Dr Zhou on Ongoing Investigations into the Treatment of Patients With Myelofibrosis and Anemia

Amy W. Zhou, MD, assistant professor, Department of Medicine, Division of Hematology, Washington University School of Medicine in St. Louis, discusses ongoing and planned trials that may enhance therapeutic options for patients with myelofibrosis and anemia that are currently taking place at the Washington University School of Medicine in St. Louis.

The team at Washington University School of Medicine in St. Louis is presently involved in a phase 1/2 clinical trial (NCT04455841), Zhou begins. This open-label, dose-finding study is designed to assess the efficacy and safety profile of the ALK2 inhibitor INCB000928, both as a standalone therapy and in combination with ruxolitinib (Rituxan), for patients with myelofibrosis who have symptomatic anemia or are transfusion dependent, she explains. This trial will evaluate the safety and tolerability, pharmacokinetics, pharmacodynamics, and efficacy of INCB000928.

Investigators have also launched another phase 1b/2a open-label clinical study (NCT05320198), Zhou expands. This investigation focuses on the utilization of DISC-0974, an agent with anti-hemojuvelin properties that is known to effectively mitigate hepcidin levels, in patients with myelofibrosis and anemia, she emphasizes. This trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DISC-0974 as well as categorize its effects on anemia response in participants with myelofibrosis and anemia. This novel treatment approach holds promise for patients, potentially bringing forth a new era of management strategies for this challenging disease, Zhou states.

As the horizon of hematology research broadens, Zhou underscores the importance of these ongoing trials, particularly as some of the investigational treatments are poised for potential FDA approval. These research initiatives illuminate the commitment of investigators as they further evaluate potential therapeutic options for patients grappling with myelofibrosis-related anemia, she states. These clinical trials aim to alleviate the burden of myelofibrosis and provide hope for patients seeking improved treatment outcomes for their disease, Zhou concludes.