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.
To evaluate the impact of bleeding episodes on patients/caregivers/families and the association between monthly QOL scores and patients' average diary experiences.
Frequently bleeding inhibitor patients (≥four bleeds in 3 months), or their caregivers, provided daily assessment of EuroQol five-dimensional questionnaire and EuroQol five-dimensional questionnaire visual analogue scale, pain (11-point Likert scale), and family anxiety/stress/activity change over 3 to 6 months. QOL scores were stratified by bleed/nonbleed days.
Patient QOL assessments were recorded by 37 of the 39 enrolled patients/caregivers (3771 of 3777 eligible dairy days, 472 bleed/3299 nonbleed days). Median (range) diary duration was 91 (66–180) days, with 8.2% (0%–72.2%) bleed days. Mean health scores were significantly worse on bleed days than on nonbleed days (P 0.001) proportions of days with abnormalities in family anxiety/stress (42% vs. 30%) and family activity changes (34% vs. 21%).
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.