Pain Interference in Juvenile Idiopathic Arthritis.

OBJECTIVE: Despite treatment advances, pain remains a serious problem for many children with juvenile idiopathic arthritis (JIA). To better understand pain in children with JIA and identify potentially modifiable factors, this study evaluated Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Pain Interference (PI) and its relationships with other pain measures and demographic, clinical, psychosocial, and functional variables. METHODS: This cross-sectional, observational, multicenter study used descriptive statistics and a mix of bivariate and multivariable analyses to describe PI and characterize relationships with other measures and variables. RESULTS: Among 355 children with JIA, 27% reported moderate or severe PI and 13.3% reported daily pain. PI correlated with other pain measures. Increasing age, decreasing disease duration, and increasing number of active joints, as well as presence of active disease, steroid treatment, and biologic treatment, were associated with greater PI. All PROMIS psychosocial and functional measures were associated with PI in the expected direction except for PROMIS Pediatric Physical Activity, which showed no association. In multivariable analyses, only PROMIS Fatigue, PROMIS Mobility, and the exploratory interaction of PROMIS Anxiety and disease-modifying antirheumatic drug treatment were significant. CONCLUSION: Moderate and severe PI was prevalent in this sample of children with JIA. PI increased with age and indicators of disease activity, but was more strongly associated with increasing fatigue and decreasing mobility. Findings support the use of PI as a short, easily administered multidimensional pain measure as part of routine clinical care. Fatigue, mobility, and disease activity should be assessed further when PI is high.

The importance of development standards for anchoring vignettes: an illustrative example from pediatric localized scleroderma quality of life.

PURPOSE: Anchoring vignettes (AVs) are a promising measurement technique to reduce bias in patient-reported outcome (PRO) measures by helping researchers understand differences in how individuals and groups interpret response options. However, little attention has been paid to ensure quality development of AVs, and their performance has not been well assessed in pediatric populations. In this study, we explore the application of a rigorous development process for AVs based upon current standards for PROs, as well as feasibility of AVs when administered to children and adolescents. METHODS: We developed AVs using a rigorous, patient-centered mixed methods process including three phases: (1) development, (2) a pilot study, and (3) a field test. Our proposed process included the generation of a conceptual framework based on the PRO, the Localized Scleroderma Quality of Life Instrument, and numerous vignette-specific considerations. We qualitatively explored readability and comprehension of the AVs (pilot study) and then analyzed ranking patterns within vignette sets (field test). RESULTS: Four sets of four vignettes were developed. Revisions were suggested at each phase of development. The pilot study demonstrated that children ≥ 10 years had no trouble indicating understanding of the AVs. In the field test, although appropriate rankings of vignettes were generally demonstrated by participants, the percentage of tied rankings was higher than expected in this pediatric group. CONCLUSIONS: This work supports the need for rigorous developmental standards for AVs, as each stage of development suggested revisions. Additionally, AVs showed initial promise for use with pediatric populations; general feasibility and understanding were supported.

Gender-Based Salary Inequities Among Pediatric Rehabilitation Medicine Physicians in the United States.

OBJECTIVE: To assess whether gender inequities exist for pediatric physiatrists and, if affirmative, what factors account for this difference. DESIGN: Cohort study. SETTING: Online REDCap survey administered via e-mail. PARTICIPANTS: Pediatric physiatrists practicing in the United States in 2017. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Respondents reported on their gender, training, practice type and location, leadership positions, years in practice and years at their current location, salary, research, and clinical productivity. RESULTS: Of the 307 surveys sent, 235 individuals responded, yielding a response rate of 76.5%. Pediatric physiatrists who identified as women were more likely to work part-time but were demographically similar to their colleagues who identified as men. The odds of having no leadership role were higher for women (odds ratio=2.17; P=.02) than men. Pediatric physiatrists who identified as men made on average (in US dollars ± SD) 244,798±52,906 annually compared with those who identified as women 224,497±60,756. The average annual difference in full-time salary was $20,311 in favor of those who identified as men (95% confidence interval, $3135-$37,486). The set of predictors in the multivariable model explained about 40% of the total variability in annual full-time salary (R2=0.389; adjusted R2=0.339; F15,197=7.734; P<.001). Gender was not a significant predictor in the model, but model prediction of the salaries of pediatric physiatrists who identified as men was better than model prediction of the salaries of those who identified as women. CONCLUSIONS: Despite representing a majority of the field, pediatric physiatrists who identified as women were paid less than their counterparts who identified as men. The traditional predictors associated with the salaries of men were not enough to explain salary variation among those who identified as women, thereby providing evidence of the importance of intangible and unmeasured aspects of a women's career, such as bias and institutional culture.

