FEV1 Minimum Important Difference versus Minimal Detectable Difference? In Search of the Unicorn.
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SUBMITTER: Crim C
PROVIDER: S-EPMC8483219 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:AimsMinimum important difference (MID) estimates the minimum degree of change in an instrument's score that correlates with subjective sense of improvement. The aim of this study was to estimate the MID for the Urogenital Distress Inventory (UDI), Incontinence Impact Questionnaire (IIQ) and Overactive Bladder Questionnaire (OAB-q) using anchor and distribution-based approaches in patients with urge-predominant incontinence and whether MID changes over time.MethodsThis was a sub-analysis of a multi-center trial of 307 women with pure urge (11) or urge-predominant (296) incontinence who completed condition-specific instruments 10 weeks and 8 months after randomization to anticholinergic medication with or without behavioral therapy. We applied anchor-based methods only when the Kendall's rank correlations between the anchors (Global Perception of Improvement (GPI), Patient Satisfaction Questionnaire (PSQ), and incontinence episodes) and the incontinence instruments (UDI, UDI irritative subscale, IIQ, and OAB-q subscales) were ≥ 0.3. We applied three distribution-based methods to all instruments: effect sizes of ± 0.2 SD (small) and ± 0.5 SD (medium), and standard error of measurement of ± 1. Analyses were performed at both time points.ResultsAnchor-based MIDs for the UDI ranged from -35 to -45 and -15 to -25 for the irritative subscale distribution-based methods MIDs for UDI and IIQ ranged between -10 to -25 and -19 to -49, respectively, reflective of a reduction in bother and symptom severity (SS). OAB-q subscale MIDs ranged from +5 to +12, denoting improved quality of life (HRQL) and -13 to -25, consistent with a reduction in SS.ConclusionsThe MID in women with urge-predominant UI for the UDI and UDI irritative are -35 and -15. Our findings are consistent with previously reported MIDs for the OAB-q subscales. Distribution-based method MIDs are lower values than anchor-based values. The MID did not typically change over the time.
| S-EPMC5373473 | biostudies-literature
Project description:Our aim was to determine the minimal important difference (MID) for 6-min walk distance (6MWD) and maximal cycle exercise capacity (MCEC) in patients with severe chronic obstructive pulmonary disease (COPD). 1,218 patients enrolled in the National Emphysema Treatment Trial completed exercise tests before and after 4-6 weeks of pre-trial rehabilitation, and 6 months after randomisation to surgery or medical care. The St George's Respiratory Questionnaire (domain and total scores) and University of California San Diego Shortness of Breath Questionnaire (total score) served as anchors for anchor-based MID estimates. In order to calculate distribution-based estimates, we used the standard error of measurement, Cohen's effect size and the empirical rule effect size. Anchor-based estimates for the 6MWD were 18.9 m (95% CI 18.1-20.1 m), 24.2 m (95% CI 23.4-25.4 m), 24.6 m (95% CI 23.4-25.7 m) and 26.4 m (95% CI 25.4-27.4 m), which were similar to distribution-based MID estimates of 25.7, 26.8 and 30.6 m. For MCEC, anchor-based estimates for the MID were 2.2 W (95% CI 2.0-2.4 W), 3.2 W (95% CI 3.0-3.4 W), 3.2 W (95% CI 3.0-3.4 W) and 3.3 W (95% CI 3.0-3.5 W), while distribution-based estimates were 5.3 and 5.5 W. We suggest a MID of 26 ± 2 m for 6MWD and 4 ± 1 W for MCEC for patients with severe COPD.
| S-EPMC5516638 | biostudies-literature
Project description:Estimates of the minimal clinically important difference (MCID) for physical activity (PA) in chronic obstructive pulmonary disease (COPD) are needed. The objective is to provide an anchor-based estimate of the MCID for daily step count. PA was promoted in persons with COPD using a pedometer (Omron HJ-720ITC) alone or a pedometer plus interactive website for 3 months. Participants wore the pedometer daily and received phone calls monthly to ascertain medical events. Medical events were counted when a participant self-reported that he/she had (1) worsening of breathing, (2) change to breathing medications, (3) medical care from an emergency room for any reason, or (4) hospitalization for any reason. Generalized linear regression models assessed daily step count as change at the end of study and averaged over the 15, 31, or 61 days centered on the event, in those with an event compared to those without one. All categories of events carried equal weight in the analyses. We studied 93 persons, 46 of whom had an event. Participants who experienced an event had a decrease of 1086 (95% confidence interval (CI): -2124 to -48) or 887 (95% CI: -2030 to 257) steps/day in the pedometer plus website or pedometer alone groups, respectively, compared to those without one. In the days centered on an event, participants who had an event experienced a decrease of 882-983 steps/day (pedometer plus website) or a decrease of 351-495 steps/day (pedometer alone), compared to those without one. The MCID for PA in COPD ranges from 350 steps/day to 1100 steps/day.
