Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: Updated recommendations by the EBMT/ELN International Working Group Review
| Authors: | Kröger, N.; Bacigalupo, A.; Barbui, T.; Ditschkowski, M.; Gagelmann, N.; Griesshammer, M.; Gupta, V.; Hamad, N.; Harrison, C.; Hernandez-Boluda, J. C.; Koschmieder, S.; Jain, T.; Mascarenhas, J.; Mesa, R.; Popat, U. R.; Passamonti, F.; Polverelli, N.; Rambaldi, A.; Robin, M.; Salit, R. B.; Schroeder, T.; Scott, B. L.; Tamari, R.; Tefferi, A.; Vannucchi, A. M.; McLornan, D. P.; Barosi, G. |
| Review Title: | Indication and management of allogeneic haematopoietic stem-cell transplantation in myelofibrosis: Updated recommendations by the EBMT/ELN International Working Group |
| Abstract: | New options for medical therapy and risk scoring systems containing molecular data are leading to increased complexity in the management of patients with myelofibrosis. To inform patients’ optimal care, we updated the 2015 guidelines on indications for and management of allogeneic haematopoietic stem-cell transplantation (HSCT) with the support of the European Society for Blood and Marrow Transplantation (EBMT) and European LeukemiaNet (ELN). New recommendations were produced using a consensus-building methodology after a comprehensive review of articles released from January, 2015 to December, 2022. Seven domains and 18 key questions were selected through a series of questionnaires using a Delphi process. Key recommendations in this update include: patients with primary myelofibrosis and an intermediate-2 or high-risk Dynamic International Prognostic Scoring System score, or a high-risk Mutation-Enhanced International Prognostic Score Systems (MIPSS70 or MIPSS70-plus) score, or a low-risk or intermediate-risk Myelofibrosis Transplant Scoring System score should be considered candidates for allogeneic HSCT. All patients who are candidates for allogeneic HSCT with splenomegaly greater than 5 cm below the left costal margin or splenomegaly-related symptoms should receive a spleen-directed treatment, ideally with a JAK-inhibitor; HLA-matched sibling donors remain the preferred donor source to date. Reduced intensity conditioning and myeloablative conditioning are both valid options for patients with myelofibrosis. Regular post-transplantation driver mutation monitoring is recommended to detect and treat early relapse with donor lymphocyte infusion. In a disease where evidence-based guidance is scarce, these recommendations might help clinicians and patients in shared decision making. © 2024 Elsevier Ltd |
| Keywords: | event free survival; myelofibrosis; gene mutation; overall survival; busulfan; fludarabine; janus kinase inhibitor; neutropenia; review; hypertension; liver cirrhosis; chemotherapy; cytarabine; methodology; gene; cd34 antigen; progression free survival; liver toxicity; erythropoietin; anemia; bone marrow; thrombocytopenia; cytogenetics; cyclophosphamide; melphalan; transplantation; carmustine; stem cell; questionnaire; chronic graft versus host disease; graft failure; karnofsky performance status; prophylaxis; splenomegaly; scoring system; graft versus host reaction; donor lymphocyte infusion; allogeneic hematopoietic stem cell transplantation; delphi study; hyperbilirubinemia; idarubicin; immunosuppressive treatment; bone marrow transplantation; decision making; conceptual framework; karyotype; granulocyte colony stimulating factor; cyclosporine; azacitidine; recurrence free survival; asxl1 gene; transcription factor ezh2; isocitrate dehydrogenase 1; liver venoocclusive disease; decitabine; international prognostic scoring system; isocitrate dehydrogenase 2; momelotinib; pacritinib; ruxolitinib; clinical significance; human; fedratinib; prognostic assessment; venetoclax; srsf2 gene; splanchnic vein thrombosis; genetically inspired prognostic scoring system |
| Journal Title: | The Lancet Haematology |
| Volume: | 11 |
| Issue: | 1 |
| ISSN: | 2352-3026 |
| Publisher: | Elsevier Science, Inc. |
| Date Published: | 2024-01-01 |
| Start Page: | e62 |
| End Page: | e74 |
| Language: | English |
| DOI: | 10.1016/s2352-3026(23)00305-8 |
| PUBMED: | 38061384 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Review -- Source: Scopus |
