or years ago,” Delaunay emphasized.
The lesions are generally nonspecific and scratching.
“This is a diagnosis of exclusion for other causes of pruritus. In the absence of specific lesions pointing to a dermatosis, eviction/reintroduction tests with treatments should be conducted one by one, which can be quite lengthy,” she explained.
Awareness for Scabies
Delaunay reminded attendees to consider scabies in older individuals when classic signs of pruritus flare up at night, with a rash affecting the face, scabs, or vesicles in the interdigital spaces of the hands, wrists, scrotal area, or the peri-mammary region.
“The incidence is increasing, particularly in nursing homes, where outbreaks pose a significant risk of rapid spread. Treatment involves three courses of topical and oral treatments administered on days 0, 7, and 14. All contact cases must also be treated. Sometimes, these thick lesions are stripped with 10% salicylated petroleum jelly. Environmental treatment with acaricides is essential, along with strict isolation measures,” Delaunay emphasized.
Adherent nits on the scalp or other hairy areas should raise suspicion of pediculosis.
Neurogenic and Psychogenic Origins
Neurogenic pruritus can occur during a stroke, presenting as contralateral pruritus, or in the presence of a brain tumor or following neurosurgery. Opioid-containing medications may also induce neurogenic pruritus.
The presence of unilateral painful or itchy sensations should prompt the investigation of shingles in older individuals.
Psychogenic pruritus is also common and can arise in the context of psychosis with parasitophobia or as part of anxiety-depression syndromes.
Supportive Measures
For managing pruritus, it is essential to:
- Keep nails trimmed short
- Wash with cold or lukewarm water
- Use lipid-rice soaps and syndets
- Avoid irritants, including antiseptics, cologne, no-rinse cleansers, and steroidal or nonsteroidal anti-inflammatory drugs
- Limit bathing frequency
- Avoid wearing nylon, wool, or tight clothing
- Minimize exposure to heat and excessive heating
“Alternatives to scratching, such as applying a moisturizing emollient, can be beneficial and may have a placebo effect,” explained the dermatologist. She further emphasized that local corticosteroids are effective only in the presence of inflammatory dermatosis and should not be applied to healthy skin. Similarly, antihistamines should only be prescribed if the pruritus is histamine-mediated.
Capsaicin may be useful in the treatment of localized neuropathic pruritus.
In cases of neurogenic pruritus, gabapentin and pregabalin may be prescribed, but tolerance can be problematic at this age. Other measures include acupuncture, cryotherapy, relaxation, hypnosis, psychotherapy, and music therapy. In cases of repeated therapeutic failure, patients may be treated with biotherapy (dupilumab) by a dermatologist.
This story was translated from Medscape’s French edition using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Management of Children and Adolescents With Long COVID
Current management of children and adolescents with long COVID was the focus of various presentations at the 3rd Long COVID Congress in Berlin in November 2024. The congress aimed to facilitate in-depth discussions on recent research projects, diagnostic procedures, and therapeutic approaches to enhance care for long COVID patients. This year, the focus was on research into long COVID in children and adolescents and how to improve their care.
Uta Behrends, MD, head of the Munich Chronic Fatigue Center, Center for Pediatric and Adolescent Medicine at the Technical University of Munich, Germany, and Nicole Toepfner, MD, a pediatrician at the University Hospital in Dresden, Germany, provided an initial overview.
Prevalence Data Are Limited
Data on the incidence and prevalence of the condition in children and adolescents are limited because most studies have primarily examined adults. A 2022 Swiss study estimated that it affects between 2% and 3.5% of children and adolescents who contract COVID-19. A recent study published in JAMA involving 5367 children and adolescents found that 20% of children aged 6-11 years and 14% of adolescents met the researchers’ criteria for long COVID.
Impaired Mental Health
Initial data from the latest wave of the population-based longitudinal COPSY (Corona and Psyche) study showed that compared with their peer group children and adolescents diagnosed with long COVID exhibit significantly higher rates of psychological issues and depressive symptoms. Although no significant differences were found in anxiety levels, study leader Ulrike Ravens-Sieberer, PhD, from the University Medical Center Hamburg-Eppendorf, Germany, told the congress that those with long COVID do also report more frequent somatic or psychological health complaints and lower health-related quality of life than peers.
