Are OMERACT recommendations followed in clinical trials on fibromyalgia? A systematic review of patient-reported outcomes and their measures.
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ABSTRACT: Purpose
Patient-Reported Outcomes (PROs) and its measures (PROMs) are key to outcome assessment in Fibromyalgia (FM) trials. The aim of this review was to investigate which domains and instruments were assessed in recent FM trials and to compare them to recommendations by the Outcome Measures in Rheumatology (OMERACT) initiative. In addition, we investigated the overlap with a generic health assessment approach, i.e. eight domains suggested by the Patient-Reported Outcome Measurement Information System® (PROMIS®).Methods
In compliance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, a systematic literature search in scientific databases including PubMed, PsycInfo, and Embase was conducted to identify studies that assessed at least two dimensions of health-related quality of life (HRQoL) from 2015 to June 2022. Non-randomized and randomized controlled trials were included in the analysis. We extracted PROs and PROMs used in each study.Results
From 1845 identified records, 107 records out of 105 studies met the inclusion criteria. Studies investigated 50 PROs using 126 different PROMs. Most frequently assessed domains were pain, depression, fatigue, and anxiety (> 95% of the studies). The disease-specific FIQ was the most frequently applied PROM (82%). Overall, only 9% of the studies covered all domains deemed mandatory by OMERACT. Very few studies covered all eight generic health domains suggested by PROMIS.Conclusion
The majority of trials covered most OMERACT domains or generic PROMIS health domains. There was, however, great variability in the instruments used to assess the domains, which points at a limited degree of standardization in the field.
SUBMITTER: Dohmen A
PROVIDER: S-EPMC10172242 | biostudies-literature | 2023 Jun
REPOSITORIES: biostudies-literature
Publications
Are OMERACT recommendations followed in clinical trials on fibromyalgia? A systematic review of patient-reported outcomes and their measures.
Döhmen Annika A Kock Milan M Fischer Felix F Rose Matthias M Rose Matthias M Obbarius Alexander A Klapproth Christoph Paul CP
Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation 20221001 6
<h4>Purpose</h4>Patient-Reported Outcomes (PROs) and its measures (PROMs) are key to outcome assessment in Fibromyalgia (FM) trials. The aim of this review was to investigate which domains and instruments were assessed in recent FM trials and to compare them to recommendations by the Outcome Measures in Rheumatology (OMERACT) initiative. In addition, we investigated the overlap with a generic health assessment approach, i.e. eight domains suggested by the Patient-Reported Outcome Measurement Inf ...[more]
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Project description:IntroductionThe subjective assessment of the adequacy of informed consent for clinical trials, and the potential difficulties associated with it, has led several studies to develop objective measures of informed consent for clinical trials. These objective measures of informed consent are often specific to a particular population or clinical condition and largely focus on understanding of (some or all of) the key elements of informed consent. Many of the developed tools are study-specific, but some validated measures exist. Of these validated measures, those which are reported by participants are of particular interest. Whether these objective tools conceptualize and measure informed consent in the same way is not known. As such, it is not clear whether meta-analyzing data from studies reporting different tools is worthwhile. The aim of this systematic review was to critically appraise the evidence on the overall conceptualisation and item content of validated patient reported measures of informed consent for clinical trials, and to identify core domains of potential importance for informed consent.MethodsA systematic search of the literature was conducted to identify relevant articles that described the development, and/or validation, of patient-reported measures of adequacy of informed consent for randomised controlled trials. Data was synthesised by classifying the items identified into domains and sub-domains which were determined by the nomenclature reported in included studies. Both for descriptions of included studies and of the instruments reported in those studies, descriptive statistics were used to describe general information and instrument detail. A narrative synthesis of the instruments and their inter-related domains and subdomains was conducted to identify areas of both convergence and divergence.ResultsThe search identified 8193 citations. After screening titles and abstracts, 29 full text articles were retrieved for further assessment. Of these 29, 14 complied with our pre-specified inclusion criteria with 15 not being eligible. Of the 14 instruments, three explicitly reported a theoretical or conceptual framework underpinning their development, a further three implicitly referred to the 'conceptual dimensions of informed consent' or 'principles of research ethics' as informing their development and eight reported no guiding theoretical framework. Only three of the 14 studies reported patient or public involvement in the development of the tool. One hundred and seventy nine items were included across the 14 instruments. The primary focus of the instruments was on understanding. Five core domains were identified which included: Autonomy; Consequences; Expectations; Purpose; and Individualisation. There was substantial variability in the coverage of different domains across measures.ConclusionsThis study demonstrated the variability in the theoretical underpinning, development and domain coverage of existing patient-reported measures of informed consent for clinical trials. The conceptualisation of informed consent could benefit from being extended from a narrow focus on understanding to include broader considerations of decision-making. Meaningful involvement of potential trial participants during development of measures critical for tool relevance is also lacking. The identification of the key domains relevant to all stakeholders which could be measured to assess the informed consent process for clinical trials is needed.
