Types of Therapy

Coagulation therapies include:

Plasma-derived substitution therapy. Producing plasma-derived products is a unique process in which several therapies are created from a single starting material — human plasma from highly motivated and selected donors. Plasma-derived proteins replace missing components in the blood to help individuals with bleeding disorders lead long, healthy lives.

Recombinant substitution therapy. Recombinant therapy is a type of product that involves cultures of animal cells that have been programmed to produce the coagulation factor by insertion of the human gene for that particular coagulation factor into the genome (DNA material) of the cell. The coagulation factor is harvested from the medium in which the cell culture grows.

Immune tolerance therapy (ITT). About 25% to 30% of hemophilia A patients develop inhibitory antibodies (inhibitors) toward the substituted factor VIII (FVIII). ITT covers different specialized treatment regimens of repeated, sustained administration of FVIII aiming at eliminating such inhibitory antibodies against FVIII. When successful, the patient will be tolerant to FVIII substitution and prophylaxis or on-demand therapy can be reinstituted.

Gene therapy. In gene therapy, an intact human gene coding for the defect coagulation factor is transferred into the patient’s liver cells (transfection) by use of a non-virulent viral vector. The gene makes the cells produce normal coagulation factor, and this technique has normalized the levels of factor IX (FIX) in animal models of hemophilia B. However, there is a risk that the transfected cells are eliminated from the body, that the rate of transfection is too low to generate sufficient levels of factor or that the body reacts to the vector with, for example, hepatitis-like symptoms. Until recently, only a few patients have undergone this experimental treatment.