Distribution-based Methods Research Articles

King's Brief Interstitial Lung Disease questionnaire: responsiveness and minimum clinically important difference.

Health status is increasingly used in clinical practice to quantify symptom burden and as a clinical trial end-point in patients with interstitial lung disease (ILD). The King's Brief Interstitial Lung Disease (KBILD) questionnaire is a brief, validated 15-item, disease-specific, health-related quality of life questionnaire that is increasingly used in clinical trials, but little data exist regarding the minimum clinically important difference (MCID). Using pulmonary rehabilitation as a model, we aimed to determine the responsiveness of KBILD and provide estimates of the MCID.KBILD scores, Chronic Respiratory Questionnaire (CRQ) scores, Medical Research Council (MRC) Dyspnoea score and incremental shuttle walk test (ISWT) distance were measured in 209 patients with ILD (105 with idiopathic pulmonary fibrosis (IPF)) before and after an outpatient pulmonary rehabilitation programme. Changes with intervention and Cohen's effect size were calculated. Anchor-based (linear regression and receiver operating characteristic plots) or distribution-based approaches (0.5 sd and standard error of measurement) were used to estimate the MCID of KBILD domain and total scores.KBILD, CRQ, MRC Dyspnoea and ISWT improved with intervention, and the effect sizes of KBILD domain and total scores ranged from 0.28 to 0.38. Using anchor-based estimates, the MCID estimates for KBILD-Psychological, KBILD-Breathlessness and activities, and KBILD-Total were 5.4, 4.4 and 3.9 points, respectively. Using distribution-based methods, the MCID estimate for KBILD-Chest symptoms was 9.8 points. The MCID estimates for KBILD in IPF patients were similar.In patients with ILD and IPF, KBILD is responsive to intervention with an estimated MCID of 3.9 points for the total score.

A new objective and distribution-based method to characterize pseudo-slug flow from wire-mesh-sensors (WMS) data

The subjective nature of intermittent (slug/pseudo-slug) flow characterizations has limited the reproducibility and consistency among independent datasets and may contribute to the perceived poor performance of available closure relationships. This deficiency is more critical for pseudo-slug flow where pseudo-slug body and film regions are not easily differentiable. This paper proposed an objective method to characterize pseudo-slug flow based on time-trace signal (time-series) of wire-mesh-sensors (WMS) data. Primarily, the front/tail threshold values and disregard film criteria are selected based on the distribution benchmarking of holdup and film length, respectively.The proposed method is validated qualitatively and quantitatively. The eye-test qualitatively shows that the proposed method is capable of identifying the pseudo-slug front consistently. The end of pseudo-slug is located in the middle of the tail region, where holdup gradually decreases from pseudo-slug (HLPS) to film (HLF) holdup. This behavior is physically intuitive as the tail region consists of both upward (similar to pseudo-slug body) and backward moving liquid bodies (similar to liquid film). Quantitatively, the variables measured with the proposed method, namely: pseudo-slug frequency (fPS), structure velocity (vTPS), body length (lPS), body holdup (HLPS), and film holdup (HLF), demonstrate self-consistency within the framework of unit cell model, thereby indicating conformance to the continuity equation of intermittent flow.

Translation into modern standard Arabic, cross-cultural adaptation and psychometric properties' evaluation of the Lower Extremity Functional Scale (LEFS) in Arabic-speaking athletes with Anterior Cruciate Ligament (ACL) injury.

BackgroundThe Lower Extremity Functional Scale evaluates the functional status of patients that have lower extremity conditions of musculoskeletal origin. Regional Arabic dialects often create barriers to clear communication and comparative research. We aimed to cross-culturally adapt the Lower Extremity Functional Scale in modern standard Arabic that is widely used and understood in the Middle East and North Africa region, and assess its psychometric properties.MethodsCross-cultural adaptation followed a combination of recommended guidelines. For psychometric evaluation 150 patients with anterior cruciate ligament injury and 65 asymptomatic individuals were recruited. All measurement properties as indicated by the Consensus-based Standards for the selection of health status Measurement Instruments recommendations were evaluated, including content-relevance analysis, structural validity, longitudinal reproducibility, anchor- and distribution-based methods of responsiveness, as well as the longitudinal pattern of change of Lower Extremity Functional Scale in anterior cruciate ligament injured patients’ functional status.ResultsThe questionnaire presented excellent internal consistency (α = 0.96), reliability (0.80–0.98), and good convergent validity (ρ = 0.85). For reproducibility testing: minimal detectable change was 9.26 points; for responsiveness assessment: minimal clinically important difference was 9 points and presented moderate effect sizes (Glass’Δ = 0.71, Cohen’s d = 0.81). Its unidimensionality was not confirmed and an exploratory factor analysis indicated a 2-factor solution explaining 78.1% of the variance.ConclusionThe Arabic Lower Extremity Functional Scale presented acceptable psychometric properties comparable to the original version. The Arabic version of Lower Extremity Functional Scale can be used in research and clinical practice to assess the functional status of Arabic-patients suffering an anterior cruciate ligament injury.

Understanding the Minimal Clinically Important Difference (MCID) of Patient-Reported Outcome Measures.

The minimal clinically important difference (MCID) of a patient-reported outcome measure (PROM) represents a threshold value of change in PROM score deemed to have an implication in clinical management. The MCID is frequently used to interpret the significance of results from clinical studies that use PROMs. However, an understanding of the many caveats of the MCID, as well as its strengths and limitations, is necessary. The objective of this article is to provide a review of the calculation, interpretation, and caveats of MCID. MEDLINE and PubMed Central. Literature search-including primary studies, review articles, and consensus statements-pertinent to the objectives of this review using PubMed. The MCID of a PROM may vary depending on the patients and clinical context in which the PROM is given. The primary approaches for calculating MCID are distribution-based and anchor-based methods. Each methodology has strengths and limitations, and the ideal determination of a PROM MCID includes synthesis of results from both approaches. The MCID of a PROM is also not perfect in detecting patients experiencing a clinically important improvement, and this is reflected in its accuracy (eg, sensitivity and specificity). Interpretation or application of MCID requires consideration of all caveats underlying the MCID, including the patients in whom it was derived, the limitations of the methodologies used to calculate it, and its accuracy for identifying patients who have experienced clinically significant improvement.

A Threshold of Meaning for Work Disability Improvement in Psoriatic Arthritis Measured by the Work Productivity and Activity Impairment Questionnaire.

IntroductionThe Work Productivity and Activity Impairment Specific Health Problem Questionnaire (WPAI:SHP) is used to assess the impact of an intervention on work productivity in patients with psoriatic arthritis (PsA). Unfortunately, studies reporting changes or improvements in domains of WPAI:SHP by patients with PsA have a limited threshold of meaning due to the absence of published minimal clinically important differences (MCIDs). Our objective was to determine the MCIDs for improvement in WPAI:SHP in patients with active PsA.MethodsMCIDs for WPAI:SHP domains (presenteeism, work productivity loss, and activity impairment) were derived for patients with active PsA who were biologic naïve or TNF inhibitor (TNFi) experienced using 24-week results from two phase 3 trials (SPIRIT-P1 and SPIRIT-P2). MCIDs were derived using the anchor-based method supplemented by the distribution-based method. Anchors included achievement of the American College of Rheumatology 20 responder index (ACR20), the minimal disease activity (MDA), and the Health Assessment Questionnaire and Disability Index (HAQ-DI) MCID (improvement ≥ 0.35). Anchor validity was assessed by biserial correlation and analysis of covariance modeling against the domains. MCIDs were triangulated using the receiver operating characteristic (ROC) method supplemented by the distribution-based method.ResultsThe analyses included 417 biologic-naïve and 363 TNFi-experienced patients. ACR20, MDA, and HAQ-DI were valid anchors. Significant differences in WPAI:SHP domain scores were observed between patients achieving ACR20, MDA, or HAQ-DI compared to patients not achieving these clinical thresholds (all P < 0.001). ROC analyses suggested that a ≥ 20% improvement in presenteeism, a 15% improvement in work productivity loss, and a 20% improvement in activity impairment represented clinically meaningful improvements in both populations. The distribution-based method supported the results.ConclusionMCIDs for the presenteeism, work productivity loss, and activity impairment domains were estimated to be 20%, 15%, and 20%, respectively, in biologic-naïve or TNFi-experienced PsA populations. These results will help improve the meaningfulness of WPAI:SHP improvements reported by PsA patients.Trial RegistrationSPIRIT-P1: NCT01695239, SPIRIT-P2: NCT02349295.FundingEli Lilly and Company.

Thresholds for clinically important deterioration versus improvement in COPD health status: results from a randomised controlled trial in pulmonary rehabilitation and an observational study during routine clinical practice.

ObjectivesChronic Obstructive Pulmonary Disease (COPD) is a progressive disease. Preventing deterioration of health status is therefore an important therapy goal. (Minimal) Clinically Important Differences ((M)CIDs) are used to interpret changes observed. It remains unclear whether (M)CIDs are similar for both deterioration and improvement in health status. This study investigates and compares these clinical thresholds for three widely-used questionnaires.Design and settingData were retrospectively analysed from an inhouse 3-week pulmonary rehabilitation (PR) randomised controlled trial in the German Klinik Bad Reichenhall (study 1), and observational research in Dutch primary and secondary routine clinical practice (RCP) (study 2).ParticipantsPatients with COPD aged ≥18 years (study 1) and aged ≥40 years (study 2) without respiratory comorbidities were included for analysis.Primary outcomesThe COPD Assessment Test (CAT), Clinical COPD Questionnaire (CCQ) and St George’s Respiratory Questionnaire (SGRQ) were completed at baseline and at 3, 6 and 12 months. A Global Rating of Change scale was added at follow-up. Anchor-based and distribution-based methods were used to determine clinically relevant thresholds.ResultsIn total, 451 patients were included from PR and 207 from RCP. MCIDs for deterioration ranged from 1.30 to 4.21 (CAT), from 0.19 to 0.66 (CCQ), and from 2.75 to 7.53 (SGRQ). MCIDs for improvement ranged from −3.78 to −1.53 (CAT), from −0.50 to −0.19 (CCQ), and from −9.20 to −2.76 (SGRQ). Thresholds for moderate improvement versus deterioration ranged from −5.02 to −3.29 vs 3.89 to 8.14 (CAT), from −0.90 to −0.72 vs 0.42 to 1.23 (CCQ), and from −15.85 to −13.63 vs 7.46 to 9.30 (SGRQ).ConclusionsMCID ranges for improvement and deterioration on the CAT, CCQ and SGRQ were somewhat similar. However, estimates for moderate and large change varied and were inconsistent. Thresholds differed between study settings.Trial registration numberRoutine Inspiratory Muscle Training within COPD Rehabilitation trial: #DRKS00004609; MCID study: #UMCG201500447.

Minimal Clinically Important Differences for PROMIS Physical Function, Upper Extremity, and Pain Interference in Carpal Tunnel Release Using Region- and Condition-Specific PROM Tools

Uncertainty exists about what change in Patient-Reported Outcomes Measurement Information System (PROMIS) scores represents a clinically relevant improvement (minimal clinically important difference [MCID]) in hand surgery care. Using a region-specific patient-reported outcome measure (PROM) (Michigan Hand Question [MHQ]) and a condition-specific PROM (Boston Carpal Tunnel Questionnaire [BCTQ]), MCID values were determined for PROMIS Physical Function (PF), Upper Extremity (UE), and Pain Interference (PI) computerized adaptive testing among patients undergoing carpal tunnel release (CTR). Patients undergoing CTR with a single surgeon from November 2014 to April 2017 were asked to complete the BCTQ, MHQ, and PROMIS PF, UE, and PI at each visit. Patients who had completed questionnaires both at a preoperative and either a 6-week or a 3-month postoperative visit were included. The PROMIS PF, UE, and PI MCID values were calculated using previously determined MCID estimates in the literature with both region- (ie, MHQ) and condition-specific (ie, BCTQ) PROM anchors. The PROMIS domain MCID estimates were also determined using the distribution-based method. A total of 70 patients fit our inclusion criteria. Using MHQ Function and Pain, PROMIS UE, PF, and PI MCIDs were 6.3, 1.8, and -8.9, respectively. Using the average of the 2 BCTQ domains, PROMIS UE, PF, and PI MCIDs were 8.0, 2.8, and -9.7, respectively. Using the distribution-based method, PROMIS UE, PF, and PI MCIDs were 4.2, 2.7, and-4.1, respectively. Using region- and condition-specific PROMs, we were able to provide MCID estimates of PROMIS UE, PF, and PI for patients undergoing CTR. Estimating PROMIS UE, PF, and PI MCIDs in CTR using validated region- and condition-specific PROMs provides hand surgeons a way to evaluate CTR outcomes not previously described in the literature. Surgeons should understand that these values are only estimates and future work is needed to verify whether they reflect clinical improvement.

Minimal clinically important differences and substantial clinical benefits for Knee Society Scores.