Addressing Data Gaps
Another study due to launch in January 2025 and run through to 2028 is the COVYOUTH data study, which aims to better understand the nature, frequency, and risk factors of COVID-related sequelae in children and adolescents.
Study centers include Ruhr University Bochum, University Hospital Cologne, the Paul-Ehrlich-Institut, and University Medical Center Hamburg-Eppendorf. Using routine data from statutory health insurance and newly developed case definitions, researchers aim to investigate:
- Psychological stress caused by COVID-19 measures
- Post-COVID syndrome and myocarditis
- Adverse effects of COVID-19 vaccinations
Specialized Diagnostics and Care
The Post-COVID Kids Bavaria project offers specialized diagnostics and care for children and adolescents, including a day clinic, telemedical follow-ups, and an inpatient pain therapy module providing age-appropriate care as close to patients’ homes as possible.
MOVE-COVID is a model project for patient-focused research on long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) involving university pediatric hospitals in Freiburg, Heidelberg, Tübingen, and Ulm. It also aims to establish a care network across the state of Baden-Württemberg, including the establishment of long COVID outpatient clinics at social pediatric centers in the network hospitals, as well as enhanced telemedical support and standardized diagnostic and treatment protocols. “MOVE-COVID has successfully consolidated competencies and capacities in patient care, health services research, and patient-focused studies across multiple centers,” Behrends said.
Chronic Pain and Fatigue
Post-COVID syndromes in children and adolescents may feature profound fatigue, unrefreshing sleep, post-exertional malaise, cognitive dysfunction, and orthostatic intolerance and overlap with conditions such as ME/CFS. According to the German patient association Fatigatio, Berlin, research and studies for these conditions in children remain limited compared with those in adults. However, the US Centers for Disease Control estimates that around 2% of ME/CFS patients are children or adolescents, with the majority being teenagers.
Two inpatient treatment concepts, SHARK and TIGER, developed by Lea Höfel, PhD, head of the Centre for Pain Therapy for Young People and the Psychological Service at the Children’s Hospital in Garmisch-Partenkirchen, address chronic pain, fatigue, and ME/CFS in young people. These programs integrate structured breaks and flexible access to multiple therapists as needed. The TIGER program focuses on those with post-exertional malaise, while the SHARK program is designed for adolescents without this symptom. Both programs last 4.5-5 weeks and emphasize symptom reduction, education, and energy management.
Preliminary Results
SHARK included 30 participants (7 men; average age, 16 years), of whom 12 had a history of SARS-CoV-2 infection. TIGER involved 100 participants (24 men; average age, 16.7 years), of whom 32 had a SARS-CoV-2 infection as a triggering event. Other triggers included Epstein-Barr virus and other infections.
Preliminary findings from the projects indicate that optimized management with outpatient and follow-up care can yield positive, sometimes lasting effects. No significant differences between SARS-CoV-2 and other triggers emerged, but pain proved more manageable in the SHARK group than in the TIGER group, suggesting they may involve different pathological mechanisms.
Hope for Improved Outcomes
“It’s important to move away from the idea that nothing can be done,” Behrends said. This is a common attitude with children and adolescents displaying these types of symptoms, but it’s simply not true. “Even in pediatrics, we have numerous therapeutic options that may offer relief, from medication to psychosocial interventions,” she concluded.
This story was translated from Medscape’s German edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Current management of children and adolescents with long COVID was the focus of various presentations at the 3rd Long COVID Congress in Berlin in November 2024. The congress aimed to facilitate in-depth discussions on recent research projects, diagnostic procedures, and therapeutic approaches to enhance care for long COVID patients. This year, the focus was on research into long COVID in children and adolescents and how to improve their care.
Uta Behrends, MD, head of the Munich Chronic Fatigue Center, Center for Pediatric and Adolescent Medicine at the Technical University of Munich, Germany, and Nicole Toepfner, MD, a pediatrician at the University Hospital in Dresden, Germany, provided an initial overview.
Prevalence Data Are Limited
Data on the incidence and prevalence of the condition in children and adolescents are limited because most studies have primarily examined adults. A 2022 Swiss study estimated that it affects between 2% and 3.5% of children and adolescents who contract COVID-19. A recent study published in JAMA involving 5367 children and adolescents found that 20% of children aged 6-11 years and 14% of adolescents met the researchers’ criteria for long COVID.
