A randomized, placebo-controlled study provides evidence that pomalidomide is an effective treatment with an acceptable safety profile for reducing nosebleed severity.
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 …
Reviewing Author
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute