Patients with haemophilia (at least those in high-income countries) currently experience an effective and safe standard of care. The major challenge continues to lie with the one-third of patients who develop inhibitors to FVIII concentrates, as inhibitors reduce FVIII efficacy and are associated with high morbidity. A related issue concerns the high economic burden of treating patients with inhibitors, whereby the direct costs of replacement factor therapy account for nearly 99% of the total medical resources absorbed by care [27]. Immune tolerance induction therapy is the first choice of treatment in patients with inhibitors, especially those with high-responding inhibitors (Fig. 7). PD-0332991 datasheet By current standards,

success may be expected in about two-thirds of such patients who can subsequently return to their original FVIII treatment. Options available for the remaining one-third of patients include a move to bypassing

agents either as prophylaxis or on-demand. A few years ago our group attempted Inhibitor Library to analyse the economic impact of treating patients with inhibitors [28]. A brief overview of available data in this area highlights some of the issues involved in conducting an analysis of this nature (Table 4). Given that inhibitor formation is a rare complication of a rare disease, available data are limited; most stem from retrospective short-term evaluations that allow only for analysis of the direct costs of different treatment

strategies (cost-effectiveness). The data are time and region specific and 上海皓元医药股份有限公司 are therefore not directly transferable between countries. Another issue relates to the heterogeneity of the ITI therapy and bypassing agent strategies employed in various studies. The use of bypassing agents represents a lifetime decision and, as such, how does this compare economically with administering massive doses of FVIII concentrate in the hope of restoring original treatment over a 1- to 3-year period? Moreover, the introduction of orthopaedic surgery for patients with inhibitors has obviated any previous assumptions of outcomes and underscores the need for a lifetime perspective of the economic consequences of treating this patient group. A cost-utility analysis which takes into account the benefits of a given treatment/intervention on patients’ health-related quality of life is likely to be the most appropriate approach. Briefly, some pertinent findings from studies which have attempted to quantify the costs of treating patients with inhibitors are as follows: 1  Average annual concentrate costs are 1.5- to 3-fold higher in patients with inhibitors vs. those without inhibitors, although ‘outliers’ account for a high proportion of these total higher costs [29–32]. Currently ongoing in Italy is the PROFIT (PROgnostic Factors in ITI of haemophiliacs A with inhibitors) study which has the aim of establishing an optimal regimen for ITI therapy.