Self Study 2023 Release II: Updates in the Management of Hemophilia: Gene Therapy for Hemophilia Management
Hemophilia is a rare inherited bleeding disorder caused by a single gene mutation, resulting in decreased production of clotting factor levels VIII (hemophilia A) and IX (hemophilia B)1. Management of hemophilia to date has focused on the replacement of the deficient clotting factor with the use of clotting factor concentrates (CFCs) or use of a bypassing agent for the treatment and prevention of bleeding which often involves frequent intravenous drug infusions. Recent approval of emicizumab, a bispecific factor activated factor IX (IXa) and factor X-directed antibody, has changed the landscape of hemophilia A by providing a subcutaneously administered agent for prophylaxis of bleeding in patients with or without factor VIII inhibitors. However, despite these treatments, breakthrough bleeding may still occur.
Gene therapy is a suitable treatment for hemophilia because it is caused by a single gene defect; efficacy of gene therapy is easily be evaluated with measurement of factor levels.
System Requirements
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Accreditation Statement
The Hematology/Oncology Pharmacy Association (HOPA) is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. An ACPE statement of credit will be issued only upon completion of the pre/post- assessment and a post-activity evaluation form.
The Hematology/Oncology Pharmacy Association (HOPA) is accredited by the Board of Pharmacy Specialties (BPS) as a provider of Board Certified Oncology Pharmacist (BCOP) credit. A BCOP statement of credit will be issued only upon completion of a post- activity evaluation form, and post-test, a passing grade of 75% or greater is achieved.
Disclosure of Conflicts of Interest
The Hematology/Oncology Pharmacy Association (HOPA) assesses conflict of interest with its instructors, planners, managers, and other individuals who are in a position to control the content of CE activities. All relevant conflicts of interest that are identified are thoroughly vetted by HOPA for fair balance, scientific objectivity of studies utilized in this activity, and patient care recommendations. HOPA is committed to providing its learners with high-quality CE activities and related materials that promote improvements of quality in healthcare and not a specific proprietary business interest of a commercial interest.
Reported Areas of Conflict
The Hematology/Oncology Pharmacy Association does not view the existence of relationships as an implication of bias or that the value of the material is decreased. The content of the activity was planned to be balanced, objective, and scientifically rigorous. Occasionally, authors may express opinions that represent their own viewpoint. Conclusions drawn by participants should be derived from objective analysis of scientific data. All faculty have indicated that they have not referenced unlabeled/unapproved uses of drugs and devices.