Core Outcome Measures Research Articles

Inclusion of participants from low-income and middle-income countries in core outcome sets development: a systematic review

ObjectiveOur study aims to describe differences or similarities in the scope, participant characteristics and methods used in core outcome sets (COS) development when only participants from high-income countries (HICs) were...

341 All Over the Road: A Systematic Review of Mobile Integrated Health, Community Paramedicine Programs

Mobile Integrated Health Community Paramedicine (MIHCP) expands the scope of EMS clinicians to provide patient-centered, mobile resources in the out-of-hospital environment to increase access to care and reduce ED usage. Prior MIHCP reviews have been limited in breadth or focused on characterizing interventions. This systematic review aims to characterize the outcomes and methodology that programs used to evaluate their effectiveness. We searched Pubmed, Embase, CINAHL, and Scopus databases for peer-reviewed literature related to MIHCP programs from January 1, 2000, through July 2020. We included all full-text English studies whose program met the National Association of Emergency Medical Technicians (NAEMT) definition, had an MIH- CP-related intervention and measured outcome, and was implemented through emergency medical services. We excluded all non-English papers, abstract-only, and incomplete studies. We identified 22 studies in our literature review. Study populations ranged from 14 to 2315 subjects and lasted from 4 to 48 months. There were three randomized controlled trials, 13 cohort studies (8 retrospective, 5 prospective), three case series, and three cross-sectional studies. Of the 13 cohort studies, 8 used a pre-post control, 2 statistically matched controls, 1 non-statistically matched control, 1 combined statistically matched and pre-post controls, and 1 did not feature a control. Case series and cross-sectional studies did not feature controls. 14 studies included ED usage as an outcome, measured by: ED visits (9), hospital admissions (8), ED length of stay (3), ED visits avoided (2), readmission rate (3), and ED transport rate (5). 12 studies included at least one Center for Medicare & Medicaid Innovation (CMMI) healthcare utilization core outcome. 4 studies reported cost-related outcomes, measured by: ambulance transport savings (2), ED visit savings (2), hospital admissions savings (1), and cost per quality adjusted life year increase (1). Three studies used at least one NAEMT core cost outcome. The NAEMT laid out a uniform set of measures (eg. health care utilization and cost) for MIHCP to optimize resource utilization and achievement of the triple aim. No individual study reported all CMMI healthcare utilization core outcomes nor NAEMT cost core outcomes. Our review of MIHCP studies revealed significant heterogeneity in study designs, control groups, and outcome measurements. Importantly, 6/22 (27%) studies used appropriate controls, while the rest were subject to selection bias, and only 9/22 (41%) were prospective, leaving the others more subject to information bias. Future MIHCP studies should focus on appropriate study designs to reduce bias and report core outcome measures to improve consistency across investigations and adherence to national organizations' recommendations.

Implementation of the Core Outcome Measures CPG: Assessing the Know-Do Gap

<h3>Research Objectives</h3> To assess the Know-Do Gap of physical therapist use of the clinical practice guideline recommended core set of outcome measures (OMs) for neurologic conditions. <h3>Design</h3> Mixed-methods knowledge translation study implementing the Knowledge to Action (KTA) Cycle. <h3>Setting</h3> One neurologic outpatient physical therapy organization with three clinical sites that primarily serves patients with neurologic conditions. <h3>Participants</h3> 19 physical therapists. <h3>Interventions</h3> Retrospective medical record review of initial evaluation and discharge documentation from a five month period (October 2019 - February 2020). <h3>Main Outcome Measures</h3> Frequency of use of outcome measures, including the six core outcome measures recommended in the neurological outcome measures clinical practice guideline. <h3>Results</h3> 225 notes were reviewed (evaluation = 134; discharge = 91), performed by 19 physical therapists. 39 unique OMs were used during the 5 month reporting period. Results of the six core OMs were performed at the following frequencies: Six Minute Walk Test 82.2% (n = 185); Berg Balance Scale 43.1% (n = 97); 5 Times Sit-to-Stand 16.9% (n = 38); 10 meter Walk Test 14.7% (n = 33); Functional Gait Assessment 3.1% (n = 7); and the Activities-Specific Balance Confidence Scale 1.8% (n =4). <h3>Conclusions</h3> Implementation of the six core OMs in this outpatient physical therapy organization was variable. This assessment of the know-do gap is the first step in the KTA Cycle. Future steps include continuing to utilize the KTA Cycle to increase uptake of the core set of OMs. <h3>Author(s) Disclosures</h3> Dr. Yorke has no disclosures. Dr. Trojanowski has no disclosures. Dr. Fritz serves on the MS scientific advisory board for Helius Medical.

