Hope for Managing Epistaxis in Hereditary Hemorrhagic Telangiectasia

Recurrent epistaxis, a common presentation in hereditary hemorrhagic telangiectasia (HHT), can lead to anemia and reduced quality of life. To evaluate the efficacy and safety of pomalidomide for treating moderate-to-severe HHT-related epistaxis, investigators randomized 144 adults to receive either pomalidomide (4 mg daily) or placebo for 24 weeks. Eligible patients had an Epistaxis Severity Score (ESS) of at least 3 (ESS range, 0 to 10) within the prior 3 months and had anemia at screening or iron infusions or red-cell transfusions in the previous 6 months.

The pomalidomide arm had a significantly greater mean reduction in ESS at 24 weeks — the primary outcome — compared with the placebo group (−1.84 vs. −0.90 points). The pomalidomide arm had shorter duration of intensity-weighted epistaxis, fewer red-cell transfusions, lower iron infusion requirements, and significant improvements in quality of life compared with the placebo arm. Adverse effects more frequent with pomalidomide included neutropenia, constipation, and rash, but they were generally well tolerated.