EFFICACY AND SAFETY OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION AS FIRST-LINE TREATMENT FOR SEVERE APLASTIC ANEMIA IN CHILDREN: A SYSTEMATIC REVIEW

@article{viana2026efficacy,
  title = {{EFFICACY AND SAFETY OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION AS FIRST-LINE TREATMENT FOR SEVERE APLASTIC ANEMIA IN CHILDREN: A SYSTEMATIC REVIEW}},
  author = {Pedro Henrique Rodrigues VIANA and Lucas De Barros ANASTáCIO},
  journal = {Hematology, Transfusion and Cell Therapy},
  year = {2026},
  month = oct,
  doi = {10.1016/j.htct.2026.106861},
  url = {https://doi.org/10.1016/j.htct.2026.106861},
  note = {CC BY},
}

TY  - JOUR
TI  - EFFICACY AND SAFETY OF HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTATION AS FIRST-LINE TREATMENT FOR SEVERE APLASTIC ANEMIA IN CHILDREN: A SYSTEMATIC REVIEW
AU  - Pedro Henrique Rodrigues VIANA
AU  - Lucas De Barros ANASTáCIO
PY  - 2026
DA  - 2026/10/23
JO  - Hematology, Transfusion and Cell Therapy
DO  - 10.1016/j.htct.2026.106861
UR  - https://doi.org/10.1016/j.htct.2026.106861
AB  - Introduction: Severe aplastic anemia (SAA) in children causes bone marrow failure and high infection and hemorrhagic risk. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the main treatment, favoring HLA-matched sibling donors. Pediatric haploidentical transplantation is becoming more relevant due to the lack of matched donors. Objective: This systematic review aimed to appraise the available evidence regarding the efficacy and safety of haploidentical transplantation as a first-line therapeutic approach for children with severe aplastic anemia. Methods: This systematic review was conducted in accordance with PRISMA guidelines, using PubMed as the sole database. The search strategy combined Medical Subject Headings (MeSH) and free-text terms across five conceptual blocks: severe aplastic anemia, hematopoietic stem cell transplantation, haploidentical donor source, frontline/first-line treatment setting, and pediatric population, connected by Boolean operators (AND/OR), with an additional NOT operator applied to exclude records pertaining to Fanconi anemia. Studies published between 2020 and 2026 were considered eligible. Inclusion criteria comprised publication within the defined date range, studies conducted in human subjects, and inclusion of a pediatric population. Exclusion criteria comprised studies restricted to an adult population, or studies in which outcome data for the pediatric subgroup could not be individually extracted or qualitatively assessed. Study selection was performed independently by two reviewers, and a qualitative synthesis was conducted to characterize the results of the selection process, the corpus of included studies, and their principal findings. Results: Twenty-seven publications were retrieved after application of the temporal filter. Following application of the exclusion criteria, five studies were selected for inclusion in this review, encompassing a total of 148 pediatric patients who underwent haploidentical HSCT (haplo-HSCT) for SAA. The included studies reported first-line overall survival rates ranging from 83.3% to 100%. Compared with matched related donor transplantation or immunosuppressive therapy, haplo-HSCT did not differ statistically in two studies but demonstrated inferior outcomes in two others, potentially attributable to small sample sizes and indication bias. Only one study provided detailed data on myeloid engraftment and graft failure. Severe acute graft-versus-host disease (grade III–IV GVHD) was observed in 0% of patients across the three studies employing universal post-transplant cyclophosphamide (PTCy), compared with 25% in the single study that did not use PTCy, suggesting a protective effect of cyclophosphamide in this context. Infection, particularly cytomegalovirus reactivation, constituted the leading cause of transplant-related mortality. Conclusion: Haploidentical HSCT-PTCy is feasible, safe, and potentially effective as first-line treatment for pediatric SAA without a matched donor, with survival rates approaching those of matched sibling donor transplantation and low severe GVHD rates. Nevertheless, the current body of evidence remains constrained by retrospective study designs, small sample sizes, heterogeneity in reported outcomes, and donor selection bias. Adequately powered prospective randomized controlled trials are warranted to validate these findings.
SN  - 2531-1379
SN  - 2531-1387
ER  -