Preventable harm because of outpatient medication errors among children with leukemia and lymphoma: A multisite longitudinal assessment.

BACKGROUND: There is little longitudinal information about the type and frequency of harm resulting from medication errors among outpatient children with cancer. We aimed to characterize rates and types of medication errors and harm to outpatient children with leukemia and lymphoma over 7 months of treatment. METHODS: We recruited children taking medications at home for leukemia or lymphoma from three pediatric cancer centers. Errors were identified by chart review, in-home medication review, observation of administration, and interviews. Physician reviewers confirmed error (Fleiss' κ = 0.95), harm (Fleiss' κ = 0.82), and suggested interventions. Generalized linear mixed models with random effects were used to account for clustering by site. RESULTS: Among 131 children taking 1669 medications with 367 home visits, 408 errors were identified, including 242 with potential for harm and 39 with harm (1.0 harm per 1000 patient-days [95% CI, 0.1-9.8]). Ten percent of children were injured by errors and 42% had errors with potential for harm. Twenty-six percent of caregivers reported that miscommunication led to missed doses or overdoses at home. Children on >13 medications had significantly more serious medication errors than those on fewer medications (77% vs 61%; p = .05). Physician reviewers judged that improved communication among caregivers and between caregivers and clinicians may have prevented the most harm (66%). CONCLUSIONS: In this longitudinal study, 10% children with leukemia or lymphoma experienced adverse drug events because of outpatient medication errors. Improvements addressing communication with and among caregivers should be codeveloped with families and based on human-factors engineering. PLAIN LANGUAGE SUMMARY: In this longitudinal study, medication errors in the clinic, pharmacy, or at home among children with leukemia or lymphoma over a 7-month period were common, and 10% suffered harm because of errors. Children on >13 medications had significantly more serious medication errors than those on fewer medications (77% vs 61%; p = .05). Physician reviewers judged that improved communication among caregivers and between caregivers and clinicians may have prevented the most harm (66%). Improvements addressing communication with and among caregivers should be codeveloped with families and based on human-factors engineering.

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

OBJECTIVES: To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. STUDY DESIGN: We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. RESULTS: Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. CONCLUSIONS: This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

A Medication Adherence Promotion System to Reduce Late Kidney Allograft Rejection: A Quality Improvement Study.

RATIONALE & OBJECTIVE: Adolescent and young adult kidney transplant recipients have a high risk of rejection related to suboptimal adherence. Multicomponent interventions improve adherence in controlled trials, but clinical implementation is lacking. We describe an initiative to reduce allograft rejection using evidence-based adherence promotion strategies. STUDY DESIGN: Interrupted time series. SETTING & PARTICIPANTS: Kidney transplant recipients cared for at Cincinnati Children's Hospital ≥ 1 year after transplant and taking ≥1 immunosuppressive medication(s) from 2014 through 2017. QUALITY IMPROVEMENT ACTIVITIES: The following interventions, collectively called MAPS (Medication Adherence Promotion System), were implemented over 14 months: (1) adherence promotion training for clinical staff, 2) electronic health record-supported adherence risk screening, (3) systematic assessment of medication adherence barriers, (4) designation of specific staff to address adherence barriers, (5) shared decision-making with the patients to overcome adherence barriers, (6) follow-up evaluation to assess progress, and (7) optional electronic medication monitoring. OUTCOMES: Primary Outcome: Late acute rejection. Process measures were conducted to assess barriers, identify barriers, and perform interventions. The secondary outcomes/balancing measures were de novo donor-specific antibodies (DSA), biopsy rate, and rejections per biopsy. ANALYTICAL APPROACH: Time series analysis using statistical process control evaluated patient-days between acute rejections as well as monthly rejections per 100 patient-months before and after implementation. To control for known rejection risk factors including changes in treatment and case mix, multivariable analyses were performed. RESULTS: The monthly rejection rate fell from 1.61 rejections per 100 patient-months in the 26 months before implementation to 0.88 rejections per 100 patient-months in the 22 months after implementation. In the multivariable analysis, MAPS was associated with a 50% reduction in rejection incidence (incidence rate ratio, 0.50 [95% CI, 0.27-0.91]; P = 0.02). DSA and time since transplant (per each additional year) were also associated with rejection incidence (incidence rate ratio, 2.27 [P = 0.02] and 0.87 [P = 0.02], respectively). LIMITATIONS: Single-center study, and potential confounding by unmeasured variables. CONCLUSIONS: Clinical implementation of evidence-based adherence-promotion strategies was associated with a 50% reduction in acute rejection incidence over 2 years.

