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BACKGROUND: Childhood interstitial lung disease (chILD) encompasses a group of rare heterogeneous respiratory conditions associated with significant morbidity and mortality. Reports suggest that many patients diagnosed with chILD continue to have potentially progressive or fibrosing disease into adulthood. Over the last decade, the spectrum of conditions within chILD has widened substantially, with the discovery of novel entities through advanced genetic testing. However, most evidence is often limited to small case series, with reports disseminated across an array of subspecialty, clinical and molecular journals. In particular, the frequency, management and outcome of paediatric pulmonary fibrosis is not well characterised, unlike in adults, where clear diagnosis and treatment guidelines are available. METHODS AND RESULTS: This review assesses the current understanding of pulmonary fibrosis in chILD. Based on registry data, we have provisionally estimated the occurrence of fibrosis in various manifestations of chILD, with 47 different potentially fibrotic chILD entities identified. Published evidence for fibrosis in the spectrum of chILD entities is assessed, and current and future issues in management of pulmonary fibrosis in childhood, continuing into adulthood, are considered. CONCLUSIONS: There is a need for improved knowledge of chILD among pulmonologists to optimise the transition of care from paediatric to adult facilities. Updated evidence-based guidelines are needed that incorporate recommendations for the diagnosis and management of immune-mediated disorders, as well as chILD in older children approaching adulthood.
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Calcinosis is a sequela of Juvenile Dermatomyositis (JDM) with significant morbidity. A retrospective study observing risk factors for JDM calcinosis, including a possible association between higher intensity of subcutaneous and myofascial edema in initial magnetic resonance imaging (MRI) and development of calcinosis was performed at a tertiary pediatric medical center. Data from the past 20 years on JDM patients with MRIs at the time of JDM diagnosis were obtained. MRIs were individually evaluated by two pediatric musculoskeletal radiologists who blindly graded the intensity of edema on a 0-4 Likert scale. Clinical data and edema scores were compared between patients who developed calcinosis and who did not. Forty-three patients (14 with calcinosis and 29 without calcinosis) were identified. The calcinosis group contained more racial and ethnic minorities, younger ages of JDM onset and longer time to reach JDM diagnosis. Muscle enzyme levels at JDM diagnosis were lower in the calcinosis group, especially Creatinine Kinase (CK) (p = 0.047) and Alanine Aminotransferase (ALT) (p = 0.015). The median score for edema in both groups was 3 (p = 0.39) with an inter-rater reliability of 95%. There was no association between increased subcutaneous and myofascial edema in MRIs at the time of JDM diagnosis and development of calcinosis. Earlier age of JDM onset, racial and ethnic minority, and delay in JDM diagnosis could be risks for developing calcinosis. The calcinosis group presented with lower muscle enzyme levels at the time of JDM diagnosis, especially CK and ALT with statistical significance. This could reflect delay in diagnosis and treatment.
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Childhood interstitial lung disease (chILD) comprises a spectrum of rare diffuse lung disorders. chILD is heterogeneous in origin, with different disease manifestations occurring in the context of ongoing lung development. The large number of disorders in chILD, in combination with the rarity of each diagnosis, has hampered scientific and clinical progress within the field. Epidemiologic and natural history data are limited. The prognosis varies depending on the etiology, with some forms progressing to lung transplant or death. There are limited treatment options for patients with chILD. Although U.S. Food and Drug Administration-approved treatments are now available for adult patients with idiopathic pulmonary fibrosis, no clinical trials have been conducted in a pediatric population using agents designed to treat lung fibrosis. This review will focus on progressive chILD disorders and on the urgent need for meaningful objective outcome measures to define, detect, and monitor fibrosis in children.
