Summary-We estimate the current burden of illness of osteoporosis in Canada is double ($4.6 billion) our previous estimates ($2.3 billion) due to improved data capture of the multiple encounters and services that accompany a fracture: emergency room, admissions to acute and stepdown non-acute institutions, rehabilitation, home-assisted or long-term residency support.

BackgroundTo estimate the long-term change in health related quality of life (HRQoL) following low-trauma fractures among individuals receiving home care (HC) services or living in long-term care (LTC) facilities using linked healthcare administrative data from Ontario, Canada.MethodsHRQoL was estimated using the Health Utility Index (HUI-2) with the InterRai Minimum Data Set (MDS), a mandatory questionnaire for LTC and HC in the province of Ontario (population 14 million). The HUI-2, a validated HRQoL instrument, allows the calculation of health utility where 0 represents death and 1 the best imaginable health state. For reference, the HUI-2 utility value for Canadians aged 80–84 years is 0.61 and the minimal clinically important difference is 0.03. The MDS was linked to Ontario acute care databases for fiscal years 2007–2011 to identify low-trauma fractures using ICD-10-CA codes. Regression models were used to identify predictors of change in HRQoL from pre-fracture levels to 3 years post fracture for several populations. Low-trauma fractures included hip, humerus, vertebral, wrist, multiple and other.ResultsTwenty-three thousand six-hundred fifty-five unique patients with low-trauma fractures were identified with pre- and post-fracture HRQoL assessments, of which 5057 individuals had at least 3 years of follow-up. Compared to patients receiving HC services (N = 3303), individuals residing in LTC (N = 1754) were older, taking more medications, and had more comorbidities. LTC patients had more hip fractures (49 % of total versus 29 %). For all fracture types, HRQoL decreased immediately following fracture. Although levels rebounded after the first month, HRQoL up to 36 months never returned to pre-fracture levels even for non-hip fracture. For both HC and LTC cohorts, clinically important and statistically significant decreases in HUI-2 utility scores were observed 36 months post fracture. Of the 6 HUI-2 domains, mobility had the largest impact on change in HRQoL. Regression analysis indicated that living with a musculoskeletal disorder or a neurological condition and living in LTC were associated with greater decrements in utility following a fracture.ConclusionsBased on the analysis of one of the largest studies on HRQoL to date, among individuals living in LTC facilities or receiving HC services, fractures have a significant permanent impact on HRQoL up to 3 years following fracture.Electronic supplementary materialThe online version of this article (doi:10.1186/s12877-016-0259-5) contains supplementary material, which is available to authorized users.

IntroductionThe goal of this study was to compare the efficacy in terms of Health Assessment Questionnaire change from baseline (HAQ CFB), 50% improvement in American College of Rheumatology criterion (ACR-50) and Disease Activity Score in 28 joints (DAS28) defined remission (< 2.6) between abatacept and other biologic disease modifying anti-rheumatic drugs (DMARDs) in patients with rheumatoid arthritis (RA) who have inadequate response to methotrexate (MTX-IR).MethodsA systematic literature review identified controlled trials investigating the efficacy of abatacept (three studies), etanercept (two studies), infliximab (two), adalimumab (two), certolizumab pegol (two) ritixumab (three), and tocilizumab (two) in MTX-IR patients with RA. The clinical trials included in this analysis were similar with respect to trial design, baseline patient characteristics and background therapy (MTX). The key clinical endpoints of interest were HAQ CFB, ACR-50 and DAS28 < 2.6 measured at 24 and 52 weeks. The results were analysed using network meta-analysis methods that enabled calculation of an estimate for expected relative effect of comparative treatments. Analysis results were expressed as the difference in HAQ CFB score and odds ratio (OR) of achieving an ACR-50 and DAS28 response and associated 95% credible intervals (CrI).ResultsThe analysis of HAQ CFB at 24 weeks and 52 weeks showed that abatacept in combination with MTX is expected to be more efficacious than MTX monotherapy and is expected to show a comparable efficacy relative to other biologic DMARDs in combination with MTX. Further, abatacept showed comparable ACR-50 and DAS28 < 2.6 response rates with other biologic DMARDs at 24 and 52 weeks, except for ACR-50 compared to certolizumab pegol at 52 weeks and for DAS28 < 2.6 compared to tocilizumab at 24 weeks. Sensitivity analyses confirmed the robustness of the findings.ConclusionsAbatacept in combination with MTX is expected to result in a comparable change from baseline in HAQ score and comparable ACR-50 and DAS28 < 2.6 response rates in MTX-IR patients compared to other approved biologic agents.

BackgroundHealth-related quality of life is often collected in clinical studies, and forms a cornerstone of economic evaluation. This study had two objectives, firstly to report and compare pre- and post-progression health state utilities in advanced melanoma when valued by different methods and secondly to explore the validity of progression-based health state utility modelling compared to modelling based upon time to death.MethodsUtilities were generated from the ipilimumab MDX010-20 trial (Clinicaltrials.gov Identifier: NCT00094653) using the condition-specific EORTC QLQ-C30 (via the EORTC-8D) and generic SF-36v2 (via the SF-6D) preference-based measures. Analyses by progression status and time to death were conducted on the patient-level data from the MDX010-20 trial using generalised estimating equations fitted in Stata®, and the predictive abilities of the two approaches compared.ResultsMean utility showed a decrease on disease progression in both the EORTC-8D (0.813 to 0.776) and the SF-6D (0.648 to 0.626). Whilst higher utilities were obtained using the EORTC-8D, the relative decrease in utility on progression was similar between measures. When analysed by time to death, both EORTC-8D and SF-6D showed a large decrease in utility in the 180 days prior to death (from 0.831 to 0.653 and from 0.667 to 0.544, respectively). Compared to progression status alone, the use of time to death gave similar or better estimates of the original data when used to predict patient utility in the MDX010-20 study. Including both progression status and time to death further improved model fit. Utilities seen in MDX010-20 were also broadly comparable with those seen in the literature.ConclusionsPatient-level utility data should be analysed prior to constructing economic models, as analysis solely by progression status may not capture all predictive factors of patient utility and time to death may, as death approaches, be as or more important. Additionally this study adds to the body of evidence showing that different scales lead to different health state values. Further research is needed on how different utility instruments (the SF-6D, EORTC-8D and EQ-5D) relate to each other in different disease areas.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-014-0140-1) contains supplementary material, which is available to authorized users.

Factor XIII congenital deficiency (FXIII CD) is a serious bleeding disorder resulting in a lifelong bleeding tendency, defective wound healing and recurrent miscarriage. The aim of this study was to review available literature on the burden and management of FXIII CD. To this end, Medline, Embase and Cochrane databases were searched. In current literature, FXIII CD is described as one of the most severe forms of a congenital coagulation disorder, primarily due to a high risk of severe bleeding events. The published literature suggests that over 50% of untreated FXIII CD patients experience severe bleeding symptoms. Intracranial haemorrhage (ICH)--a major cause of death and morbidity--is reported to occur in up to one-third of patients. Nonetheless, data on the social and financial burden in patients with FXIII CD are sparse. Identified reports on the effectiveness and safety of recommended treatments support that patients with FXIII CD should receive prophylactic treatment as early as possible in their lives to prevent the occurrence of bleeds, including potentially life-threatening ICHs. In conclusion, limited data on the social and economic consequences related specifically to FXIII CD have been published to date. However, it is widely acknowledged that the high risk of severe bleeds and ICH results in a high level of burden in patients with bleeding disorders. To inform future clinical decision-making and reimbursement decisions, further research is required to gain insight in how specifically FXIII CD affects quality of life and to fully understand associated economic consequences.