Safety and Efficacy of Fedratinib in Patients With Primary or Secondary Myelofibrosis

Abstract

clinicaltrials.gov identifier: NCT01437787.

A phase 1/2, open-label study evaluating twice-daily administration of momelotinib in myelofibrosis

“…The mechanism by which momelotinib might contribute to sensory neuropathy has not been identified. 10,13,14 Spleen response rate by MRI at 24 weeks was similar to that which has been described for the JAK1/2 inhibitor ruxolitinib. 1,2 Additionally, momelotinib demonstrated evidence of improvement in clinically important, anemiarelated endpoints of transfusion independence and increased hemoglobin, using both 8-week criteria and the more clinically relevant 12-week criteria.…”

Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis

“…Notably, INCB039110 overall was less effective in providing spleen size reductions than ruxolitinib and some JAK2 inhibitors tested in phase III clinical trials. 11,12,15,16 Few patients in our study experienced ≥35% spleen volume reduction; however, among patients who remained on treatment, the proportion achieving ≥35% spleen volume reduction increased from week 12 to 24. Additionally, more than half of the patients treated with 200 mg twice daily and nearly half of those treated with 600 mg once daily experienced a ≥10% spleen volume reduction at week 24, which was associated with clinically meaningful symptom improvement in patients treated with ruxolitinib in COMFORT-I.…”

Myeloproliferative neoplasms: A decade of discoveries and treatment advances

“…The phase-3 study (n 5 289) compared fedratinib at two different doses (400 or 500 mg day 21 ) with placebo and confirmed the efficacy of the drug in inducing spleen (36,40, and 1%, respectively) and symptom response (36,34, and 7%) [142]. Side effects of fedratinib included anemia, gastrointestinal symptoms, and increased levels of liver transaminases, serum creatinine, and pancreatic enzymes.…”

“…The results of the phase 2 and phase 3 studies using fedratinib (a selective JAK2 inhibitor) are now published [142,143]. The phase-3 study (n 5 289) compared fedratinib at two different doses (400 or 500 mg day 21 ) with placebo and confirmed the efficacy of the drug in inducing spleen (36,40, and 1%, respectively) and symptom response (36,34, and 7%) [142].…”