Evaluating measures of quality of life in adult scoliosis: a protocol for a systematic review and narrative synthesis

BackgroundAdult scoliosis represents a distinct subgroup of scoliosis patients for whom the diagnosis can have a large impact on their health-related quality of life (HR-QOL). Therefore, HR-QOL patient-reported outcome measures (PROMs) are essential to assess disease progression and the impact of interventions. The objective of this systematic review is to evaluate the measurement properties of HR-QOL PROMs in adult scoliosis patients.MethodsWe will conduct a literature search, from their inception onwards, of multiple electronic databases including AMED, CINAHL, EMBASE, Medline, PsychINFO and PubMed. The searches will be performed in two stages. For both stages of the search, participants will be aged 18 and over with a diagnosis of scoliosis. The primary outcome of interest in the stage one searches will be studies which use PROMs to investigate HR-QOL as defined by the Core Outcome Measures in Effectiveness Trials (COMET) taxonomy, the secondary outcome will be to assess the frequency of use of the various PROMs. In stage two, the primary outcome of interest will be studies which assess the measurement properties of the HR-QOL PROMs identified in stage one. No specific measurement property will be given priority. No planned secondary outcomes have been identified but will be reported if discovered. In stage one, the only restriction on study design will be the exclusion of systematic reviews. In Stage two the only restriction on study design will be the exclusion of full-text articles not available in the English language.Two reviewers will independently screen all citations and abstract data. Potential conflicts will be resolved through discussion. The study methodological quality (or risk of bias) will be appraised using the Consensus-based Standards for the selection of Health Measurement Instruments (COSMIN) checklist. The overall strength of the body of evidence will then be assessed using the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) approach. A narrative synthesis will be provided with information presented in the main text and tables to summarise and explain the characteristics and findings of the included studies. The narrative synthesis will explore the evidence for currently used PROMs in adult scoliosis patients and any areas that require further study.DiscussionThe review will help clinicians and researchers identify a HR-QOL PROM for use in patients with adult scoliosis. Findings from the review will be published and disseminated through a peer-reviewed journal and conference presentations.Systematic review registrationThis systematic review has been registered with the International Prospective Register of Systematic Reviews (PROSPERO), reference number: CRD42020219437

Development of an extension of the OMERACT Summary of Measurement Properties table to capture equity considerations: SOMP-Equity

ObjectiveTo develop an equity extension of the OMERACT Summary of Measurement Properties (SOMP) Table, SOMP Equity to describe whether a patient reported outcome measure (PROM) works well among patients of diverse languages and cultures, education levels, and other population characteristics. MethodsWe used the PROGRESS-Plus framework to categorize equity characteristics assessed in trials of PROM. PROGRESS refers to Place of residence, Race/ethnicity/culture/language, Occupation, Gender/sex, Religion, Education, Socioeconomic status, and Social Capital, while the ‘plus’ captures additional characteristics, such as age. We pilot tested our SOMP Equity Extension using the Health Assessment Questionnaire (HAQ) as a prototypical PROM. ResultsThe SOMP Equity Extension retains the same columns as the original OMERACT SOMP (domain match, feasibility, construct validity, test-retest reliability, longitudinal construct validity, clinical trial discrimination, thresholds of meaning) but uses the PROGRESS-Plus characteristics as rows. We found several examples of studies of the HAQ which had assessed one or more PROGRESS-Plus characteristics. ConclusionsThe most commonly reported equity considerations were related to language. OMERACT Equity virtual meeting participants were polled and they indicated that the SOMP Equity Extension is useful for highlighting and tracking equity considerations for OMERACT Core Outcome Measurement Instruments.

