Abdul Mannan: HAVEN 7 and the Future of Early Haemophilia A Care
Abdul Mannan, Consultant Haematologist at Betsi Cadwaladr University Health Board, shared a post on LinkedIn:
”The number that stayed with me from HAVEN 7 is zero.
Zero intracranial haemorrhages in 55 infants with severe haemophilia A receiving emicizumab prophylaxis.
That matters. A brain bleed in early infancy is every haemophilia team’s nightmare.
Here is what the primary analysis reported:
- 55 boys aged 12 months or younger
- Median treatment duration: about 100 weeks
- Treated bleed rate: 0.4 per year
- 54.5 percent had no treated bleeds
- Every treated bleed was traumatic
- No deaths, thromboembolic events, thrombotic microangiopathy, or anti-drug antibodies
But there is an equally important caveat.
This was an open-label, single-arm study. It describes outcomes in this cohort. It does not prove superiority over another prophylaxis strategy.
And inhibitors remain part of the conversation. Two infants developed factor VIII inhibitors after factor VIII exposure. Long-term follow-up will matter, especially for inhibitor surveillance and joint health.
Early prophylaxis is changing what childhood haemophilia care can look like. The question is no longer only “Can we prevent bleeds?” It is also “How do we monitor what remains uncertain?”
How are teams approaching early emicizumab, factor VIII exposure, and inhibitor surveillance in infants?
Source: Pipe et al. Blood 2024, HAVEN 7 primary analysis.”
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