Growth and puberty following treosulfan HSCT for thalassemia
Ganesan K, Muthukumar V, Nair A, et al. A 10-Year Follow-Up Study of Children With Thalassemia Major Post-Transplantation Using Treosulfan, Thiotepa, and Fludarabine-Based Conditioning Regimen and Its Impact on Growth and Puberty. Transplantation and Cellular Therapy. 2025; (doi: 10.1016/j.jtct.2025.07.011).
Among children with thalassemia major, researchers say treosulfan-based conditioning prepares them for hematopoietic stem cell transplantation (allo-HSCT) without harming their natural growth cycle. A retrospective analysis considered 202 children who underwent allo-HSCT between 2010 and 2020. The median height standard deviation score was -0.574 at baseline and -0.669 at outcome assessment a minimum of 2 years later, a narrow margin interpreted to signify no meaningful reduction in growth potential. In all, 79.3% of participants — primarily those with Class 1 or 2 thalassemia — achieved optimal growth velocity with sufficient catch-up growth following allo-HSCT. Those with delayed growth velocity typically had Class 3 thalassemia and/or developed chronic graft-versus-host disease post-transplantation. The data also showed that only 14 patients needed growth hormone supplementation and that 54.2% of the 83 girls evaluated experienced spontaneous menarche. The authors conclude that the conditioning regimen generally did not disrupt growth potential in these young patients and that pubertal growth for most held near where it should have been based on age.