Standardised Outcome Reporting for the Nutrition Management of Complex Chronic Disease: A Rapid Review.

Individuals with coexisting chronic diseases or with complex chronic disease are among the most challenging and costly patients to treat, placing a growing demand on healthcare systems. Recommending effective treatments, including nutrition interventions, relies on standardised outcome reporting from randomised controlled trials (RCTs) to enable data synthesis. This rapid review sought to determine how the scope and consistency of the outcomes reported by RCTs investigating nutrition interventions for the management of complex chronic disease compared to what is recommended by the core outcome sets (COS) for individual disease states. Peer-reviewed RCTs published between January 2010 and July 2020 were systematically sourced from PubMed, CINAHL and Embase, and COS were sourced from the International Consortium for Health Outcomes Measurements (ICHOM) and the Core Outcome Measures in Effectiveness Trials (COMET) database. A total of 45 RCTs (43 studies) and 7 COS were identified. Outcomes were extracted from both the RCTs and COS and were organised using COMET Taxonomy Core Areas. A total of 66 outcomes and 439 outcome measures were reported by the RCTs. The RCTs demonstrated extensive outcome heterogeneity, with only five outcomes (5/66, 8%) being reported with relative consistency (cited by ≥50% of publications). Furthermore, the scope of the outcomes reported by studies was limited, with a notable paucity of patient-reported outcomes. Poor agreement (25%) was observed between the outcomes reported in the RCTs and those recommended by the COS. This review urges greater uptake of the existing COS and the development of a COS for complex chronic disease to be considered so that evidence can be better synthesised regarding effective nutrition interventions.

Core outcome measurement instrument selection for physical function in hand osteoarthritis using the OMERACT Filter 2.1 process

ObjectivePhysical function is one of the Outcome Measures in Rheumatology (OMERACT) core outcome domains for hand osteoarthritis studies. Our aim was to select appropriate instrument(s) to measure this domain, as part of the development of a core outcome measurement set. MethodsFollowing the OMERACT Filter 2.1 instrument selection process, the (function subscale of) the Australian/Canadian Hand Osteoarthritis Index (AUSCAN), Functional Index for Hand Osteoarthritis (FIHOA) and Michigan Hand Outcomes Questionnaire (MHQ) were assessed for domain match, feasibility, truth and discrimination. Data gathered from available literature, working group and patient surveys, and additional analyses in two hand osteoarthritis cohorts were used to inform a consensus process. Results were summarized in Summary of Measurements Properties tables and reviewed by the OMERACT technical advisory group. ResultsMHQ passed the assessment of domain match and feasibility by the working group and patient research partners. For AUSCAN important limitations in feasibility were noted, but domain match was good. FIHOA did not pass the assessment and was not taken through the follow-up assessment. Based on published literature, reliability and construct/longitudinal validity of both MHQ and AUSCAN fulfilled OMERACT standards. While clinical trial discrimination and thresholds of meaning were good for AUSCAN, results for MHQ were ambiguous. ConclusionMHQ was provisionally endorsed as OMERACT core outcome measure for the core domain physical function. While AUSCAN may have better metric properties than MHQ, it received provisional endorsement as a second measure of function due to important feasibility issues. A research agenda to merit full endorsement was set.

ACTonFood. Acceptance and Commitment Therapy-Based Group Treatment Compared to Cognitive Behavioral Therapy-Based Group Treatment for Weight Loss Maintenance: An Individually Randomized Group Treatment Trial.

