Gagelmann Nico, Witte Moniek de, Peczynski Christophe, Boreland William, Broers Annoek E C, Jost Edgar, Kulagin Alexander, Esquirol Albert, Sica Simona, Kuball Jürgen, Errico Gerardo, Bethge Wolfgang, Maertens Johan, Stölzel Friedrich, Forcade Edouard, Collin Matthew, Parma Matteo, Choi Goda, Kröger Nicolaus, Di Chio Maria Chiara, Finazzi Maria Chiara, López Corral Lucia, Rifón Jose, Mussetti Alberto, Bloor Adrian, Ladetto Marco, Schoemans Hélène, Penack Olaf, Moiseev Ivan, Peric Zinaida
University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Hematology, University Medical Center Utrecht, Utrecht, The Netherlands.
Blood Cancer J. 2025 Jun 4;15(1):106. doi: 10.1038/s41408-025-01315-8.
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.
纯红细胞再生障碍性贫血（PRCA）是ABO血型不匹配的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法，临床实践也存在差异。在本研究中，我们利用国际合作来描述首次异基因HCT后接受达雷妥尤单抗治疗PRCA的患者的特征和结局。我们确定了45例符合这些标准的患者（中位患者年龄56岁）。从HCT到PRCA的中位时间为55天（四分位间距，36 - 116天），所有患者在开始使用达雷妥尤单抗时均依赖输血。16例患者（36%）将达雷妥尤单抗作为一线治疗，大多数患者（67%）接受静脉注射达雷妥尤单抗，从PRCA诊断到开始使用达雷妥尤单抗的中位时间为88天（四分位间距，59 - 219天）。6个月时输血独立的发生率为69%（95%置信区间[CI]，52 - 80%），12个月时为80%（95%CI，62 - 90%）。6个月时血红蛋白和网织红细胞恢复的发生率分别为56%和78%，12个月时分别为65%和83%。12个月时的生存率为81%，在8例死亡病例中，7例与移植物抗宿主病（GVHD）或感染相关。1例死亡与溶血性贫血有关。这是关于异基因HCT后使用达雷妥尤单抗治疗PRCA的第一项国际且规模最大的研究，显示出高于其他治疗方法报道的高缓解率。7例严重不良事件（大多为感染）突出表明需要密切监测、积极管理以及进行比较研究以确定达雷妥尤单抗在PRCA治疗中的作用。最后，基于这些数据和全面的文献综述，我们为现代PRCA治疗提供了实际的考虑因素。
