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达雷妥尤单抗用于异基因造血干细胞移植后纯红细胞再生障碍的管理

Daratumumab in the Management of Red Cell Aplasia Following Allogeneic Hematopoietic Stem Cell Transplantation.

作者信息

Desai Nihar, Viswabandya Auro, Kim Dennis Dong Hwan, Lipton Jeffrey H, Mattsson Jonas, Law Arjun Datt

机构信息

Hans Messner Allogeneic Blood and Marrow Transplant Program, Division of Medical Oncology and Hematology, Princess Margaret Hospital, Toronto, Canada.

Temerty faculty of Medicine, University of Toronto, Toronto, Canada.

出版信息

Eur J Haematol. 2025 Feb;114(2):310-314. doi: 10.1111/ejh.14341. Epub 2024 Nov 4.

DOI:10.1111/ejh.14341
PMID:39494777
Abstract

Pure red cell aplasia (PRCA) is a rare but significant complication following major ABO-incompatible allogeneic hematopoietic stem cell transplantation (HSCT). The persistence of recipient B lymphocytes producing anti-donor isohemagglutinins leads to reticulocytopenia and anemia, often resulting in transfusion dependence. Current treatment options for post-HSCT PRCA are limited and frequently yield suboptimal responses, complicating patient management. Herein, we report three cases of post-HSCT PRCA successfully managed with daratumumab, a monoclonal antibody targeting CD38-expressing plasma cells. All patients demonstrated rapid reticulocyte recovery and transfusion independence after daratumumab treatment, despite prior treatment failures. These findings suggest that daratumumab may provide a more effective therapeutic approach, with a favorable safety profile compared to traditional therapies. Given its demonstrated efficacy and safety, daratumumab warrants consideration as a first-line treatment for post-HSCT PRCA, potentially improving patient quality of life and reducing transfusion-related complications. Further studies should explore optimal dosing and long-term outcomes.

摘要

纯红细胞再生障碍性贫血(PRCA)是主要ABO血型不合的异基因造血干细胞移植(HSCT)后一种罕见但严重的并发症。产生抗供体同种血凝素的受者B淋巴细胞持续存在会导致网织红细胞减少和贫血,常导致输血依赖。HSCT后PRCA的当前治疗选择有限,且常常产生次优反应,使患者管理复杂化。在此,我们报告3例HSCT后PRCA患者,使用靶向表达CD38的浆细胞的单克隆抗体达雷妥尤单抗成功治疗。尽管先前治疗失败,但所有患者在达雷妥尤单抗治疗后均表现出网织红细胞快速恢复且不再依赖输血。这些发现表明,与传统疗法相比,达雷妥尤单抗可能提供一种更有效的治疗方法,且安全性良好。鉴于其已证实的疗效和安全性,达雷妥尤单抗值得考虑作为HSCT后PRCA的一线治疗药物,可能改善患者生活质量并减少输血相关并发症。进一步的研究应探索最佳剂量和长期疗效。

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