Objective Performance Goals Based on a Systematic Review and Meta-Analysis of Clinical Outcomes for Bare-Metal Stents and Percutaneous Transluminal Angioplasty for Hemodialysis-Related Central Venous Obstruction.

PURPOSE: To use safety and efficacy outcomes following treatment with percutaneous transluminal angioplasty (PTA) and/or stent placement for thoracic central venous obstruction in hemodialysis-dependent patients to establish objective performance goals (OPGs). METHODS: A systematic literature review and meta-analysis were conducted for articles published between January 1, 2000, and August 31, 2021. Efficacy outcomes included primary patency rates at 6 and 12 months, and safety outcomes included adverse events (AEs) categorized as access loss, procedure-related AEs, and serious AEs (SAEs). OPGs were derived from the upper and lower bounds of the 95% confidence intervals for primary patency and SAE rates. RESULTS: Of 66 articles reviewed, 17 met the inclusion criteria (PTA, n = 4; stent placement, n = 5; PTA/stent, n = 8). The 6- and 12-month primary patency rates for PTA were 50.9% and 36.7%, respectively. Based on these findings, the proposed 6- and 12-month primary patency OPGs identifying superiority against PTA were 66.5% and 52.6%, respectively, and those for noninferiority were 39.0% and 25.7%, respectively. For stent placement, the 6- and 12-month primary patency rates were 69.7% and 47.9%, respectively. The proposed 6- and 12-month primary patency OPGs identifying superiority were 82.1% and 64.1%, respectively, and those for noninferiority were 59.3% and 35.8%, respectively. SAE rates for PTA and stent placement were 3.8% and 8.1%, respectively. Proposed safety OPGs for noninferiority versus superiority for PTA and stent placement were 10.1% versus 1.4% and 13.6% versus 4.8%, respectively. CONCLUSION: The OPGs derived from real-world studies of PTA and stent placement may serve as a benchmark for future interventions indicated for this patient population.

A population-based study of scoliosis among males diagnosed with a dystrophinopathy identified by the Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet).

INTRODUCTION/AIMS: Scoliosis is a common comorbidity among individuals diagnosed with a dystrophinopathy. We examined associations between clinical predictors and scoliosis in childhood-onset dystrophinopathy. METHODS: The progression and treatment of scoliosis were obtained from data collected by the US population-based Muscular Dystrophy Surveillance, Tracking, and Research Network. Associations between loss of independent ambulation (LoA) and corticosteroid use and scoliosis outcomes (ages at or exceeding Cobb angle thresholds [10°, 20°, 30°]; surgery) were estimated using Kaplan-Meier curve estimation and extended Cox regression modeling. RESULTS: We analyzed curvature data for 513 of 1054 individuals ascertained. Overall, approximately one-half had at least one radiograph and one-quarter had a curvature of at least 20°. The average maximum curvature was 25.0° (SD = 21.5°) among all individuals and 42.8° (SD = 18.8°) among those recommended for surgery. Higher adjusted hazards ratio of curvature (aHR(curvature) [95% confidence interval]) were found among individuals with LoA compared to those without LoA (aHR(10)  = 6.2 [4.4, 8.7], aHR(20)  = 15.3 [7.4, 31.7], aHR(30)  = 31.6 [7.7, 128.9]), among individuals who did not use corticosteroids compared to those who did (aHR(10)  = 1.2 [0.9, 1.7], aHR(20)  = 1.8 [1.1, 2.7], aHR(30)  = 2.3 [1.3, 4.0]), and among non-ambulatory individuals who used corticosteroids after LoA compared to those who did not (aHR(10)  = 1.8 [1.2, 2.8], aHR(20)  = 1.6 [1.0, 2.6], aHR(30)  = 3.6 [1.6, 7.9]). Scoliosis surgery among individuals with LoA who did not use corticosteroids was more than double compared to those who used (aHR = 2.3 [1.3, 4.2]). DISCUSSION: Our retrospective observational study suggests corticosteroids may delay spinal curvature progression and need for scoliosis surgery. Continuing corticosteroids after LoA also showed potential benefits of delaying curvature progression, additional studies are needed to confirm this finding or address the magnitude of benefit.

Using machine learning to identify factors associated with practice location of the healthcare workforce.

INTRODUCTION: Past studies examined factors associated with rural practice, but none employed newer machine learning (ML) methods to explore potential predictors. The primary aim of this study was to identify factors related to practice in a rural area. Secondary aims were to capture a more precise understanding of the demographic characteristics of the healthcare professions workforce in Utah (USA) and to assess the viability of ML as a predictive tool. METHODS: This study incorporated four datasets - the 2017 dental workforce, the 2016 physician workforce, the 2014 nursing workforce and the 2017 pharmacy workforce - collected by the Utah Medical Education Council. Supervised ML techniques were used to identify factors associated with practice location, the outcome variable of interest. RESULTS: The study sample consisted of 11 259 healthcare professionals with an average age of 46.6 years, of which 36.6% were males and 94.5% Caucasian. Four ML methods were applied to assess model performance by comparing accuracy, sensitivity, specificity and area under the receiver operating characteristic (ROC) curve. Of the methods used, support vector machine performed the best (accuracy 99.7%, precision 100%, sensitivity 100%, specificity 99.4% and ROC 0.997). The models identified income and rural upbringing as the top factors associated with rural practice. CONCLUSION: By far, income emerged as the most important factor associated with rural practice, suggesting that attractive income offers might help rural communities address health professional shortages. Rural upbringing was the next most important predictive factor, validating and updating earlier research. The performance of the ML algorithms suggests their usefulness as a tool to model other databases for individualized prediction.

The Charcot-Marie-Tooth Health Index: Evaluation of a Patient-Reported Outcome.

OBJECTIVE: The development of a disease-specific patient-reported outcome for Charcot-Marie-Tooth disease is an important step in the preparation for therapeutic trials. This study describes the development of the Charcot-Marie-Tooth Health Index (CMTHI). METHODS: Inherited Neuropathy Consortium Contact Registry participants were queried on the symptoms that most impacted their lives. The CMTHI was developed based on these responses. Factor analysis, assessment of test-retest reliability, known group validity, and patient interviews were utilized to refine the instrument. RESULTS: The final CMTHI contains 18 themes that capture Charcot-Marie-Tooth disease (CMT) burden. The CMTHI has a high internal consistency and test-retest reliability. The CMTHI was able to discriminate between patient groups expected to have different disease burden. The CMTHI was able to discriminate levels of disability as measured by the CMT examination score and the mobility-Disability Severity Index. INTERPRETATION: The CMTHI represents a valid and reliable outcome to assess patient-reported disease burden in CMT. Ann Neurol 2018;84:225-233.

Patient-reported study of the impact of pediatric-onset myotonic dystrophy.

INTRODUCTION: The prevalence and impact of symptoms affecting individuals with pediatric forms of myotonic dystrophy type-1 (DM1) are not well understood. METHODS: Patients from the United States, Canada, and Sweden completed a survey that investigated 20 themes associated with pediatric-onset DM1. Participants reported the prevalence and importance of each theme affecting their lives. Surveys from participants were matched with surveys from their caregivers for additional analysis. RESULTS: The most prevalent symptomatic themes included problems with hands or fingers (79%) and gastrointestinal issues (75%). Problems with urinary/bowel control and gastrointestinal issues were reported to have the greatest impact on patients' lives. Responses from participants and their caregivers had varying levels of agreement among symptomatic themes. DISCUSSION: Many symptoms have meaningful impact on disease burden. The highest levels of agreement between caregivers and individuals with pediatric forms of myotonic dystrophy were found for physical activity themes.

Validation of a photophobia symptom impact scale.

