Proper Dosing, Bleeding Awareness Essential Roles of Pharmacists for Hemophila A and B, Thombocytopenic Purpura Treatment

Hemophilia A and B are rare, complex bleeding disorders that often require complicated treatment management and specialized care for the patient. Medications are available to treat or ease the associated symptoms of hemophilia but are often at high price tags and require close management to ensure the safety and medication adherence of the patient.¹

Hemophilia A and B are rare blood conditions that can affect both adults and children. | Image Credit: © Fernando Cortés | stock.adobe.com

Pharmacists play an outsized role in the management of patients with hemophilia A or B due to the unpredictable nature of the disease. Bleeding can begin for no reason unexpectedly, and minor bumps or bruises can become major issues if bleeding persists for an unusually long time. Patients with hemophilia need to be consistently monitored by pharmacists as they are often the first point-of-contact for patients and can spot any notable hemophilia symptoms.²

Regarding treatment, pharmacists should be collaborating with other members of a patient care team, including providers that might be encountered in a hospital setting or members of a health system specialty pharmacy that specialize in bleeding disorder management. What treatment works best for each patient with hemophilia depends on their bleeding status and clotting factor levels, in addition to a patient’s physical and mental capacity to handle treatment, aspects that a pharmacist is in the best position to analyze.^1,2

Luckily, a variety of treatment options for both hemophilia A or B and associated blood-clotting disorders have been approved by regulatory agencies and are available for patients. Options range from replacement therapy prophylaxis to recombinant therapy, and pharmacists should be aware of this range of options to be best prepared to counsel patients with hemophilia A or B.

Eloctate (antihemophilic factor [recombinant], Fc fusion protein; Sanofi) was approved over a decade ago for the control and prevention of bleeding episodes, routine prophylaxis, and surgical management of hemophilia A in children and adults. According to investigators, it was the first recombinant therapy with hemophilia A to feature prolonged circulation in the body.³

This treatment comes with the benefit of possibly extending the interval between prophylactic infusions for patients. A major burden with many hemophilia treatments is the arduous process for administration, which could turn patients away from seeking out treatment or deter adherence. Pharmacists play an essential role in recommending treatments for patients that align with their comfortability regarding frequent infusions, and Eloctate could be a suitable option for many.³

One of the more recently approved treatments is Altuviiio, [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl; Sanofi], a von Willebrand factor (VWF) independent recombinant factor VIII therapy for patients with hemophilia A. It is designed to treat hemophilia-associated bleeding episodes as routine prophylaxis or on-demand treatment.⁴

According to Sanofi, Altuviiio is the first and only hemophilia A therapy that can provide normal to near-normal factor activity levels for most of the week with a once-weekly dosing regimen. Furthermore, the FDA updated Altuviiio’s label in May 2024, providing further study results indicating the medicine’s use for pediatric patients in addition to adults. Pharmacists can counsel patients on the robust efficacy and safety data surrounding Altuviiio when recommending the drug for patients. Given Altuviiio’s approval in pediatric patients, pharmacists can also offer the drug as an early treatment option for caregivers struggling to manage their child’s hemophilia who want to stay ahead of bleeds and reduce potential future complications.^4,5

Alprolix (Coagulation factor IX [recombinant], Fc fusion protein; Sanofi) is another effective medication that can help patients with hemophilia B control or prevent bleeding episodes. According to Alprolix’s prescribing information accessible from its website, the drug was approved by the FDA in 2014 for the on-demand treatment and control of bleeding episodes, perioperative management of bleeding, and routine prophylaxis in both adults and children.⁶

There are some important considerations for pharmacists considering Alprolix for a patient with hemophilia B. Primarily, Alprolix’s prescribing information notes it is not indicated for the induction of immune tolerance in patients with hemophilia B. Additionally, as with many treatments for hemophilia, dosing depends on the type of bleeding a patient is experiencing. Pharmacists are in a pivotal position of monitoring patient bleeding tendency to determine the correct dosing regimen for their patients.⁶

Another hematologic condition, acquired thrombotic thrombocytopenic purpura (aTTP), presents similarly to hemophilia but are distinct in their mechanisms. In aTTP, an immune-mediated deficiency of the protease ADAMTS13 allows for unrestrained adhesion of von Willebrand factor multimers to platelets and microthrombosis, resulting in thrombocytopenia and anemia. aTTP is a rare disorder, but because of its similarity to hemophilia and the prospect of both co-occurring in a patient, pharmacists must be aware of treatments if they become necessary.⁷

Caplacizumab-yhdp (Cablivi; Sanofi) injection, in combination with immunosuppressive therapy and plasma exchange, was approved by the FDA in 2019, becoming the first therapy specifically indicated for adults with aTTP. This approval allows for more targeted inhibition of the formation of blood clots in patients, while allowing for easier management and treatment with no need to endure difficult daily plasma exchange.⁸

Proper awareness of a patient’s clinical presentation and symptoms will ensure an accurate diagnosis is made if blood clotting is observed. For patients who do present with hemophilia A or B, there are a litany of treatment options available that pharmacists can educate themselves on and provide to constituents.

REFERENCES

1. Antrim A. Bleeding disorder management at a health system specialty pharmacy improves quality of life. Pharmacy Times. Published January 9, 2025. Accessed January 16, 2025. https://www.pharmacytimes.com/view/bleeding-disorder-management-at-a-health-system-specialty-pharmacy-improves-quality-of-life

2. Cleveland Clinic. Hemophilia. Last Reviewed November 14, 2022. Accessed January 16, 2025. https://my.clevelandclinic.org/health/diseases/14083-hemophilia

3. Biogen Idec. FDA approves Biogen Idec’s ELOCTATE, first hemophilia A therapy to extend the interval between prophylactic infusions, for both adults and children. News Release. Released June 6, 2014. Accessed January 16, 2025. https://www.businesswire.com/news/home/20140606005891/en/FDA-Approves-Biogen-Idec%E2%80%99s-ELOCTATE%E2%84%A2-Hemophilia-Therapy#.U5SBiC9KutB

4. PT Staff. FDA approves first-in-class factor VIII therapy for hemophilia A. Pharmacy Times. Published February 24, 2023. Accessed January 16, 2025. https://www.pharmacytimes.com/view/fda-approves-first-in-class-factor-viii-therapy-for-hemophilia-a

5. Gallagher A. FDA approves updated label for altuviiio to include fall data on pediatric patients with hemophilia A. Pharmacy Times. Published May 13, 2024. Accessed January 16, 2025. https://www.pharmacytimes.com/view/fda-approves-updated-label-for-altuviiio-to-include-full-data-on-pediatric-patients-with-hemophilia-a

6. Alprolix. How ALPROLIX works. Accessed Online January 16, 2025. https://www.alprolix.com/how-alprolix-works

7. Scully M, Cataland SR, Peyvandi F, et al. Caplacizumab treatment for acquired thrombotic thrombocytopenic purpura. N Engl J Med. 2019;380(4):335-346. doi:10.1056/NEJMoa1806311

8. FDA. FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder. News Release. Released February 6, 2019. Accessed January 16, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-treatment-adult-patients-rare-blood-clotting-disorder