Andrew Kuykendall, MD, from the Moffitt Cancer Center discusses updated data of an ongoing Phase II clinical trial assessing the safety and efficacy of PTG-300 to treat individuals with polycythemia vera. The data was presented at the American Society of Hematology (ASH) Annual Meeting.

Polycythemia vera is a rare blood disorder characterized by an increased concentration of blood cells. In most cases, polycythemia vera is acquired and predominantly affects older adults. It is associated with mutations in the JAK2 and TET2 genes. The most common treatment for polycythemia vera includes frequent phlebotomies. However, as Dr. Kuykendall explains in the video, this can exacerbate iron deficiency.

The updated data from the clinical trial assessing the safety and efficacy of PTG-300 are promising. As Dr. Kuykendall, who is the lead investigator in the study, explains, participants recruited for the study all needed frequent phlebotomies prior to treatment with PTG-300. Following treatment, the majority of patients in the trial did not require any phlebotomies and were able to maintain a target hematocrit level of less than 45%. Treatment with PTG-300 also reversed iron deficiency and reduced symptoms such as fatigue and itching. Finally, the data suggest high tolerability of PTG-300 with injection site reactions and bruise the only observed adverse effects.

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