Yao Di, Tian Yuanyuan, Li Jie, Li Bohan, Lu Jun, Ling Jing, Zheng Defei, Yao Yanhua, Xiao Peifang, Meng Lijun, Hu Shaoyan
Department of Hematology, Children's Hospital of Soochow University, Suzhou, China.
Jiangsu Children's Hematology & Oncology Center, Suzhou, China.
Ther Adv Hematol. 2022 Oct 28;13:20406207221134409. doi: 10.1177/20406207221134409. eCollection 2022.
Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) based on granulocyte colony-stimulating factor plus anti-thymocyte regimens ('Beijing Protocol') provides a salvage treatment for patients of acquired severe aplastic anemia (SAA) in China. However, graft--host disease (GVHD) is a major impediment of haplo-HSCT due to human leukocyte antigen disparity. Recently, haplo-HSCT combined with umbilical cord blood (UCB) (haplo-cord HSCT) is performed in clinical trials to potentially reduce the risk of severe GVHD. Nevertheless, studies comparing GVHD in pediatric patients receiving haplo and haplo-cord HSCT for SAA are limited.
The objective of this study was to investigate the impact of UCB co-infusion on GVHD in pediatric patients receiving haplo-HSCT for SAA.
We conducted a retrospective study of 91 consecutive SAA children undergoing haploidentical transplantation based on the 'Beijing Protocol' with or without co-infusion of UCB in our center.
All patients received uniform non-myeloablative conditioning and GVHD prophylaxis. We compared baseline characteristics and transplant outcomes between the haplo ( = 35) and haplo-cord ( = 56) recipients.
All 91 patients achieved hematopoietic recovery from haploidentical donors, with a higher incidence of peri-engraftment syndrome observed with the haplo-cord group as compared with the haplo group (75.0% 48.6%, = 0.029). Notably, the haplo-cord group showed a lower incidence of II-IV acute GVHD (aGVHD) than the haplo group (16.1% 42.9%, = 0.002). Observed incidences of chronic GVHD (cGVHD) and moderate to severe cGVHD in the haplo-cord group were also lower than that in the haplo group (25.6% 51.3%, = 0.019; 16.2% 41.3%, = 0.016, respectively). Haplo-cord HSCT was identified as the only factor associated with a lower incidence of II-IV aGVHD and cGVHD in multivariate analysis. However, no differences were observed between the two groups for infections and survival outcomes.
Our data indicated that co-infusion of UCB in 'Beijing Protocol'-based haplo-HSCT may be effective for reducing the risk of severe GVHD in SAA children.
基于粒细胞集落刺激因子加抗胸腺细胞方案的单倍型造血干细胞移植（单倍型HSCT，即“北京方案”）为中国获得性重型再生障碍性贫血（SAA）患者提供了一种挽救性治疗方法。然而，由于人类白细胞抗原不相合，移植物抗宿主病（GVHD）是单倍型HSCT的主要障碍。最近，单倍型HSCT联合脐血（UCB）（单倍型-脐血HSCT）已在临床试验中开展，以潜在降低严重GVHD的风险。尽管如此，比较接受单倍型和单倍型-脐血HSCT治疗SAA的儿科患者中GVHD情况的研究仍然有限。
本研究旨在探讨UCB共输注对接受单倍型HSCT治疗SAA的儿科患者中GVHD的影响。
我们对在本中心接受基于“北京方案”的单倍型移植且有或无UCB共输注的91例连续SAA儿童进行了一项回顾性研究。
所有患者均接受统一的非清髓性预处理和GVHD预防。我们比较了单倍型（n = 35）和单倍型-脐血（n = 56）受者的基线特征和移植结局。
所有91例患者均从单倍型供者实现了造血恢复，与单倍型组相比，单倍型-脐血组植入综合征的发生率更高（75.0% vs 48.6%，P = 0.029）。值得注意的是，单倍型-脐血组II-IV级急性GVHD（aGVHD）的发生率低于单倍型组（16.1% vs 42.9%，P = 0.002）。单倍型-脐血组慢性GVHD（cGVHD）以及中度至重度cGVHD的观察发生率也低于单倍型组（分别为25.6% vs 51.3%，P = 0.019；16.2% vs 41.3%，P = 0.016）。在多因素分析中，单倍型-脐血HSCT被确定为与II-IV级aGVHD和cGVHD发生率较低相关的唯一因素。然而，两组在感染和生存结局方面未观察到差异。
我们的数据表明，在基于“北京方案”的单倍型HSCT中，UCB共输注可能对降低SAA儿童严重GVHD的风险有效。
