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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