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OBJECTIVE: To assess the quality of medical care in childhood-onset systemic lupus erythematosus (SLE) at tertiary pediatric rheumatology centers as measured by observance of SLE quality indicators (SLE-QIs). METHODS: International consensus has been achieved for childhood-onset SLE-QIs capturing medical care provision in 9 domains: diagnostic testing, education of cardiovascular (CV) risk and lifestyles, lupus nephritis (LN), medication management, bone health, ophthalmologic surveillance, transition, pregnancy, and vaccination. Using medical record information, the level of performance of these childhood-onset SLE-QIs was assessed in childhood-onset SLE populations treated at 4 tertiary pediatric rheumatology centers in the US, 2 in Brazil, and 1 center in India. RESULTS: A total of 483 childhood-onset SLE patients were assessed. Care for the 310 US patients differed markedly for childhood-onset SLE-QIs addressing LN, bone health, vaccinations, education on CV risk, and transition planning. Performance of safety blood testing for medications was high at all centers. Despite often similar performance on the childhood-onset SLE-QI, access to kidney biopsies was lower in Brazil than in the US. Irrespective of the country of practice, larger centers tended to meet the childhood-onset SLE-QIs more often than smaller centers. CONCLUSION: The childhood-onset SLE-QIs, evidence-based minimum standards of medical care, are not consistently met in the US or some other countries outside the US. This has the potential to contribute to suboptimal childhood-onset SLE outcomes.
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Benchmarking , Lúpus Eritematoso Sistêmico/terapia , Indicadores de Qualidade em Assistência à Saúde , Adolescente , Adulto , Feminino , Humanos , Masculino , Adulto JovemRESUMO
The Patient-Reported Outcomes Measurement Information System (PROMIS) initiative is a comprehensive strategy by the National Institutes of Health to support the development and validation of precise instruments to assess self-reported health domains across healthy and disease-specific populations. Much progress has been made in instrument development, but there remains a gap in the validation of PROMIS measures for pediatric chronic pain. The purpose of this study was to investigate the construct validity and responsiveness to change of 7 PROMIS domains for the assessment of children (ages: 8-18) with chronic pain--Pain Interference, Fatigue, Anxiety, Depression, Mobility, Upper Extremity Function, and Peer Relationships. The PROMIS measures were administered at the initial visit and 2 follow-up visits at an outpatient chronic pain clinic (CPC; N = 82) and at an intensive amplified musculoskeletal pain day-treatment program (N = 63). Aim 1 examined construct validity of PROMIS measures by comparing them with corresponding "legacy" measures administered as part of usual care in the CPC sample. Aim 2 examined sensitivity to change in both CPC and amplified musculoskeletal pain samples. Longitudinal growth models showed that PROMIS' Pain Interference, Anxiety, Depression, Mobility, Upper Extremity, and Peer Relationship measures and legacy instruments generally performed similarly with slightly steeper slopes of improvement in legacy measures. All 7 PROMIS domains showed responsiveness to change. Results offered initial support for the validity of PROMIS measures in pediatric chronic pain. Further validation with larger and more diverse pediatric pain samples and additional legacy measures would broaden the scope of use of PROMIS in clinical research.
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Dor Crônica/psicologia , Dor Crônica/terapia , Manejo da Dor , Medição da Dor , Resultado do Tratamento , Adolescente , Criança , Dor Crônica/fisiopatologia , Depressão/diagnóstico , Depressão/etiologia , Avaliação da Deficiência , Registros Eletrônicos de Saúde/estatística & dados numéricos , Feminino , Humanos , Estudos Longitudinais , Masculino , Clínicas de Dor , Escalas de Graduação Psiquiátrica , Reprodutibilidade dos Testes , AutorrelatoRESUMO
UNLABELLED: As initial steps in a broader effort to develop and test pediatric pain behavior and pain quality item banks for the Patient-Reported Outcomes Measurement Information System (PROMIS), we used qualitative interview and item review methods to 1) evaluate the overall conceptual scope and content validity of the PROMIS pain domain framework among children with chronic/recurrent pain conditions, and 2) develop item candidates for further psychometric testing. To elicit the experiential and conceptual scope of pain outcomes across a variety of pediatric recurrent/chronic pain conditions, we conducted 32 semi-structured individual and 2 focus-group interviews with children and adolescents (8-17 years), and 32 individual and 2 focus-group interviews with parents of children with pain. Interviews with pain experts (10) explored the operational limits of pain measurement in children. For item bank development, we identified existing items from measures in the literature, grouped them by concept, removed redundancies, and modified the remaining items to match PROMIS formatting. New items were written as needed and cognitive debriefing was completed with the children and their parents, resulting in 98 pain behavior (47 self, 51 proxy), 54 quality, and 4 intensity items for further testing. Qualitative content analyses suggest that reportable pain outcomes that matter to children with pain are captured within and consistent with the pain domain framework in PROMIS. PERSPECTIVE: PROMIS pediatric pain behavior, quality, and intensity items were developed based on a theoretical framework of pain that was evaluated by multiple stakeholders in the measurement of pediatric pain, including researchers, clinicians, and children with pain and their parents, and the appropriateness of the framework was verified.
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Dor Crônica/psicologia , Medição da Dor/métodos , Adolescente , Criança , Bases de Dados Factuais , Feminino , Humanos , Entrevistas como Assunto , Masculino , Pais , Psicometria/métodos , Pesquisa Qualitativa , AutorrelatoRESUMO
PURPOSE: To conduct a comparative analysis of eight pediatric self-report scales for ages 8-17 years from the National Institutes of Health (NIH) Patient Reported Outcomes Measurement Information System (PROMIS(®)) in six pediatric chronic health conditions, using indicators of disease severity. METHODS: Pediatric patients (N = 1454) with asthma, cancer, chronic kidney disease, obesity, rheumatic disease, and sickle cell disease completed items from the PROMIS pediatric mobility, upper extremity functioning, depressive symptoms, anxiety, anger, peer relationships, pain interference, and fatigue self-report scales. Comparisons within the six pediatric chronic health conditions were conducted by examining differences in groups based on the disease severity using markers of severity that were specific to characteristics of each disease. A comparison was also made across diseases between children who had been recently hospitalized and those who had not. RESULTS: In general, there were differences in self-reported health outcomes within each chronic health condition, with patients who had higher disease severity showing worse outcomes. Across health conditions, when children with recent hospitalizations were compared with those who had not been hospitalized in the past 6 months, we found significant differences in the expected directions for all PROMIS domains, except anger. CONCLUSIONS: PROMIS measures discriminate between different clinically meaningful subgroups within several chronic illnesses. Further research is needed to determine the responsiveness of the PROMIS pediatric scales to change over time.
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Ira , Doença Crônica/psicologia , Qualidade de Vida/psicologia , Autorrelato , Adolescente , Anemia Falciforme/complicações , Anemia Falciforme/psicologia , Asma/complicações , Asma/psicologia , Criança , Depressão/complicações , Depressão/psicologia , Fadiga/complicações , Fadiga/psicologia , Feminino , Humanos , Masculino , Neoplasias/complicações , Neoplasias/psicologia , Obesidade Infantil/complicações , Obesidade Infantil/psicologia , Insuficiência Renal Crônica/complicações , Insuficiência Renal Crônica/psicologia , Doenças Reumáticas/complicações , Doenças Reumáticas/psicologia , Inquéritos e Questionários , Estados UnidosRESUMO
OBJECTIVE: The study objective was to describe the individual item-level discrepancies between children ages 8-17 years and their parents for the PROMIS(®) pediatric scales. Contextual effects on item-level informant discrepancies for the pediatric pain interference items were further analyzed conditional on whether the child, the parent, or anyone else in the household experienced chronic pain. METHODS: Parallel pediatric self-report and parent proxy-report items were completed by approximately 300 parent-child dyads depending on form assignment and individual nonresponse. Agreement between parent and child responses to individual items was measured using the polychoric correlation coefficient and weighted κ. The Chi-square test of symmetry was utilized for a comparison of the pattern of parent-child item discrepancies on the response scales, and the differences between the child and parent responses on the 1-5 item response scale are summarized . RESULTS: A continuum of higher item-level parent-child discrepancies was demonstrated starting with peer relationships, anger, anxiety, and depressive symptoms, followed by progressively lower parent-child discrepancies for energy, fatigue, asthma impact, pain interference, upper extremity, and mobility items. Parent-child discrepancies for pain interference items were lower in the context of chronic pain either in the child or in the parent. CONCLUSIONS: Parent-child item-level discrepancies were lower for more objective or visible items than for items measuring internal states or less observable items measuring latent variables such as peer relationships and fatigue. Future research should focus on the child and parent characteristics that influence domain-specific item-level discrepancies, and under what conditions item-level parent-child discrepancies predict child health outcomes.
