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

Pediatr Transplant. 2022 Nov;26(7):e14340. doi: 10.1111/petr.14340. Epub 2022 Jun 23.

Abstract

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.

Keywords: children; hematopoietic stem cell transplantation; non-severe aplastic anemia.

MeSH terms

  • Anemia, Aplastic* / therapy
  • Child
  • Graft vs Host Disease* / etiology
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Retrospective Studies
  • Transplantation Conditioning