Bone Abstracts

Objective: The potential for clinical trials to impact patient care may be limited if the outcomes reported vary by trial and lack direct relevance to patients. We aimed to systematically assess the scope and consistency of outcomes reported in randomised trials of interventions for children with osteogenesis imperfecta.

Methods: We systematically searched for all published and unpublished randomised trials of interventions for children with osteogenesis imperfecta. Trials including adults were excluded but there were no other exclusion criteria. We extracted and analysed the frequency and characteristics of the outcome domains and measures.

Results: From 21 publications of 19 trials with 857 children included, 1782 different measurements of 60 different outcome domains were reported. There was a median of 14 outcome domains reported per trial (interquartile range 12 to 17). Overall, 33 domains (55%) were surrogate e.g. serum calcium, 23 (38%) were clinical e.g. vertebral fracture, and 4 (7%) were patient reported e.g. pain. From all outcome measures assessed, bone density (288 or 16%, 16 trials) and bone turnover markers (122 or 7%, 14 trials) were the two most commonly reported, while pain (32 or 2%, 6 trials) and fatigue (4 or <1%, 1 trial) were infrequently reported.

Conclusion: The outcomes reported in clinical trials involving children with osteogenesis imperfecta are extremely heterogeneous and are often focused on surrogate outcomes, rather than clinical and patient-focussed outcomes. There is also extreme diversity and heterogeneity at every level: domain, measure, and time point. A limitation of this study is that the data extracted was limited by the quality of the available reports, but this also reflects the real-life experience of end users of the medical literature. Efforts to ensure consistent reporting of outcomes and international patient registries with pre-defined outcomes that are important to patients and clinicians will improve the value of trials in guiding clinical decision-making.

Disclosure: The authors declared no competing interests.