Dataset Information

Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study.

ABSTRACT:

Background

The Food and Drug Administration considers patient-reported outcome measures (PROMs) an essential part of clinical research studies for approval of new drugs and new indications for existing drugs. GALAXY evaluated the feasibility of electronic PROMs (ePROMS) to conduct a comprehensive evaluation of gastrointestinal (GI) symptoms in persons with cystic fibrosis (pwCF).

Methods

Three validated GI ePROMs (PAC-SYM, PAGI-SYM and PAC-QOL) were combined with a Stool-Specific questionnaire to make up the GALAXY ePROMs and administered prospectively across 26 CF centers in the United States. The ePROMs were completed at enrollment visit and then electronically at weeks 1, 2 and 4. PwCF at least 2 years and older were eligible for the study. Reminders were only provided by the mobile application during the study window.

Results

There were 402 participants enrolled in GALAXY. Of those, 169 (42%) were under 18 years old and 193 (48%) were female. The proportion of all follow-up weeks with at least 1 ePROM fully completed was 80%, slightly higher in those ≥18 years of age (82.5%) compared to those <18 years of age (76.5%). When assessing the completion for all 4 ePROMs, the percentage was 77.6%, also higher among those ≥18 year of age (81.5% versus 72.2% for < 18 years of age).

Conclusion

Using ePROMs, our study demonstrated that GI symptoms can be feasibly collected with good reproducibility and with minimal involvement of research coordinator time. This mechanism of symptom collection may provide an efficient tool for future CF trials.

SUBMITTER: Sathe M

PROVIDER: S-EPMC8403637 | biostudies-literature | 2021 Jul

REPOSITORIES: biostudies-literature

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Publications

Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study.

Sathe Meghana M Moshiree Baha B Vu Phuong T PT Khan Umer U Heltshe Sonya L SL Romasco Melita M Freedman Steven D SD Schwarzenberg Sarah Jane SJ Goss Christopher H CH Freeman A Jay AJ

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society 20210722 4

<h4>Background</h4>The Food and Drug Administration considers patient-reported outcome measures (PROMs) an essential part of clinical research studies for approval of new drugs and new indications for existing drugs. GALAXY evaluated the feasibility of electronic PROMs (ePROMS) to conduct a comprehensive evaluation of gastrointestinal (GI) symptoms in persons with cystic fibrosis (pwCF).<h4>Methods</h4>Three validated GI ePROMs (PAC-SYM, PAGI-SYM and PAC-QOL) were combined with a Stool-Specific ...[more]

PMID: 34305007

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Project description:BACKGROUND:Patients with cystic fibrosis (CF) can struggle with burdensome symptoms and treatment regimens that negatively affect every aspect of their life. As physiological parameters can fail to capture these complications, the assessment of health-related quality of life (HRQOL) has gained prominence. HRQOL can be measured using standardized patient questionnaires called patient-reported outcome measures (PROMs). The Australian Cystic Fibrosis Data Registry (ACFDR) collects clinical data on adult and pediatric patients with CF. The incorporation of PROMs into the ACFDR would enable monitoring of HRQOL trends, benchmarking of HRQOL outcomes, and support of HRQOL research in CF. OBJECTIVE:Prior to incorporation of a PROM in the ACFDR, this systematic review was planned to evaluate whether any suitable PROMs are currently being used for CF. METHODS:This systematic review will be conducted in compliance with the PRISMA-P (Preferred Reporting Items for Systematic Reviews and Meta-Analyses Protocols) guidelines. MEDLINE, EMBASE, Scopus, CINAHL (Cumulative Index of Nursing and Allied Health Literature), PsycINFO, and Cochrane Library databases were searched for articles published between January 2009 and February 2019 on the use of PROMs to measure HRQOL in adult and pediatric patients with CF. Study designs such as observational studies, reviews and validation studies were included. Studies describing randomized controlled trials, dissertations, books, guideline statements, and abstracts were excluded. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) risk of bias checklist was used to assess the methodological quality of included studies. A descriptive synthesis of the results will be undertaken in line with the outcomes of this study. RESULTS:As of July 2019, the search has been conducted and 4530 records were screened. After two phases of screening, 97 studies were included in the final review and subjected to data extraction. Reviewers are currently in the process of critical appraisal. CONCLUSIONS:This review will identify any PROM(s) that may be used to measure HRQOL in the ACFDR. TRIAL REGISTRATION:PROSPERO International Prospective Register of Systematic Reviews CRD42019126931; https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=126931.

