Dataset Information

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

ABSTRACT: BACKGROUND:Patient-Reported Outcomes provide an opportunity for patients to establish dialogue with pharmaceutical or biotechnology companies about their health conditions without interpretation by a clinician or anyone else. However, Patient-Reported Outcomes that can be widely applicable for use in patient-focused drug development or clinical trial designs are not yet validated for all diseases. The aim of this study report was to provide supportive evidence of the construct and content validity of selected Patient-Reported Outcomes Measurement Information System (PROMIS®) questionnaires compared with other disease-relevant clinical outcome measures, including the 6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test, in late-onset Pompe disease and to provide supportive evidence that the selected PROMIS measures are relevant and important to these patients. METHODS:Thirty patients with late-onset Pompe disease completed five PROMIS questionnaires that were chosen based on patient and provider feedback, along with discussion with key opinion leaders who are experts in Pompe disease. The Amicus Pompe Patient Advisory Board also provided patient experience feedback using the PROMIS questionnaires. Clinical outcome measures (6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test) were collected at the Duke University Pompe Disease Clinical Research Program during a single visit. RESULTS:The Patient Advisory Board rated the questionnaires as representative of an unmet need. Correlation data demonstrated moderate to strong correlations of PROMIS questionnaires with the specified clinical outcome measures (6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test). These data supported the construct and content validity of the PROMIS questionnaires because they confirmed the motor signs and symptoms of functional disability observed in patients with Pompe disease. CONCLUSIONS:The correlations indicate that the clinical outcome measures assess important concepts related to patient-reported experiences. The Patient Advisory Board findings suggest that the selected PROMIS questionnaires are meaningful and address important concepts to patients with Pompe disease. The data were collected from a small number of patients at a single time point; further studies are needed with additional PROMIS questionnaires, which should include measures of motor function and health-related quality of life, in a larger number of patients followed up longitudinally.

SUBMITTER: Harfouche M

PROVIDER: S-EPMC7547055 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature

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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

Harfouche Melodi M Kishnani Priya S PS Krusinska Eva E Gault Jamie J Sitaraman Sheela S Sowinski Amanda A Katz Irina I Austin Stephanie S Goldstein Margi M Mulberg Andrew E AE

Journal of patient-reported outcomes 20201009 1

<h4>Background</h4>Patient-Reported Outcomes provide an opportunity for patients to establish dialogue with pharmaceutical or biotechnology companies about their health conditions without interpretation by a clinician or anyone else. However, Patient-Reported Outcomes that can be widely applicable for use in patient-focused drug development or clinical trial designs are not yet validated for all diseases. The aim of this study report was to provide supportive evidence of the construct and conten ...[more]

PMID: 33034771

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Project description:This is the first study of the measurement equivalence of the Patient Reported Outcomes Measurement Information System® (PROMIS®) Anxiety short forms in a large ethnically diverse sample. The psychometric properties and differential item functioning (DIF) were examined across different racial/ethnic, educational, age, gender and language groups. METHODS:These data are from individuals selected from cancer registries in the United States. For the analyses of race/ethnicity the reference group was non-Hispanic Whites (n = 2,263), the studied groups were non-Hispanic Blacks (n = 1,117), Hispanics (n = 1,043) and Asians/Pacific Islanders (n = 907). Within the Hispanic subsample, there were 335 interviews conducted in Spanish and 703 in English. The 11 anxiety items were from the PROMIS emotional disturbance item bank. DIF hypotheses were generated by content experts who rated whether or not they expected DIF to be present, and the direction of the DIF with respect to several comparison groups. The primary method used for DIF detection was the Wald test for examination of group differences in item response theory (IRT) item parameters accompanied by magnitude measures. Expected item scores were examined as measures of magnitude. The method used for quantification of the difference in the average expected item scores was the non-compensatory DIF (NCDIF) index. DIF impact was examined using expected scale score functions. Additionally, precision and reliabilities were examined using several methods. RESULTS:Although not hypothesized to show DIF for Asians/Pacific Islanders, every item evidenced DIF by at least one method. Two items showed DIF of higher magnitude for Asians/Pacific Islanders vs. Whites: "Many situations made me worry" and "I felt anxious". However, the magnitude of DIF was small and the NCDIF statistics were not above threshold. The impact of DIF was negligible. For education, six items were identified with consistent DIF across methods: fearful, anxious, worried, hard to focus, uneasy and tense. However, the NCDIF was not above threshold and the impact of DIF on the scale was trivial. No items showed high magnitude DIF for gender. Two items showed slightly higher magnitude for age (although not above the cutoff): worried and fearful. The scale level impact was trivial. Only one item showed DIF with the Wald test after the Bonferroni correction for the language comparisons: "I felt fearful". Two additional items were flagged in sensitivity analyses after Bonferroni correction, anxious and many situations made me worry. The latter item also showed DIF of higher magnitude, with an NCDIF value (0.144) above threshold. Individual impact was relatively small. CONCLUSIONS:Although many items from the PROMIS short form anxiety measures were flagged with DIF, item level magnitude was low and scale level DIF impact was minimal; however, three items: anxious, worried and many situations made me worry might be singled out for further study. It is concluded that the PROMIS Anxiety short form evidenced good psychometric properties, was relatively invariant across the groups studied, and performed well among ethnically diverse subgroups of Blacks, Hispanic, White non-Hispanic and Asians/Pacific Islanders. In general more research with the Asians/Pacific Islanders group is needed. Further study of subgroups within these broad categories is recommended.

