The minimal important change (MIC) and minimal clinically important difference (MCID) of the patient and observer scar assessment scale (POSAS) 2.0.

BACKGROUND: The Patient and Observer Scar Assessment Scale (POSAS) is frequently used to assess scar quality after burns. It is important to be aware of the minimal important change (MIC) and the minimal clinically important difference (MCID) to establish if a POSAS score represents a clinically relevant change or difference. The aim of this study is to explore the MIC and MCID of POSAS version 2.0. METHODS: This prospective study included 127 patients with deep dermal burns that underwent split thickness skin grafting with a mean age of 44 years (range 0 - 87) and total body surface area burned of 10 % (range 0.5 - 55). POSAS data was obtained for one burn scar area at three, six, and 12 months after split skin grafting. At the second and third visits, patients rated the degree of clinical change in scar quality in comparison to the previous visit. At 12 months, they completed the POSAS for a second burn scar area and rated the degree of clinical difference between the two scar areas. Two anchor-based methods were used to determine the MIC and MCID. RESULTS: MIC values of the patient POSAS ranged from - 0.59 to - 0.29 between three and six months and from - 0.75 to - 0.38 between six and 12 months follow-up. Both had a poor discriminatory value. MCID values ranged from - 0.39 and - 0.08, with a better discriminatory value. CONCLUSION: Results suggest that patients consider minor differences (less than 0.75 on the 1-10 scale) in POSAS scores as clinically important scar quality changes. MCID values can be used to evaluate the effects of burn treatment and perform sample-size calculations.

TENDINopathy Severity assessment-Achilles (TENDINS-A): evaluation of reliability and validity in accordance with COSMIN recommendations.

OBJECTIVE: To evaluate the construct validity (structural validity and hypothesis testing), reliability (test-retest reliability, measurement error and internal consistency) and minimal important change (MIC) of the 13-item TENDINopathy Severity assessment-Achilles (TENDINS-A). METHODS: Participants with Achilles pain completed an online survey including: demographics, TENDINS-A, Foot and Ankle Outcome Score (FAOS) and Victorian Institute of Sport Assessment-Achilles (VISA-A). Exploratory factor analysis (EFA) assessed dimensionality. Confirmatory factor analysis (CFA) assessed structural validity (root mean square error of approximation (RMSEA); Comparative Fit Index (CFI); Tucker-Lewis Index (TLI); standardised root measure square (SRMS)). Correlations between TENDINS-A and the FAOS or VISA-A assessed hypothesis testing. Intraclass correlation (ICC) assessed test-retest reliability. Cronbach's alpha assessed internal consistency. SE of the measurement (SEM) assessed measurement error. A distribution-based approach assessed MIC. RESULTS: 79 participants (51% female) with a mean (SD) age=42.6 (13.0) years, height=175.0 (11.7) cm and body mass=82.0 (19.1) kg were included. EFA identified three meaningful factors, proposed as pain, symptoms and function. The best model identified using CFA for TENDINS-A had structural validity (RMSEA=0.101, CFI=0.959, TLI=0.947, SRMS=0.068), which included three factors (pain, symptoms and function), but excluded three items from the original TENDINS-A. TENDINS-A exhibited moderate positive correlation with FAOS (r=0.598, p<0.001) and a moderate negative correlation with VISA-A (r=-0.639, p<0.001). Reliability of the TENDINS-A was excellent (ICC=0.930; Cronbach's α=0.808; SEM=6.54 units), with an MIC of 12 units. CONCLUSIONS: Our evaluation of the revised 10-item TENDINS-A determined it has construct validity and excellent reliability, compared with the VISA-A and FAOS which lack content and construct validity. The TENDINS-A is recommended as the preferred patient-reported outcome measure to assess disability in people with Achilles tendinopathy.

Assessing Clinically Important Differences During Rehabilitation for Stroke: A Pilot Study Evaluating Anchor and Distribution Derived Estimates of Physical Function Change in Classically Summed and Rasch Models of Section GG of the Inpatient Rehabilitation Facility Patient Assessment Instrument.

OBJECTIVE: To determine clinically important differences (CIDs) on Section GG physical functioning scores on the Centers for Medicare and Medicaid Services (CMS) Inpatient Rehabilitation Facility Assessment Instrument (IRF-PAI) for patients with stroke, using anchor and distribution-based approaches. DESIGN: Pilot prospective observational cohort study. SETTING: Inpatient rehabilitation facility. PARTICIPANTS: Patients with stroke (N=208). INTERVENTIONS: Physicians assessed improvements during rehabilitation using the modified Rankin scale (mRS). Improvements (≥1 point) on the mRS were used as the anchor for establishing CIDs. MAIN OUTCOME MEASURES: Classically summed and Rasch transformed Section GG change scores associated with clinically important improvements on the mRS. RESULTS: A total of 166 patients (79.8%) improved ≥1 point on the mRS. Change scores of 27, 9, and 16 on Section GG total physical functioning (self-care + mobility), self-care, and mobility/walk scales, respectively, had high sensitivity (0.82-0.85) but low specificity (0.52-0.69) in identifying patients improving on the mRS. Positive predictive values ranged from 0.87 to 0.91, and negative predictive values ranged from 0.42 to 0.52. Total physical functioning and selfcare anchor-derived change scores were similar to the reliable change index (RCI [2.77 × SEM]), calculated as 28 and 10 points, respectively, whereas anchor-derived mobility/walk scale change scores were equivalent to 1.96 × SEM. Exploratory Rasch modeling identified 3 Section GG subscales (R-Self-Care, R-Mobility, and R-Walking). Improvements on the R-Walking subscale were most correlated with mRS improvements (ρ=-0.47); however, accuracy of CID estimates was not improved. CONCLUSIONS: Cut-off scores obtained using the mRS anchor aligned with more robust estimates of change, as estimated by distribution-based measures. While patients achieving anchor-derived cut-offs have a high probability of mRS improvement, change scores may fail to detect clinically meaningful improvements at these same thresholds. Alternative criteria for determining MCID/CIDs, should be explored. Rasch models require further validation.

Use of Area Deprivation Index to Predict Minimal Clinically Important Difference for Patient Reported Outcomes Measurement Information System After Arthroscopic Rotator Cuff Repair.

BACKGROUND: Socioeconomic disparities correlate with worse outcomes after arthroscopic rotator cuff repair. However, use of a surrogate to describe socioeconomic disadvantage has been a challenge. The Area Deprivation Index (ADI) is a tool that encompasses 17 socioeconomic variables into a single metric based on census location. HYPOTHESIS: Higher ADI would result in a worse minimal clinically important difference (MCID) for the Patient Reported Outcomes Measurement Information System (PROMIS) and have less improvement in range of motion (ROM) following arthroscopic rotator cuff repair (ARCR). STUDY DESIGN: Cohort study; Level of evidence, 3. METHOD: A retrospective review was performed for patients who underwent arthroscopic rotator cuff repair. Patients in the most socioeconomically disadvantaged quartile (ADIHigh) were compared with the least disadvantaged quartile (ADILow) in the ability to reach MCID. Demographic and surgical features were assessed for attainment of MCID. RESULTS: In total 1382 patients were identified who underwent ARCR, of which a total of 306 patients met final inclusion criteria. A higher percentage of patients within the ADIHigh cohort identified as "Black" or "other" race and had government-issued insurance compared with the ADILow cohort (P < .05). The ADIHigh cohort had significantly worse postoperative forward flexion compared with the ADILow cohort (145.0°± 32.5° vs 156.3°± 23.4°; P = .001) despite starting with comparable preoperative ROM (P = .17). Logistic regression showed that ADI was the only variable significant for predicting achievement of MCID for all 3 PROMIS domains, with the ADIHigh cohort having significantly worse odds of achieving MCID Physical Function (odds ratio [OR], 0.31; P = .001), Pain Interference (OR, 0.21; P = .001), and Depression (OR, 0.28; P = .001). Meanwhile, age, sex, body mass index, and smoking history were nonsignificant. Moreover, "other" for race and Medicare insurance were significant for achievement of MCID Depression but not Physical Function or Pain Interference. Finally, ADI was the main feature for predictive logistic regression modeling. CONCLUSION: ADI served as the only significant predictor for achieving MCID for all 3 PROMIS domains after arthroscopic rotator cuff repair. Patients who face high levels of socioeconomic disadvantage have lower rates of achieving MCID. In addition, patients with greater neighborhood disadvantage demonstrated significantly worse improvement in active forward flexion. Further investigation is required to understand the role of ADI on physical therapy compliance and to identify the barriers that prevent equitable postoperative care.

Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy.