The purpose of this Individually Randomized Group Treatment Trial was to compare an Acceptance and Commitment Therapy-based (ACT) group intervention and a Cognitive Behavioral Therapy-based (CBT) group intervention for weight loss maintenance in a sample of adult patients with obesity seeking treatment for weight loss. One hundred and fifty-five adults (BMI: Kg/m2 = 43.8 [6.8]) attending a multidisciplinary rehabilitation program for weight loss were randomized into two conditions: ACT and CBT. Demographical, physical, and clinical data were assessed at the beginning of the program (t0), at discharge (t1), and at 6-month follow-up (t2). The following measures were administered: The Acceptance and Action Questionnaire-II (AAQ-II) and the Clinical Outcome in Routine Evaluation-Outcome Measure (CORE-OM). Generalized linear mixed models were performed to assess differences between groups. Moderation effects for gender and Eating Disorders (ED) have been considered. From baseline to discharge, no significant differences between interventions were found, with the only exception of an improvement in the CORE-OM total score and in the CORE-OM subjective wellbeing subscale for those in the CBT condition. From discharge to follow-up, ACT group participants showed significant results in terms of weight loss maintenance, CORE-OM total score, and CORE-OM and AAQ-II wellbeing, symptoms, and psychological problems subscales. Gender moderated the effects of time and intervention on the CORE-OM subscale reporting the risk for self-harm or harm of others. The presence of an eating disorder moderated the effect of time and intervention on the CORE-OM total score, on the CORE-OM symptoms and psychological problems subscales, and on the AAQ-II. Patients who received the ACT intervention were more likely to achieve a ≥5% weight loss from baseline to follow-up and to maintain the weight loss after discharge. The ACT intervention was thus effective in maintaining weight loss over time.

Standardised Outcomes in Nephrology \u2013 Chronic Kidney Disease (SONG-CKD): a protocol for establishing a core outcome set for adults with chronic kidney disease who do not require kidney replacement therapy

BackgroundGlobally, over 1.2 million people die from chronic kidney disease (CKD) every year. Patients with CKD are up to 10 times more likely to die prematurely than progress to kidney failure requiring kidney replacement therapy. The burden of symptoms and impaired quality of life in CKD may be compounded by comorbidities and treatment side effects. However, patient-important outcomes remain inconsistently and infrequently reported in trials in patients with CKD, which can limit evidence-informed decision-making. The Standardised Outcomes in Nephrology – Chronic Kidney Disease (SONG-CKD) aims to establish a consensus-based core outcome set for trials in patients with CKD not yet requiring kidney replacement therapy to ensure outcomes of relevance to patients, caregivers and health professionals are consistently reported in trials.MethodsSONG-CKD involves four phases: a systematic review to identify outcomes (domains and measures) that have been reported in randomised controlled trials involving adults with CKD who do not require kidney replacement therapy; stakeholder key informant interviews with health professionals involved in the care of adults with CKD to ascertain their views on establishing core outcomes in CKD; an international two-round online Delphi survey with patients, caregivers, clinicians, researchers, policy makers and industry representatives to obtain consensus on critically important outcome domains; and stakeholder consensus workshops to review and finalise the set of core outcome domains for trials in CKD.DiscussionEstablishing a core outcome set to be reported in trials in patients with CKD will enhance the relevance, transparency and impact of research to improve the lives of people with CKD.Trial registrationNot applicable. This study is registered with the Core Outcome Measures in Effectiveness Trials (COMET) database: http://www.comet-initiative.org/Studies/Details/1653.

APTA Cross Sections and Academies Recommendations for COVID-19 Core Outcome Measures.

Purpose:The novel coronavirus (COVID-19) emerged as a major health concern within the United States in early 2020. Because this is a novel virus, little guidance exists for best practice to evaluate this population within the field of physical therapy.Methods:An expert task force appointed by the leadership of 9 different academies or sections of the American Physical Therapy Association was formed to develop recommendations for a set of core outcome measures for individuals with or recovering from COVID-19.Results:This perspective provides guidance on a best practice recommendation to physical therapists and researchers regarding the use of core outcome measures for individuals with or recovering from COVID-19. The process for the selection of core measures for this population is presented and discussed.Conclusions:Core outcome measures improve the ability to track progress and change across the continuum of care at both the patient and population levels.

Patient-rated outcome after atlantoaxial (C1-C2) fusion: more than a decade of evaluation of 2-year outcomes in 126 patients.