Impaired Mental Health
Initial data from the latest wave of the population-based longitudinal COPSY (Corona and Psyche) study showed that compared with their peer group children and adolescents diagnosed with long COVID exhibit significantly higher rates of psychological issues and depressive symptoms. Although no significant differences were found in anxiety levels, study leader Ulrike Ravens-Sieberer, PhD, from the University Medical Center Hamburg-Eppendorf, Germany, told the congress that those with long COVID do also report more frequent somatic or psychological health complaints and lower health-related quality of life than peers.
Addressing Data Gaps
Another study due to launch in January 2025 and run through to 2028 is the COVYOUTH data study, which aims to better understand the nature, frequency, and risk factors of COVID-related sequelae in children and adolescents.
Study centers include Ruhr University Bochum, University Hospital Cologne, the Paul-Ehrlich-Institut, and University Medical Center Hamburg-Eppendorf. Using routine data from statutory health insurance and newly developed case definitions, researchers aim to investigate:
- Psychological stress caused by COVID-19 measures
- Post-COVID syndrome and myocarditis
- Adverse effects of COVID-19 vaccinations
Specialized Diagnostics and Care
The Post-COVID Kids Bavaria project offers specialized diagnostics and care for children and adolescents, including a day clinic, telemedical follow-ups, and an inpatient pain therapy module providing age-appropriate care as close to patients’ homes as possible.
MOVE-COVID is a model project for patient-focused research on long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) involving university pediatric hospitals in Freiburg, Heidelberg, Tübingen, and Ulm. It also aims to establish a care network across the state of Baden-Württemberg, including the establishment of long COVID outpatient clinics at social pediatric centers in the network hospitals, as well as enhanced telemedical support and standardized diagnostic and treatment protocols. “MOVE-COVID has successfully consolidated competencies and capacities in patient care, health services research, and patient-focused studies across multiple centers,” Behrends said.
Chronic Pain and Fatigue
Post-COVID syndromes in children and adolescents may feature profound fatigue, unrefreshing sleep, post-exertional malaise, cognitive dysfunction, and orthostatic intolerance and overlap with conditions such as ME/CFS. According to the German patient association Fatigatio, Berlin, research and studies for these conditions in children remain limited compared with those in adults. However, the US Centers for Disease Control estimates that around 2% of ME/CFS patients are children or adolescents, with the majority being teenagers.
Two inpatient treatment concepts, SHARK and TIGER, developed by Lea Höfel, PhD, head of the Centre for Pain Therapy for Young People and the Psychological Service at the Children’s Hospital in Garmisch-Partenkirchen, address chronic pain, fatigue, and ME/CFS in young people. These programs integrate structured breaks and flexible access to multiple therapists as needed. The TIGER program focuses on those with post-exertional malaise, while the SHARK program is designed for adolescents without this symptom. Both programs last 4.5-5 weeks and emphasize symptom reduction, education, and energy management.
Preliminary Results
SHARK included 30 participants (7 men; average age, 16 years), of whom 12 had a history of SARS-CoV-2 infection. TIGER involved 100 participants (24 men; average age, 16.7 years), of whom 32 had a SARS-CoV-2 infection as a triggering event. Other triggers included Epstein-Barr virus and other infections.
Preliminary findings from the projects indicate that optimized management with outpatient and follow-up care can yield positive, sometimes lasting effects. No significant differences between SARS-CoV-2 and other triggers emerged, but pain proved more manageable in the SHARK group than in the TIGER group, suggesting they may involve different pathological mechanisms.
Hope for Improved Outcomes
“It’s important to move away from the idea that nothing can be done,” Behrends said. This is a common attitude with children and adolescents displaying these types of symptoms, but it’s simply not true. “Even in pediatrics, we have numerous therapeutic options that may offer relief, from medication to psychosocial interventions,” she concluded.
This story was translated from Medscape’s German edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
Current management of children and adolescents with long COVID was the focus of various presentations at the 3rd Long COVID Congress in Berlin in November 2024. The congress aimed to facilitate in-depth discussions on recent research projects, diagnostic procedures, and therapeutic approaches to enhance care for long COVID patients. This year, the focus was on research into long COVID in children and adolescents and how to improve their care.
Uta Behrends, MD, head of the Munich Chronic Fatigue Center, Center for Pediatric and Adolescent Medicine at the Technical University of Munich, Germany, and Nicole Toepfner, MD, a pediatrician at the University Hospital in Dresden, Germany, provided an initial overview.
Prevalence Data Are Limited
Data on the incidence and prevalence of the condition in children and adolescents are limited because most studies have primarily examined adults. A 2022 Swiss study estimated that it affects between 2% and 3.5% of children and adolescents who contract COVID-19. A recent study published in JAMA involving 5367 children and adolescents found that 20% of children aged 6-11 years and 14% of adolescents met the researchers’ criteria for long COVID.
Impaired Mental Health
Initial data from the latest wave of the population-based longitudinal COPSY (Corona and Psyche) study showed that compared with their peer group children and adolescents diagnosed with long COVID exhibit significantly higher rates of psychological issues and depressive symptoms. Although no significant differences were found in anxiety levels, study leader Ulrike Ravens-Sieberer, PhD, from the University Medical Center Hamburg-Eppendorf, Germany, told the congress that those with long COVID do also report more frequent somatic or psychological health complaints and lower health-related quality of life than peers.
Addressing Data Gaps
Another study due to launch in January 2025 and run through to 2028 is the COVYOUTH data study, which aims to better understand the nature, frequency, and risk factors of COVID-related sequelae in children and adolescents.
Study centers include Ruhr University Bochum, University Hospital Cologne, the Paul-Ehrlich-Institut, and University Medical Center Hamburg-Eppendorf. Using routine data from statutory health insurance and newly developed case definitions, researchers aim to investigate:
- Psychological stress caused by COVID-19 measures
- Post-COVID syndrome and myocarditis
- Adverse effects of COVID-19 vaccinations
Specialized Diagnostics and Care
The Post-COVID Kids Bavaria project offers specialized diagnostics and care for children and adolescents, including a day clinic, telemedical follow-ups, and an inpatient pain therapy module providing age-appropriate care as close to patients’ homes as possible.
MOVE-COVID is a model project for patient-focused research on long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) involving university pediatric hospitals in Freiburg, Heidelberg, Tübingen, and Ulm. It also aims to establish a care network across the state of Baden-Württemberg, including the establishment of long COVID outpatient clinics at social pediatric centers in the network hospitals, as well as enhanced telemedical support and standardized diagnostic and treatment protocols. “MOVE-COVID has successfully consolidated competencies and capacities in patient care, health services research, and patient-focused studies across multiple centers,” Behrends said.
Chronic Pain and Fatigue
Post-COVID syndromes in children and adolescents may feature profound fatigue, unrefreshing sleep, post-exertional malaise, cognitive dysfunction, and orthostatic intolerance and overlap with conditions such as ME/CFS. According to the German patient association Fatigatio, Berlin, research and studies for these conditions in children remain limited compared with those in adults. However, the US Centers for Disease Control estimates that around 2% of ME/CFS patients are children or adolescents, with the majority being teenagers.
Two inpatient treatment concepts, SHARK and TIGER, developed by Lea Höfel, PhD, head of the Centre for Pain Therapy for Young People and the Psychological Service at the Children’s Hospital in Garmisch-Partenkirchen, address chronic pain, fatigue, and ME/CFS in young people. These programs integrate structured breaks and flexible access to multiple therapists as needed. The TIGER program focuses on those with post-exertional malaise, while the SHARK program is designed for adolescents without this symptom. Both programs last 4.5-5 weeks and emphasize symptom reduction, education, and energy management.
Preliminary Results
SHARK included 30 participants (7 men; average age, 16 years), of whom 12 had a history of SARS-CoV-2 infection. TIGER involved 100 participants (24 men; average age, 16.7 years), of whom 32 had a SARS-CoV-2 infection as a triggering event. Other triggers included Epstein-Barr virus and other infections.
Preliminary findings from the projects indicate that optimized management with outpatient and follow-up care can yield positive, sometimes lasting effects. No significant differences between SARS-CoV-2 and other triggers emerged, but pain proved more manageable in the SHARK group than in the TIGER group, suggesting they may involve different pathological mechanisms.
Hope for Improved Outcomes
“It’s important to move away from the idea that nothing can be done,” Behrends said. This is a common attitude with children and adolescents displaying these types of symptoms, but it’s simply not true. “Even in pediatrics, we have numerous therapeutic options that may offer relief, from medication to psychosocial interventions,” she concluded.
