Positive Outlook for Hemophilia Treatment

Mar 1, 2018
In recognition of bleeding disorders awareness month, Crystal Blankenship, PharmD, senior clinical consultant from the Accredo Bleeding Disorders Therapeutic Resource Center highlights the complexity of treating hemophilia and the outlook for new treatments on the horizon.
  • Hemophilia

Bleeding disorders such as hemophilia can be complex – and expensive – diseases to manage. Therefore, it’s critical to have a specialized medical and pharmaceutical team to help prevent episodes and manage the symptoms and therapy. Bleeding disorders are not curable at this time, but they are treatable. The experienced specialist pharmacists and nurses in the Accredo Bleeding Disorders Therapeutic Resource Center (TRC) are dedicated to counseling patients and their families. With their disease-specific expertise, they can help people with bleeding disorders such as hemophilia A or B and their caregivers manage their conditions in order to lead full, normal lives.

For example, Accredo Specialty Pharmacy's trained clinicians provide tips for patients with bleeding who plan to travel to ensure the proper treatment, storage and transportation requirements. They also offer onsite and in-school education and coaching for school staff who may need to care for a child with a bleeding disorder. One study found that patients and caregivers who received education from Accredo nurses had better symptom recognition and fewer bleeds.

Complex, Costly Treatment

Although the population of patients with hemophilia is small, cost of treatment for an individual patient can be extremely high, from tens of thousands of dollars for each monthly prescription to over a million dollars per year.

Current treatments include both recombinant and plasma-derived intravenous direct factor replacement that requires intravenous administration. Standard half-life factor products have been available in various forms for many years. In recent years, a number of extended half-life (EHL) products have been approved for both hemophilia A and B, which allows for longer intervals between IV infusions. This has had a more significant impact on the daily life of people with hemophilia B because the EHL products have extended the interval between required infusions from one to two weeks depending on the individual patient. Because patients with hemophilia A have a shorter residence time within the body, the may still require infusions several times per week.

The most recent product to be approved – Hemlibra® (emicizumab-kxwh) – is a monoclonal antibody with a novel mechanism of action, as well as subcutaneous administration, has the potential to revolutionize therapy in some patients. It is currently FDA- approved to treat people with hemophilia A and inhibitors, and is being studies in those without inhibitors. There are also several other drugs in the pipeline with novel mechanisms of action that require subcutaneous rather than intravenous administration.

An article recently published in Managed Healthcare Magazine highlights the active drug pipeline of new treatments for hemophilia. In addition to new extended half-life products, the most exciting thing on the frontier for hemophilia A and B is gene therapy. Gene therapy has the potential to significantly decrease the number of bleeds that a patient experiences or even provide an effective cure.

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