There is a paucity of literature defining the minimal clinically important difference (MCID) for the Knee Society Scores (KSS) after total knee arthroplasty (TKA), and no data on the substantial clinical benefit (SCB) for KSS have been reported. The purpose of this study was to determine MCID and SCB for the KSS in patients with primary TKA. The median age of patients was 71.6 (range 50-88) years, and 60.3% were females 507 patients with TKA were prospectively enrolled. Patients completed the KSS before surgery and at second postoperative year. The MCID values of the KSS were estimated using anchor-based method, distribution-based method and receiver operating characteristic (ROC) curve analysis with calculation of the area under curve (AUC). SCB was estimated using ROC. The MCID for KSS-knee score was 7.2 points by the anchor-based method, 7.2 by the distribution-based method, and using a ROC analysis the cutoff point was 8.9 points with an AUC of 0.75. For KSS-function score, the MCID values were 9.7, 6.3, and 10.3 (AUC 0.71), respectively. SCB values were 39.7 points (AUC 0.74) for the KSS-knee score, and 38.6 (AUC 0.76) for the KSS-function score. Logistic regression showed age and Charlson index to negatively affect the changes in KSS. Different methods for MCID calculation lead to different results. With the use of ROC curve analysis, patients with an improvement of at least 9 points for KSS-knee and 10 points for KSS-function scores experience a clinically important change, whereas those who have at least an improvement of 40 points for KSS-knee and 39 points for KSS-function scores experience a substantial clinical benefit. These findings can ensure clinical improvement from the patient's perspective and also aid in interpreting results from clinical studies. III.

Patient-reported Outcomes and Late Toxicity After Postprostatectomy Intensity-modulated Radiation Therapy

BackgroundLimited long-term data characterize patient-reported quality of life (QOL) following postprostatectomy intensity-modulated radiation therapy (PPRT), and predictors of decline are poorly defined. ObjectiveTo identify modifiable dosimetric and clinical risk factors impacting QOL and late toxicity following PPRT. Design, setting, and participantsA prospective cohort study of consecutive men with prostate cancer who received PPRT between 2007 and 2015 at a single academic institution. InterventionPatients were prospectively evaluated using the Expanded Prostate Cancer Index Composite (EPIC-26) QOL instrument. Radiation Therapy Oncology Group/Common Toxicity Criteria for Adverse Events toxicity grades were assigned at every follow-up visit. Treatment was delivered to the prostate bed (median 68Gy)±pelvic lymphatics (65%, median 50.4Gy) with daily image guidance. Androgen deprivation therapy was concomitantly administered to 132 (66%) men for a median of 4mo. Outcome measurements and statistical analysisChanges were deemed relevant if they exceeded the minimally clinically important difference (MCID), as calculated by a distribution-based method. Generalized estimating equation models and Cox regression were used for QOL and late toxicity univariate and multivariable analysis. Results and limitationsOverall, 199 men were identified with a median follow-up of 33mo. Overall urinary function (UF), bowel function (BF), sexual function (SF), and urinary irritation/obstruction (UI/UO) scores were never lower than the MCID. Between 8% and 18% of men experienced a small multidomain (1× MCID) decline, and 0–8% experienced a moderate multidomain decline (2× MCID) at a given time point up to 84mo after PPRT. The rates of freedom from grade 2 or higher (Gr2+) genitourinary (GU) and gastrointestinal (GI) toxicity were 94% and 95%, respectively, at 4yr. Factors associated with worse QOL or toxicity included longer time to PPRT (UC and UF), higher BMI (UF, BF, and late GI toxicity), older age (BF, SF, and late GU toxicity); hormone therapy (SF), total dose (late GI toxicity), tobacco history (BF), and higher bladder V70Gy (UC, UF, and late GU toxicity). ConclusionsLong-term QOL and late toxicity are favorable following postprostatectomy radiation therapy. Identifiable clinical and dosimetric risk factors may guide decision making to optimize urinary, sexual, and bowel function. Patient summaryThe following study provides a detailed report of favorable patient-reported quality of life and late side-effect profiles of radiation therapy following surgery for localized prostate cancer. Our findings provide patients guidance on what symptoms to expect if they are planning to undergo radiation therapy in this setting. It also allows physicians to counsel patients appropriately, and modify certain clinical and radiation-related risk factors to optimize quality of life.

Minimal clinically important decline in physical function over one year: EPOSA study