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Dor Crônica/psicologia , Qualidade de Vida/psicologia , Autorrelato , Adolescente , Adulto , Ira , Asma/terapia , Criança , Depressão/psicologia , Fadiga/psicologia , Feminino , Humanos , Masculino , Pais , Pediatria , ProcuradorRESUMO
OBJECTIVE: To develop and validate the Juvenile Spondyloarthritis Disease Activity Index (JSpADA) for use in clinical practice and research. METHODS: Using modified Delphi consensus techniques, 10 items were selected by participants in the international pediatric rheumatology listserv, the Childhood Arthritis and Rheumatology Research Alliance, and the listserv for the pediatric section of the American College of Rheumatology. Validation was performed in a retrospective multicenter cohort of 244 children. RESULTS: In total, 106 physicians representing 14 countries completed the initial questionnaire. Completion rates for the subsequent questionnaires were 84%, 75%, and 77% of the original respondents. Ten items exceeded 80% consensus: arthritis, enthesitis, patient pain assessment, inflammatory markers, morning stiffness, clinical sacroiliitis, uveitis, back mobility, and patient and physician assessments of disease activity. After item analysis, 2 items were eliminated (patient and physician assessments of disease activity). Factor analysis identified 3 primary domains that explained 58% of the variance: peripheral disease, axial disease, and uveitis. The Cronbach's α coefficient was 0.66. The JSpADA had high or moderate correlations with the Juvenile Arthritis Disease Activity Score (r = 0.81), patient and physician assessments of disease activity (r = 0.70 and r = 0.66, respectively), and the Childhood Health Assessment Questionnaire (r = 0.56). The JSpADA discriminated well between subjects with active versus inactive disease (P < 0.001) and was responsive to improvement or worsening in disease activity over time (P < 0.001). CONCLUSION: Using international input and consensus formation techniques, we developed and validated the first disease activity assessment for juvenile spondyloarthritis. Future studies should validate the JSpADA in a prospective multicenter cohort.
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Medição da Dor/métodos , Reumatologia/métodos , Espondilartrite/diagnóstico , Adolescente , Criança , Técnica Delphi , Feminino , Nível de Saúde , Humanos , Masculino , Estudos Retrospectivos , Índice de Gravidade de Doença , Inquéritos e QuestionáriosRESUMO
OBJECTIVE: There is no standardized approach to the initial treatment of polyarticular juvenile idiopathic arthritis (JIA) among pediatric rheumatologists. Understanding the comparative effectiveness of the diverse therapeutic options available will result in better health outcomes for polyarticular JIA. The Childhood Arthritis and Rheumatology Research Alliance (CARRA) developed consensus treatment plans (CTPs) for use in clinical practice to facilitate such studies. METHODS: A case-based survey was administered to CARRA members to identify the common treatment approaches for new-onset polyarticular JIA. Two face-to-face consensus conferences employed modified nominal group technique to identify treatment strategies, operational case definition, end points, and data elements to be collected. A core workgroup reviewed the relevant literature, refined plans, and developed medication dosing and monitoring recommendations. RESULTS: The initial case-based survey identified significant variability among treatment approaches for new-onset polyarticular JIA. We developed 3 CTPs based on treatment strategies for the first 12 months of therapy, as well as case definitions and clinical and laboratory monitoring schedules. The CTPs include a step-up plan (nonbiologic disease-modifying antirheumatic drug [DMARD] followed by a biologic DMARD), an early combination plan (nonbiologic and biologic DMARD combined within a month of treatment initiation), and a biologic only plan. This approach was approved by 96% of the CARRA JIA Research Committee members attending the 2013 CARRA face-to-face meeting. CONCLUSION: Three standardized CTPs were developed for new-onset polyarticular JIA. Coupled with data collection at defined intervals, use of these CTPs will enable the study of their comparative effectiveness in an observational setting to optimize initial management of polyarticular JIA.
