Hemophilia

Results of a real-world study that included people with hemophilia from 33 countries show that people with hemophilia B, especially those with inhibitors, have the greatest unmet need.

Qfitlia (fitusiran) is the first therapy for both hemophilia A or B, with or without inhibitors, available in the United States.

The context matters — and it is not a matter of semantics.

The FDA recently approved the treatment by Novo Nordisk for hemophilia A or B with inhibitors, designed to prevent or reduce bleeding episodes in patients aged 12 and older.

Hympavzi is the first anti-tissue factor pathway inhibitor approved in the United States for hemophilia.

Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.

Marstacimab is being reviewed to prevent or reduce the frequency of bleeding episodes in people with hemophilia A or B. The FDA has set an action date in the first quarter of 2024.

CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.

People with moderate or severe hemophilia had worse joint status, lower pain thresholds, and poorer quality of life compared with people with mild hemophilia or healthy controls.

Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.

Expensive gene therapy has grabbed the headlines, but three promising drugs that work in the coagulation pathway are in late-stage development.

A hemophilia expert makes some comparisons between the two approved gene therapies for hemophilia.

Data from clinical trials suggest that the vast majority of the patients respond to the gene therapy for hemophilia A, says Courtney Thornburg, M.D., M.S. But there is also decreasing factor expression.

Roctavian for hemophilia A and Hemgenix for hemophilia B could spare some patients from treatment that involves intravenous infusions of clotting factors several times a week. But a hemophilia expert cautions that the high-priced gene therapies aren’t a cure and that the reprieve won’t last a lifetime.

The National Bleeding Disorders Foundation (NBDF) has joined a petition demanding that Payer Matrix stop representing itself as a patient advocacy organization.

Specialty pharmacies can help patients with hemophilia A maintain hemostasis and ensure patient safety through medication therapy management, utilizing regional care coordinators and direct pharmacist outreach, and by maintaining consistent provider communication.

Despite positive safety and quality of life results for FEIBA, the emergency of Hemlibra created difficulties for the FEIBA study and resulted in it being halted.

Recognizing the significance of patient comfort, familiarity with treatment, and established support networks is crucial for adherence if treatment switches are needed.

Ensuring that patients with hemophilia A have the necessary injection supplies, appropriately-sized syringes, and access to tracking tools can help to foster treatment adherence.

Joe Pugliese, president and CEO of the patient advocacy group Hemophilia Alliance, discusses recent announcements by manufacturers of two ultra-expensive gene therapies for hemophilia that they will offer payers partial or full reimbursement if patients fail on their therapies.

Counseling for patients with hemophilia A should include medication storage requirements, the importance of maintaining sterile injections, and administration considerations, such as how to use blunt fill needles.

Diverse factors can influence treatment decisions for patients with hemophilia A, such as the evolving landscape of insurance coverage, challenges related to vein access and medication storage, and mode of administration.

BioMarin Pharmaceutical and CSL Behring are offering valu\ed-based contracts for their hemophilia gene therapies.

The British cost-effectiveness agency said the evidence from a pivotal clinical trial wasn't strong enough to clarify Hemgenix's effectiveness relative to other therapies or satisfy concerns about its long-term durability.

University of Pittsburgh’s Margaret Ragni, M.D., M.P.H., discussed fitusiran, the investigational small interfering RNA (siRNA) therapeutic, with Managed Healthcare Executive and also some of the possible limitations of gene therapy for hemophilia.

A yearlong study of the newly approved half-life extender showed no evidence of factor VIII inhibitors.

Promising results were reported in The Lancet and The Lancet Haematology.