| S-EPMC6302974 | biostudies-literature
Project description:BACKGROUND:Changes in physical activity (PA) are difficult to interpret because no framework of minimal important difference (MID) exists. We aimed to determine the minimal important difference (MID) in physical activity (PA) in patients with Chronic Obstructive Pulmonary Disease and to clinically validate this MID by evaluating its impact on time to first COPD-related hospitalization. METHODS:PA was objectively measured for one week in 74 patients before and after three months of rehabilitation (rehabilitation sample). In addition the intraclass correlation coefficient was measured in 30 patients (test-retest sample), by measuring PA for two consecutive weeks. Daily number of steps was chosen as outcome measurement. Different distribution and anchor based methods were chosen to calculate the MID. Time to first hospitalization due to an exacerbation was compared between patients exceeding the MID and those who did not. RESULTS:Calculation of the MID resulted in 599 (Standard Error of Measurement), 1029 (empirical rule effect size), 1072 (Cohen's effect size) and 1131 (0.5SD) steps.day-1. An anchor based estimation could not be obtained because of the lack of a sufficiently related anchor. The time to the first hospital admission was significantly different between patients exceeding the MID and patients who did not, using the Standard Error of Measurement as cutoff. CONCLUSIONS:The MID after pulmonary rehabilitation lies between 600 and 1100 steps.day-1. The clinical importance of this change is supported by a reduced risk for hospital admission in those patients with more than 600 steps improvement.
| S-EPMC4849755 | biostudies-literature
Project description:Pedometer step count improves with pulmonary rehabilitation and deteriorates with time. The MCID for improvement and deterioration is 427 and -456 steps, respectively, but there is uncertainty about the reliability of these estimates. https://bit.ly/3ci97Jh.
| S-EPMC7983253 | biostudies-literature
Project description:BackgroundA minimal clinically important difference has not been established for the Abnormal Involuntary Movement Scale in patients with tardive dyskinesia. Valbenazine is a vesicular monoamine transporter 2 inhibitor approved for the treatment of tardive dyskinesia in adults. Efficacy in randomized, double-blind, placebo-controlled trials was defined as the change from baseline in Abnormal Involuntary Movement Scale total score (sum of items 1-7).ObjectivesTo estimate an minimal clinically important difference for the Abnormal Involuntary Movement Scale using valbenazine trial data and an anchor-based method.MethodsData were pooled from three 6-week double-blind, placebo-controlled trials: KINECT (NCT01688037), KINECT 2 (NCT01733121), and KINECT 3 (NCT02274558). Valbenazine doses were pooled for analyses as follows: "low dose," which includes 40 or 50 mg/day; and "high dose," which includes 75 or 80 mg/day. Mean changes from baseline in Abnormal Involuntary Movement Scale total score were analyzed in all participants (valbenazine- and placebo-treated) with a Clinical Global Impression of Change-Tardive Dyskinesia or Patient Global Impression of Change score of 1 (very much improved) to 3 (minimally improved).ResultsThe least squares mean improvement from baseline to week 6 in Abnormal Involuntary Movement Scale total score was significantly greater with valbenazine (low dose: -2.4; high dose: -3.2; both, P < 0.001) versus placebo (-0.7). An minimal clinically important difference of 2 points was estimated based on least squares mean changes in Abnormal Involuntary Movement Scale total score in participants with a Clinical Global Impression of Change-Tardive Dyskinesia score ≤3 at week 6 (mean change: -2.2; median change: -2) or Patient Global Impression of Change score ≤3 at week 6 (mean change: -2.0; median change: -2).ConclusionsResults from an anchor-based method indicate that a 2-point decrease in Abnormal Involuntary Movement Scale total score may be considered clinically important. © 2019 The Authors. Movement Disorders published by Wiley Periodicals, Inc. on behalf of International Parkinson and Movement Disorder Society.
| S-EPMC6772010 | biostudies-literature
Project description:In the European registration procedure for pesticides, microcosm and mesocosm studies are the highest aquatic experimental tier to assess their environmental effects. Evaluations of microcosm/mesocosm studies rely heavily on no observed effect concentrations (NOECs) calculated for different population-level endpoints. Ideally, a power analysis should be reported for the concentration-response relationships underlying these NOECs, as well as for measurement endpoints for which significant effects cannot be demonstrated. An indication of this statistical power can be provided a posteriori by calculated minimum detectable differences (MDDs). The MDD defines the difference between the means of a treatment and the control that must exist to detect a statistically significant effect. The aim of this paper is to expand on the Aquatic Guidance Document recently published by the European Food Safety Authority (EFSA) and to propose a procedure to report and evaluate NOECs and related MDDs in a harmonised way. In addition, decision schemes are provided on how MDDs can be used to assess the reliability of microcosm/mesocosm studies and for the derivation of effect classes used to derive regulatory acceptable concentrations. Furthermore, examples are presented to show how MDDs can be reduced by optimising experimental design and sampling techniques.
