Patient-centered outcomes used in pediatric focused manual therapies research studies: a secondary data analysis of a systematic review

Abstract Background Patient-reported outcome measurements (PROM) are instruments that seek a patient’s health or functional status. Inclusion of standardized PROMs in research studies and clinical practice provides a more comprehensive understanding of an intervention from the patient’s viewpoint. This secondary analysis identified PROM usage and appropriateness of references for property measurements from clinical trials included in a recent systematic review of pediatric manual therapy. Methods All included manuscripts within a recent systematic review had two authors extract PROM and associated property measurement data, including the property measurements supporting citations. Inclusion criteria for the articles were published clinical trials (observational or experimental) of pediatric children receiving manual therapy (any profession) for any condition between 2001-March 2018. For each PROM’s associated property measurement, two authors used the COSMIN study design checklist to appraise the quality of the cited study to evaluate the property measurement. Results Of the 50 manuscripts included in the systematic review, 20 manuscripts reported the use of 52 PROMs. Of the 52 PROMs assessed, 31 did not make a statement about the instrument’s property measurement, 7 PROMs had property measurements stated but not referenced, 4 PROMs stated that the property measurement information was unknown, and 10 had property measurement data with reference(s). These 10 PROMs with referenced property measurements were from 7 unique PROMs: constipation assessment scale, satisfaction visual analog scale (VAS), crying time diary, sleep diary, fear avoidance belief questionnaire (FABQ), pain VAS, and autism treatment evaluation checklist. The assessment of the referenced property measurements found that several property measurement’s dimensions had not been assessed and those that had were evaluated were done so with poor or fair standards. Conclusions This secondary analysis finds that clinical studies of pediatric manual therapy lack consistent use of PROMs with high quality property measurements. Further research to establish and implement PROMs to be used in future research studies and in clinical settings should become a priority for professions using manual therapy in children.

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Measurement properties of patient-reported outcome measures (PROMs) in hyperhidrosis: a systematic review

Quality of Life Research ◽

10.1007/s11136-021-02958-3 ◽

2021 ◽

Author(s):

Michaela Gabes ◽

Helge Knüttel ◽

Gesina Kann ◽

Christina Tischer ◽

Christian J. Apfelbacher

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Measurement Properties ◽

High Quality ◽

High Quality Evidence ◽

Patient Reported ◽

Quality Evidence

Abstract Purpose To critically appraise, compare and summarize the quality of all existing PROMs that have been validated in hyperhidrosis to at least some extend by applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Thereby, we aim to give a recommendation for the use of PROMs in future clinical trials in hyperhidrosis. Methods We considered studies evaluating, describing or comparing measurement properties of PROMs as eligible. A systematic literature search in three big databases (MEDLINE, EMBASE and Web of Science) was performed. We assessed the methodological quality of each included study using the COSMIN Risk of Bias checklist. Furthermore, we applied predefined quality criteria for good measurement properties and finally, graded the quality of the evidence. Results Twenty-four articles reporting on 13 patient-reported outcome measures were included. Three instruments can be further recommended for use. They showed evidence for sufficient content validity and moderate- to high-quality evidence for sufficient internal consistency. The methodological assessment showed existing evidence gaps for eight other PROMs, which therefore require further validation studies to make an adequate decision on their recommendation. The Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) and the short-form health survey with 36 items (SF-36) were the only questionnaires not recommended for use in patients with hyperhidrosis due to moderate- to high-quality evidence for insufficient measurement properties. Conclusion Three PROMs, the Hyperhidrosis Quality of Life Index (HidroQoL), the Hyperhidrosis Questionnaire (HQ) and the Sweating Cognitions Inventory (SCI), can be recommended for use in future clinical trials in hyperhidrosis. Results obtained with these three instruments can be seen as trustworthy. Nevertheless, further validation of all three PROMs is desirable. Systematic review registration PROSPERO CRD42020170247

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The impact of patient-reported outcome (PRO) data from clinical trials: a systematic review and critical analysis

Health and Quality of Life Outcomes ◽

10.1186/s12955-019-1220-z ◽

2019 ◽

Vol 17 (1) ◽

Cited By ~ 12

Author(s):

Samantha Cruz Rivera ◽

Derek G. Kyte ◽

Olalekan Lee Aiyegbusi ◽

Anita L. Slade ◽

Christel McMullan ◽

...

Keyword(s):