The North Star ambulatory assessment (NSAA) is a functional motor outcome measure in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural history studies, as well as in clinical practice. However, little has been reported on the minimal clinically important difference (MCID) of the NSAA. The lack of established MCID estimates for NSAA presents challenges in interpreting the significance of the results of this outcome measure in clinical trials, natural history studies and clinical practice. Combining statistical approaches and patient perspectives, this study estimated MCID for NSAA using distribution-based estimates of 1/3 standard deviation (SD) and standard error of measurement (SEM), an anchor-based approach, with six-minute walk distance (6MWD) as the anchor, and evaluation of patient and parent perception using participant-tailored questionnaires. The MCID for NSAA in boys with DMD aged 7 to 10 years based on 1/3 SD ranged from 2.3-2.9 points, and that on SEM ranged from 2.9-3.5 points. Anchored on the 6MWD, the MCID for NSAA was estimated as 3.5 points. When the impact on functional abilities was considered using participant response questionnaires, patients and parent perceived a complete loss of function in a single item or deterioration of function in one to two items of the assessment as an important change. Our study examines MCID estimates for total NSAA scores using multiple approaches, including the impact of patient and parent perspective on within scale changes in items based on complete loss of function and deterioration of function, and provides new insight on evaluation of differences in these widely used outcome measure in DMD.

Defining Clinically Significant Outcomes Following Superior Capsular Reconstruction With Acellular Dermal Allograft.

PURPOSE: To define clinically significant outcomes (CSO) thresholds for minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient-acceptable symptomatic state (PASS) in patients undergoing superior capsular reconstruction (SCR) with an acellular dermal allograft. We also evaluated patient-specific variables predictive of achieving CSO thresholds. METHODS: The American Shoulder and Elbow Surgeons Standardized Shoulder Assessment Form (ASES), Single Assessment Numeric Evaluation (SANE), and subjective Constant-Murley (Constant) scores were collected preoperatively and at the most recent follow up for patients undergoing SCR from 2010 to 2019. A distribution-based approach was used to calculate MCID, and an anchor-based approach was used to calculate SCB and PASS. Logistic regression was used to determine factors associated with CSO achievement. RESULTS: Fifty-eight patients were identified (n = 39 males; n = 19 females) with a mean age of 53.4 ± 14.1 years at surgery and an average follow-up of 23 months. The MCID, SCB, and PASS were 11.2, 18.02, and 68.82 for ASES, 14.5, 23.13, and 69.9 for SANE, and 3.6, 10, and 18 for Constant, respectively. Subscapularis tear, female sex, and workers compensation (WC) status reduced odds of achieving MCID. Reduced odds of achieving Constant SCB were associated with older age, female sex, and WC status, while concomitant distal clavicle excision during SCR and lower preoperative ASES increased odds of achieving ASES SCB. Reduced odds for achieving ASES PASS were associated with female sex and WC status, while reduced odds for achieving SANE PASS were associated with subscapularis tearing preoperatively. CONCLUSION: On the basis of calculated values for MCID, SCB, and PASS, subscapularis tearing, WC status, age, and sex are associated with failure to achieve clinically significant outcomes following SCR. Concomitant distal clavicle excision during SCR and lower preoperative ASES was predictive for achievement of MCID and SCB. By defining the thresholds and variables predictive of achieving CSOs following SCR, surgeons may better counsel patients prior to SCR. LEVEL OF EVIDENCE: Level IV, case series.

Minimal Clinically Important Differences (MCID) for the Functional Assessment of Chronic Illness Therapy Fatigue Scale in Patients with Systemic Sclerosis.

(1) Background: Systemic sclerosis (SSc) is characterized by significant fatigue, causing diminished quality of life (QoL). The aim of this study was to examine fatigue levels and their associations with clinical factors and determine the minimal clinically important difference (MCID) value for the Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-FS). (2) Methods: A total of 160 SSc patients and 62 individuals without SSc were followed-up over a 12-month period by measuring the FACIT-FS and the Visual Analogue Scale and the Short Form 36 Vitality Score analyzing changes in exhaustion. (3) Results: Fatigue was strongly correlated with HRQoL, level of pain, emotional disorders, physical capability and functionality. The MCID values for FACIT-FS were calculated as -3 for deterioration and +4 for improvement after a 12-month follow-up. The predictors of improvement of fatigue from baseline parameters were the significant disease activity, the patients' poorer functionality and the short disease duration. Patients with scleroderma-related interstitial lung disease at baseline had approximately tripled risks for worsening fatigue. The independent influential factors regarding the changing of FACIT-FS were improving or worsening in the same direction in reference to physical condition, gastrointestinal and emotional factors. (4) Conclusions: Fatigue is a multi-dimensional symptom, which is strongly correlated to HRQoL. MCID values of FACIT-FS can be useful tools in monitoring the changes of HRQoL in clinical trials and in daily practice among patients with SSc.

Minimal clinically important difference of the gait assessment and intervention tool for adults with stroke.

BACKGROUND: The Gait Assessment and Intervention Tool (GAIT) provides a valid and reliable gait quality measure for adults with stroke, however a minimal clinically important difference (MCID) is yet to be determined. RESEARCH QUESTION: What is the GAIT MCID in community dwelling adults with a stroke diagnosis? METHODS: The GAIT scores for a consecutive series of 63 adults with stroke, referred for video gait assessment within an outpatient rehabilitation program, were retrospectively identified from a gait laboratory database. Patients were classified by Functional Ambulation Category (FAC) and had walking speed measured, which classified them into one of three walking speed-based ambulatory levels (I.e. household, limited community or community ambulator). Linear regression models were fitted to assess the association between GAIT score and FAC level and GAIT score and ambulatory level. MCIDs were determined based on estimates calculated in the two models RESULTS: The FAC of patients ranged from 3 to 5. GAIT score was negatively correlated with FAC level and ambulatory level (ρ = -0.73, p < 0.001 and ρ =-0.69, p < 0.001, respectively). Pairwise comparisons from the models showed absolute mean differences between estimated GAIT scores of 10.84 (95% confidence interval 7.59-14.09) for changes from FAC level 3-4 and 12.13 (8.90-15.36) for household to limited community ambulator, and 5.90 (3.44-8.37) for changes from FAC level 4-5 and 4.39 (2.01-6.76) for limited community to community ambulator. The proposed MCID for FAC level 3 or household ambulators is 11.48, and for FAC level 4 and 5 or limited community/community ambulators is 5.19. SIGNIFICANCE: The proposed MCIDs represent real changes in gait quality measured by the GAIT for adults with stroke who exhibit lower or higher functional mobility levels. The MCIDs will assist clinicians and researchers using the tool to determine if meaningful change in gait quality has taken place for adults with stroke undergoing rehabilitation.

Functional capacity assessment and Minimal Clinically Important Difference in post-acute cardiac patients: the role of Short Physical Performance Battery.

BACKGROUND: The Short Physical Performance Battery (SPPB) test is a well-established tool to assess physical performance, and to identify frail patients. Assessment of the SPPB in a specific population of elder patients in cardiac rehabilitation phase after a cardiac event is missing. AIM: The aim of this study was to correlate SPPB and the cardiac rehabilitation outcome in a group of elder patients after a cardiac event and to identify the Minimal Clinically Important Difference (MCID) of the SPPB. METHODS: Consecutive (n = 392) patients aged ≥75 years, in the rehabilitation phase after cardiac surgery (70.1%), congestive heart failure (7.4%), or acute coronary syndrome (22.5%), were enrolled. SPPB was performed twice: on admission and discharge. The MCID was assessed with the 'anchor method', and the Patient Global Impression of Change was employed as the anchor. RESULTS: On admission, SPPB classified 56, 117, 116, and 94 patients as severe, moderate, mild, or minimal/no limitations, respectively. Patients with the lower SPPB had the longer length of stay, and the higher complications rate. At receiver operating characteristic analysis, an SPPB improvement >1 was identified as the MCID (area-under-curve 0.77, 95% CI 0.67-0.85). Overall, 285 patients (74.2%) had a 'clinically significant' improvement in SPPB, with a rate of improvement higher in patients with severe/moderate limitations (83.0%) and lower in those with mild (78.9%) or minimal/no limitations (53.6%). CONCLUSION: A lower SPPB score is associated with a higher complications rate in the post-acute phase. An improvement >1 point of SPPB was identified as the MCID; this reference value could serve as the goal for rehabilitation interventions.

Validation of the minimal clinically important difference for modified activities assessment scale.

BACKGROUND: The minimal clinically important difference (MCID) is the smallest change in patient-derived scores that is clinically important. We sought to validate the MCID of the modified activities assessment scale (mAAS). METHODS: Patients were surveyed prior to undergoing abdomen/pelvis CT scans and resurveyed one year later. Before resurvey, patients were asked if they had no change, worsening, or improvement in AW-QOL. The anchor-based MCID was calculated by taking a weighted mean of the difference between control (no change) and study (worsening/improved) groups. Distribution-based approach was calculated by one-half of the standard deviation in the QOL change. RESULTS: 52.8% of 181 patients self-reported no change, 39.2% reported improvement, and 8.3% reported worsening AW-QOL. The anchor-based approach MCID was 4. The distribution-based MCID was 16. CONCLUSION: Our study results validate prior work demonstrating similar ranges of the mAAS MCID. We recommend adopting an MCID of 5 and 15 for AW-QOL with mAAS.

Reliability, validity, and minimal clinically important differences of the Japanese version of the early clinical assessment of balance in children with cerebral palsy.

PURPOSE: We aimed to translate the Early Clinical Assessment of Balance (ECAB) from English to Japanese and examine the content validity, inter-rater reliability, intra-rater reliability, construct validity, and minimal clinically important difference (MCID) for children with cerebral palsy (CP). METHODS: The ECAB was translated into Japanese per international standards. The study included 106 children with CP and, aged 1.5-12 years. The ECAB, the Gross Motor Function Classification System (GMFCS), and the Gross Motor Function Measure 66 Basal & Ceiling (GMFM-66-B&C) were measured. The content and construct validity were examined based on therapist feedback and correlations between the ECAB and GMFM-66-B&C. The inter-rater reliability and the intra-rater reliability were examined by the intra-class correlation coefficient (ICC). The MCID was calculated by the anchor-based method with the GMFM-66-B&C. RESULTS: High content validity (more than 80% agreement), inter-rater and intra-rater reliability (ICC = 0.99 & 0.99, respectively), and construct validity (r = 0.96) were demonstrated, with MCID values of 7.39, 5.32, and 6.88 observed for the GMFCS I/II, III, and IV/V, respectively. CONCLUSION: The Japanese version of the ECAB is a reliable and valid measure of balance ability in children with CP. Furthermore, the MCID of the ECAB was established, appears to be useful in helping to provide rehabilitation.Implications for RehabilitationThe Japanese version of the Early Clinical Assessment of Balance is easy, safe, and low-cost, and has high reliability and validity for assessing balance ability in children with cerebral palsy.The use of the Japanese version of the Early Clinical Assessment of Balance is beneficial for determining the therapeutic effect, appropriate treatment, and prediction of prognosis regarding balance ability in children with cerebral palsy.The minimal detectable change of the Japanese version of the ECAB suggest that a score exceeding 6 is a true change and the minimal clinically important difference of the Japanese version of the ECAB suggest that the scores exceeding 8, 6, and 7 for the GMFCS I/II, III, and IV/V, respectively, is a clinically useful change.

The minimal clinically important change in the motor section of the Burke-Fahn-Marsden Dystonia Rating Scale for generalized dystonia: Results from deep brain stimulation.

BACKGROUND: The minimal clinically important difference (MCID) describes the smallest change in an outcome that is considered clinically meaningful. The Burke-Fahn-Marsden Dystonia Rating Scale (BFMDRS) is the most frequently rating scale assessing the efficacy of deep brain stimulation therapy (DBS) for dystonia. To expand our understanding, we evaluated the MCID thresholds for the BFMDRS motor subscale (MS) using physician-reported outcomes. METHODS: We assessed the MCID thresholds for the BFMDRS using movement disorder specialist ratings of videotapes from patients with genetically determined dystonia (Tor1A and THAP1) who underwent bilateral globus pallidum internum (GPi) DBS. We calculated the effect size of the BFMDRS-MS change and determined the MCID thresholds using the Clinical Global Impression of Change (CGIC). RESULTS: Twelve participants with a median age at DBS of 44.5 (range:27-68) had baseline and follow-up BFMDRS-MS with a median post-DBS follow-up of 5.5 years. Based on descriptive analysis, patients with good improvement after DBS according to the CGIC [8/12 (67%)] had a median BFMDRS-MS score reduction of 77% [Interquartile range (IQR):66.2;91.0) with an effect size of 0.39, and those with non-improvement [4/12 (33%)], had a median BFMDRS-MS score reduction of 62% (IQR:36.6;83.6). CONCLUSIONS: Our MCID estimates can be utilized in clinical practice in judging clinical relevance. However, further larger, powered studies are needed to simultaneously determine and compare MCID using patient and physician-reported outcomes in segmental and generalized dystonia in genetic and non-genetic populations.

