The current state of adverse event reporting in hemophilia

Abstract

Text word count: 3827; abstract word count: 187; number of tables: 5; number of references: 73.2 Abstract Introduction: Replacement of the missing clotting factor is the mainstay of haemophilia treatment. Whilst historically many haemophilia 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.

Establishment of a framework for assessing mortality in persons with congenital hemophilia A and its application to an adverse event reporting database

“…Thrombotic complications have been reported in PwcHA using clotting factor concentrates and BPAs 32,33 . For example, myocardial infarction (MI) and pulmonary embolism (PE) have been observed in PwcHA taking either recombinant FVIII, aPCC, or rFVIIa 34‐37 and more recently these complications have been observed in those using non‐factor substitution treatments 27 .…”