TY - JOUR
T1 - Allogeneic hematopoietic cell transplantation in patients with CALR-mutated myelofibrosis
T2 - a study of the Chronic Malignancies Working Party of EBMT
AU - Hernández-Boluda, Juan Carlos
AU - Eikema, Diderik Jan
AU - Koster, Linda
AU - Kröger, Nicolaus
AU - Robin, Marie
AU - de Witte, Moniek
AU - Finke, Jürgen
AU - Finazzi, Maria Chiara
AU - Broers, Annoek
AU - Raida, Ludek
AU - Schaap, Nicolaas
AU - Chiusolo, Patrizia
AU - Verbeek, Mareike
AU - Hazenberg, Carin L.E.
AU - Halaburda, Kazimierz
AU - Kulagin, Aleksandr
AU - Labussière-Wallet, Hélène
AU - Gedde-Dahl, Tobias
AU - Rabitsch, Werner
AU - Raj, Kavita
AU - Drozd-Sokolowska, Joanna
AU - Battipaglia, Giorgia
AU - Polverelli, Nicola
AU - Czerw, Tomasz
AU - Yakoub-Agha, Ibrahim
AU - McLornan, Donal P.
N1 - Publisher Copyright:
© 2023, The Author(s), under exclusive licence to Springer Nature Limited.
PY - 2023/12
Y1 - 2023/12
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85169890003&partnerID=8YFLogxK
U2 - 10.1038/s41409-023-02094-1
DO - 10.1038/s41409-023-02094-1
M3 - Article
C2 - 37679647
AN - SCOPUS:85169890003
SN - 0268-3369
VL - 58
SP - 1357
EP - 1367
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 12
ER -