IPMDS-Sponsored Scale Translation Program: Process, Format, and Clinimetric Testing Plan for the MDS-UPDRS and UDysRS.
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ABSTRACT:
SUBMITTER: Goetz CG
PROVIDER: S-EPMC5065071 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:This study was undertaken to define the number of missing values permissible to render valid total scores for each Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part. To handle missing values, imputation strategies serve as guidelines to reject an incomplete rating or create a surrogate score. We tested a rigorous, scale-specific, data-based approach to handling missing values for the MDS-UPDRS. From two large MDS-UPDRS datasets, we sequentially deleted item scores, either consistently (same items) or randomly (different items) across all subjects. Lin's Concordance Correlation Coefficient (CCC) compared scores calculated without missing values with prorated scores based on sequentially increasing missing values. The maximal number of missing values retaining a CCC greater than 0.95 determined the threshold for rendering a valid prorated score. A second confirmatory sample was selected from the MDS-UPDRS international translation program. To provide valid part scores applicable across all Hoehn and Yahr (H&Y) stages when the same items are consistently missing, one missing item from Part I, one from Part II, three from Part III, but none from Part IV can be allowed. To provide valid part scores applicable across all H&Y stages when random item entries are missing, one missing item from Part I, two from Part II, seven from Part III, but none from Part IV can be allowed. All cutoff values were confirmed in the validation sample. These analyses are useful for constructing valid surrogate part scores for MDS-UPDRS when missing items fall within the identified threshold and give scientific justification for rejecting partially completed ratings that fall below the threshold.
| S-EPMC5072275 | biostudies-literature
Project description:BackgroundThe Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) comprises 50 items, consisting of historical questions and motor ratings, typically taking around 30 minutes to complete. We sought to identify an abbreviated version that could facilitate use in clinical practice or used remotely via telemedicine.MethodsTo create an 8-item version we conducted an "exhaustive search" of all possible subsets. We measured explained variance in comparison to the 50-item version using linear regression, with the "optimal" subset maximising this while also meeting remote assessment practicality constraints. The subset was identified using a dataset collected by the Parkinson's Progression Markers Initiative and validated using an MDS Non-Motor Symptoms Scale validation study dataset.ResultsThe optimal remote version comprised items from all parts of the MDS-UPDRS and was found to act as an unbiased estimator of the total 50-item score. This version had an explained variance score of 0.844 and was highly correlated with the total MDS-UPDRS score (Pearson's r = 0.919, p-value <0.0001). Another subset that maximised explained variance score without adhering to remote assessment practicality constraints provided similar results.ConclusionThis result demonstrates that the total scores of an abbreviated form identified by computational statistics had high agreement with the MDS-UPDRS total score. Whilst it cannot capture the richness of information of the full MDS-UPDRS, it can be used to create a total score where practicality limits the application of the full MDS-UPDRS, such as remote monitoring. Further validation will be required, including in specific subgroups and advanced disease stages, and full validation of clinimetric properties.
| S-EPMC9197633 | biostudies-literature
Project description:BACKGROUND:Care for infants born preterm or with major birth defects is costly. Specific estimates of financial burden for different payers are lacking, in part because use of administrative data to identify preterm infants and costs is challenging. METHODS:We used private health insurance claims data and billing codes to identify live births during 2013 and calculated first-year expenditures for employer-sponsored health plans for infants born preterm, both overall and stratified by major birth defects. RESULTS:We conservatively estimated that 7.7% of insured infants born preterm accounted for 37% of $2.0 billion spent by participating plans on the care of infants born during 2013. With a mean difference in plan expenditures of ?$47?100 per infant, preterm births cost the included plans an extra $600 million during the first year of life. Extrapolating to the national level, we projected aggregate employer-sponsored plan expenditures of $6 billion for infants born preterm during 2013. Infants with major birth defects accounted for 5.8% of preterm births but 24.5% of expenditures during infancy. By using an alternative algorithm to identify preterm infants, it was revealed that incremental expenditures were higher: $78?000 per preterm infant and $14 billion nationally. CONCLUSION:Preterm births (especially in conjunction with major birth defects) represent a substantial burden on payers, and efforts to mitigate this burden are needed. In addition, researchers need to conduct studies using linked vital records, birth defects surveillance, and administrative data to accurately and longitudinally assess per-infant costs attributable to preterm birth and the interaction of preterm birth with major birth defects.
