Distribution-based Methods Research Articles

Quality of Life Effect of the Anti-CCR4 Monoclonal Antibody Mogamulizumab Versus Vorinostat in Patients With Cutaneous T-cell Lymphoma

BackgroundSézary syndrome (SS) and mycosis fungoides (MF), 2 types of cutaneous T-cell lymphoma, cause significant morbidity and adversely affect patients’ quality of life (QoL). The present study assessed the QoL measurement changes in patients receiving mogamulizumab versus vorinostat. Patients and MethodsA multicenter phase III trial was conducted of patients with stage IB-IV MF/SS with ≥ 1 failed systemic therapy. The QoL measures included Skindex-29 and the Functional Assessment of Cancer Therapy–General. The symptoms, function, and QoL subdomains were longitudinally modeled using mixed models with prespecified covariates. Meaningful change thresholds (MCTs) were defined using distribution-based methods. The categorical changes by group over time and the time to clinically meaningful worsening were analyzed. ResultsOf the 372 randomized patients, mogamulizumab demonstrated improvement in Skindex-29 symptoms (cycles 3, 5, and 7; P < .05) and functional (cycles 3 and 5; P < .05) scales. A significantly greater proportion of mogamulizumab-treated patients improved by MCTs or more from baseline in the Skindex-29 symptoms domain (cycles 3, 5, 7, and 11) and functioning domain (cycle 5). Significant differences in the Functional Assessment of Cancer Therapy–General physical well-being (cycles 1, 3, and 5; P < .05) were observed in favor of mogamulizumab and a greater proportion of patients had declined by MCTs or more at cycles 1, 3, 5, and 7 with vorinostat treatment. The median time to symptom worsening using Skindex-29 was 27.4 months for mogamulizumab versus 6.6 months for vorinostat. In the patients with SS, the time to worsening favored mogamulizumab (P < .005) for all Skindex-29 domains. The time to worsening was similar for the 2 MF treatment arms. ConclusionThe symptoms, function, and overall QoL of patients with MF/SS favored mogamulizumab over vorinostat across all time points. Patients with the greatest symptom burden and functional impairment derived the most QoL benefit from mogamulizumab.

Minimal important difference and patient acceptable symptom state for PFDI-20 and POPDI-6 in POP surgery

Introduction and hypothesisPatient-reported outcome measures are fundamental tools when assessing effectiveness of treatments. The challenge lies in the interpretation: which magnitude of change in score is meaningful for the patients? The minimal important difference (MID) is defined as the smallest difference in score that patients perceive as important. The Patient Acceptable Symptom State (PASS) represents the value of score beyond which patients consider themselves well. We aimed to determine the MID and PASS for Pelvic Floor Distress Inventory-20 (PFDI-20) and Pelvic Organ Prolapse Distress Inventory-6 (POPDI-6) in pelvic organ prolapse (POP) surgery.MethodsWe used data from 2704 POP surgeries from a prospective, population-based cohort. MID was determined with three anchor-based and one distribution-based method. PASS was defined using two different methods. Medians of the estimates were identified.ResultsThe MID estimates with (1) mean change, (2) receiver-operating characteristic (ROC) curve, (3) 75th percentile, and (4) distribution-based method varied between 22.9–25.0 (median 24.2) points for PFDI-20 and 9.0–12.5 (median 11.3) for POPDI-6. The PASS cutoffs with (1) 75th percentile and (2) ROC curve method varied between 57.7–62.5 (median 60.0) for PFDI-20 and 16.7–17.7 (median 17.2) for POPDI-6.ConclusionA mean difference of 24 points in the PFDI-20 or 11 points in the POPDI-6 can be used as a clinically relevant difference between groups. Postoperative scores ≤ 60 for PFDI-20 and ≤ 17 for POPDI-6 signify acceptable symptom state.

260. Preoperative medical comorbidities do not affect minimum clinically important difference (MCID) for lumbar fusion in grade one spondylolisthesis

