Minimal Clinically Important Change Research Articles

Post Hoc Analysis of Data from Two Clinical Trials Evaluating the Minimal Clinically Important Change in International Restless Legs Syndrome Sum Score in Patients with Restless Legs Syndrome (Willis-Ekbom Disease).

Determine the minimal clinically important change (MCIC), a measure determining the minimum change in scale score perceived as clinically beneficial, for the international restless legs syndrome (IRLS) and restless legs syndrome 6-item questionnaire (RLS-6) in patients with moderate to severe restless legs syndrome (RLS/Willis-Ekbom disease) treated with the rotigotine transdermal system. This post hoc analysis analyzed data from two 6-mo randomized, double-blind, placebo-controlled studies (SP790 [NCT00136045]; SP792 [NCT00135993]) individually and as a pooled analysis in rotigotine-treated patients, with baseline and end of maintenance IRLS and Clinical Global Impressions of change (CGI Item 2) scores available for analysis. An anchor-based approach and receiver operating characteristic (ROC) curves were used to determine the MCIC for the IRLS and RLS-6. We specifically compared "much improved vs minimally improved," "much improved/very much improved vs minimally improved or worse," and "minimally improved or better vs no change or worse" on the CGI-2 using the full analysis set (data as observed). The MCIC IRLS cut-off scores for SP790 and SP792 were similar. Using the pooled SP790+SP792 analysis, the MCIC total IRLS cut-off score (sensitivity, specificity) for "much improved vs minimally improved" was -9 (0.69, 0.66), for "much improved/very much improved vs minimally improved or worse" was -11 (0.81, 0.84), and for "minimally improved or better vs no change or worse" was -9 (0.79, 0.88). MCIC ROC cut-offs were also calculated for each RLS-6 item. In patients with RLS, the MCIC values derived in the current analysis provide a basis for defining meaningful clinical improvement based on changes in the IRLS and RLS-6 following treatment with rotigotine.

Responsiveness of the Japanese Osteoporosis Quality of Life questionnaire in women with postmenopausal osteoporosis.

BackgroundThe Japanese Osteoporosis Quality of Life (JOQOL) questionnaire measures quality of life in Japanese patients with osteoporosis. However, several important aspects of the psychometric properties of individual domains, including responsiveness, have not been addressed to enable valid clinical application. This analysis examined the internal and external responsiveness of the JOQOL questionnaire.MethodsThis was a post hoc analysis of a 24-week prospective postmarketing study of raloxifene (60 mg/day) administered to postmenopausal Japanese women with osteoporosis (JapicCTI-070465). Internal responsiveness was assessed using Standardized Response Mean (SRM) statistics and changes in JOQOL domain scores. Patients were also stratified into those who did or did not achieve a minimal clinically important change (MCIC) in pain, assessed by a visual analogue scale for pain (VAS pain): comparisons were made between treated patients who achieved VAS pain reduction ≥ 20 mm versus VAS pain reduction < 20 mm. External responsiveness was assessed using Pearson’s correlation coefficient (r) for changes in JOQOL domain scores with Short Form-8 Health Survey and European Quality of Life Instrument scores.ResultsOf 506 patients analyzed, 421 had a baseline value for VAS pain; of these, 152 patients (36.1%) had a MCIC, whereas 264 patients (62.7%) did not. The JOQOL domains pain, overall health, and falls/psychological factors had small to moderate SRM values (0.3-0.5) in all patients, but consistently showed significantly larger changes in patients whose pain score changes exceeded the MCIC. Together, these findings suggest some degree of internal responsiveness for these domains. However, activities of daily living domain had a SRM value as low as 0.2, and recreation/social activities and posture/physique domains had SRM values close to 0. Moderate correlation (defined as r ≥ 0.4 to < 0.6) was noted between the domains pain, activities of daily living, and overall health and some Short Form-8 Health Survey subscales and the European Quality of Life total score, suggesting external responsiveness of these domains.ConclusionsThe inconsistent responsiveness among individual JOQOL domains in treated patients suggests the need for improving several JOQOL domains, especially the activities of daily living, recreation/social activities and posture/physique domains, before application to clinical research.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-014-0178-0) contains supplementary material, which is available to authorized users.

