Hemophilia
The main treatment for hemophilia is “factor replacement therapy,” giving or replacing the clotting factor that is too low or missing. Concentrates of the clotting factor are infused or injected directly into the bloodstream.
Replacement therapy can be used to prevent bleeding (prophylactic therapy) or to stop bleeding when it occurs (episodic therapy/acute therapy). Mild and moderate hemophilia usually only requires episodic therapy whereas severe hemophilia usually requires prophylactic therapy.
The clotting factors that are used in replacement therapy come from one of two sources: recombinant DNA or human plasma.
Recombinant DNA products: Produced in a sterile facility, these clotting factor genes are grown synthetically.
Human plasma products: Produced in a sterile facility, these clotting factors are derived from human plasma and go through viral inactivation steps in the production process to greatly decrease the risk of viral contamination.
Human plasma derived
- FEIBA
- FEIBA NF
Description
FEIBA is used to control spontaneous bleeding episodes or to cover surgical interventions in hemophilia A and hemophilia B patients with inhibitors. Typically, FEIBA is used to supplement immune tolerance induction (ITI) therapy to treat inhibitors in breakthrough bleeds. FEIBA may also be used as prophylaxis therapy in selected patients.
Dosing
The dose of FEIBA is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Chest pain, fast or irregular heartbeat, tightness in chest; difficulty breathing, wheezing, dizziness or faintness, fever or chills, skin rash, itching, pain, redness or swelling at the injection site, headache.
Recombinant (porcine sequence)
- Obizur
Recombinant (pegylated)
- Adynovate
Human plasma derived
- Hemofil M
- Koate DVI
- Monoclate-P
Recombinant
- Advate
- Afstyla
- Eloctate
- Helixate FS
- Kogenate FS
- Kovaltry
- Novoeight
- Nuwiq
- Recombinate
- Xyntha
Description
Factor VIII products are used to control and prevent bleeding episodes in adults and children (0-16 years) with Hemophilia A. Recombinant factor VIII products are the most frequently used products because it does not carry any risk of blood-borne infections. Human plasma-derived products are less frequently used but may still be the treatment of choice in selected patients.
Extended half-life Factor VIII Products
Eloctate and Adynovate are known as extended half-life factor VIII products or extended half-life factor VIII products. These products may be injected less frequently than regular factor VIII products in selected patients.
Dosing
The dose of factor VIII products is based on the patient’s body weight. The frequency of administration depends on the severity of the disease and the type of product used.
Side Effects
Allergic reactions (difficulty breathing, hives, rash, tightness in throat, swelling of tongue or wheezing), chest pain, fast or irregular heartbeat, dizziness or fainting, blurred vision, fatigue, pain, redness or swelling at the injection site, signs of viral infection (cough, fever, headache), jaundice, stomach pain, nausea, vomiting, skin flushing, numbness or tingling in hands and/or feet.
Recombinant
- Tretten
Description
Tretten is used to control spontaneous bleeding episodes or to cover surgical interventions in patients with factor XIII deficiency.
Dosing
The dose of Tretten is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Headache, pain in the extremities, injection site pain.
Human plasma-derived & Recombinant
Recombinant (albumin fusion protein)
- Idelvion
Complex
- Bebulin
- Profilnine
Description
Factor IX products are used to control and prevent bleeding episodes in adults and children (0-16 years) with Hemophilia B. Recombinant factor IX products are the most frequently used products because it does not carry the risk of blood-borne infections. Human plasma-derived products are less frequently used but may still be the treatment of choice in selected patients.
Extended half-life Factor VIII Products
Alprolix and Idelvion are known as extended half-life factor IX products. These products may be injected less frequently than regular factor IX products in selected patients.
Dosing
The dose of factor IX products is based on the patient’s body weight. The frequency of administration depends on the severity of the disease and the type of product used.
Side Effects
Chest pain, fast or irregular heartbeat, tightness in chest, difficulty breathing, wheezing, fever or chills, skin rash, itching, pain, redness or swelling at the injection site, flushing, headache, nausea, vomiting, fatigue.
Recombinant
- NovoSeven RT
Description
NovoSeven is used to control spontaneous bleeding episodes or to cover surgical interventions in hemophilia (hemophilia A, hemophilia B, and acquired hemophilia) patients with inhibitors. In addition, it can also be used to treat factor VII deficiencies. Typically, NovoSeven is used to supplement immune tolerance induction (ITI) therapy to treat inhibitors in breakthrough bleeds. NovoSeven may also be used as prophylaxis therapy in selected patients.
Dosing
The dose of NovoSeven is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Allergic reactions (difficulty breathing, hives, rash, tightness in throat, swelling of tongue or wheezing), bruising or continued bleeding, cough or signs of infection, fever or chills, pain, redness or swelling at the injection site, swelling of the legs, headache, vomiting.
Human plasma derived
- Coagadex
Description
Coagadex is used to control spontaneous bleeding episodes or to cover surgical interventions in patients with factor X deficiency.
Dosing
The dose of Coagadex is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Infusion site erythema (reddening of the skin), infusion site pain, fatigue and back pain.
Human plasma derived
- Corifact
Description
Corifact is used to control spontaneous bleeding episodes or to cover surgical interventions in patients with factor XIII deficiency.
Dosing
The dose of Corifact is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Allergic reactions (difficulty breathing, hives, rash, tightness in throat, swelling of tongue or wheezing), joint inflammation, hypersensitivity, rash, pruritus, erythema, hematoma, arthralgia, headache, elevated thrombin-antithrombin levels, and increased blood lactate dehydrogenase.
Recombinant
Description
Vonvendi is used to control spontaneous bleeding episodes or to cover surgical interventions in patients with factor XIII deficiency.
Dosing
The dose of Vonvendi is based on the patient’s body weight. The frequency of administration depends on the severity of the disease.
Side Effects
Severe itching.
Complex
- Alphanate
- Humate-P
- Wilate
Description
Factor VIII/von Willebrand factor combination products are used to control and prevent bleeding episodes in adults and children (0-16 years) with Hemophilia A or von Willebrand disease. All factor VIII/vWF combination products are human plasma derived, which may be associated with the risk of blood borne pathogens. However, recent data suggest that these products may be associated with less risk for inhibitor development.
Dosing
The dose of factor VIII/vWF combination products is based on the patient’s body weight. The frequency of administration depends on the severity of the disease and the type of product used.
Side Effects
Allergic reactions (difficulty breathing, hives, rash, tightness in throat, swelling of tongue or wheezing), and in patients undergoing surgery, postoperative wound and injection-site bleeding, and nose bleed.