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Fibrose Pulmonar Idiopática/diagnóstico , Fibrose Pulmonar Idiopática/terapia , Doenças Pulmonares Intersticiais/diagnóstico , Doenças Pulmonares Intersticiais/terapia , Criança , Pré-Escolar , Ensaios Clínicos como Assunto , Humanos , Projetos de PesquisaRESUMO
OBJECTIVE: To investigate the characteristics and risk factors of a novel parenchymal lung disease (LD), increasingly detected in systemic juvenile idiopathic arthritis (sJIA). METHODS: In a multicentre retrospective study, 61 cases were investigated using physician-reported clinical information and centralised analyses of radiological, pathological and genetic data. RESULTS: LD was associated with distinctive features, including acute erythematous clubbing and a high frequency of anaphylactic reactions to the interleukin (IL)-6 inhibitor, tocilizumab. Serum ferritin elevation and/or significant lymphopaenia preceded LD detection. The most prevalent chest CT pattern was septal thickening, involving the periphery of multiple lobes ± ground-glass opacities. The predominant pathology (23 of 36) was pulmonary alveolar proteinosis and/or endogenous lipoid pneumonia (PAP/ELP), with atypical features including regional involvement and concomitant vascular changes. Apparent severe delayed drug hypersensitivity occurred in some cases. The 5-year survival was 42%. Whole exome sequencing (20 of 61) did not identify a novel monogenic defect or likely causal PAP-related or macrophage activation syndrome (MAS)-related mutations. Trisomy 21 and young sJIA onset increased LD risk. Exposure to IL-1 and IL-6 inhibitors (46 of 61) was associated with multiple LD features. By several indicators, severity of sJIA was comparable in drug-exposed subjects and published sJIA cohorts. MAS at sJIA onset was increased in the drug-exposed, but was not associated with LD features. CONCLUSIONS: A rare, life-threatening lung disease in sJIA is defined by a constellation of unusual clinical characteristics. The pathology, a PAP/ELP variant, suggests macrophage dysfunction. Inhibitor exposure may promote LD, independent of sJIA severity, in a small subset of treated patients. Treatment/prevention strategies are needed.
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Artrite Juvenil/complicações , Pneumopatias/epidemiologia , Pulmão/diagnóstico por imagem , Biópsia , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Incidência , Lactente , Pneumopatias/diagnóstico , Pneumopatias/etiologia , Masculino , Prognóstico , Estudos Retrospectivos , Taxa de Sobrevida/tendências , Tomografia Computadorizada por Raios X , Estados Unidos/epidemiologiaRESUMO
OBJECTIVES: To investigate the burden of systemic juvenile idiopathic arthritis (SJIA) on health-related quality of life (HRQOL) and resource use of patients and caregivers (families) on biologic therapy. METHODS: This international study assessed SJIA burden in patients on biologics, using a caregiver questionnaire and retrospective chart review. Validated measures included: Child Health Questionnaire Parent-Form 50 (CHQ-PF50), 36-Item Short-Form Health Survey (SF-36v2) and Work Productivity and Activity Impairment questionnaire: Specific Health Problem (WPAI:SHP). Caregivers completed function, treatment satisfaction and resource utilisation questions. RESULTS: Sixty-one biologic treated patients participated (12 anakinra, 25 canakinumab, 24 tocilizumab). Mean age at diagnosis and survey completion was 6.4 and 11.3 years, respectively. Mean (±SD: standard deviation) CHQ-PF50 physical (PhS) and psychosocial (PsS) summary scores were significantly lower in SJIA patients than a normative population (PhS: 40.0±18.2 vs. 53.0±8.8; PsS: 46.6±11.3 vs. 51.2±9.1) as was caregivers' mean SF-36v2 mental component score (MCS; 46.2±10.7 vs. 50.0±10). Assistive devices were required by 54%; 20% required home/car alterations. According to caregivers, biologic treatment completely improved SJIA symptoms in 48% on canakinumab or tocilizumab and 32% on anakinra. Over 2 months, patients missed 2.9 school days due to SJIA (10% yearly loss). Caregivers lost 25 work days annually and 27.5 days of productivity (WPAI-SHP: mean absenteeism 10%; presenteeism 11%). Yearly SJIA travel/treatment costs averaged $1,130. CONCLUSIONS: SJIA patients on biologic therapy experience HRQOL impairment, caregivers' mental well-being suffers and productivity losses and expenses are incurred. Therapeutic interventions that reduce the burden of SJIA are required.