Validation of a new questionnaire to assess the impact of Whiplash Associated Disorders: The Whiplash Activity and participation List (WAL)

Valid questionnaires for measuring functional limitations in patients with Whiplash Associated Disorders (WAD) are lacking, since existing measures are not suitable for addressing the specific limitations of these patients and because of cross contamination between theoretical constructs.The objective of this study was to evaluate dimensionality, test-retest reliability, measurement error, construct validity, and responsiveness of a new condition-specific questionnaire for WAD as well as to estimate the minimally important change score.Patients with WAD grade I or II were recruited from physical therapy practices and rehabilitation centers. Dimensionality was examined by internal consistency analysis (Cronbach's alpha) and factor analysis. Test-retest reliability was estimated by intraclass correlations and measurement error was calculated by the minimal detectable change (MDC) scores. Construct validity was investigated by testing predefined hypotheses on correlations of the WAL scores with generic health measures and by using the known group method. Responsiveness was expressed as the minimal clinically important change (MCIC) score.73 patients (53 women) were included. Cronbach's alpha was high (0.95) and unidimensionality was plausible because factor analysis showed 40.3% variance explained by one dominant factor, which was more than 4.5 times larger than the second largest factor. Test-retest reliability was excellent (0.92, 95% CI 0.87–0.95). Construct validity was supported by 14 out of 15 confirmed hypotheses and the WAL showed statistically significant differences between known groups. MDC was 16 points while the MCIC was 18 points.In conclusion, the present study suggests that the WAL has adequate measurement properties, but additional research is needed.

Responsiveness of outcome measures in patients with superior labral anterior and posterior lesions.

Evaluation of patients with superior labral anterior and posterior (SLAP) lesions requires outcome measures validated for the purpose. The present study aimed to evaluate the responsiveness of the Rowe score, Oxford Instability Shoulder Score (OISS), Western Ontario Shoulder Instability Index (WOSI) and EuroQol (EQ-5D and EQ-VAS) in patients treated for SLAP lesions. Eighty-nine patients were included: 34 had arthroscopic labral repair, 28 had mini-open biceps tenodesis and 27 had physical treatment. The outcome measures were administrated before treatment and after 6 months. Responsiveness was evaluated using standardized response mean (SRM), area under receiver-operating characteristic curve (ROCAUC), reliable chance proportion (RCP) statistics and hypothesis testing. Minimal clinically important change (MCIC) estimates were reported. All outcome measures had high values of SRM (0.86-1.92). RCPs for the improved group were 68% to 79% for OISS, WOSI and Rowe score, and 15% to 49% for EuroQol. ROCAUC was >0.70 for all outcomes. MCIC estimates were 8 and 10 for OISS; 451 and 569 for WOSI; 17 and 18 for Rowe score; 0.39 and 0.53 for EQ-5D; and 35 and 41 for EQ-VAS. Responsiveness tested with hypotheses favours the shoulder-specific outcomes. OISS, WOSI and Rowe score are more responsive than EuroQol in evaluation of patients with SLAP lesions.

Minimally important change was estimated for the Manchester–Oxford Foot Questionnaire after foot/ankle surgery

ObjectivesTo ascertain the smallest amounts of change for the three Manchester–Oxford Foot Questionnaire (MOXFQ) domains that are likely to be clinically meaningful and beyond measurement error for conditions affecting the foot/ankle. Estimates were compared with those from the Short-Form 36 (SF-36). Study Design and SettingA prospective observational study of 671 consecutive patients undergoing foot or ankle surgery at an orthopedic hospital. Before and 9 months after surgery, patients completed the MOXFQ and SF-36; transition items (anchor) asked about perceived changes in foot/ankle pain or problems since the surgery. ResultsFour hundred ninety-one patients completed pre- and postoperative questionnaires. Anchor-based minimal clinically important change (MCIC) values were ∼13 points for each of the MOXFQ Walking/standing (W/S), Pain, and Social Interaction (S-I) domains [and greater than the standard error of measurement (SEM)]. MCIC values for all SF-36 domains fell within the SEM. Between-group MCIDs for the MOXFQ were W/S, 16.2; Pain, 9.9; S-I, 9.3. Distribution-based minimal detectable change (MDC90) values for the MOXFQ were ∼11, ∼12, and ∼16 score points for the W/S, Pain, and S-I scales, respectively. ConclusionThis article provides information for aiding the interpretability of MOXFQ outcomes data and for planning future studies. The SF-36 is not recommended as a primary outcome for foot/ankle surgery.

Guideline‐based development and practice test of quality indicators for physiotherapy care in patients with neck pain

Little is currently known about the quality of physiotherapy care for patients with musculoskeletal pain. Neck pain was used as an example. The aim is to develop a set of quality indicators, including a practice test. A systematic method is used to develop potential process and outcome indicators. An expert and user panel is used to appraise the potential quality indicators regarding clarity, relevancy, feasibility, acceptability and improvement potential. An invitation to participate in the practice test was sent to physiotherapy practices. The resulting algorithm is used to calculate the degree to which physiotherapists met these indicators (0-100%). Differences in valid outcomes are tested for significance (Student's t-test; α = 0.05) and compared with established values for clinical relevance [minimal clinically important change (MCIC)]. A representative set of 40 quality indicators (28 process indicators and 12 outcome indicators) is selected from 44 initial guidelines and literature-based recommendations. The process indicators (n = 28) are classified per step of the clinical reasoning process of physiotherapy care. Of the 106 potential participants from 27 practices, 38 physiotherapists (35.8%) submitted data on 96 patients with non-specific neck pain. On average, the participating physiotherapists showed a 55.6% adherence to process indicators with a great variation in scores per step of the clinical reasoning process. The outcomes for 'pain', 'headache' and 'daily functioning' were significantly better compared with baseline, and the mean differences exceeded established values for MCICs. Guardedly, we can conclude that a systematic approach is a valuable means to develop a preliminary set of process and outcome indicators for physiotherapy care for patients with non-specific neck pain, and a practice test should be an intrinsic part of such a systematic approach as it provides valuable information on the key attributes of the set indicators.

Clinimetric properties of the EuroQol-5D in patients with chronic low back pain

Clinimetric properties of the EuroQol-5D (EQ-5D) in patients with nonspecific chronic low back pain (CLBP) are largely unknown. To study the criterion validity, responsiveness, and minimal clinically important change (MCIC) of EQ-5D in patients with CLBP. Prospective study design carried out in a multispecialist Spine Center in The Netherlands. One hundred fifty-one patients with CLBP. Quality of life (QOL) was measured with EQ-5D, consisting of two scales: one scale measuring QOL with five categorical questions and the other measuring health state on a visual analog scale (0-100). Criterion measures were disability, measured with the Pain Disability Index (PDI) and the Roland Morris Disability Questionnaire (RMDQ), and pain intensity, measured with a numeric rating scale (NRS). Pearson correlation coefficients between the EQ-5D and RMDQ, PDI, and NRS were calculated to test the criterion validity. Correlations were interpreted based on predefined criteria. Responsiveness of the EQ-5D was calculated with area under the receiver operating characteristics (ROC) curve. Minimal clinically important change was calculated with the optimal cutoff point under the ROC curve, and sensitivity and specificity were also calculated. Correlations between EQ-5D and criterion measures ranged between 0.39 and 0.59 and were considered moderate to good. Areas under the ROC curve ranged from 0.59 to 0.72 depending on the external criterion and EQ-5D subscale. The MCIC was 0.03 points for the categorical scales of the EQ-5D and 10.5 points for the EQ-5D visual analog scale. The EQ-5D is a valid and responsive QOL scale in patients with CLBP.