BACKGROUND CONTEXT Medical comorbidities, particularly anxiety or depression, may potentially influence how patients perceive clinical benefit after surgery. The current study defined and compared minimum clinically important difference (MCID) thresholds in patients with and without anxiety/depression and obesity undergoing fusion for grade 1 degenerative spondylolisthesis. PURPOSE To determine if preoperative psychiatric diagnoses, more than obesity, alters the MCID threshold in patients undergoing fusion for spondylolisthesis. STUDY DESIGN/SETTING Retrospective analysis of multicenter, prospective database. PATIENT SAMPLE Patients who underwent lumbar fusion for grade 1 degenerative spondylolisthesis from January 2014 to August 2017 enrolled in the Quality Outcomes Database (QOD). OUTCOME MEASURES Oswestry Disability Index (ODI), EuroQOL5D (EQ-5D), back (BP, 0-10) and leg pain (LP, 0-10) scores METHODS The QOD was queried for patients who underwent lumbar fusion for grade 1 degenerative spondylolisthesis from January 2014 to August 2017. Patient Reported Outcomes (PROs) included ODI, EQ-5D, BPand LP scores. Both anchor-based and distribution-based methods for MCID calculation were used. RESULTS Of 462 eligible cases, 356 (77%) had complete baseline and 12-month PROs. The MCID values for ODI were similar in patients with and without a diagnosis of obesity (20.58 and 20.69, respectively), and with and without anxiety/depression (24.72 and 22.56, respectively). The MCID values for BP, LP, and EQ-5D were also similar among all groups regardless of method used to determine MCID. CONCLUSIONS MCID thresholds for ODI, EQ5D, back pain and leg pain in patients with and without anxiety/depression and obesity undergoing spinal fusion for grade 1 degenerative spondylolisthesis are similar. Preoperative clinical and shared decision-making may be improved by understanding that preop medical comorbidities do not affect how patients determine an important clinical change postoperatively. FDA DEVICE/DRUG STATUS This abstract does not discuss or include any applicable devices or drugs.

Determining Thresholds for Meaningful Change for the Headache Impact Test (HIT-6) Total and Item-Specific Scores in Chronic Migraine.

ObjectiveThe objective of the analyses described here was to develop thresholds defining clinically meaningful response on the total and item scores of the 6‐item short‐form Headache Impact Test (HIT‐6) in a population of patients with chronic migraine (CM).BackgroundThe HIT‐6 is a short, easily understood, and useful measure of the impact of headache on daily life. Though widely used, limited literature supports a threshold value for clinically meaningful response within individuals over time for the HIT‐6 total score and for the item scores, especially in the CM population.MethodsPROMISE‐2 is a randomized, double‐blind, multicenter study comparing intravenous eptinezumab 100 and 300 mg with placebo for the preventive treatment of CM. Responder definitions for HIT‐6 total and items scores using data from PROMISE‐2 study were calculated via distribution‐based and anchor‐based methods. Distribution‐based methods included half of the baseline standard deviation and baseline standard error of measurement. The change from baseline to week 12 in HIT‐6 scores was assessed using the following anchors: patient global impression of change, reduction in migraine frequency, and change in EuroQol 5 dimensions 5 levels visual analog scale. Values from the literature and PROMISE‐2 analyses were plotted against the cumulative distribution function of change values (baseline to week 12) and used to triangulate to empirically support clinically meaningful change definitions for the HIT‐6 total and item scores in patients with CM.ResultsFrom the literature, 5 articles provided 7 candidate values for a responder threshold for the HIT‐6 total score. From distribution‐ and anchor‐based methods, 5 candidate values were derived from PROMISE‐2 data. Using the median of all candidate values, a HIT‐6 total score responder definition estimate of −6 (ie, ≥6‐point improvement in the total score) appears most appropriate for discriminating between individuals with CM who have experienced meaningful change over time and those who have not. For item‐level analyses using anchor‐based methods, the responder definition for items 1‐3 (“severe pain,” “limits daily activities,” and “lie down”) was a 1‐category improvement in response (eg, from Sometimes to Rarely); for items 4‐6 (“too tired,” “felt fed up or irritated,” and “limits concentration”), a 2‐category improvement in response (eg, from Always to Sometimes) was clinically meaningful.ConclusionsUsing a multifaceted, statistically‐based approach, the recommended responder definition for the HIT‐6 total score in the CM population is a ≥6‐point decrease, consistent with previous literature. Anchor‐based item‐level responder thresholds were defined as a decrease of 1 or 2 categories, depending on the item. These CM‐specific values will provide researchers and clinicians a means to interpret clinically meaningful change in the HIT‐6 total and item scores and may facilitate the measurement of treatment benefits in specific functional domains of the HIT‐6.

Preoperative patient-reported outcome score thresholds predict the likelihood of reaching MCID with surgical correction of adult spinal deformity.

