Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis: a study of the Chronic Malignancies Working Party of EBMT

Juan Carlos Hernández-Boluda*, Diderik Jan Eikema, Linda Koster, Nicolaus Kröger, Marie Robin, Moniek de Witte, Jürgen Finke, Maria Chiara Finazzi, Annoek Broers, Ludek Raida, Nicolaas Schaap, Patrizia Chiusolo, Mareike Verbeek, Carin L.E. Hazenberg, Kazimierz Halaburda, Aleksandr Kulagin, Hélène Labussière-Wallet, Tobias Gedde-Dahl, Werner Rabitsch, Kavita RajJoanna Drozd-Sokolowska, Giorgia Battipaglia, Nicola Polverelli, Tomasz Czerw, Ibrahim Yakoub-Agha, Donal P. McLornan*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

1 Citation (Scopus)

Abstract

Allogeneic hematopoietic cell transplantation (allo-HCT) is curative for myelofibrosis (MF) but assessing risk-benefit in individual patients is challenging. This complexity is amplified in CALR-mutated MF patients, as they live longer with conventional treatments compared to other molecular subtypes. We analyzed outcomes of 346 CALR-mutated MF patients who underwent allo-HCT in 123 EBMT centers between 2005 and 2019. After a median follow-up of 40 months, the estimated overall survival (OS) rates at 1, 3, and 5 years were 81%, 71%, and 63%, respectively. Patients receiving busulfan-containing regimens achieved a 5-year OS rate of 71%. Non-relapse mortality (NRM) at 1, 3, and 5 years was 16%, 22%, and 26%, respectively, while the incidence of relapse/progression was 11%, 15%, and 17%, respectively. Multivariate analysis showed that older age correlated with worse OS, while primary MF and HLA mismatched transplants had a near-to-significant trend to decreased OS. Comparative analysis between CALR- and JAK2-mutated MF patients adjusting for confounding factors revealed better OS, lower NRM, lower relapse, and improved graft-versus-host disease-free and relapse-free survival (GRFS) in CALR-mutated patients. These findings confirm the improved prognosis associated with CALR mutation in allo-HCT and support molecular profiling in prognostic scoring systems to predict OS after transplantation in MF.

Original languageEnglish
Pages (from-to)1357-1367
Number of pages11
JournalBone Marrow Transplantation
Volume58
Issue number12
DOIs
Publication statusPublished - Dec 2023

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© 2023, The Author(s), under exclusive licence to Springer Nature Limited.

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