Dataset Information

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

ABSTRACT:

Objectives

To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness.

Study design

We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity.

Results

Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain.

Conclusions

This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

SUBMITTER: Miller TL

PROVIDER: S-EPMC8882140 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature

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Publications

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

Miller Talya L TL Schuchard Julia J Carle Adam C AC Forrest Christopher B CB Kappelman Michael D MD

The Journal of pediatrics 20211103

<h4>Objectives</h4>To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness.<h4>Study design</h4>We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical tri ...[more]

PMID: 34740588

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Project description:BackgroundDespite the acceptability and efficacy of e-patient-reported outcome (ePRO) systems, implementation in routine clinical care remains challenging.ObjectiveThis pragmatic trial implemented the PROMPT-Care (Patient Reported Outcome Measures for Personalized Treatment and Care) web-based system into existing clinical workflows and evaluated its effectiveness among a diverse population of patients with cancer.MethodsAdult patients with solid tumors receiving active treatment or follow-up care in four cancer centers were enrolled. The PROMPT-Care intervention supported patient management through (1) monthly off-site electronic PRO physical symptom and psychosocial well-being assessments, (2) automated electronic clinical alerts notifying the care team of unresolved clinical issues following two consecutive assessments, and (3) tailored online patient self-management resources. Propensity score matching was used to match controls with intervention patients in a 4:1 ratio for patient age, sex, and treatment status. The primary outcome was a reduction in emergency department presentations. Secondary outcomes were time spent on chemotherapy and the number of allied health service referrals.ResultsFrom April 2016 to October 2018, 328 patients from four public hospitals received the intervention. Matched controls (n=1312) comprised the general population of patients with cancer, seen at the participating hospitals during the study period. Emergency department visits were significantly reduced by 33% (P=.02) among patients receiving the intervention compared with patients in the matched controls. No significant associations were found in allied health referrals or time to end of chemotherapy. At baseline, the most common patient reported outcomes (above-threshold) were fatigue (39%), tiredness (38.4%), worry (32.9%), general wellbeing (32.9%), and sleep (24.1%), aligning with the most frequently accessed self-management domain pages of physical well-being (36%) and emotional well-being (23%). The majority of clinical feedback reports were reviewed by nursing staff (729/893, 82%), largely in response to the automated clinical alerts (n=877).ConclusionsAlgorithm-supported web-based systems utilizing patient reported outcomes in clinical practice reduced emergency department presentations among a diverse population of patients with cancer. This study also highlighted the importance of (1) automated triggers for reviewing above-threshold results in patient reports, rather than passive manual review of patient records; (2) the instrumental role nurses play in managing alerts; and (3) providing patients with resources to support guided self-management, where appropriate. Together, these factors will inform the integration of web-based PRO systems into future models of routine cancer care.Trial registrationAustralian New Zealand Clinical Trials Registry ACTRN12616000615482; https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=370633.International registered report identifier (irrid)RR2-10.1186/s12885-018-4729-3.

Project description:PurposeTo analyze time to completion of preoperative legacy patient-reported outcomes (PROs) and more recent computer adaptive Patient-Reported Outcomes Measurement Information System (PROMIS) questionnaires in patients with symptomatic femoroacetabular impingement syndrome undergoing primary hip arthroscopy.MethodsA retrospective analysis was conducted on patients undergoing hip arthroscopy by a single fellowship-trained hip arthroscopist. Inclusion criteria were patients undergoing primary arthroscopic hip surgery and completion of at least 1 legacy PRO or PROMIS questionnaire at the preoperative time point. Exclusion criteria were history of contralateral or ipsilateral hip surgery, non-English-speaking patients, patients who completed PROs by phone or by paper form, and patients who did not complete preoperative PROs. Legacy PROs included modified Harris Hip Score (mHHS), Hip Outcome Score (HOS), International Hip Outcome Tool (iHOT-12), and Hip Pain Visual Analog Scale (VAS-Pain). PROMIS PROs included Physical Function (PROMIS-PF), Pain Interference (PROMIS-PI), and Depression (PROMIS-D). Only preoperative PROs were included in the analysis. Completion time was calculated using the questionnaire start and stop time reported by the survey collecting software. The median and interquartile range of each PRO were reported for analysis of central tendency and statistical dispersion, respectively.ResultsA total of 1,901 patients and 269 patients were included in the legacy and PROMIS groups, respectively. The median time required for completion of each PRO in (minutes: seconds) format was as follows: mHHS (1:29), HOS (3:58), iHOT (2:11), VAS-Pain (0:32), PROMIS-PF (0:46), PROMIS-PI (0:37), and PROMIS-D (0:43). The interquartile range of the middle 50% of respondents was as follows; mHHS (0:58), HOS (2:46), iHOT (1:22), VAS-Pain (0:28), PROMIS-PI (0:19), PROMIS-D (0:29), and PROMIS-PF (0:20).ConclusionsThis study supports that preoperative PROMIS forms require less time to complete than preoperative legacy PROs and are not significantly influenced by age, race, or workers compensation status.Level of evidenceLevel IV, case series.