The Mental and Physical Health of Mothers of Children with Special Health Care Needs in the United States.

OBJECTIVE: To determine the prevalence of poor mental and physical health among mothers of children with special health care needs (CSHCN) and to determine the association between maternal health and the child's number of special health care needs (SHCN) and severity of ability limitation. METHODS: We used the combined 2016-2018 National Survey of Children's Health Dataset of 102,341 children ages 0-17 including 23,280 CSHCN. We used regression models to examine the associations of a child's number of SHCN and ability limitations with maternal health. RESULTS: Twice as many mothers of CSHCN had poor mental and physical health compared to non-CSHCN (mental 10.3% vs. 4.0%, p < .001; physical 11.9% vs 5.0%, p < .001). In regression models, increased number of SHCN and severity of activity limitations were associated with significantly increased odds of poor maternal health. CONCLUSIONS FOR PRACTICE: Mothers of CSHCN have worse health compared to mothers of non-CSHCN, especially those who experience social disadvantage and those with children with complex SHCN or severe ability limitations. Interventions to improve the health of these particularly vulnerable caregivers of CSHCN are warranted.

Predicting acute rejection in children, adolescents, and young adults with a kidney transplant by assessing barriers to taking medication.

BACKGROUND: Nonadherence to immunosuppression is common among pediatric, adolescent, and young adult kidney transplant recipients and a leading cause of graft loss. Assessing barriers to medication adherence in clinical practice may identify patients at risk for rejection and provide therapeutic targets. METHODS: Kidney transplant patients and/or their caregivers were assessed for 14 barriers to medication adherence using the barriers assessment tool. We compared rejection rates between patients with at least one reported adherence barrier to those without reported adherence barriers using a Kaplan-Meier estimator and Cox proportional hazard models to adjust for other mediators of acute rejection at 2 years following barriers assessment. RESULTS: Ninety-eight patients were assessed for barriers to adherence. Over the 2-year observation period, 22 patients developed biopsy-proven acute rejection (BPAR). Kaplan-Meier estimates show that patients with an identified barrier to adherence were more likely to have BPAR (p = 0.02) than patients without an identified barrier in the 24 months following barriers assessment. The median time to rejection for patients who experienced acute rejection was 175.5 days (IQR 63-276 days) from the time of barriers assessment. An identified barrier to adherence remained the only statistically significant predictor of BPAR with Cox modeling (HR 2.6, p = 0.04), after accounting for age, sex, and race. CONCLUSIONS: Pediatric and adolescent kidney transplant recipients with identified adherence barriers are at increased risk for acute rejection. Barriers to adherence provide a potentially modifiable therapeutic target that can be assessed in clinic to guide targeted interventions.

Understanding Variation in Longitudinal Data Using Latent Growth Mixture Modeling.

