Transitioning patients with severe haemophilia A from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: Real‐world clinical experience.

Klamroth, Robert; Gottstein, Saskia

This article discusses the transition of patients with severe hemophilia A from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy. Emicizumab is a routine prophylaxis for patients with severe hemophilia A, while valoctocogene roxaparvovec is a gene therapy that delivers a functional copy of the factor VIII-encoding gene to prevent bleeding. The article presents a real-world clinical experience of transitioning a patient from emicizumab to gene therapy without a washout period. The patient experienced no notable adverse events and continues to do well after the transition. The article provides additional confidence for clinicians considering transitioning their patients from emicizumab to valoctocogene roxaparvovec gene therapy.

BISPECIFIC antibodies; LOW-molecular-weight heparin; BLOOD coagulation factor VIII antibodies; THROMBOSIS; EMICIZUMAB

Haemophilia, 2024, Vol 30, Issue 5, p1247

1351-8216

Academic Journal

10.1111/hae.15086