Michael Makris on Rising Malignancy Concerns After Hemophilia Gene Therapy: Key Challenges Highlighted in NEJM
Michael Makris, Emeritus Professor of Haemostasis and Thrombosis at the University of Sheffield, Editor-in-Chief of RPTH Journal, posted 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? ”
Stay updated with Hemostasis Today.
-
Jun 26, 2026, 18:28Daniel Pereira Monteiro: Why Is Beta Thalassaemia Major so Severe?
-
Jun 26, 2026, 18:27Pradip Pawar: Cell and Gene Therapy – Transforming Medicine in the 21st Century
-
Jun 26, 2026, 18:26Aviva Schwartz: How Can We Better Protect Patients After an Ischemic Stroke?
-
Jun 26, 2026, 18:25Does Reversing Factor Xa Inhibitors Really Carry as Much Thrombotic Risk as We Think? – RPTH Journal
-
Jun 26, 2026, 18:25Akshat Jain: Novel Therapies in Childhood Blood and Cancer Disorders
-
Jun 26, 2026, 16:57Abdul Muqtadir Abbasi: Anticoagulation in DVT Patients With Hidden Varices Requires Extreme Caution
-
Jun 26, 2026, 16:42Anesa Mulabecirovic: Why Von Willebrand Disease Takes Years to Diagnose in Women
-
Jun 26, 2026, 16:32Kaitlin Lichty: A New Identity Reflecting a Shared Legacy of Bleeding Disorders
-
Jun 26, 2026, 16:07Toong Youttananukorn: The WFH Registry Driving Better Care and Advocacy