Article Abstract

Role of virus inactivated cryoprecipitate in the treatment of bleeding disorders

Authors: Jean-Claude Faber


Background: Treatment of patients with bleeding disorders has significantly improved in terms of quality, safety and efficacy. Despite impressive medical and pharmaceutical progress, serious problems continue to exist with the treatment of these patients: (I) in developing countries, treatment of patients with bleeding disorders is inadequate. In total, 75% to 80% of all hemophiliacs in the world have no access to any form of effective treatment, for different reasons: needed blood products are unavailable, inaccessible and/or unaffordable. Even more striking, this situation has hardly changed over the past 20 to 30 years and remains catastrophic and unacceptable in resource limited countries; (II) although many complications resulting from hemophilia can be avoided nowadays, one major side-effect continues to exist with the use of clotting factor concentrates in hemophilia patients: the formation of inhibitors, neutralizing alloantibodies against injected coagulation factors. This potentially life-threatening complication arises frequently in previously untreated patients with severe hemophilia A (according to a recent study, in at least 25% to 35%) and poses them at serious risk.
Methods: Recently, significant developments have occurred with the potential to change the situation: blood systems (including blood centers and services) in developing countries have in general tangibly improved and novel technologies for viral inactivation (VI) of blood components are now available and marketed (using amotosalen, riboflavin, solvent-detergent, etc.).
Results: In a few countries, local preparation of virus inactivated cryoprecipitate (Cryo-VI) is used, like in Egypt where about 1/3 of the national factor VIII (FVIII) consumption is covered by Cryo-VI, and it has proven to be a safe and effective therapeutic for patients with bleeding disorders, at an affordable cost. If rolled out into developing countries, it is foreseeable that supply, availability, accessibility and affordability of therapeutic hemostatic products will sensibly improve. At the same time, inhibitor occurrence can be drastically lowered as it is well known from historical data and long-term clinical experience that only around 5% of hemophilia A patients treated with cryo develop an inhibitor. Despite these promising facts, introduction and use of Cryo-VI remains limited in the world. There are many different obstacles for the use of this simple and inexpensive technology.
Conclusions: A worldwide coordinated movement involving all stakeholders is needed to facilitate introduction, implementation and maintenance of local preparation of Cryo-VI. Cryo-VI has undoubtedly the potential to improve the existing situation by alleviating shortage or absence of needed hemostatic products in low Human Development Index (l-HDI) and by lowering the incidence of inhibitors in newly treated hemophilia A patients.