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

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

*Corresponding author af dette arbejde
1 Citationer (Scopus)

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.

OriginalsprogEngelsk
TidsskriftBritish Journal of Haematology
Vol/bind204
Udgave nummer1
Sider (fra-til)e1-e5
Antal sider5
ISSN0007-1048
DOI
StatusUdgivet - jan. 2024

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