Harmonization Of Outcome Measures Research Articles

Approaches to outcome and covariate harmonization--the perfect as the “enemy of the good”

S21: When the Answer is “Big(ger) Data” in Environmental Epidemiology: What are the Questions?, Room 315, Floor 3, August 26, 2019, 1:30 PM - 3:00 PM As discussed in other talks in this session, pooling multiple generally similar studies allows more flexibility in data analysis compared to meta-analysis methods but requires careful attention to details of harmonization. Though the design of pooling projects may consider similarities in available data when selecting cohorts for inclusion, it is nearly inevitable that harmonization of exposures, outcomes, and covariates pose specific challenges. This talk will discuss approaches to harmonization for cohort studies, acknowledging the tradeoffs with each choice made. Even when cohorts use the same methods and tools to collect data, there are differences in cohort design including differences in time periods data were collected, differences in locations of participants, and meaningful differences in how environmental exposures differ over time and space. Harmonization of outcome measures may pose additional challenges, particularly due to changes over time in both standard definitions of health endpoints and data collection methods. For example, blood pressure may be measured, taken from medical records, a self-reported value or self-reported as “high”. It may be reported at baseline only, or reported during follow-ups. Also, standard definitions of high blood pressure have changed over time. When considering how to harmonize all of these aspects must be considered. Most existing pooling projects have harmonized covariates by attempting to create unified variables; is this sufficient? What other considerations should we be taking into account, particularly in environmental epidemiology? Are there other methodological approaches that can be considered? An open discussion of the advantages and disadvantages to different approaches to harmonization will help guide future pooling projects.

Reporting of outcomes in randomized controlled trials on nail psoriasis: a systematic review.

Harmonization of outcome measures is needed to increase the value of clinical trials on nail psoriasis. To provide the first step in core outcome set (COS) development for nail psoriasis. A systematic review was performed to identify outcome instruments and corresponding outcome domains used in (ongoing) randomized controlled trials. Identified outcome domains included clinical signs, quality of life, symptoms and delivery of care. The Nail Psoriasis Severity Index (NAPSI) was the most commonly used measure to assess clinical signs (74% of studies). Other outcome instruments used included the Nail Area Severity score, composite fingernail score, a Physician's Global Assessment, individual nail features or a combination of these. Heterogeneity in type and reporting (e.g. NAPSI 50, NAPSI 75) of outcome instruments was high and characteristics were often insufficiently reported. In total 43% of studies assessed quality of life, with 3% of studies using a nail psoriasis-specific tool. Assessment of symptoms and delivery of care was limited. Heterogeneity in the type and reporting of nail psoriasis outcome instruments needs to be addressed in the process towards COS development. Sufficient reporting of instrument characteristics should be encouraged. As nail psoriasis is generally assessed secondarily to psoriasis of the skin or joints, collaboration between different research groups in COS development is needed.

Systematic review of exercise for Charcot-Marie-Tooth disease.

Charcot-Marie-Tooth disease (CMT) is a slowly progressive hereditary degenerative disease and one of the most common neuromuscular disorders. Exercise may be beneficial to maintain strength and function for people with CMT, however, no comprehensive evaluation of the benefits and risks of exercise have been conducted. A systematic review was completed searching numerous electronic databases from earliest records to February 2015. Studies of any design including participants of any age with confirmed diagnosis of CMT that investigated the effects of exercise were eligible for inclusion. Of 13,301 articles identified following removal of duplicates, 11 articles including 9 unique studies met the criteria. Methodological quality of studies was moderate, sample sizes were small, and interventions and outcome measures used varied widely. Although the majority of the studies identified changes in one or more outcome measurements across exercise modalities, the majority were non-significant, possibly due to Type II errors. Significant effects described included improvements in strength, functional activities, and physiological adaptations following exercise. Despite many studies showing changes in strength and function following exercise, findings of this review should be met with caution due to the few studies available and moderate quality of evidence. Well-powered studies, harmonisation of outcome measures, and clearly described interventions across studies would improve the quality and comparability of the evidence base. The optimal exercise modality and intensity for people with CMT as well as the long-term safety of exercise remain unclear.

Towards harmonization of outcome measures in children with mitochondrial disorders

Towards harmonization of outcome measures in children with mitochondrial disorders

Towards the harmonization of outcome measures in children with mitochondrial disorders

A clinical trial is only as reliable as its outcomes, therefore the careful and systematic selection of outcome measures is extremely important. Currently, the selection of outcome measures for clinical trials designed to evaluate new drugs in patients with mitochondrial disorders is inefficient and has not been addressed systematically. Given that meaningful data can be obtained only from trials in which outcomes are assessed using valid instruments, one should first focus on the validation of a set of selected instruments in the target population. The aim of this review is to systematically select a 'toolbox' of robust outcome measures that are relevant to all patients. Using an extensive search of published literature, we systematically compiled a toolbox with outcome measures based on a primary search for possible instruments Subsequently, we reduced this toolbox using strict criteria that were adapted from the United States Food and Drug Administration. A toolbox with clinically relevant and psychometrically robust instruments for performing clinical research in children with mitochondrial disorders was compiled. In coming years, more experience using these outcome measures in children with various mitochondrial disease phenotypes must be obtained before reliable conclusions regarding the validity of these instruments can be drawn.