BACKGROUND: Routine assessment of photophobia in the clinical setting may underestimate the presence and severity of this condition. We aimed to develop and validate a questionnaire to improve evaluation of the impact of photophobia on activities of daily living, and to determine the relationship of this questionnaire to psychophysical assessment of light sensitivity thresholds. METHODS: We developed the 17-item Utah Photophobia Symptom Impact Scale (UPSIS-17) and compared its psychometric properties to the 8-item Korean Photophobia Questionnaire (KUMC-8). Ninety five subjects with or without light sensitivity completed both questionnaires; 72 also completed laboratory-based assessment of light sensitivity thresholds. We used Rasch analysis to evaluate instrument targeting, including internal consistency and reliability. Correlation analysis was used to assess the relationship between questionnaire scores and light sensitivity thresholds. RESULTS: We observed correlation between UPSIS-17 and KUMC-8, r = 0.72 (p < 0.0001). Higher UPSIS-17 scores correlated with light sensitivity thresholds, r = -0.42 (p < 0.0001), whereas KUMC-8 scores did not significantly correlate with light sensitivity thresholds, r = -0.21 (p = 0.072). UPSIS-17 showed better instrument targeting than KUMC-8 on Rasch analysis. Person-item maps allowed for identification of questions that could be removed without affecting questionnaire validity measures. CONCLUSION: This study resulted in a shortened, 12-item questionnaire. The UPSIS-12 retained significant correlation with both the KUMC-8 and light sensitivity thresholds, yielding a simpler tool for symptom assessment, while retaining validity. This expanded tool may be useful in clinical, as well as research settings, for collection of data about disability due to photophobia.

Minimal Clinically Important Difference After Carpal Tunnel Release Using the PROMIS Platform.

PURPOSE: In light of recently-proposed quality measures for carpal tunnel release (CTR), elucidating the minimal clinically important difference (MCID) for selected outcome measures will be important when interpreting treatment responses. Our purpose was to estimate the MCID of the Patient-Reported Outcomes Measurement Information System (PROMIS) instruments and the short Disabilities of the Arm, Shoulder, and Hand (QuickDASH) following CTR. METHODS: Adult patients undergoing isolated unilateral CTR between July 2014 and October 2016 were identified. Outcomes included the PROMIS Upper Extremity (UE) Computer Adaptive Test (CAT), Physical Function (PF) CAT, QuickDASH, and Pain Interference (PI) CAT. For inclusion, pretreatment baseline (within 60 days of surgery) and postoperative (6-90 days) UE or PF CAT scores were required, as well as a response on a 5-point Likert scale to the question "How much relief and/or improvement do you feel you have experienced as a result of your treatment?" The MCID was calculated using SD and minimum detectable change (MDC) distribution methods. RESULTS: In response to the Likert scale question, 88.6% of patients reported improvement at a mean of 14.8 days after surgery. The infrequency of patients reporting no change (5 of 44; 11.4%) precluded calculation of a statistically sound anchor-based MCID value. The MCID values, as calculated using the one-half SD method, were 3.6, 4.6, 10.4, and 3.4 for the UE CAT, PF CAT, QuickDASH, and PI CAT, respectively. CONCLUSIONS: We have calculated MCID values for the UE CAT, PF CAT, QuickDASH, and PI CAT for patients undergoing CTR. Although the small number of patients reporting no change and minimal change after surgery precluded an anchor-based MCID calculation, we report estimates using the one-half SD method for the MCID following CTR. CLINICAL RELEVANCE: These MCID estimates will be helpful when interpreting CTR clinical outcomes and for powering prospective trials.

Evaluation of Version 2.0 of the PROMIS Upper Extremity Computer Adaptive Test in Nonshoulder Upper Extremity Patients.

PURPOSE: The Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity Computer Adaptive Test (UE CAT) was recently updated to version 2.0 (v2.0). We hypothesized that the PROMIS UE CAT v2.0 would exhibit improved performance characteristics compared with the Quick Disabilities of the Arm, Shoulder, and Hand (QuickDASH) when administered to a nonshoulder upper extremity patient population. METHODS: The UE CAT v2.0, Physical Function (PF) CAT v2.0, and the QuickDASH were each prospectively administered via tablet computer to all patients presenting to a tertiary hand and upper extremity clinic between April 2017 and October 2017. Patient responses were analyzed, and the mean, range, floor and ceiling effect, and correlations between instruments were calculated. RESULTS: Among 825 patients, the mean UE CAT v2.0 score was 38.3 (SD 10.7) with a range of 15 to 61 and interquartile range of 15.4. The UE CAT v2.0 had a strong correlation with the QuickDASH (r = -0.749) and the PF CAT v2.0 (r = 0.719). No patient scored between 56 and 60, indicating a gap in scoring in that range. The UE CAT v2.0 demonstrated a floor effect of 1%, a ceiling effect of 6.9%, and a high internal consistency with a Cronbach alpha of 0.99. CONCLUSIONS: The PROMIS UE CAT v2.0 demonstrated improved ceiling effects, range, and a decreased gap in scoring compared with prior versions. Limitations of the PROMIS UE CAT v2.0 are still present, but updates have led to an incremental improvement over prior versions, demonstrating the ability to influence PROMIS instrument performance through upgrades. CLINICAL RELEVANCE: The updated PROMIS UE CAT v2.0 still demonstrates a ceiling effect and gap in scores at the upper end of the instrument, both of which may limit discrimination between different levels of upper extremity function for high-functioning patients.

Psychometric evaluation of the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function and Pain Interference Computer Adaptive Test for subacromial impingement syndrome.

BACKGROUND: The Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Computer Adaptive Test (CAT) was previously validated for rotator cuff disease and shoulder instability. This study evaluated the psychometric properties of the PROMIS Physical Function (PF) CAT, PROMIS Pain Interference (PI) CAT, and the American Shoulder and Elbow Surgeons (ASES) Shoulder Function Score for subacromial impingement syndrome. METHODS: PROMIS PF CAT, PI CAT, and ASES (Pain, Function, Total) were collected on all visits for 2 surgeons between January 2016 and August 2016. New patients, aged 18 years and older, were selected by International Classification of Diseases code for impingement syndrome of the shoulder. The mean number of questions answered determined efficiency. Person-item maps were created to determine ceiling and floor effects as well as person reliability. Convergent validity was determined by comparison of PROMIS domains to ASES scores with Pearson correlations. RESULTS: For PROMIS PF CAT, the mean number of items answered was 4.54 (range 4-12). The ceiling effect was 1.56%, and the floor effect was 3.13%. The person reliability was 0.94. Pearson correlation coefficients between the PF CAT and ASES were 0.664 (ASES Function), 0.426 (ASES Pain), and 0.649 (ASES Total). For PROMIS PI CAT, the mean number of items answered was 4.27 (range 3-11). The ceiling effect was 4.69%, and the floor effect was 8.33%. The person reliability was 0.92. Pearson correlation coefficients between the PI CAT and ASES were: 0.667 (ASES Function), 0.594 (ASES Pain), and 0.729 (ASES Total). CONCLUSIONS: The psychometric properties of PROMIS PF and PI CATs were favorable for subacromial impingement syndrome.

Application of machine learning for diagnostic prediction of root caries.

OBJECTIVE: This study sought to utilise machine learning methods in artificial intelligence to select the most relevant variables in classifying the presence and absence of root caries and to evaluate the model performance. BACKGROUND: Dental caries is one of the most prevalent oral health problems. Artificial intelligence can be used to develop models for identification of root caries risk and to gain valuable insights, but it has not been applied in dentistry. Accurately identifying root caries may guide treatment decisions, leading to better oral health outcomes. METHODS: Data were obtained from the 2015-2016 National Health and Nutrition Examination Survey and were randomly divided into training and test sets. Several supervised machine learning methods were applied to construct a tool that was capable of classifying variables into the presence and absence of root caries. Accuracy, sensitivity, specificity and area under the receiver operating curve were computed. RESULTS: Of the machine learning algorithms developed, support vector machine demonstrated the best performance with an accuracy of 97.1%, precision of 95.1%, sensitivity of 99.6% and specificity of 94.3% for identifying root caries. The area under the curve was 0.997. Age was the feature most strongly associated with root caries. CONCLUSION: The machine learning algorithms developed in this study perform well and allow for clinical implementation and utilisation by dental and nondental professionals. Clinicians are encouraged to adopt the algorithms from this study for early intervention and treatment of root caries for the ageing population of the United States, and for attaining precision dental medicine.