Minimal clinically important change on the Headache Impact Test-6 questionnaire in patients with chronic tension-type headache

To determine the minimal clinically important change (MCIC) in Headache Impact Test-6 (HIT-6) score in patients with chronic tension-type headache (CTTH). The HIT-6 was administered at baseline and at 8 weeks follow-up in a cohort of 186 participants with CTTH who received manual therapy or usual care by their general practitioner. An anchor-based method was used to determine the MCIC, with the external criterion (anchor) being based on general perceived improvement ('much improved or very much improved' on a 7-point Likert scale) in combination with 50% reduction in headache days. Using receiver operating characteristic (ROC) curve analysis we defined an optimal cut-off score discriminating between improved and not improved participants. The optimal cut-off point for the MCIC for the HIT-6 was -8 points, on a total scale range of 42 points. A clinically relevant improvement in patients with CTTH is reflected by a decrease of at least 8 points on the HIT-6.

Responsiveness and Minimal Clinically Important Change of the Pain Disability Index in Patients With Chronic Back Pain

Prospective cohort study. The objective of this study was to test the responsiveness and minimal clinically important change (MCIC) of the Pain Disability Index (PDI) in patients with chronic back pain (CBP). Treatment of patients with CBP is primarily focused on reduction of disability. For disability measurement, the PDI is a widely used questionnaire. There are, however, no data available on responsiveness and MCIC. Two hundred forty-two patients with CBP were included in this study. Patients filled in the PDI at baseline and at discharge. The PDI consists of 2 subscales: 1 measuring voluntary activities and 1 measuring obligatory activities. PDI was anchored at 2 self-reported global perceived effect (GPE) scales for complaints and self-care, respectively. Responsiveness was considered sufficient when Area Under the Receiver Operating Characteristics (ROC) Curve (AUC) was higher than 0.70. To test interpretability, change scores and MCIC were calculated. MCIC was tested by determination of optimal cut-off point of the ROC curve and determination of specificity and sensitivity of the optimal cut-off point. AUCs were 0.76 and 0.77 depending on the external criterion. The subscale obligatory activities did not meet the criteria for responsiveness (AUC: 0.63-0.69). MCIC of the PDI was 9.5 points for GPE "complaints" and 8.5 for GPE "self-care." The total score of the PDI as well as the subscale of voluntary activities is responsive. Partly because of floor effects, the subscale obligatory activities are not sufficiently responsive in patients with CBP. However, the responsiveness of this subscale in other patient groups should be further tested. In patients with CBP, change can be considered clinically important when PDI score has decreased 8.5 to 9.5 points.

Poster #165 EFFICACY AND SAFETY OF DEEP BRAIN STIMULATION IN PATIENTS WITH A PSYCHIATRIC ILLNESS AND TARDIVE DYSKINESIA AND/OR DYSTONIA: A SYSTEMATIC REVIEW

To characterize the minimal clinically important change (MCIC) after treatment in cervical dystonia patients using the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS).Changes in the TWSTRS from an observational study of abobotulinumtoxinA in the routine management of cervical dystonia (NCT01314365) were analyzed using the Patient Global Impression of Change (PGIC) as anchor.For the overall population (N = 304, baseline TWSTRS-Total score 43.4 ± 19.4), the MCIC for the TWSTRS Total score was −11.9 (95%CI: −13.9, −10.0; p < 0.0001). However, thresholds ranged from −3.2 to −18.0 dependent on baseline severity. TWSTRS-Total scores improved linearly by 3 points for every one-point PGIC increase. There was similar linearity between the graded PGIC categories and TWSTRS subscale scores (severity, disability, and pain).A 3-point change is the minimal clinically important change after treatment using TWSTRS as endpoint with higher cutoffs for greater baseline disease severity. For an average trial population (TWSTRS-total: 40–45), a 12-point decrease is clinically meaningful.

