Exploring Fedratinib in Rare Myeloid Neoplasms

Andrew Kuykendall, MD, an assistant member at the H. Lee Moffitt Cancer Center of University of South Florida in Tampa, FL, discusses the background, methods, and design of a phase 2, multi-institutional, investigator-initiated study (NCT05177211) which evaluated fedratinib (Inrebic) for the treatment of patients with atypical chronic myeloid leukemia (CML), chronic neutrophilic leukemia, myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN)-unclassifiable, and MDS/MPN-ring sideroblasts and thrombocytosis.

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0:10 | When we designed the study, we wanted to serve an underserved population of patients that often get excluded from clinical trials, and so we were in a bit of a unique situation as a standalone cancer center where we see a lot of these rare diseases more frequently than they are seen out in the community. We feel like we can offer something to these patients that often get excluded and not enrolled in trials because people think it would be hard to accrue.

0:41 | We designed this study specifically for patients with MDS/MPN overlap syndromes and chronic neutrophilic leukemia. We excluded patients with chronic myelomonocytic leukemia, as those are more common, and individual trials can often be conducted for that specific diagnosis. This trial included patients with MDS/MPN-unclassifiable, atypical CML, MDS/MPN with ring sideroblasts and thrombocytosis, and chronic neutrophilic leukemia. These diseases fall under the category of MDS/MPN overlap syndromes.

1:15 | Prior studies have suggested that these are not as distinct as we would like them to be; instead, they should be viewed more as a spectrum of diseases. This perspective was the rationale for including them together in this study. [For this] phase 2 trial, we enrolled patients with these diagnoses based on specific criteria. Patients needed to have either disease-related symptoms or an enlarged spleen, as we believe this is where JAK inhibitors could offer a benefit. We used a Simon two-stage design for this study. Our goal was to enroll around 25 patients, but after the first 9, we conducted an interim analysis to ensure the treatment showed some benefit. If we observed benefit in the initial group, we would proceed with enrolling the remaining patients.