| S-EPMC4544645 | biostudies-literature
Project description:Background: The minimum clinical important differences (MCIDs) of resilience instruments in patients with cancer have not been comprehensively described. This study was designed to evaluate MCIDs of 10-item and 25-item resilience scales specific to cancer (RS-SC-10 and RS-SC-25).Methods: From June 2015 to December 2018, RS-SCs were longitudinally measured in 765 patients with different cancer diagnoses at baseline (T0) and 3?months later (T1). The EORTC QLQ-C30, Connor-Davidson Resilience Scale, Hospital Anxiety and Depression Scale, and Allostatic Load Index were measured concurrently as anchors. Anchor-based methods (linear regression, within-group), distribution-based methods(within-group), and receiver operating characteristic curves (ROCs, within-subject) were performed to evaluate the MCIDs.Results: 623 of 765 (84.1%) patients had paired RS-SCs scores. Moderate correlations were identified between the change in RS-SCs and change in anchors (r?=?0.38-0.44, all p?<?0.001). Linear regression estimated +?8.9 and?-?6.7 as the MCIDs of RS-SC-25, and?+?3.4 and?-?2.5 for RS-SC-10. Distribution-based methods estimated +?9.9 and?-?9.9 as the MCIDs of RS-SC-25, and?+?4.0 and?-?4.0 for RS-SC-10. ROC estimated +?5.5 and?-?4.5 as the MCIDs of RS-SC-25, and?+?2.0 and?-?1.5 for RS-SC-10.Conclusions: The most reliable MCID is around 5 points for RS-SC-25 and 2 points for RS-SC-10. RS-SCs are more responsive to the worsening status of resilience in patients with cancer and these estimates could be useful in future resilience-based intervention trials.
| S-EPMC7724855 | biostudies-literature
Project description:PurposeThe Dementia-Related Quality of Life (DEMQOL) measure and the DEMQOL-Utility Score (DEMQOL-U) are validated tools for measuring quality of life (QOL) in people with dementia. What score changes translate to a clinically significant impact on patients' lives was unknown. This study establishes the minimal important differences (MID) for these two instruments.MethodsAnchor-based and distribution-based methods were used to estimate the MID scores from patients enrolled in a randomised controlled trial. For the anchor-based method, the global QOL (Q29) item from the DEMQOL was chosen as the anchor for DEMQOL and both Q29 and EQ-5D for DEMQOL-U. A one category difference in Q29, and a 0.07 point difference in EQ-5D score, were used to classify improvement and deterioration, and the MID scores were calculated for each category. These results were compared with scores obtained by the distribution-based methods.ResultsA total of 490 people with dementia had baseline DEMQOL data, of these 386 had 8-month data, and 344 had 12-month DEMQOL data. The absolute change in DEMQOL for a combined 1-point increase or decrease in the Q29 anchor was 5.2 at 8 months and 6.0 at 12 months. For the DEMQOL-U, the average absolute change at 8 and 12 months was 0.032 and 0.046 for the Q29 anchor and 0.020 and 0.024 for EQ-5D anchor.ConclusionWe present MID scores for the DEMQOL and DEMQOL-U instruments obtained from a large cohort of patients with dementia. An anchored-based estimate of the MID for the DEMQOL is around 5 to 6 points; and 0.02 to 0.05 points for the DEMQOL-U. The results of this study can guide clinicians and researchers in the interpretation of these instruments comparisons between groups or within groups of people with dementia.Trial registration number and date of registrationISRCTN17993825 on 11th October 2016.
| S-EPMC8481142 | biostudies-literature
Project description:Background and Aims. The aim of the present study was to determine the estimates of minimal clinically important difference for Parkinson's Disease Sleep Scale 2nd version (PDSS-2) total score and dimensions. Methods. The subject population consisted of 413 PD patients. At baseline, MDS-UPDRS, Hoehn-Yahr Scale, Mattis Dementia Rating Scale, and PDSS-2 were assessed. Nine months later the PDSS-2 was reevaluated with the Patient-Reported Global Impression Improvement Scale. Both anchor-based techniques (within patients' score change method and sensitivity- and specificity-based method by receiver operating characteristic analysis) and distribution-based approaches (effect size calculations) were utilized to determine the magnitude of minimal clinically important difference. Results. According to our results, any improvements larger than -3.44 points or worsening larger than 2.07 points can represent clinically important changes for the patients. These thresholds have the effect size of 0.21 and -0.21, respectively. Conclusions. Minimal clinically important differences are the smallest change of scores that are subjectively meaningful to patients. Studies using the PDSS-2 as outcome measure should utilize the threshold of -3.44 points for detecting improvement or the threshold of 2.07 points for observing worsening.
| S-EPMC4619979 | biostudies-other