Hemophilia: Care and Treatment for Bleeding Disorders

First published July 2017
Updated September 2025

Hemophilia is not just one condition but a group of conditions caused by decreased amounts of clotting proteins. This decrease results in excessive bleeding tendencies. There are two major forms: Hemophilia A which is factor VIII deficiency and Hemophilia B which is factor IX deficiency. Both are caused by a mutation in a gene located on the X chromosome.

In most cases females carry hemophilia and males are affected by the condition. However, some females also have increased bleeding risk. Hemophilia is rare with only about 30,000 cases in the United States. Without treatment, severe bleeding episodes—especially into joints and muscles—are common.

Care is complex and patients treated in a comprehensive hemophilia treatment center (HTC) have the best results. With good care, life expectancy is nearly normal. Unfortunately, about 75% of persons affected don’t have access to expert care.

Traditional treatments include prophylactic intravenous infusions of clotting factors, mimetics or rebalancing agents, and avoiding high-risk medications and contact sports.

Several non-factor, subcutaneous (under the skin) rebalancing agent treatments have emerged:

• Fitusiran: A small interfering RNA therapy that reduces antithrombin levels. Approved by the FDA in March 2025 for hemophilia A or B, with or without inhibitors.
• Marstacimab: A monoclonal antibody targeting Tissue Factor Pathway Inhibitor (TFPI), approved in late 2024 for use in patients with hemophilia A or B without inhibitors. Trials have shown 93% reduction in annualized bleeding rates, including joint and spontaneous bleeds.
• Concizumab: Another anti-TFPI antibody approved in December 2024 (U.S. and EU), initially for patients with inhibitors. In July 2025, FDA expanded its indication to include hemophilia A or B with or without inhibitors, allowing regular administration to prevent or reduce bleeding.

These therapies represent a shift toward less invasive, patient-friendly prophylaxis options—offering more independence and improved quality of life.

Efanesoctocog alfa is a once-weekly, extended half-life recombinant factor VIII replacement therapy that continues to play a key role in traditional prophylaxis regimens by reducing infusion frequency.

Additional recommendations are Protect, Rest, Ice, Compression and Elevation (PRICE) to reduce the complications of bleeds.

Protect: Protect the affected joints or muscles. This may involve using crutches, a walker or a sling.

Rest: Rest the site of a bleed especially if it is in a joint or muscle

Ice: Apply a cool pack or ice to the injured site for 20 minutes at a time (with a 20-minute break in between), making sure to use a towel or other barrier between the ice and skin to prevent frostbite.

Compression: Apply compression with an elastic bandage to the affected area.

Elevate: If an arm or leg is hurt, place it on a pillow or folded blanket so it’s higher than the patient’s body.

Finally, don’t leave home without a plan for breakthrough bleeds. Persons with hemophilia always need to plan to have a supply of factor, or desmopressin if indicated, since only certain specialty pharmacies carry factor products. Children should have factor easy to access anytime and anywhere and should always have a bleeding action plan in place with their HTC.