Allogeneic hematopoietic stem-cell transplantation for myelofibrosis

Abstract

Myelofibrosis is one of the Philadelphia chromosome (Ph)-negative myeloproliferative neoplasms with heterogeneous clinical course. Though many treatment options, including Janus kinase (JAK) inhibitors, have provided clinical benefits and improved survival, allogeneic hematopoietic stem-cell transplantation (AHSCT) remains the only potentially curative therapy. Considering the significant transplant-related morbidity and mortality, it is crucial to decide who to proceed to AHSCT, and when. In this review, we discuss recent updates in patient selection, prior splenectomy, conditioning regimen, donor type, molecular mutation, and other factors affecting AHSCT outcomes. Relapse is a major cause of treatment failure; we also describe recent data on minimal residual disease monitoring and management of relapse. In addition, emerging studies have reported pretransplant therapy with ruxolitinib for myelofibrosis showing favorable results, and further research is needed to explore its use in the post-transplant setting.

Advances in management of primary myelofibrosis and polycythaemia vera: Implications in clinical practice

“…[31] Hence, if a suitable donor is available, we also consider AHSCT in intermediate‐1 risk PMF patients with these adverse mutations. [30, 32]…”

“…haploidentical and umbilical cord blood), which provide an attractive alternative source of stem cells due to widespread availability and ease of stem cell procurement. [30, 32, 37, 39]…”

“…[20] Splenomegaly may negatively affect AHSCT outcomes, as it may be associated with increased engraftment failure and mortality. [20, 32, 37] The mainstay of splenomegaly treatment in PMF is the use of oral JAK inhibitors, which are able to suppress inflammatory cytokines and inhibit JAK2‐mediated haematopoietic stem cell migration. [2, 33, 40] Ruxolitinib is the first JAK inhibitor approved by the United States Food and Drug Administration (FDA) in 2011 for the treatment of patients with intermediate‐ or high‐risk myelofibrosis and by the European Medicines Agency (EMA) in 2012 for the treatment of splenomegaly and/or other symptoms in patients with myelofibrosis, irrespective of mutational status.…”

“…However, ruxolitinib may be considered in patients who relapse after AHSCT to decrease the symptom burden. [32]…”

CircRNA-016901 silencing attenuates irradiation-induced injury in bone mesenchymal stem cells via regulating the miR-1249-5p/HIPK2 axis

“…Recently, research on the treatment of blood diseases at the molecular level has received widespread attention (15)(16)(17).…”