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Patient-reported outcome instruments provide valuable insight into disease-related morbidity known only to the patient and complement more objective outcome tools in the clinical trial setting. They are of particular importance in systemic sclerosis owing to the challenges around defining disease activity, the episodic nature of many disease-specific manifestations and the paucity of validated objective surrogate outcome measures for use in clinical trials. Early clinical trials of systemic sclerosis often incorporated legacy patient-reported outcome instruments, but the last 20 years has witnessed the emergence of several scleroderma-specific instruments that are now being routinely used alongside other outcomes in systemic sclerosis clinical trials. More recently, the value of patient-reported outcomes has been highlighted by their prominence in the American College of Rheumatology Combined Response Index for Systemic Sclerosis that has been utilized as the primary endpoint of recent clinical trials of early diffuse systemic sclerosis. This review considers the role and performance of the various patient-reported outcome instruments utilized in systemic sclerosis clinical trials, the current positioning of patient-reported outcome instruments within clinical trial endpoint models across the range of systemic sclerosis disease manifestations and, where applicable, we shall highlight areas for future research.

Background: Lack of uniformity in reported outcomes makes comparisons between acromioclavicular joint (ACJ) injury studies challenging. Knowledge of common outcome measures and standardization will help orthopaedic surgeons report and compare outcomes more consistently. Purpose: To identify the most commonly reported outcome measures for ACJ injuries. Study Design: Systematic review. Methods: A systematic review was performed to identify all English-language original articles assessing any type of management of ACJ injuries (acute and chronic) in PubMed and Scopus from 2007 to 2017. Review articles, meta-analyses, studies with less than 5 patients, pediatric studies, technique articles, and biomechanical studies were excluded. The 100 top orthopaedic journals in the English literature were selected for review. Included studies were assessed for patient characteristics and the use of outcome variables, including range of motion (ROM), strength, patient-reported outcomes (PROs), satisfaction, return to work, return to sport, and complications. Results: A total of 605 unique articles were identified; 92 met the inclusion criteria. The average number of ACJ injuries per study was 37, with a mean weighted patient age of 36 years (range, 20.1-57.3 years). The mean follow-up was 36 months (range, 5-290 months). Acute injuries were reported in 59% of studies. ROM and strength measurements were reported in 22.8% and 5.4% of studies. Sixteen different PRO instruments were used. The most commonly reported measures were Constant score (75%), visual analog scale for pain (VAS-pain; 33%) score, American Shoulder and Elbow Surgeons (ASES) score (21%), Simple Shoulder Test (SST) score (19%), and University of California Los Angeles (UCLA; 17%) shoulder score. An average of 2.5 outcome measures per study were reported. The use of 4 or more outcome scores was associated with publication in higher–impact factor journals. Conclusion: Inconsistent reporting of multiple outcome measures is present in the ACJ injury literature. The best scoring system for assessing ACJ injury and treatment has not yet been agreed upon. Until improved scoring systems come into general use, we recommend that future literature on ACJ injuries use at least 4 outcome scores and include the commonly used outcome measures (Constant, VAS-pain, ASES, and SST scores) to enable future comparison of patient outcomes across publications.

Study Design Literature review. Objective To identify outcomes instruments used in spinal trauma surgery over the past decade, their frequency of use, and usage trends. Methods Five top orthopedic journals were reviewed from 2004 to 2013 for clinical studies of surgical intervention in spinal trauma that reported patient-reported outcome instruments use or neurologic function scale use. Publication year, level of evidence (LOE), and outcome instruments were collected for each article and analyzed. Results A total of 58 studies were identified. Among them, 26 named outcome instruments and 7 improvised questionnaires were utilized. The visual analog scale (VAS) for pain was used most frequently (43.1%), followed by the Short Form 36 (34.5%), Frankel grade scale (25.9%), Oswestry Disability Index (20.7%) and American Spinal Injury Association Impairment Scale (15.5%). LOE 4 was most common (37.9%), and eight LOE 1 studies were identified (10.3%). Conclusions The VAS pain scale is the most common outcome instrument used in spinal trauma. The scope of this outcome instrument is limited, and it may not be sufficient for discriminating between more and less effective treatments. A wide variety of functional measures are used, reflecting the need for a disease-specific instrument that accurately measures functional limitation in spinal trauma.

In this review we discuss health measurement with a focus on psychometric methods and methodology. In particular, we examine some of the key issues currently facing the use of clinician and patient rating scales to measure the health outcomes of disease and treatment. We present three key facts and flag one crucial problem. First, the numbers generated by scales are increasingly used as the measurements of the central dependent variables upon which clinical decisions are frequently made. The rising profile of rating scales has significant implications for scale construction, evaluation, and selection, as well as for interpreting studies. Second, rating scale science is well established. Therefore, it is important to learn the lessons from those who have built and established the science over the last century. Finally, the goal of a rating scale is to measure. As such, over the last half century, developments in rating scale (psychometric) methods have caused a refocus in the way we should be measuring health. In particular, newer methods have significant clinical advantages over traditional approaches. These should be seriously considered for inclusion in everyday practice. This leads us to the central problem with health measurement, which is that we cannot currently be sure what most rating scales are measuring. This is because the methods we have in place to ensure the validity of rating scales fall short of what is actually required. We expand on this point, and provide some potential routes forward to help address this important problem.