Project description:BackgroundPatient-reported experience measures are tools used to gather feedback from patients about their experiences of healthcare services, which are crucial for improving the quality of care from the perspective of patients. The aim of this systematic review was to identify surgery-related patient-reported experience measures, evaluate their psychometric properties, appraise and identify recurring themes within qualitative studies on surgical care, and identify potential bias in study designs.MethodsPubMed, MEDLINE, Embase, the Cumulative Index to Nursing and Allied Health Literature database, and the Cochrane Library, along with clinical trial registries, were searched for articles on surgery-specific patient-reported experience measures and qualitative studies on patients' experiences up to 21 September 2023. Manual coding was used for theme identification and grouping based on thematic synthesis principles. Joanna Briggs Institute tools were used for risk-of-bias assessment and a revised version of the COnsensus-based Standards for the selection of health Measurement INstruments checklist was used for appraisal of patient-reported experience measures.ResultsA total of 14 studies met the inclusion criteria, identifying seven patient-reported experience measures. Key patient experience themes included communication with healthcare providers, care setting environment, overall satisfaction, pre-admission information, coordination of care, waiting time, surgical experience, post-surgery support, impact on life, and healthcare information and technology management. Internal consistency was reported adequately across all patient-reported experience measures. Other psychometric properties were questionable.ConclusionInadequate psychometric evaluations of some patient-reported experience measures in surgery highlight the need for rigorous validity and reliability assessments. Identification of thematic patterns emphasizes the importance of ongoing research to explore patients' experiences in surgical contexts. Clinical staff can use this information to enhance communication, reduce waiting time, and improve the overall patient experience by addressing highlighted areas.

Project description:ObjectiveTo systematically describe the use and outcomes of Patient-Reported Outcomes Measurement Information System (PROMIS) measures in clinical studies in populations with stroke.Data sourcesA systematic search on the use of PROMIS measures in clinical stroke studies in 9 electronic databases.Study selectionStudies had to be original, reporting on outcome data using PROMIS measures in populations with stroke (ischemic and/or hemorrhagic), from January 1st, 2007. Initially, 174 unique studies met the inclusion criteria. In 2 steps, titles, abstracts and full-text articles were screened for eligibility (2 authors independently).Data extractionFrom the selected articles, study characteristics, type of PROMIS measures, and its outcomes were extracted by 2 authors independently. The authors discussed their views to achieve consensus. A third author was consulted if necessary.Data synthesisIn total, 27 studies (24,366 patients) were included, predominantly from the United States (22); most study populations were hospital-based (20); the number of patients ranged from 30-3283. In general, patients had no or mild symptoms (median modified Rankin scale 1). Two different generic PROMIS measures were reported (PROMIS Global Health, PROMIS 29) and 9 PROMIS measures focusing on specific domains (sleep, pain, physical functioning, self-efficacy, satisfaction with social roles, depression, anxiety, cognition, fatigue). These match the International Classification of Functioning, Disability, and Health (ICF) domains mentioned in the Core Set for Stroke. The measures were administered 1-55 months after stroke. Outcome data are provided. Pooling of data was not achieved because of a large variety in study characteristics (inclusion criteria, follow-up moments, data processing).ConclusionsThe PROMIS measures in this review could be relevant from a patient's perspective, covering ICF core set domains for patients with stroke. The large variety in study characteristics hampers comparisons across populations. Many different outcome measures are used to report results of stroke rehabilitation studies.

Project description:PurposeTo evaluate and compare the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) computerized adaptive test and PROMIS Pain Interference (PI) instruments versus legacy patient-reported outcome measures (PROMs) in patients undergoing hip arthroscopy for femoroacetabular impingement syndrome at 1-year follow-up.MethodsPatients undergoing primary hip arthroscopy for the treatment of femoroacetabular impingement syndrome without concomitant procedures performed by a single surgeon between August 2018 and January 2019 were identified. The PROMIS PF score, PROMIS PI score, Hip Outcome Score-Activities of Daily Living Subscale (HOS-ADL), Hip Outcome Score-Sports Subscale (HOS-SS), modified Harris Hip Score (mHHS), International Hip Outcome Tool 12 (iHOT-12), and visual analog scale (VAS) pain score were obtained preoperatively and at 6 months and 1 year postoperatively. Bivariate correlation analyses between PROMIS and legacy PROMs were performed. The floor and ceiling effects, responsiveness, and relative efficiency (RE) of each PROM were calculated.ResultsThis study included 124 patients with an average age of 32.7 ± 12.3 years. The PROMIS PF score showed very good correlations with the HOS-ADL (r = 0.73, P < .001) and mHHS (r = 0.70, P < .001) and good correlations with the HOS-SS (r = 0.62, P < .001), iHOT-12 score (r = 0.62, P < .001), and VAS pain score (r = -0.64, P < .001). The PROMIS PI score showed very good inverse correlations with the HOS-ADL (r = -0.72, P < .001) and mHHS (r = -0.79, P < .001) and good correlations with the HOS-SS (r = -0.64, P < .001), iHOT-12 score (r = -0.65, P < .001), and VAS pain score (r = 0.65, P < .001). No floor effect was observed for any measure. Ceiling effects were not observed in the PROMIS PROMs but were detected for the HOS-ADL (16.1%) and mHHS (19.3%). The effect size was large for all outcomes: iHOT-12 score, d = 1.77; HOS-ADL, d = 1.37; HOS-SS, d = 1.45; PROMIS PI score, d = 1.05; and PROMIS PF score, d = 1.01. The iHOT-12 score was more responsive than the PROMIS PI score (RE = 1.69), PROMIS PF score (RE = 1.75), HOS-ADL (RE = 1.29), and HOS-SS (RE = 1.22).ConclusionsAt 1-year follow-up, PROMIS PROMs displayed very good correlations with legacy PROMs. However, PROMIS PROMs had lower effect sizes and were not as responsive as legacy PROMs.Level of evidenceLevel II, development of diagnostic criteria on the basis of consecutive patients.

Dataset Information

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

Objectives

Study design

Results

Conclusions

Publications

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

Similar Datasets

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