Assessing spousal support and health in an aging population: support and strain amidst changing social dynamics.

This study examined the role of relationship quality on physical and psychological health among older adults. It included 2,298 adults aged 50 and older who participated in the Midlife in the US national longitudinal study of health and well-being. We assessed the effect of spousal support and strain on psychological and physical health, controlling for age, education, income, depression levels and prior health. Results indicated that spousal support and strain affected psychological health but not physical health. Despite prior research showing an association between marital quality and physical health, this study did not support the conceptualization that relationship quality measured by spousal support or strain has a direct effect on long-term health in this sample of older adults. This study does not preclude the presence of a mediated or moderated association between relationship quality and physical health. Higher levels of spousal support are associated with positive psychological health among adults over age 50 while spousal strain is associated with negative psychological health. This study supports the premise that relationship quality has an ongoing impact on the psychological health of mature adults, bolstering arguments to include psychological health screening and couples relationship education among health services provided to older adults.

Orofacial strength, dysarthria, and dysphagia in congenital myotonic dystrophy.

INTRODUCTION: Herein we present an exploratory study of orofacial function in children with congenital myotonic dystrophy (CDM) vs. healthy controls. METHODS: We evaluated 41 children with CDM and 29 healthy controls for speech and swallow function and for lingual and labial strength. RESULTS: The Iowa Oral Performance Instrument (IOPI), measuring tongue strength, and a lip force meter (LFM), measuring lip strength, had excellent interrater reliability with intraclass correlation coefficients (ICCs) of 0.75 (n = 19, P < 0.001) and 0.96 (n = 20, P < 0.001), respectively. Mean overall lingual strength was 3.5-fold less and labial strength was about 7-fold less in CDM patients than in healthy controls. Eighteen of 24 children with CDM demonstrated dysarthria and an additional 11 participants were nonverbal. Dysarthria correlated moderately with lingual strength, age, and dysphagia. Strength measures correlated moderately with dysphagia. DISCUSSION: Children with CDM have impaired orofacial functioning that affects communication and swallowing. Reliability of strength measures may be useful for future therapeutic trials. Muscle Nerve 58: 413-417, 2018.

Modified dynamic gait index and limits of stability in myotonic dystrophy type 1.

INTRODUCTION: The purpose of this study was to describe and compare the performance of balance and walking tests in relation to self-reported fall history in adults with myotonic dystrophy type 1 (DM1). METHODS: Twenty-two (13 male) participants with DM1 completed, a 6-month fall history questionnaire, the modified Dynamic Gait Index (mDGI), limits of stability (LoS) testing, and 10-m walking tests. RESULTS: Mean (SD) falls in 6 months was 3.7 (3.1), and 19 (86%) participants reported at least 1 fall. Significant differences in mDGI scores (P = 0.006) and 10-m fast walking gait velocity (P = 0.02) were found between those who had been classified as "fallers" and those who had been classified as "nonfallers." Significant correlations were found between mDGI scores and 10-m walking time. DISCUSSION: Falls are common in DM1, and the mDGI may have potential to distinguish fallers from nonfallers, whereas the LoS failed to detect such impairment. Future studies should further explore use of the mDGI in DM1. Muscle Nerve 58: 694-699, 2018.

The Relationship of PROMIS Pain Interference and Physical Function Scales.

Objectives: To examine the relationship between the Patient Reported Outcome Measurement Information System (PROMIS) Pain Interference (PI) and PROMIS Physical Function (PF) scales in patients with spinal pain at a university spine center. Design: Retrospective analysis of prospectively collected patient-reported outcome data at a university spine clinic. Pearson correlation was done to examine the relationship of the PROMIS PF and PROMIS PI scores. Age, gender, and race were analyzed by subgroups on the PROMIS Physical Function and Pain Interference score. Linear regression analyzed predictive relationships. Statistical significance was set at P < 0.05. Results: A total of 1,992 participants completed an assessment, with 1,923 completing the PF CAT and 1,927 the PI CAT. Participants' mean age was 52.8 years (range = 18-94 years, SD = 6.5 years). Correlation analysis of the PROMIS PF with the PROMIS PI showed a Pearson correlation value of -0.717 (P < 0.05). There was a strong linear relationship with a high negative correlation between PF CAT and PI CAT. The PI CAT predicted PF CAT scores (ß = -0.707, P < 0.001). Conclusions: For patients with pain from spinal origin, there is a strong negative correlation between self-reported physical function and pain interference related to physical, social, and mental health. The predictive relationship of function from pain scores supports the PROMIS PI being used as an important adjunct measure of physical function in patients with spinal pain.