BackgroundThe Australian/Canadian hand Osteoarthritis Index (AUSCAN) and the Western Ontario and McMaster Universities knee and hip Osteoarthritis Index (WOMAC) are the most commonly used clinical tools to manage and monitor osteoarthritis (OA). Few studies have as yet reported longitudinal changes in the AUSCAN index regarding the hand. While there are published data regarding WOMAC assessments of the hip and the knee, the two sites have always evaluated separately. The current study therefore sought to determine the minimal clinically important difference (MCID) in decline in the AUSCAN hand and WOMAC hip/knee physical function scores over 1 year using anchor-based and distribution-based methods.MethodsThe study analysed data collected by the European Project on Osteoarthritis, a prospective observational study investigating six adult cohorts with and without OA by evaluating changes in the AUSCAN and WOMAC physical function scores at baseline and 12–18 months later. Pain and stiffness scores, the performance-based grip strength and walking speed and health-related quality of life measures were used as the study’s anchors. Receiver operating characteristic curves and distribution-based methods were used to estimate the MCID in the AUSCAN and WOMAC physical function scores; only the data of those participants who possessed paired (baseline and follow up-measures) AUSCAN and WOMAC scores were included in the analysis.ResultsOut of the 1866 participants who were evaluated, 1842 had paired AUSCAN scores and 1845 had paired WOMAC scores. The changes in the AUSCAN physical function score correlated significantly with those in the AUSCAN pain score (r = 0.31). Anchor- and distribution-based approaches converged identifying 4 as the MCID for decline in the AUSCAN hand physical function. Changes in the WOMAC hip/knee physical function score were significantly correlated with changes in both the WOMAC pain score (r = 0.47) and the WOMAC stiffness score (r = 0.35). The different approaches converged identifying two as the MCID for decline in the WOMAC hip/knee physical function.ConclusionsThe most reliable MCID estimates of decline over 1 year in the AUSCAN hand and WOMAC hip/knee physical function scores were 4 and 2 points, respectively.

Clostridium difficile Infection\u2013Daily Symptoms (CDI-DaySyms\u2122) questionnaire: psychometric characteristics and responder thresholds

BackgroundThe purpose of the current study was to determine the final content validation, psychometric characteristics, clinically meaningful improvement, and responder thresholds of the Clostridium difficile infection (CDI)–Daily Symptoms (CDI-DaySyms™) patient-reported outcome (PRO) questionnaire.MethodsThis validation study was part of two phase III studies (NCT01987895 and NCT01983683) conducted in patients with mild-to-moderate or severe CDI who completed the CDI-DaySyms™ daily throughout the treatment period. The questionnaire was evaluated in three stages: final PRO item content validation (Stage I); psychometric evaluation of reliability and construct validity (Stage II); and determination of clinically meaningful improvement and responder thresholds using distribution-based methods (Stage III).ResultsThe analysis included 168 patients. Most patients were female and Caucasian with mild-to-moderate CDI. The mean age was 57.1 years. Initial item analysis supported by confirmatory factor analysis demonstrated the relevance of 10 items grouped into three distinct domains (Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms). Domain scores demonstrated acceptable internal consistency and test-retest reliability, were sensitive to change, and correlated in expected directions with other relevant symptom and disease-severity measures. Responder thresholds were defined as score changes of − 1.00, − 0.80, and − 0.70 in the Diarrhea Symptoms, Abdominal Symptoms, and Systemic/Other Symptoms domains, respectively.ConclusionsThe CDI-DaySyms™ is a valid measure of patient-reported CDI symptoms, with good measurement properties, which supports its utility as an endpoint in clinical studies. Further studies confirming responder thresholds based on anchor-based methods are required.Trial registrationNCT01987895, registered November 20, 2013; NCT01983683, registered November 14, 2013.

Minimal clinically important differences in Toronto Extremity Salvage Score for patients with lower extremity sarcoma

BackgroundThe Toronto Extremity Salvage Score (TESS) is the most widely used patient-reported outcome measure for orthopaedic oncology patients. However, minimal clinically important differences (MCIDs) in the TESS have not been analyzed. The aim of this study was to define the MCIDs of TESS in patients with lower extremity sarcoma. MethodsA total of 85 patients were investigated to calculate the MCIDs for TESS. Three different methods were used: 1) distribution-based methods based on one-half of the standard deviation and standard error of measurement (SEM) at the baseline, 2) anchor-based and receiver operating characteristic (ROC) analysis, and 3) anchor-based using Akaike's Information Criterion (AIC) analysis. ResultsThe MCIDs at 6 months were 4.9–7.8 by distribution-based methods and 4.3–4.4 by anchor-based methods. The MCIDs at 12 months were 4.0–6.9 by distribution-based methods and 10.6–11.6 by anchor-based methods. ConclusionsWe calculated MCID values for the TESS based on distribution- and anchor-based approaches. Our results seem reasonable since MCIDs calculated by the different approaches had similar values. This knowledge will enable clinicians to identify meaningful functional improvements in sarcoma patients.

Responsiveness, Minimal Clinically Important Difference, and Validity of the MoCA in Stroke Rehabilitation.