Various surgical techniques have been introduced for atlantoaxial (C1-C2) fusion, the most common being Magerl's (transarticular) or the Harms/Goel screw fixation. Common indications include degenerative osteoarthritis (OA), trauma or rheumatoid arthritis (RA). Only few, small studies have evaluated patient-reported outcomes after C1-C2 fusion. We investigated 2-year outcomes in a large series of consecutive patients undergoing isolated C1-C2 fusion. We analysed prospectively collected data (2005-2016) from our Spine outcomes database, collected within the framework of EUROSPINE's Spine Tango Registry. It included 126patients (34 (27%) men, 92 (73%) women; mean (SD) age 67 ± 19 y) who had undergone first-time isolated C1-C2 fusion (61% Magerl, 39% Harms(-Goel)) at least 2 years ago for OA (83 (66%)), RA (20 (16%)), fracture (15 (12%)) or other (8 (6%)). Patients completed the multidimensional Core Outcome Measures Index (COMI; 0-10) and various single item outcomes. Questionnaires were returned by 118/126 (94%) patients, 2 years post-operative. Mean COMI scores showed a significant reduction from baseline: 6.9 ± 2.4 to 2.7 ± 2.5 (p < 0.0001). Overall, 75% patients achieved the MCIC of ≥ 2.2points reduction in COMI and 88% reported a good global outcome. 91% patients were satisfied/very satisfied with their care. Self-reported complications were declared by 16% patients and further surgery at the same segment, by 2.5%. In this large series with almost complete follow-up, C1-C2 fusion showed extremely good results. Despite the complexity of the intervention, outcomes surpassed those typically reported for simple procedures such as ACDF and lumbar discectomy, suggesting reservations about the procedure should perhaps be reviewed.

Development of a core outcome set for evaluative research into paediatric cerebral visual impairment (CVI), in the UK and Eire

ObjectivesCerebral visual impairment (CVI) comprises a heterogeneous group of brain-related vision problems. A core outcome set (COS) represents the most important condition-specific outcomes according to patients, carers, professionals and researchers....

Outcomes and measures of delirium interventional studies in palliative care to inform a core outcome set: A systematic review

Background: Trials of interventions for delirium in various patient populations report disparate outcomes and measures but little is known about those used in palliative care trials. A core outcome set promotes consistency of outcome selection and measurement. Aim: To inform core outcome set development by examining outcomes, their definitions, measures and time-points in published palliative care studies of delirium prevention or treatment delirium interventions. Design: Prospectively registered systematic review adhering to Preferred Reporting Items for Systematic Reviews and Meta-Analyses. Data sources: We searched six electronic databases (1980–November 2020) for original studies, three for relevant reviews and the International Clinical Trials Registry Platform for unpublished studies and ongoing trials. We included randomised, quasi-randomised and non-randomised intervention studies of pharmacological and non-pharmacological delirium prevention and/or treatment interventions. Results: From 13/3244 studies (2863 adult participants), we identified 9 delirium-specific and 13 non-delirium specific outcome domains within eight Core Outcome Measures in Effectiveness Trials (COMET) taxonomy categories. There were multiple and varied outcomes and time points in each domain. The commonest delirium specific outcome was delirium severity (n = 7), commonly using the Memorial Delirium Assessment Scale (6/8 studies, 75%). Four studies reported delirium incidence. Non-delirium specific outcomes included mortality, agitation, adverse events, other symptoms and quality of life. Conclusion: The review identified few delirium interventions with heterogeneity in outcomes, their definition and measurement, highlighting the need for a uniform approach. Findings will inform the next stage to develop consensus for a core outcome set to inform delirium interventional palliative care research.

Progression of Clinical Features in Lewy Body Dementia Can Be Detected Over 6 Months.