This story was translated from Medscape’s German edition using several editorial tools, including artificial intelligence, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.
FROM THE 3RD LONG COVID CONGRESS
CDK 4/6 Blocker Prolongs Survival in HER2+ Metastatic Breast Cancer
according to the results of the phase 3 PATINA study.
This regimen “may represent a new standard of care” for these patients, said principal investigator and presenter Otto Metzger, MD, a medical breast oncologist at the Dana-Farber Cancer Institute in Boston, Massachusetts, who presented the findings at the San Antonio Breast Cancer Symposium (SABCS) 2024.
The open-label PATINA trial, which was conducted in Europe, Australia, New Zealand, and the United States, included a total of 518 patients. Patients received first-line treatment of six to eight cycles of induction chemotherapy plus anti-HER2 therapy. Researchers then randomized patients to either palbociclib plus anti-HER2 and endocrine therapy (n = 261) or to anti-HER2 and endocrine therapy alone (n = 257).
Patients did not progress on induction therapy, which likely would have signaled early resistance to anti-HER2 treatment. For anti-HER2 therapy, 97.3% received a combination of trastuzumab and pertuzumab. For endocrine therapy, 90.9% received an aromatase inhibitor.
Metzger and colleagues found that median progression-free survival was 1.3 years longer in patients receiving palbociclib — 3.7 years in the palbociclib arm vs 2.4 years in the control group (hazard ratio [HR], 0.74; P = .0074).
Although overall survival outcomes are immature, 5-year survival rates were slightly better in the palbociclib arm — 74.3% with palbociclib vs 69.8% without it — but the difference was not statistically significant.
Grade 3 neutropenia was the most frequent adverse event in the palbociclib arm (63.2% vs 2%). Grades 2 and 3 fatigue, stomatitis, and diarrhea were also more common with palbociclib. Grade 4 adverse events occurred in 12.3% of those receiving palbociclib and 8.9% of those who did not. There were no treatment-related deaths.
“We’re very impressed with the results,” said Metzger.
On the basis of previous studies, it’s believed that CDK 4/6 inhibition counteracts the development of resistance to anti-HER2 and endocrine therapies, which likely explains the benefit found in the trial.
But even without CDK 4/6 inhibition, the progression-free survival of 2.4 years in the control arm “far exceed[ed] our expectations,” Metzger reported. This may have occurred because the control arm received endocrine therapy, something previous trials of anti-HER2 therapy have avoided because of tolerability and other concerns.
These findings, however, support “the common use of endocrine therapy,” Metzger said.
‘Incredible’ Results
The progression-free survival as well as overall survival results in the trial are “incredible,” said study discussant Sara Hurvitz, MD, a medical breast oncologist at the Fred Hutch Cancer Center in Seattle, Washington. This is “historic and very important data.”
Hurvitz even suggested the results might mean that patients who fit the PATINA criteria can avoid the toxicity of upfront trastuzumab deruxtecan and use the PATINA regimen instead, potentially preserving their quality of life for longer.
Another study discussant, Virginia Kaklamani, MD, a medical breast oncologist at the University of Texas MD Anderson Cancer Center, San Antonio, had a similar thought.
In PATINA, “we’re talking about patients being on a treatment that’s well tolerated, where patients continue to work and continue with their lives despite being on treatment for metastatic breast cancer for 4 years, which is remarkable,” Kaklamani said.
Many of us have dabbled with giving CDK 4/6 inhibitors in triple-positive breast cancer, but “now we have more definitive data,” she said. The approach can help “maintain the quality of life of our patients for a longer period of time” and delay the use of chemotherapy in the second line, she added.
Metzger said Pfizer, the maker of palbociclib, plans to file for a HER2-positive indication with the Food and Drug Administration based on the trial results.
For now, the CDK 4/6 blocker is only indicated in combination with endocrine therapy for HR-positive, HER2-negative metastatic disease.
In response to a question about using the PATINA regimen in patients who don’t get chemotherapy induction, Metzger noted that, “while the study didn’t test this directly, I would argue that this data is quite compelling” for using palbociclib plus anti-HER2 and endocrine therapy, even without chemotherapy induction.