OBJECTIVE: This article guides researchers through the process of specifying, troubleshooting, evaluating, and interpreting latent growth mixture models. METHODS: Latent growth mixture models are conducted with small example dataset of N = 117 pediatric patients using Mplus software. RESULTS: The example and data show how to select a solution, here a 3-class solution. We also present information on two methods for incorporating covariates into these models. CONCLUSIONS: Many studies in pediatric psychology seek to understand how an outcome changes over time. Mixed models or latent growth models estimate a single average trajectory estimate and an overall estimate of the individual variability, but this may mask other patterns of change shared by some participants. Unexplored variation in longitudinal data means that researchers can miss critical information about the trajectories of subgroups of individuals that could have important clinical implications about how one assess, treats, and manages subsets of individuals. Latent growth mixture modeling is a method for uncovering subgroups (or "classes") of individuals with shared trajectories that differ from the average trajectory.

Using nationally representative percentiles to interpret PROMIS pediatric measures.

PURPOSE: This study's aim was to use a representative sample of the US pediatric population to estimate percentiles for several PROMIS pediatric measures: Anger, Anxiety, Depressive Symptoms, Family Relationships, Fatigue, Global Health, Life Satisfaction, Meaning and Purpose, Pain Behavior, Pain Interference, Physical Activity, Physical Function Mobility, Physical Function Upper Extremity, Physical Stress Experiences, Positive Affect, Psychological Stress Experiences, Sleep Disturbance, Sleep Impairment, and Peer Relationships. METHODS: We used two separate, nationally representative samples of parents and children aged 5-17 years drawn in different years from the GfK Knowledge Panel, a dual-frame online probability panel. RESULTS: All measures that were developed using a representative sample had a median at or near the expected value of 50. For the other measures, the 50th percentile was often 10 points or more from 50. Several domains had high floors or low ceilings. No domain's percentiles completely corresponded to the percentiles associated with a normal distribution with a mean of 50 and standard deviation of 10. CONCLUSIONS: This work allows users to interpret a child's self-reported quality of life relative to children in the US general population. When attempting to evaluate whether a child falls above or below other children in the US, one should use the values presented in this study. In addition, we recommend that users should focus on whether a child's score falls into one of a few broad severity groups rather than on specific percentile scores.

Differences in Reporting Child Health Needs and Status and Developmental Concerns between Mothers and Fathers: Findings from the National Survey of Children's Health.

Nationally representative data of 140 000 children in 2-parent households showed that fathers were more likely than mothers to report that a child was in good health and less likely to report the presence of a specific health condition, special health care needs, or unmet health service needs.

A phase I/II study of ribociclib following radiation therapy in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG).

PURPOSE: Cyclin-dependent kinase-retinoblastoma (CDK-RB) pathway is dysregulated in some diffuse intrinsic pontine gliomas (DIPG). We evaluated safety, feasibility, and early efficacy of the CDK4/6-inhibitor ribociclib, administered following radiotherapy in newly-diagnosed DIPG patients. METHODS: Following radiotherapy, eligible patients received ribociclib in 28-day cycles (350 mg/m2; 21 days on/7 days off). Feasibility endpoints included tolerability for at least 6 courses, and a less than 2-week delay in restarting therapy after 1 dose reduction. Early efficacy was measured by 1-year and median overall survival (OS). Patient/parent-by-proxy reported outcomes measurement information system (PROMIS) assessments were completed prospectively. RESULTS: The study included 10 evaluable patients, 9 DIPG and 1 diffuse midline glioma (DMG)-all 3.7 to 19.8 years of age. The median number of courses was 8 (range 3-14). Three patients required dose reduction for grade-4 neutropenia, and 1 discontinued therapy for hematological toxicity following course 4. The most common grade-3/4 toxicity was myelosuppression. After 2 courses, MRI evaluations in 4 patients revealed increased necrotic volume, associated with new neurological symptoms in 3 patients. The 1-year and median OS for DIPG was 89% and 16.1 months (range 10-30), respectively; the DMG patient died at 6 months post-diagnosis. Five patients donated brain tissue and tumor; 3 were RB+ . CONCLUSIONS: Ribociclib administered following radiotherapy is feasible in DIPG and DMG. Increased tumor necrosis may represent a treatment effect. These data warrant further prospective volumetric analyses of tumors with necrosis. Feasibility and stabilization findings support further investigation of ribociclib in combination therapies. TRIAL REGISTRATION: NCT02607124.

