Health-related quality of life and a cost-utility simulation of adults in the UK with osteogenesis imperfecta, X-linked hypophosphatemia and fibrous dysplasia.

BACKGROUND: Health-related quality of life of adults with osteogenesis imperfecta (OI), fibrous dysplasia (FD) and X-linked hypophosphatemia (XLH) remains poorly described. The aim of this study was to describe the HRQoL of adults with osteogenesis imperfecta, fibrous dysplasia and X-linked hypophophataemia and perform a cost-utility simulation to calculate the maximum cost that a health care system would be willing to pay for a hypothetical treatment of a rare bone disease. RESULTS: Participants completed the EQ-5D-5 L questionnaire between September 2014 and March 2016. For the economic simulation, we considered a hypothetical treatment that would be applied to OI participants in the lower tertile of the health utility score. A total of 109 study participants fully completed the EQ-5D-5 L questionnaire (response rate 63%). Pain/discomfort was the most problematic domain for participants with all three diseases (FD 31%, XLH 25%, OI 16%). The economic simulation identified an expected treatment impact of +2.5 QALYs gained per person during the 10-year period, which led to a willing to pay of £14,355 annually for a health care system willing to pay up to £50,000 for each additional QALY gained by an intervention. CONCLUSIONS: This is the first study to quantitatively measure and compare the HRQoL of adults with OI, FD and XLH and the first to use such data to conduct an economic simulation leading to healthcare system willingness-to-pay estimates for treatment of musculoskeletal rare diseases at various cost-effectiveness thresholds

A structural equation analysis on the relationship between maternal health services utilization and newborn health outcomes: a cross-sectional study in Eastern Uganda

Abstract Background Neonatal and maternal health services have a bearing on neonatal mortality. Direct and indirect factors affecting neonatal health outcomes therefore require understanding to enable well-targeted interventions. This study, therefore, assessed the interrelationship between newborn health outcomes and maternal service utilization factors. Methods We investigated maternal health utilization factors using health facility delivery and at least four Antenatal Care (ANC) visits; and newborn health outcomes using newborn death and low birth weight (LBW). We used data from a household cross-sectional survey that was conducted in 2015 in Kamuli, Pallisa and Kibuku districts. We interviewed 1946 women who had delivered in the last 12 months. The four interrelated (Endogenous) outcomes were ANC attendance, health facility delivery, newborn death, and LBW. We performed analysis using a structural equation modeling technique. Results A history of newborn death (aOR = 12.64, 95% CI 5.31–30.10) and birth of a LBW baby (aOR = 3.51, 95% CI 1.48–8.37) were directly related to increased odds of newborn death. Factors that reduced the odds of LBW as a mediating factor for newborn death were ANC fourth time attendance (aOR = 0.62, 95% CI 0.45–0.85), having post-primary level education (aOR = 0.68, 95% CI 0.46–0.98) compared to none and being gravida three (aOR = 0.49, 95% CI 0.26–0.94) compared to being gravida one. Mother’s age group, 20–24 (aOR = 0.24, 95% CI 0.08–0.75) and 25–29 years (aOR = 0.20, 95% CI 0.05–0.86) compared to 15–19 years was also associated with reduced odds of LBW. Additionally, ANC visits during the first trimester (aOR = 2.04, 95% CI 1.79–2.34), and village health teams (VHTs) visits while pregnant (aOR = 1.14, 95% CI 1.01–1.30) were associated with increased odds of at least four ANC visits, which is a mediating factor for health facility delivery, LBW and newborn death. Surprisingly, newborn death was not significantly different between health facility and community deliveries. Conclusions Attending ANC at least four times was a mediating factor for reduced newborn death and low birth weight. Interventions in maternal health and newborn health should focus on factors that increase ANC fourth time attendance and those that reduce LBW especially in resource-limited settings. Targeting women with high-risk pregnancies is also crucial for reducing newborn deaths