Therapeutic choices for patients with hemophilia and high-titer inhibitors
β Scribed by Roshni Kulkarni; Louis M. Aledort; Erik Berntorp; Hans H. Brackman; Deborah Brown; Alan R. Cohen; Nadia P. Ewing; Alessandro Gringeri; Ralph Gruppo; Keith Hoots; Cindy Leissenger; Katheliijne Peerlinck; Man-Chiu Poon; Wing-Yen Wong
- Publisher
- John Wiley and Sons
- Year
- 2001
- Tongue
- English
- Weight
- 41 KB
- Volume
- 67
- Category
- Article
- ISSN
- 0361-8609
- DOI
- 10.1002/ajh.1123
No coin nor oath required. For personal study only.
β¦ Synopsis
Effective treatment of bleeding episodes in hemophilia with high titer inhibitors (HTI) remains a challenge, despite the fact that the therapeutic armamentarium has expanded considerably over the past few years. Treatment safety has improved with the availability of porcine factor VIII (FVIII) and bypassing products such as recombinant factor VIIa (rFVIIa), and plasma-derived activated Prothrombin Complex Concentrates (aPCCs) that are virally inactivated. The major drawbacks of rFVIIa and aPCCs are their unpredictable hemostatic effect, lack of laboratory assays to monitor efficacy and dosing frequency, and the risk of thrombosis. The proceedings of a one-day workshop of physicians who specialized in treating patients with hemophilia held in Vienna on May 13, 2000 have been summarized. In making a decision regarding the choice of product, physicians often consider the type of bleeding episode (life or limb threatening), age of the patient, volume of the reconstituted product, previous exposure to plasma derived products, cost, efficacy, and safety. For plasma naΓ―ve patients, to achieve rapid hemostasis a majority of the panelists used porcine FVIII (for patients who lack porcine inhibitory antibodies) or rFVIIa. For patients previously treated with plasma derived factors, in addition to the above concentrates, aPCCs were recommended. Although no data exists regarding safety and efficacy, switching products was routinely practiced either because of availability or cost. Furthermore, the panelists were uncertain about the efficacy of bypassing agents in the prevention of joint disease in inhibitor patients. The workshop participants felt that future research offers the best solution to resolve some of the dilemmas faced by clinicians and may help individualise treatment in a hemophilia patient with a high titer inhibitor.
π SIMILAR VOLUMES
## Abstract Achieving adequate hemostasis in patients with hemophilia and highβtiter inhibitor antibodies remains a challenge despite the development of plasmaβderived and recombinant therapies to bypass the inhibitory effect. A group of hemophilia treaters met to examine the factors that influence