ObjectiveThis study aimed to quantify the trajectory and magnitude of change of the key clinical features and corresponding symptom domains of dementia with Lewy bodies (DLB) and Parkinson disease dementia (PDD), including global cognition, parkinsonism, recurrent visual hallucinations, cognitive fluctuations, and sleep disturbance.MethodsOne hundred sixteen patients with Lewy body dementia (DLB = 72, PDD = 44) underwent assessment at baseline and 3 and 6 months as part of a prospective multicenter randomized controlled trial. Linear mixed models were constructed for core outcome measures using the Mini-Mental State Examination (MMSE), motor section of the Unified Parkinson's Disease Rating Scale (UPDRS-III), Dementia Cognitive Fluctuations Scale (DCFS), and Neuropsychiatric Inventory (NPI).ResultsWithin the time frame of our study (6 months), we were able to identify a significant cognitive decline of 1.3 points on the MMSE (p = 0.002) and significant worsening of motor parkinsonism with an increase in UPDRS-III score of 3.2 points (p = 0.018). Fluctuation severity also increased using the DCFS with a 6-month change in score of 1.3 points (p = 0.001). Uniquely, a signal for increased severity of sleep symptoms of 1.2 points (NPI-sleep) was also detectable (p = 0.04). Significant changes in neuropsychiatric symptoms were not detected. There was no difference in rates of change of scores between DLB and PDD.DiscussionClinically significant rates of change in core clinical features can be detected and quantified in Lewy body dementia over a relatively short period (6 months) using common clinical instruments and thus may be useful as clinical endpoints for therapeutic trials of disease-modifying and symptomatic agents.

Core Outcome Measures for Palliative and End-of-Life Research After Severe Stroke: Mixed-Method Delphi Study.

Stroke is the second commonest cause of death worldwide and a leading cause of severe disability, yet there are no published trials of palliative care in stroke. To design and evaluate palliative care interventions for people with stroke, researchers need to know what measurable outcomes matter most to patients and families, stroke professionals, and other service providers. A multidisciplinary steering group of professionals and laypeople managed the study. We synthesized recommendations from respected United Kingdom and international consensus documents to generate a list of outcome domains and then performed a rapid scoping literature review to identify potential outcome measures for use in future trials of palliative care after stroke. We then completed a 3-round, online Delphi survey of professionals, and service users to build consensus about outcome domains and outcome measures. Finally, we held a stakeholder workshop to review and finalize this consensus. We generated a list of 36 different outcome domains from 4 key policy documents. The rapid scoping review identified 43 potential outcome measures that were used to create a shortlist of 16 measures. The 36 outcome domains and 16 measures were presented to a Delphi panel of diverse healthcare professionals and lay service users. Of 48 panelists invited to take part, 28 completed all 3 rounds. Shared decision-making and quality of life were selected as the most important outcome domains for future trials of palliative care in stroke. Additional comments highlighted the need for outcomes to be feasible, measurable, and relevant beyond the initial, acute phase of stroke. The stakeholder workshop endorsed these results. Future trials of palliative care after stroke should include pragmatic outcome measures, applicable to the evolving patient and family experiences after stroke and be inclusive of shared decision-making and quality of life.

Establishing a core outcome measure for pain in patients with autosomal dominant polycystic kidney disease: a consensus workshop report.

BackgroundPain is the highest prioritized patient-reported outcome in people with autosomal dominant polycystic kidney disease (ADPKD) but remains infrequently and inconsistently measured in clinical trials and poorly managed in clinical settings. A recently completed systematic review of pain in ADPKD identified 26 different outcome measures. None of these measures were considered appropriate as a core outcome measure due to the lack of patient-important dimensions, inadequate content, relatively long duration of completion time and limited evidence to support psychometric robustness.MethodsWe convened an international Standardized Outcomes in Nephrology–Polycystic Kidney Disease consensus workshop involving 21 patients/caregivers and 40 health professionals (clinicians, nurses, researchers, policy makers and industry representatives) from 18 countries to discuss the identification or development of a core outcome measure for pain.ResultsFour themes were identified highlighting fundamental issues for the measurement of pain in ADPKD: distressing and disrupting life participation; variability and ambiguity in defining pain; stigma, frustration and adaptation to pain; and ensuring validity and feasibility of pain measures.ConclusionsExisting measures were found to be insufficient in capturing pain as a core outcome and there was consensus on the need for a new validated measure that is simple, succinct and addresses the impact of pain on life participation. This measure will facilitate the appropriate prioritization of pain in all trials and guide clinical decision making in people with ADPKD.

