Alan Nurden: rFVIIa Prophylaxis May Reduce Bleeding-Related Healthcare Burden in Glanzmann Thrombasthenia
Alan Nurden, Emeritus Research Director at CNRS, Co-Founder of the French National Reference Centre for Inherited Platelet Disorders (CRPP), shared a post on LinkedIn about a recent article by Ahmad Tarawah et al, published in Blood VTH, adding:
“An excellent and much-needed paper now published in Blood VTH, examines in detail the use of rVIIa as prophylaxis in a group of 11 children with severe Glanzmann thrombasthenia in Saudi Arabia.
They show the benefits following a mean 18-month period and relate the high cost of the treatment to savings in hospitalization and blood transfusions over the treatment period.
The paper should be read together with that of Garcia-Jaen P et al who examined rFVIIa as prophylaxis in adult GT patients in Spain.
As, regrettably, gene therapy or gene repair is still not on the horizon, the search for prophylaxis for severe bleeders with GT is gaining strength.
Despite the promise of the above papers, the high cost of rVIIa will prove prohibitive in many countries and particularly for ethnic groups.
Furthermore, the need for a minimum of 3 intravenous administrations weekly will represent a heavy burden for the patients and their families.
The potential for twice monthly subcutaneous injections of the Hemab bivalent monoclonal antibody, sutacimig, that recruits endogenous FVIIa to the sites of vascular injury through its binding to TLT-1 of activated platelets remains if safety concerns can be met.
With these potential advances, I hope that pharmaceutical companies will make every effort to ensure that all GT patients can profit from the new treatments no matter where they live.”
Title: Clinical and economic impact of rFVIIa prophylaxis in Glanzmann thrombasthenia: cohort analysis in Saudi Arabia
Authors: Ahmad Tarawah, Sami Albattat, Ayman Shahba, Mohamed Nashat, Sarah Brighton, Emily Back, Sam Bristow, George Morgan
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