Understanding the Role of Past Health Care Discrimination in Help-Seeking and Shared Decision-Making for Depression Treatment Preferences.

As a part of a larger, mixed-methods research study, we conducted semi-structured interviews with 21 adults with depressive symptoms to understand the role that past health care discrimination plays in shaping help-seeking for depression treatment and receiving preferred treatment modalities. We recruited to achieve heterogeneity of racial/ethnic backgrounds and history of health care discrimination in our participant sample. Participants were Hispanic/Latino (n = 4), non-Hispanic/Latino Black (n = 8), or non-Hispanic/Latino White (n = 9). Twelve reported health care discrimination due to race/ethnicity, language, perceived social class, and/or mental health diagnosis. Health care discrimination exacerbated barriers to initiating and continuing depression treatment among patients from diverse backgrounds or with stigmatized mental health conditions. Treatment preferences emerged as fluid and shaped by shared decisions made within a trustworthy patient-provider relationship. However, patients who had experienced health care discrimination faced greater challenges to forming trusting relationships with providers and thus engaging in shared decision-making processes.

Marked Variation Exists Among Surgeons and Hospitals in the Use of Secondary Cleft Lip Surgery.

OBJECTIVE: To identify child-, surgeon-, and hospital-specific factors at the time of primary cleft lip repair that are associated with the use of secondary cleft lip surgery. DESIGN: Retrospective cohort study. SETTING: Forty-nine pediatric hospitals. PARTICIPANTS: Children who underwent cleft lip repair between 1999 and 2015. MAIN OUTCOME MEASURE: Time from primary cleft lip repair to secondary lip surgery. RESULTS: By 5 years after primary lip repair, 24.0% of children had undergone a secondary lip surgery. In multivariable analysis, primary lip repair before 3 months had a 1.22-fold increased hazard of secondary surgery (95% confidence interval [CI]: 1.02-1.46) compared to repair at 7 to 12 months of age, and children with multiple congenital anomalies had a 0.77-fold decreased hazard of secondary surgery (95% CI: 0.68-0.87). After adjusting for cleft type, age at repair, presence of multiple congenital anomalies, and procedure volume, there remained substantial variation in secondary surgery use among surgeons and hospitals (P < .01). For children with unilateral cleft lip repaired at 3 to 6 months of age, the predicted proportion of children undergoing secondary surgery within 5 years of primary repair ranged from 4.9% to 21.8% across surgeons and from 4.5% to 24.7% across hospitals. CONCLUSIONS: There are substantial differences among surgeons and hospitals in the rates of secondary lip surgery. Further work is needed to identify causes for this variation among providers.

Assessing Children's Eudaimonic Well-Being: The PROMIS Pediatric Meaning and Purpose Item Banks.

OBJECTIVE: To describe the development of the Patient-Reported Outcome Measurement Information System (PROMIS) Pediatric Meaning and Purpose item banks, child-report and parent-proxy editions. METHODS: Data were collected from two samples. The first comprised 1,895 children (8-17 years old) and 927 parents of children 5-17 years old recruited from an Internet panel, medical clinics, and schools. The second comprised a nationally representative sample of 990 children 8-17 years old and 1,292 parents of children 5-17 years old recruited from a different Internet panel. Item pool evaluation was done with Sample 1 and analyses were used to support decisions about item retention. The combined sample was used for item response theory (IRT) calibration of the item bank. Both samples were used in validation studies. RESULTS: Eleven items were deleted from the item pool because of poor psychometric performance. The final versions of the scales showed excellent reliability (>0.90). Short form scales (4 or 8 items) had a high degree of precision across over 4 SD units of the latent variable. The item bank positively correlated with extant measures of positive psychological functioning, and negatively correlated with measures of emotional distress, pessimism, and pain. Lower meaning and purpose scores were associated with adolescence and presence of a special healthcare need. CONCLUSION: The PROMIS Pediatric Meaning and Purpose item banks and their short forms are ready for use in clinical research and practice. They are measures of children's eudaimonic well-being and indicative of children's hopefulness, optimism, goal-directedness, and feelings that life is worth living.