Development of a core outcome set for therapeutic studies in eosinophilic esophagitis (COREOS)

End points used to determine treatment efficacy in eosinophilic esophagitis (EoE) have evolved over time. With multiple novel therapies in development for EoE, harmonization of outcomes measures will facilitate evidence synthesis and appraisal when comparing different treatments. We sought to develop a core outcome set (COS) forcontrolled and observational studies of pharmacologicanddiet interventions in adult and pediatric patients with EoE. Candidate outcomes were generated from systematic literature reviews and patient engagement interviews and surveys. Consensus was established using an iterative Delphi process, with items voted on using a 9-point Likert scale and with feedback from other participants to allow score refinement. Consensus meetings were held to ratify the outcome domains of importance and the core outcome measures. Stakeholders were recruited internationally and included adult and pediatric gastroenterologists, allergists, dieticians, pathologists, psychologists, researchers, and methodologists. The COS consists of 4 outcome domains for controlled and observational studies: histopathology, endoscopy, patient-reported symptoms, and EoE-specific quality of life. Atotal of 69 stakeholders (response rate 95.8%) prioritized 42 outcomes in a 2-round Delphi process, and the final ratification meeting generated consensus on 33 outcome measures. These included measurement of the peak eosinophil count, Eosinophilic Esophagitis Histology Scoring System, Eosinophilic Esophagitis Endoscopic Reference Score, and patient-reported measures of dysphagia and quality of life. This interdisciplinary collaboration involving global stakeholders has produced a COS that can be applied to adult and pediatric studies of pharmacologic and diet therapies for EoE and will facilitate meaningful treatment comparisons and improve the quality of data synthesis.

Plasma Rich in Growth Factors (PRGF) in the Treatment of Cervical and Lumbar Back Pain: A Retrospective Observational Clinical Study

Plasma rich in growth factors (PRGF) is a leukocyte-free platelet-rich plasma (PRP) that is an effective biological approach to tissue repair and has been demonstrated to significantly improve multiple conditions, including low back pain and degenerative disc pathology. The objective of this retrospective study was to analyze the effectiveness of treating both cervical and lumbar spine pain with minimally invasive infiltrations of PRGF-Endoret. Retrospective study design. Outpatient private practice facility. The effectiveness of PRGF has been assessed by patient-reported outcomes (PRO) through validated questionnaires, namely Numeric Rating Scale (NRS) for back pain, Spine Tango Core Outcome Measure Index (COMI) Questionnaire for lumbar and cervical pain, and OSWESTRY Disability Index questionnaire for lumbar pain cases. Score differences between pre- and post-treatment have also been assessed stratified for multiple sub-groups of patients based on the sector of the column treated, gender, and age. This study includes 65 patients (18 with cervical pain and 47 with lumbar back pain). The average time of back pain evolution was 10 years. Patients received at least 2 PRGF infiltration series about one month apart. Each patient received intervertebral disc and epidural infiltrations, root infiltrations, in case of radicular injury, and intraarticular infiltrations, in case of osteoarthritis of the facet joints. Results show statistically significant (P < 0.05) improvements on all analyzed scores. Regarding the stratified analyses, 249 (99%) sub-groups showed an improvement in all tests (252 cases overall). From the 249 improving cases, 154 (62%) showed a statistically significant difference between the pre- and the post- treatment periods. This research is a retrospective study with a relatively small sample cohort. Only PRO have been assessed. According to the results obtained in this study, and taking into account their limitations, PRGF infiltrations are an effective and minimally invasive biological strategy in the treatment of both cervical and lumbar pain, evaluated according to PRO.

A systematic review of outcome reporting and outcome measures in studies investigating uterine-sparing treatment for adenomyosis.