Preoperative (pre-op) identification of patients likely to achieve a clinically meaningful improvement following surgery for adult spinal deformity (ASD) is critical, especially given the substantial cost and comorbidity associated with surgery. Even though pain is a known indication for surgical ASD correction, we are not aware of established thresholds for baseline pain and function to guide which patients have a higher likelihood of improvement with corrective surgery. We aimed to establish pre-op patient-reported outcome measure (PROM) thresholds to identify patients likely to improve by at least one minimum clinically important difference (MCID) with surgery for ASD. This is a retrospective cohort study using prospectively collected data. We reviewed 172 adult patients' charts who underwent corrective surgery for spinal deformity. Included measures were the Visual Analog Scale for pain (VAS), Oswestry Disability Index (ODI), and Scoliosis Research Society-22 (SRS-22). Our primary outcome of interest was improvement by at least one MCID on the ODI and SRS-22 at 2years after surgery. As part of usual care, the VAS, ODI, and SRS-22 were collected pre-op and re-administered at 1, 2, and 5years after surgery. MCIDs were calculated using a distribution-based method. Determining significant predictors of MCID at two years was accomplished by Firth bias corrected logistic regression models. Significance of predictors was determined by Profile Likelihood Chi-square. We performed a Youden analysis to determine thresholds for the strongest pre-op predictors. At year two, 118 patients (83%) reached MCID for the SRS and 127 (75%) for the ODI. Lower pre-op SRS overall, lower pre-op SRS pain, and higher pre-op SRS function predicted a higher likelihood of reaching MCID on the overall SRS (p < 0.05). Higher pre-op ODI, lower SRS pain and self-image, and higher SRS overall predicted a higher likelihood of reaching MCID on the ODI (p < 0.05). An ODI threshold of 29 predicted reaching MCID with a sensitivity of 0.89 and a specificity of 0.64 (AUC = 0.7813). An SRS threshold of 3.89 predicted reaching MCID with a sensitivity of 0.93 and specificity of 0.68 (AUC = 0.8024). We identified useful thresholds for ODI and SRS-22 with acceptable predictive ability for improvement with surgery for ASD. Pre-op ODI, SRS, and multiple SRS subscores are predictive of meaningful improvement on the ODI and/or SRS at 2years following corrective surgery for spinal deformity. These results highlight the usefulness of PROMs in pre-op shared decision-making.

Effects of preoperative obesity and psychiatric comorbidities on minimum clinically important differences for lumbar fusion in grade 1 degenerative spondylolisthesis: analysis from the prospective Quality Outcomes Database registry.

Medical comorbidities, particularly preoperatively diagnosed anxiety, depression, and obesity, may influence how patients perceive and measure clinical benefit after a surgical intervention. The current study was performed to define and compare the minimum clinically important difference (MCID) thresholds in patients with and without preoperative diagnoses of anxiety or depression and obesity who underwent spinal fusion for grade 1 degenerative spondylolisthesis. The Quality Outcomes Database (QOD) was queried for patients who underwent lumbar fusion for grade 1 degenerative spondylolisthesis during the period from January 2014 to August 2017. Collected patient-reported outcomes (PROs) included the Oswestry Disability Index (ODI), health status (EQ-5D), and numeric rating scale (NRS) scores for back pain (NRS-BP) and leg pain (NRS-LP). Both anchor-based and distribution-based methods for MCID calculation were employed. Of 462 patients included in the prospective registry who underwent a decompression and fusion procedure, 356 patients (77.1%) had complete baseline and 12-month PRO data and were included in the study. The MCID values for ODI scores did not significantly differ in patients with and those without a preoperative diagnosis of obesity (20.58 and 20.69, respectively). In addition, the MCID values for ODI scores did not differ in patients with and without a preoperative diagnosis of anxiety or depression (24.72 and 22.56, respectively). Similarly, the threshold MCID values for NRS-BP, NRS-LP, and EQ-5D scores were not statistically different between all groups. Based on both anchor-based and distribution-based methods for determination of MCID thresholds, there were no statistically significant differences between all cohorts. MCID thresholds were similar for ODI, EQ-5D, NRS-BP, and NRS-LP in patients with and without preoperative diagnoses of anxiety or depression and obesity undergoing spinal fusion for grade 1 degenerative spondylolisthesis. Preoperative clinical and shared decision-making may be improved by understanding that preoperative medical comorbidities may not affect the way patients experience and assess important clinical changes postoperatively.