Objective Persons with stroke frequently suffer from cognitive impairment. The Montreal Cognitive Assessment (MoCA), a recently developed screening tool, is sensitive to poststroke cognitive deficits. The present study assessed its psychometric and clinimetric properties (i.e., responsiveness, minimal clinically important difference (MCID), and criterion validity) in stroke survivors receiving rehabilitative therapy. Method The MoCA and the Stroke Impact Scale (SIS) were administered to 65 stroke survivors before and after 4 to 5 weeks of therapy. The effect size and standardized response mean (SRM) were calculated for responsiveness. Anchor- and distribution-based methods were used to estimate the MCID. Criterion validity was measured with the Spearman correlation coefficient. Results The responsiveness of the MoCA was moderate (SRM = 0.67). Participants exceeding the MCID according to the anchor- and distribution-based approaches were 33 (50.77%) and 20 (30.77%), respectively. Fair to good concurrent validity was reported between the MoCA and the SIS communication subscale. The MoCA had satisfactory predictive validity with the SIS communication and memory subscales. Conclusion This study may support the responsiveness, MCID, and criterion validity of the MoCA in stroke populations. Future studies with larger sample sizes are needed to validate the current findings.

Minimal clinically important difference of the EORTC QLQ-CIPN20 for worsening peripheral neuropathy in patients receiving neurotoxic chemotherapy.

This is the first study to determine the minimal clinically important difference (MCID) of the European Organisation of Research and Treatment of Cancer Quality of Life Questionnaire-CIPN twenty-item scale (EORTC QLQ-CIPN20), a validated instrument designed to elicit cancer patients' experience of symptoms and functional limitations related to chemotherapy-induced peripheral neuropathy. Cancer patients receiving neurotoxic chemotherapy completed EORTC QLQ-CIPN20 and the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity [FACT/GOG-NTX] at baseline, second cycle of chemotherapy (T2, n = 287), and 12months after chemotherapy (T3, n = 191). Anchor-based approach used the validated FACT/GOG-NTX neurotoxicity (Ntx) subscale to identify optimal MCID cutoff for deterioration. Distribution-based approach used one-third standard deviation (SD), half SD, and one standard error of measurement of the total EORTC QLQ-CIPN20 score. There was a moderate correlation between the change scores of the Ntx subscale and sensory and motor subscales of QLQ-CIPN20 (T2: r = - 0.722, p < 0.001 and r = - 0.518, p < 0.001, respectively; T3: r = - 0.699; p < 0.001 and r = - 0.523, p < 0.001, respectively). The correlation between the change scores of the Ntx subscale and the QLQ-CIPN20 autonomic subscale was poor (T2: r = - 0.354, p < 0.001; T3: r = 0.286, p < 0.001). Based on the MCID derived using distribution-based method, the MCID for the QLQ-CIPN20 sensory subscale was 2.5-5.9 (6.9% to 16.4% of the subdomain score) and for motor subscale was 2.6-5.0 (8.1%-15.6% of the subdomain score). The MCID for the EORTC QLQ-CIPN20 established using distribution-based approaches was 2.5-5.9 for the sensory subscale and 2.6-5.0 for the motor subscale. When noted in assessments even with small change in scores, clinicians can be alerted for appropriate intervention.

Minimal Clinically Important Difference for PROMIS Physical Function in Patients With Distal Radius Fractures

This study was conducted to determine the minimal clinically important difference (MCID) of the Patient-Reported Outcomes Information System (PROMIS) Physical Function computer adaptive test (CAT) after distal radius fracture. This study retrospectively analyzed data from 187 adults receiving nonsurgical care for a unilateral distal radius fracture at a single institution between February 2016 and November 2017. All patients completed the PROMIS Physical Function v1.2/2.0 CAT at each visit. At follow-up, patients also completed 2 multiple-choice clinical anchor questions querying their overall response to treatment. The MCID estimate was then calculated with an anchor-based method as the mean PROMIS Physical Function score change for the group reporting mild improvement and with a distribution-based method considering effect sizes of change and the minimum detectable change (MDC). The MCID estimate was examined for the influence of patient age, follow-up interval, and initial PROMIS score. Change in PROMIS Physical Function scores between visits was significantly different between patients reporting no change, mild improvement, and much improvement on the anchor questions. The anchor-based MCID estimate for PROMIS Physical Function was 3.6 points (SD, 8.4). Among patients reporting mild improvement, individual changes in PROMIS Physical Function were not correlated with patient age or time between visits but were moderately negatively correlated with the initial absolute PROMIS Physical Function score. Applying the effect size parameters to our data when patients indicated minimal change, the distribution-based MCID estimate was 4.6 (SD, 1.8). Both the anchor-based and the distribution-based MCID estimates were judged sufficient because they exceeded the MDC value of 2.3. The MCID value for PROMIS Physical Function is estimated between 3.6 and 4.6 in patients treated nonsurgically for distal radius fractures. Clinical improvement is associated with smaller magnitudes of change on PROMIS Physical Function when patients present with better reported function. The MCID estimations are needed to determine the clinical relevance of changes in PROMIS scores and to more accurately calculate sample sizes needed for research incorporating PROMIS.

