METHODS: It is timely at this juncture - as clinicians and researchers begin to grapple with the "invasion" of digital technologies - to review the strengths and weaknesses of these outcome measures.
RESULTS: This paper discusses advances (including an enhanced understanding of PD itself, and the development of clinimetrics as a field) that have led to improvements in the COMs used in PD; their strengths and limitations; and factors to consider when selecting and using a measuring instrument.
CONCLUSIONS: It is envisaged that in the future, a combination of COMs and technology-based objective measures will be utilized, with different methods having their own strengths and weaknesses. Judgement is required on the part of the clinician and researcher in terms of which instrument(s) are appropriate to use, depending on the particular clinical or research setting or question.
METHODS: VALID is a prospective, multi-center, multinational validation study that will assess the accuracy and feasibility of measuring VA function, defined as the need for interventions to enable and maintain the use of a VA for HD. The primary objective is to determine whether VA function can be measured accurately by clinical staff as part of routine clinical practice (Assessor 1) compared to the reference standard of documented VA procedures collected by a VA expert (Assessor 2) during a 6-month follow-up period. Secondary outcomes include feasibility and acceptability of measuring VA function and the time to, rate of, and type of VA interventions. An estimated 612 participants will be recruited from approximately 10 dialysis units of different size, type (home-, in-center and satellite), governance (private versus public), and location (rural versus urban) across Australia, Canada, Europe, and Malaysia. Validity will be measured by the sensitivity and specificity of the data acquisition process. The sensitivity corresponds to the proportion of correctly identified interventions by Assessor 1, among the interventions identified by Assessor 2 (reference standard). The feasibility of measuring VA function will be assessed by the average data collection time, data completeness, feasibility questionnaires and semi-structured interviews on key feasibility aspects with the assessors.
DISCUSSION: Accuracy, acceptability, and feasibility of measuring VA function as part of routine clinical practice are required to facilitate global implementation of this core outcome across all HD trials. Global use of a standardized, patient-centered outcome measure for VA function in HD research will enhance the consistency and relevance of trial evidence to guide patient-centered care.
TRIAL REGISTRATION: Clinicaltrials.gov: NCT03969225. Registered on 31st May 2019.
MATERIALS AND METHODS: Overall methods were guided by the Core Outcome Set Measures in Effectiveness Trials (COMET) initiative. Initial outcome identification was achieved from focus groups with PWLE employing calibrated methods across two low-middle-income countries (China and Malaysia) and two high-income countries (Spain and the United Kingdom). Following consolidation of the results, the outcomes were incorporated into a three-stage Delphi process with PWLE participation. Finally, consensus between PWLE and DPs was achieved using a mixed live and recorded platform. The experiences of PWLE involvement in the process was also evaluated.
RESULTS: Thirty-one PWLE participated in four focus groups. Thirty-four outcomes were suggested across the focus groups. Evaluation of the focus groups revealed a high level of satisfaction with the engagement process and some new learning. Seventeen PWLE contributed to the first 2 Delphi rounds and 7 to the third round. The final consensus included 17 PWLE (47%) and 19 DPs (53%). Out of the total of 11 final consensus outcomes considered essential by both PWLE and health professionals, 7 (64%) outcomes mapped across to ones that PWLE initially identified, broadening their definition. One outcome (PWLE effort required for treatment and maintenance) was entirely novel.
CONCLUSIONS: We conclude that engaging PWLE in COS development can be achieved across widely different communities. Furthermore, the process both broadened and enriched overall outcome consensus, yielding important and novel perspectives for health-related research.
DESIGN: A psychometric systematic review.
DATA SOURCES: Articles about the translation, adaptation, or validation of the MOS-SSS in Medline, PubMed, CINAHL, and Web of Science and their reference lists published before 11 November 2022.
REVIEW METHODS: The review followed the Consensus Standards for the Selection of Health Measurement Instruments guidelines.
RESULTS: The review included 35 articles. Eleven versions of MOS-SSS (3, 4, 5, 6, 8, 12, 13, 16, 18, 19, and 22 items) have been validated in various populations and 13 languages. Of 14 studies developing a translated version of MOS-SSS, four studies performed both an experts' evaluation of content validity and a face validity test; two studies reported translation evaluation in the form of a content validity index. Of 35 studies, six performed both exploratory factor analysis and confirmatory factor analysis for structural validity; hypotheses and measurements for construct validity testings were often not clearly stated; two examined criterion validity; and four assessed cross-cultural validity. Internal consistency reliabilities were commonly examined by calculating Cronbach's alpha and reported satisfactory. Five studies analysed test-retest reliabilities using intra correlation coefficient. Methodological concerns exist.
CONCLUSION: The English 19-item, Farsi Persian 19-item, and Vietnamese 19-item versions are recommended for future use in research and practice. Italian 19-item and Malaysian 13-item versions are not recommended to be used in future research and practice. All other versions considered in this review have potential use in future research and practice. Proper procedures for developing a translated version of MOS-SSS and validating the scale are recommended.