Establishing clinically significant outcome thresholds for the Single Assessment Numeric Evaluation 2 years following total shoulder arthroplasty

Single Assessment Numerical Evaluation (SANE) is a simple, time-efficient patient-reported outcome measure (PROM) used to assess postoperative shoulder function. Clinically significant outcome values and ability to correlate with longer legacy PROM scores at 2 years following shoulder arthroplasty are unknown. A retrospective analysis was performed using SANE, American Shoulder and Elbow Surgeons Standardized Shoulder Assessment Form (ASES), and Constant scores that were collected at a minimum 2-year follow-up. A total of 153 patients who underwent anatomic total shoulder arthroplasty (TSA) or reverse total shoulder arthroplasty (RTSA) were included. A distribution-based method was used to determine the minimal clinically important difference (MCID). An anchor-based method was used to determine substantial clinical benefit (SCB). The following anchor question was collected alongside the PROMs and graded on a 15-point Likert-type scale to establish the SCB: "Since your surgery, has there been any change in the pain in your shoulder?" Linear regression was used to assess correlations between PROMs. SANE showed moderate correlation with ASES (R2= 0.493) and Constant (R2 = 0.586) scores (P < .001). The MCID value was 14.9, and the SCB absolute value was 80.4 (area under the curve = 0.663) for SANE. Multivariate logistic regression demonstrated that patients undergoing RTSA were less likely to achieve SCB on all 3 outcome measures (P < .02). This study establishes concurrent construct validity for SANE and suggests that it is a valid metric to assess the MCID and SCB at 2 years following anatomic TSA and RTSA. SANE demonstrated moderate correlations with ASES and Constant scores. Patients undergoing RTSA demonstrated a lower propensity to achieve SCB at 2 years postoperatively compared with anatomic TSA.

Patients with a Hypoplastic Labrum Achieve Similar Outcomes After Primary Labral Repair in the Treatment of Femoracetablular Impingement Syndrome Compared to Patients with a Normal Labrum

Objectives:A preoperative or intraoperative finding of an acetabular labrum width of <4mm is often cited as an indication for performing primary labral reconstruction over repair in the surgical correction of Femoracetablular Impingement Syndrome (FAIS). The purpose of this study is to compare patient reported clinical outcomes and rates of reaching Minimum Clinical Important Differences (MCID) and Patient Acceptable Symptomatic State (PASS) between patients with a hypoplastic labrum compared to those with a normal labrum width at a minimum 1 year from arthroscopic treatment of FAIS.Methods:Data from consecutive patients who underwent primary hip arthroscopy between November 2015 and July 2018 for the treatment of FAIS were analyzed. Baseline demographic data, preoperative, and minimum 1 year post-operative clinical outcomes including Hip Outcome Score-Activities of Daily Living (HOS-ADL), HOS-Sports Subscale (HOS-SS), modified Harris Hip Score (mHHS), international Hip Outcome Tool 12 questions (iHOT-12), and visual analog scale (VAS) for pain and satisfaction were recorded. The labrum size was documented by the senior surgeon for all patients using the tip of an arthroscopic probe measuring 4mm. Labrum hypoplasia was defined as a labrum width at the 12 o’clock position of <4mm and normal labrum was defined as labrum width between 4 and 7mm. Patients with hypoplastic labrum were matched 1:1 by age and BMI to patients with normal labrum width. All patients underwent arthroscopic labral repair in addition to impingement resection and capsular repair. The groups were further compared after separating by acetabular coverage, in which a Lateral Center Edge Angle of 18-24.9 was considered borderline dysplastic and 25-40 was considered normal. Rates of achieving MCID calculated using a distribution-based method and PASS calculated using an anchor-based method.Results:A total of 360 patients were included in the study with 180 in each of the normal and hypoplastic groups. There was no significant differences seen in 1-year post operative outcome scores or score improvement between the two groups. When examining only borderline dysplastic patients there were no significant differences in outcomes between normal and hypoplastic labrum patients. In the subset with a normal LCEA only post-operative VAS satisfaction (85.5 ± 19.3 vs 78.2 ± 27.4; p=0.050) was significantly higher in the normal labrum patients compared to hypoplastic labrum. Patients with hypoplastic labrum achieved MCID and PASS at the same rate as patients with normal labrum across all outcome measures.Conclusions:Patients with an intraoperative finding of labral hypoplasia achieve 1-year meaningful clinical outcome at the same rate as those with normal labral width following arthroscopic labral repair. These results bring into question the need for primary labral reconstruction in patients with labral hypoplasia with a normal or borderline dysplastic acetabulum.

Defining the MCID, PASS and SCB for Arthroscopic Hip Preservation Surgery at Minimum Five-Year Follow-up

Objectives:Minimal clinically important difference (MCID), substantial clinical benefit (SCB) and patient acceptable symptomatic state (PASS) have gained prominence as important variables in the orthopedic outcomes literature. In hip preservation surgery, much attention has been given to defining early clinically significant outcome, however, it is unknown what represents meaningful patient reported outcome improvement in the medium to long-term. The purpose of the present study was to define MCID, PASS and SCB at a minimum five years after hip arthroscopy for femoroacetabular impingement syndrome (FAIS). Secondarily, we sought to evaluate the time dependent nature of MCID, PASS and SCB.Methods:Patients undergoing hip arthroscopy for FAIS between January 2012 and March 2014 were included. Clinical and demographic data were collected in an institutional hip preservation registry. MCID, PASS, and SCB were calculated for each outcome score at 1-, 2-, and 5- years. MCID was calculated using a distribution-based method while PASS and SCB were calculated using an anchor method.Results:Two hundred and eighty-three patients were included with an average age of 34.2+11.9 years. The one year, two year and five-year MCID scores were as follows respectively: HOS-ADL (8.8, 9.7, 10.2); HOS-SS (13.9, 14.3, 15.2); mHHS (6.9, 9.2, 11.4) and iHOT-12 (15.1, 13.9, 15.1). PASS scores were as follows: HOS-ADL (89.7, 88.2, 99.2); HOS-SS (72.2, 76.4, 80.9); mHHS (84.8, 83.3, 83.6) and iHOT-12 (69.1, 72.2, 74.3). SCB scores were as follows: HOS-ADL (89.7, 91.9, 94.6); HOS-SS (78.1, 77.9, 85.8); mHHS (86.9, 85.8, 94.4) and iHOT-12 (72.6, 76.8, 87.5). More patients achieved MCID, SCB and PASS at two-year follow-up than at one-year follow-up. However, 79.3% of the patients achieved MCID by 5 years.Conclusion:The greatest proportion of patients achieve clinically significant outcome improvement at two-year follow-up after arthroscopic treatment of FAIS. Improvements are maintained out to five-year follow-up although there is a slight decrease in the proportion of patients achieving clinical significance.Table 1.Frequency of patients achieving any MCID, PASS, or SCB threshold at 1,2, and 5-years 1-year 2-year 5-year p-value Any MCID233 (82.6%)247 (87.3%)224 (79.3%)0.035Any PASS191 (67.6%)211 (74.9%)191 (67.5%)0.114Any SCB176 (62.3%)190 (67.2%)160 (56.6%)0.034

Solving Portfolio Optimization Problems Using MOEA/D and Lévy Flight

Portfolio optimization is a financial task which requires the allocation of capital on a set of financial assets to achieve a better trade-off between return and risk. To solve this problem, recent studies applied multi-objective evolutionary algorithms (MOEAs) for its natural bi-objective structure. This paper presents a method injecting a distribution-based mutation method named Lévy Flight into a decomposition based MOEA named MOEA/D. The proposed algorithm is compared with three MOEA/D-like algorithms, NSGA-II, and other distribution-based mutation methods on five portfolio optimization benchmarks sized from 31 to 225 in OR library without constraints, assessing with six metrics. Numerical results and statistical test indicate that this method can outperform comparison methods in most cases. We analyze how Lévy Flight contributes to this improvement by promoting global search early in the optimization. We explain this improvement by considering the interaction between mutation method and the property of the problem. We additionally show that our method perform well with a round-lot constraint on Nikkei.

The responsiveness and minimally important difference for the Accidental Bowel Leakage Evaluation questionnaire.

We describe the responsiveness and minimally important difference (MID) of the Accidental Bowel Leakage Evaluation (ABLE) questionnaire. Women with bowel leakage completed ABLE, Patient Global Impression of Improvement, Colo-Rectal Anal Distress Inventory, and Vaizey questionnaires pretreatment and again at 24weeks post-treatment. Change scores were correlated between questionnaires. Student's t tests compared ABLE change scores for improved versus not improved based on other measures. The MID was determined by anchor- and distribution-based approaches. In 266 women, the mean age was 63.75 (SD = 11.14) and 79% were white. Mean baseline ABLE scores were 2.32 ± 0.56 (possible range 1-5) with a reduction of 0.62 (SD = 0.79) by 24weeks. ABLE change scores correlated with related measures change scores (r = 0.24 to 0.53) and differed between women who improved and did not improve (all p < 0.001). Standardized response means for participants who improved were large ranging from -0.89 to -1.12. Distribution-based methods suggest a MID of -0.19 based on the criterion of one SEM and -0.28 based on half a standard deviation. Anchor-based MIDs ranged from -0.10 to -0.45. We recommend a MID of -0.20. The ABLE questionnaire is responsive to change, with a suggested MID of -0.20.

