1. Lenalidomide and dexamethasone for Rosai-Dorfman disease: a single arm, single center, prospective phase 2 studyResearch in context
- Author
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Long Chang, Min Lang, Ting Liu, He Lin, Zheng-Zheng Liu, Hao Cai, Dao-bin Zhou, and Xin-xin Cao
- Subjects
Rosai-Dorfman disease ,Treatment ,Lenalidomide ,Dexamethasone ,Medicine (General) ,R5-920 - Abstract
Summary: Background: Rosai-Dorfman disease (RDD) is a rare heterogeneous histiocytic disorder lacking standardized first-line treatment. Methods: This single-center, phase 2 prospective study enrolled 13 newly diagnosed and 10 recurrent RDD patients from June 2021 to March 2023 at Peking Union Medical College Hospital (Beijing, China). Lenalidomide 25 mg days 1–21 plus dexamethasone 40 mg days 1, 8, 15, 22 was administered in 28-day cycles, totaling 12 cycles. The primary endpoint was progression-free survival (PFS). Secondary endpoints were overall response rate (ORR) to lenalidomide and dexamethasone (RD) regimen, toxicity, and overall survival (OS) measured from RD start to death or last follow-up. OS and PFS were estimated according to Kaplan–Meier survival analysis and compared with the log-rank test. For OS and OR rate, 95% confidence limits were obtained using the Clopper-Pearson method, with standard methods used for PFS. p
- Published
- 2024
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