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
- Cseh, Annamária
- Galimard, Jacques‐Emmanuel
- de la Fuente, Josu
- Isgro, Antonella
- Zecca, Marco
- Garwer, Birgit
- Biffi, Alexandra
- Aljurf, Mahmoud
- Sundin, Mikael
- Belendez, Cristina
- Locatelli, Franco
- Balduzzi, Adriana
- Lawson, Sarah
- Sengeloev, Henrik
- Ifversen, Marianne
- Saccardi, Riccardo
- Wynn, Robert
- Lankester, Arjan C.
- Corbacioglu, Selim
- Peters, Christina
DOIAbstract
: 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 acu...
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