Clinically meaningful change in itch intensity scores: An evaluation in patients with chronic kidney disease–associated pruritus

Clinically meaningful change in itch intensity scores: An evaluation in patients with chronic kidney disease–associated pruritus

Minimal clinically important differences after subpectoral biceps tenodesis: definition and retrospective assessment of predictive factors

Minimal clinically important differences (MCIDs) at 1 year after subpectoral biceps tenodesis are unknown for the American Shoulder and Elbow Surgeons (ASES) scale, Subjective Shoulder Value (SSV), and visual analog scale (VAS) for pain. Our objectives were to determine MCIDs for these measures at 1 year after biceps tenodesis and to identify preoperative factors that predict attainment of MCIDs. We included 52 patients who underwent arthroscopic débridement, decompression, and mini-open biceps tenodesis from 2016-2018. We analyzed age, sex, body mass index value, arm dominance, diagnosis, range of shoulder motion, and preoperative and 1-year postoperative ASES, SSV, and VAS scores. MCIDs were calculated using a distribution-based method of one-half the standard deviation. Preoperative thresholds predictive of MCIDs were calculated with univariate logistic regression. Multiple logistic regression was used to determine factors that predict MCIDs. Significance was set at a 2-tailed P value of <.05. MCIDs for the ASES, SSV, and VAS were 13, 12, and 1.6 points, respectively. Preoperative ASES score <59 predicted MCID on the ASES (P = .03); VAS score >3 predicted MCID on the VAS (P < .01); external shoulder rotation >40° predicted MCID on the SSV (P = .02); and age >41 years predicted MCID on the VAS (P = .02). At 1 year after débridement, decompression, and biceps tenodesis, MCIDs were 13, 12, and 1.6 points for the ASES, SSV, and VAS, respectively. Patients most likely to attain MCIDs were those aged >41 years, those with the most preoperative pain, and those with the poorest preoperative shoulder function.

Role of preoperative PROMIS scores in predicting postoperative outcomes and likelihood of achieving MCID following reverse shoulder arthroplasty

BackgroundThe patient-reported outcomes measurement information system (PROMIS) has emerged as an efficient and valid outcome measure in various shoulder surgeries. The purpose of this study was to investigate the influence of preoperative PROMIS scores in predicting postoperative PROMIS scores and the likelihood of achieving a minimal clinically important difference (MCID) following primary reverse total shoulder arthroplasty for cuff tear arthropathy. We hypothesize that preoperative PROMIS scores will influence both postoperative PROMIS scores and the probability of achieving MCID. Methods73 patients undergoing reverse shoulder arthroplasty by a board-certified shoulder and elbow surgeon were given three PROMIS CAT forms: PROMIS Upper Extremity Physical Function CAT v2.0 (“PROMIS-UE”), PROMIS Pain Interference v1.1 (“PROMIS-PI”), and PROMIS Depression v1.0 (“PROMIS-D”).). PROMIS CAT domain t scores were assessed for significance between both time points using a Paired Samples t test. Minimal clinically important difference (MCID) was calculated using the distribution method and each PROMIS domain was subsequently assessed for its discriminatory ability in predicting postoperative improvement equal to or greater than the MCID through receiver operating characteristic (ROC) curve analysis. ResultsOur cohort consisted of 73 patients (49.3% male) and an average age of 69.7 years (standard deviation, 11.9). Mean follow-up time point was 9.6 months (standard deviation, 5.0) after surgery. Preoperative PROMIS-UE, PROMIS-PI, and PROMIS-D were 29.5 ± 6.2, 63.3 ± 5.4, and 50.1 ± 9.2, respectively. Each domain significantly improved at 10-months, on average, to 40.9 ± 7.8, 51.4 ± 8.5, 42.6 ± 8.1, respectively. Following the distribution-based method for MCID calculation, we found the following MCID values for PROMIS-UE, PROMIS-PI, and PROMIS-D: 3.1, 2.7, and 4.6, respectively. ROC analysis revealed strong predictive ability for PROMIS-UE (AUC = 0.717, p < 0.05), moderative predictive ability for PROMIS-PI (AUC = 0.634, p < 0.05), and excellent predictive ability for PROMIS-D (AUC = 0.864, p < 0.05). Specifically, preoperative cutoff values of <26.0, >70.0, and >52.5 for PROMIS-UE, PROMIS-PI, and PROMIS-D are especially predictive of achieving MCID. ConclusionsPreoperative baseline scores can serve as strong predictors of success in patients undergoing primary reverse shoulder arthroplasty and can be used to both counsel patients on surgery and to tailor postoperative protocols. Level of evidenceLevel II.