Victorian Institute of Sport Assessment-Achilles (VISA-A) Questionnaire-Minimal Clinically Important Difference for Active People With Midportion Achilles Tendinopathy: A Prospective Cohort Study.

OBJECTIVE: To determine the minimal clinically important difference (MCID) for the Victorian Institute of Sport Assessment-Achilles (VISA-A) score in patients with midportion Achilles tendinopathy (AT). DESIGN: Prospective cohort study. METHODS: We included physically active patients with midportion AT who received exercises and an injection. We measured the VISA-A score (ranging from 0 to 100 points, where 100 points represents a healthy tendon) at baseline and at 12 weeks and 24 weeks after treatment, and the 7-point Global Assessment Scale (ranging from "worse than ever" to "completely recovered") at 12 weeks and 24 weeks after treatment. We dichotomized the Global Assessment Scale to not improved ("worse than ever" to "unchanged") or improved ("moderately improved" to "completely recovered"). The area under the curve and the Youden's index value closest to 1 were determined for both MCIDs (12 and 24 weeks), with corresponding sensitivity and specificity. RESULTS: Sixty-four patients were included, and 61 patients (95%) completed the 24-week follow-up. The MCID was 14 points (95% confidence interval [CI]: 3, 19) over a 12-week period, corresponding to 57% sensitivity and 88% specificity. The MCID was 7 points (95% CI: -10, 28) over a 24-week period, with 85% sensitivity and 62% specificity. CONCLUSION: A change in VISA-A score of at least 14 points after 12 weeks or at least 7 points after 24 weeks of exercise therapy and an injection reflects a meaningful change for physically active patients with midportion AT. J Orthop Sports Phys Ther 2021;51(10):510-516. doi:10.2519/jospt.2021.10040.

Validation of the Oxford Depression Questionnaire: Sensitivity to change, minimal clinically important difference, and response threshold for the assessment of emotional blunting.

BACKGROUND: The Oxford Depression Questionnaire (ODQ) is a patient-reported scale for assessing emotional blunting in patients with major depressive disorder (MDD). This analysis was undertaken to further validate the scale in patients experiencing emotional blunting while receiving antidepressant treatment. METHODS: Patients with MDD who experienced inadequate depressive-symptom resolution and emotional blunting on selective serotonin reuptake inhibitor or serotonin-noradrenaline reuptake inhibitor monotherapy (adequate dose for ≥6 weeks) were switched to vortioxetine 10-20 mg/day. ODQ total scores were assessed excluding and including the "antidepressant-as-cause" domain (ODQ-20 and ODQ-26, respectively). Anchor- and distribution-based methods were used to determine the minimal clinically important difference in ODQ scores in terms of change from baseline to week 8 of antidepressant treatment. RESULTS: After 8 weeks of vortioxetine treatment, the mean change in ODQ-20 and ODQ-26 scores from baseline was -24.8 and -30.1 points, respectively. Greater mean changes from baseline in ODQ-20 and ODQ-26 scores were seen in patients reporting no emotional blunting vs those still experiencing emotional blunting after 8 weeks of vortioxetine treatment (ODQ-20: -27.0 vs -22.6 points; ODQ-26: -32.8 vs -27.5 points, respectively). In patients considered clinically minimally improved (Clinical Global Impression-Improvement score, 3) after 8 weeks of vortioxetine treatment, respective mean (standard deviation) change in ODQ-20 and ODQ-26 score from baseline was -15.5 (18.1) and -20.0 (20.5) points. LIMITATIONS: Short study duration. CONCLUSIONS: These results provide further validation of the clinical utility of the ODQ for assessing emotional blunting in patients with MDD. The suggested minimal clinically important difference for change in ODQ-20 and ODQ-26 scores is 16 and 20 points, respectively, after 8 weeks of antidepressant treatment. TRIAL REGISTRATION: ClinicalTrials.gov identifier: NCT03835715.

Minimal clinically important difference of Gait Assessment and Intervention Tool (GAIT) in patients with sub-acute stroke.