| S-EPMC6029853 | biostudies-literature
Project description:BackgroundThe Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) is a newly developed tool to assess Parkinson's disease (PD). Changes in scores on the scale over the course of PD, including increasing disease duration and Hoehn and Yahr (HY) stages, have not been described. The objectives of this study were to analyze MDS-UPDRS scores on Parts I through IV and their differences based on HY stage and disease duration in a large cohort of patients with PD.MethodsFor this cross-sectional study, demographic data and MDS-UPDRS scores were collected, including HY stage. Subscores on MDS-UPDRS Parts I through IV were analyzed using 1-way analyses of variance for each HY stage and in 5-year increments of disease duration. Part III (motor assessment) scores were analyzed separately for on and off states.ResultsThe mean age of the 3206 patients was 65.8 ± 10.6 years, 53.3% were men, the mean disease duration was 11.5 ± 4.6 years, and the median HY stage was 2 (range, 0-5); 2156 patients were examined in an on state and 987 were examined in an off state. Scores for all MDS-UPDRS parts increased significantly through HY stages 1 through 5, with an average increase of 3.8, 7.7, 14.6, and 2.0 points consecutively for parts I through IV, respectively. For the 5-year increments of disease duration, MDS-UPDRS subscores increased by an average of 1.6, 3.3, 4.2, and 1.4 points consecutively for parts I through IV, respectively. This increase was significant only during the first 15 years of disease for all 4 parts, including part III scores evaluated in both on and off states.ConclusionsMDS-UPDRS scores for all 4 parts increase significantly with every HY stage and also with 5-year increments of disease duration in the first 15 years of the disease.
| S-EPMC6174385 | biostudies-literature
Project description:BackgroundAn important challenge in Parkinson's disease research is how to measure disease progression, ideally at the individual patient level. The MDS-UPDRS, a clinical assessment of motor and nonmotor impairments, is widely used in longitudinal studies. However, its ability to assess within-subject changes is not well known. The objective of this study was to estimate the reliability of the MDS-UPDRS when used to measure within-subject changes in disease progression under real-world conditions.MethodsData were obtained from the Parkinson's Progression Markers Initiative cohort and included repeated MDS-UPDRS measurements from 423 de novo Parkinson's disease patients (median follow-up: 54 months). Subtotals were calculated for parts I, II, and III (in on and off states). In addition, factor scores were extracted from each part. A linear Gaussian state space model was used to differentiate variance introduced by long-lasting changes from variance introduced by measurement error and short-term fluctuations. Based on this, we determined the within-subject reliability of 1-year change scores.ResultsOverall, the within-subject reliability ranged from 0.13 to 0.62. Of the subscales, parts II and III (OFF) demonstrated the highest within-subject reliability (both 0.50). Of the factor scores, the scores related to gait/posture (0.62), mobility (0.45), and rest tremor (0.43) showed the most consistent behavior.ConclusionsOur results highlight that MDS-UPDRS change scores contain a substantial amount of error variance, underscoring the need for more reliable instruments to forward our understanding of the heterogeneity in PD progression. Focusing on gait and rest tremor may be a promising approach for an early Parkinson's disease population. © 2019 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.