STUDY QUESTIONWhich outcomes and outcome measures are reported in interventional trials evaluating the treatment of adenomyosis?SUMMARY ANSWERWe identified 38 studies, reporting on 203 outcomes using 133 outcome measures.WHAT IS KNOWN ALREADYHeterogeneity in outcome evaluation and reporting has been demonstrated for several gynaecological conditions and in fertility studies. In adenomyosis, previous systematic reviews have failed to perform a quantitative analysis for central outcomes, due to variations in outcome reporting and measuring.STUDY DESIGN, SIZE, DURATIONA systematic search of Embase, Medline and Cochrane Register of Controlled Trials (CENTRAL) was performed with a timeframe from 1950 until February 2021, following the preferred reporting items for systematic reviews and meta-analysis (PRISMA).PARTICIPANTS/MATERIALS, SETTING, METHODSStudies reporting on any uterus-sparing intervention to treat adenomyosis, both prospective and retrospective, were eligible for inclusion. Inclusion criteria were a clear definition of diagnostic criteria for adenomyosis and the modality used to make the diagnosis, a clear description of the intervention, a follow-up time of ≥6 months, a study population of n ≥ 20, a follow-up rate of at least 80%, and English language. The population included premenopausal women with adenomyosis. Risk of bias was assessed using the Evidence Project risk of bias tool.MAIN RESULTS AND THE ROLE OF CHANCEWe included 38 studies (6 randomized controlled trials and 32 cohort studies), including 5175 participants with adenomyosis. The studies described 10 interventions and reported on 203 outcomes, including 43 classified as harms, in 29 predefined domains. Dysmenorrhoea (reported in 82%), heavy menstrual bleeding (HMB) (in 79%) and uterine volume (in 71%) were the most common outcomes. Fourteen different outcome measures were used for dysmenorrhoea and 17 for HMB. Quality of life was reported in 9 (24%) studies, patient satisfaction with treatment in 1 (3%). A clear primary outcome was stated in only 18%.LIMITATIONS, REASONS FOR CAUTIONThis review includes studies with a high risk of bias.WIDER IMPLICATIONS OF THE FINDINGSShortcomings in the definition and choice of outcomes and outcome measures limit the value of the conducted research. The development and implementation of a core outcome set (COS) for interventional studies in adenomyosis could improve research quality. This review suggests a lack of patient-centred research in adenomyosis and people with adenomyosis should be involved in the development and implementation of the COS.STUDY FUNDING/COMPETING INTERESTSNo funds specifically for this work were received. T.T. receives fees from General Electrics for lectures on ultrasound independently of this project.TRIAL REGISTRATION NUMBERThis review is registered with the International Prospective Register of Systematic Reviews (PROSPERO; registration number CRD42020177466) and the Core Outcome Measures in Effectiveness Trials (COMET) initiative (registration number 1649).

Development of a core outcome domain set for clinical research on capillary malformations (the COSCAM project).

BackgroundDue to a large variety in treatment outcomes reported in therapeutic trials and lacking patient‐relevant outcomes, it is hard to adequately compare and improve current therapies for patients with capillary malformations (CMs). The Core Outcome Set for Capillary Malformations (COSCAM) project aims to develop a core outcome set (COS) for use in future CM trials, in which we will first develop a core outcome (sub)domain set (CDS). Here, we describe the methods for the development of a CDS and present the results of the first development stage.MethodsThe COSCAM project is carried out according to the recommendations of the Cochrane Skin Core OUtcomes Set INitiative (CS‐COUSIN) and the Core Outcome Measures in Effectiveness Trials (COMET) initiative. During the first stage, we identified all potentially relevant outcome subdomains based on a systematic review, two focus group sessions and input from patient representatives of Dutch patient organizations and the COSCAM‐founding group. In stage two, we will present the subdomains in a three‐round e‐Delphi study and online consensus meeting, in which CM patients, parents/caregivers and CM experts worldwide rate the importance of the proposed subdomains, hereby finalizing the core outcome (sub)domains of the CDS.ResultsA total of 67 potential outcome subdomains were included; sixteen were previously used in the literature, 20 were proposed by Dutch patients and their parents/caregivers (n = 13) in focus group sessions and 38 were suggested by the experts of the COSCAM‐founding group. Seven were excluded because of overlap.ConclusionThe final CDS may serve as a minimum standard in future CM trials, thereby facilitating adequate comparison of treatment outcomes. After this CDS development, we will select appropriate outcome measurement instruments to measure the core outcome subdomains.