Michael Makris Shares Insights from Global Forum Meeting of the WFH Meeting in Montreal
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, Editor-in-Chief of RPTH Journal, shared on LinkedIn:
”FVIII inhibitors and Haemophilia A Gene Therapy.
I have just returned from the excellent Global Forum meeting of the World Federation for Hemophilia Meeting in Montreal.
This meeting is held every 2 years and discusses safety and supply issues in haemophilia.
There were 2 highly informative presentations on inhibitors and gene therapy by David Matino (Canada) and Gabriela Yamaguti-Hayakawa (Brazil).
The messages:
a) One patient developed an FVIII inhibitor after gene therapy. He was a previously treated patient (PTP) with a negative inhibitor before infusion. Initially he responded well but needed 3 courses of steroids due to transaminitis. One year after his gene therapy, he developed an FVIII inhibitor and lost expression.
b) Seven haemophilia A patients with previous inhibitors received gene therapy, and their response was the same as those without previous inhibitors. There was no inhibitor recurrence.
c) Three patients with active inhibitors received gene therapy. One cleared the inhibitor, in one the inhibitor titre went up, and in the other the titre stayed the same.
My interpretation of the above:
a) Inhibitors in PTPs are well described but very rare. Considering less than 300 haemophilia A patients have received gene therapy so far, development of an FVIII inhibitor should be considered an adverse event and mentioned during shared decision making discussions when considering the treatment.
b) A past history of an inhibitor should not be an exclusion for gene therapy, but the small number treated with gene therapy should be appreciated.
c) Gene therapy should not be considered for persons with haemophilia and inhibitors outside clinical trials.”
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