Five-Fold Variation Among Surgeons and Hospitals in the Use of Secondary Palate Surgery.

OBJECTIVE: To identify child-, surgeon- and hospital-specific factors at the time of primary cleft palate repair that are associated with the use of secondary palate surgery. DESIGN: Retrospective cohort study. SETTING: Forty-nine pediatric hospitals. PARTICIPANTS: Children who underwent cleft palate repair between 1998 and 2015. MAIN OUTCOME MEASURE: Time from primary cleft palate repair to secondary palate surgery. RESULTS: By 5 years after the primary palate repair, 27.5% of children had undergone secondary palate surgery. In multivariable analysis, cleft type and age at primary palate repair were both associated with secondary surgery ( P < .01). Children with unilateral cleft lip and palate had a 1.69-fold increased hazard of secondary surgery (95% confidence interval [CI]: 1.54-1.85) compared to children with cleft palate alone. Primary palate repair before 9 months had a 3.99-fold increased hazard of secondary surgery (95% CI: 3.39-4.07) compared to repair at 16 to 24 months of age. After adjusting for cleft type, age at repair, and procedure volume, there remained substantial variation in secondary surgery use among surgeons and hospitals ( P < .01). For children with isolated cleft palate, the predicted proportion of children undergoing secondary surgery within 5 years of primary repair ranged from 8.5% to 46.0% across surgeons and 9.1% to 49.4% across hospitals. CONCLUSIONS: There are substantial differences among surgeons and hospitals in the rates of secondary palate surgery. Further work is needed to identify causes for this variation among providers and develop interventions to reduce the need for secondary surgery.

Factor Structure of the Cultural Competence Items in the National Home and Hospice Care Survey.

BACKGROUND: There is a need for validated measures of cultural competency practices in home health and hospice care (HHHC). OBJECTIVE: To establish the factor structure of the cultural competency items included in the agency-component of the 2007 public-use National Home and Hospice Care Survey file. DATA SOURCE: We used weighted survey data from 1036 HHHC agencies. RESEARCH DESIGN AND PARTICIPANTS: We used exploratory factor analyses to identify a preliminary factor structure, and then performed confirmatory factor analysis to provide further support for identified factor structure. MEASURES: We examined 9 cultural competency items. RESULTS: Exploratory factor analyses suggested an interpretable 2-factor solution: (1) the provision of mandatory cultural competency training; and (2) the provision of cultural competency communication practices. Each factor consisted of 3 items. The remaining 3 items did not load well on these factors. A similar, but more restrictive, confirmatory factor analysis model without cross-loadings supported the 2-factor model: (Equation is included in full-text article.)=9.50, P=0.30, root mean square error of approximation (RMSEA)=0.01, comparative fit index (CFI)=0.99, Tucker-Lewis Index (TLI)=0.99. CONCLUSIONS: Two constructs with 3 items each appeared to be internally valid measures of cultural competency in this nationally representative survey of HHHC agencies: cultural competency training and cultural competency communication practices. These measures could be used by HHHC managers in quality improvement efforts and by policy makers in monitoring cultural competency practices.

Psychometric Evaluation of the PROMIS® Pediatric Psychological and Physical Stress Experiences Measures.