BACKGROUND: The Gait Assessment and Intervention Tool (GAIT) is a well-accepted tool to determine gait characteristics in neurological patients. AIM: The aim of this study was to determine the minimal clinically importance difference (MCID) of the GAIT in sub-acute stroke patients. DESIGN: Prospective study. SETTING: An in- and out-patient rehabilitation clinic in a university hospital in Thailand. POPULATION: Patients with sub-acute stroke who had an ongoing rehabilitation between October 2017 to March 2019. METHODS: We used an anchor-based method to determine change in GAIT score needed to achieve MCID. Participants were dichotomized to 'no change group' or 'positive change group' based on different anchors of objective and subjective perceived changes in gait function after 4 weeks of rehabilitation. The groups were determined based on whether 1) participants achieved an increase in comfortable gait speed (CGS) of ≥ 0.06 m/s, 2) averaged Global Rating of Change (GROC) score was ≥+3 evaluated by two physiatrists 3) GROC score of was ≥+3 rated by the participants. The best cut-off point was the score which most successfully separated these two groups within the ROC curve. RESULTS: Thirty-one participants with sub-acute stroke (18 males, 13 female) completed both assessments. Their average age was 60.3±11.4 years. The best cut-off point were a 2.5- and 4-point improvements in GAIT score based on changes in CGS (AUC 0.76, 95% CI 0.58-0.95) and clinicians' perceived changes (AUC 0.88, 95% CI 0.76-1.00). Additionally, the best cut-off point was 1.5 (AUC 0.71 95% CI 0.31-1.00) when determined by participants' perception. All anchors yielded adequate discriminative ability. The positive likelihood ratio (LR) was in the range of 2.7-5.5 and the negative LR range was 0.1-0.3. CONCLUSIONS: A change in GAIT score should exceed 1.5, 2.5 and 4 to be considered MCID based on participants' GROC, CGS and Clinicians' GROC anchors. CLINICAL REHABILITATION IMPACT: This information will be useful for the determination of changes after rehabilitation and for tracking sub-acute stroke patients' progress.

The need to show minimum clinically important differences in Alzheimer's disease trials.

Deciding on the smallest change in an outcome that constitutes a clinically meaningful treatment effect (ie, the minimum clinically important difference [MCID]) is fundamental to interpreting clinical trial outcomes, making clinical decisions, and designing studies with sufficient statistical power to detect any such effect. There is no consensus on MCIDs for outcomes in Alzheimer's disease trials, but the US Food and Drug Administration's consideration of aducanumab clinical trials data has exposed the uncertainty of the clinical meaning of statistically significant but small improvements. Although MCIDs for outcomes, including Clinical Dementia Rating-Sum of Boxes and Mini-Mental State Examination in Alzheimer's disease have been reported, the Food and Drug Administration's guidelines, drafted in 1989 to facilitate regulatory approval of substantially effective antidementia drugs, do not specify quantified minimum differences. Although it is important that regulatory requirements encourage drug development and approval, without MCIDs, sponsors are motivated to power trials to detect statistical significance for only small and potentially inconsequential effects on clinical outcomes. MCIDs benefit patients, family members, caregivers, and health-care systems and should be incorporated into clinical trials and drug development guidance for Alzheimer's disease.

Minimal clinically important difference in walking velocity, gait profile score and two minute walk test for individuals with lower limb amputation.

BACKGROUND: Individuals with lower limb amputation are routinely assessed with a variety outcome measures, however there is a lack of published data to indicate minimal clinically important differences (MCID) for many of these outcome measures. Three such important gait-specific outcome measures include walking velocity, gait profile score (GPS) and the two minute walk test (2MWT). RESEARCH QUESTION: Determine the MCIDs for walking velocity, GPS and 2MWT for individuals with lower limb amputation. METHODS: Walking velocity and GPS (n = 60), and 2MWT (n = 119) data for individuals with unilateral transfemoral or knee disarticulation were identified retrospectively from a database held at the study centre. An anchor-based method was used with Medicare functional classification level (MFCL) acting as the impairment-related criterion, and a least-squares linear regression approach was used to calculate the gradient required for a change between MFCL levels. RESULTS: An increase of 0.21 m/s (95 % CI: 0.13,0.29) for walking velocity, a reduction of 1.7° (95 % CI: -2.449,-1.097) for GPS and an increase of 37.2 m (95 % CI: 28.8,45.5) for 2MWT were found to correspond to an increase in MFCL of one level. Walking velocity, GPS and 2MWT correlated with MFCL with R2 values of 0.333, 0.322 and 0.398 respectively (p < 0.00001). The authors propose that 0.21 m/s for walking velocity, 1.7° for GPS and 37.2 m for 2MWT be used as MCID values for individuals with lower limb amputation. SIGNIFICANCE: The results of this study can be used to help both researchers and clinicians to objectively evaluate if interventions for individuals with lower limb amputation are effective.