What Are the MCIDs for PROMIS, NDI, and ODI Instruments Among Patients With Spinal Conditions?

BACKGROUND: As new Patient-Reported Outcomes Measurement Information System (PROMIS) instruments are incorporated into clinical practice, determining how large a change on these instruments represents a clinically relevant difference is important; the metric that describes this is the minimum clinically important difference (MCID). Prior research on MCIDs of the Neck Disability Index (NDI) and Oswestry Disability Index (ODI) has produced values ranging from 5 to 10 points, but these measures have not been presented in relation to MCID values of PROMIS instruments. QUESTIONS/PURPOSES: To establish a comprehensive repository of MCID values calculated both with distribution-based and anchor-based methods for four outcomes instruments in spine care, we asked: (1) What are the MCIDs of the PROMIS Physical Function (PF); (2) the PROMIS Pain Interference (PI); (3) the NDI; and (4) the ODI among spine patients? METHODS: We conducted a prospective study of previously tested diagnostic measures on 1945 consecutive patients with a reference standard applied. All patients aged 18 years and older visiting an orthopaedic spine clinic between October 2013 and January 2017 completed the PROMIS PF and PI, NDI, and ODI on tablet computers before their clinic visits. Patients were grouped by change level (self-report of meaningful change versus slight or no change) using an anchor question in comparison to baseline. Descriptive statistics, two anchor-based MCID values (mean change and receiver operating characteristic curve), and five distribution-based values (SD at 1/2 and 1/3 values and minimum detectable change [MDC] at 90%, 95%, and 99%) were analyzed four different times between 3 months and > 6 months of followup. A total of 1945 included patients with a wide range of spine conditions and varying treatments had a mean age of 58 years (SD = 15.5), were 51% (988 of 1945) male, 90% (1754 of 1945) self-identified as white, and 5% (94 of 1945) as Hispanic with 1% to 2% of patients refusing participation. RESULTS: The PROMIS PF mean change scores in the changed group (much worse, worse, improved, or much improved) ranged between 7 and 8 points. MCID values ranged from 3 to 23 points depending on the method of calculation with a median of 8. For the PROMIS PI, mean change scores ranged from 8 to 9 points and MCID values from 1 to 24 points with a median of 8. For the NDI, mean change scores ranged from 13 to 18 points and MCID values ranged from 6 to 43 points with a median of 18. For the ODI, mean change ranged from 17 to 19 points and MCID values ranged from 7 to 51 points with a median of 24. For each instrument, distribution-based SD yielded the smallest values, followed by anchor-based methods, with MDC yielding the largest MCID values. CONCLUSIONS: This study uses a range of methods for determining MCIDs of the PROMIS PF and PI, NDI, and ODI from anchor-based to distribution-based methods. MCIDs do not have a static value for a given outcome measure, but have a range of values and are dependent on the method calculated. The lowest MCIDs identified for the NDI and ODI are consistent with prior studies, but those at the upper range are much higher. Anchor-based methods are thought to be most relevant in the clinical setting and are more easily understood by clinicians, whereas the distribution-based MCIDs are useful in understanding population breadth. Lower MCID values may be most appropriate for screening purposes or low-risk effects, and the median or higher MCID values should be used for high-risk effects or outcomes. LEVEL OF EVIDENCE: Level I, diagnostic study.

Psychometrics of the Patient-Reported Outcomes Measurement Information System Physical Function instrument administered by computerized adaptive testing and the Disabilities of Arm, Shoulder and Hand in the orthopedic elbow patient population.

