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

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     «

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, Do...     »

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.      «

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) rat...     »