Pomalidomide Significantly Reduces Epistaxis Severity in HHT Patients, Study Finds

Results

• At baseline for all patients the mean Epistaxis Severity Score was 5.0 ± 1.5, denoting moderate to severe nosebleeds.

• At week 24, the mean change in Epistaxis Severity Score was -0.94 points for the pomalidomide group compared with the placebo group (95% CI, −1.57 to −0.31; P=0.004), crossing the clinically significant threshold of 0.71 points.

• Compared with the placebo group, the mean difference in HHT-specific quality of life between baseline and end was −1.4 points (95% CI, −2.6 to −0.3), suggesting improvement in daily functioning and reduction of disease-related limitations.

• Neutropenia, constipation, and rash were more common in the pomalidomide group, but no new safety signals emerged.

Pomalidomide markedly decreased the severity of epistaxis, a HHT, on the criterion of clinical significance for more than half of those affected. The treatment also seemed to improve quality of life, and safety. Therefore, pomalidomide could be a promising therapeutic intervention in HHT, but more studies are needed to adequately describe its long-term benefits and safety across the larger populations of affected individuals.

Reference:

Al-Samkari, H., Kasthuri, R. S., Iyer, V. N., Pishko, A. M., Decker, J. E., Weiss, C. R., Whitehead, K. J., Conrad, M. B., Zumberg, M. S., Zhou, J. Y., Parambil, J., Marsh, D., Clancy, M., Bradley, L., Wisniewski, L., Carper, B. A., Thomas, S. M., & McCrae, K. R. (2024). Pomalidomide for epistaxis in hereditary hemorrhagic telangiectasia. The New England Journal of Medicine, 391(11), 1015–1027. https://doi.org/10.1056/nejmoa2312749