Advances in the Management of Myelofibrosis

Abstract

Significant advances have been made in understanding the pathophysiology of MF, leading to novel therapeutic approaches. The discovery of the JAK2 mutation and the development of JAK2 inhibitors provide clinicians with a new effective treatment option. Ruxolitinib is the first JAK1/2 inhibitor approved by the Food and Drug Administration (FDA) for the treatment of patients with intermediate- or high-risk MF. In clinical studies, ruxolitinib produced a significantly greater reduction in spleen size and improved quality of life compared with placebo or best available therapy. Several future therapies, including combination therapies with ruxolitinib, are currently under investigation.

Myeloproliferative neoplasms: trends in incidence, prevalence and survival in Norway

“…Although we do not have the result of survival among females with MF diagnosed during the period 2003–2008 due to small number of patients, it seems that the five‐year relative survival improved for patients with MF during this period. This may account for the overall slight improvement in relative survival for the total MPN population related to improved diagnostics and therapies such as use of stem cell transplantation, interferon and possible improved supportive care . Myelofibrosis remains, however, the MPN subtype with the poorest relative five‐year survival of 56.7% in females and 59.5% in males (Fig.…”

Life for patients with myelofibrosis: the physical, emotional and financial impact, collected using narrative medicine—Results from the Italian ‘Back to Life’ project

“…PMF is a rare disorder with an estimated annual incidence of approximately 1 per 100,000 and a prevalence of 4-6 per 100,000 persons in Europe [8]. It affects primarily older individuals with a median age of 67 years, when subjects may be still in their productive phase [9]. Prognosis in PMF can be stratified according to several indexes and median survival is different among risk categories, ranging from 1.5 to 14.2 years [10], but it may extend to over 20 years in low-risk patients [11].…”

Results of a phase 2 study of pacritinib (SB1518), a JAK2/JAK2(V617F) inhibitor, in patients with myelofibrosis

“…The hallmarks of MF include bone marrow fibrosis, splenomegaly, anemia and constitutional symptoms. 1 The outcome of MF varies based on the presence of certain risk factors, and survival ranges from ,2 years to .10 years. 6 The frequency of the JAK2 (V617F) mutation in patients with PMF is 50% to 60% and .95% in patients with PPV-MF.…”

“…1 The dominant gain-of-function mutation in the Janus kinase 2 (JAK2) gene in patients with MPN involves the substitution of valine for phenylalanine at position 617 (V617F). This was the first acquired, somatic mutation in the hematopoietic stem cells described in these disorders.…”