Navigating the New Era of Hemophilia Care: Insights From the Pharmacy Times Peer Exchange

The management of hemophilia is undergoing a significant transformation, moving from traditional intravenous factor replacement toward a diverse landscape of subcutaneous nonfactor therapies and rebalancing agents.¹

In a recent Pharmacy Times Peer Exchange series, a panel of experts led by Robert Sidonio, MD, pediatric hematologist and medical director of the Hemophilia Treatment Center at the Aflac Cancer and Blood Disorders Center, discussed the complexities of this evolving field. Joining him were Alexis Kuhn, PharmD, BCOP pediatric oncology pharmacist at Mayo Clinic; Dana Smith, PharmD, hemophilia pharmacy manager at Fairview Health Services; Catherine Boutsikakis, PharmD, pharmacy manager at Cure 4 the Kids Foundation; and Cherelyn Karras, PharmD, director of pharmacy at the New York Blood Center. The discussion highlighted the burden of the disease, the nuances of novel therapies, and the essential role of the pharmacist in optimizing patient care.^1,2

Understanding the Spectrum of Hemophilia

Hemophilia A and B are X-linked recessive bleeding disorders characterized by deficiencies in clotting factors VIII and IX, respectively. While predominantly affecting males, with approximately 33,000 affected in the US, the experts noted a rising awareness of female patients and carriers who often experience symptomatic bleeding.^1,2

"A [patient with severe hemophilia] has very, very low [risk], less than 1%. Bleeding is often spontaneous, and it can happen without injury. Common areas of bleeds are joints, muscles, intracranial; they’re obviously very painful and can cause swelling,” Karras emphasized.³

Beyond the physical risks, the panel stressed the emotional and social toll on families. Spontaneous bleeds into joints or muscles can lead to long-term damage and chronic pain, while the diagnosis itself often alters family dynamics, sometimes requiring a caregiver to stop working.^2,4

The Financial and Clinical Burden on the Health Care System

The daily reality for patients involves a high treatment burden, particularly for those requiring frequent intravenous infusions that are time-consuming and stressful. The financial impact on the health care system is substantial, as hemophilia treatment is expensive to stock and hospital admissions for complications, such as inhibitors, can drive costs significantly higher.⁴

In the discussion, Kuhn highlighted the economic and clinical value of integrating pharmacists into the multidisciplinary care team to mitigate these costs.⁴

"There actually was a wonderful publication out of Kaiser Permanente a couple years ago where they evaluated the impact of pharmacists when added to their hemophilia treatment teams, and they found numerically lower rates of hospitalizations and [emergency department visits], but monetarily, I think it was close to $1 million in savings per year,” Kuhn noted.⁴

Evolving Therapeutic Strategies: Rebalancing Hemostasis

Traditional care has centered on factor replacement to achieve 100% factor levels, though these levels drop quickly due to the product’s half-life. The introduction of mimetics and rebalancing agents, such as anti-tissue factor pathway inhibitors (TFPIs) and small interfering RNA (siRNA), has shifted the goal toward consistent hemostasis. Fitusiran (Qfitlia; Sanofi), an siRNA targeting antithrombin, has shown a 90% reduction in annualized bleeding rates in phase 3 ATLAS trials.^1,5

Sidonio reflected on the extensive clinical data supporting the safety and efficacy of fitusiran. "When patients say, ‘Oh, I'm not sure I want to try it,’ I think we can tell them, ‘Well, this trial series went on for like 10 years. They learned a lot. They followed the patients for a long time.’”⁵

Other novel options include the TFPI inhibitors concizumab-mtci (Alhemo; Novo Nordisk) and marstacimab (Hympavzi; Pfizer). Concizumab offers treatment via a daily subcutaneous pen, which Boutsikakis described as "easy peasy, light and squeezy," whereas marstacimab provides a fixed-dose weekly option without the need for routine lab monitoring.^1,5

Managing Breakthrough Bleeds in a New Therapeutic Landscape

With the abundance of options, shared clinical decision-making is paramount to ensure the therapy aligns with a patient's lifestyle and activity levels. While prophylaxis aims to prevent joint damage, or hemarthroses, patients must still be prepared for breakthrough bleeding. Recognizing early signs, such as a tingling sensation or pain before swelling occurs, is critical for prompt treatment.^5-8

Boutsikakis emphasized the critical shift in dosing strategies required for managing acute bleeds when a patient is using a rebalancing agent. "With concizumab, they give you a nice handy-dandy chart. You're using significantly less factor and less frequency. For factor XI, for example, you're using 10 to 20 units per kg as opposed to 50 because you want to prevent complications like thromboembolic events, deep vein thrombosis, and pulmonary embolism.”⁹