The early outcome of surgical treatment for femoroacetabular impingement: success depends on how you measure it

To evaluate the proportion of "successes" after surgery for femoroacetabular impingement (FAI) using different external criteria, "feeling better" and "feeling good", and to determine the corresponding cut-off scores indicating "success" for the Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) (0-10-point response scale), Oxford Hip Score (OHS) and EuroQoL-5D (EQ-5D and EQ-VAS). Prospective, observational study based in an orthopaedic hospital. Ninety-nine consecutive patients with FAI completed the questionnaires before and 6 months after surgery (arthroscopy or mini-open surgical dislocation). Patient-ratings of change in state ("feeling better") were assessed using a global treatment outcome (GTO) item. Acceptability of the current health state was assessed using the symptom-specific well-being (SSWB) item. Cut-off (threshold) scores for the different instruments indicating the minimal clinically important change (MCIC) and acceptable symptom state were calculated using Receiver Operating Characteristics (ROC) analyses. Significant improvements in all scores (P < 0.001) were recorded 6 months after surgery. The proportion of good outcomes measured with GTO was 60%; 55% of patients reported having achieved an acceptable symptom state. The MCIC scores for improvement were ≥6 for the OHS (0-48 total score range), ≥15 for EQ-VAS, ≥0.16 for EQ-5D index, and ≥22 for the WOMAC-total score (0-100 total score range); absolute scores of ≥40, ≥80, ≥0.682 and ≤8, respectively, were associated with an acceptable symptom state. The results show that feeling better does not always equate to feeling good, and that improvements in outcome scores, even large, do not necessarily indicate acceptability of the current state. The cut-off values may help in the interpretation of trial results and individual change-scores recorded in clinical practice.

Responsiveness and Minimal Clinically Important Change in Overactive Bladder Symptom Score

To assess the responsiveness and minimal clinically important change (MCIC) in the Overactive Bladder Symptom Score (OABSS), a single score used to quantify overactive bladder symptoms. The data were derived from a clinical trial of propiverine in patients with overactive bladder. The analysis included participants who completed the OABSS, a bladder diary, and patient-reported outcome measures (urgency and incontinence impact) at baseline and 12 weeks. Responsiveness was assessed with effect sizes, correlations between the OABSS items and bladder diary variables, and comparisons between the score changes and symptom improvement. The MCIC was comprehensively estimated from the discriminating thresholds for minimal symptom improvement, and the receiver operating characteristics curve analyses were used to derive cutoff scores for symptom improvement. A total of 282 participants were included in the present analysis. The effect sizes for the OABSS ranged from -0.369 to -1.485, and correlations between the changes in the OABSS items and the corresponding bladder diary variables were moderate to large. A linear tendency was found between the changes in the OABSS and symptom improvement. The mean change for urgency and incontinence impact was -2.59 and -2.49 for "no change" and -3.67 and -3.78 for "minor improvement," respectively. The optimal cutoff score ranged from -4 to -3. Integrating the MCIC analyses, -3 was estimated as the minimal threshold for a meaningful change. With good responsiveness, the OABSS is a useful tool for assessing the treatment of OAB symptoms. Furthermore, the results of our study suggest that the MCIC of the OABSS is -3.

Determination of minimal clinically important change in early and advanced Parkinson's disease

Two common primary efficacy outcome measures in Parkinson's disease (PD) are change in Unified Parkinson's Disease Rating Scale (UPDRS) scores in early PD and change in "off" time in patients with motor fluctuations. Defining the minimal clinically important change (MCIC) in these outcome measures is important to interpret the clinical relevance of changes observed in clinical trials and other situations. We analyzed data from 2 multicenter, placebo-controlled, randomized clinical trials of rasagiline; TEMPO studied 404 early PD subjects, and PRESTO studied 472 levodopa-treated subjects with motor fluctuations. An anchor-based approach using clinical global impression of improvement (CGI-I) was used to determine MCIC for UPDRS scores and daily "off" time. MCIC was defined as mean change in actively treated subjects rated minimally improved on CGI-I. Receiver operating characteristic (ROC) curves defined optimal cutoffs discriminating between changed and unchanged subjects. MCIC for improvement in total UPDRS score (parts I-III) in early PD was determined to be -3.5 points based on mean scores and -3.0 points based on ROC curves. In addition, we found an MCIC for reduction in "off" time of 1.0 hours as defined by mean reduction in "off" time in active treated subjects self-rated as minimally improved on CGI-I minus mean reduction in "off" time in placebo-treated subjects self-rated as unchanged (1.9-0.9 hours). We hypothesize that many methodological factors can influence determination of the MCIC, and a range of values is likely to emerge from multiple studies.