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Antirreumáticos/uso terapêutico , Artrite Juvenil/tratamento farmacológico , Produtos Biológicos/uso terapêutico , Cuidadores/psicologia , Efeitos Psicossociais da Doença , Qualidade de Vida , Absenteísmo , Antirreumáticos/efeitos adversos , Antirreumáticos/economia , Artrite Juvenil/economia , Artrite Juvenil/epidemiologia , Artrite Juvenil/psicologia , Produtos Biológicos/efeitos adversos , Produtos Biológicos/economia , Criança , Estudos Transversais , Custos de Medicamentos , Eficiência , Emprego/economia , Europa (Continente)/epidemiologia , Feminino , Gastos em Saúde , Inquéritos Epidemiológicos , Humanos , Masculino , Presenteísmo/economia , Indução de Remissão , Estudos Retrospectivos , Resultado do Tratamento , Estados Unidos/epidemiologiaRESUMO
PURPOSE OF REVIEW: The present review intends to provide an overview of the diversity and complexity of pulmonary manifestations of rheumatologic diseases and gaps in knowledge to effectively manage them. RECENT FINDINGS: Diffuse lung disease in children with rheumatologic diseases represents a heterogeneous group of autoimmune disorders. Despite their significant morbidity and mortality, we have limited understanding about their pathogenesis. Here, we provide an overview of the pathophysiology and current management approach of these disorders, highlighting tools which assist with diagnosis, risk stratification and therapy. In this context, we address the need to develop a standardized approach to diagnose at-risk patients with rheumatologic disease and to predict their progression and the need to develop robust studies which evaluate the factors and interventions that influence pulmonary disease outcome. SUMMARY: Diffuse lung disease in children with rheumatologic diseases represents a heterogeneous group of severe autoimmune disorders. By adopting a collaborative research approach among multicenters to help diagnose, risk stratify, and understand disease progression, effective management decisions can be optimized to improve clinical outcome.
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Doenças Autoimunes/etiologia , Pneumopatias/etiologia , Doenças Reumáticas/fisiopatologia , Doenças Autoimunes/diagnóstico , Doenças Autoimunes/epidemiologia , Doenças Autoimunes/terapia , Criança , Humanos , Pneumopatias/diagnóstico , Pneumopatias/epidemiologia , Pneumopatias/terapia , Prevalência , Doenças Reumáticas/imunologia , Medição de RiscoRESUMO
Starting in 2015, pediatric rheumatology fellowship training programs were required by the Accreditation Council for Graduate Medical Education to assess fellows' academic performance within 21 subcompetencies falling under six competency domains. Each subcompetency had four or five milestone levels describing developmental progression of knowledge and skill acquisition. Milestones were standardized across all pediatric subspecialties. As part of the Milestones 2.0 revision project, the Accreditation Council for Graduate Medical Education convened a workgroup in 2022 to write pediatric rheumatology-specific milestones. Using adult rheumatology's Milestones 2.0 as a starting point, the workgroup revised the patient care and medical knowledge subcompetencies and milestones to reflect requirements and nuances of pediatric rheumatology care. Milestones within four remaining competency domains (professionalism, interpersonal and communication skills, practice-based learning and improvement, and systems-based practice) were standardized across all pediatric subspecialties, and therefore not revised. The workgroup created a supplemental guide with explanations of the intent of each subcompetency, 25 in total, and examples for each milestone level. The new milestones are an important step forward for competency-based medical education in pediatric rheumatology. However, challenges remain. Milestone level assignment is meant to be informed by results of multiple assessment methods. The lack of pediatric rheumatology-specific assessment tools typically result in clinical competency committees determining trainee milestone levels without such collated results as the foundation of their assessments. Although further advances in pediatric rheumatology fellowship competency-based medical education are needed, Milestones 2.0 importantly establishes the first pediatric-specific rheumatology Milestones to assess fellow performance during training and help measure readiness for independent practice.
