1. Allogeneic haematopoietic cell transplantation for myelofibrosis: a real-life perspective.
- Author
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Savani M, Dulery R, Bazarbachi AH, Mohty R, Brissot E, Malard F, Bazarbachi A, Nagler A, and Mohty M
- Subjects
- Allografts, Clinical Trials as Topic, Combined Modality Therapy, Cyclophosphamide administration & dosage, Cyclophosphamide therapeutic use, Disease Progression, Donor Selection, Hematopoiesis, Extramedullary, Humans, Janus Kinase 2 antagonists & inhibitors, Janus Kinase 2 genetics, Middle Aged, Mutation, Nitriles administration & dosage, Nitriles therapeutic use, Premedication, Primary Myelofibrosis drug therapy, Primary Myelofibrosis genetics, Primary Myelofibrosis surgery, Prognosis, Protein Kinase Inhibitors therapeutic use, Pyrazoles administration & dosage, Pyrazoles therapeutic use, Pyrimidines administration & dosage, Pyrimidines therapeutic use, Recurrence, Risk Assessment, Salvage Therapy, Severity of Illness Index, Splenectomy, Transplantation Conditioning methods, Hematopoietic Stem Cell Transplantation, Primary Myelofibrosis therapy
- Abstract
Myelofibrosis (MF) is a clonal stem cell neoplasm with heterogeneous clinical phenotypes and well-established molecular drivers. Allogeneic haematopoietic stem cell transplantation (HSCT) offers an important curative treatment option for primary MF and post-essential thrombocythaemia/polycythaemia vera MF or secondary MF. With a disease course that varies from indolent to highly progressive, we are now able to stratify risk of mortality through various tools including patient-related clinical characteristics as well as molecular genetic profile. Owing to the high risk of mortality and morbidity associated with HSCT for patients with myelofibrosis, it is important to improve patient selection for transplant. Our primary goal is to comprehensively define our understanding of current practices including the role of Janus Kinase (JAK) inhibitors, to present the data behind transplantation before and after leukaemic transformation, and to introduce novel personalization of MF treatment with a proposed clinical-molecular prognostic model to help elucidate a timepoint optimal for consideration of HSCT., (© 2021 British Society for Haematology and John Wiley & Sons Ltd.)
- Published
- 2021
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