Performance Tests in People With Chronic Low Back Pain

Cohort study. To assess the responsiveness and minimal clinically important change (MCIC) of 6 commonly-used performance tests (5-minute walking, 50-ft walking, sit-to-stand, 1 minute stair climbing, loaded forward reach, Progressive Isoinertial Lifting Evaluation). Performance tests are used to evaluate physical function in people with low back pain. Little is known about their clinimetric properties. Performance tests were administered in people with chronic nonspecific low back pain (n = 198) before and after 10 weeks of treatment. At 10 weeks, the global perceived effect scale was used to determine if participants judged themselves as worsened, unchanged, or improved. The mean change scores for each performance test were calculated. A performance test was considered responsive if the area under the receiver operating characteristic curve (AUC) was equal to or greater than 0.70. We used 2 methods to evaluate MCIC: the optimal cut-off point based on the receiver operating characteristic curve, which takes into account both sensitivity and specificity, and the minimal detectable change for improvement, which considers test specificity only. In general, the mean change scores were the smallest in participants who judged themselves worsened and largest in those reporting to be improved. Sit-to-stand (AUC = 0.75) and stair climbing (AUC = 0.72) were the only performance tests that showed adequate responsiveness. For sit-to-stand, the MCIC ranged from 4.1 to 9.8 seconds (19%-45% of the mean baseline score). For stair climbing, the MCIC ranged from 14.5 to 23.9 steps (19%-31% of the mean baseline score). Only 2 of the 6 performance tests were responsive. Both had acceptable MCIC values. Developing individualized performance tests might partly overcome the general lack of responsiveness of performance tests. Future research should focus on the clinimetric testing of performance tests in subgroups.

Responsiveness of the Bournemouth Questionnaire in Determining Minimal Clinically Important Change in Subgroups of Low Back Pain Patients

Prospective single cohort. To determine the ability of the Bournemouth Questionnaire (BQ) to distinguish between improved and nonimproved patients who present with either short (acute) or long (subacute/chronic) duration low back pain (LBP), and with either high or low baseline scores (severity). Recent evidence suggests that the responsiveness of outcome measures used to determine clinical change is dependent on the chronicity and severity of the condition. Data from 437 back patients undergoing chiropractic treatment were used for analysis. Patients completed the BQ before treatment and 4 weeks later. Patients also completed the Patient Global Impression of Change scale at follow-up. Responsiveness was determined by calculating Standardized Response Means (SRM) and by the area under the receiver operator curve (ROC) with best cut-point analysis. The minimal clinically important change (MCIC) was calculated by the change score with the best balanced sensitivity and specificity. The responsiveness of the BQ at 4 weeks was dependent on both duration and severity of the condition. As expected, the responsiveness of the total BQ was greater in improved compared to nonimproved patients in the acute (SRM [95% confidence interval], 1.9 [1.7-2.0] and 1.2 [0.9-1.5], respectively), as well as in the subacute/chronic group (SRM, 1.7 [1.5-1.8] and 0.5 [0.3-0.7]), respectively. For the psychological domains, SRMs in the acute patients failed to distinguish improved from nonimproved patients (SRM [95% confidence interval], 1.3 [1.1-1.4] and 0.9 [0.5-1.2] for anxiety, and 0.9 [0.8-1.0] and 0.8 [0.5-1.2] for depression). In acute and subacute/chronic patients, the MCIC for the total BQ was 26 and 18 points, respectively. In patients with lower and higher BQ scores at baseline, the MCIC was 10 and 31 points, respectively. The BQ can distinguish between improved and nonimproved LBP patients but the amount of change needed to achieve this is lower in more chronic patients and in individuals with less severe presentation at baseline.