New evidence for construct validity and interpretability of the German Quality of Life in Hand Eczema Questionnaire (QOLHEQ).

The Quality of Life in Hand Eczema Questionnaire (QOLHEQ) is a disease-specific instrument used to assess health-related quality of life (HRQoL) in patients with hand eczema according to the domains of (a) symptoms, (b) emotions, (c) functioning, and (d) treatment/prevention. Today it is not clear what a single score of the QOLHEQ in its German-language version means to a patient. It was the aim of this study to band the QOLHEQ score to an anchor question (AQ) in order to obtain meaningful categories of the QOLHEQ to aid its interpretation. In addition, we assessed the minimal important change (MIC) by using anchor- and distribution-based methods. Overall n = 440 hand eczema patients were included in the study. Mean age was 47.5 years (SD 11.9); 38.4% of the sample were female. With a weighted kappa of 0.62, the total QOLHEQ score showed the best agreement for the following band: QOLHEQ of <17 = no impairment; QOLHEQ of 18-28 = slight impairment; QOLHEQ of 29-41 = moderate impairment; QOLHEQ of 42-79 = severe impairment; and QOLHEQ of >79 = very severe impairment. The MIC for the total score was found to be 16.5 points. This banding represents a standardized means of interpreting the QOLHEQ total score. Our results indicate that a banding study should be performed for each language version of the QOLHEQ.

Influence of workers' compensation status on postoperative outcomes in patients following biceps tenodesis: a matched-pair cohort analysis

Although the literature on the association of workers' compensation (WC) status with negative outcomes after orthopedic surgery is extensive, there is a paucity of evidence on outcomes in WC recipients undergoing biceps tenodesis. We hypothesized that WC patients would report significantly worse outcomes postoperatively on patient-reported outcome measures (PROMs). Functional and health-related quality-of-life PROMs and a visual analog scale score for pain were administered preoperatively and at 12 months postoperatively to consecutive patients undergoing isolated biceps tenodesis between 2014 and 2018 at our institution. Thirty-eight WC patients were matched 1:2 to non-WC patients by age, body mass index, and operative limb. The minimal clinically important difference, substantial clinical benefit, and patient acceptable symptom state were calculated for all patients via anchor- and distribution-based methods. Rates of achievement and the likelihood of achievement were determined. All patients showed significant improvements in all outcome measures (P < .001). WC patients reported inferior postoperative scores on all PROMs examined. WC status significantly predicted a reduced likelihood of achieving substantial clinical benefit for the American Shoulder and Elbow Surgeons score (odds ratio [OR], 0.38; 95% confidence interval [CI], 0.17-0.81; P = .01) and the patient acceptable symptom state (OR, 0.28; 95% CI, 0.12-0.65; P = .003) for the American Shoulder and Elbow Surgeons score, Single Assessment Numeric Evaluation score (OR, 0.24; 95% CI, 0.10-0.61; P = .003), Constant-Murley Subjective Assessment (OR, 0.25; 95% CI, 0.08-0.77; P = .016), and visual analog scale pain score (OR, 0.27; 95% CI, 0.16-0.47; P < .001). WC patients reported inferior scores on all postoperative PROMs and demonstrated lower odds of achieving substantial benefit and satisfaction regarding improvements in both function and paincompared with non-WC patients.

Defining the Minimal Clinically Important Difference and Patient-acceptable Symptom State Score for Disability Assessment in Surgical Patients.

The World Health Organization Disability Assessment Schedule 2.0 has been used to measure postoperative disability in several clinical trials and cohort studies. It is uncertain what the minimal clinically important difference or patient-acceptable symptom state scores are for this scale in patients recovering from surgery. The authors analyzed prospectively collected data from three studies that measured disability 3 and 6 months after surgery. Three distribution-based methods (0.3 multiplied by SD, standard error of the measurement, and 5% range) and two anchor-based methods (anchored to two patient-rated health status questions and separately to unplanned hospital readmission) were averaged to estimate the minimal clinically important difference for the World Health Organization Disability Assessment Schedule 2.0 score converted to a percentage scale. Scores consistent with a patient-acceptable symptom state and clinically significant disability were determined by an anchored 75th centile method. Data from 4,361 patients were analyzed. The average minimal clinically important difference estimate for the World Health Organization Disability Assessment Schedule 2.0 was 5%, with similar estimates in patients with or without preoperative disability. The patient-acceptable symptom state score was 16%, and the score consistent with at least moderate clinically significant disability was 35%. Using these estimates, between baseline and 6 months after surgery, 21% of patients had a significant increase in disability, and 73% achieved a patient-acceptable symptom state. A change in World Health Organization Disability Assessment Schedule 2.0 score of 5% or more after surgery is consistent with a clinically important change in disability. Patients with a score less than 16% after surgery have an acceptable symptom state and can be considered as disability-free, whereas patients with a score of 35% or more can be considered as having at least moderate clinically significant disability.

