2021 Update on allogeneic hematopoietic stem cell transplant for myelofibrosis: A review of current data and applications on risk stratification and management

Abstract

The number of patients with myelofibrosis (MF) undergoing an allogeneic hemopoietic stem cell transplantation (HSCT) is increasing: in the analysis of the European Group for Blood and Marrow Transplantation (EBMT) the number of MF has increased from 515 in 2014 to 748 in 2018 . This reflects the fact that HSCT is currently the only curative treatment, capable of inducing prolonged disease-free survival. Nevertheless, several problems prevent more patients from undergoing an allogeneic HSCT: we will be discussing indications for HSCT, comorbidities, splenomegaly, older age and disease phase. Donor type and stem cell source are less of a problem. Several transplant platforms exist, including different strategies for graft versus host disease (GvHD) prophylaxis, Age tailored conditioning regimens need to be implemented, to allow older and fragile patients to undergo an allogeneic HSCT.

Momelotinib for myelofibrosis: 12‐year survival data and retrospective comparison to ruxolitinib

“…Outcome of AHSCT in myelofibrosis is likely to be improved by further refinements of pre-and post-transplant measures to curb graft failure, delayed count recovery, graft versus host disease, infection risk, and disease relapse. [10][11][12][13][14] Finally, we noted that a small number (<10%) of patients on momelotinib remained on active therapy for over 10 years, without undergoing AHSCT, an observation that is intriguing and worthy of further investigation.…”

Momelotinib (JAK1/JAK2/ACVR1 inhibitor): mechanism of action, clinical trial reports, and therapeutic prospects beyond myelofibrosis

“…At present, allogeneic stem cell transplantation (ASCT) is the only treatment modality in MF with the potential to cure the disease or prolong survival. 11 In a multicenter retrospective study of 4,142 patients with myelofibrosis receiving ASCT and followed for a median of 48 months, 3-year survival, relapse, and nonrelapse mortality (NRM) rates were 58%, 22% and 29%, respectively. 12 The study showed a significant trend in terms of older age distribution (median 59.3 years) and utilization of matched unrelated donors (45.2%), in more recent times.…”

Jaktinib (JAK1/2 inhibitor): A momelotinib derivative with similar activity and optimized dosing schedule

“…At present, allogeneic hematopoietic stem cell transplant (AHSCT) remains the only treatment modality in MF that secures long-term drug-free survival, including for patients with advanced age or who lack HLA-identical sibling donor. 20,21 For patients who are unable to receive AHSCT for one reason or another, optimal palliative therapy requires attention to all three major disease complications that impact QOL: anemia, splenomegaly, and constitutional symptoms. 4 The prospect of addressing all three of these issues with one drug is appealing, but requires consideration of other factors including toxicity profile, drug access, and cost (Table 1).…”