We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
ITI choice for the optimal management of inhibitor patients - from a clinical and pharmacoeconomic perspective.
- Authors
Oldenburg, J.; Austin, S. K.; Kessler, C. M.
- Abstract
The development of alloantibody inhibitors against factor VIII ( FVIII) represents the most significant complication of haemophilia care. Inhibitors tend to develop early in the course of treatment in about 20-30% of patients with severe haemophilia who receive on-demand or prophylactic FVIII therapy. Many factors are associated with inhibitor formation, including disease severity, major FVIII gene defects, family history and non-Caucasian race, as well as age at first treatment, intensity of early treatment, use of prophylaxis and product choice. As these latter treatment-related variables are modifiable, they provide opportunity to minimize inhibitor incidence at the clinical level. Data from the Bonn Centre in Germany have indicated an overall success rate of 78% for immune tolerance induction ( ITI) therapy, with a failure rate of 15% and with some treatments either ongoing (3%) or withdrawn (4%). Similarly, data from the G- ITI study, the largest international multicentre ITI study using a single plasma-derived (pd) FVIII/von Willebrand factor ( VWF) product, have demonstrated success rates (complete and partial) in primary and rescue ITI of 87% and 74%, respectively, with 85% of poor prognosis patients achieving success. Favourable clinical results based on success rates and time to tolerization continue to be reported for use of pd FVIII/ VWF in ITI, with pd FVIII/ VWF having a particular role in patients who require rescue ITI and those with a poor prognosis for success. Data from prospective, randomized, controlled clinical studies, such as RES.I.ST (Rescue Immune Tolerance Study), are eagerly awaited. Another factor to consider with ITI therapy is cost; preliminary data from an updated decision analytic model have provided early evidence that ITI has an economic advantage compared with on-demand or prophylactic therapy.
- Subjects
HEMOPHILIA; BLOOD coagulation disorders; BLOOD diseases; PROGNOSIS; FAMILY history (Medicine); CLINICAL trials
- Publication
Haemophilia, 2014, Vol 20, Issue 6, p17
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.12466