Fedratinib can reduce symptoms and spleen volume in patients with myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPNs), according to research presented at the ASH Annual Meeting 2024.
Researchers are evaluating fedratinib, a JAK2 inhibitor, in a phase 2 trial. The trial (NCT05177211) enrolled 25 patients with atypical chronic myeloid leukemia (n=6), chronic neutrophilic leukemia (n=5), MDS/MPN-unclassifiable (n=8), and MDS/MPN with ring sideroblasts and thrombocytosis (n=6).
At baseline, the median patient age was 68.8 (range, 39.9-84.7) years, and the median time from diagnosis to treatment was 7.1 months. Most patients had splenomegaly (83%), and the median MPN-Symptom Assessment Form Total Symptom Score was 21 (range, 1-73). Prior treatments included hydroxyurea (36%), ruxolitinib (20%), luspatercept (8%), and hypomethylating agents (12%). Patients had a median of 3 pathogenic mutations.
“Most of these patients had multiple mutations, and most had a signaling mutation, an epigenetic mutation, and a splicing mutation,” said study presenter Andrew Kuykendall, MD, of the Moffitt Cancer Center in Tampa, Florida.
The patients received fedratinib at a dose of 400 mg daily in 28-day cycles. They could continue on treatment as long as they had a clinical benefit. At last follow-up, 11 patients were still on study treatment.
The median duration of fedratinib treatment was 10.8 months, and 21 patients were evaluable for efficacy at 24 weeks. Three patients discontinued fedratinib prior to 24 weeks for reasons unrelated to toxicity or lack of efficacy (eg, cost) and were considered non-responders.