
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
Yang Xi, Li Chenglong, Ye Xuemei, Wang Yu, Mao Yan, Pu Wenqi, Huang Xiaobing
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. Univariate analysis showed that the extended course of RAP treatment was correlated with a trend of survival improvement(P=0.089). ③Glucocorticoid exposure:all 14 cases had a long-term history of glucocorticoid 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.
hormone-free regimen / allogeneic hematopoietic stem cell transplantation / bronchiolitis obliterans syndrome / retrospective analysis
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中华医学会血液学分会造血干细胞应用学组,中国抗癌协会血液病转化委员会. 慢性移植物抗宿主病(cGVHD)诊断与治疗中国专家共识(2021年版)[J]. 中华血液学杂志,2021,42(4):265-275.
Hematopoietic stem cell Application Group,
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