The Arrival of Gene Therapy for Patients with Hemophilia A

G. Castaman, G. Di Minno, Raimondo De Cristofaro, F. Peyvandi

Risultato della ricerca: Contributo in rivistaArticolo in rivista

Abstract

Historically, the standard of care for hemophilia A has been intravenous administration of exogenous factor VIII (FVIII), either as prophylaxis or episodically. The development of emicizumab, a humanized bispecific monoclonal antibody mimicking activated FVIII, was a subsequent advance in treatment. However, both exogenous FVIII and emicizumab require repeated and lifelong administration, negatively impacting patient quality of life. A recent breakthrough has been the development of gene therapy. This allows a single intravenous treatment that could result in long-term expression of FVIII, maintenance of steady-state plasma concentrations, and minimization (or possibly elimination) of bleeding episodes for the recipient’s lifetime. Several gene therapies have been assessed in clinical trials, with positive outcomes. Valoctocogene roxaparvovec (an adeno-associated viral 5-based therapy encoding human B domain-deleted FVIII) is expected to be the first approved gene therapy in European countries, including Italy, in 2022. Some novel challenges exist including refining patient selection criteria, managing patient expectations, further elucidation of the durability and variability of transgene expression and long-term safety, and the development of standardized ‘hub and spoke’ centers to optimize and monitor this innovative treatment. Gene therapy represents a paradigm shift, and may become a new reference standard for treating patients with hemophilia A.
Lingua originaleEnglish
pagine (da-a)N/A-N/A
RivistaInternational Journal of Molecular Sciences
Volume23
DOI
Stato di pubblicazionePubblicato - 2022

Keywords

  • adeno-associated viral vector
  • emicizumab
  • valoctogene roxaparvovec
  • gene therapy
  • hemophilia A
  • exogenous factor VIII

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