A Comparison of Multilevel Mediation Modeling Methods: Recommendations for Applied Researchers.

Multilevel structural equation modeling (MSEM) has been proposed as a valuable tool for estimating mediation in multilevel data and has known advantages over traditional multilevel modeling, including conflated and unconflated techniques (CMM & UMM). Recent methodological research has focused on comparing the three methods for 2-1-1 designs, but in regards to 1-1-1 mediation designs, there are significant gaps in the published literature that prevent applied researchers from making educated decisions regarding which model to employ in their own specific research design. A Monte Carlo study was performed to compare MSEM, UMM, and CMM on relative bias, confidence interval coverage, Type I Error, and power in a 1-1-1 model with random slopes under varying data conditions. Recommendations for applied researchers are discussed and an empirical example provides context for the three methods.

Endpoints and outcomes for localized scleroderma/morphea: a scoping literature review.

BACKGROUND: Current treatment for localized scleroderma (LS) has been shown to halt disease activity, but little is still known about patient experiences with these treatments, nor is there consensus about optimal measurement strategies for future clinical trials. OBJECTIVE: Conduct a scoping review of the literature for the types of outcomes and measures (i.e. clinician-, patient-, and caregiver-reported) utilized in published treatment studies of LS. METHODS: Online databases were searched for articles related to the evaluation of treatment efficacy in LS with a special focus on pediatrics. RESULTS: Of the 168 studies, the most common outcomes used were cutaneous disease activity and damage measured via clinician-reported assessments. The most frequently cited measure was the Localized Scleroderma Cutaneous Assessment Tool (LoSCAT). Few patient-reported outcome measures (PROMs) were used. LIMITATIONS: Some studies only vaguely reported the measures utilized, and the review yielded a low number of clinical trials. CONCLUSION: In addition to evaluating disease activity with clinician-reported measures, the field could obtain critical knowledge on the patient experience by including high-quality PROMs of symptoms and functioning. More clinical trials using a variety of outcomes and measures are necessary to determine the most suitable course of treatment for LS patients.

Understanding reliability of the observer-reported communication ability measure within Angelman syndrome through the lens of generalizability theory.

AIMS: Caregivers rate improved communication ability as one of the most desired outcomes for successful interventions for individuals with Angelman syndrome (AS). When measuring communication ability in clinical trials, the reliability of such measures is critical for detecting significant changes over time. This study examined the reliability of the Observed-Reported Communication Ability (ORCA) measure completed by caregivers of individuals with AS. METHODS: The ORCA measure was completed by 249 caregivers with 170 caregivers completing the ORCA measure again after 5-12 days. Generalizability theory was used to examine the following sources of measurement error in ORCA scores: concepts, subdomains, assessment points, and the interactions among those facets and the object of measurement: communication ability. Three generalizability studies were conducted to understand the reliability of the ORCA measure for different measurement designs. Decision studies were carried out to demonstrate the optimization of measurement procedures of the ORCA measure. RESULTS: G and Phi coefficients of the original measurement design exceeded the 0.80 threshold considered sufficiently reliable to make relative and absolute decisions about the communication ability of individuals with AS based on their caregivers' observed scores. The optimization procedures indicated that increasing the number of communication concepts and/or assessment points leads to more reliable estimates of communication. CONCLUSION: The ORCA measure was able to reliably distinguish different levels of communication ability among individuals with AS. Multiple assessment points and or more concepts would provide more precise estimates of an individual's communication ability but at the cost of survey fatigue.

Collecting patient-reported outcome measures in the electronic health record: Lessons from the NIH pragmatic trials Collaboratory.

The NIH Pragmatic Trials Collaboratory supports the design and conduct of 27 embedded pragmatic clinical trials, and many of the studies collect patient reported outcome measures as primary or secondary outcomes. Study teams have encountered challenges in the collection of these measures, including challenges related to competing health care system priorities, clinician's buy-in for adoption of patient-reported outcome measures, low adoption and reach of technology in low resource settings, and lack of consensus and standardization of patient-reported outcome measure selection and administration in the electronic health record. In this article, we share case examples and lessons learned, and suggest that, when using patient-reported outcome measures for embedded pragmatic clinical trials, investigators must make important decisions about whether to use data collected from the participating health system's electronic health record, integrate externally collected patient-reported outcome data into the electronic health record, or collect these data in separate systems for their studies.

Current Utilization of Qualitative Methodologies in Dermatology: A Scoping Review.

The focus of this review was to determine how qualitative methods are used in dermatology research and whether published manuscripts meet current standards for qualitative research. A scoping review of manuscripts published in English between January 1, 2016 and September 22, 2021 was conducted. A coding document was developed to collect information on authors, methodology, participants, research theme, and the presence of quality criteria as outlined by the Standards for Reporting Qualitative Research. Manuscripts were included if they described original qualitative research about dermatologic conditions or topics of primary interest to dermatology. An adjacency search yielded 372 manuscripts, and after screening, 134 met the inclusion criteria. Most studies utilized interviews or focus groups, and researchers predominantly selected participants on the basis of disease status, including over 30 common and rare dermatologic conditions. Research themes frequently included patient experience of disease, development of patient-reported outcomes, and descriptions of provider and caregiver experiences. Although most authors explained their analysis and sampling strategy and included empirical data, few referenced qualitative data reporting standards. Missed opportunities for qualitative methods in dermatology include examination of health disparities, exploration of surgical and cosmetic dermatology experiences, and determination of the lived experience of and provider attitudes toward diverse patient populations.

Patient experience of spontaneous intracranial hypotension (SIH): qualitative interviews for concept elicitation.

BACKGROUND & OBJECTIVES: Spontaneous intracranial hypotension (SIH) is an underdiagnosed and debilitating condition caused by a spinal cerebrospinal fluid (CSF) leak. Although SIH can lead to substantial morbidity and disability, little data exists about patients' perspectives. Without hearing directly from patients, our understanding of the full experience of having SIH is limited, as is our ability to identify and use appropriate patient-reported outcome measures (PROMs) within clinical care and research. The purpose of this study was to conduct qualitative interviews with confirmed SIH patients to fully describe their experiences and identify relevant concepts to measure. METHODS: Patients were recruited from an SIH specialty clinic at a large, U.S.-based healthcare center. Patients undergoing an initial consultation who were ≥ 18 years old, English-speaking, met the International Classification of Headache Disorders-3 criteria for SIH, and had a brain MRI with contrast that was positive for SIH were eligible to participate. During semi-structured qualitative interviews with a trained facilitator, participants were asked to describe their current SIH symptoms, how their experiences with SIH had changed over time, and the aspects of SIH that they found most bothersome. Analysts reviewed the data, created text summaries, and wrote analytic reports. RESULTS: Fifteen participants completed interviews. Common symptoms reported by patients included headache, tinnitus, ear fullness/pressure/pain, and neck or interscapular pain. Patients reported that their symptoms worsened over the course of their day and with activity. The most bothersome aspect of SIH was disruption to daily activities and limits to physical activities/exercise, which were severe. With regard to symptoms, the most bothersome and impactful included physical pain and discomfort (including headache), as well as fatigue. CONCLUSIONS: Patients reported a diverse set of symptoms that were attributed to SIH, with devastating impacts on functioning and high levels of disability. Researchers considering use of PROMs for SIH should consider inclusion of both symptom scales and aspects of functioning, and future work should focus on evaluating the validity of existing measures for this patient population using rigorous qualitative and quantitative methods in diverse samples. Additionally, these data can be used to assist clinicians in understanding the impacts of SIH on patients.

Validation of the Observer-Reported Communication Ability (ORCA) Measure for Individuals With Angelman Syndrome.

There is a critical need for high-quality clinical outcome assessments to capture the important aspects of communication ability of individuals with Angelman syndrome (AS). To center the perspective of caregivers, our team developed the novel Observer-Reported Communication Ability (ORCA) measure using best practice guidelines, with the goal of developing a measure that could be administered to caregivers directly without the need for a certified administrator for use in clinical trials. To refine the draft measure, we conducted two rounds of cognitive interviews with 24 caregivers and a quantitative study including 249 caregivers. The results from both studies support the overall content validity, construct validity, and the reliability of the ORCA measure for individuals with AS > 2 years old for use in research contexts. Future work should explore the responsiveness of ORCA measures to changes over time in a diverse sample.

Exploring Communication Ability in Individuals With Angelman Syndrome: Findings From Qualitative Interviews With Caregivers.

Communication deficits have a substantial impact on quality of life for individuals with Angelman syndrome (AS) and their families, but limited qualitative work exists to support the necessary content of measures aiming to assess communication for these individuals. Following best practices for concept elicitation studies, we conducted individual qualitative interviews with caregivers and clinicians to elicit meaningful aspects of communication for individuals with AS. Caregivers were able to discuss their child's specific communication behaviors within a large number of expressive, receptive, and pragmatic functions via numerous symbolic and non-symbolic modalities. These results aligned well with published literature on communication in AS and will be used to inform the design of a novel caregiver-reported measure. Future studies on communication in individuals with AS should focus on gathering quantitative data from large samples of diverse caregivers, which would allow for estimations of the frequency of specific behaviors across the population.