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Artrite Juvenil/terapia , Sistema de Registros , Reumatologia/normas , Artrite Juvenil/diagnóstico , Criança , Protocolos Clínicos , Consenso , Diagnóstico Precoce , Humanos , Pediatria/normas , Reumatologia/organização & administraçãoRESUMO
Comparative effectiveness research (CER) is a growing area of investigation aimed at determining the most beneficial treatments for patients in view of their clinical characteristics. CER provides personalized treatment information that cannot be obtained from traditional clinical trials. However, many analytical challenges to performing CER remain, particularly in nonexperimental settings. The main obstacles to CER include bias from variation in use of treatments, and heterogeneity in types and quality of data across registries. Increasing standardization of care and consensus among stakeholders regarding CER methodology will strengthen the validity of CER from observational data. Innovations in outcomes measurement, and the ability to repurpose electronic health record data for research will increase the capability to assess treatment effects by CER in clinical practice. Investment in infrastructure, informatics, and data management to sustain high-quality registries, along with engagement of stakeholders to maintain a co-ordinated research agenda, are essential for successful CER in rheumatology.
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Pesquisa Comparativa da Efetividade/métodos , Doenças Reumáticas/terapia , Reumatologia/métodos , Humanos , Avaliação de Resultados em Cuidados de Saúde , Projetos de Pesquisa , Resultado do TratamentoRESUMO
OBJECTIVE: The objective of the present study is to describe the extension of the National Institutes of Health Patient-Reported Outcomes Measurement Information System (PROMIS(®)) pediatric parent proxy-report item banks for parents of children ages 5-7 years, and to investigate differential item functioning (DIF) between the data obtained from parents of 5-7-year-old children with the data obtained from parents of 8-17 year-old children in the original construction of the scales. METHODS: Item response theory (IRT) analyses of DIF were conducted comparing data from the 5-7 age group with data from the established scales for ages 8-17 across 5 generic health domains (physical functioning, pain, fatigue, emotional health, and social health) and asthma. RESULTS: IRT DIF analyses revealed that the majority of the items functioned similarly with responses from parents of younger and older children. A small number of items were removed from the item bank for younger children, and a few items that exhibited statistical DIF were retained in the pools with the caveat that they should not be used in studies that involve comparisons of younger children with older children. CONCLUSIONS: The study confirms that most of the items in the PROMIS parent proxy-report item banks can be used with parents of children ages 5-7. It is anticipated that these new scales will have application for younger pediatric populations when pediatric self-report is not feasible.
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Avaliação de Resultados em Cuidados de Saúde , Pais/psicologia , Pediatria/estatística & dados numéricos , Procurador , Perfil de Impacto da Doença , Adolescente , Distribuição por Idade , Asma/terapia , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Modelos Teóricos , National Institutes of Health (U.S.) , Psicometria , Estados UnidosRESUMO
This article provides an introduction to key aspects of outcomes research in pediatric rheumatology, focusing on arthritis. Patient-centered outcomes research addresses questions of interest to multiple stakeholders in order to guide the best health care decisions suited to a particular patient's circumstances and preferences. Discussion includes the importance of maintaining high-quality longitudinal patient registries and use of valid clinical and patient-reported outcome measures. Rapid, reliable translation of research on best practices into clinical care, as facilitated by quality improvement learning networks, leads to timely and meaningful improvement in patient outcomes.
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Artrite Juvenil/terapia , Doenças Autoimunes/terapia , Avaliação de Resultados em Cuidados de Saúde/métodos , Pediatria/métodos , Sistema de Registros , Reumatologia/métodos , Criança , Humanos , Avaliação de Resultados da Assistência ao PacienteAssuntos
Artrite Juvenil/tratamento farmacológico , Produtos Biológicos/efeitos adversos , Hospedeiro Imunocomprometido , Imunossupressores/efeitos adversos , Programas de Rastreamento/normas , Reumatologia/normas , Sociedades Médicas/normas , Tuberculose/diagnóstico , Artrite Juvenil/imunologia , Criança , Humanos , Valor Preditivo dos Testes , Fatores de Risco , Tuberculose/imunologia , Tuberculose/microbiologia , Estados UnidosRESUMO
OBJECTIVE: To create upper-extremity and mobility subdomain scores from the Patient-Reported Outcomes Measurement Information System (PROMIS) physical functioning adult item bank. DESIGN: Expert reviews were used to identify upper-extremity and mobility items from the PROMIS item bank. Psychometric analyses were conducted to assess empirical support for scoring upper-extremity and mobility subdomains. SETTING: Data were collected from the U.S. general population and multiple disease groups via self-administered surveys. PARTICIPANTS: The sample (N=21,773) included 21,133 English-speaking adults who participated in the PROMIS wave 1 data collection and 640 Spanish-speaking Latino adults recruited separately. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: We used English- and Spanish-language data and existing PROMIS item parameters for the physical functioning item bank to estimate upper-extremity and mobility scores. In addition, we fit graded response models to calibrate the upper-extremity items and mobility items separately, compare separate to combined calibrations, and produce subdomain scores. RESULTS: After eliminating items because of local dependency, 16 items remained to assess upper extremity and 17 items to assess mobility. The estimated correlation between upper extremity and mobility was .59 using existing PROMIS physical functioning item parameters (r=.60 using parameters calibrated separately for upper-extremity and mobility items). CONCLUSIONS: Upper-extremity and mobility subdomains shared about 35% of the variance in common, and produced comparable scores whether calibrated separately or together. The identification of the subset of items tapping these 2 aspects of physical functioning and scored using the existing PROMIS parameters provides the option of scoring these subdomains in addition to the overall physical functioning score.
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Pessoas com Deficiência/reabilitação , Avaliação de Resultados em Cuidados de Saúde , Adulto , Criança , Doença Crônica , Análise Fatorial , Humanos , Movimento , Aptidão Física , PsicometriaRESUMO
PURPOSE: This paper reports on the development and psychometric properties of self-reported pediatric fatigue item banks as part of the Patient-Reported Outcomes Measurement Information System (PROMIS). METHODS: Candidate items were developed by using PROMIS qualitative methodology. The resulting 39 items (25 tiredness related and 14 energy related) were field tested in a sample that included 3,048 participants aged 8-17 years. We used confirmatory factor analysis (CFA) to evaluate dimensionality and differential item functioning (DIF) analysis to evaluate parameter stability between genders and by age; we examined residual correlations to evaluate local dependence (LD) among items and estimated the parameters of item response theory (IRT) models. RESULTS: Of 3,048 participants, 48 % were males, 60 % were white, and 23 % had at least one chronic condition. CFA results suggest two moderately correlated factors. Two items were removed due to high LD, and three due to gender-based DIF. Two item banks were calibrated separately using IRT: Tired and (Lack of) Energy, which consisted of 23 and 11 items, respectively; 10- and 8-item short-forms were created. CONCLUSION: The PROMIS assessment of self-reported fatigue in pediatrics includes two item banks: Tired and (Lack of) Energy. Both demonstrated satisfactory psychometric properties and can be used for research settings.
