Allogeneic haematopoietic cell transplantation for myelofibrosis: a real‐life perspective

Abstract

Summary 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.

Novel Usefulness of Krebs von den Lungen 6 (KL-6) with Hemoglobin and Lactate Dehydrogenase for Assessing Bone Marrow Fibrosis

“…Third, somatic mutations of Janus kinases 2 (Jak2), myeloproliferative leukemia (MPL), and calreticulin (CALR) genes are known as a driver mutation of the MPN phenotype with BMF [1,2]. Novel treatments for BMF, including Jak2 inhibitors and transplantation, were introduced [31]. In this study, we did not investigate the association between the KL-6 level and Jak2, MPL, or CALR genes and could not elucidate the relevant mechanisms.…”

The application of JAK inhibitors in the peri-transplantation period of hematopoietic stem cell transplantation for myelofibrosis

“…Joanne E. Davis [45] et al observed in a preclinical mouse model that ruxolitinib-treated mice had reduced NK and CD8 + T-cells, reduced acute GVHD incidence without affecting the stem cell engrafting, prolonged survival of experimental animals, and a significant GVT effect. In a retrospective cohort study by Pu [46] et al, patients were divided into three cohorts, A (n = 3) with cytokine release syndrome (CRS) [35]. In a survey of 251 patients whose RUX therapy was interrupted conducted by Palandri [36] et al, RDS occurred in 34 patients (13.5%) at a median time of 7 days (range 2-21 days) after RUX discontinuation, among whom 21 patients had mild RDS that was manifested as splenomegaly in 13 patients (61.8%), somatic symptoms (fever, weight loss, night sweats) in 2 patients, and other MF-related symptoms (malaise, pruritus, bone pain, abdominal discomfort) in 6 patients.…”