Treatment of Children with Severe Haemophilia A and Inhibitors: a Health Economic Evaluation for Germany

Abstract

'ITI with risk assessment' is cost-saving with comparable outcomes to HD ITI. However, patient-related factors like bleeding frequency have to determine treatment decisions in individual patients. More clinical data is needed to increase the significance of model -calculations.

The socioeconomic burden of patients affected by hemophilia with inhibitors

“…The treatment regimens used for inhibitors in hemophilia care are on demand therapy, ITI, and prophylaxis (Figure ). According to the retrieved studies (Table ), annual treatment costs of ITI and prophylaxis with bypassing agents were higher than those of on demand treatment; however, the evidences have demonstrated the long‐term benefit of prophylaxis and ITI compared to on demand treatment in patients with hemophilia and inhibitors, in preventing bleeding episodes, in improving clinical outcomes, and in reducing long‐term total costs . Therefore, the high initial costs of ITI are generally balanced against its long‐term data on the costs and efficacy .…”

“…According to the retrieved studies (Table 1), annual treatment costs of ITI and prophylaxis with bypassing agents were higher than those of on demand treatment; however, the evidences have demonstrated the long-term benefit of prophylaxis and ITI compared to on demand treatment in patients with hemophilia and inhibitors, in preventing bleeding episodes, in improving clinical outcomes, and in reducing long-term total costs. [54][55][56] Therefore, the high initial costs of ITI are generally balanced against its long-term data on the costs and efficacy. [55][56][57] In the last 40 years, various regimens using different FVIII dosages and intervals have been acknowledged to eradicate the inhibitors through different ITI regimens.…”

Making economic evaluations more helpful for treatment choices in haemophilia

“…Twenty‐one economic evaluations met our inclusion criteria and were grouped under the following topics: prophylaxis vs. treatment on‐demand (five studies) ; bypassing therapy use (six studies) ; immune tolerance induction (four studies) ; and other topics within haemophilia (six studies) . Details of the CHEERS assessments for the 15 studies discussing the three main topics are given in Appendix S2 (available online) and described below.…”

“…All four of the reviewed studies considered alternative strategies for treating patients with inhibitors . These strategies included prophylaxis or on‐demand treatment with a bypassing agent, low‐ and high‐dose immune tolerance induction (ITI) regimens and ITI treatment based on risk assessment.…”

Immunotolerance induction in a pediatric hemophilic patient in Costa Rica

“…3 Once an inhibitor develops, regular prophylaxis with FVIII needs to be discontinued and only by-passing agents can be used, which rise both on demand and prophylaxis costs and also represent a less effective haemostatic therapy. 2,8 ITI is the best way to eradicate the inhibitors permanently, to restore prophylactic FVIII therapy, to stop bleedings and thus to improve the patient's quality of life. 2,7,8 Several good prognostic factors for ITI have been described, as: 1) FVIII inhibitor titer at the beginning of therapy less than 10 BU/ml; 2) historical peak inhibitor titer less than 200 BU/ ml; 3) peak inhibitor titer during ITI less than 250 BU/ml; 4) Initiation of ITI within 5 years of FVIII inhibitor appearance.…”