01/12/2022 The CDCN also collaborated on a study that was presented at the world’s largest cancer meeting (ASCO) this year. https://ascopubs.org/doi/abs/10.1200/JCO.2021.39.15_suppl.7048
The team, led by first author, Dr Aaron Cohen and senior author, Dr. David Fajgenbaum, studied idiopathic multicentric CD (iMCD) patients and described their clinical characteristics, treatment patterns, and real-world overall survival. As a background, previous studies have found that outcomes for patients with iMCD are poor and treatment options are limited, with only one FDA-approved therapy (siltuximab in April 2014).(rwOS). 747 patients with possible CD were identified, of whom 453 were confirmed to have CD by expert reviewers (172 UCD, 100 iMCD, 36 HHV-8 MCD, and 145 unclassified). IMCD patients were predominantly female (53%), white (58%), and treated at community sites (70%). The most common first-line therapies were siltuximab-based therapy (42.3%), rituximab monotherapy (36.5%), and chemotherapy-based treatment (13.5%). Among 60 iMCD patients diagnosed on or after siltuximab approval in April 2014 (including those without evidence of any treatment), 26 (43%) received siltuximab at some point. 5-year overall survival rate for the 100 iMCD patients was 75% [95% CI: 63-89%], which means that the exact 5 year survival across the entire US population of iMCD patients is likely somewhere between 63% and 89%.
Conclusions: This is the first study to utilize a large EHR-derived database to describe characteristics, treatment patterns, and overall survival of iMCD patients in real-world practice. Less than half of iMCD patients diagnosed on or after the date of FDA approval for siltuximab received it at some point. Future work led by the CDCN and collaborators will focus on characterizing the drivers of poor patient outcomes.