Researchers at the Universitat Aut๒noma de Barcelona Teaching Unit at Vall d'Hebron Hospital have developed a therapy to treat acquired haemophilia A. The therapy is based on the use of cyclosporin, which produces less side effects than the usual treatment based on using corticosteroids.
Acquired haemophilia A is an extremely rare disease that causes the sudden appearance of antibodies against coagulation factor VIII. This is therefore an autoimmune disease. This autoantibody, or inhibitor, impedes the activity of the factor VIII that collaborates in the normal coagulation cascade, meaning the patients have various haemorrhages in different parts of the body, especially on the skin, muscles and mucosae, which can be serious or life-threatening (the mortality rate without treatment is 30%).
The immunosuppressive treatments used to eradicate these autoantibodies are based mainly on using only corticosteroids or by combining corticosteroids with cytotoxic drugs (cyclophosphamide, vincristine, azathioprine). With cytotoxic drugs, however, there are many hematological or infectious side effects.
The UAB researchers have studied a more specific immunosuppressive therapy that has fewer side effects and is more tolerated by patients. They chose to use cyclosporin, which fits this pharmacological profile and had already been effective in a few cases of acquired haemophilia that did not improve when the traditional treatments were used.
From March 2001 to October 2003, five patients at Vall d'Hebron Hospital with acquired haemophilia were assessed. As soon as they were diagnosed they were treated with oral cyclosporin (3 to 5 mg/kg/day) and a daily intravenous infusion of corticosteroids (methylprednisolone 1g/day) over a period of three days. On day four, the treatment was changed to oral corticosteroids (prednisone 1mg/kg/day).
Cyclosporin and prednisone continued to be used until there was complete remission of the disease, ie, the complete disappearance of autoantibodies against coagulation factor VIII. Complete remission, with the disappearance of the inhibitor, was achieved for all patients after an average of four weeks from the start of the treatment.
Cyclosporin tolerance was good in all patients. Only one unusual neurological side effect was observed in one patient, but this quickly disappeared once the drug was no longer administered. In two years, no reappearance of the disease has been observed.
The researchers believe their study shows that it is probably beneficial to treat the disease first using cyclosporin associated with corticosteroids. This therapeutical option should be assessed in future studies.
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