Allogeneic Hematopoietic Cell Transplantation for Pediatric Unstable Hemoglobinopathies: An Aggregated Cohort with Long-Term Follow-Up from a Brazilian Center

Background/Objectives: Severe unstable hemoglobinopathies may cause transfusion dependence from early childhood, and allogeneic hematopoietic cell transplantation (allo-HCT) is the only established curative option. Pediatric experience with allo-HCT for unstable hemoglobinopathies is limited to isolated reports, and long-term outcomes remain poorly characterized. The objective of this study was to characterize the clinical and molecular features, transplant approaches, and long-term outcomes of two Brazilian patients with unstable hemoglobinopathies, in the context of the published pediatric literature.

Methods

We performed a retrospective analysis of two children with unstable hemoglobinopathies who underwent allo-HCT at a Brazilian center. PubMed/MEDLINE and Scopus were searched for pediatric cases published between 2002 and 2025 using terms related to unstable hemoglobin variants and hematopoietic transplantation. Published cases were combined with the Brazilian patients to form an aggregated cohort. Clinical, molecular, transplant, and follow-up data were analyzed descriptively.