Dr. Hoffman on Advantages of Self-Administration With PTG-300 in Polycythemia Vera

Ronald Hoffman, MD, professor of medicine, hematology, and medical oncology and director of the Myeloproliferative Diseases Program at The Icahn School of Medicine at Mount Sinai, discusses the advantages of self-administration with the investigational hepcidin mimetic PTG-300 for patients with polycythemia vera (PV).

PTG-300 is being evaluated in an ongoing phase 2 clinical trial (NCT04057040). Preliminary data have shown encouraging clinical activity and a tolerable safety profile in patients with PV.

The first-in-class agent is to be self-administered by patients subcutaneously, says Hoffman. Conventional treatment of patients with PV consists of repeated phlebotomies to maintain a hematocrit level below 45%.

Phlebotomies can cause elderly patients to become syncopal or exacerbate congestive heart failure, explains Hoffman. Additionally, the treatment can be frightening or traumatic for some patients.

Between phlebotomies, patients’ hematocrit levels are likely elevated, Hoffman adds. However, this often goes undetected as continuous testing is not currently implemented.

As such, the self-administration of PTG-300 may provide sustained hematocrit control, reduction of long-term thrombotic events, and systemic symptom relief. Additionally, patients may have improved satisfaction with the agent as self-administering medication will provide them more independence, concludes Hoffman.