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.
OBJECTIVE:Congenital hemophiliapatients 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.