1. Busulfan-fludarabine- or treosulfan-fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties.
- Author
-
Cseh A, Galimard JE, de la Fuente J, Isgro A, Zecca M, Garwer B, Biffi A, Aljurf M, Sundin M, Belendez C, Locatelli F, Balduzzi A, Lawson S, Sengeloev H, Ifversen M, Saccardi R, Wynn R, Lankester AC, Corbacioglu S, and Peters C
- Subjects
- Humans, Child, Busulfan therapeutic use, Siblings, Vidarabine therapeutic use, Transplantation Conditioning, Retrospective Studies, Hematopoietic Stem Cell Transplantation, Graft vs Host Disease etiology, Graft vs Host Disease prevention & control, Anemia, Sickle Cell therapy
- Abstract
How important is choice of conditioning regimen in allogeneic haematopoietic stem cell transplantation (HSCT) for sickle cell disease (SCD)? We compared HSCT outcomes by conditioning regimen in paediatric patients with SCD from the EBMT registry. In 2010-2020, 251 patients aged <18 years underwent a first matched sibling donor (MSD) HSCT with conditioning based on busulfan-fludarabine (bu-flu; n = 89) or treosulfan-fludarabine (treo-flu; n = 162). In the bu-flu and treo-flu groups, 51.7% and 99.4% of patients, respectively, received thiotepa. Median follow-up was 2.7 years. Two-year overall survival (OS) was 98.7% (95% confidence interval [CI]: 90.9-99.8) with bu-flu and 99.3% (95% CI: 95.2-99.9) with treo-flu (p = 0.63). Grade III-IV acute graft-versus-host disease (GVHD) at 100 days was 2.4% (95% CI: 0.4-7.5) and 0.6% (0.1%-3.2%) for bu-flu and treo-flu respectively (p = 0.25). The 2-year incidence of extensive chronic GVHD was 1.5% (95% CI: 0.1-7.3) with bu-flu and 8.0% (95% CI: 4.1-13.3) with treo-flu (p = 0.057). These multinational data confirm the excellent curative capacity of MSD HSCT with myeloablative conditioning. Both conditioning regimens yielded excellent OS, low rates of acute and chronic GVHD, and low rates of graft failure., (© 2023 British Society for Haematology and John Wiley & Sons Ltd.)
- Published
- 2024
- Full Text
- View/download PDF