Abdul Mannan: HAVEN 7 – Zero Intracranial Hemorrhages
Abdul Mannan, Consultant Haematologist at Betsi Cadwaladr University Health Board, shared a post on LinkedIn about a recent article by Steven W Pipe et al., published in Blood, adding:
“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% 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?”
Title: Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial
Authors: Steven W Pipe, Peter Collins, Christophe Dhalluin, Gili Kenet, Christophe Schmitt, Muriel Buri, Víctor Jiménez-Yuste, Flora Peyvandi, Guy Young, Johannes Oldenburg, Maria Elisa Mancuso, Kaan Kavakli, Anna Kiialainen, Sonia Deb, Markus Niggli, Tiffany Chang, Michaela Lehle, Karin Fijnvandraat

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