Project description:AIMS:The goals of these analyses were to examine the psychometric properties and measurement equivalence of a self-reported cognition measure, the Patient Reported Outcome Measurement Information System® (PROMIS®) Applied Cognition - General Concerns short form. These items are also found in the PROMIS Cognitive Function (version 2) item bank. This scale consists of eight items related to subjective cognitive concerns. Differential item functioning (DIF) analyses of gender, education, race, age, and (Spanish) language were performed using an ethnically diverse sample (n = 5,477) of individuals with cancer. This is the first analysis examining DIF in this item set across ethnic and racial groups. METHODS:DIF hypotheses were derived by asking content experts to indicate whether they posited DIF for each item and to specify the direction. The principal DIF analytic model was item response theory (IRT) using the graded response model for polytomous data, with accompanying Wald tests and measures of magnitude. Sensitivity analyses were conducted using ordinal logistic regression (OLR) with a latent conditioning variable. IRT-based reliability, precision and information indices were estimated. RESULTS:DIF was identified consistently only for the item, brain not working as well as usual. After correction for multiple comparisons, this item showed significant DIF for both the primary and sensitivity analyses. Black respondents and Hispanics in comparison to White non-Hispanic respondents evidenced a lower conditional probability of endorsing the item, brain not working as well as usual. The same pattern was observed for the education grouping variable: as compared to those with a graduate degree, conditioning on overall level of subjective cognitive concerns, those with less than high school education also had a lower probability of endorsing this item. DIF was also observed for age for two items after correction for multiple comparisons for both the IRT and OLR-based models: "I have had to work really hard to pay attention or I would make a mistake" and "I have had trouble shifting back and forth between different activities that require thinking". For both items, conditional on cognitive complaints, older respondents had a higher likelihood than younger respondents of endorsing the item in the cognitive complaints direction. The magnitude and impact of DIF was minimal. The scale showed high precision along much of the subjective cognitive concerns continuum; the overall IRT-based reliability estimate for the total sample was 0.88 and the estimates for subgroups ranged from 0.87 to 0.92. CONCLUSION:Little DIF of high magnitude or impact was observed in the PROMIS Applied Cognition - General Concerns short form item set. One item, "It has seemed like my brain was not working as well as usual" might be singled out for further study. However, in general the short form item set was highly reliable, informative, and invariant across differing race/ethnic, educational, age, gender, and language groups.

Project description:BackgroundThe Patient-Reported Outcomes Measurement Information SystemⓇ (PROMISⓇ) is a dynamic system of psychometrically sound patient-reported outcome (PRO) measures. There has been a recent increase in the use of PROMIS measures, yet little has been written about the reporting of these measures in the field of orthopedics. The purpose of this study was to conduct a systematic review to determine the uptake of PROMIS measures across orthopedics and to identify the type of PROMIS measures and domains that are most commonly used in orthopedic research and practice.MethodsWe searched PubMed, Embase, and Scopus using keywords and database-specific subject headings to capture orthopedic studies reporting PROMIS measures through November 2018. Our inclusion criteria were use of PROMIS measures as an outcome or used to describe a population of patients in an orthopedic setting in patients ≥ 18 years of age. We excluded non-quantitative studies, reviews, and case reports.ResultsOur final search yielded 88 studies published from 2013 through 2018, with 57% (50 studies) published in 2018 alone. By body region, 28% (25 studies) reported PROMIS measures in the upper extremity (shoulder, elbow, hand), 36% (32 studies) reported PROMIS measures in the lower extremity (hip, knee, ankle, foot), 19% (17 studies) reported PROMIS measures in the spine, 10% (9 studies) reported PROMIS measures in trauma patients, and 6% (5 studies) reported PROMIS measures in general orthopedic patients. The majority of studies reported between one and three PROMIS domains (82%, 73 studies). The PROMIS Computerized Adaptive Test (CAT) approach was most commonly used (81%, 72 studies). The most frequently reported PROMIS domains were physical function (81%, 71 studies) and pain interference (61%, 54 studies).ConclusionOur review found an increase in the reporting of PROMIS measures over the recent years. Utilization of PROMIS measures in orthopedic populations is clinically appropriate and can facilitate communication of outcomes across different provider types and with reduced respondent burden.RegistrationThe protocol for this systematic review was designed in accordance with the PRISMA guidelines and is registered with the PROSPERO database (CRD42018088260).