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Fadiga , Psicometria/instrumentação , Adolescente , Criança , Análise Fatorial , Fadiga/fisiopatologia , Fadiga/psicologia , Feminino , Humanos , Masculino , Modelos Teóricos , Qualidade de Vida , Autorrelato , Inquéritos e QuestionáriosRESUMO
OBJECTIVE: To evaluate the comparative effectiveness of nonbiologic disease-modifying antirheumatic drugs (DMARD) versus biologic DMARD (bDMARD) for treatment of rheumatoid arthritis (RA), using 2 common analytic approaches. METHODS: We analyzed change in Clinical Disease Activity Index (CDAI) scores in patients with RA enrolled in a US-based observational registry from 2001 to 2008 using multivariable (MV) regression and propensity score (PS) matching. Among patients who initiated treatment with a nonbiologic DMARD (n = 1729), we compared patients who switched to, or added, another nonbiologic (n = 182) or a bDMARD (n = 342) at 5, 9, and 24 months after treatment change. RESULTS: Both analytic approaches showed that patients switching to or adding another nonbiologic DMARD demonstrated improvement across 9 and 24 months (both p < 0.001). Both approaches also demonstrated greater improvement in CDAI among recipients of bDMARD relative to a second nonbiologic DMARD at 5 months (p < 0.02). The MV regression approach upheld these results at 9 and 24 months (p < 0.03). In contrast, the PS-matching approach did not show a sustained advantage with bDMARD at these later timepoints, possibly because of lower statistical power and/or lower baseline disease activity in the PS-matched cohort. CONCLUSION: Patients in both treatment groups generally experienced lower CDAI scores across time. Patients switching to bDMARD demonstrated greater improvement than patients switching to nonbiologic DMARD with both analytic approaches at 5 months. Relative advantages with bDMARD were observed at 9 and 24 months only with MV regression. These analyses provide a practical example of how findings in comparative effectiveness research can diverge with different methodological approaches.
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Antirreumáticos/uso terapêutico , Artrite Reumatoide/tratamento farmacológico , Produtos Biológicos/uso terapêutico , Pesquisa Comparativa da Efetividade/métodos , Índice de Gravidade de Doença , Adulto , Idoso , Artrite Reumatoide/fisiopatologia , Estudos de Coortes , Feminino , Seguimentos , Humanos , Masculino , Pessoa de Meia-Idade , Pontuação de Propensão , Sistema de Registros , Análise de Regressão , Resultado do TratamentoRESUMO
Patient-reported outcomes (PRO) are questionnaire measures of patients' symptoms, functioning, and health-related quality of life. They are designed to provide important clinical information that generally cannot be captured with objective medical testing. In 2004, the National Institutes of Health launched a research initiative to improve the clinical research enterprise by developing state-of-the-art PROs. The NIH Patient-Reported Outcomes Measurement System (PROMIS) and Assessment Center are the products of that initiative. Adult, pediatric, and parent-proxy item banks have been developed by using contemporary psychometric methods, yielding rapid, accurate measurements. PROMIS currently provides tools for assessing physical, mental, and social health using short-form and computer-adaptive testing methods. The PROMIS tools are being adopted for use in clinical trials and translational research. They are also being introduced in clinical medicine to assess a broad range of disease outcomes. Recent legislative developments in the United States support greater efforts to include patients' reports of health experience in order to evaluate treatment outcomes, engage in shared decision-making, and prioritize the focus of treatment. PROs have garnered increased attention by the Food and Drug Administration (FDA) for evaluating drugs and medical devices. Recent calls for comparative effectiveness research favor inclusion of PROs. PROs could also potentially improve quality of care and disease outcomes, provide patient-centered assessment for comparative effectiveness research, and enable a common metric for tracking outcomes across providers and medical systems.
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OBJECTIVES: To examine the content validity of the Patient-Reported Outcomes Measurement Information System pediatric measures, including the pain interference scale, among children and adolescents (aged 8-18 years) who experience chronic pain. To describe children's understandings of the health domain constructs and elucidate verbal and conceptual aspects of self-reported pain-related functioning, which shape disclosure and reporting. METHODS: 34 children and youth with diagnoses of juvenile idiopathic arthritis or noninflammatory chronic pain completed semistructured and cognitive interviews exploring the meaning, experience, and expression of up to 4 of the Patient-Reported Outcomes Measurement Information System pediatric domains: anger, anxiety, depressive symptoms, fatigue, pain interference, and peer relationships. Team-based thematic and content analyses were conducted. RESULTS: Clear verbal and social-cognitive differences were observed in representations and accounts of the domain-experiences across age-groups, but we noted little, if any, evidence of problems with content validity. CONCLUSIONS: Findings suggest the importance of a rigorous developmental approach for understanding the verbal and cognitive dimensions of pediatric self-reports and patient-reported outcomes.
