Cardio-oncology & otherRandomised controlled trial
Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia
- Randomised controlled trial
- placebo-controlled
- 2:1 randomisation
- prespecified interim analysis
Authors' conclusion
Among patients with HHT, pomalidomide treatment resulted in a significant, clinically relevant reduction in epistaxis severity. No unexpected safety signals were identified.
Study context
- Population
- Patients with hereditary hemorrhagic telangiectasia (HHT)
- Participants
- 144 patients
- Comparison
- Pomalidomide vs placebo
- Pomalidomiden = 95
- Placebon = 49reference
- Primary endpoint
- Change from baseline in Epistaxis Severity Score (0–10, higher = worse bleeding; reduction ≥0.71 points clinically significant)
- Follow-up
- Through week 24
Primary result
Change from baseline in Epistaxis Severity Score · 24 weeks
Mean difference
−0.94 points
95% CI −1.57 to −0.31
P = 0.004
Pomalidomide vs Placebo
mean difference −0.94 points (95% CI −1.57 to −0.31); P = 0.004
As reported in the abstract
Secondary outcomes
HHT-specific quality-of-life score (key secondary; range 0–16, higher = more limitations) · 24 weeks
- Mean difference between the groups in the changes in HHT-specific quality-of-life score
- −1.4 points (95% CI, −2.6 to −0.3)
Safety
Adverse events
- More common with pomalidomide than placebo: neutropenia, constipation, rash
Original publication
Pomalidomide for Epistaxis in Hereditary Hemorrhagic Telangiectasia. N Engl J Med. Published 19 September 2024. doi:10.1056/nejmoa2312749
Summarised from the published abstract; every number is checked against it