Methods for interpreting change over time in patient-reported outcome measures

Objective: To investigate patient-reported outcome (PRO) usage in phase I oncology clinical trials, including types of PRO measures and changes over time. Methods: We analyzed ClinicalTrials.gov records of phase I oncology clinical trials completed by December 2019. Results: Of all eligible trials, 2.3% (129/5,515) reported ≥1 PRO, totaling 181 instances of PRO usage. PRO usage increased over time, from 0.6% (trials initiated before 2000) to 3.4% (trials starting between 2015 and 2019). The most common PRO measures were unspecified (29%), tumor-specific (24%), and generic cancer (19%). Conclusion: Although uncommon in phase I oncology clinical trials, PRO usage is increasing over time. PRO measures were often unspecified on ClinicalTrials.gov, suggesting that more precise reporting and standardization are needed.

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Testing the Responsiveness of and Defining Minimal Important Difference (MID) Values for the CARe Burn Scales: Patient-Reported Outcome Measures to Assess Quality of Life for Children and Young People Affected by Burn Injuries, and Their Parents/Caregivers

European Journal of Burn Care ◽

10.3390/ebj2040019 ◽

2021 ◽

Vol 2 (4) ◽

pp. 249-280

Author(s):

Catrin Griffiths ◽

Philippa Tollow ◽

Danielle Cox ◽

Paul White ◽

Timothy Pickles ◽

...

Keyword(s):

Young People ◽

Burn Injury ◽

Minimal Important Difference ◽

Patient Reported Outcome Measures ◽

Young Person ◽

Patient Reported Outcome ◽

Change Over Time ◽

Patient Reported ◽

Assess Quality ◽

Over Time

The CARe Burn Scales are a portfolio of burn-specific PROMs for people affected by burns, including a Child Form (for children < 8 years (parent-proxy)), a Young Person Form (for young people aged 8–17 years), an Adult Form, and a Parent Form (for parents/carers of children aged 0–17 years). This study aimed to determine the responsiveness and minimal important difference (MID) values of the three scales developed for use in paediatric burn services and research. Participants were recruited by 15 UK Burn Services. Participants completed the appropriate CARe Burn Scale and a set of appropriate comparison validated measures, at three time points: 4 weeks (T1), 3 months (T2) and 6 months (T3) post-burn injury. Spearman’s correlation analysis and effect sizes based on Cohen’s d thresholds were reported and MID values were calculated. At baseline, 250 participants completed the Child Form, 69 completed the Young Person Form, and 320 completed the Parent Form. A total of 85–92% of participants were retained at follow up. The tested CARe Burn Scales were all responsive to change over time. MID values were created for all subscales and ranged from 2 to 11 for the Child Form, 3 to 14 for the Young Person Form and 3 to 10 for the Parent Form. The CARe Burn Scales for children, young people and parents are responsive to change over time. The scales are freely available for clinical and research use.

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FRI0215 Variability Over Time in Patient-Reported Outcome Measures in Patients with Ankylosing Spondylitis

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2015-eular.2203 ◽

2015 ◽

Vol 74 (Suppl 2) ◽

pp. 502.2-502

Author(s):

I. Essers ◽

M. Busch ◽

A. Boonen ◽

S. Ramiro ◽

D. van der Heijde ◽

...

Keyword(s):

Ankylosing Spondylitis ◽

Outcome Measures ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Patient Reported ◽

Over Time

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Patient-reported outcome measures for patients with cerebral aneurysms acquired via social media: data from a large nationwide sample

Journal of NeuroInterventional Surgery ◽

10.1136/neurintsurg-2014-011492 ◽

2014 ◽

Vol 8 (1) ◽

pp. 42-46 ◽

Cited By ~ 6

Author(s):

Michael Chen ◽

Erwin Mangubat ◽

Bichun Ouyang

Keyword(s):

Social Media ◽

Cerebral Aneurysm ◽

Outcome Measures ◽

Study Design ◽

Cerebral Aneurysms ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Physical Impairment ◽

Patient Reported ◽

Over Time

BackgroundWith greater survival rates, patient-reported outcome measures (PROMs) among survivors of ruptured cerebral aneurysm should be an increasing concern among neurointerventionalists. Prior studies were limited in scale and generalizability. Our study aims were to (1) evaluate the validity of cerebral aneurysm PROMs obtained from social media; (2) determine the persistence of PROMs over time; and (3) determine what PROMs still exist in those with no physical impairments.MethodsBy engaging national brain aneurysm support groups and using an online questionnaire modeled after the generic EQ-5D instrument, we asked respondents to classify their health in five dimensions including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression using a 3-point Likert scale.ResultsIn 2 months we received 604 responses from 46 states in the USA. Our cohort of ruptured aneurysm respondents reported PROMs similar to previously published series. Over time, headache and anxiety improved while depression, level of exercise, and return to work remained unchanged. We found that memory worsened after 2 years. Among those without any physical impairment, rates of 20.6%, 14.9%, 12.6%, and 23% were seen for significant headaches, significant memory loss, significant depression, and sense of life being negatively affected, respectively.ConclusionsDespite this novel study design, we obtained results comparable to prior studies. These results suggest that many patients with ruptured cerebral aneurysms, regardless of whether they are >2 years after the event and/or free of physical impairment, struggle with a poor quality of life. The latency, scale, and low cost of this study design may accelerate future cerebral aneurysm PROM research.

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Review of response rates over time in registry-based studies using patient-reported outcome measures

BMJ Open ◽

10.1136/bmjopen-2019-030808 ◽

2020 ◽

Vol 10 (8) ◽

pp. e030808

Author(s):

Katherine Wang ◽

Cathrine N Eftang ◽

Rune Bruhn Jakobsen ◽

Asbjørn Årøen

Keyword(s):

Outcome Measures ◽

Response Rates ◽

Patient Reported Outcome Measures ◽

Primary Outcome Measure ◽

Patient Reported Outcome ◽

Annual Reports ◽

Eligibility Criteria ◽

Time Points ◽

Patient Reported ◽

Over Time

ObjectivesGain an overview of expected response rates (RRs) to patient-reported outcome measures (PROMs) in clinical quality registry-based studies and long-term cohorts in order to better evaluate the validity of registries and registry-based studies. Examine the trends of RRs over time and how they vary with study type, questionnaire format, and the use of reminders.DesignLiterature review with systematic search.Data sourcesPubMed, MEDLINE, EMBASE, kvalitetsregistre.no, kvalitetsregister.se and sundhed.dk.Eligibility criteriaArticles in all areas of medical research using registry-based data or cohort design with at least two follow-up time points collecting PROMs and reporting RRs. Annual reports of registries including PROMs that report RRs for at least two time points.Primary outcome measureRRs to PROMs.ResultsA total of 10 articles, 12 registry reports and 6 registry articles were included in the review. The overall RR at baseline was 75%±22.1 but decreased over time. Cohort studies had a markedly better RR (baseline 97%±4.7) compared with registry-based data at all time points (baseline 72%±21.8). For questionnaire formats, paper had the highest RR at 86%±19.4, a mix of electronic and paper had the second highest at 71%±15.1 and the electronic-only format had a substantially lower RR at 42%±8.7. Sending one reminder (82%±16.5) or more than one reminder (76%±20.9) to non-responders resulted in a higher RR than sending no reminders (39%±6.7).ConclusionsThe large variation and downward trend of RRs to PROMs in cohort and registry-based studies are of concern and should be assessed and addressed when using registry data in both research and clinical practice.

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