FEV1 Minimum Important Difference versus Minimal Detectable Difference? In Search of the Unicorn.
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ABSTRACT:
SUBMITTER: Crim C
PROVIDER: S-EPMC8483219 | biostudies-literature | 2021 Jun
REPOSITORIES: biostudies-literature
Publications
FEV<sub>1</sub> Minimum Important Difference versus Minimal Detectable Difference? In Search of the Unicorn.
Crim Courtney C Frith Lucy J LJ Midwinter Dawn D Donohue James F JF
American journal of respiratory and critical care medicine 20210601 12
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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:BackgroundEchocardiography (2DE) is integral for screening and longitudinal evaluation of pulmonary arterial hypertension (PAH) in systemic sclerosis (SSc). In the present study, we sought to establish the reliability, repeatability, and reproducibility of 2DE parameters in SSc patients with and without PAH and to define the minimal detectable difference (MDD), the smallest change detected beyond measurement error.MethodsSSc patients without known PAH and with invasively confirmed PAH on stable therapies underwent 2DE with strain at two time points. Analysis of variance (ANOVA) and coefficients of variation (CV) were calculated to assess for repeatability, reliability, and reproducibility. Intra- and inter-observer agreement were assessed using intraclass correlation. Bland-Altman analysis explored the level of agreement between evaluations. MDD was calculated using the standard error of measurement for each parameter by cohort.ResultsANOVA demonstrated few significant differences between evaluations across groups. Global right ventricular longitudinal systolic strain (GRVLSS, 9.7%) and fractional area change (FAC, 21.3%) had the largest CV, while tricuspid annular plane excursion (TAPSE), S' wave, and right ventricular outflow track velocity time integral (RVOT VTI) were 0.87%, 3.2%, and 6.0%, respectively. Intra- and inter-observer agreement was excellent. MDD for TAPSE, FAC, S' wave, RVOT VTI, GRVLSS, and RVSP were 0.11 cm, 0.03%, 1.27 cm/s, 0.81 cm, 1.14%, and 6.5 mmHg, respectively.ConclusionsWe demonstrate minimal measurement error in clinically important 2DE-based measures in SSc patients with and without PAH. Defining the MDD in this population has important implications for PAH screening, assessment of therapeutic response, and sample size calculations for future clinical trials.
| S-EPMC9206258 | biostudies-literature
Project description: Not available
| S-EPMC6336372 | 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:BackgroundImpulse oscillometry (IOS) allows an effort-independent evaluation of small airway function in asthma. Unfortunately, well-determined minimal clinically important differences (MCIDs) for IOS measures are lacking. Here, we provide MCIDs for frequently used IOS measures, namely frequency dependence of resistance (FDR) and area of reactance (AX), in patients with asthma.MethodsWe performed IOS at baseline and 1 year later in adult patients with mild-to-severe asthma (n=235). In a two-step approach, we first applied a distribution-based method to statistically determine the MCID. Next, we validated the proposed MCID according to patient-reported outcome measures (PROMs): Asthma Quality of Life Questionnaire (AQLQ), Asthma Control Questionnaire-7 (ACQ-7) and Asthma Control Test (ACT). We used multivariable analyses to investigate the proposed MCIDs as predictors for improvements in PROMs compared with the established MCID of forced expiratory volume in 1 s (FEV1).ResultsThe proposed MCID was a decline of ≥0.06 kPa·L-1·s-1 and ≥0.65 kPa·L-1 for FDR and AX, respectively. Patients who had changes beyond the MCIDs for both FDR and AX showed greater improvements in all PROMs than those who had not. The mean improvements in PROMs were beyond the established MCIDs for ACQ-7 and AQLQ, and approximated the MCID for ACT. Multivariable analyses demonstrated the MCIDs for both FDR and AX as independent predictors for the MCIDs of all PROMs. The MCID for FDR was a stronger predictor of all PROMs than the MCID for FEV1.ConclusionsThis study provides MCIDs for IOS-derived measures in adult patients with asthma and emphasises that small airway function is a distinguished end-point beyond the conventional measure of FEV1.
| S-EPMC10160799 | 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: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:BackgroundChanges 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.MethodsPA 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.ResultsCalculation 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.ConclusionsThe 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:The minimum clinically important difference (MCID) is a measure of the minimal clinically relevant change. The MCID represents the smallest difference in score on the measure or domain of interest which patients or clinicians perceive as beneficial or as meaningful decline. The MCID is not an alternative clinical trial outcome; it does not apply to group measures and is used as a means of determining whether an individual patient has reached a threshold of change. MCIDs have been derived for symptomatic treatments and for disease targeted therapies. MCIDs have been derived for nearly all clinical trial instruments used in AD therapeutic research. Application of the MCID to patients on disease-targeted therapies requires awareness of the expected increasing treatment-no treatment difference exhibited by these agents. The MCID complements other strategies for assessing the meaningfulness of interventions including effect size, number needed to treat, responder analyses, time saved, quality of life, and quality-adjusted life years. MCID is not a required measure for regulatory approval of a therapeutic since it is applicable to individual patients and not to group outcomes or mean differences used to determine treatment benefit in clinical trials.HighlightsMCID is a key measure of within-person change in cognition, function, or behavior when it is applied to metrics of Alzheimer's disease progression.In Alzheimer's disease, MCID or minimum within person change (MWPC) can function as useful means of determining if a patient has progressed to thresholds of detectable change.In Alzheimer's disease, MCID/MWPC can be used to determine the number or percent of individuals who have progressed to detectable levels of within-person change, with differences anticipated in active treatment and placebo groups.MCID/MWPC are not measures that are appropriately applied to group outcomes of clinical trials.
| S-EPMC11891559 | 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