The work was funded by palbociclib maker Pfizer. Metzger had no disclosures. Hurvitz has numerous industry ties, including being a researcher and advisor to Pfizer. Kaklamani also has numerous industry ties, including reporting personal/consulting fees from Pfizer Canada.
A version of this article first appeared on Medscape.com.
according to the results of the phase 3 PATINA study.
This regimen “may represent a new standard of care” for these patients, said principal investigator and presenter Otto Metzger, MD, a medical breast oncologist at the Dana-Farber Cancer Institute in Boston, Massachusetts, who presented the findings at the San Antonio Breast Cancer Symposium (SABCS) 2024.
The open-label PATINA trial, which was conducted in Europe, Australia, New Zealand, and the United States, included a total of 518 patients. Patients received first-line treatment of six to eight cycles of induction chemotherapy plus anti-HER2 therapy. Researchers then randomized patients to either palbociclib plus anti-HER2 and endocrine therapy (n = 261) or to anti-HER2 and endocrine therapy alone (n = 257).
Patients did not progress on induction therapy, which likely would have signaled early resistance to anti-HER2 treatment. For anti-HER2 therapy, 97.3% received a combination of trastuzumab and pertuzumab. For endocrine therapy, 90.9% received an aromatase inhibitor.
Metzger and colleagues found that median progression-free survival was 1.3 years longer in patients receiving palbociclib — 3.7 years in the palbociclib arm vs 2.4 years in the control group (hazard ratio [HR], 0.74; P = .0074).
Although overall survival outcomes are immature, 5-year survival rates were slightly better in the palbociclib arm — 74.3% with palbociclib vs 69.8% without it — but the difference was not statistically significant.
Grade 3 neutropenia was the most frequent adverse event in the palbociclib arm (63.2% vs 2%). Grades 2 and 3 fatigue, stomatitis, and diarrhea were also more common with palbociclib. Grade 4 adverse events occurred in 12.3% of those receiving palbociclib and 8.9% of those who did not. There were no treatment-related deaths.
“We’re very impressed with the results,” said Metzger.
On the basis of previous studies, it’s believed that CDK 4/6 inhibition counteracts the development of resistance to anti-HER2 and endocrine therapies, which likely explains the benefit found in the trial.
But even without CDK 4/6 inhibition, the progression-free survival of 2.4 years in the control arm “far exceed[ed] our expectations,” Metzger reported. This may have occurred because the control arm received endocrine therapy, something previous trials of anti-HER2 therapy have avoided because of tolerability and other concerns.
These findings, however, support “the common use of endocrine therapy,” Metzger said.
‘Incredible’ Results
The progression-free survival as well as overall survival results in the trial are “incredible,” said study discussant Sara Hurvitz, MD, a medical breast oncologist at the Fred Hutch Cancer Center in Seattle, Washington. This is “historic and very important data.”
Hurvitz even suggested the results might mean that patients who fit the PATINA criteria can avoid the toxicity of upfront trastuzumab deruxtecan and use the PATINA regimen instead, potentially preserving their quality of life for longer.
Another study discussant, Virginia Kaklamani, MD, a medical breast oncologist at the University of Texas MD Anderson Cancer Center, San Antonio, had a similar thought.
In PATINA, “we’re talking about patients being on a treatment that’s well tolerated, where patients continue to work and continue with their lives despite being on treatment for metastatic breast cancer for 4 years, which is remarkable,” Kaklamani said.
Many of us have dabbled with giving CDK 4/6 inhibitors in triple-positive breast cancer, but “now we have more definitive data,” she said. The approach can help “maintain the quality of life of our patients for a longer period of time” and delay the use of chemotherapy in the second line, she added.
Metzger said Pfizer, the maker of palbociclib, plans to file for a HER2-positive indication with the Food and Drug Administration based on the trial results.
For now, the CDK 4/6 blocker is only indicated in combination with endocrine therapy for HR-positive, HER2-negative metastatic disease.
In response to a question about using the PATINA regimen in patients who don’t get chemotherapy induction, Metzger noted that, “while the study didn’t test this directly, I would argue that this data is quite compelling” for using palbociclib plus anti-HER2 and endocrine therapy, even without chemotherapy induction.
The work was funded by palbociclib maker Pfizer. Metzger had no disclosures. Hurvitz has numerous industry ties, including being a researcher and advisor to Pfizer. Kaklamani also has numerous industry ties, including reporting personal/consulting fees from Pfizer Canada.