Project description:Short form measures from the Patient Reported Outcomes Measurement Information System® (PROMIS®) are used widely. The present study was among the first to examine differential item functioning (DIF) in the PROMIS Depression short form scales in a sample of over 5000 racially/ethnically diverse patients with cancer. DIF analyses were conducted across different racial/ethnic, educational, age, gender and language groups.MethodsDIF hypotheses, generated by content experts, informed the evaluation of the DIF analyses. The graded item response theory (IRT) model was used to evaluate the five-level ordinal items. The primary tests of DIF were Wald tests; sensitivity analyses were conducted using the IRT ordinal logistic regression procedure. Magnitude was evaluated using expected item score functions, and the non-compensatory differential item functioning (NCDIF) and T1 indexes, both based on group differences in the item curves. Aggregate impact was evaluated with expected scale score (test) response functions; individual impact was assessed through examination of differences in DIF adjusted and unadjusted depression estimates.ResultsMany items evidenced DIF; however, only a few had slightly elevated magnitude. No items evidenced salient DIF with respect to NCDIF and the scale-level impact was minimal for all group comparisons. The following short form items might be targeted for further study because they were also hypothesized to evidence DIF. One item showed slightly higher magnitude of DIF for age: nothing to look forward to; conditional on depression, this item was more likely to be endorsed in the depressed direction by individuals in older groups as contrasted with the cohort aged 21 to 49. This item was also hypothesized to show age DIF. Only one item (failure) showed DIF of slightly higher magnitude (just above threshold) for Whites vs. Asians/Pacific Islanders in the direction of higher likelihood of endorsement for Asians/Pacific Islanders. This item was also hypothesized to show DIF for minority groups. The impact of DIF was negligible. Conditional on depression, the items, worthless and hopeless were more likely to be endorsed in the depressed direction by respondents with less than high school education vs. those with a graduate degree; the magnitude of DIF was slightly above the T1 threshold, but not that of NCDIF. These items were also hypothesized to show DIF in the direction of more feelings of worthlessness by groups with lower education. While the magnitude and aggregate impact of DIF was small, in a few instances, individual impact was observed. Information provided was relatively high, particularly in the middle upper (depressed) tail of the distribution. Reliability estimates were high (> 0.90) across all studied groups, regardless of estimation method.ConclusionsThis was the first study to evaluate measurement equivalence of the PROMIS Depression short forms across large samples of ethnically diverse groups. There were few items with DIF, and none of high magnitude, thus supporting the use of PROMIS Depression short form measures across such groups. These results could be informative for those using the short forms in minority populations or clinicians evaluating individuals with the depression short forms.

Project description:PurposeThis study aimed to validate the PROMIS Pediatric item bank v2.0 Peer Relationships and compare reliability of the full item bank to its short form, computerized adaptive test (CAT) and the social functioning (SF) subscale of the Pediatric Quality of Life Inventory (PedsQL™).MethodsChildren aged 8-18 (n = 1327), representative of the Dutch population completed the Peer Relationships item bank. A graded response model (GRM) was fit to the data. Structural validity was assessed by checking item-fit statistics (S-X2, p < 0.001 = misfit). For construct validity, a moderately strong correlation (> 0.50) was expected between Peer Relationships and the PedsQL SF subscale. Cross-cultural DIF between U.S. and NL was assessed using logistic regression, where an item with McFadden's pseudo R2 > 0.02 was considered to have DIF. Percentage of participants reliably measured was assessed using the standard error of measurement (SEM) < 0.32 as a criterion (reliability of 0.90). Relative efficiency ((1-SEM2)/nitems) was calculated to compare how well the instruments performed relative to the amount of items administered.ResultsIn total, 527 (response rate: 39.7%) children completed the PROMIS v2.0 Peer Relationships item bank (nitems = 15) and the PedsQL™ (nitems = 23). Structural validity of the Peer Relationships item bank was sufficient, but one item displayed misfit in the GRM model (S-X2 < 0.001); 5152R1r ("I played alone and kept to myself"). The item 733R1r ("I was a good friend") was the only item that displayed cross-cultural DIF (R2 = 0.0253). The item bank correlated moderately high (r = 0.61) with the PedsQL SF subscale Reliable measurements were obtained at the population mean and > 2SD in the clinically relevant direction. CAT outperformed all other measures in efficiency. Mean T-score of the Dutch general population was 46.9(SD 9.5).ConclusionThe pediatric PROMIS Peer Relationships item bank was successfully validated for use within the Dutch population and reference data are now available.

Dataset Information

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

Publications

Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

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