BACKGROUND: This study evaluated the psychometric properties of the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) instrument administered through computerized adaptive testing (CAT) compared with the traditional full-length Disabilities of the Arm, Shoulder and Hand (DASH). METHODS: The PROMIS PF CAT and the DASH were administered to 1759 patients seeking care for elbow conditions. This study used Rasch partial credit modeling to analyze the instruments with item fit, internal reliability, response category thresholds, dimensionality, local independence, gender differential item functioning, and floor and ceiling effects. RESULTS: The PROMIS PF CAT and DASH had satisfactory item fit for all but 1 item on both measures. Internal reliabilities were high for both measures. Two items on the DASH and 4 items on the PF CAT showed nonordered category thresholds. Unidimensionality was adequate, and local independence was supported for both instruments. Gender bias was found for 4 items on the PF CAT and 12 items on the DASH. Both measures had adequate instrument targeting and satisfactory floor and ceiling effects. CONCLUSION: The PROMIS PF CAT and the DASH both showed sufficient unidimensionality, good item fit, and good local independence with the exception of high levels of gender item bias, particularly for the DASH. Further scale evaluation should address item bias and item response categories for these instruments. Overall, the PROMIS PF CAT is an effective outcome instrument to measure function in patients with elbow disorders that requires significantly fewer questions than the DASH.

Profiling Arthritis Pain with a Decision Tree.

BACKGROUND: Arthritis is the leading cause of work disability and contributes to lost productivity. Previous studies showed that various factors predict pain, but they were limited in sample size and scope from a data analytics perspective. OBJECTIVES: The current study applied machine learning algorithms to identify predictors of pain associated with arthritis in a large national sample. METHODS: Using data from the 2011 to 2012 Medical Expenditure Panel Survey, data mining was performed to develop algorithms to identify factors and patterns that contribute to risk of pain. The model incorporated over 200 variables within the algorithm development, including demographic data, medical claims, laboratory tests, patient-reported outcomes, and sociobehavioral characteristics. RESULTS: The developed algorithms to predict pain utilize variables readily available in patient medical records. Using the machine learning classification algorithm J48 with 50-fold cross-validations, we found that the model can significantly distinguish those with and without pain (c-statistics = 0.9108). The F measure was 0.856, accuracy rate was 85.68%, sensitivity was 0.862, specificity was 0.852, and precision was 0.849. CONCLUSION: Physical and mental function scores, the ability to climb stairs, and overall assessment of feeling were the most discriminative predictors from the 12 identified variables, predicting pain with 86% accuracy for individuals with arthritis. In this era of rapid expansion of big data application, the nature of healthcare research is moving from hypothesis-driven to data-driven solutions. The algorithms generated in this study offer new insights on individualized pain prediction, allowing the development of cost-effective care management programs for those experiencing arthritis pain.

Physical function and mobility in children with congenital myotonic dystrophy.

INTRODUCTION: Congenital myotonic dystrophy (CDM) occurs when symptoms of myotonic dystrophy present at birth. In this study we evaluated the relationship between physical function, muscle mass, and age to provide an assessment of the disease and help prepare for therapeutic trials. METHODS: CDM participants performed timed functional tests (TFTs), the first 2 minutes of 6-minute walk tests (2/6MWTs), and myometry tests, and also performed dual-energy X-ray absorption (DEXA) scans. Healthy controls (HCs) performed TFTs, 6MWTs, and myometry. RESULTS: Thirty-seven children with CDM and 27 HCs (age range 3-13 years) participated in the study. There were significant differences in the 10-meter walk (11.3 seconds in CDM vs. 6.8 seconds in HC) and 2MWT (91 meters in CDM vs. 193 meters in HCs). DEXA lean mass of the right arm correlated with grip strength (r = 0.91), and lean mass of the right leg correlated with 6MWT (r = 0.62). CONCLUSION: Children with CDM have significant limitations in strength and mobility. The tests performed were reliable, and lean muscle mass may serve as a useful biomarker. Muscle Nerve 56: 224-229, 2017.

