The evolving treatment paradigm in myelofibrosis

Research output: Contribution to journalReview article

15 Citations (Scopus)

Abstract

Myelofibrosis (MF) is a BCR-ABL1-negative myeloproliferative neoplasm diagnosed de novo or developed from essential thrombocythemia (ET) or polycythemia vera (PV). Average survival of a patient with MF is 5-7 years. Disease complications include fatigue, early satiety, pruritus, painful splenic infarcts, infections and leukemic transformation. Allogeneic hematopoietic stem cell transplant (HSCT) is the only potentially curative option for MF, but carries a risk of treatment-related mortality and is reserved for the few high-risk patients fit enough to endure the procedure. Other traditional therapies are palliative and supported by few randomized, controlled trials; thus, novel treatment strategies are needed. Discovery of the Janus kinase 2 (JAK2) gain-of-function mutation, JAK2V617F, in the majority (50-60%) of patients with MF led to increased understanding of the biology underlying MF and the development of JAK2 inhibitors to treat MF. Recent Food and Drug Administration (FDA) approval of the first JAK2 inhibitor, ruxolitinib, signaled a new era for treatment of MF. Additional JAK2 inhibitors, such as SAR302503, may become commercially available in the near future, and their distinct pharmacologic and efficacy profiles will help determine their use across the patient population. Data on JAK2 inhibitors, their role in an evolving treatment paradigm, and future directions for treatment of MF are discussed.

Original languageEnglish (US)
Pages (from-to)242-251
Number of pages10
JournalLeukemia and Lymphoma
Volume54
Issue number2
DOIs
StatePublished - Feb 1 2013
Externally publishedYes

Fingerprint

Primary Myelofibrosis
Janus Kinase 2
Therapeutics
Splenic Infarction
Essential Thrombocythemia
Drug Approval
Polycythemia Vera
United States Food and Drug Administration
Pruritus
Hematopoietic Stem Cells
Palliative Care
Fatigue
Randomized Controlled Trials
Transplants
Mutation
Survival
Mortality
Infection

Keywords

  • Dysplasias
  • JAK2V617F
  • Janus kinase 2 (JAK2)
  • Myelofibrosis
  • Myeloid leukemias
  • Myeloproliferative disorders
  • Signaling therapies

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Cite this

The evolving treatment paradigm in myelofibrosis. / Mesa, Ruben.

In: Leukemia and Lymphoma, Vol. 54, No. 2, 01.02.2013, p. 242-251.

Research output: Contribution to journalReview article

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