Nathan Connell: Interesting Data in The Management of HAC Has Been Published in Blood from Benoit Guillet and Colleagues
Nathan Connell, Clinical Chief of Hematology at Brigham and Women’s Faulkner Hospital, shared on LinkedIn:
”Interesting data in the management of hemophilia A Carriers (HAC) has been published in Blood from authors Benoit Guillet and colleagues
The article, “F8 gene variants influence the response to desmopressin in hemophilia A carriers,” provides crucial insights for clinical practice regarding the use of desmopressin to correct factor VIII (FVIII) levels in this population.
This large-scale study (n=361) leveraged a population pharmacokinetic/pharmacodynamic model to uncover key factors dictating treatment efficacy:
- Genotype Matters: The study found that HAC with null F8 gene variants exhibited a significantly poorer FVIII response to desmopressin, characterized by a lower mean peak FVIII level and higher FVIII clearance, compared to those with non-null variants.
- Body Weight Influence: Patients with a body weight <35 kg also showed a diminished response, including lower FVIII peaks and a shorter duration of FVIII:C levels ≥0.8 IU/mL.
These findings underscore the need for genotype-informed and weight-considered dosing strategies when utilizing desmopressin to move us toward a more personalized approach to managing bleeding in hemophilia A carriers.
Read the full article for a detailed analysis.”
Read the full article in Blood.
Article: F8 gene variants influence the response to desmopressin in hemophilia A carriers
Authors: Benoît Guillet Corresponding Author , Roseline D’Oiron, Marc Trossaert, Bénédicte Wibaut, Brigitte Pan-Petesch, Birgit Frotscher, Fabienne Genre-Volot, Laurent Ardillon, Stéphanie Desage, Céline Falaise, Christine Biron-Andreani, Vincent Cussac, Brigitte Tardy, Yoann Huguenin, Herve Chambost, Sophie Bayart, Sabine-Marie Castet, Xavier Delavenne
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