Pure red cell aplasia (PRCA) is a relevant complication after ABO-mismatched allogeneic hematopoietic cell transplantation (HCT). No standard treatment exists, and practice is heterogenous. In this study, we took advantage of an international collaboration to describe characteristics and outcomes of patients receiving daratumumab for PRCA following first allogeneic HCT. We identified 45 patients meeting these criteria (median patient age, 56 years). The median time from HCT to PRCA was 55 days (IQR, 36–116) and all patients were transfusion-dependent at time of daratumumab start. Daratumumab was first-line treatment in 16 patients (36%), most patients (67%) received daratumumab intravenously, and median time from PRCA diagnosis and daratumumab start was 88 days (IQR, 59–219). Incidence of transfusion independence was 69% (95% confidence interval [CI], 52–80%) at 6 months and 80% (95% CI, 62–90%) at 12 months. Incidences of hemoglobin and reticulocyte recoveries were respectively 56 and 78% at 6 months and 65 and 83% at 12 months. Survival at 12 months was 81%, and of 8 deaths, 7 were GVHD- or infection-related. One death was associated with hemolytic anemia. This is the first international and largest study on the use of daratumumab for PRCA after allogeneic HCT, showing high response rates superior to that reported for other treatments. Seven incidents of severe adverse events (mostly infections) underscore the need for close monitoring, proactive management, and comparative studies to determine the role for daratumumab for PRCA. Last, based on these data and a comprehensive literature review, we provide practical consideration for modern PRCA treatment.
纯红细胞再生障碍（PRCA）是ABO血型不合异基因造血细胞移植（HCT）后的一种相关并发症。目前尚无标准治疗方案，临床实践存在差异。本研究通过国际合作，描述了首次异基因HCT后因PRCA接受达雷妥尤单抗治疗患者的特征与结局。共纳入45例符合标准的患者（中位年龄56岁）。从HCT至发生PRCA的中位时间为55天（IQR，36-116），所有患者在开始达雷妥尤单抗治疗时均处于输血依赖状态。其中16例患者（36%）将达雷妥尤单抗作为一线治疗，多数患者（67%）接受静脉给药，从PRCA诊断至开始达雷妥尤单抗治疗的中位时间为88天（IQR，59-219）。6个月和12个月时脱离输血依赖的发生率分别为69%（95% CI，52-80%）和80%（95% CI，62-90%）。血红蛋白恢复与网织红细胞恢复的发生率在6个月时分别为56%和78%，12个月时分别为65%和83%。12个月总生存率为81%，8例死亡患者中7例与移植物抗宿主病（GVHD）或感染相关，1例与溶血性贫血相关。本研究是首个关于异基因HCT后使用达雷妥尤单抗治疗PRCA的国际性最大规模研究，结果显示其疗效优于既往报道的其他治疗方案，且缓解率较高。7例严重不良事件（主要为感染）的发生提示需加强监测、积极管理并进行对比研究，以明确达雷妥尤单抗在PRCA治疗中的作用。最后，基于本研究数据及全面的文献综述，我们为现代PRCA治疗提供了实践参考。
肿瘤(癌症)患者之家