异基因造血干细胞移植治疗≤50岁高危多发性骨髓瘤患者14例临床分析

Clinical analysis of 14 patients aged≤50 years with high-risk multiple myeloma treated with allogeneic hematopoietic stem cell transplantation

目的观察异基因造血干细胞移植(allo-HSCT)对年轻(≤50岁)高危多发性骨髓瘤(HRMM)患者的疗效。方法纳入2016年11月至2022年11月期间于中国医学科学院血液病医院造血干细胞移植中心接受allo-HSCT的14例具有高危细胞遗传学改变或高危疾病生物学因素的年轻(≤50岁)HRMM患者,对其临床资料进行回顾性分析。结果14例患者中,男7例,女7例,移植时中位年龄为39.5(31~50)岁。患者移植前中位治疗线数为2(1~6)线,移植前6例患者未达完全缓解(CR),5例患者微小残留病(MRD)阳性。14例患者均获得造血重建,中性粒细胞、血小板中位植入时间分别为11(10~14)d、13(9~103)d。5例患者发生Ⅱ~Ⅳ度急性移植物抗宿主病(GVHD),2例患者发生中重度慢性GVHD。移植后3个月疗效评估,所有患者均为严格意义的完全缓解(sCR)。移植后中位随访时间为18.9(4.1~72.5)个月,移植后2年移植相关死亡率为7.1%(95%CI 0%~21.1%),总生存率为92.9%(95%CI 80.3%~100.0%);移植后1、2年无进展生存率分别为92.9%(95%CI 80.3%~100.0%)、66.0%(95%CI 39.4%~100.0%),2年累积复发率为28.9%(95%CI 0%~56.7%)。结论年轻HRMM患者在诱导治疗后桥接allo-HSCT可进一步提高疗效。

Objective To evaluate the efficacy of allogeneic hematopoietic stem cell transplantation(allo-HSCT)in young patients with high-risk multiple myeloma(HRMM)and analyzed the factors affecting patient prognosis.Methods In this retrospective study,we analyzed the clinical data of 14 patients with HRMM with cytogenetic abnormalities or high-risk biological factors who underwent allo-HSCT at the Hematopoietic Stem Cell Transplantation Center of the Institute of Hematology&Blood Diseases Hospital between November 2016 and November 2022.Results There were seven males and seven females included in the study,with a median age of 39.5(31-50)years at the time of allo-HSCT.The median number of treatment lines before transplantation was 2(1-6).Before allo-HSCT,42.9%(6/14)of the patients did not achieve complete remission,while 35.7%(5/14)of the patients achieved measurable residual disease positivity.After transplantation,all patients were evaluated for their treatment response,and the overall response rate was 100%(14/14).All 14 patients successfully underwent allo-HSCT,with median engraftment times for neutrophils and platelets of 11(10-14)days and 13(9-103)days,respectively.Acute gradeⅡ-Ⅳgraft-versus-host disease(GVHD)occurred in five patients(35.7%),and two patients(14.3%)developed moderate-to-severe chronic GVHD.The median follow-up time after allo-HSCT was 18.93(4.10-72.53)months,with an expected 2-year transplant-related mortality rate of 7.1%(95%CI 0%-21.1%)and an expected 2-year overall survival rate of 92.9%(95%CI 80.3%–100.0%).Moreover,the expected 1-year and 2-year progression-free survival rates were 92.9%(95%CI 80.3%-100.0%)and 66.0%(95%CI 39.4%-100.0%),respectively,and the 2-year cumulative incidence of relapse was 28.9%(95%CI 0%-56.7%).Upfront allo-HSCT following complete remission after induced therapy and the presence of chronic GVHD might be favorable prognostic factors.Conclusion allo-HSCT is an effective treatment for improving the prognosis of young patients with HRMM.