ArticleHaemophilia : the official journal of the World Federation of Hemophilia
Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels.
Article in Haemophilia : the official journal of the World Federation of Hemophilia. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
introductionValoctocogene roxaparvovec, an adeno-associated virus (AAV)-based gene therapy, enables endogenous factor VIII (FVIII) expression in patients with severe hemophilia A without the need for regular FVIII infusions. Long-term follow-up assesses durability, safety, and immune-related challenges following gene therapy.
aimEvaluation of six years of post-infusion outcomes of a patient receiving valoctocogene roxaparvovec, focusing on FVIII expression, immune response management, and adverse events (AEs).
methodsA 44-year-old male with severe hemophilia A received a single infusion of valoctocogene roxaparvovec (6 × 10
resultsPost-infusion, the patient experienced an initial ALT elevation and FVIII decline, requiring oral prednisolone (60 mg/day) at week five. ALT elevation grade 3 (peaking at 555 U/L) was treated with intravenous methyl-prednisolone (week 6), followed by oral prednisolone. To mitigate cortisone side effects, prednisolone was switched to budesonide therapy at week 19 and continued for 6 months. FVIII activity (measured with a chromogenic FVIII assay) reached a peak of 202 IU/dL at month 6 and declined subsequently to levels of 50-70 IU/dL. After six years, FVIII activity remained normal, thus eliminating prophylactic FVIII infusions and bleeding episodes. A total of 20 AEs occurred, including two serious adverse events related to ALT elevation and a traumatic acetabulum fracture.
conclusionValoctocogene roxaparvovec can offer durable FVIII expression in severe hemophilia A. This case provides valuable insights into personalised immunosuppression therapy with a favourable outcome for long-term efficacy and safety.
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