Rare Disease - Episode 10

Episode 10 - Treating Mild and Severe Hemophilia

During this segment, Patrick F. Fogarty, MD, discusses the treatment goals for hemophilia, based on disease severity.

Dr Fogarty defines prophylaxis, explains primary and secondary prophylaxis for the treatment of mild and severe hemophilia, and discusses the recommended target trough levels and dosage requirements based on disease severity.

He continues by discussing how treatment expectations are similar between patients, their family members, and providers. From a pediatric standpoint, Dr Fogarty stresses the importance of providing patients the best opportunity for a normal lifestyle, and explains that as longer-acting proteins continue to develop, there is an opportunity to administer intravenous therapy less frequently.

Dr Fogarty concludes by briefly discussing the benefits of the agents that are currently available to treat patients with mild and severe hemophilia, and cites a recent study that treated patients with a regimen of recombinant clotting factor 8 concentrate and revealed positive results on patients’ quality of life.