Minimal Clinically Important Difference, Maximum Impact.

Minimal Clinically Important Difference, Maximum Impact.

Minimum clinically important difference of major patient-reported outcome measures in patients undergoing decompression surgery for lumbar spinal stenosis

ObjectiveA minimum clinically important difference (MCID) has been increasingly well known in the current era of patient-centered care because it reflects a smallest change that is meaningful for patients following a clinical intervention. Previous studies suggested MCID values are disease and/or procedure dependent. No MCID values have been reported on the lumbar spinal stenosis (LSS) following decompression surgery despite LSS is the most common spinal disease and the main treatment is decompression surgery. Therefore, this study aimed to determine the MCID values as major outcome measures including the Numeric Rating Scale (NRS) of back pain, leg pain and numbness, Roland-Morris Disability Questionnaire (RMDQ), and Physical Component Summary (PCS) and Mental Component Summary (MCS) of Short Form 8 (SF-8) for patients with LSS undergoing decompression surgery. Patients and MethodsThis is a retrospective cohort study using prospectively collected data from consecutive patients who underwent lumbar decompression without fusion for LSS at a single institution between May 2014 and March 2016. Inclusion criteria were 1) minimum 1-year follow-up 2) a complete set of preoperative and final follow-up questionnaires available, including the NRS, RMDQ, and SF-8. Revision surgery or non-degenerative etiology such as infection or tumor was excluded. MCIDs of each outcome measure were determined using two major approaches, distribution- and anchor-based methods. The distribution-based method uses the distributional characteristics of the sample. This method expresses the observed degree of variation to obtain a standardized metric such as the standard deviation or standard error of measurement. The anchor-based method uses an external criterion known as anchor to determine the factors that should be considered by patients for an important improvement. Anchor-based methods assess how much changes in the measurement instrument correspond with a minimal important change defined on the anchor. We used symptom severity, physical function, and satisfaction scores from Zurich Claudication Questionnaire as anchors for NRS and RMDQ, PCS, and MCS, respectively. ResultsA total of 126 patients were included. From the anchor-based method, MCIDs were determined to be 2 points for back pain, 4 points for leg pain and numbness, 5 points for RMDQ, 5 points for PCS, and 2 points for MCS. From the distribution-based method, MCIDs were determined to be 2 points for back pain, leg pain and numbness, 3–4 points for RMDQ, 6 points for PCS, and 5 points for MCS. ConclusionWe first identified the MCIDs of the NRS, RMDQ, and SF-8 specific to patients undergoing decompression surgery for LSS.

Practical issues encountered while determining Minimal Clinically Important Difference in Patient-Reported Outcomes

BackgroundUsing a real dataset, we highlighted several major methodological issues raised by the estimation of the Minimal Clinically Important Difference (MCID) of a Patient-Reported Outcomes instrument. We especially considered the management of missing data and the use of more than two times of measurement. While inappropriate missing data management and inappropriate use of multiple time points can lead to loss of precision and/or bias in MCID estimation, these issues are almost never dealt with and require cautious considerations in the context of MCID estimation.MethodsWe used the LIGALONGO study (French Randomized Controlled Trial). We estimated MCID on the SF-36 General Health score by comparing many methods (distribution or anchor-based). Different techniques for imputation of missing data were performed (simple and multiple imputations). We also consider all measurement occasions by longitudinal modeling, and the dependence of the score difference on baseline.ResultsThree hundred ninety-three patients were studied. With distribution-based methods, a great variability in MCID was observed (from 3 to 26 points for improvement). Only 0.2 SD and 1/3 SD distribution methods gave MCID values consistent with anchor-based methods (from 4 to 7 points for improvement). The choice of missing data imputation technique clearly had an impact on MCID estimates. Simple imputation by mean score seemed to lead to out-of-range estimate, but as missing not at random mechanism can be hypothesized, even multiple imputations techniques can have led to an slight underestimation of MCID. Using 3 measurement occasions for improvement led to an increase in precision but lowered estimates.ConclusionThis practical example illustrates the substantial impact of some methodological issues that are usually never dealt with for MCID estimation. Simulation studies are needed to investigate those issues.Trial registrationNCT01240772 (ClinicalTrials.gov) registered on November 15, 2010.