Efficacy of hematopoietic stem cell transplantation in the treatment of children with non‐severe aplastic anemia

• Published in: Pediatric transplantation 2022-11, Vol.26 (7), p.e14340-n/a
• Main Authors: Zhang, Man, Zhu, Guanghua, Cai, Lixiao, Yuan, Meng, Wu, Runhui, Jia, Chenguang, Wang, Bin, Zheng, Jie, Ma, Jie, Qin, Maoquan, Li, Sidan
• Format: Article
• Language: English
• Subjects: Anemia; Aplastic anemia; children; hematopoietic stem cell transplantation; Hematopoietic stem cells; non‐severe aplastic anemia; Patients; Pediatrics; Stem cell transplantation
• ISSN: 1397-3142; 1399-3046
• DOI: 10.1111/petr.14340

Background 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. Methods 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. Results 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%. Conclusions Hematopoietic stem cell transplantation is a practicable, safe, and effective treatment option for non‐severe aplastic anemia pediatric patients who are suitable for transplant.