| S-EPMC6851993 | biostudies-literature
Project description:X-linked hypophosphatemia (XLH), a dominant disorder caused by pathogenic variants in the PHEX gene, affects both sexes of all ages and results in elevated serum fibroblast growth factor 23 (FGF23) and below-normal serum phosphate. In XLH, rickets, osteomalacia, short stature, and lower limb deformity may be present with muscle pain and/or weakness/fatigue, bone pain, joint pain/stiffness, hearing difficulty, enthesopathy, osteoarthritis, and dental abscesses. Invitae and Ultragenyx collaborated to provide a no-charge sponsored testing program using a 13-gene next-generation sequencing panel to confirm clinical XLH or aid diagnosis of suspected XLH/other genetic hypophosphatemia. Individuals aged ≥6 months with clinical XLH or suspected genetic hypophosphatemia were eligible. Of 831 unrelated individuals tested between February 2019 and June 2020 in this cross-sectional study, 519 (62.5%) individuals had a pathogenic or likely pathogenic variant in PHEX (PHEX-positive). Among the 312 PHEX-negative individuals, 38 received molecular diagnoses in other genes, including ALPL, CYP27B1, ENPP1, and FGF23; the remaining 274 did not have a molecular diagnosis. Among 319 patients with a provider-reported clinical diagnosis of XLH, 88.7% (n = 283) had a reportable PHEX variant; 81.5% (n = 260) were PHEX-positive. The most common variant among PHEX-positive individuals was an allele with both the gain of exons 13-15 and c.*231A>G (3'UTR variant) (n = 66/519). Importantly, over 80% of copy number variants would have been missed by traditional microarray analysis. A positive molecular diagnosis in 41 probands (4.9%; 29 PHEX positive, 12 non-PHEX positive) resulted in at least one family member receiving family testing. Additional clinical or family member information resulted in variant(s) of uncertain significance (VUS) reclassification to pathogenic/likely pathogenic (P/LP) in 48 individuals, highlighting the importance of segregation and clinical data. In one of the largest XLH genetic studies to date, 65 novel PHEX variants were identified and a high XLH diagnostic yield demonstrated broad insight into the genetic basis of XLH. © 2021 The Authors. Journal of Bone and Mineral Research published by Wiley Periodicals LLC on behalf of American Society for Bone and Mineral Research (ASBMR).
| S-EPMC9298723 | biostudies-literature
Project description:Movement Disorder Society-sponsored Unified Parkinson's Disease Rating Scale (MDS-UPDRS) has separate items for measuring sleep problems (item 1.7) and daytime sleepiness (1.8). The aim of our study was to evaluate the screening sensitivity and specificity of these items to the PD Sleep Scale 2nd version (PDSS-2) and Epworth Sleepiness Scale (ESS). In this nationwide, cross-sectional study 460 PD patients were enrolled. Spearman's rank correlation coefficients were calculated between the individual items, domains, and the total score of PDSS-2 and item 1.7 of MDS-UPDRS. Similarly, the items and the total score of ESS were contrasted to item 1.8 of MDS-UPDRS. After developing generalized ordinal logistic regression models, the transformed and observed scores were compared by Lin's Concordance Correlation Coefficient. Only item 3 difficulties staying asleep and the "disturbed sleep" domain of PDSS-2 showed high correlation with "sleep problems" item 1.7 of the MDS-UPDRS. Total score of PDSS-2 had moderate correlation with this MDS-UPRDS item. The total score of ESS showed the strongest, but still moderate, correlation with "daytime sleepiness" item 1.8 of MDS-UPDRS. As intended, the MDS-UPDRS serves as an effective screening tool for both sleep problems and daytime sleepiness and identifies subjects whose disabilities need further investigation.