Objective: To provide psychometric evaluation of the PROMIS® Pediatric Psychological and Physical Stress Experiences measures. Methods: Across two studies, Psychological and Physical Stress Experiences items were administered to 2,875 children aged 8-17 years and 2,212 parents of children aged 5-17 years. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning (DIF), and assessment of construct validity. Items were calibrated using item response theory to estimate item parameters representative of the United States. Recommended eight- and four-item short forms were constructed for child- and parent-report versions of the Psychological and Physical Stress Experiences item banks. Results: Final item banks were unidimensional and items were locally independent and free from impactful DIF. Psychological Stress banks include 19 child-report and 12 parent-proxy items. Physical Stress banks include 26 child-report and 26 parent-proxy items. All instruments have strong internal consistency and retest-reliability, and provide precise estimates of varying stress levels. The instruments' construct validity was evidenced by known-group comparisons and convergence with legacy measures. Conclusions: The Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Psychological and Physical Stress item banks and short forms provide efficient, precise, and valid assessments of children's stress experiences.

Development and psychometric evaluation of the PROMIS Pediatric Life Satisfaction item banks, child-report, and parent-proxy editions.

PURPOSE: To describe the psychometric evaluation and item response theory calibration of the PROMIS Pediatric Life Satisfaction item banks, child-report, and parent-proxy editions. METHODS: A pool of 55 life satisfaction items was administered to 1992 children 8-17 years old and 964 parents of children 5-17 years old. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning, and assessment of construct validity. Thirteen items were deleted because of poor psychometric performance. An 8-item short form was administered to a national sample of 996 children 8-17 years old, and 1294 parents of children 5-17 years old. The combined sample (2988 children and 2258 parents) was used in item response theory (IRT) calibration analyses. RESULTS: The final item banks were unidimensional, the items were locally independent, and the items were free from impactful differential item functioning. The 8-item and 4-item short form scales showed excellent reliability, convergent validity, and discriminant validity. Life satisfaction decreased with declining socio-economic status, presence of a special health care need, and increasing age for girls, but not boys. After IRT calibration, we found that 4- and 8-item short forms had a high degree of precision (reliability) across a wide range (>4 SD units) of the latent variable. CONCLUSIONS: The PROMIS Pediatric Life Satisfaction item banks and their short forms provide efficient, precise, and valid assessments of life satisfaction in children and youth.

Relationship-focused vs. Structural Activities in Medical Home Measurement in Pediatrics.

Objectives The Family-Centered Medical Home (FCMH) has become a model of effective and efficient primary care. However, efforts to measure the FCMH may ignore its complexity. We sought to determine whether U.S. primary care pediatricians view structure-focused and relationship-focused practice activities of the FCMH as distinct constructs and how these constructs are associated with practice functions. Methods We analyzed data from the 2012 American Academy of Pediatrics Periodic Survey of Fellows #79 (n = 572) addressing opinions and practice activities related to the FCMH. Through a modified Delphi expert process, we selected items believed to be primarily structure-focused and items that were relationship-focused. Confirmatory factor analysis was used to test whether these constructs were distinct. Separate structural equation models assessed whether structure-focused and relationship-focused activities predicted three broader survey items: (1) interest in FCMH; (2) application for FCMH recognition; and (3) whether a team meets to discuss FCMH improvements. Results The initial two-factor model did not fit well, but improved with movement of two items from the structure to the relationship-focused group. The two factors correlated at r = 0.70. Respondents with increased relationship-focused activities had statistically higher odds of having medium/high interest in FCMH, and having a team meet to discuss FCMH improvements. Respondents with increased structure-focused activities also had higher odds of having team meetings to discuss FCMH improvements, but lower odds of applying for FCMH recognition. Conclusions for Practice The FCMH is multi-dimensional, with relationship- and structure-focused activities differentially linked to pediatrician reports of broader FCMH functions.

Development and Evaluation of the PROMIS® Pediatric Positive Affect Item Bank, Child-Report and Parent-Proxy Editions.

