The only potentially curative therapy for Myelofibrosis (MF) is hematopoietic stem cell transplantation (HSCT), applicable only to patients with a compatible donor, and of suitable age and functional status to withstand treatment-related toxicity. The JAK inhibitor ruxolitinib has been the mainstay of MF treatment since it was approved in 2011, and until recently was the only targeted agent approved by the US Food & Drug Administration for management of MPNs. The recent addition of a novel JAK inhibitor, the JAK2-selective fedratinib, is a significant milestone in the therapeutic landscape of MF.
Despite this critical advancement, MF management is suboptimal, owing to a lack of clinician awareness of the substantial symptom burden and quality-of-life impact of MF. The development of individualized care concepts have changed with increasing understanding of the role of HSCT, JAK inhibitors, and other patient-/disease-specific factors in the treatment paradigm for MF.
This activity will review recent advances and critical concepts that affect outcomes, including symptom burden, molecular diagnostics, prognostic risk stratification scores, and the treatment and management of MF.