| S-EPMC4251816 | biostudies-literature
Project description:Rationale & objectiveDespite growing interest in individualizing care, routine dialysis processes, including the interdisciplinary plan of care, often fail to account for patient-identified priorities. To better align dialysis care with patient priorities and improve care planning experiences, we implemented a person-centered care plan program at a single clinic. We also sought to gain insight into key implementation considerations and areas for program improvement.Study design6-month quality improvement project with research substudy.Setting & participants49 hemodialysis patients and 14 care team members at a North Carolina dialysis clinic.Quality improvement activitiesImplementation of My Dialysis Plan, a person-centered care plan program.OutcomesParticipant perspectives and care plan meeting characteristics (quality improvement); pre- to postprogram change in patient-reported autonomy support, patient-centeredness of care, and dialysis care individualization (research).Analytical approachWe used the Consolidated Framework for Implementation Research to guide implementation and evaluation. We conducted pre-, intra-, and post-project interviews with clinic stakeholders (patients, clinic personnel, and medical providers) to identify implementation barriers, facilitators, and perceptions. We compared pre- and post-project care plan meeting content and patient-reported outcome survey scores.ResultsWe conducted 54 care plans with 49 patients. Overall, care teams successfully used My Dialysis Plan to elicit and link patient priorities to actionable aspects of dialysis care. Participants identified interdisciplinary team commitment, accountability, and the structured yet flexible meeting approach as key implementation elements. Throughout the project, stakeholder input guided program modifications (eg, implementation practices and resources) to better meet clinic needs, but follow-up on care plan-identified action items remained challenging. Among the 28 substudy participants, there was no difference in pre- to post-project patient-reported outcome survey scores.LimitationsSingle clinic implementation.ConclusionsMy Dialysis Plan has the potential to enhance dialysis care individualization and care plan experiences. Evaluation of program impact on patient-reported and clinical outcomes is needed.
| S-EPMC8039412 | biostudies-literature
Project description:BACKGROUND: Neck pain is a common problem that can greatly affect a person's activities of daily living. Functional status questionnaires are important in assessing this effect, and are used to follow up neck pain management programs. The Neck Disability Index (NDI) is the first-created scale for neck pain-related disability and is widely translated and in common used in many countries. Our aim is investigate to clinometric properties of a Turkish version of modified NDI and to give a choice in daily practise of versions to be used. METHODS: The modified NDI was applied to 30 patients for reliability. 185 patients participated in the validity study. All patients were recruited from the outpatient clinic of our department. The scale was translated by the forward and backward translation procedure according to the COSMIN criteria. The test was repeated at 48 hours interval for reliability study. SPSS-10.0, software was used for statistical analyses. The Intraclass correlation coefficient was used for the test- retest reliability of the modified NDI. Cronbach ? was used for internal consistency. Factor analysis was used for construct validity. The validity of the modified NDI with respect to the SF-36, HAD, VAS pain, VAS disability was assessed using Spearman correlations. RESULTS: The Intraclass correlation coefficient between first and second (within 48 hours) evaluation of test (rs) was 0.92. Questions 1,4,6,8,10 were shown to have excellent reliability. (rs > 0.9). Question 10 was the most frequently challenged question because "recreational and social activities" do not have not the same meanings in Turkey than in western countries. This required that detailed explanations be provided by the investigators. Cronbach's alpha for the total index was 0.88. A single factor accounting for 80.2% of the variance was obtained. Validity studies demonstrated good and moderate correlations (rs) among NDI, HAD, VAS, physical function subtitle of SF 36 (0.62, 0.76, 0.68). CONCLUSIONS: The modified NDI-Turkish version is a reliable and valid test and is suitable for daily practise.
| S-EPMC3305639 | biostudies-literature
Project description:BackgroundThe Movement Disorder Society Unified Parkinson Disease Rating Scale (MDS-UDPRS) is a commonly used tool to measure Parkinson disease (PD) progression. Longitudinal changes in MDS-UPDRS scores in de novo PD have not been established.ObjectiveDetermine progression rates of MDS-UPDRS scores in de novo PD.Methods362 participants from the Parkinson's Progression Markers Initiative, a multicenter longitudinal cohort study of de novo PD, were included. Longitudinal progression of MDS-UPDRS total and subscale scores were modeled using mixed model regression.ResultsMDS-UPDRS scores increased in a linear fashion over five years in de novo PD. MDS-UPDRS total score increased an estimated 4.0 points/year, Part I 0.25 points/year, Part II 1.0 points/year, and Part III 2.4 points/year.ConclusionsThe expected average progression of MDS-UPDRS scores in de novo PD from this study can assist in clinical monitoring and provide comparative data for detection of disease modification in treatment trials.
| S-EPMC5898442 | biostudies-literature