Effect of acute bleeding on daily quality of life assessments in patients with congenital hemophilia with inhibitors and their families: Observations from the dosing observational study in hemophilia

Abstract

Introduction: Quality-of-life (QOL) assessments in frequently bleeding patients with congenital hemophilia with inhibitors and their families are confounded by preexisting arthropathy and family circumstances. Periodic QOL assessments typically made on nonbleed days may not provide complete reflections of the burden on patients/families. Aim: To evaluate the impact of bleeding episodes on patients/caregivers/families and the association between monthly QOL scores and patients' average diary experiences. Methods: Frequently bleeding inhibitor patients ( < 0.0001 for all): EuroQol five-dimensional questionnaire index, 0.66 versus 0.82; visual analogue scale health, 69.7 versus 77.4; and pain score, 4.1 versus 1.8. Bleed days also had higher (P < 0.001) proportions of days with abnormalities in family anxiety/stress (42% vs. 30%) and family activity changes (34% vs. 21%). Conclusions: Assessing the impact of hemophilia with inhibitors on patient/family QOL typically includes periodic (likely nonbleed day) evaluations reflecting baseline abnormalities. Daily assessment, however, indicated that frequent acute bleeds impair QOL beyond patient's nonbleed day baseline. New approaches are required to assess the cumulative impact of frequent acute bleeds on patients and their families. © 2012 International Society for Pharmacoeconomics and Outcomes Research (ISPOR).