A questionnaire found disease-specific WORC index is not more responsive than SPADI and OSS in rotator cuff disease

Objectives To compare responsiveness and minimal clinically important change (MCIC) for the disease-specific Western Ontario Rotator Cuff index (WORC) and the two region-specific questionnaires Shoulder Pain and Disability Index (SPADI) and Oxford Shoulder Scale (OSS) in patients with rotator cuff disease receiving corticosteroid injection therapy. Study Design and Setting One hundred twenty-one patients with rotator cuff disease. Western Ontario Rotator Cuff index, SPADI, and OSS were administered before treatment and at 2 and 6 weeks after corticosteroid injection. Responsiveness was compared between questionnaires using the standardized response mean (SRM), area under the receiver operating characteristic curve, and reliable change proportion (RCP) statistics. Minimal clinically important change estimates were reported. Results The differences between questionnaires were small and not consistent across the different responsiveness indices. Shoulder Pain and Disability Index was significantly more responsive than OSS measured by SRM and RCP at 2 and 6 weeks. Western Ontario Rotator Cuff index was significantly more responsive than OSS in RCP and area under receiver operating characteristic curve at 6 weeks. Shoulder Pain and Disability Index was significantly more responsive than WORC measured by RCP at 2 weeks. Minimal clinically important change was estimated to 5, 275, and 20 points for OSS, WORC, and SPADI, respectively. Conclusions All questionnaires are suitable for measuring change in patients with rotator cuff disease. Disease-specific WORC index is not more responsive than the region-specific SPADI and OSS in rotator cuff disease.

A Study of Minimal Change in Nocturia Affecting Quality of Life

Purpose: It is well known that nocturia is a bothersome symptom that affects the quality of life (QoL). However, it is not known how much of a decrease in nocturnal frequency is needed to improve the QoL. We determined the minimal change in nocturnal frequency affecting the QoL of patients with nocturia. Materials and Methods: A total of 87 patients with BPH and nocturia were treated with α-blocker and desmopressin for 8 weeks. Considering that the minimal clinically important change (MCIC) is different according to the baseline value, we divided the patients into two groups (group I, II). Group I had mild to moderate nocturia with voiding ＜4 times per night, and Group II had severe nocturia with voiding ≥4 times per night. Voiding diaries and King’s Health Questionnaires (KHQs) were evaluated before and after treatment. To determine the minimal change in nocturia affecting the QoL, statistical analysis was performed by Student's t-test and ANOVA test with the change in KHQ score according to decreased numbers of nocturnal frequency. Results: Groups I and II had 35 and 52 patients, respectively. The minimal decrease in nocturnal frequency associated with a statistically significant decrease in sleep/energy domain scores was 1 time in Group I and 2 times in Group II. Conclusions: These data show that decreases in nocturia of at least 1 time in patients with mild to moderate nocturia (＜4 times/night) and of 2 times in patients with severe nocturia (≥4 times/night) are needed to

Evidence-Based Medicine, Systematic Reviews, and Guidelines in Interventional Pain Management: Part 2: Randomized Controlled Trials

