New directions to develop therapies for people with hemophilia

Pooja Gogia, Michael Tarantino, Wolfgang Schramm, Louis Aledort

Research output: Contribution to journalReview articlepeer-review


Introduction: The past few decades have seen a tremendous advancement in the management of hemophilia. Whether it is improved methods to attenuate critical viruses, recombinant bioengineering with decreased immunogenicity, extended half-life replacement therapies to mitigate the burden of repeated infusion treatments, novel nonreplacement products to avoid the drawback of inhibitor development with its attractive subcutaneous administration and then the introduction of gene therapy, the management has trodden a long way. Areas covered: This expert review describes the progress in the treatment of hemophilia over the years. We discuss, in detail, the past and current therapies, their benefits, drawbacks, along with relevant studies leading to approval, efficacy and safety profile, ongoing trials, and future prospects. Expert opinion: The technological advances in the treatment of hemophilia with convenient modes of administration and innovative modalities offer a chance for a normal existence of the patients living with this disease. However, it is imperative for clinicians to be aware of the potential adverse effects and the need for further studies to establish causality or chance association of these events with novel agents. Thus, it is crucial for clinicians to engage patients and their families in informed decision-making and tailor individual concerns and necessities.

Original languageEnglish
Pages (from-to)417-433
Number of pages17
JournalExpert Review of Hematology
Issue number6
StatePublished - 2023


  • Hemophilia
  • extended halflife factor IX
  • extended halflife factor VIII
  • gene therapy
  • inhibitor
  • replacement factor IX
  • replacement factor VIII


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