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

Autor: Hernández-Boluda, Juan Carlos, Eikema, Diderik-Jan, Koster, Linda, Kröger, Nicolaus, Robin, Marie, de Witte, Moniek, Finke, Jürgen, Finazzi, Maria Chiara, Broers, Annoek, Raida, Ludek, Schaap, Nicolaas, Chiusolo, Patrizia, Verbeek, Mareike, Hazenberg, Carin L. E., Halaburda, Kazimierz, Kulagin, Aleksandr, Labussière-Wallet, Hélène, Gedde-Dahl, Tobias, Rabitsch, Werner, Raj, Kavita, Drozd-Sokolowska, Joanna, Battipaglia, Giorgia, Polverelli, Nicola, Czerw, Tomasz, Yakoub-Agha, Ibrahim, McLornan, Donal P.
Zdroj: Bone Marrow Transplantation; 20230101, Issue: Preprints p1-11, 11p
Abstrakt: 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 CALRmutation in allo-HCT and support molecular profiling in prognostic scoring systems to predict OS after transplantation in MF.
Databáze: Supplemental Index