Updated results of the placebo-controlled, phase III JAKARTA trial of fedratinib in patients with intermediate-2 or high-risk myelofibrosis

Br J Haematol. 2021 Oct;195(2):244-248. doi: 10.1111/bjh.17727. Epub 2021 Jul 30.

Abstract

Fedratinib, an oral Janus kinase-2 (JAK2) inhibitor, reduces splenomegaly and improves symptom burden in patients with myelofibrosis. Regulatory approval of fedratinib 400-mg daily was based on results of an updated analysis of the pivotal phase III, placebo-controlled JAKARTA trial in patients with JAK-inhibitor-naïve myelofibrosis. At week 24, spleen volume response rate was 47% and symptom response rate was 40% with fedratinib 400 mg, versus 1% and 9% respectively, with placebo. Common adverse events were diarrhoea, nausea, anaemia, and vomiting. No Wernicke encephalopathy occurred in patients receiving fedratinib 400 mg/day. These updated data support use of first-line fedratinib in patients with myelofibrosis.

Keywords: JAK inhibitor; fedratinib; myelofibrosis.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Aged, 80 and over
  • Case-Control Studies
  • Follow-Up Studies
  • Humans
  • Janus Kinase 2 / antagonists & inhibitors*
  • Janus Kinase Inhibitors / administration & dosage
  • Janus Kinase Inhibitors / adverse effects
  • Janus Kinase Inhibitors / pharmacology
  • Janus Kinase Inhibitors / therapeutic use*
  • Middle Aged
  • Placebos / administration & dosage
  • Primary Myelofibrosis / complications
  • Primary Myelofibrosis / diagnosis
  • Primary Myelofibrosis / drug therapy*
  • Pyrrolidines / administration & dosage
  • Pyrrolidines / adverse effects
  • Pyrrolidines / pharmacology
  • Pyrrolidines / therapeutic use*
  • Safety
  • Spleen / drug effects
  • Splenomegaly / drug therapy
  • Sulfonamides / administration & dosage
  • Sulfonamides / adverse effects
  • Sulfonamides / pharmacology
  • Sulfonamides / therapeutic use*

Substances

  • Janus Kinase Inhibitors
  • Placebos
  • Pyrrolidines
  • Sulfonamides
  • fedratinib
  • JAK2 protein, human
  • Janus Kinase 2