ORIGINAL RESEARCH article
Front. Immunol.
Sec. Alloimmunity and Transplantation
Volume 16 - 2025 | doi: 10.3389/fimmu.2025.1622326
This article is part of the Research TopicBone Marrow Failure Syndromes: From Biology to Cure - Volume IIView all articles
Unveiling new horizons in severe aplastic anemia management: A two-decade study on intensive immunosuppressive therapy combined with unrelated cord blood efficacy
Provisionally accepted
- 960th Hospital of the PLA, Jinan, China
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Background: In the absence of a human leukocyte antigen (HLA)-matched donor for severe aplastic anemia (SAA) in patients who had failed first-line immunosuppressive therapy (IST), intensive IST combined with unrelated cord blood (IIST-UCB) is a salvage treatment option. With improvements in transplantation technology, the treatment outcome of IIST-UCB has improved considerably in recent years. Here, we will focus on the differential effects of IIST-UCB on patients' survival and the risk of GVHD, and evaluate the therapeutic efficacy between the SAA and VSAA patients.Methods: Between August 2004 and May 2024, 115 SAA patients were screened at enrollment. The overall survival (OS) rates and failure-free survival (FFS) rates were evaluated and compared using Kaplan-Meier curves and log-rank tests. Cumulative incidences of cytomegalovirus (CMV), hematopoietic recovery, and Epstein-Barr virus (EBV) were estimated by a competing risk regression model.The median age was 16 years (range, 2-74). In 6 months, 27 patients (27%) were in complete response (CR) and 44 patients (44%) were in partial response (PR). The median period for neutrophil engraftment was 25 days and platelet engraftment was 44 days. The 250-day cumulative incidences of hemoglobin recoveries were 87.8% (95% CI, 77.7%-93.6%). The 100-day cumulative incidences of neutrophil engraftment were 88.5% (95%CI, 80.6%-93.3%). The 400-day cumulative incidences of platelet engraftment were 86.7% (95%CI, 77.5%-92.4%). The 5-year overall survival was 86.1% ± 6.66% and the 5-year failure-free survival was 72% ± 8.62% in the whole cohort. Transplantation-related mortality was 12.5% (95% CI, 7.2%-19.4%). No acute graft-versus-host disease (GVHD) and chronic GVHD were observed over a whole period. The cumulative incidences of CMV and EBV were 7.18% 3 (95%CI, 3.34%-13%) and 16.8 (95%CI, 10.6%-24.3%), respectively. The majority of patients were at microchimerism status and maintained hematopoiesis for a long term. SAA group patients who received UCB treatment showed a significantly higher hematopoietic reconstitution efficiency (P = 0.004, P = 0.001, P = 0.001) and OS rate compared with the VSAA group (P = 0.028).These data support IIST-UCB as an alternative therapeutic approach for SAA patients.
Keywords: unrelated cord blood, Severe aplastic anemia, immunosuppressive therapy, sAA, Chimerism
Received: 03 May 2025; Accepted: 30 Jun 2025.
Copyright: © 2025 Zhou, Li, Yu, Song, Li, Deng, Kong, Wang and Ni. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Fang Zhou, 960th Hospital of the PLA, Jinan, China
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