The current state of adverse event reporting in hemophilia

ABSTRACT

Introduction: Replacement of the missing clotting factor is the mainstay of hemophilia treatment. Whilst historically many hemophilia patients were infected with blood-borne viruses transmitted via plasma-derived products, nowadays the formation of alloantibodies against the missing clotting factor is the main adverse event of treatment.

Areas covered: This paper provides an overview of the current national and international adverse event reporting systems, what these surveillance schemes taught us about side effects of the products presently in use, and elaborates on how to adapt these systems to the challenges we face with the changing treatment landscape.

Expert commentary: Treatment of inherited bleeding disorders was accompanied by severe complications in the past, resulting in major morbidity and mortality. Current products are much safer, but still require monitoring via efficient safety surveillance systems. Adverse events are reported in national and international systems. With many new products entering the market, as well as non-factor replacement therapies, new safety issues may arise. It is important to identify potential adverse events early by making surveillance systems suitable to pick up unknown or unexpected effects, and to recognize and communicate patterns of adverse events rapidly.

KEYWORDS:

• Bleeding disorders
• haemophilia
• safety
• surveillance
• concentrate

Declaration of interest

M Makris has consulted for CSL Behring, Novo Nordisk and Grifols and has been a project leader for EUHASS which receives funding from 12 pharmaceutical manufacturers. A Iorio’s institution has received project based funding via research or service agreements with Bayer, Baxalta, Biogen Idec, Grifols, NovoNordisk, Pfizer and Octapharma. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Additional information

Funding

This paper was not funded.