Allogeneic hematopoietic cell transplantation in patients with myelodysplastic syndrome using treosulfan based compared to other reduced-intensity or myeloablative conditioning regimens. A report of the chronic malignancies working party of the EBMT

Avichai Shimoni, Marie Robin, Simona Iacobelli, Dietrich Beelen, Ghulam J. Mufti, Fabio Ciceri, Wolfgang Bethge, Liisa Volin, Didier Blaise, Arnold Ganser, Thomas Luft, Patrice Chevallier, Rainer Schwerdtfeger, Linda Koster, Theo de Witte, Nicolaus Kröger, Arnon Nagler, Ibrahim Yakoub-Agha

Research output: Contribution to journalArticlepeer-review

Abstract

Allogeneic haematopoietic-cell transplantation (allo-HCT) is a potentially curative therapy for high-risk myelodysplastic syndrome (MDS). Reduced-intensity conditioning (RIC) is usually associated with lower non-relapse mortality (NRM), higher relapse rate and similar overall-survival (OS) as myeloablative-conditioning (MAC). Fludarabine/treosulfan (FT) is a reduced-toxicity regimen with intense anti-leukaemia activity and a favourable toxicity profile. We investigated post-transplant outcomes in 1722 MDS patients following allo-HCT with FT (n = 367), RIC (n = 687) or MAC (n = 668). FT and RIC recipients were older than MAC recipients, median age 59, 59 and 51 years, respectively (P < 0·001) but other disease characteristics were similar. The median follow-up was 64 months (1–171). Five-year relapse rates were 25% (21–30), 38% (34–42) and 25% (22–29), after FT, RIC and MAC, respectively, (P < 0·001). NRM was 30% (25–35), 27% (23–30) and 34% (31–38, P = 0·008), respectively. Five-year OS was 50% (44–55), 43% (38–47), and 43% (39–47), respectively (P = 0·03). In multivariate analysis, FT was associated with a lower risk of relapse (HR 0·55, P < 0·001) and better OS (HR 0·72, P = 0·01). MAC was associated with higher NRM (HR 1·44, P = 0·001). In conclusion, FT is associated with similar low relapse rates as MAC and similar low NRM as RIC, resulting in improved OS. FT may be the preferred regimen for allo-HCT in MDS.

Original languageEnglish
Pages (from-to)417-428
Number of pages12
JournalBritish Journal of Haematology
Volume195
Issue number3
DOIs
StatePublished - Nov 2021

Keywords

  • allogeneic haematopoietic cell transplantation
  • myeloablative conditioning
  • myelodysplastic syndrome
  • reduced-intensity conditioning
  • treosulfan

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