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Competência Clínica , Educação de Pós-Graduação em Medicina , Bolsas de Estudo , Pediatria , Reumatologia , Reumatologia/educação , Reumatologia/normas , Humanos , Competência Clínica/normas , Educação de Pós-Graduação em Medicina/normas , Pediatria/educação , Pediatria/normasRESUMO
BACKGROUND: After introducing IL-1/IL-6 inhibitors, some patients with Still and Still-like disease developed unusual, often fatal, pulmonary disease. This complication was associated with scoring as DReSS (drug reaction with eosinophilia and systemic symptoms) implicating these inhibitors, although DReSS can be difficult to recognize in the setting of systemic inflammatory disease. OBJECTIVE: To facilitate recognition of IL-1/IL-6 inhibitor-DReSS in systemic inflammatory illnesses (Still/Still-like) by looking at timing and reaction-associated features. We evaluated outcomes of stopping or not stopping IL-1/IL-6 inhibitors after DReSS reaction began. METHODS: In an international study collaborating primarily with pediatric specialists, we characterized features of 89 drug-reaction cases versus 773 drug-exposed controls and compared outcomes of 52 cases stopping IL-1/IL-6 inhibitors with 37 cases not stopping these drugs. RESULTS: Before the reaction began, drug-reaction cases and controls were clinically comparable, except for younger disease-onset age for reaction cases with preexisting cardiothoracic comorbidities. After the reaction began, increased rates of pulmonary complications and macrophage activation syndrome differentiated drug-reaction cases from drug-tolerant controls (P = 4.7 × 10-35 and P = 1.1 × 10-24, respectively). The initial DReSS feature was typically reported 2 to 8 weeks after initiating IL-1/IL-6 inhibition. In drug-reaction cases stopping versus not stopping IL-1/IL-6-inhibitor treatment, reaction-related features were indistinguishable, including pulmonary complication rates (75% [39 of 52] vs 76% [28 of 37]). Those stopping subsequently required fewer medications for treatment of systemic inflammation, had decreased rates of macrophage activation syndrome, and improved survival (P = .005, multivariate regression). Resolution of pulmonary complications occurred in 67% (26 of 39) of drug-reaction cases who stopped and in none who continued inhibitors. CONCLUSIONS: In systemic inflammatory illnesses, recognition of IL-1/IL-6-inhibitor-associated reactions followed by avoidance of IL-1/IL-6 inhibitors significantly improved outcomes.
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COVID-19 hypoxemic patients although sharing a same etiology (SARS-CoV-2 infection) present themselves quite differently from one another. Patients also respond differently to prescribed medicine and to prone Vs supine bed positions. A severe pulmonary ventilation-perfusion mismatch usually triggers moderate to severe COVID-19 cases. Imaging can aid the physician in assessing severity of COVID-19. Although useful for their portability X-ray and ultrasound serving on the frontline to evaluate lung parenchymal abnormalities are unable to provide information about pulmonary vasculature and blood flow redistribution which is a consequence of hypoxemia in COVID-19. Advanced imaging modalities such as computed tomography, single-photon emission tomography, and electrical impedance tomography use a sharp algorithm visualizing pulmonary ventilation-perfusion mismatch in the abnormal and in the apparently normal parenchyma. Imaging helps to access the severity of infection, lung performance, ventilation-perfusion mismatch, and informs strategies for medical treatment. This review summarizes the capacity of these imaging modalities to assess ventilation-perfusion mismatch in COVID-19. Despite having limitations, these modalities provide vital information on blood volume distribution, pulmonary embolism, pulmonary vasculature and are useful to assess severity of lung disease and effectiveness of treatment in COVID-19 patients.
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COVID-19 , Embolia Pulmonar , COVID-19/diagnóstico por imagem , Humanos , Pulmão/diagnóstico por imagem , Perfusão , SARS-CoV-2 , Tomografia Computadorizada de Emissão de Fóton Único/métodosRESUMO
Diffuse alveolar hemorrhage often presents as dyspnea, cough, or hemoptysis, and it is mediated by both immune and nonimmune processes. Isolated pauci-immune capillaritis (IPPC) is a rare diagnosis in which capillaritis, small-vessel vasculitis of the lung, is found on biopsy in the absence of an underlying systemic disorder. Traditionally, IPPC has been treated similarly to anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis with cyclophosphamide and glucocorticoids. However, few cases describing management options are available in the literature, especially among pediatric patients. Our report of successful induction of remission in an adolescent girl suggests that the combination of IV rituximab and pulse methylprednisolone may be a viable option for disease control in pediatric patients with IPPC.
