A European survey on allogeneic haematopoietic cell transplantation for myelofibrosis on behalf of the Chronic Malignancies Working Party of the EBMT: focus on ‘real world’ experience of JAK inhibitors, splenomegaly management and novel agents in the transplant algorithm
Year: 2025
Type: article
Study design: Observational Study
Abstract: Allogeneic haematopoietic cell transplantation (allo-HCT) remains the only potentially curative option for patients with myelofibrosis (MF), yet the integration of JAK inhibitors (JAKi) and novel agents into transplant pathways has created increasing complexity. To capture current real-world practice, the EBMT Chronic Malignancies Working Party conducted a survey of 19 high-volume European centres performing MF allo-HCT. Most centres (68%) routinely initiated JAKi, primarily ruxolitinib, in transplant-eligible patients prior to conditioning, with goals of splenomegaly reduction and symptom control. Management of ruxolitinib intolerance or resistance was heterogeneous, with strategies including switching to alternative JAKi, proceeding directly to allo-HCT, or enroling in clinical trials. Peri-transplant approaches also varied: over half of centres continued ruxolitinib throughout conditioning, while others employed tapering or abrupt discontinuation. Experience with newer JAKi and investigational therapies was limited. Post-transplant, most centres did not routinely reintroduce JAKi, although some used them for relapse or GVHD mitigation. Notably, many centres reported transplant delays due to prolonged medical therapy, with adverse consequences including disease progression. These findings highlight significant heterogeneity in practice, which is likely to increase as more novel agents are integrated in treatment algorithms. Harmonised, multidisciplinary guidelines to optimise timing and outcomes for MF patients eligible for allo-HCT are needed.
Source: Bone Marrow Transplantation
Authors: Alexandros Rampotas, Jose Maria Aspa-Cilleruelo, Linda Koster, Daniele Avenoso, Jakob Passweg, Elisa Sala, Marie Robin, Anders Eivind Myhre, Moniek de Witte, Erfan Nur, Patrice Chevallier, Thomas Schroeder, Micha Srour, Patrizia Chiusolo, Urpu Salmenniemi, Mareike Verbeek, Maria Chiara Finazzi, Cristina Castilla-Llorente, Marie Therese Rubio, Patryk Sobieralski, Katja Sockel, Ahmad Alabdulkarim, Joanna Drozd-Sokolowska, Kavita Raj, Giorgia Battipaglia, Tomasz Czerw, Nicola Polverelli, Juan Carlos Hernández-Boluda, Donal P. McLornan
Institutions: University College London, Sheffield Teaching Hospitals NHS Foundation Trust, European Society for Blood and Marrow Transplantation, Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia, University of Brescia, University Hospital of Basel, University Hospital Ulm, Université Paris Cité, Assistance Publique – Hôpitaux de Paris, Hôpital Saint-Louis, Oslo University Hospital, University Medical Center Utrecht, Amsterdam University Medical Centers, University of Amsterdam, Centre Hospitalier Universitaire de Nantes, Essen University Hospital, Centre Hospitalier Universitaire de Lille, Agostino Gemelli University Polyclinic, Helsinki University Hospital, TUM Klinikum, Technical University of Munich, Ospedale Papa Giovanni XXIII, Institut Gustave Roussy, Centre Hospitalier Régional et Universitaire de Nancy, University Clinical Centre, Gdańsk Medical University, University Hospital Carl Gustav Carus, Medical University of Warsaw, Federico II University Hospital, University of Naples Federico II, The Maria Sklodowska-Curie National Research Institute of Oncology, Policlinico San Matteo Fondazione, Hospital Clínico Universitario de Valencia
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,990
OpenAlex ID: W7117129571
