https://orcid.org/0000-0001-6013-1254
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ABSTRACT
Introduction
Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.
Areas covered
This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II–III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.
Expert opinion
Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.
Funding
This paper was not funded.
Abbreviation table
| FVIII | = | Factor VIII |
| PWH | = | Patients with hemophilia A |
| HCV | = | Hepatitis C virus |
| HIV | = | Human immunodeficiency virus |
| pdFVIII | = | Plasma derived FVIII |
| rFVIII | = | Recombinant FVIII |
| PUP | = | Previously untreated patients |
| QOL | = | Quality of life |
| BPA | = | Bypassing agents |
| aPCC | = | Activated prothrombin complex concentrate |
| rFVIIa | = | Activated recombinant factor VII |
| VWF | = | von Willebrand factor |
| ITI | = | Immune tolerance induction |
| BDD | = | B-domain deletion |
| CVV | = | Central venous catheter |
| EHL FVIII | = | Extended half-life FVIII |
| SHL FVIII | = | Standard half-life FVIII |
| FDA | = | Food and Drug Administration |
| EMA | = | European Medicines Agency |
| ABR | = | Annualized bleeding rate |
| siRNA | = | Small interfering RNA |
| AAV | = | Adeno-associated virus |
| PLC | = | Human placental cells |
Declaration of interest
G Young has received consulting fees and honoraria from BioMarin, Bioverativ/Sanofi, CSL Behring, Freeline, Genentech/Roche, Grifols, Novo Nordisk, Spark, Takeda, and UniQure and research grants from Grifols, Genentech and Takeda. 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.