Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Ruxolitinib in Patients Who Can’t Take Hydroxyurea

Blood; 2017 Oct 12; Verstovsek, Passamonti, et al

Patients with essential thrombocythemia (ET) who do not respond to or cannot tolerate hydroxyurea can benefit from ruxolitinib treatment, according to a small open-label phase 2 study involving 39 individuals. Participants received a median dose of 30 mg/day of ruxolitinib. Among the results:

  • Treatment with a starting dose of 25 mg twice daily was linked with improved symptoms.
  • 47% of patients experienced a ≥50% improvement in bone pain at week 12.
  • Pruritus improved in 50%, night sweats in 75%, numbness/tingling in the fingers/toes in 65%, and weakness in 35%.
  • At baseline, 62% of patients were positive for the JAK2V617F mutation.
  • Minimal decrease from baseline in JAK2V617F allele burden was seen after 24 weeks of treatment.
  • Allele burden was reduced with longer exposure.
  • The most common nonhematologic adverse events during the first 8 weeks were weight increase, cough, diarrhea, and pyrexia.
  • The most common hematologic abnormalities were anemia and leukopenia.


Verstovsek S, Passamonti F, Rambaldi A, et al. Ruxolitinib for essential thrombocythemia refractory to or intolerant of hydroxyurea: Long-term phase 2 study results. Blood. 2017;130(15):1768-1771. doi:10.1182/blood-2017-02-765032.

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