Because using traditional high doses of factor alongside rebalancing agents could be “potentially catastrophic,” the panel stressed the importance of updating prescriptions and “bleeding action plans” immediately upon transitioning to a new therapy.^9,10

Safety Monitoring and the Pharmacist’s Clinical Role

Newer therapies require diligent monitoring for specific adverse events, including hepatotoxicity and thrombotic risk. For fitusiran, health care professionals must monitor liver enzymes and maintain antithrombin levels between 15% and 35%. Pharmacists serve as a bridge in this process, educating families on regimen selection and helping them recognize when “something’s not right.”^11,12

Smith summarized the holistic focus of modern individualized care, which looks beyond just factor levels to the patient's overall quality of life.⁷

"Are their thrombotic events and joints improving? Are we slowing the progression of joint disease? Is it aligning with the patient’s goals? Is that because there is shared or clinical decision-making? So, we want to make sure we're meeting their needs,” Smith said.⁷

Expert Insights on Personalized Care

As the therapeutic landscape continues to expand with exciting advancements, the focus of modern hemophilia care has shifted toward a highly personalized approach. Experts agree that therapy selection is no longer a one-size-fits-all model, but rather a dynamic process tailored to each patient’s clinical phenotype and lifestyle goals.¹¹

In this context, the pharmacist’s role remains vital, providing the specialized insights necessary to navigate these complex treatment options and ensure that every patient receives the most appropriate, individualized care.¹¹

Conclusion: Toward a Hemophilia-Free Mind

The goal of modern management is to move toward a “hemophilia-free mind” where patients can lead spontaneous, active lives without constant fear. Sidonio concluded by noting the long-term rewards of building relationships with patients as they grow and thrive.^5,7,12

“That’s part of why this job is nice—you get to see [patients] grow up. [They are] not going to live a normal life, but it’s relatively normal, and you want to see them thrive and avoid all the terrible things that could potentially happen,” Sidonio said.¹³

As new real-world data and consensus guidelines emerge, pharmacists will remain vital in navigating these complex protocols to ensure patient safety and efficacy.

Watch the full Peer Exchange series here.

REFERENCES

1. Evolving treatment strategies in hemophilia: mechanisms, benefits, and practical considerations. Pharmacy Times. January 7, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/evolving-treatment-strategies-in-hemophilia-mechanisms-benefits-and-practical-considerations

2. Understanding hemophilia A and B: essential Insights for pharmacists. Pharmacy Times. December 23, 2025. Accessed April 1, 2026. https://www.pharmacytimes.com/view/understanding-hemophilia-a-and-b-essential-insights-for-pharmacists

3. Characterizing hemophilia severity and its impact on patient outcomes. Pharmacy Times. December 23, 2025. Accessed April 1, 2026. https://www.pharmacytimes.com/view/characterizing-hemophilia-severity-and-its-impact-on-patient-outcomes

4. The burden of hemophilia and the expanding role of pharmacists in patient support. Pharmacy Times. January 7, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/the-burden-of-hemophilia-and-the-expanding-role-of-pharmacists-in-patient-support

5. Novel non-factor therapies in hemophilia: insights from phase 3 trials. Pharmacy Times. January 28, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/novel-non-factor-therapies-in-hemophilia-insights-from-phase-3-trials

6. Individualizing prophylaxis and acute management in hemophilia care. Pharmacy Times. January 14, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/individualizing-prophylaxis-and-acute-management-in-hemophilia-care

7. Real-world insights and monitoring of non-factor therapies in hemophilia. Pharmacy Times. February 4, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/real-world-insights-and-monitoring-of-non-factor-therapies-in-hemophilia

8. Recognizing and managing breakthrough bleeding in hemophilia. Pharmacy Times. January 14, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/recognizing-and-managing-breakthrough-bleeding-in-hemophilia

9. Managing breakthrough bleeding in hemophilia: strategies and considerations. Pharmacy Times. January 21, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/managing-breakthrough-bleeding-in-hemophilia-strategies-and-considerations

10. Transitioning from prophylaxis to bleed management: creating effective plans for hemophilia patients. Pharmacy Times. January 21, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/transitioning-from-prophylaxis-to-bleed-management-creating-effective-plans-for-hemophilia-patients

11. Personalized therapy selection and expert insights in modern hemophilia care. Pharmacy Times. February 11, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/personalized-therapy-selection-and-expert-insights-in-modern-hemophilia-care

12. Monitoring and managing adverse events in hemophilia prophylaxis. Pharmacy Times. February 4, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/monitoring-and-managing-adverse-events-in-hemophilia-prophylaxis

13. Assessing risks and comfort in managing breakthrough bleeding with rebalancing agents. Pharmacy Times. January 28, 2026. Accessed April 1, 2026. https://www.pharmacytimes.com/view/assessing-risks-and-comfort-in-managing-breakthrough-bleeding-with-rebalancing-agents