IMPACT: The review identified quality versions of MOS-SSS to measure social support in future research and practice. The study also indicated methodological issues in current validation studies. Application of the study findings and recommendations can be useful to improve outcome measurement quality and maximize the efficiency of resource use in future research and practice.
NO PATIENT OR PUBLIC CONTRIBUTION: This systematic review synthesized the evidence from previous research and did not involve any human participation.
METHODS: Prospectively collected clinical and EDx data were available in 957 IGOS patients from 115 centers. Only the first EDx study was included in the current analysis.
RESULTS: Median timing of the EDx study was 7 days (interquartile range 4-11) from symptom onset. Methodology varied between centers, countries and regions. Reference values from the responding 103 centers were derived locally in 49%, from publications in 37% and from a combination of these in the remaining 15%. Amplitude measurement in the EDx studies (baseline-to-peak or peak-to-peak) differed from the way this was done in the reference values, in 22% of motor and 39% of sensory conduction. There was marked variability in both motor and sensory reference values, although only a few outliers accounted for this.
CONCLUSIONS: Our study showed extensive variation in the clinical practice of EDx in GBS patients among IGOS centers across the regions.
SIGNIFICANCE: Besides EDx variation in GBS patients participating in IGOS, this diversity is likely to be present in other neuromuscular disorders and centers. This underlines the need for standardization of EDx in future multinational GBS studies.
METHODS: Medical records of all HSCR patients who underwent pull-through at the Dr. Sardjito Hospital, Indonesia between January 2010 and August 2016 were reviewed for their growth outcomes before and after the surgery.
RESULTS: We included 64 HSCR patients, 45 males and 19 females, of which 14, 17, and 33 patients underwent Duhamel, Soave, and TEPT respectively. There were no nutritional status differences in HSCR patients after Duhamel, Soave, and TEPT surgery (p=0.07, 0.17, and 0.79, respectively). Z-score average of weight-for-age did not differ between three surgical methods (p=0.77 and 0.15 for preoperative and postoperative, respectively). In addition, the improvement of nutritional status was achieved in 21.2% HSCR patients after TEPT, 14.3% post Duhamel and 5.9% following Soave procedure, but these differences did not reach a significant level (p=0.34).
DISCUSSION: Our study shows no difference in effect on the growth outcomes in HSCR patients following Duhamel, Soave and TEPT procedure. Further study with a larger sample size is important to give valuable long-term growth outcomes for HSCR patients after pull-through.
SUMMARY OF BACKGROUND DATA: A previous systematic review identified 115 outcomes in 60 trials and systematic reviews evaluating treatments for children with appendicitis, suggesting the need for a COS.
METHODS: The development process consisted of 4 phases: (1) an updated systematic review identifying all previously reported outcomes, (2) a 2-stage international Delphi study in which parents with their children and surgeons rated these outcomes for inclusion in the COS, (3) focus groups with young people to identify missing outcomes, and (4) international expert meetings to ratify the final COS.
RESULTS: The systematic review identified 129 outcomes which were mapped to 43 unique outcome terms for the Delphi survey. The first-round included 137 parents (8 countries) and 245 surgeons (10 countries), the second-round response rates were 61% and 85% respectively, with 10 outcomes emerging with consensus. After 2 young peoples' focus groups, 2 additional outcomes were added to the final COS (12): mortality, bowel obstruction, intraabdominal abscess, recurrent appendicitis, complicated appendicitis, return to baseline health, readmission, reoperation, unplanned appendectomy, adverse events related to treatment, major and minor complications.
CONCLUSION: An evidence-informed COS based on international consensus, including patients and parents has been developed. This COS is recommended for all future studies evaluating treatment ofsimple appendicitis in children, to reduce heterogeneity between studies and facilitate data synthesis and evidence-based decision-making.
MATERIALS AND METHODS: Overall methods were guided by the Core Outcome Set Measures in Effectiveness Trials (COMET) initiative. Initial outcome identification was achieved from focus groups with PWLE employing calibrated methods across two low-middle-income countries (China and Malaysia) and two high-income countries (Spain and the United Kingdom). Following consolidation of the results, the outcomes were incorporated into a three-stage Delphi process with PWLE participation. Finally, consensus between PWLE and DPs was achieved using a mixed live and recorded platform. The experiences of PWLE involvement in the process was also evaluated.
RESULTS: Thirty-one PWLE participated in four focus groups. Thirty-four outcomes were suggested across the focus groups. Evaluation of the focus groups revealed a high level of satisfaction with the engagement process and some new learning. Seventeen PWLE contributed to the first 2 Delphi rounds and 7 to the third round. The final consensus included 17 PWLE (47%) and 19 DPs (53%). Out of the total of 11 final consensus outcomes considered essential by both PWLE and health professionals, 7 (64%) outcomes mapped across to ones that PWLE initially identified, broadening their definition. One outcome (PWLE effort required for treatment and maintenance) was entirely novel.
CONCLUSIONS: We conclude that engaging PWLE in COS development can be achieved across widely different communities. Furthermore, the process both broadened and enriched overall outcome consensus, yielding important and novel perspectives for health-related research.