Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia.

ABSTRACT:

Background

Hereditary hemorrhagic telangiectasia (HHT) is characterized by extensive telangiectasias and arteriovenous malformations. The primary clinical manifestation is epistaxis that results in iron-deficiency anemia and reduced health-related quality of life.

Methods

We conducted a randomized, placebo-controlled trial to evaluate the safety and efficacy of pomalidomide for the treatment of HHT. We randomly assigned patients, in a 2:1 ratio, to receive pomalidomide at a dose of 4 mg daily or matching placebo for 24 weeks. The primary outcome was the change from baseline through week 24 in the Epistaxis Severity Score (a validated bleeding score in HHT; range, 0 to 10, with higher scores indicating worse bleeding). A reduction of 0.71 points or more is considered clinical

SUBMITTER: Al-Samkari H 

PROVIDER: S-EPMC11412318 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature