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Use of machine learning techniques to predict poor survival after hematopoietic cell transplantation for myelofibrosis

. 2025 Jun 26 ; 145 (26) : 3139-3152.

Language English Country United States Media print

Document type Journal Article

With the incorporation of effective therapies for myelofibrosis (MF), accurately predicting outcomes after allogeneic hematopoietic cell transplantation (allo-HCT) is crucial for determining the optimal timing for this procedure. Using data from 5183 patients with MF who underwent first allo-HCT between 2005 and 2020 at European Society for Blood and Marrow Transplantation centers, we examined different machine learning (ML) models to predict overall survival after transplant. The cohort was divided into a training set (75%) and a test set (25%) for model validation. A random survival forests (RSF) model was developed based on 10 variables: patient age, comorbidity index, performance status, blood blasts, hemoglobin, leukocytes, platelets, donor type, conditioning intensity, and graft-versus-host disease prophylaxis. Its performance was compared with a 4-level Cox regression-based score and other ML-based models derived from the same data set, and with the Center for International Blood and Marrow Transplant Research score. The RSF outperformed all comparators, achieving better concordance indices across both primary and postessential thrombocythemia/polycythemia vera MF subgroups. The robustness and generalizability of the RSF model was confirmed by Akaike information criterion and time-dependent receiver operating characteristic area under the curve metrics in both sets. Although all models were prognostic for nonrelapse mortality, the RSF provided better curve separation, effectively identifying a high-risk group comprising 25% of patients. In conclusion, ML enhances risk stratification in patients with MF undergoing allo-HCT, paving the way for personalized medicine. A web application (https://gemfin.click/ebmt) based on the RSF model offers a practical tool to identify patients at high risk for poor transplantation outcomes, supporting informed treatment decisions and advancing individualized care.

Ematologia e Terapie Cellulari IRCCS Ospedale Policlinico San Martino Genova Italy

European Group for Blood and Marrow Transplantation Leiden Study Unit Leiden The Netherlands

Hematology Department Central Clinical Hospital The Medical University of Warsaw Warsaw Poland

Hematology Department Centre Hospitalier Universitaire de Lille INSERM U1286 Infinite Lille France

Hematology Department Erasmus MC Cancer Institute Rotterdam The Netherlands

Hematology Department Federico 2 University of Naples Naples Italy

Hematology Department Hospital Clínico Universitario Instituto de Investigación Sanitaria del Hospital Clínico de Valencia University of Valencia Valencia Spain

Hematology Department Maria Skłodowska Curie National Research Institute of Oncology Gliwice Poland

Hematology Department Medical Clinic and Policlinic Leipzig Germany

Hematology Department Rigshospitalet Copenhagen Denmark

Hematology Department Saint Louis Hospital Bone Marrow Transplantation Unit Paris France

Hematology Department University College London Hospitals National Health Service Trust London United Kingdom

Hematology Department University Hospital Basel Basel Switzerland

Hematology Department University Hospital Essen Duesseldorf Germany

Hematology Department University Hospital of Santiago de Compostela Instituto de Investigación Sanitaria de Santiago de Compostela Santiago de Compostela Spain

Hematology Department University Hospital Technische Universität Dresden Dresden Germany

Hematology Department University Hospital Uppsala Uppsala Sweden

Hematology Department University Medical Center Hamburg Eppendorf Hamburg Germany

Hematology Department University Medical Centre Utrecht The Netherlands

Hematology Department University of Liege Liege Belgium

Hematology Department University of Muenster Muenster Germany

Institute of Hematology and Blood Transfusion Prague Czech Republic

Medizinische Klinik m S Hämatologie Onkologie und Tumorimmunologie Berlin Germany

Unit of Bone Marrow Transplantation Division of Hematology Fondazione IRCCS Policlinico San Matteo Pavia Italy

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