Transplantation for myelofibrosis patients in the ruxolitinib era: a registry study from the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire

• Published in: Bone marrow transplantation (Basingstoke) Vol. 59; no. 7; pp. 965 - 973
• Main Authors: Villar, Sara, Chevret, Sylvie, Poire, Xavier, Joris, Magalie, Chevallier, Patrice, Bourhis, Jean-Henri, Forcade, Edouard, Chantepie, Sylvain, Beauvais, David, Raus, Nicole, Bay, Jacques-Olivier, Loschi, Michael, Devillier, Raynier, Duléry, Remy, Ceballos, Patrice, Rubio, Marie Thérèse, Servais, Sophie, Nguyen, Stephanie, Robin, Marie
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.07.2024; Nature Publishing Group
• Series: Bone Marrow Transplantation
• Subjects: 692/699/1541/1990; 692/700; Adult; Aged; Allografts; Cell Biology; Female; Fludarabine; Hematology; Hematopoietic Stem Cell Transplantation - methods; Humans; Internal Medicine; Leukemia; Life Sciences; Male; Medicine; Medicine & Public Health; Melphalan; Middle Aged; Mortality; Myelofibrosis; Nitriles; Primary Myelofibrosis - drug therapy; Primary Myelofibrosis - mortality; Primary Myelofibrosis - therapy; Public Health; Pyrazoles - therapeutic use; Pyrimidines - therapeutic use; Registries; Risk factors; Spleen; Stem cell transplantation; Stem Cells; Survival Rate; Transplantation; Transplantation Conditioning - methods
• ISSN: 0268-3369; 1476-5365; 1476-5365
• DOI: 10.1038/s41409-024-02268-5

In this SFGM-TC registry study, we report the results after stem cell transplantation (HSCT) in 305 myelofibrosis patients, in order to determine potential risk factors associated with outcomes, especially regarding previous treatment with ruxolitinib. A total of 102 patients were transplanted from an HLA-matched-sibling donor (MSD), and 143 patients received ruxolitinib. In contrast with previous studies, our results showed significantly worse outcomes for ruxolitinib patients regarding overall survival (OS) and non-relapse mortality (NRM), especially in the context of unrelated donors (URD). When exploring reasons for potential confounders regarding the ruxolitinib effect, an interaction between the type of donor and the use of ATG was found, therefore subsequent analyses were performed separately for each type of donor. Multivariable analyses did not confirm a significant negative impact of ruxolitinib in transplantation outcomes. In the setting of URD, only age and Fludarabine-Melphalan (FM) conditioning were associated with increased NRM. For MSD, only Karnoksfy <70% was associated with reduced OS. However, a propensity score analysis showed that ruxolitinib had a negative impact on OS but only in non-responding patients, consistent with previous data. To conclude, with all the precautions due to confounders and bias, ruxolitinib itself does not appear to increase mortality after HSCT.