A version of this article first appeared on Medscape.com.
according to the results of the phase 3 PATINA study.
This regimen “may represent a new standard of care” for these patients, said principal investigator and presenter Otto Metzger, MD, a medical breast oncologist at the Dana-Farber Cancer Institute in Boston, Massachusetts, who presented the findings at the San Antonio Breast Cancer Symposium (SABCS) 2024.
The open-label PATINA trial, which was conducted in Europe, Australia, New Zealand, and the United States, included a total of 518 patients. Patients received first-line treatment of six to eight cycles of induction chemotherapy plus anti-HER2 therapy. Researchers then randomized patients to either palbociclib plus anti-HER2 and endocrine therapy (n = 261) or to anti-HER2 and endocrine therapy alone (n = 257).
Patients did not progress on induction therapy, which likely would have signaled early resistance to anti-HER2 treatment. For anti-HER2 therapy, 97.3% received a combination of trastuzumab and pertuzumab. For endocrine therapy, 90.9% received an aromatase inhibitor.
Metzger and colleagues found that median progression-free survival was 1.3 years longer in patients receiving palbociclib — 3.7 years in the palbociclib arm vs 2.4 years in the control group (hazard ratio [HR], 0.74; P = .0074).
Although overall survival outcomes are immature, 5-year survival rates were slightly better in the palbociclib arm — 74.3% with palbociclib vs 69.8% without it — but the difference was not statistically significant.
Grade 3 neutropenia was the most frequent adverse event in the palbociclib arm (63.2% vs 2%). Grades 2 and 3 fatigue, stomatitis, and diarrhea were also more common with palbociclib. Grade 4 adverse events occurred in 12.3% of those receiving palbociclib and 8.9% of those who did not. There were no treatment-related deaths.
“We’re very impressed with the results,” said Metzger.
On the basis of previous studies, it’s believed that CDK 4/6 inhibition counteracts the development of resistance to anti-HER2 and endocrine therapies, which likely explains the benefit found in the trial.
But even without CDK 4/6 inhibition, the progression-free survival of 2.4 years in the control arm “far exceed[ed] our expectations,” Metzger reported. This may have occurred because the control arm received endocrine therapy, something previous trials of anti-HER2 therapy have avoided because of tolerability and other concerns.
These findings, however, support “the common use of endocrine therapy,” Metzger said.
‘Incredible’ Results
The progression-free survival as well as overall survival results in the trial are “incredible,” said study discussant Sara Hurvitz, MD, a medical breast oncologist at the Fred Hutch Cancer Center in Seattle, Washington. This is “historic and very important data.”
Hurvitz even suggested the results might mean that patients who fit the PATINA criteria can avoid the toxicity of upfront trastuzumab deruxtecan and use the PATINA regimen instead, potentially preserving their quality of life for longer.
Another study discussant, Virginia Kaklamani, MD, a medical breast oncologist at the University of Texas MD Anderson Cancer Center, San Antonio, had a similar thought.
In PATINA, “we’re talking about patients being on a treatment that’s well tolerated, where patients continue to work and continue with their lives despite being on treatment for metastatic breast cancer for 4 years, which is remarkable,” Kaklamani said.
Many of us have dabbled with giving CDK 4/6 inhibitors in triple-positive breast cancer, but “now we have more definitive data,” she said. The approach can help “maintain the quality of life of our patients for a longer period of time” and delay the use of chemotherapy in the second line, she added.
Metzger said Pfizer, the maker of palbociclib, plans to file for a HER2-positive indication with the Food and Drug Administration based on the trial results.
For now, the CDK 4/6 blocker is only indicated in combination with endocrine therapy for HR-positive, HER2-negative metastatic disease.
In response to a question about using the PATINA regimen in patients who don’t get chemotherapy induction, Metzger noted that, “while the study didn’t test this directly, I would argue that this data is quite compelling” for using palbociclib plus anti-HER2 and endocrine therapy, even without chemotherapy induction.
The work was funded by palbociclib maker Pfizer. Metzger had no disclosures. Hurvitz has numerous industry ties, including being a researcher and advisor to Pfizer. Kaklamani also has numerous industry ties, including reporting personal/consulting fees from Pfizer Canada.
A version of this article first appeared on Medscape.com.
FROM SABCS 2024