The current state of adverse event reporting in hemophilia

Expert Rev Hematol. 2017 Feb;10(2):161-168. doi: 10.1080/17474086.2017.1272410. Epub 2016 Dec 26.

Abstract

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; concentrate; haemophilia; safety; surveillance.

Publication types

  • Review

MeSH terms

  • Drug Hypersensitivity / etiology
  • Drug-Related Side Effects and Adverse Reactions*
  • Factor IX / adverse effects
  • Factor IX / immunology
  • Factor IX / therapeutic use
  • Factor VIII / adverse effects
  • Factor VIII / immunology
  • Factor VIII / therapeutic use
  • Hemophilia A / complications*
  • Hemophilia A / drug therapy
  • Hemophilia B / complications*
  • Hemophilia B / drug therapy
  • Humans
  • Isoantibodies / immunology
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / immunology
  • Recombinant Proteins / therapeutic use
  • Virus Diseases / etiology
  • Virus Diseases / transmission

Substances

  • Isoantibodies
  • Recombinant Proteins
  • Factor VIII
  • Factor IX