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 14, 2025, 06:19Kay Sharma: I Feel Incredibly Proud and Honored to Share the Final Agenda for The ISTH Continuing Education Forum
-
Oct 14, 2025, 06:12Murray Adams: Professor Favaloro Presented Talks On The 100-Year History of von Willebrand Disease
-
Oct 14, 2025, 06:10Samuel Nweke on Significance of MPV in a CBC in Diagnosis
-
Oct 14, 2025, 06:09Francois Delvoye: 5 Days to Go Before the Belgian Stroke Council Scientific Workshop!
-
Oct 14, 2025, 06:08David Smadja: Honored to Take Part in This Special Event for World Thrombosis Day at UM6P University
-
Oct 14, 2025, 06:13Amaliris Guerra: Driving True Innovation in Preclinical Drug Development Related to Iron-Metabolism and Inflammation is One of My Passions!
-
Oct 13, 2025, 03:32Wolfgang Miesbach: This Study from Children's Hospital of Philadelphia Provides Crucial Insights Into Elevidys
-
Oct 13, 2025, 02:43Lukas Meyer and Colleagues on Reperfusion-dependent Treatment Effects of Thrombectomy in Large Ischemic Infarcts
-
Oct 12, 2025, 10:14Hatim Lokhandwala: Landmark HI-PRO Trial Redefines VTE Treatment Guidelines
-
Oct 12, 2025, 07:28Roberta Gualtierotti: rFVIIIFc and rFIXFc Ensured Effective Haemostasis and Favourable Safety in Major Orthopaedic Surgery
-
Oct 13, 2025, 02:46Flora Peyvandi: First Proposed Blood Test for Chronic Fatigue Syndrome - What Scientists Think
-
Oct 13, 2025, 02:44Silvio Maresca: Honored to be part of this international research project - ThrombUS+
-
Oct 10, 2025, 12:25Robert Lewis: T Cells Are Wonderful - But They Can't Do Everything
-
Oct 9, 2025, 13:03Peter Graves: When the Bleeding Does Not Stop
-
Oct 8, 2025, 12:44Bobur Kholikov: Thrombosis Diagnostics with YHLO
-
Oct 14, 2025, 06:11Souad Ahmed: It Was Such A Treat to Attend the 2025 AIMS Travelling Orator Session
-
Oct 14, 2025, 06:06Tara Miller: One of My Favorite Parts of My Job Is Partnering With Passionate Teams Like ISTH
-
Oct 14, 2025, 06:05Pierre Grès: Honored to Share With You My Recent Award for Best Oral Presentation And My Experiences at The Congress of SFTH
-
Oct 14, 2025, 01:16Louise Bannon: Thank You to My Dear Friend, Justin Romanello, Who Shared His Blood Clot Survivor Story With ISTH World Thrombosis Day Campaign
-
Oct 13, 2025, 03:44Jorge Monge: It Was An Honor to Take Part in the 3rd EBA Workshop on Rare Blood Provision