Recombinate for hemophilia

Last updated Oct. 14, 2022, by Teresa Carvalho, MS

Fact-checked by Joana Carvalho, PhD


What is Recombinate for hemophilia?

Recombinate is a recombinant, or man-made version, of factor VIII (FVIII), prescribed to prevent and control bleeding episodes in people with hemophilia A.

It was originally developed by the Genetics Institute, in Boston, and marketed by Baxter, which is now part of Takeda Pharmaceuticals.

How does Recombinate work?

FVIII plays an important role in regulating the formation of blood clots. Hemophilia A is an inherited bleeding disorder in which genetic mutations lower the production of interfere with the function of FVIII, leading to excessive bleeding.

Recombinate is a lab-made form of FVIII that is produced by genetically engineered Chinese hamster ovary cells. It is structurally and functionally similar to naturally occurring human FVIII, and can therefore, compensate the lack of human FVIII in hemophilia A patients.

Who can take Recombinate?

The U.S. Food and Drug Administration approved Recombinate for the treatment of hemophilia A on Dec. 10, 1992. The same day, the treatment was approved by Canada’s Health Protection Branch.

Recombinate is indicated for the prevention and control of bleeding episodes in people with hemophilia A, including children of all ages. Patients also may be given Recombinate to prevent bleeding events occurring during surgery.

Who should not take Recombinate?

Recombinate should not be used if the patient is allergic to proteins of mouse, hamster, or bovine origin, or if they are allergic to any of treatment’s other constituents.

Recombinate also is not indicated to treat von Willebrand disease, another inherited bleeding disorder.

How is Recombinate administered?

Recombinate is administered by intravenous (into-the-vein) injection. The infusion can be performed at a healthcare center or at home after proper training. The medication is supplied as a dry powder that comes in five different doses, ranging from 250 to 2,000 international units (IU), in single-dose vials. The five different strengths are coded, as follows:

  • light blue, for a dosage strength of around 250 IU per vial (220–400 IU/vial)
  • pink, for a dosage strength of around 500 IU per vial (401–800 IU/vial)
  • green, for a dosage strength of around 1,000 IU per vial (801–1,240 IU/vial)
  • purple, for a dosage strength of around 1,500 IU per vial (1,241-1,800 IU/vial)
  • orange, for a dosage strength of around 2,000 IU per vial (1,801–2,400 IU/vial)

For treating bleeds, Recombinate should be given to maintain FVIII activity levels at:

  • 20 to 40 IU/dL, with dosing every 12–24 hours for one to three days until the bleed is resolved or healing has been achieved, in the case of minor bleeds.
  • 30 to 60 IU/dL, with dosing every 12–24 hours for usually three days or more until the bleed is resolved, in the case of more extensive bleeds.
  • 60 to 100 IU/dL, with dosing every 8–24 hours until the bleed is resolved, in the case of life-threatening bleeds.

For minor surgical procedures, such as tooth extraction, a single infusion of Recombinate plus additional antifibrinolytic agents (treatments that prevent the blood clots from breaking down) within one hour of the procedure is sufficient to maintain FVIII activity levels at 60 to 80 IU/dL and control bleeds in 70% of the cases.

For major surgical procedures, patients should receive Recombinate every 8–24 hours until healing is achieved to maintain FVIII activity levels at 80 to 100 IU/dL. Treatment should then be given both before and after surgery.

Recombinate should be reconstituted, or diluted, before injection, in 5 or 10 mL of sterile water using double-ended needles or a needleless transfer device called Baxject II. The maximum infusion rate is 5 or 10 mL per minute if Recombinate is dissolved with 5 or 10 mL of sterile water, respectively. The treatment should be given within three hours after reconstitution.

Vials containing the powdered product should be stored in the fridge or at room temperature. If patients choose to store vials at room temperature, these should remain at room temperature until infused. Vials should be stored in their original box and protected from light. The treatment should not be frozen.

Recombinate in clinical trials

The safety and efficacy of Recombinate was evaluated in two prospective, open-label, multicenter studies in patients with hemophilia A.

Previously treated patients

The first was a 48-month study that included 69 patients who were previously treated with plasma-derived FVIII. Patients received Recombinate as an on-demand treatment to control bleeding episodes, and also for prophylaxis, or to prevent bleeds.

Results showed Recombinate could successfully control bleeds, with a response to treatment classified as good or excellent in 91.2% of the 3,481 assessed bleeding events. The study also found Recombinate was effective at preventing bleeds in a total of 24 surgeries in 13 patients. Notably, none of the patients developed inhibitors, or neutralizing antibodies against FVIII during the study.

Previously untreated patients

The other study involved 79 previously untreated patients with hemophilia A. Patients were treated on-demand for bleeding episodes or for bleed prevention.

Results showed Recombinate was well-tolerated and had similar clinical efficacy as plasma-derived human FVIII in controlling blood loss. Treatment also was effective in controlling spontaneous bleeds, as well as bleeds occurring inside the skull and those taking place during surgical procedures.

Common side effects of Recombinate

The most common side effects related to Recombinate are:

  • chills
  • flushing
  • rash
  • nose bleeds

Allergic reactions

Recombinate contains residual amounts of bovine proteins, mouse antibodies, and hamster proteins. Patients treated with Recombinate may develop hypersensitivity, or allergies, to these proteins. Severe allergic reactions (anaphylaxis) may also occur. If these occur, Recombinate should be discontinued and patients should receive emergency treatment right away.

Development of inhibitors

FVIII inhibitors have been reported in patients treated with Recombinate, mainly in previously untreated patients and in those minimally treated. The risk of developing inhibitors is highest during the first 20 days of treatment. Inhibitors may stop Recombinate from working correctly. Therefore, patients should be closely monitored for the development of inhibitors and talk with their healthcare team right away if bleeding does not stop after treatment.

Use in pregnancy and breastfeeding

It is currently unknown if Recombinate can affect the developing fetus or pass to breast milk. Patients who are pregnant, plan to become pregnant, are breastfeeding, or plan to do so should discuss potential risks and benefits talk with their physicians.

 


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