Estimating the potential cost of a high dose immune tolerance induction (ITI) therapy relative to the cost of a combined therapy of a low dose ITI therapy with bypassing agent prophylaxis.

Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Original Publication: Osney Mead, Oxford, UK : Blackwell Science, [1994-

Cost-Benefit Analysis* ; Drug Costs* ; Immune Tolerance*; Factor VIII/*administration & dosage ; Hemophilia A/*drug therapy ; Hemorrhage/*prevention & control; Blood Coagulation Factor Inhibitors/immunology ; Clinical Decision-Making ; Disease Management ; Factor VIII/adverse effects ; Factor VIII/immunology ; Hemophilia A/complications ; Hemophilia A/immunology ; Humans ; Isoantibodies/immunology ; Models, Economic ; Recombinant Proteins/administration & dosage ; Recombinant Proteins/adverse effects ; Recombinant Proteins/immunology

Introduction: The International Immune Tolerance Study (I-ITI) demonstrated comparable success rates between low (FVIII 50 IU/kg/TIW) and high dose (FVIII 200 IU/kg/day) regimens. While costlier, the high dose ITI regimen achieved shorter time-to-treatment success with fewer bleeding episodes compared to the low dose ITI regimen. Adding bypassing agent prophylaxis (BAP) to a low dose ITI regimen may reduce bleeding while still being less costly than high dose ITI.
Aim and Methods: An economic model was developed to compare high dose ITI to low dose ITI with BAP. All model inputs were derived from clinical trials. The I-ITI study indicated a median time to negative inhibitor titre of 4.6 and 9.2 months and average number of bleeds/patient of 4.2 and 9.9 for the high and low dose regimens respectively. Based on the BAP trials, aPCC (85 U/kg/TIW) and rFVIIa (90 μg/kg/day) achieved a 62% and 45% reduction in bleeding frequency respectively. Cost analysis was from a US third party payer perspective and limited to drug costs. One-way, two-way and probabilistic sensitivity analyses were performed.
Results: Costs of low dose ITI with aPCC prophylaxis until negative inhibitor titre is achieved was 24.0% less compared to high dose ITI. Low dose ITI with rFVIIa prophylaxis cost 46.5% more compared to high dose ITI. Model results were robust in the majority of the sensitivity analyses.
Conclusion: A low dose ITI regimen with aPCC prophylaxis may be cost saving compared to a high dose ITI regimen with the potential to reduce morbidity by lowering the risk for breakthrough bleeds.
(© 2017 John Wiley & Sons Ltd.)

Keywords: bypassing agent; cost; factor VIII; haemophilia; immune tolerance; prophylaxis

0 (Blood Coagulation Factor Inhibitors)
0 (Isoantibodies)
0 (Recombinant Proteins)
9001-27-8 (Factor VIII)

Date Created: 20170623 Date Completed: 20180521 Latest Revision: 20180521

20250114

10.1111/hae.13294

28641362