The Single Assessment Numeric Evaluation (SANE) after anterior cervical discectomy and fusion: A pilot study.

STUDY DESIGN: Retrospective cohort. OBJECTIVES: To evaluate the single assessment numerical evaluation (SANE) as a patient reported outcomes measure (PROM) after anterior cervical discectomy and fusion (ACDF), by comparing to legacy measures. METHODS: We included all patients undergoing ACDF with at least one year of follow up with complete PROM data. Patients completed the Neck Disability Index (NDI), the RAND-36 and the EuroQual Five Dimension (EQ-5D) scale, as well as the one-question SANE, pre- and post-operatively. Validity of SANE compared with other PROMs was determined utilizing Pearson's correlation (ρ), proportional bias (B), responsiveness, minimal clinically important difference (MCID) and agreement. RESULTS: Sixty-nine patients were included. There were moderate-to-strong correlations at a minimum of one-year follow-up between the SANE and NDI (ρ = -0.73, P < 0.0001), RAND (ρ = 0.80, P < 0.0001), and EQ-5D (ρ = -0.66, P < 0.0001). No significant proportional bias was found for the SANE when compared to the RAND (B = 0.03, p = 0.99), NDI (B = -0.003, p = 0.99), or EQ-5D (B = -0.0007, p = 0.99). Responsiveness for SANE was statistically similar to all other PROMs. The MCID for SANE was determined to be 10.5, with 42% of patients achieving the MCID. Bland-Altman plots demonstrated high agreement between all PROMs. CONCLUSION: We found the SANE score provides clinically important patient outcomes data after ACDF, despite only requiring answering one question. The SANE performs comparably to more burdensome health questionnaires. The SANE score may offer spine surgeons the option to easily and quickly collect clinically relevant data on their surgical patients.

Assessment of Food and Drug Administration- and European Medicines Agency-Approved Systemic Oncology Therapies and Clinically Meaningful Improvements in Quality of Life: A Systematic Review.

Importance: For patients with cancer treated with palliative intent, quality of life (QOL) is a critical aspect of treatment decision-making, alongside survival. However, regulatory approval can be based solely on survival measures or antitumor activities, without QOL evidence. Objective: To investigate whether recently approved oncology therapies demonstrate clinically meaningful improvements in QOL. Evidence Review: This systematic review study identified oncology drug indications approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) from January 2006 to December 2017 and supporting clinical trials (QOL publications identified to October 2019). Indications were evaluated for the presence of published QOL evidence; QOL benefits according to the American Society of Clinical Oncology Value Framework version 2.0 (ASCO-VF) and European Society of Medical Oncology Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS) QOL bonus criteria; and clinically meaningful improvements in QOL beyond minimal clinically important differences. Hematology trials were not evaluated by ESMO-MCBS. Associations between QOL evidence and approval year were examined using logistic regression models. Findings: In total, 214 FDA-approved (77 [36%] hematological) and 170 EMA-approved (52 [31%] hematological) indications were included. QOL evidence was published for 40% and 58% of FDA- and EMA-approved indications, respectively. QOL bonus criterion for ASCO-VF and ESMO-MCBS was met in 13% and 17% of FDA-approved and 21% and 24% of EMA-approved indications, respectively. Clinically meaningful improvements in QOL beyond minimal clinically important differences were noted in 6% and 11% of FDA- and EMA-approved indications, respectively. Availability of published QOL evidence at the time of approval increased over time for EMA (odds ratio [OR], 1.13; P = .03), however not for FDA (OR, 1.10; P = .12). Over time, no increase in awarded QOL bonuses or clinically meaningful improvements in QOL were found. Conclusions and Relevance: The findings of this systematic review suggest that approved systemic oncology therapies often do not have published evidence to suggest QOL improvement, despite its recognized importance. Of indications with evidence of statistical improvement, few have demonstrated clinically meaningful improvements.