RAP hormone-free regimen in the treatment of moderate to severe bronchiolitis obliterans syndrome after allogeneic hematopoietic stem cell transplantation:a single-center retrospective analysis
Objective:To retrospectively analyze the clinical efficacy of the RAP(Ruxolitinib,Azithromycin,and Pirfenidone)hormone-free regimen in the treatment of moderate to severe BOS and its influencing factors.Methods:This study included 14 patients who developed moderate to severe BOS after undergoing allo-HSCT at our center from January 1,2018 to December 31,2023.These patients were retrospectively analyzed for the clinical efficacy of the RAP hormone-free regimen and its influencing factors.Results:①The overall response rates at 3,6 and 12 months of treatment were 50.0％,57.1％,and 50.0％,respectively.At the six-months post RAP regimen,the FEV1％predicted value(t=2.187,P=0.033),6 minute walk test(t=2.338,P=0.023),ECOG physical state score(t=2.681,P=0.013)and cGVHD severity(Z=2.155,P=0.032)of 8 patients who responded to the RAP treatment were significantly improved.②Survival:During a median follow-up of 13 months,5 patients(35.7％)died of severe pneumonia complications.Univari-ate analysis showed that the extended course of RAP treatment was correlated with a trend of survival improvement(P=0.089).③Glu-cocorticoid exposure:all 14 cases had a long-term history of gluco-corticoid use,8 cases(57.1％)discontinued glucocorticoid use within 1 month,and 2 cases reduced their dose by more than 50％.④The safety profile was favorable,and the main adverse reactions were gastrointestinal reactions and hematological toxicity.Conclusion:The RAP hormone-free regimen shows a favorable effect in the treatment of moderate to severe BOS.It allows for the rapid withdrawal of glucocorticoids and improves the quality of life and long-term survival of patients.However,it needs to be further verified by prospective studies due to its small sample size and retrospective design.
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