Validation of the Observer-Reported Communication Ability (ORCA) measure for individuals with Rett syndrome.

PURPOSE: The study goal was to validate the Observer-Reported Communication Ability (ORCA) measure for use with females with Rett Syndrome (RTT). METHODS: Qualitative interviews, including concept elicitation and cognitive interviewing methods, were conducted with 19 caregivers of individuals with RTT ages 2 and older. A quantitative study was then conducted in 279 caregivers to evaluate construct validity and reliability. RESULTS: After minor modifications were made, the modified ORCA measure was well understood and captured key communication concepts. Quantitative data showed evidence for reliable scores (α = 0.90, test-retest intraclass correlation = 0.88), minimal floor and no ceiling effects, and strong correlation with the Communication and Symbolic Behaviors Scale (r = 0.73). CONCLUSIONS: This study provided initial support that the modified ORCA measure is an acceptable caregiver-reported measure of communication ability for females with RTT. Future work should include evaluation of longitudinal validity of the measure and its associations with clinician- and performance-based measures in diverse samples.

Equity and bias in electronic health records data.

Embedded pragmatic clinical trials (ePCTs) are conducted during routine clinical care and have the potential to increase knowledge about the effectiveness of interventions under real world conditions. However, many pragmatic trials rely on data from the electronic health record (EHR) data, which are subject to bias from incomplete data, poor data quality, lack of representation from people who are medically underserved, and implicit bias in EHR design. This commentary examines how the use of EHR data might exacerbate bias and potentially increase health inequities. We offer recommendations for how to increase generalizability of ePCT results and begin to mitigate bias to promote health equity.

Potential bias and lack of generalizability in electronic health record data: reflections on health equity from the National Institutes of Health Pragmatic Trials Collaboratory.

Embedded pragmatic clinical trials (ePCTs) play a vital role in addressing current population health problems, and their use of electronic health record (EHR) systems promises efficiencies that will increase the speed and volume of relevant and generalizable research. However, as the number of ePCTs using EHR-derived data grows, so does the risk that research will become more vulnerable to biases due to differences in data capture and access to care for different subsets of the population, thereby propagating inequities in health and the healthcare system. We identify 3 challenges-incomplete and variable capture of data on social determinants of health, lack of representation of vulnerable populations that do not access or receive treatment, and data loss due to variable use of technology-that exacerbate bias when working with EHR data and offer recommendations and examples of ways to actively mitigate bias.

Assessing Patient-Reported Outcomes in Pediatric Rheumatic Diseases: Considerations and Future Directions.

For children with pediatric rheumatic diseases (PRDs), the inclusion of patient-reported outcomes (PROs) is critical to inform decision making in health care delivery and research settings. PROs are direct reports from a child on their health status, without interpretation by anyone else. PROs improve understanding of the patient experience, allow clinicians to provide patient-centered care, and add value to clinical trials. When PROs cannot be collected directly from the patient, caregiver-proxy reports can provide important information on the child's more observable symptoms and functioning. In this article, we describe the current use of PROs in specific PRDs, align current research with best practice recommendations for both clinical care and research settings, highlight exciting new developments, and identify areas for future research.

Responding to signals of mental and behavioral health risk in pragmatic clinical trials: Ethical obligations in a healthcare ecosystem.

BACKGROUND: Ethical responsibilities for monitoring and responding to signals of behavioral and mental health risk (such as suicidal ideation, opioid use disorder, or depression) in general clinical research have been described; however, pragmatic clinical trials (PCTs) raise new contextual challenges. METHODS: We use our experience with the PRISM (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing) program, which is a component of the Helping End Addiction Long-Term (HEAL) Initiative, to provide examples of research studying nonpharmacologic interventions for pain that collect sensitive data. Members of the PRISM Ethics and Regulatory Core and Patient-Centered Outcome Core Working Group discussed and refined considerations and recommendations. RESULTS: PCT researchers can help identify the extent of their ethical obligations to monitor and respond to signals of potential behavioral and mental health risks by understanding and aligning stakeholder expectations; considering characteristics of the trial and study population; defining triggers, thresholds, and responsibilities for action; identifying appropriate response mechanisms and capabilities; integrating responses with health systems; and addressing privacy. Based on such an assessment, researchers should proactively identify if, when, and how a response will be triggered. Doing so necessitates that stakeholders understand their roles in managing such risks. Finally, consent forms and other study disclosures should clearly state what if any responses might be taken. CONCLUSION: Early and ongoing bi-directional communication with relevant stakeholders is critical to identifying and meeting the ethical challenges for PCTs when managing and responding to behavioral and mental health data that potentially signal elevated risk to individuals.

