Wolfgang Miesbach, Flora Peyvandi and Glenn F Pierce on Malignancy in Persons with Haemophilia
Michael Makris, Professor of Haemostasis and Thrombosis at University of Sheffield, Editor-in-Chief of Research and Practice in Thrombosis and Haemostasis, shared an insightful post on LinkedIn:
”There is an interesting discussion about the issue of malignancy in persons with haemophilia who have had gene therapy in the New England Journal this week.
The number of malignancies is increasing as more patients undergo gene therapy.
The key issues are:
a) Need for a standardised set of investigations
b) Need for advice on what tissue sample to collect and how to store them, to allow for the more specialised investigations
c) The interpretation of the results
d) Payment for these very expensive investigations. Manufacturers have funded these so far because most of the cases were seen in the clinical trials, but will they continue to fund it long term?”
Read the full article in NEJM.
Article: AAV Gene Therapy in Severe Hemophilia B
Authors: Wolfgang Miesbach, Flora Peyvandi, Glenn F Pierce
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