Health-related quality of life in haemophilia patients with inhibitors and their caregivers.

Data on the health-related quality of life (HRQoL) of congenital haemophilia patients with inhibitors (CHwI) and their caregivers are limited. To understand the association between patient demo-graphics/clinical characteristics with HRQoL among CHwI patients and caregivers, a survey was developed to assess HRQoL with haemophilia-specific QoL questionnaires (HAEMO-QoL/HAEM-A-QoL). In the cross-sectional study, paper-pencil questionnaires were mailed to 261 US CHwI patients/caregivers in July 2010. Descriptive analyses were performed to characterize HRQoL by age and to identify drivers of impairment, from both patient/caregiver perspectives. HRQoL scores were transformed on a scale of 0-100, with higher scores indicating higher impairment in HRQoL. Ninety-seven respondents completed the HRQoL assessment. HRQoL impairment was higher in adult patients. In children ages 8-16 years, mean HAEMO-QoL total score was 33.8 (SD = 15.5), and 35.0 (SD = 16.1) in children ages 4-7 years; for adult patients the mean HAEM-A-QoL total score was 42.2 (SD = 14.8). Adults reported highest impairment in the 'sports/leisure' subscale (Mean = 62.5, SD = 18.7), whereas patients 8-16 years reported highest impairment in the 'physical health' subscale (Mean = 50.8, SD = 30.5).Caregivers of patients ages 4-7 years reported greatest impairment within the 'family' subscale (Mean = 55.6, SD = 19.4). Caregivers were ''considerably/very much'' bothered by their child's inhibitors and reported higher QoL impairment for their child than parents who were not bothered. Within ChwI patients, HRQoL impairments increased with age and existed across a range of physical/psychosocial domains. In addition, caregiver burden also affected the perceived HRQoL of paediatric CHwI patients. Additional research is considered necessary to further understand the support caregivers need while caring for children with CHwI.

Patient/caregiver perceived benefits and barriers to elective orthopedic surgery (EOS) in patients with congenital hemophilia with inhibitors.

OBJECTIVE: Congenital hemophilia patients with inhibitors are at greater risk for developing arthropathy and orthopedic complications compared to those without inhibitors. Elective orthopedic surgeries (EOS) may be an option for these patients and may provide long-term cost savings due to reduced bleed frequency. However, patient motivations and goals for undergoing or delaying such surgeries are not well understood. A US-based patient/caregiver survey was designed to describe inhibitor patient experiences and outcomes following EOS and to develop a comprehensive understanding of patient preferences for EOS, which are lacking in the literature. METHODS: The paper-pencil questionnaire was mailed to 261 US inhibitor patients/caregivers and included history and timing of EOS, quality-of-life (QoL) and potential benefits of and barriers to receiving EOS. Univariate/bivariate descriptive analyses were performed to characterize those with/without a history of EOS. RESULTS: For 103 subjects who responded, the mean age was 20.9 years. Approximately 25% (n = 26) of respondents underwent EOS, most commonly on the knee (21, 81%); 73.1% of surgery recipients reported the surgery improved or greatly improved their QoL based on single-item response. The highest ranked perceived benefits were less pain, fewer bleeds, and improved mobility. However, the leading concerns reported were lack of improved mobility (62.2%), fear of uncontrolled bleeding (61.3%), and surgical complications, such as blood clot (60.0%). LIMITATIONS: The study consisted of a small sample size, primarily due to the difficulty in trying to reach inhibitor patients or their caregivers, thereby restricting inferential and stratification analysis. CONCLUSIONS: QoL improved for most inhibitor patients who reported having EOS. For those considering surgery, there is optimism about the potential benefits, but realistic concerns associated with bleed control and post-op complications.