Project description:IntroductionCancer patients experience different complications and outcomes during or after medical treatments. Electronic reporting of the outcomes by patients is a solution that facilitates communication with physicians and improve patient health status. The aim of this study was to develop a smartphone-based application for electronic reporting of outcomes by patients with prostate cancer.MethodsThe present research was conducted in 2021 in two phases. In the first phase, initially, users' requirements were identified based on reviewing the related literature, existing applications, and guidelines. Then, a questionnaire was designed and the specialists' opinions about the users' requirements were investigated. The specialties included urologists, hemato-oncologists, uro-oncologists, and radiotherapists (n = 15). In the second phase, the application was designed, and patients with prostate cancer (n = 21) and specialists (n = 10) evaluated it using the post-study system usability questionnaire (PSSUQ). Data were analyzed using descriptive statistics.ResultsThe findings of the first phase of the research showed that out of 108 data elements and functions proposed for the application, 91 items were found essential by the specialists. Data elements were categorized into the patient data, general complications of prostate cancer and side effects of drug therapy, surgery, chemotherapy, cryotherapy, radiation therapy, and hormone therapy. Necessary functions for the application included presenting a patient care summary, communication between the patient and the specialist, free text explanation for complications and sides effects, generating reports, reminder and alert, completing quality of life questionnaire, and calculating the score for the questionnaire. In the second phase of the research, the application was developed and evaluated. The mean value for user satisfaction was (5.95 ± 0.55) out of 7.ConclusionThe developed application can help to accelerate communication with the specialists. It can improve quality of care, reduce unnecessary treatment visits and side effects, and improve timely data collection for a variety of research purposes. However, further research on the cost-effectiveness and usefulness of the collected data is recommended.

Project description:BackgroundMobile health (mHealth) technologies are increasingly used in various medical fields. However, the potential of mHealth to improve patient care in radiotherapy by acquiring electronic patient reported outcome measures (ePROMs) during treatment has been poorly studied so far.ObjectiveThe aim of this study was to develop and implement a novel Web app (PROMetheus) for patients undergoing radiotherapy. Herein, we have reported our experience with a focus on feasibility, patient acceptance, and a correlation of ePROMs with the clinical course of the patients.MethodsIn the period between January and June 2018, 21 patients used PROMetheus to score side effects, symptoms, and quality of life-related parameters during and after their treatment. Items of the Patient Reported Outcome version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) were chosen based on the primary site of disease, 27 items for head and neck tumors, 21 items for thoracic tumors, and 24 items for pelvic tumors.ResultsIn total, 17 out of the 21 patients (81%) regularly submitted ePROMs and more than 2500 data points were acquired. An average of 5.2, 3.5, and 3.3 min was required to complete the head and neck, thorax, and pelvis questionnaires, respectively. ePROMS were able to detect the occurrence of both expected and unexpected side effects during the treatment. In addition, a gradual increase in the severity of side effects over the course the treatment and their remission afterward could be observed with ePROMs. In total, 9 out of the 17 patients (53%), mostly those with head and neck and thoracic cancers, reported PRO-CTCAE grade III or IV fatigue with severe impairments of activities of daily life.ConclusionsThis study shows the successful implementation of an ePROM system and a high patient acceptance. ePROMs have a great potential to improve patient care in radiotherapy by providing a comprehensive documentation of symptoms and side effects, especially of ones that are otherwise underreported.

Dataset Information

Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study.

Background

Methods

Results

Conclusion

Publications

Utilization of electronic patient-reported outcome measures in cystic fibrosis research: Application to the GALAXY study.

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