Systematic Review ◽

Clinical Trial ◽

Clinical Trials ◽

Case Studies ◽

Real World ◽

Clinical Trial Data ◽

Research Impact ◽

Trial Data ◽

Patient Reported ◽

The Impact

Abstract Background Patient-reported outcomes (PROs) are commonly collected in clinical trials and should provide impactful evidence on the effect of interventions on patient symptoms and quality of life. However, it is unclear how PRO impact is currently realised in practice. In addition, the different types of impact associated with PRO trial results, their barriers and facilitators, and appropriate impact metrics are not well defined. Therefore, our objectives were: i) to determine the range of potential impacts from PRO clinical trial data, ii) identify potential PRO impact metrics and iii) identify barriers/facilitators to maximising PRO impact; and iv) to examine real-world evidence of PRO trial data impact based on Research Excellence Framework (REF) impact case studies. Methods Two independent investigators searched MEDLINE, EMBASE, CINAHL+, HMIC databases from inception until December 2018. Articles were eligible if they discussed research impact in the context of PRO clinical trial data. In addition, the REF 2014 database was systematically searched. REF impact case studies were included if they incorporated PRO data in a clinical trial. Results Thirty-nine publications of eleven thousand four hundred eighty screened met the inclusion criteria. Nine types of PRO trial impact were identified; the most frequent of which centred around PRO data informing clinical decision-making. The included publications identified several barriers and facilitators around PRO trial design, conduct, analysis and report that can hinder or promote the impact of PRO trial data. Sixty-nine out of two hundred nine screened REF 2014 case studies were included. 12 (17%) REF case studies led to demonstrable impact including changes to international guidelines; national guidelines; influencing cost-effectiveness analysis; and influencing drug approvals. Conclusions PRO trial data may potentially lead to a range of benefits for patients and society, which can be measured through appropriate impact metrics. However, in practice there is relatively limited evidence demonstrating directly attributable and indirect real world PRO-related research impact. In part, this is due to the wider challenges of measuring the impact of research and PRO-specific issues around design, conduct, analysis and reporting. Adherence to guidelines and multi-stakeholder collaboration is essential to maximise the use of PRO trial data, facilitate impact and minimise research waste. Trial registration Systematic Review registration PROSPERO CRD42017067799.

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Patient-Reported Outcome Measures for Use in Clinical Trials and Clinical Practice in Inflammatory Bowel Diseases: A Systematic Review

Clinical Gastroenterology and Hepatology ◽

10.1016/j.cgh.2017.10.019 ◽

2018 ◽

Vol 16 (5) ◽

pp. 648-663.e3 ◽

Cited By ~ 38

Author(s):

Marin J. de Jong ◽

Roxanne Huibregtse ◽

Ad A.M. Masclee ◽

Daisy M.A.E. Jonkers ◽

Marie J. Pierik

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Clinical Practice ◽

Outcome Measures ◽

Inflammatory Bowel Diseases ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Patient Reported ◽

Bowel Diseases ◽

Inflammatory Bowel

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A Systematic Review of Patients’ Treatment Satisfaction and/or Preference Patient-Reported Oucomes Measures Used in Clinical Trials

Value in Health ◽

10.1016/j.jval.2013.08.1673 ◽

2013 ◽

Vol 16 (7) ◽

pp. A596

Author(s):

S. Pease ◽

I. Savre ◽

K. Rudell ◽

C. Perret ◽

T. Symonds

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Treatment Satisfaction ◽

Patient Reported

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Integration of subclassification strategies in randomised controlled clinical trials evaluating manual therapy treatment and exercise therapy for non-specific chronic low back pain: a systematic review

British Journal of Sports Medicine ◽

10.1136/bjsm.2009.063289 ◽

2009 ◽

Vol 44 (14) ◽

pp. 1054-1062 ◽

Cited By ~ 54

Author(s):

K. V. Fersum ◽

W. Dankaerts ◽

P. B. O'Sullivan ◽

J. Maes ◽

J. S. Skouen ◽

...

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Low Back Pain ◽

Back Pain ◽

Manual Therapy ◽

Chronic Low Back Pain ◽

Low Back ◽

Controlled Clinical Trials ◽

Randomised Controlled ◽

Therapy Treatment

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Barriers to participation in clinical trials of cancer: a meta-analysis and systematic review of patient-reported factors

The Lancet Oncology ◽

10.1016/s1470-2045(06)70576-9 ◽

2006 ◽

Vol 7 (2) ◽

pp. 141-148 ◽

Cited By ~ 309

Author(s):

Edward J Mills ◽

Dugald Seely ◽

Beth Rachlis ◽

Lauren Griffith ◽

Ping Wu ◽

...

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Meta Analysis ◽

Barriers To Participation ◽

Patient Reported

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Gaps in patient-reported outcome measures in randomized clinical trials of cardiac catheter ablation: a systematic review

European Heart Journal - Quality of Care and Clinical Outcomes ◽

10.1093/ehjqcco/qcaa022 ◽

2020 ◽

Vol 6 (4) ◽

pp. 234-242

Author(s):

Yang Chen ◽

Myura Nagendran ◽

Manuel Gomes ◽

Peter V Wharton ◽

Rosalind Raine ◽

...

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Catheter Ablation ◽

Outcome Measures ◽

Comparative Effectiveness ◽

Randomized Clinical Trials ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Cardiac Catheter ◽

Patient Reported

Abstract The aim of this systematic review was to evaluate randomized clinical trials (RCTs) of cardiac catheter ablation (CCA) and to assess the prevalence, characteristics and reporting standards of clinically relevant patient-reported outcome measures (PROMs). Electronic database searches of Medline, Embase, CENTRAL, and the WHO Trial Registry were conducted in March 2019. The study protocol was registered on PROSPERO (CRD42019133086). Of 7125 records identified, 237 RCTs were included for analysis, representing 35 427 patients with a mean age of 59 years. Only 43 RCTs (18%) reported PROMs of which 27 included a generic PROM that measured health-related quality of life (HRQL) necessary to conduct comparative effectiveness research. There was notable under-representation of certain patient groups—only 31% were women and only 8% were of non-Caucasian ethnicity, in trials which reported such data. The reporting standard of PROMs was highly variable with 8–62% adherence against CONSORT PRO-specific items. In summary, PROMs play a crucial role in determining the clinical and cost-effectiveness of treatments which primarily offer symptomatic improvement, such as CCA. Their underuse significantly limits evaluation of the comparative effectiveness of treatments. Using CCA as an exemplar, there are additional issues of infrequent assessment, poor reporting and under-representation of many population groups. Greater use of PROMs, and specifically validated HRQL questionnaires, is paramount in giving patients a voice in studies, generating more meaningful comparisons between treatments and driving better patient-centred clinical and policy-level decision-making.

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