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.

1st State Pavlov Medical University of St Petersburg St Petersburg Russian Federation

BMT Unit Internal Medicine 1 Medical University of Vienna Vienna Austria

Central Clinical Hospital The Medical University of Warsaw Warsaw Poland

Centre Hospitalier Lyon Sud Lyon France

CHU de Lille Univ Lille INSERM U1286 Infinite 59000 Lille France

EBMT Leiden Study Unit Leiden the Netherlands

EBMT Statistical Unit Leiden the Netherlands

Erasmus MC Cancer Institute Rotterdam the Netherlands

Hematology Department Federico 2 University of Naples Naples Italy

Hôpital Saint Louis APHP Université de Paris Cité Paris France

Hospital Clínico Universitario INCLIVA University of Valencia Valencia Spain

Institute of Hematology and Transfusion Medicine Warsaw Poland

Maria Skłodowska Curie National Research Institute of Oncology Gliwice Poland

Olomouc University Hospital Olomouc Czech Republic

Oslo University Hospital Hematology dep Stem cell transplantation and Institute for Clinical Medicine University of Oslo Oslo Norway

Radboud University Medical Centre Nijmegen the Netherlands

Sezione di Ematologia Dipartimento di Scienze Radiologiche ed Ematologiche Università Cattolica del Sacro Cuore Dipartamento di Diagnostica per Immagini Radioterapia Oncologica ed Ematologia Fondazione Policlinico A Gemelli IRCCS Rome Italy

Technical University of Munich School of Medicine Klinikum rechts der Isar Clinic and Policlinic for Internal Medicine 3 Munich Germany

Unit of Blood Diseases and Stem Cell Transplant ASST Spedali Civili University of Brescia Brescia Italy

University College London Hospitals NHS Trust London UK

University Hospital Eppendorf Hamburg Germany

University Medical Center Groningen University of Groningen Groningen the Netherlands

University Medical Center Utrecht the Netherlands

University of Freiburg and Medical Faculty Freiburg Germany

University of Milan and ASST Papa Giovanni XXIII Bergamo Italy

Bone Marrow Transplant. 2024 Jan;59(1):1-3. doi: 10.1038/s41409-023-02112-2. PubMed

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