Successful Hematopoietic Stem Cell Transplantation from a Matched Related Donor with Beta-Thalassemia Minor for Severe Aplastic Anemia

Abstract

The first-line treatment for severe aplastic anemia (SAA) patients is hematopoietic stem cell transplantation (HSCT), with full-matched related donors considered the most suitable. We report a case of SAA in which the patient successfully underwent HSCT from a donor with β-thalassemia minor. The patient in this case underwent HSCT from a human leukocyte antigen (HLA)-matched younger brother with β-thalassemia minor. A 7-year-old girl was referred to our facility following a 6-month history of easy bruising and pallor. Laboratory examinations showed pancytopenia and hypocellular bone marrow with cellularity of <5%. She was diagnosed with acquired SAA, and HLA typing of her family members was performed. Her younger brother was an HLA-matched sibling but had β-thalassemia minor. Since his hemoglobin levels were maintained at 10–11 d/dL, he was considered a suitable HSCT donor. The conditioning regimen included fludarabine, cyclophosphamide, and anti-thymocyte globulin. The CD34+ and CD3+ cell counts were 6.6 × 106/kg and 0.48 × 108/kg, respectively. White blood cell engraftment was evident on day +11. Regimen-associated toxicities, such as anorexia and enteritis, were mild; no infections occurred, and no symptoms of acute graft-versus-host disease (GVHD) were observed. The 30-day follow-up bone marrow examination revealed normocellular marrow with 80%–90% cellularity. Acute or chronic GVHD has not been reported, and good performance status has been observed throughout the 5 years after HSCT. β-thalassemia minor patients can be considered as bone marrow donors for SAA patients.

Case report: First report of haploidentical allogeneic hematopoietic stem cell transplantation from donors with mild alpha-thalassemia for acute leukemia

“…Mi YJ et al reported a rare case in which allo-HSCT for a 7-year-old severe aplastic anemia patient from a MSD with mild b-thalassemia was performed successful. In this case, the engraftment of erythrocytes was evident, good performance status has been observed throughout the 5 years after HSCT and the PBSCs mobilized by G-CSF were sufficient (11). Li K et al also reported that for the efficiency of mobilization of PBCSs in patients with thalassemia, there were no significant differences in the CD34+ cell subsets and lymphocyte subsets after G-CSF administration when compared with those in healthy donors (12).…”

“…reported a rare case in which allo-HSCT for a 7-year-old severe aplastic anemia patient from a MSD with mild β-thalassemia was performed successful. In this case, the engraftment of erythrocytes was evident, good performance status has been observed throughout the 5 years after HSCT and the PBSCs mobilized by G-CSF were sufficient ( 11 ). Li K et al.…”

Unraveling trajectories from aplastic anemia to hematologic malignancies: genetic and molecular insights

“…However, advances in medical science have resulted in innovative treatment strategies. Hematopoietic stem cell transplantation from a matched sibling donor has been considered the gold standard for treatment, offering a potential cure ( 8 , 9 ). However, the availability of suitable donors is limited, and the procedure carries risks of complications ( 3 ).…”