Evidence-based medicine (EBM) is a shift in medical paradigms and about solving clinical problems, acknowledging that intuition, unsystematic clinical experience, and pathophysiologic rationale are insufficient grounds for clinical decision-making. The importance of randomized trials has been created by the concept of the hierarchy of evidence in guiding therapy. Even though the concept of hierarchy of evidence is not absolute, in modern medicine, most researchers synthesizing the evidence may or may not follow the principles of EBM, which requires that a formal set of rules must complement medical training and common sense for clinicians to interpret the results of clinical research. N of 1 randomized controlled trials (RCTs) has been positioned as the top of the hierarchy followed by systematic reviews of randomized trials, single randomized trial, systematic review of observational studies, single observational study, physiologic studies, and unsystematic clinical observations. However, some have criticized that the hierarchy of evidence has done nothing more than glorify the results of imperfect experimental designs on unrepresentative populations in controlled research environments above all other sources of evidence that may be equally valid or far more applicable in given clinical circumstances. Design, implementation, and reporting of randomized trials is crucial. The biased interpretation of results from randomized trials, either in favor of or opposed to a treatment, and lack of proper understanding of randomized trials, leads to a poor appraisal of the quality. Multiple types of controlled trials include placebo-controlled and pragmatic trials. Placebo-controlled RCTs have multiple shortcomings such as cost and length, which limit the availability for studying certain outcomes, and may suffer from problems of faulty implementation or poor generalizability, despite the study design which ultimately may not be the prime consideration when weighing evidence for treatment alternatives. However, in practical clinical trials, interventions compared in the trial are clinically relevant alternatives, participants reflect the underlying affected population with the disease, participants come from a heterogeneous group of practice settings and geographic locations, and endpoints of the trial reflect a broad range of meaningful clinical outcomes. Key words: Randomized controlled trial (RCT), placebo-controlled trial, pragmatic controlled trial, randomization, allocation concealment, sample size, blinding, consolidated standards of reporting trials (CONSORT) statement, minimal clinically important change (MCIC), minimal clinical important difference (MCID)

Minimal Clinically Important Change for Pain Intensity and Disability in Patients With Nonspecific Low Back Pain

Cohort study. To estimate the minimal clinically important change (MCIC) on the pain intensity numerical rating scale (PI-NRS) and the Roland Morris Disability Questionnaire (RMQ) in subacute and chronic patients with low back pain (LBP), with and without referred pain to the leg (LP), seen in the routine clinical practice of the Spanish National Health Service. MCIC have been explored in Anglo-Saxon and Northern European LBP patients. No data on the influence of LP on MCIC are available. In Southern European patients, determinants of disability have shown to be different, and MCIC for pain and disability are unknown. Data from the postmarketing surveillance of 1349 LBP subacute and chronic patients treated in routine clinical practice were used for this study. Three different methods were used to estimate the MCIC over a 12-week period: the mean change score (MCS), the minimal detectable change (MDC), and the optimal cutoff point in receiver operant curves (OCP). Patients' own "global perceived effect" was used as the external criterion. The effect on MCIC of initial scores, duration of pain, and existence of LP were assessed. Different methods led to different MCIC values, with those deriving from OCP being the smallest. Depending on the methods which were used, the MCIC for LBP ranged from 1.5 to 3.2 PI-NRS points in patients with a baseline score below 7 points, and from 2.5 to 4.3 in patients with a baseline score >or=9 points. The MCIC for disability ranged from 2.5 to 6.8 RMQ points in those with baseline scores below 10 points, and from 5.5 to 13.8 in those baseline scores >or=15 points. These values were similar for patients with LP, and were not influenced by the duration of pain. In subacute and chronic patients, improvements in LBP of <or=1.5 PI-NRS points, or in disability of <or=2.5 RMQ points should be seen as irrelevant. In those patients, MCIC range values are similar in patients with LP, are consistent across different cultural settings and remain stable no matter what the duration of pain is.

Minimal Clinically Important Change of the Neck Disability Index and the Numerical Rating Scale for Patients With Neck Pain

Prospective, single-cohort study. To assess the minimal clinically important change (MCIC) on the Neck Disability Index (NDI) and the Numerical Rating Scale (NRS) for pain in patients with neck pain. Both measurement instruments are frequently used in research and clinical practice, but which changes are clinically relevant is still unknown. The MCIC was estimated with 2 different methods, both integrating an anchor-based and distribution-based approach: the minimal detectable change (MDC) and the optimal cutoff point of the ROC curve. The study population consisted of 183 patients with nonspecific neck pain. The results show an MDC of 10.5 points for the NDI (scale range, 0-50) and 4.3 points for the NRS (scale range, 0-10), and optimal cutoff points of the ROC curve of 3.5 for the NDI and 2.5 for the NRS. The estimated MCIC should be used as an indication for relevant changes in clinical practice. Using the optimal cutoff point of the ROC curve, false positives and false negatives are equally weighted; and if there are no objections doing so, the optimal cutoff point of the ROC curve may be a good choice.