Longitudinal validity of the hemophilia caregiver impact measure

IntroductionThe hemophilia caregiver impact (HCI) measure is a 36-item self-report tool that has documented reliability and validity in a large cross-sectional study, but its longitudinal construct validity is unknown. This study’s objective was to evaluate the responsiveness of the HCI to clinically important change, and to provide interpretation guidelines.MethodsThis web-based study invited 458 hemophilia caregivers involved in the HCI’s validation study to provide follow-up data. Measures included the HCI, and a Likert item querying Global Assessment of Change (GAC) for caregiver burden. Responsiveness was estimated using anchor- and distribution-based methods. The anchor-based method computed the minimally important difference (MID) by computing the mean change separately for those who reported lesser or more caregiver burden on the GAC. The distribution-based method computed the Modified Standardized Response Mean (MSRM) separately for people who reporting reduced or increased burden as compared to the ‘same’ groups.ResultsThe study sample included 323 caregivers (71% response rate), with mean follow-up of 21.9 months. The HCI Burden Summary score and all negative-burden subscales but not the Positive Emotions subscale evidenced responsiveness to clinically important differences, showing statistically significant differences by transition group. The MIDs were relatively small mean changes over time (e.g., Burden Summary MID ranged from − 2.2 to 2.6, for reduced versus increased burden), and the MSRMs were small effect sizes. The Burden Summary score was equally sensitive to reduced versus increased burden (MSRM of − 0.32 and 0.35, respectively).ConclusionsThe HCI demonstrated longitudinal construct validity. The HCI shows promise for clinical hemophilia studies as a caregiver-based tool for evaluating treatments.

Determination of Minimum Clinically Important Difference (MCID) in Visual Analog Scale (VAS) Pain and Foot and Ankle Ability Measure (FAAM) Scores After Hallux Valgus Surgery

Minimum clinically important difference (MCID) defines a threshold when determining clinically significant treatment improvement. Visual analog scale (VAS) and Foot and Ankle Ability Measure activities of daily living (FAAM-ADL) are commonly used for measuring hallux valgus correction. This study aimed to determine MCID in VAS pain and FAAM-ADL scores for hallux valgus correction and additionally, to identify variables influencing achievement of the VAS pain MCID. Patients undergoing hallux valgus surgery were retrospectively included. VAS pain, FAAM-ADL, and pain satisfaction surveys were collected preoperatively and minimum 1-year postoperatively. Using a 6-point Likert-type pain satisfaction scale, patients reporting low postoperative satisfaction scores 1 through 3 were categorized as "dissatisfied," and high satisfaction scores 4 through six as "satisfied." One distribution-based method and 2 anchor-based methods were used to calculate MCID. Further, a logistic regression was calculated to determine if one group (defined by sex, pain satisfaction, preoperative VAS pain, concomitant lesser toe deformity correction, and specific hallux valgus correction procedure) had a greater likelihood of achieving the VAS pain MCID threshold. This study included 170 patients with postoperative follow-up averaging 23.6 months. Calculated MCID scores ranged from 1.8 to 5.2 points for VAS pain and 11.1 to 22.7 points for FAAM-ADL. Moderate deformity correction with proximal first metatarsal osteotomy (Ludloff) (OR=2.236, P = .036) or severe deformity correction with first tarsometatarsal arthrodesis (Lapidus) (OR=3.145, P = .046); and higher preoperative pain scores (OR=1.045, P < .010) had significantly higher odds of meeting VAS pain MCID. This study demonstrated MCID values that may indicate significant pain and function improvement after hallux valgus correction. Higher preoperative pain, and utilization of proximal metatarsal osteotomy or first tarsometatarsal arthrodesis for moderate or severe deformity correction resulted in significantly greater likelihood of reaching the VAS pain MCID than utilizing distal metatarsal and/or proximal phalanx osteotomy for mild deformity treatment. Level IV, validating outcome measures.

Preoperative Patient Reported Outcomes Measurement Information System Scores Assist in Predicting Early Postoperative Success in Lumbar Discectomy.

