British journal of haematology · 2021
Allogeneic haematopoietic cell transplantation for myelofibrosis: a real-life perspective.
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Savani Malvi, Dulery Rémy, Bazarbachi Abdul Hamid, Mohty Razan, Brissot Eolia, Malard Florent, Bazarbachi Ali, Nagler Arnon, Mohty Mohamad
Аннотация
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.
Предметные рубрики MeSH
AllograftsClinical Trials as TopicCombined Modality TherapyCyclophosphamideDisease ProgressionDonor SelectionHematopoiesis, ExtramedullaryHematopoietic Stem Cell TransplantationHumansJanus Kinase 2Middle AgedMutationNitrilesPremedicationPrimary MyelofibrosisPrognosisProtein Kinase InhibitorsPyrazolesPyrimidinesRecurrenceRisk AssessmentSalvage TherapySeverity of Illness IndexSplenectomyTransplantation Conditioning