Zhang Man, Zhu Guanghua, Cai Lixiao, Yuan Meng, Wu Runhui, Jia Chenguang, Wang Bin, Zheng Jie, Ma Jie, Qin Maoquan, Li Sidan
Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Ministry of Education, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Department of Stem Cell Transplantation, National Center for Children's Health, Baoding Children's Hospital, Baoding, China.
Pediatr Transplant. 2022 Nov;26(7):e14340. doi: 10.1111/petr.14340. Epub 2022 Jun 23.
Non-severe aplastic anemia is more likely to develop into severe aplastic anemia, and there is no widely accepted treatment plan at present. Hematopoietic stem cell transplantation might be a new therapeutic strategy.
Retrospectively analyzed 32 patients with non-severe aplastic anemia who underwent hematopoietic stem cell transplantation from September 2007 to September 2020, and the 5-year estimated overall survival rate and the incidence of graft-versus-host disease were analyzed to evaluate the efficacy and safety of hematopoietic stem cell transplantation in the treatment of pediatric non-severe aplastic anemia.
Thirty-two patients who underwent transplantation, 29 patients (90.6%) survived, 3 patients (9.4%) died. The incidence of acute graft-versus-host disease was 51.6% (16/31), including 15 cases (48.4%) of grade I-II and 1 case (3.2%) of grade III-IV. The incidence of chronic graft-versus-host disease was 38.7% (12/31). The 5-year overall survival rate was 91.8%.
Hematopoietic stem cell transplantation is a practicable, safe, and effective treatment option for non-severe aplastic anemia pediatric patients who are suitable for transplant.
非重型再生障碍性贫血更易发展为重型再生障碍性贫血，目前尚无广泛接受的治疗方案。造血干细胞移植可能是一种新的治疗策略。
回顾性分析2007年9月至2020年9月接受造血干细胞移植的32例非重型再生障碍性贫血患儿，分析其5年总生存率及移植物抗宿主病发生率，以评估造血干细胞移植治疗儿童非重型再生障碍性贫血的疗效和安全性。
32例接受移植的患者中，29例（90.6%）存活，3例（9.4%）死亡。急性移植物抗宿主病发生率为51.6%（16/31），其中Ⅰ-Ⅱ级15例（48.4%），Ⅲ-Ⅳ级1例（3.2%）。慢性移植物抗宿主病发生率为38.7%（12/31）。5年总生存率为91.8%。
造血干细胞移植是适合移植的儿童非重型再生障碍性贫血患者可行、安全且有效的治疗选择。