OBJECTIVE: The purpose of this study is to describe the psychometric evaluation and item response theory calibration of the PROMIS Pediatric Positive Affect item bank, child-report and parent-proxy editions. METHODS: The initial item pool comprising 53 items, previously developed using qualitative methods, was administered to 1,874 children 8-17 years old and 909 parents of children 5-17 years old. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning, and construct validity. A total of 14 items were deleted, because of poor psychometric performance, and an 8-item short form constructed from the remaining 39 items was administered to a national sample of 1,004 children 8-17 years old, and 1,306 parents of children 5-17 years old. The combined sample was used in item response theory (IRT) calibration analyses. RESULTS: The final item bank appeared unidimensional, the items appeared locally independent, and the items were free from differential item functioning. The scales showed excellent reliability and convergent and discriminant validity. Positive affect decreased with children's age and was lower for those with a special health care need. After IRT calibration, we found that 4 and 8 item short forms had a high degree of precision (reliability) across a wide range of the latent trait (>4 SD units). CONCLUSION: The PROMIS Pediatric Positive Affect item bank and its short forms provide an efficient, precise, and valid assessment of positive affect in children and youth.

Establishing clinical meaning and defining important differences for Patient-Reported Outcomes Measurement Information System (PROMIS®) measures in juvenile idiopathic arthritis using standard setting with patients, parents, and providers.

BACKGROUND: Patient-Reported Outcomes Measurement Information System (PROMIS) measures are used increasingly in clinical care. However, for juvenile idiopathic arthritis (JIA), scores lack a framework for interpretation of clinical severity, and minimally important differences (MID) have not been established, which are necessary to evaluate the importance of change. METHODS: We identified clinical severity thresholds for pediatric PROMIS measures of mobility, upper extremity function (UE), fatigue, and pain interference working with adolescents with JIA, parents of JIA patients, and clinicians, using a standard setting methodology modified from educational testing. Item parameters were used to develop clinical vignettes across a range of symptom severity. Vignettes were ordered by severity, and panelists identified adjacent vignettes considered to represent upper and lower boundaries separating category cut-points (i.e., from none/mild problems to moderate/severe). To define MIDs, panelists reviewed a full score report for the vignettes and indicated which items would need to change and by how much to represent "just enough improvement to make a difference." RESULTS: For fatigue and UE, cut-points among panels were within 0.5 SD of each other. For mobility and pain interference, cut-scores among panels were more divergent, with parents setting the lowest cut-scores for increasing severity. The size of MIDs varied by stakeholders (parents estimated largest, followed by patients, then clinicians). MIDs also varied by severity classification of the symptom. CONCLUSIONS: We estimated clinically relevant severity cut-points and MIDs for PROMIS measures for JIA from the perspectives of multiple stakeholders and found notable differences in perspectives.

Children's family experiences: development of the PROMIS® pediatric family relationships measures.

PURPOSE: To describe the development of pediatric family relationships measures, with versions for child self-report (8-17 years) and parent-report for children 5-17 years old. Measures were created for integration into the Patient Reported Outcomes Measurement Information System (PROMIS®). METHODS: Semi-structured interviews with 10 experts, 24 children, and 8 parents were conducted to elicit and clarify essential elements of family relationships. A systematic literature review was conducted to identify item concepts representative of each element. The concepts were transformed into items that were iteratively revised based on cognitive interviews (n = 43 children) and item translatability review. Psychometric studies involving 2846 children and 2262 parents were conducted to further refine and validate the instruments. RESULTS: Qualitative procedures supported the development of content valid Family Relationships item banks. Final child- and parent-report item banks each contain 47 items. Unidimensional item banks were calibrated using IRT-modeling to estimate item parameters representative of the US population and to enable computerized adaptive test administration. Four- and eight-item short forms were constructed for standard fixed format administration. All instruments have strong internal consistency, retest-reliability, and provide precise estimates of various levels of family relationship quality. Preliminary evidence of the instruments' validity was provided by known-group comparisons and convergence with legacy measures. CONCLUSION: The PROMIS pediatric Family Relationships measures can be applied in research focused on determinants, outcomes, and the protective effects of children's subjective family relationship experiences.