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Adaptação Psicológica , Dor Crônica/psicologia , Emoções , Saúde Mental , Autorrevelação , Adolescente , Afeto , Ira , Criança , Depressão/psicologia , Feminino , Nível de Saúde , Humanos , Masculino , Pesquisa Qualitativa , Qualidade de Vida , AutorrelatoRESUMO
OBJECTIVE: To characterize disease-modifying antirheumatic drug (DMARD) use for children with juvenile idiopathic arthritis (JIA) in the United States and to determine patient factors associated with medication use. METHODS: We analyzed cross-sectional baseline enrollment data from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry from May 2010 through May 2011 for children with JIA. Current and prior medication use was included. We used parsimonious backward stepwise logistic regression models to calculate OR to estimate associations between clinical patient factors and medication use. RESULTS: We identified 2748 children with JIA with a median disease duration of 3.9 years from 51 US clinical sites. Overall, 2023 (74%) had ever received a nonbiologic DMARD and 1246 (45%) had ever received a biologic DMARD. Among children without systemic arthritis, methotrexate use was most strongly associated with uveitis (OR 5.2, 95% CI 3.6-7.6), anticitrullinated protein antibodies (OR 4.5, 95% CI 1.7-12), and extended oligoarthritis (OR 4.1, 95% CI 2.5-6.6). Among children without systemic arthritis, biologic DMARD use was most strongly associated with rheumatoid factor (RF)-positive polyarthritis (OR 4.3, 95% CI 2.9-6.6), psoriatic arthritis (PsA; OR 3.0, 95% CI 2.0-4.4), and uveitis (OR 2.8, 95% CI 2.1-3.7). Among children with systemic arthritis, 160 (65%) ever received a biologic DMARD; tumor necrosis factor inhibitor use was associated with polyarthritis (OR 2.5, 95% CI 3.8-16), while interleukin 1 inhibitor use was not. CONCLUSION: About three-quarters of all children with JIA in the CARRA Registry received nonbiologic DMARD. Nearly one-half received biologic DMARD, and their use was strongly associated with RF-positive polyarthritis, PsA, uveitis, and systemic arthritis.
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Antirreumáticos/uso terapêutico , Artrite Juvenil/tratamento farmacológico , Fator de Necrose Tumoral alfa/antagonistas & inibidores , Adalimumab , Adolescente , Anticorpos Monoclonais/uso terapêutico , Anticorpos Monoclonais Humanizados/uso terapêutico , Criança , Pré-Escolar , Estudos Transversais , Feminino , Humanos , Infliximab , Masculino , Metotrexato/uso terapêutico , Padrões de Prática Médica , Sistema de RegistrosRESUMO
OBJECTIVES: Transport of ions to generate epithelial rehydration (TIGER)-1 was a randomized trial conducted to evaluate the safety and efficacy of denufosol versus placebo in patients with cystic fibrosis with mild impairment in lung function. The trial met its primary end point at 24 weeks, but a subsequent trial did not show a sustained effect of denufosol at 48 weeks. By using the 48-week data, we characterized resource use, direct medical costs, indirect costs, and utility estimates. METHODS: Data on medications, outpatient and emergency visits, hospital admissions, tests, procedures, and home nursing were captured on study case report forms. Sources for unit costs included the Medicare Physician Fee Schedule, the Nationwide Inpatient Sample, and the Red Book. Health utilities were derived from the Health Utilities Index Mark 2/3. We used multivariable regression to evaluate the impact of baseline covariates on costs. RESULTS: Characteristics of the 352 participants at enrollment included mean age of 14.6 years, history of Pseudomonas aeruginosa colonization in 45.2%, use of dornase alfa in 77.0%, and long-term use of inhaled antibiotics in 37.2%. Over 48 weeks, 22.4% of participants were hospitalized and, on average, participants missed 7.4 days of school or work. Mean total costs (excluding denufosol) were $39,673 (SD $26,842), of which 85% were attributable to medications. Female sex and P. aeruginosa colonization were independently associated with higher costs. CONCLUSIONS: Prospective economic data collection alongside a clinical trial allows for robust estimates of cost of illness. The mean annual cost of care for patients with cystic fibrosis with mild impairment in lung function exceeds $43,000 and is driven by medication costs.