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
Saty updated on the latest scientific advances in the field of Hemophilia with Hemostasis Today.
-
Oct 17, 2026, 12:21Jalen Hurts Sidelined by a Superficial Blood Clot: What to Know
-
Oct 17, 2026, 09:33Robert Lookstein: Mechanical Thrombectomy Shows Functional Benefits in Intermediate-High-Risk PE
-
Oct 17, 2026, 09:18Sarra Hmidi: Tunisia Highlights Thrombosis Prevention on World Thrombosis Day
-
Oct 17, 2026, 09:05Addressing the Hidden Burden of Bleeding Disorders in Women – Hemophilia Society Mumbai Chapter
-
Oct 17, 2026, 08:49Maria Magnusson: Karolinska Experts Explore Gene Therapy in Sickle Cell Disease and Hemophilia
-
Oct 17, 2026, 04:07Weihao Li: Global HEPATIC Registry Findings on Post-Transplant Arterial Thrombosis
-
Oct 17, 2026, 03:56Ghaith Abu-Zeinah: ASH Publishes 2026 Guidelines for Myelofibrosis Management in Blood Advances
-
Oct 17, 2026, 03:48Tristan Siokos: Why Vascular Occlusion Is More Than Just Clots – Breakdown of the 8 Pathways
-
Oct 17, 2026, 02:36Archil Jaliashvili: Oxygen Transport in Capillaries