Raj Kasthuri, MBBS, professor in the UNC Division of Hematology and director of the UNC HHT Center of Excellence, and a team of researchers published their study evaluating the safety and efficacy of pomalidomide for the treatment of Hereditary Hemorrhagic Telangiectasias in the New England Journal of Medicine.
Hereditary Hemorrhagic Telangiectasias (HHT) is an inherited disorder characterized by dysregulated angiogenesis and development of arteriovenous malformations. The most common manifestation is recurrent nose bleeds that result in development of iron deficiency anemia. HHT is the second most common inherited bleeding disorder. There are currently no approved pharmacological treatments for HHT.
Study Findings
Dr. Kasthuri was part of the team that conducted a randomized, placebo-controlled trial to evaluate the safety and efficacy of pomalidomide, an antineoplastic agent with antiangiogenic properties, for the treatment of HHT. A total of 144 patients underwent randomization with 95 patients assigned to receive pomalidomide and 49 to receive placebo. The trial was closed to enrollment in June 2023 after a planned interim analysis met a pre-specified threshold for efficacy. There was significant improvement in nosebleeds and HHT-specific quality of life in the group treated with pomalidomide at 24 weeks. Pomalidomide was well tolerated and the most common adverse events included neutropenia, constipation, and rash. This is the largest randomized controlled trial conducted thus far in HHT.
“The identification of a therapeutic agent that led to a clinically meaningful improvement in nosebleeds and quality of life is exciting and long awaited by the HHT community,” Dr. Kasthuri said.
The UNC HHT Center of Excellence was established by Dr. Kasthuri in 2011 and is one of the largest HHT centers in the country.
Read the full article at The New England Journal of Medicine.