Reliability Testing of the Balance Error Scoring System in Children Between the Ages of 5 and 14.

OBJECTIVE: Pediatric concussion is an extensive public health concern with a complex clinical presentation. Balance assessment has been well-studied in the adult population, but has been limited in children. We aimed to assess the reliability and minimum detectable change (MDC) of the Balance Error Scoring System (BESS) in healthy children. DESIGN: This prospective observational study included 373 healthy children aged 5 to 14. Interrater reliability was assessed by having 4 assessors review videos of 50 random subjects distributed evenly by age and sex across the entire cohort. Intrarater reliability was performed by having assessors review videos of other assessors' live evaluations. Test-retest reliability was obtained by comparing BESS scores as recorded live at the 2 separate time points by the same rater. SETTING: Local elementary and junior high schools. PARTICIPANTS: Three hundred and seventy three healthy children between the ages of 5 and 14. INTERVENTIONS: The BESS was performed on all children. MAIN OUTCOME MEASURES: Intraclass Correlation Coefficients (ICCs) and MDC Scores. RESULTS: The overall interrater ICC was determined to be 0.93 [95% confidence interval (CI), 0.79-0.97] and intrarater ICC was 0.96 (95% CI, 0.95-0.97) with individual intrarater ICCs ranging between 0.69 and 0.99. The test-retest reliability was 0.90 (95% CI, 0.88-0.92). The MDCs were 9.6, 4.6, and 7.3 points at the 95% CIs for interrater, intrarater, and test-retest comparisons, respectively. No learning effect was seen. CONCLUSIONS: The BESS demonstrates excellent reliability in the pediatric population without evidence of a learning effect.

Association of Physical Function, Anxiety, and Pain Interference in Nonshoulder Upper Extremity Patients Using the PROMIS Platform.

PURPOSE: The relationship between psychosocial factors and self-reported physical function among hand and upper extremity patients is complex. The Patient-Reported Outcomes Measurement Information System (PROMIS) platform has attempted to create a variety of specifically targeted metrics that can be administered using computer adaptive testing (CAT). Three metrics measuring self-reported physical function (herein referred to in combination as "functional" metrics) include the PROMIS Physical Function (PF) CAT, PROMIS Upper Extremity (UE) CAT, and the Quick Disabilities of the Arm, Shoulder, and Hand (QuickDASH). Two metrics assessing psychosocial factors include the PROMIS Anxiety and Pain Interference (PI) CATs ("nonfunctional" metrics). This study evaluates whether the functional metrics were correlated with nonfunctional metrics. METHODS: The 5 questionnaires were administered prospectively on a tablet computer to all consecutive adult patients presenting to an outpatient hand and upper extremity (nonshoulder) clinic at a tertiary academic medical center from January 1 to November 1, 2014. For patients with multiple visits during the study period, only the first was included. Data were evaluated retrospectively to assess the relationship between functional and nonfunctional measures, with Pearson correlation coefficients to understand the relationship between continuous variables, and 1-way analysis of variance to examine for differences in outcome measures across demographic groups. Multivariable linear regression analyses were performed to determine factors predicting functional disability. RESULTS: We included 1,299 patients: mean age was 46.8 years, 53% were female, and 23% were unemployed or on disability. The PROMIS PF CAT, PROMIS UE CAT, and QuickDASH scores were all significantly correlated with PROMIS Anxiety CAT (Pearson correlation coefficients, -0.46, -0.48, and 0.53, respectively) and PROMIS PI CAT (-0.60, -0.65, and 0.76, respectively) scores. Multivariable regression analyses demonstrated that increased PROMIS Anxiety and PI CAT scores each independently and adversely influenced PROMIS PF CAT, PROMIS UE CAT, and QuickDASH scores. CONCLUSIONS: Increasing levels of patient anxiety and pain interference are independently associated with decreased patient-reported upper extremity function. CLINICAL RELEVANCE: This study provides further support of the biopsychosocial model by highlighting that increased anxiety is associated with decreased self-reported function using the PROMIS platform.