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Project description:BACKGROUND: Fibromyalgia is a chronic health condition characterized by widespread musculoskeletal pain, multiple tender points on physical examination, generalized muscular aching, stiffness, fatigue, nonrestorative sleep pattern, cognitive dysfunction, and mood disturbance. Recently, the Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) Fibromyalgia Syndrome Workshop ranked and prioritized the domains that should be consistently measured in fibromyalgia clinical trials, specifically, pain, generic health-related quality of life, fatigue, sleep quality, and physical function. The focus of these deliberations was exclusively on adult patients, and to our knowledge, these domains have not been previously tested within a multidimensional framework in children and adolescents with fibromyalgia. METHODS: An analysis to determine the feasibility, reliability, and validity of the PedsQL 4.0 (Pediatric Quality of Life Inventory) Generic Core Scales, PedsQL Multidimensional Fatigue Scale, and PedsQL Rheumatology Module Pain and Hurt Scale as patient-reported outcome (PRO) measures for pediatric patients with fibromyalgia. The PedsQL Scales were completed by 59 families in a pediatric rheumatology clinic in a large children's hospital. RESULTS: The PedsQL evidenced minimal missing responses (0.53% patient self-report, 0.70% parent proxy-report), achieved excellent reliability for the Generic Core Scales Total Scale Score (alpha = 0.88 patient self-report, 0.87 parent proxy-report), the Multidimensional Fatigue Scale Total Scale Score (alpha = 0.94 patient self-report, 0.94 parent proxy-report), and acceptable reliability for the 4-item Rheumatology Module Pain and Hurt Scale (alpha = 0.68 patient self-report, 0.75 parent proxy-report). The PedsQL Generic Core Scales and Multidimensional Fatigue Scale significantly distinguished between pediatric patients with fibromyalgia and healthy children. Pediatric patients with fibromyalgia self-reported severely impaired physical and psychosocial functioning, significantly lower on most dimensions when compared to pediatric cancer patients receiving cancer treatment, and significantly lower on all dimensions than pediatric patients with other rheumatologic diseases. Patients with fibromyalgia self-reported significantly greater pain and fatigue than pediatric patients with other rheumatologic conditions, and generally more fatigue than pediatric patients receiving treatment for cancer. CONCLUSION: The results demonstrate the excellent measurement properties of the PedsQL Scales in fibromyalgia. These PedsQL Scales measure constructs consistent with the recommended OMERACT Fibromyalgia Syndrome Workshop domains. The findings highlight the severely impaired HRQOL of pediatric patients with fibromyalgia. Regular monitoring of pediatric patients with fibromyalgia will help identify children and adolescents at risk for severely impaired HRQOL. These PedsQL Scales are appropriate outcome measures for clinical trials and health services research for pediatric patients with fibromyalgia.
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Project description:BackgroundLymphoma treatment can lead to long-term consequences such as fatigue, infertility and organ damage. In clinical trials, survival outcomes, clinical response and toxicity are extensively reported while the assessment of treatment on quality of life (QoL) and symptoms is often lacking.ObjectiveWe evaluated the use and frequency of patient-reported outcome (PRO) instruments used in randomized controlled trials (RCTs) for Hodgkin lymphoma (HL) and their consistency of reporting.MethodsMEDLINE, CENTRAL and trial registries for RCTs investigating HL were systematically searched from 01/01/2016 to 31/05/2022. Following trial selection, trial, patient characteristics and outcome data on the use of PRO measures (PROMs) and reporting of PROs using a pre-defined extraction form were extracted. To assess reporting consistency, trial registries, protocols and publications were compared.ResultsWe identified 4,222 records. Following screening, a total of 317 reports were eligible for full-text evaluation. One hundred sixty-six reports of 51 ongoing/completed trials were included, of which 41% of trials were completed and 49% were ongoing based on registry entries. Full-text or abstract were available for 33 trials. Seventy percent of trials were conducted in the newly diagnosed disease setting, the majority with advanced HL. In 32 trials with published follow-up data, the median follow-up was 5.2 years. Eighteen (35%) completed/ongoing trials had mentioned PRO assessment in registry entries, protocol or publications. Twelve trials (67%) had published results and only 6 trials (50%) reported on PROs in part with the exception of 1 trial where PROs were evaluated as secondary/exploratory outcome. The most referenced global PROM was the EORTC-QLQ-C30 (12 studies), the EQ-5D (3 studies) and the FACT-Neurotoxicity (3 studies). FACT-Lymphoma, a disease-specific PROM for non-HL was mentioned in one ongoing trial. None of the trials referenced the EORTC QLQ-HL27, another disease-specific PROM developed specifically for HL patient's QoL assessment.DiscussionsOnly one-third of RCTs in HL report PROs as an outcome and only half present the outcome in subsequent publications, showcasing the underreporting of PROs in trials. Disease-specific PROMs are underutilized in the assessment of QoL in HL patients. Guidance on the assessment of PROs is needed to inform on comprehensive outcomes important to patients.Systematic review registrationhttps://www.crd.york.ac.uk/PROSPERO/display_record.php?RecordID=391552, identifier CRD42023391552.
