An Italian experience

Background: Haemophilia with inhibitors is a rare condition, which requires a substantial amount of economical and human resources representing one of the most challenging complications of haemophilia treatment. Quality of life and self-management abilities of patients are affected by this complication. In the Italian COHIBA Study haemophilia patients with inhibitors were asked about their treatment preferences, HRQoL and treatment satisfaction. Methods: HRQoL was evaluated with the Core Instrument of the haemophilia-specific Haem-A-QoL consisting of 25 items (allowing the comparison between children and adults), treatment satisfaction was assessed with the Hemo-Sat questionnaire (for adults and parents of haemophilic children) consisting of 34 items pertaining to 6 dimensions (ease & convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction). Results:In total 23 adult and 7 paediatric inhibitor patients, severely affected by haemophilia A, were enrolled from 9 Italian Haemophilia Centers. 56% received on-demand treatment, 22% prophylaxis and 22% ITI. Median age in adults was 40 years (17–60), in children 10 years (4–16). Patients and parents were mainly unsatisfied with haemophilia treatment in terms of 'efficacy' (MA= 56.25 ± 18.7; MP= 72.22 ± 11.7) and 'ease' (MA= 53.38±10.42; MP= 61.21±8.6). Adult patients showed main impairments in HRQoL in the dimensions 'sport', 'physical health' and 'future'. Whereas children were less impaired in their HRQoL than adult patients; areas of main impairments were 'sport', 'dealing' and 'view'. Conclusions: Adult patients were more impaired in their HRQoL compared to children, even though both were mainly limited in sports activities. Findings are important to understand specific problems of inhibitor patients and to improve their individual treatment