Assessing the Field of Pediatric Rehabilitation Medicine's Interest in Expanding Fellowship Training to Pediatricians.

BACKGROUND: Training opportunities to pursue a career in pediatric rehabilitation medicine (PRM) have evolved over the last 40 years, with the most recent change being the development and accreditation of PRM fellowships and subspecialty certification in PRM. Currently the American Board of Physical Medicine & Rehabilitation (ABPMR) requires all candidates for PRM subspecialty certification to have completed a physical medicine and rehabilitation (PM&R) residency. The small number of certified PRM physicians has prompted debate within the field about permitting pediatricians to enter PRM fellowships without having to complete a PM&R residency. OBJECTIVE: To assess the level of interest within the field of PRM in creating a pathway to PRM for pediatricians. DESIGN: Survey of pediatric physiatrists in the United States in 2017. SETTING: National. PARTICIPANTS: Pediatric physiatrists. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASUREMENTS: Favorability toward pediatricians becoming certified in PRM. RESULTS: Most respondents (62%) were in favor of pediatricians having the opportunity to pursue training in PRM, with an increase in support (70%) after being introduced to workforce issues in PRM. Training time for pediatricians was the largest concern identified by respondents who were not in favor (80%), with additional themes identified including dilution of the essence of the field and operationalization issues. CONCLUSIONS: With a small number of practicing pediatric physiatrists, the growing number of children with disabilities, and the limited access to our services, most pediatric physiatrists are in agreement that it is time to consider the opportunity to expand the PRM workforce by creating a fellowship pathway to subspecialty board certification in PRM after pediatric residency.

Malaria Diagnosis by U.S. Providers on Short-term Medical Mission Trips to Uganda.

Malaria is one of the top 10 leading causes of death in Uganda. Short-term medical missions (STMMs) to address unmet medical needs in lower-resource settings are increasingly common. Th is study evaluates correlations between patient and clinician variables and accurate malaria diagnosis by providers on STMMs to Uganda. We surveyed 18 U.S. providers and performed a retrospective chart review of 246 patients seen by those providers on STMMs in Uganda between 2016 to 2017. All providers recorded their clinical level of suspicion for patients who met inclusion criteria, following which a rapid diagnostic test (RDT) was performed. Fift y-four percent of the patients tested positive for malaria. Level of provider accuracy ranged widely from 30.0% to 95.5% correct. Our fi ndings reaffirm that signs and symptoms of malaria are too nonspecifi c to be used alone without diagnostic testing by STMM providers. Pre-departure STMM training on malaria diagnosis is a necessity.

Enhancing the use of EHR systems for pragmatic embedded research: lessons from the NIH Health Care Systems Research Collaboratory.

OBJECTIVE: We identified challenges and solutions to using electronic health record (EHR) systems for the design and conduct of pragmatic research. MATERIALS AND METHODS: Since 2012, the Health Care Systems Research Collaboratory has served as the resource coordinating center for 21 pragmatic clinical trial demonstration projects. The EHR Core working group invited these demonstration projects to complete a written semistructured survey and used an inductive approach to review responses and identify EHR-related challenges and suggested EHR enhancements. RESULTS: We received survey responses from 20 projects and identified 21 challenges that fell into 6 broad themes: (1) inadequate collection of patient-reported outcome data, (2) lack of structured data collection, (3) data standardization, (4) resources to support customization of EHRs, (5) difficulties aggregating data across sites, and (6) accessing EHR data. DISCUSSION: Based on these findings, we formulated 6 prerequisites for PCTs that would enable the conduct of pragmatic research: (1) integrate the collection of patient-centered data into EHR systems, (2) facilitate structured research data collection by leveraging standard EHR functions, usable interfaces, and standard workflows, (3) support the creation of high-quality research data by using standards, (4) ensure adequate IT staff to support embedded research, (5) create aggregate, multidata type resources for multisite trials, and (6) create re-usable and automated queries. CONCLUSION: We are hopeful our collection of specific EHR challenges and research needs will drive health system leaders, policymakers, and EHR designers to support these suggestions to improve our national capacity for generating real-world evidence.