Retrospective review of prospective data. Determine whether patient reported outcome (PRO) data collected prior to lumbar discectomy predicts achievement of a minimal important difference (MID) after surgery. Compare ability of PRO and clinical information to predict achievement of MID in short term follow-up after discectomy. We investigated the ability of patient reported outcomes measurement information system (PROMIS) and clinical factors at the preoperative time point to determine patients achieving MID after surgery. PROMIS physical function (PF), pain interference (PI), and depression (D) scores were assessed at evaluation and follow-up for consecutive visits between February, 2015 and September, 2017. Patients with preoperative scores within 30 days prior to surgery and with scores 40 days or more after surgery who completed all PROMIS domains were included yielding 78 patients. MIDs were calculated using a distribution-based method. A multivariate logistic regression model was created, and the ability to predict achieving MID for each of the PROMIS domains was assessed. Cut-off values and prognostic probabilities were determined for this model and models combining preoperative PROMIS with clinical data. Preoperative PROMIS scores modestly predict reaching MID after discectomy (areas under the curve [AUC] of 0.62, 0.68, and 0.76 for PF, PI, and D, respectively). Preoperative cut-off scores show patients who have PF and PI scores more than 2 standard deviations, and D more than 1.5 standard deviations worse-off than population mean are likely to achieve MID. The combination of PROMIS with clinical data was the most powerful predictor of reaching MID with AUCs of 0.87, 0.84, and 0.83 for PF, PI, and D. PROMIS scores before discectomy modestly predict improvement after surgery. Preoperative PROMIS combined with clinical factors was more predictive of achieving MID than either clinical factors or PROMIS alone. 3.

Longitudinal assessment of exercise capacity and quality of life outcome measures in cystic fibrosis: A year‐long prospective pilot study

Multiple outcomes measures including exercise capacity and quality of life are necessary to get complete and accurate picture of cystic fibrosis (CF) progression. In this pilot study, we investigated these measures in CF longitudinally for a year to determine (a) minimal clinically important difference (MCID) for 6-minute walk distance (6MWD) and CF Health-Related Quality of Life Questionnaire (CFQ-R) domains and (b) how 6MWD, CFQ-R, and spirometry change during times of exacerbation and baseline health and their relationship among each other. Subjects with moderate and severe CF (baseline FEV1 less than 70% predicted) at Akron Children's Hospital CF centre were followed for a full year longitudinally. All three tests (6MWT, CFQ-R, and spirometry) were done during each outpatient visit and weekly during inpatient admission. MCID was estimated for these parameters using distribution-based methods. Finally, data were examined visually using longitudinal graphs for each subject. Twelve CF subjects (eight [67%] males and age range 13-46years) were followed for a full year resulting in at least four encounters per subject. 6MWD (m) and CFQ-R respiratory had an MCID of 33m and 7.3, respectively. MCIDs for FEV1 (percentage predicted) and CFQ-R physical were 7.1 and 11.4, respectively. The longitudinal evaluation of multiple outcome measures during periods of disease exacerbation and baseline health showed that these parameters did not appear to change in accordance with each other. In this pilot study, MCIDs for 6MWD and CFQ-R domains were calculated for the first time to facilitate their use as additional outcome measures in CF. The disparity among multiple outcome measures highlights that these measures together may provide a more complete picture in CF than any single measure alone.

Responsiveness and minimal clinically important difference for the EQ-5D in chronic rhinosinusitis.

The 5-dimensional EuroQol questionnaire (EQ-5D) is validated to measure general health-related quality of life (QOL). Our objective was to determine the responsiveness and minimal clinically important difference (MCID) of the EQ-5D health utility value (EQ-5D HUV) and visual analog scale (EQ-5D VAS) in chronic rhinosinusitis (CRS). 203 adults undergoing medical management for CRS were prospectively recruited. General health-related QOL (using EQ-5D HUV and EQ-5D VAS) and CRS-specific QOL (using the 22-item Sinonasal Outcome Test [SNOT-22]) were measured at enrollment and a subsequent follow-up time point 2-12 months later. At follow-up, participants also rated change in general health as Much worse, A little worse, About the same, A little better or Much better compared to enrollment. The EQ-5D HUV and EQ-5D VAS MCIDs were calculated using distribution-based, anchor-based, and receiver operator characteristic (ROC) curve-based methods. Change in SNOT-22 score was correlated with EQ-5D HUV and EQ-5D VAS change. Using the different methods of calculating MCID, we find the EQ-5D HUV MCID to be 0.04 and EQ-5D VAS MCID to be 8.0. The calculated EQ-5D MCIDs had approximately a sensitivity of 40-50% and specificity of 80% in detecting patients experiencing noticeable improvement in general health. The EQ-5D responds well to changing CRS symptomatology. We propose MCIDs for EQ-5D HUV of 0.04 and EQ-5D VAS of 8 in CRS patients, which although specific, are not sensitive for detecting patients experiencing improvement in general health.