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Description:
As the care of persons with hemophilia continues to evolve, both new opportunities for managing bleeding in patients with hemophilia, and new challenges in the care of aging patients are emerging. This session will focus on new therapies for extending the half life of FVIII and FIX, as well as advances in gene therapy for hemophilia and new pharmacological advances to augment hemostasis. Lastly, we will review emerging clinical concerns affecting older patients with hemophilia.
Dr. Amy Dunn will summarize recent clinical advances with the new long acting factor products for hemophilia A and B. She will compare strategies used for extending the pharmacokinetic properties of factor VIII and IX and review the clinical data supporting their use. Dr. Dunn will provide recommendations for the use of the long acting factor products and identify their potential role in clinical care. She will also touch briefly on other FDA approved factor products, such as porcine FVIII.
Dr. Paul Monahan will discuss new therapies in the pipeline for augmenting hemostasis, including both gene therapy and new pharmacological advances. He will review the current status of gene therapy clinical trials in hemophilia, as well as novel compounds under development and in early clinical trials to treat bleeding disorders.
Dr. Suman Sood will discuss clinical challenges that are becoming increasingly common in the management of older patients with hemophilia, including cardiovascular, liver, renal disease, cancer, and joint arthropathy. She will summarize the available evidence, discuss optimal strategies for management, and highlight current challenges and areas of uncertainty.