Anchor-based Minimal Clinically Important Difference Research Articles

Minimal Important Clinical Difference Values Are Not Uniformly Valid in the Active Duty Military Population Recovering from Shoulder Surgery

There are multiple methods for calculating the minimal clinically important difference (MCID) threshold, and previous reports highlight heterogeneity and limitations of anchor-based and distribution-based analyses. The Warfighter Readiness Survey assesses the perception of a military population's fitness to deploy and may be used as a functional index in anchor-based MCID calculations. The purpose of the current study in a physically demanding population undergoing shoulder surgery was to compare the yields of two different anchor-based methods of calculating MCID for a battery of PROMs, a standard receiver operator curve (ROC) -based MCIDs and baseline-adjusted ROC MCIDs. All service members enrolled prospectively in a multicenter database with prior shoulder surgery that completed pre- and postoperative PROMs at a minimum of 12 months were included. The PROMs battery included Single Assessment Numeric Evaluation (SANE), American Shoulder and Elbow Surgeons score (ASES), Patient Reported Outcome Management Information System (PROMIS) Physical Function (PF), PROMIS Pain Interference (PI), and the Warfighter Readiness survey. Standard anchor-based and baseline-adjusted ROC MCIDs were employed to determine if the calculated MCIDs were both statistically and theoretically valid (95% confidence interval either completely negative or positive). There were 117 patients (136 operations) identified, comprised of 83% males with a mean age of 35.7 ± 10.4 years and 47% arthroscopic labral repair/capsulorrhaphy. Using the standard, anchor-based ROC MCID calculation, the area under the curve (AUC) for SANE, ASES, PROMIS PF, and PROMIS PI were greater than 0.5 (statistically valid). For ASES, PROMIS PF, and PROMIS PI, the calculated MCID 95% CI all crossed 0 (theoretically invalid). Using the baseline-adjusted ROC MCID calculation, the MCID estimates for SANE, ASES, and PROMIS PI were both statistically and theoretically valid if the baseline score was less than 70.5, 69, and 65.7. When MCIDs were calculated and anchored to the results of standard, anchor-based MCID, a standard ROC analysis did not yield statistically or theoretically valid results across a battery of PROMs commonly used to assess outcomes after shoulder surgery in the active duty military population. Conversely, a baseline-adjusted ROC method was more effective at discerning changes across a battery of PROMs among the same cohort.

Stratification of the Minimal Clinically Important Difference, Substantial Clinical Benefit, and Patient Acceptable Symptomatic State after Total Shoulder Arthroplasty by Implant Type, Preoperative Diagnosis, and Sex

Clinical significance, as opposed to statistical significance, has increasingly been utilized to evaluate outcomes after total shoulder arthroplasty (TSA). The purpose of this study was to identify thresholds of the minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptomatic state (PASS) for TSA outcome metrics and determine if these thresholds are influenced by prosthesis type (anatomic or reverse TSA), sex, or preoperative diagnosis. A prospectively collected international multicenter database inclusive of 38 surgeons was queried for patients receiving a primary aTSA or rTSA between 2003 and 2021. Prospectively, outcome metrics including ASES, shoulder function score (SFS), SST, UCLA, Constant, VAS Pain, shoulder arthroplasty smart (SAS) score, forward flexion, abduction, external rotation, and internal rotation was recorded preoperatively and at each follow-up. A patient satisfaction question was administered at each follow-up. Anchor-based MCID, SCB, and PASS were calculated as defined previously overall and according to implant type, preoperative diagnosis, and sex. The percentage of patients achieving thresholds was also quantified. A total of 5,851 total shoulder arthroplasties including aTSA (n=2,236) and rTSA (n=3,615) were included in the study cohort. The following were identified as MCID thresholds for the overall (aTSA + rTSA irrespective of diagnosis or sex) cohort: VAS Pain (-1.5), SFS (1.2), SST (2.1), Constant (7.2), ASES (13.9), UCLA (8.2), SPADI (-21.5), and SAS (7.3), Abduction (13°), Forward elevation (16°), External rotation (4°), Internal rotation score (0.2). SCB thresholds for the overall cohort were: VAS Pain (-3.3), SFS (2.9), SST 3.8), Constant (18.9), ASES (33.1), UCLA (12.3), SPADI (-44.7), and SAS (18.2), Abduction (30°), Forward elevation (31°), External rotation (12°), Internal rotation score (0.9). PASS thresholds for the overall cohort were: VAS Pain (0.8), SFS (7.3), SST (9.2), Constant (64.2), ASES (79.5), UCLA (29.5), SPADI (24.7), and SAS (72.5), Abduction (104°), Forward elevation (130°), External rotation (30°), Internal rotation score (3.2). MCID, SCB, and PASS thresholds varied depending on preoperative diagnosis and sex. MCID, SCB, and PASS thresholds vary depending on implant type, preoperative diagnosis, and sex. A comprehensive understanding of these differences as well as identification of clinically-relevant thresholds for legacy and novel metrics is essential to assist surgeons in evaluating their patient's outcomes, interpreting the literature, and counseling their patients preoperatively regarding expectations for improvement. Given that PASS thresholds are fragile and vary greatly depending on cohort variability, caution should be exercised in conflating them across different studies.

Validation of a Dyspnea Visual Analogue Scale in Fibrotic Interstitial Lung Disease.

A visual analogue scale (VAS) is a simple and easily administered tool for measuring impact of disease; however, little is known about the use of a Dyspnea VAS in interstitial lung disease (ILD). To validate the use of a Dyspnea VAS in a large and heterogenous cohort of patients with fibrotic ILD, including its minimal clinically important difference (MCID), responsiveness to change, and prognostic significance. Patients with fibrotic ILD were identified from a large prospective registry. Validity of a 100mm Dyspnea VAS was assessed by testing its correlation in change score with other measures of ILD severity, including the University of California San Diego Shortness of Breath Questionnaire (UCSDSOBQ), King's Brief Interstitial Lung Disease quality of life questionnaire Breathlessness and Activities Domain (KBILD-B), European Quality of Life visual analogue scale (EQ-VAS), forced vital capacity (FVC), and diffusion capacity of the lung for carbon monoxide (DLCO). Responsiveness of the Dyspnea VAS was qualitatively confirmed based on there being an observable difference in the change in Dyspnea VAS across tertiles of change in anchor variables. The minimum clinically important difference (MCID) in Dyspnea VAS was calculated using both anchor (linear regression) and distribution (one-half standard deviation) approaches, with anchors including the above variables that had a correlation with Dyspnea VAS (|r|0.30). The association of Dyspnea VAS with time to death or transplant was determined. The cohort included 826 patients with fibrotic ILD, including 127 patients with follow-up measurements at 6 months. Mean baseline Dyspnea VAS was 5324mm. Dyspnea VAS change scores were moderately correlated with UCSDSOBQ (|r|=0.55) and KBILD-B (|r|=0.44), and weakly correlated with EQ-VAS (|r|=0.19), FVC% (|r|=0.21) and DLCO% (|r|=0.05). MCID was 2.7 to 4.5 using the more reliable anchor-based methods and 12.0 based on distribution-based methods. Dyspnea VAS was associated with time to death or transplant in unadjusted models and after adjustment for age and sex (hazard ratio 1.16 and 1.15 respectively, p<0.05 for both). This study provides support for the use of Dyspnea VAS in patients with fibrotic ILD, with an estimated anchor-based MCID of 5mm.

Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

BackgroundThe construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) questionnaire were evaluated for patients with late-onset Pompe disease (LOPD), a rare, autosomal recessive, progressive neuromuscular disorder treatable by enzyme replacement therapy (ERT).MethodsIn the phase 3 PROPEL study, adults with LOPD underwent testing of physical functioning and had PRO measurements at baseline and at weeks 12, 26, 38, and 52 while receiving experimental or standard-of-care ERT. All patients were pooled for analyses, without comparisons between treatment groups. Associations and correlations between PROMIS PF20a scores and the 6-minute walk distance (6MWD), % predicted forced vital capacity (FVC), manual muscle test (MMT) of the lower extremities, Gait, Stairs, Gowers’ maneuver, Chair (GSGC) score, and Rasch-built Pompe-specific Activity (R-PAct) scale were evaluated by calculating regression coefficients in linear regression models and Pearson correlation coefficients (R); patients’ age, sex, race, ERT prior to study, body mass index, and study treatment were included as covariables. The minimal clinically important difference (MCID) of PROMIS PF20a was determined using distribution- and anchor-based methods.Results123 patients received at least 1 dose of ERT. In multivariable analyses, PROMIS PF20a scores had strong correlations with R-PAct scores (R = 0.83 at baseline and R = 0.67 when evaluating changes between baseline and 52 weeks) and moderate correlations with the 6MWD (R = 0.57 at baseline and R = 0.48 when evaluating changes between baseline and 52 weeks). Moderate correlations were also observed between PROMIS PF20a and MMT (R = 0.54), GSGC (R=-0.51), and FVC (R = 0.48) at baseline. In multivariable linear regression models, associations were significant between PROMIS PF20a and 6MWD (P = 0.0006), MMT (P = 0.0034), GSGC (P = 0.0278), and R-PAct (P < 0.0001) at baseline, between PROMIS PF20a and 6MWD (P < 0.0001), FVC (P = 0.0490), and R-PAct (P < 0.0001) when combining all measurements, and between PF20a and 6MWD (P = 0.0016) and R-PAct (P = 0.0001) when evaluating changes in scores between baseline and 52 weeks. The anchor-based and distribution-based MCID for a clinically important improvement for PROMIS PF20a were 2.4 and 4.2, respectively.ConclusionsPROMIS PF20a has validity as an instrument both to measure and to longitudinally follow physical function in patients with LOPD.Trial registrationClinicalTrials.gov, NCT03729362. Registered 2 November 2018, https://www.clinicaltrials.gov/search?term=NCT03729362.

3 Ethnoracial Differences in Anchor Agreement and MCID Estimation in Alzheimer’s Disease

Objective:Alzheimer’s disease (AD) clinical trials lack diverse representation, limiting their generalizability. In addition, the clinical meaningfulness of observed changes during treatment may vary as a function of participant characteristics. Defining meaningful change in AD within diverse ethnoracial groups is therefore greatly needed. Meaningful change in AD trials can be assessed by three different anchors: participants, informants, or clinicians. Previous research has suggested that estimations of the minimal clinically important difference (MCID) vary by disease severity, choice of anchor, and anchor agreement. These relationships have been studied primarily within non-Hispanic white (NHW) samples. This project evaluates anchor-based MCID within and across the three most prevalent ethnoracial groups in the United States, non-Hispanic White (NHW), Hispanic/Latino (H/L), and Black/African-American (B/AA).Participants and Methods:Data from the National Alzheimer’s Coordinating Center Uniform Dataset (NACC UDS) were used to investigate MCID within older adults (ages 50+) diagnosed as cognitively normal or cognitively impaired due to suspected AD. Data were taken from all versions of the UDS and consisted of all available participants with two consecutive annual visits. The identified sample (N=22,043) is approximately 83.6% NHW, 4.7% H/L, and 11.7% B/AA. Participant, informant, and clinician anchor variables were utilized to compare proportions of anchor agreement within and across ethnoracial groups. MCID on the Mini-Mental State Exam (MMSE) was estimated within each ethnoracial group and compared across the independent variables of anchor agreement and disease severity (cognitively normal (CN), mild cognitive impairment (MCI), and dementia) in 2x3 ANOVAs.Results:Participant age (M = 71.56, SD = 9.03) did not significantly differ across ethnoracial groups; years of education significantly differed across groups, p &lt; .001, with NHW (M=15.83 SD=3.05), H/L (M=12.49, SD=5.01), and B/AA (M=14.42, SD=3.22). Across all three anchors (participant, informant, clinician), unanimous agreement about the presence or absence of a decline in functioning was present in about 75.1% of the full sample. To further explore agreement differences across groups, anchor agreement was classified into a 3-level variable: 1) agreement that the participant remained stable over time, 2) agreement that the participant declined, and 3) disagreement. The proportion of each level within each ethnoracial group was significantly different, (x2(4, n = 22,043) = 179.16, p &lt; .001, phi = .09, NHW (34.5% agreement-stable, 41.4% agreement-declined, 24.1% disagreement), H/L (30.5%, 42.6%, 26.9%, respectively), and B/AA (42.2%, 28.1%, 29.7%, respectively). MCID estimates on the MMSE followed similar trends within each ethnoracial group. There was a significant main effect of disease severity, such that MCID estimates increased in magnitude with increasing disease severity. There were no significant main effects of anchor agreement for any ethnoracial group. Within the NHW sample only, an interaction effect between diagnostic severity and anchor agreement was significant (p = .007).Conclusions:Across ethnoracial groups, MCID estimates on the MMSE are reliably influenced by the severity of disease. However, the benefit of anchor-based MCID estimates may vary by ethnoracial group with respect to both anchor choice and use of anchor agreement. The origins of anchor disagreement and perceived stability in functioning warrant further exploration.

Quantifying success after first revision reverse total shoulder arthroplasty: the minimal clinically important difference, substantial clinical benefit, and patient acceptable symptomatic state.

When patients require revision of primary shoulder arthroplasty, revision reverse total shoulder arthroplasty (rTSA) is most commonly performed. However, defining clinically important improvement in these patients is challenging because benchmarks have not been previously defined. Our purpose was to define the minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptomatic state (PASS) for outcome scores and range of motion (ROM) after revision rTSA and to quantify the proportion of patients achieving clinically relevant success. This retrospective cohort study used a prospectively collected single-institution database of patients undergoing first revision rTSA between August 2015 and December 2019. Patients with a diagnosis of periprosthetic fracture or infection were excluded. Outcomes scores included the ASES, raw and normalized Constant, SPADI, SST, and University of California, Los Angeles (UCLA) scores. ROM measures included abduction, forward elevation (FE), external rotation (ER), and internal rotation (IR) score. Anchor-based and distribution-based methods were used to calculate the MCID, SCB, and PASS. The proportions of patients achieving each threshold were assessed. Ninety-three revision rTSAs with minimum 2-year follow-up were evaluated. Mean age was 67 years, 56% were female, and average follow-up was 54 months. Revision rTSA was performed most commonly for failed anatomic TSA (n=47), followed by hemiarthroplasty (n=21), rTSA (n=15), and resurfacing (n=10). The indication for revision rTSA was most commonly glenoid loosening (n=24), followed by rotator cuff failure (n=23), subluxation and unexplained pain (n=11 for both). The anchor-based MCID thresholds (% of patients achieving) were as follows: ASES,20.1 (42%); normalized Constant,12.6 (80%); UCLA,10.2 (54%); SST,0.9 (78%); SPADI,-18.4 (58%); abduction,13° (83%); FE,18° (82%); ER,4° (49%); and IR,0.8 (34%). The SCB thresholds (% of patients achieving) were as follows: ASES,34.1 (25%); normalized Constant,26.6 (43%); UCLA,14.1 (28%); SST,3.9 (48%); SPADI,-36.4 (33%); abduction,20° (77%); FE,28° (71%); ER,15° (15%); and IR,1.0 (29%). The PASS thresholds (% of patients achieving) were as follows: ASES,63.5 (53%); normalized Constant,59.1 (61%); UCLA,25.4 (48%); SST,7.0 (55%); SPADI,42.4 (59%); abduction,98° (61%); FE,110° (56%); ER,19° (73%); and IR,3.3 (59%). This study establishes thresholds for the MCID, SCB, and PASS at minimum 2-years after revision rTSA, providing physicians an evidence-based method to counsel patients and assess patient outcomes postoperatively.

Assessment of Thresholds for Clinically Relevant Change in the Pediatric/Adolescent Shoulder Survey After Shoulder Instability Surgery: Factors Associated With Meaningful Improvement in Outcomes.

The pediatric/adolescent shoulder survey (PASS) score is a subjective measure of shoulder symptomology in younger patients. To establish the minimal clinically important difference (MCID) and minimal detectable change (MDC) for the PASS score in adolescents after surgical treatment for shoulder instability. Case series; Level of evidence, 4. Included were patients aged 12.5 to 23 years who underwent surgical treatment for shoulder instability and who had completed PASS forms preoperatively and at 3 months postoperatively. The MCID was established using an anchor-based approach, with the Single Assessment Numeric Evaluation (SANE) and shortened version of the Disabilities of the Arm, Shoulder and Hand (QuickDASH) as anchors. Change in PASS score between anchor groups was determined using receiver operating characteristic curve analysis. MDC with 90% confidence (MDC90) was also calculated. Range of motion and strength data at 3-month follow-up were evaluated to identify the optimal postoperative PASS score. Factors associated with improvement in PASS score beyond the MDC90 and MCID were determined in a subset of patients with ≥6-month follow-up data. A total of 95 patients were included. The mean PASS score improved significantly from preoperatively to postoperatively (57 ± 15 to 75 ± 16; P < .001). The anchor-based MCID ranged from 12.5 to 13.2 points, with an area under the receiver operating characteristic (AUC) curve of 0.87 for the SANE and 0.99 for the QuickDASH. The MDC90 was 16.5 points. The optimal PASS score at 3 months after surgery was ≥85 (AUC, 0.66). Shorter duration of symptoms, lower preoperative forward elevation, and higher preoperative external rotation were associated with improvement in PASS score above the MDC90 and/or MCID for the subset of patients (n = 25) with ≥6-month follow-up data. Increased number of suture anchors, less preoperative external rotation deficit, and number of previous dislocations had a moderate effect on improvement in outcomes. A postoperative increase in PASS score of ≥16.5 points had a 90% chance of being a true-positive change, while a score change of approximately 13 points was likely clinically relevant. The optimal PASS score after surgery was ≥85. Shorter duration of symptoms, preoperative range of motion, number of surgical anchors, and number of previous dislocations were associated with achieving a clinically relevant improvement in PASS score at minimum 6 months postoperatively.

Pearls and pitfalls of PROMIS clinically significant outcomes in orthopaedic surgery.

Patient-Reported Outcome Measurement Information System (PROMIS) was developed as a uniform and generalizable PROM system using item response theory and computer adaptive testing. We aimed to assess the utilization of PROMIS for clinically significant outcomes (CSOs) measurements and provide insights into its use in orthopaedic research. We reviewed PROMIS CSO reports for orthopaedic procedures via PubMed, Cochrane Library, Embase, CINAHL, and Web of Science from inception to 2022, excluding abstracts and missing measurements. Bias was assessed using the Newcastle-Ottawa Scale (NOS) and questionnaire compliance. PROMIS domains, CSO measures, and study populations were described. A meta-analysis compared distribution and anchor-based MCIDs in low-bias (NOS ≥ 7) studies. Overall, 54 publications from 2016 to 2022 were reviewed. PROMIS CSO studies were observational with increasing publication rates. Evidence-level was II in 10/54, bias low in 51/54, and compliance ≥ 86% in 46/54. Most (28/54) analysed lower extremity procedures. PROMIS domains examined Pain Function (PF) in 44/54, Pain Interference (PI) in 36/54, and Depression (D) in 18/54. Minimal clinically important difference (MCID) was reported in 51/54 and calculated based on distribution in 39/51 and anchor in 29/51. Patient acceptable symptom state (PASS), substantial clinical benefit (SCB), and minimal detectable change (MDC) were reported in ≤ 10/54. MCIDs were not significantly greater than MDCs. Anchor-based MCIDs were greater than distribution based MCIDs (standardized mean difference = 0.44, p < 0.001). PROMIS CSOs are increasingly utilized, especially for lower extremity procedures assessing the PF, PI, and D domains using distribution-based MCID. Using more conservative anchor-based MCIDs and reporting MDCs may strengthen results. Researchers should consider unique pearls and pitfalls when assessing PROMIS CSOs.

Defining Clinical Meaningfulness in Huntington's Disease.

Minimal clinically important difference (MCID) represents the smallest within-person change on an outcome measure considered meaningful to the patient. Anchor-based MCID methods evaluate the relationship between changes in an outcome measure and the patient-reported clinical importance of that change. This study aims to estimate longitudinal MCID for clinically relevant outcome measures for individuals who have Stages 2 or 3 disease as measured by the Huntington's Disease Integrated Staging System (HD-ISS). Data were drawn from Enroll-HD, a large global longitudinal, observational study and clinical research platform for HD family members. We analyzed HD participants (N = 11,070) by staging group using time frames ranging from 12 to 36 months. The anchor was the physical component summary score of the 12-item short-form health survey. HD-relevant motor, cognitive, and functional outcome measures were independent, external criterion outcomes. Complex analysis was conducted using multiple, independent, linear mixed effect regression models with decomposition to calculate MCID for each external criterion by group. MCID estimates varied by progression stage. MCID estimates increased as stage progression increased and as the time frame increased. MCID values for key HD measures are provided. For example, starting in HD-ISS stage 2, meaningful group change over 24 months equals an average increase of 3.6 or more points on the Unified Huntington's Disease Rating Scale Total Motor Score. This is the first study to examine MCID estimation thresholds for HD. The results can be used to improve clinical interpretation of study outcomes and enable treatment recommendations to support clinical decision-making and clinical trial methodology. © 2023 International Parkinson and Movement Disorder Society.

129Xe MRI Ventilation Defects in Asthma: What is the Upper Limit of Normal and Minimal Clinically Important Difference?

The minimal clinically important difference (MCID) and upper limit of normal (ULN) for MRI ventilation defect percent (VDP) were previously reported for hyperpolarized 3He gas MRI. Hyperpolarized 129Xe VDP is more sensitive to airway dysfunction than 3He, therefore the objective of this study was to determine the ULN and MCID for 129Xe MRI VDP in healthy and asthma participants. We retrospectively evaluated healthy and asthma participants who underwent spirometry and 129XeMRI on a single visit; participants with asthma completed the asthma control questionnaire (ACQ-7). The MCID was estimated using distribution- (smallest detectable difference [SDD]) and anchor-based (ACQ-7) methods. Two observers measured VDP (semiautomated k-means-cluster segmentation algorithm) in 10 participants with asthma, five-times each in random order, to determine SDD. The ULN was estimated based on the 95% confidence interval of the relationships between VDP and age. Mean VDP was 1.6 ± 1.2% for healthy (n=27) and 13.7 ± 12.9% for asthma participants (n=55). ACQ-7 and VDP were correlated (r=.37, p=.006; VDP=3.5·ACQ+4.9). The anchor-based MCID was 1.75% while the mean SDD and distribution-based MCID was 2.25%. VDP was correlated with age for healthy participants (p=.56, p =.003; VDP=.04·Age-.01). The ULN for all healthy participants was 2.0%. By age tertiles, the ULN was 1.3% ages 18-39 years, 2.5% for 40-59 years and 3.8% for 60-79 years. The 129Xe MRI VDP MCID was estimated in participants with asthma; the ULN was estimated in healthy participants across a range of ages, both of which provide a way to interpret VDP measurements in clinical investigations.

Minimal clinically important difference (MCID) and substantial clinical benefit (SCB) of upper extremity PROMIS scores following arthroscopic rotator cuff repairs.

To calculate and determine what factors are associated with achieving the Minimal Clinically Important Difference (MCID) and the Substantial Clinical Benefit (SCB) of Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity Computer Adaptive Testing v2.0 (UE), Pain Interference (P-Interference), and Pain Intensity (P-Intensity) in patients undergoing arthroscopic rotator cuff repair (aRCR). The change in PROMIS scores representing the optimal cutoff for a ROC curve with an area under the curve analysis was used to calculate the anchor-based MCID and SCB. To assess the responsiveness of each PROM, effect sizes and standardized response means (SRM) were calculated. To identify factors associated with attaining the MCID and SCB, univariate and multivariate logistic regression analyses were performed. A total of 323 patients with an average age of 59.9 ± 9.5 were enrolled in this study, of which, 187/323 [57.9%] were male and 136/323 [42.1%] were female. The anchor-based MCID for PROMIS UE, P-Interference, and P-Intensity was: 9.0, 7.5, and 11.2, respectively. The respective SCB was 10.9, 9.3, and 12.7. Effect size and SRM were: PROMIS UE (1.4, 1.3), P-Interference (1.8, 1.5), and P-Intensity (2.3, 2.0). Lower preoperative P-Intensity scores (p = 0.02), dominant arm involvement (p = 0.03), and concomitant biceps tenodesis (p = 0.03) were associated with patients achieving the SCB for PROMIS UE. A large responsiveness for each of the PROMIS instruments due to the majority of patients reporting great improvement after aRCR and a small standard deviation across all outcome measures was shown in our study. Lower preoperative P-Intensity scores and concomitant biceps tenodesis were associated with higher odds of achieving the SCB for PROMIS UE. The knowledge of MCID and SCB values for PROMIS instruments will allow the surgeon to determine whether the improvements in the PROMIS scores after aRCR are clinically meaningful. Level III.

Minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptomatic state (PASS) of upper extremity PROMIS scores in idiopathic adhesive capsulitis.

The purpose of this study is to calculate the minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptom state (PASS) of Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity Computer Adaptive Testing v2.0 (UE), Pain Interference (P-Interference), and Pain Intensity (P-Intensity) in patients treated nonoperatively for idiopathic adhesive capsulitis (IAC). The anchor-based MCID, SCB, and PASS were calculated as the change in PROMIS scores representing the optimal cutoff for a ROC curve with an area under the curve (AUC) analysis. The distribution-based MCID was calculated as a range between the average standard error of measurement multiplied by two different constants: 1 and 2.77. Effect sizes and standardized response means (SRM) were calculated to assess the responsiveness of each PROMIS instrument while regression analyses were performed to identify factors associated with achieving these thresholds. This study enrolled 115 patients. The anchor-based MCID for PROMIS UE, P-Interference, and P-Intensity was 5.11, 4.16, and 8.16, respectively. The respective SCB was 8.44, 6.65, and 10.05. The respective PASS was 8.47, 7.01, and 10.41. The odds of achieving MCID values in adhesive capsulitis were negatively affected by gender (male), higher forward elevation at the time of presentation, higher pain scores (P-Interference), need for ≥ 2 corticosteroid injections, and a concomitant diagnosis of diabetes. The MCID, SCB, and PASS parameters for PROMIS scores can be utilized to determine the clinical meaningfulness of patient-reported improvements in these instruments during the nonoperative treatment and as a research tool to compare the efficacy of new treatments for adhesive capsulitis. Level III, basic science study, validation of outcome instruments. Key points • This is the first study to calculate the minimal clinically important difference (MCID), substantial clinical benefit (SCB), and patient acceptable symptomatic state (PASS) for the Patient-Reported Outcome Measurement Information System (PROMIS) Upper Extremity and Pain instruments in patients with idiopathic adhesive capsulitis (IAC) of the shoulder.• This study determined the impact of symptom severity, demographics, and comorbidities on achieving the MCID, SCB, and PASS for PROMIS instruments in IAC patients treated nonoperatively.

Functional Outcomes of a Musculoskeletal Integrated Practice Providing Comprehensive Whole Person Care for Hip Osteoarthritis.

Outcomes of hip osteoarthritis (OA) management within integrated practice units (IPUs) are lacking. This study reports 6-month and 1-year patient-reported outcomes (PROs) of IPU care, the proportion of patients achieving minimal clinically important difference (MCID) and substantial clinical benefit (SCB) at 1 year, and baseline factors associated with the likelihood of achieving MCID and SCB. We retrospectively evaluated 1009 new patients presenting to an IPU with hip OA between October 2017 and June 2020. Patients experienced multidisciplinary team-based management. Individuals with baseline and 6-month PROs or baseline and 1-year PROs (Hip Disability and Osteoarthritis Outcome Score Joint Replacement, HOOS JR) were included. We used anchor-based MCID and SCB thresholds and multivariable binary logistic regression models to identify baseline factors associated with achieving 1-year MCID and SCB. HOOS JR increased from baseline to 6 months (Δ= 19.1±2.1, P= .065) and baseline to 1 year (Δ= 35.8±2.9, P < .001). At 1 year, 72.7% (IPU only) and 88% (IPU-based total hip arthroplasty [THA]) achieved MCID (P < .001), and 62.3% (IPU only) and 88% (IPU-based THA) achieved SCB (P < .001). In multivariable regression, lower baseline HOOS JR scores (r= 0.96, P= .04), undergoing THA (r= 0.213, P < .001), and fewer symptoms of generalized anxiety (r= 0.932, P= .018) were independently associated with achieving MCID at 1 year. The same factors were independently associated with achieving SCB at 1 year. Lower baseline anxiety (Generalized Anxiety Disorder Questionnaire-7 item) and greater hip-related preoperative limitations result in greater likelihood of achieving MCID and SCB. Significant improvements in patient outcomes can be achieved by IPUs providing comprehensive care for hip OA including the management of psychological distress. Future prospective studies should compare the outcomes of IPUs with traditional care in managing diverse patient phenotypes.

Risk factors for failure to achieve minimal clinically important difference and significant clinical benefit in PROMIS computer adaptive test domains in patients undergoing rotator cuff repair.

The Patient-Reported Outcomes Measurement Information System (PROMIS) has emerged as a valid and efficient means of collecting outcomes in patients with rotator cuff tears. The purpose of this study was to establish threshold score changes to determine minimal clinically important difference (MCID) and substantial clinical benefit (SCB) in PROMIS computer adaptive test (CAT) scores following rotator cuff repair (RCR). Additionally, we sought to identify potential risk factors for failing to achieve MCID and SCB. Patients undergoing arthroscopic RCR were identified over a 24-month period. Only patients who completed both preoperative and postoperative PROMIS CAT assessments were included in this cohort. PROMIS CAT forms for upper extremity physical function (PROMIS-UE), pain interference (PROMIS-PI), and depression (PROMIS-D) were used with a minimum of 1.5-year follow-up. Statistical analysis was performed to determine threshold score changes to determine anchor-based MCID and SCB, as well as risk factors for failure to achieve significant clinical improvement following surgery. Of 198 eligible patients, 168 (84.8%) were included in analysis. ΔPROMIS-UE values of 5.8 and 9.7 (area under the curve [AUC]=0.906 and 0.949, respectively) and ΔPROMIS-PI values of -11.4 and -12.9 (AUC=0.875 and 0.938, respectively) were identified as threshold predictors of MCID and SCB achievement. On average, 81%, 65%, and 55% of patients achieved MCID for PROMIS-UE, PROMIS-PI, and PROMIS-D whereas 71%, 61%, and 38% of patients in the cohort, respectively, achieved SCB. MCID achievement in PROMIS-UE significantly differed according to risk factors, including smoking status (likelihood ratio [LR]: 9.8, P=.037), tear size (LR: 10.4, P<.001), distal clavicle excision (LR: 6.1, P=.005), and prior shoulder surgery (LR: 19.2, P<.001). Factors influencing SCB achievement for PROMIS-UE were smoking status (LR: 9.3, P=.022), tear size (LR: 8.0, P=.039), and prior shoulder surgery (11.9, P<.001). Significantly different rates of MCID and SCB achievement in PROMIS-PI for smoking status (LR: 7.0, P=.030, and LR: 5.2, P=.045) and prior shoulder surgery (LR: 9.1, P=.002, and LR: 7.4, P=.006) were also identified. The majority of patients showed clinically significant improvements that exceeded the established MCID for PROMIS-UE and PROMIS-PI following RCR. Patients with larger tear sizes, a history of prior shoulder surgery, tobacco users, and those who received concomitant distal clavicle excision were at risk for failing to achieve MCID in PROMIS-UE. Additionally, smokers and patients who underwent prior shoulder surgery demonstrated significantly lower improvements in pain scores following surgery.

Occupational therapy for cognitive impairment in stroke patients.

Occupational therapy for cognitive impairment in stroke patients.

Outcomes Vary Significantly Using a Tiered Approach to Define Success After Total Knee Arthroplasty

BackgroundPatient-reported outcome measures (PROM) allow assessment of clinical outcomes following primary total knee arthroplasty (TKA). The purpose of this study is to use progressively more stringent definitions of success to examine clinical outcomes of primary TKA at 1 year postoperatively and to determine which demographic variables were associated with achieving clinical success. MethodsThe American Joint Replacement Registry was queried from 2012 to 2020 for primary TKA. Patients who completed the following PROMs preoperatively and 1 year postoperatively were included: Western Ontario and McMaster Universities Arthritis Index (WOMAC), Knee Injury and Osteoarthritis Outcome Score (KOOS), and KOOS for Joint Replacement (KOOS JR). Mean PROM scores were determined for each visit and between-visit changes were evaluated using paired t-tests. Rates of achievement of minimal clinically important difference (MCID) by distribution-based and anchor-based criteria, Patient Acceptable Symptom State, and substantial clinical benefit were calculated. Logistic regression was used to evaluate the association between demographic variables and odds of clinical success. ResultsIn total, 12,341 TKAs were included. Mean improvement in PROM scores were as follows: KOOS JR, 29; WOMAC-Pain, 33; and WOMAC-Function, 31 (P < .0001 for all). Rates of achievement of each metric were the following: distribution-based MCID, 84%-87%; anchor-based MCID, 46%-79%; Patient Acceptable Symptom State, 54%-82%; and substantial clinical benefit, 68%-81%. Patient age and gender were the most influential demographic factors on achievement of clinical success. ConclusionClinical outcomes at 1 year following TKA vary significantly when using a tiered approach to define success. A tiered approach to interpretation of PROMs should be considered for future research and clinical assessment.

Minimal Clinically Important Difference for PROMIS Physical Function and Pain Interference in Patients Following Surgical Treatment of Distal Radius Fracture

We estimated the minimal clinically important difference (MCID) for the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) and Pain Interference (PI) computer adaptive tests (CATs) following surgical treatment of distal radius fracture (DRF). Adult patients surgically treated between November 2017 and November 2020 for isolated DRF were identified. Demographic and patient-reported outcome data were extracted from the electronic health record. Outcomes of interest were the PROMIS PF and PI CATs. Inclusion criteria were met if: (1) PROMIS PF and PI scores were available at preoperative and postoperative visits; and (2) a postoperative clinical anchor question asking about overall response to treatment was answered. An anchor-based MCID estimate was determined by calculating the average absolute score change in PROMIS PF and PI for patients who indicated a mild change to the anchor question. A distribution-based MCID estimate was also calculated using the standard error of measurement and effect sizes of change. The changes in PROMIS PF and PI scores were significantly different between patients who gave responses of much change (n= 73), mild change (n= 51), and no change (n= 19) to the clinical anchor question. The average score changes in the mild change group for PROMIS PF and PI were 5.2 (SD, 3.7) and 6.8 (SD, 4.3) points, respectively, representing the anchor-based MCID estimates. The PROMIS PI anchor-based estimate was moderately correlated with the preoperative score (r=-0.41), time between visits (r=-0.39), and age (r= 0.30). The distribution-based MCID estimates were 3.8 (SD, 1.3) and 3.7 (SD, 1.3) points for the PROMIS PF and PI, respectively. The MCIDs were estimated as 5.2 and 6.8 for the PROMIS PF and PI CATs, respectively, following surgery for DRF. As reports continue to publish a consistent range of MCID values, researchers can be confident in these values and begin using them across a broader spectrum of conditions treated by hand surgeons.

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

BackgroundThe 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). MethodsPatients 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. Results52.8% 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. ConclusionOur 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.

Establishing the Minimal Clinically Important Difference for the PROMIS Upper Extremity Computer Adaptive Test Version 2.0 in a Nonshoulder Hand and Upper Extremity Population

Our primary purpose was to calculate the minimal clinically important difference (MCID) for the Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity (UE) Computer Adaptive Test (CAT) version 2.0 (v2.0) for a nonshoulder hand and upper extremity population. Secondarily, we calculated the PROMIS Physical Function (PF) CAT v2.0 and the abbreviated version of the Disabilities of the Arm, Shoulder, and Hand (QuickDASH) MCID. Adult patients treated by 1 of 5 fellowship-trained hand surgeons between March 2015 and September 2019 at an academic tertiary institution were identified. The PROMIS UE CAT v2.0, PROMIS PF CAT v2.0, and QuickDASH were collected via tablet computer. Inclusion required response to at least 1 of the instruments at both baseline and follow-up (6 ± 4 weeks), and a response to the anchor question: "Compared to your first evaluation at the University Orthopaedic Center, how would you describe your physical function level now?" An additional anchor question assessing treatment-related improvement was also asked. The MCID was calculated using an anchor-based approach using the mean change difference between groups reporting no change and slight change for both anchor questions, and with the 1/2 SD method. Of 2,106 participants, mean age was 48 ± 17 years, 53% were female, and 53% were recovering from surgery. Of these patients, 381 completed the PROMISE UE CAT v2.0, 497 completed the PROMIS PF CAT v2.0, and 2,018 completed the QuickDASH. The score change between baseline and follow-up was significantly different between anchor groups for both anchor-based MCID calculations. Anchor-based MCID values were 3.0 to 4.0 for the UE CAT, 2.1 to 3.6 for the PF CAT, and 10.3 for the QuickDASH. The MCID values per the 1/2 SD method were 4.1, 4.1, and 10.2, respectively. We propose MCID ranges of 3.0 to 4.1 for the PROMIS UE CAT v2.0, and 2.1 to 4.1 for the PROMIS PF CAT v2.0. The observed QuickDASH MCID values (10.2-10.3) are within the range of previously published values. These MCID estimates will aid in interpreting clinical outcomes and in powering clinical studies.

What constitutes a clinically important change in Mayo Elbow Performance Index and range of movement after open elbow arthrolysis?

AimsThis study aimed to determine the minimal detectable change (MDC), minimal clinically important difference (MCID), and substantial clinical benefit (SCB) under distribution- and anchor-based methods for the Mayo Elbow Performance Index (MEPI) and range of movement (ROM) after open elbow arthrolysis (OEA). We also assessed the proportion of patients who achieved MCID and SCB; and identified the factors associated with achieving MCID.MethodsA cohort of 265 patients treated by OEA were included. The MEPI and ROM were evaluated at baseline and at two-year follow-up. Distribution-based MDC was calculated with confidence intervals (CIs) reflecting 80% (MDC 80), 90% (MDC 90), and 95% (MDC 95) certainty, and MCID with changes from baseline to follow-up. Anchor-based MCID (anchored to somewhat satisfied) and SCB (very satisfied) were calculated using a five-level Likert satisfaction scale. Multivariate logistic regression of factors affecting MCID achievement was performed.ResultsThe MDC increased substantially based on selected CIs (MDC 80, MDC 90, and MDC 95), ranging from 5.0 to 7.6 points for the MEPI, and from 8.2° to 12.5° for ROM. The MCID of the MEPI were 8.3 points under distribution-based and 12.2 points under anchor-based methods; distribution- and anchor-based MCID of ROM were 14.1° and 25.0°. The SCB of the MEPI and ROM were 17.3 points and 43.4°, respectively. The proportion of the patients who attained anchor-based MCID for the MEPI and ROM were 74.0% and 94.7%, respectively; furthermore, 64.2% and 86.8% attained SCB. Non-dominant arm (p = 0.022), higher preoperative MEPI rating (p < 0.001), and postoperative visual analogue scale pain score (p < 0.001) were independent predictors of not achieving MCID for the MEPI, while atraumatic causes (p = 0.040) and higher preoperative ROM (p = 0.005) were independent risk factors for ROM.ConclusionIn patients undergoing OEA, the MCID for the increased MEPI is 12.2 points and 25° increased ROM. The SCB is 17.3 points and 43.3°, respectively. Future studies using the MEPI and ROM to assess OEA outcomes should report not only statistical significance but also clinical importance.Cite this article: Bone Joint J 2021;103-B(2):366–372.