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Project description:BackgroundNeurofibromatosis type 2-related schwannomatosis is a genetic disease characterized by the development of bilateral vestibular schwannomas, ependymomas, meningiomas, and cataracts. Mild to profound hearing loss and tinnitus are common symptoms reported by individuals with neurofibromatosis type 2. While tinnitus is known to have a significant and negative impact on the quality of life of individuals from the general population, the impact on individuals with neurofibromatosis type 2 is unknown. Consensus regarding the selection of suitable patient-reported outcome measures for assessment could advance further research into tinnitus in neurofibromatosis type 2 patients. The purpose of this work is to achieve a consensus recommendation by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration for patient-reported outcome measures used to evaluate quality of life in the domain of tinnitus for neurofibromatosis type 2 clinical trials.MethodsThe Response Evaluation in Neurofibromatosis and Schwannomatosis Patient-Reported Outcomes Communication Subgroup systematically evaluated patient-reported outcome measures of quality of life in the domain of tinnitus for individuals with neurofibromatosis type 2 using previously published Response Evaluation in Neurofibromatosis and Schwannomatosis rating procedures. Of the 19 identified patient-reported outcome measures, 3 measures were excluded because they were not validated as an outcome measure or could not have been used as a single outcome measure for a clinical trial. Sixteen published patient-reported outcome measures for the domain of tinnitus were scored and compared on their participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials.ResultsThe Tinnitus Functional Index was identified as the most highly rated measure for the assessment of tinnitus in populations with neurofibromatosis type 2, due to strengths in the areas of item content, psychometric properties, feasibility, and available scores.DiscussionResponse Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.
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Project description:BackgroundThere is a considerable amount of research showing an association between continuity of care and improved health outcomes. However, the methods used in most studies examine only the pattern of interactions between patients and clinicians through administrative measures of continuity. The patient experience of continuity can also be measured by using patient reported experience measures. Unlike administrative measures, these can allow elements of continuity such as the presence of information or how joined up care is between providers to be measured. Patient experienced continuity is a marker of healthcare quality in its own right. However, it is unclear if, like administrative measures, patient reported continuity is also linked to positive health outcomes.MethodsCohort and interventional studies that examined the relationship between patient reported continuity of care and a health outcome were eligible for inclusion. Medline, EMBASE, CINAHL and the Cochrane Library were searched in April 2021. Citation searching of published continuity measures was also performed. QUIP and Cochrane risk of bias tools were used to assess study quality. A box-score method was used for study synthesis.ResultsNineteen studies were eligible for inclusion. 15 studies measured continuity using a validated, multifactorial questionnaire or the continuity/co-ordination subscale of another instrument. Two studies placed patients into discrete groups of continuity based on pre-defined questions, one used a bespoke questionnaire, one calculated an administrative measure of continuity using patient reported data. Outcome measures examined were quality of life (n = 11), self-reported health status (n = 8), emergency department use or hospitalisation (n = 7), indicators of function or wellbeing (n = 6), mortality (n = 4) and physiological measures (n = 2). Analysis was limited by the relatively small number of hetrogenous studies. The majority of studies showed a link between at least one measure of continuity and one health outcome.ConclusionWhilst there is emerging evidence of a link between patient reported continuity and several outcomes, the evidence is not as strong as that for administrative measures of continuity. This may be because administrative measures record something different to patient reported measures, or that studies using patient reported measures are smaller and less able to detect smaller effects. Future research should use larger sample sizes to clarify if a link does exist and what the potential mechanisms underlying such a link could be. When measuring continuity, researchers and health system administrators should carefully consider what type of continuity measure is most appropriate.
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Project description:The aim of this review was to summarize literature in view of patient-reported outcome (PRO) instruments for glaucoma and provide guidance on how outcomes are best assessed based on evidence about their content and validity. A systematic literature review was performed on papers describing the developmental process and/or psychometric properties of glaucoma or vision-specific PRO-instruments. Each of them was assessed on their adherence to a framework of quality criteria. Fifty-three articles were identified addressing 27 PRO-instruments. In all, 18 PRO's were developed for glaucoma and 9 for diverse ophthalmologic conditions. Seven instruments addressed functional status, 11 instruments quality of life and 9 instruments disease and treatment-related factors. Most of the instruments demonstrated only partially adherence to predefined quality standards. The tools for assessing functional status were of poor quality, while the Glaucoma Quality of Life Questionnaire and the Vision Quality of Life Index were well-developed QoL measures, yet only validated using classical techniques. The Rasch-scaled QoL-tools, IVI and VCM1 need to improve their item-content for glaucoma patients. The questionnaires to measure adherence should improve their validity and the Treatment Satisfaction Survey for Intra Ocular Pressure pops out as the highest quality tool for measuring topical treatment side effects. This review revealed that most PRO-instruments demonstrated poor developmental quality, more specifically a lack of conceptual framework and item generation strategies not involving the patients' perspective. Psychometric characteristics were mostly tested using classical validation techniques.
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