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Hemoptise , Doenças Pulmonares Intersticiais , Metilprednisolona/administração & dosagem , Rituximab/administração & dosagem , Vasculite , Adolescente , Capilares/patologia , Quimioterapia Combinada , Dispneia/diagnóstico , Dispneia/etiologia , Feminino , Hemoptise/diagnóstico , Hemoptise/etiologia , Hemoptise/terapia , Humanos , Fatores Imunológicos/administração & dosagem , Pulmão/diagnóstico por imagem , Doenças Pulmonares Intersticiais/diagnóstico , Doenças Pulmonares Intersticiais/etiologia , Doenças Pulmonares Intersticiais/fisiopatologia , Doenças Pulmonares Intersticiais/terapia , Pulsoterapia/métodos , Indução de Remissão/métodos , Tomografia Computadorizada por Raios X/métodos , Resultado do Tratamento , Vasculite/complicações , Vasculite/diagnóstico , Vasculite/fisiopatologia , Vasculite/terapiaRESUMO
e-Learning has become a popular medium for delivering instruction in medical education. This innovative method of teaching offers unique learning opportunities for medical trainees. The purpose of this article is to define the present state of e-learning in pediatrics and how to best leverage e-learning for educational effectiveness and change in medical education. Through addressing under-examined and neglected areas in implementation strategies for e-learning, its usefulness in medical education can be expanded. This study used a systematic database review of published studies in the field of e-learning in pediatric training between 2003 and 2013. The search was conducted using educational and health databases: Scopus, ERIC, PubMed, and search engines Google and Hakia. A total of 72 reference articles were suitable for analysis. This review is supplemented by the use of "e-Learning Design Screening Questions" to define e-learning design and development in 10 randomly selected articles. Data analysis used template-based coding themes and counting of the categories using descriptive statistics.Our search for pediatric e-learning (using Google and Hakia) resulted in six well-defined resources designed to support the professional development of doctors, residents, and medical students. The majority of studies focused on instructional effectiveness and satisfaction. There were few studies about e-learning development, implementation, and needs assessments used to identify the institutional and learners' needs. Reviewed studies used various study designs, measurement tools, instructional time, and materials for e-learning interventions. e-Learning is a viable solution for medical educators faced with many challenges, including (1) promoting self-directed learning, (2) providing flexible learning opportunities that would offer continuous (24h/day/7 days a week) availability for learners, and (3) engaging learners through collaborative learning communities to gain significant learning and augment continuous professional development. Several important recommendations for faculty instructors interested in providing and/or improving e-learning activities for today's learners are detailed.
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Instrução por Computador/tendências , Educação Médica/tendências , Aprendizagem , Pediatria , Ensino , Competência Clínica , Instrução por Computador/normas , Educação Médica/normas , Medicina Baseada em Evidências , Humanos , Internet , Pediatria/educação , Desenvolvimento de Pessoal , Ensino/métodos , Ensino/tendênciasRESUMO
OBJECTIVE: To examine trends in the specialty care hospitalization of pediatric rheumatology patients and determine how nonclinical factors influence access. METHODS: This study used California's Office of Statewide Health Planning and Development discharge database to perform a retrospective population analysis of pediatric rheumatology hospitalizations in California between 1999 and 2007. We used logistic regression to examine the relationship between hospitalization in specialty care centers with a pediatric rheumatologist and nonclinical patient characteristics. RESULTS: A total of 18,641 pediatric discharges revealed that 57% were discharged from a specialty care center with a pediatric rheumatologist. Multivariate analysis showed that the factors associated with increased utilization of specialty care centers with a pediatric rheumatologist were public insurance (odds ratio [OR] 1.62, 95% confidence interval [95% CI] 1.51-1.74; P < 0.0001), being Hispanic (OR 1.29, 95% CI 1.19-1.40; P < 0.0001) or Asian non-Hispanic (OR 1.39, 95% CI 1.26-1.54; P < 0.0001), and high pediatric rheumatology specialty care bed supply (OR 2.79, 95% CI 2.49-3.14; P < 0.0001). A decreased utilization of specialty care centers with a pediatric rheumatologist was seen for patients ages <1 year (OR 0.45, 95% CI 0.40-0.52; P < 0.0001), ages 1-4 years (OR 0.50, 95% CI 0.46-0.55; P < 0.0001), ages 5-9 years (OR 0.68, 95% CI 0.62-0.75; P < 0.0001), ages 15-18 years (OR 0.51, 95% CI 0.47-0.56; P < 0.0001), males (OR 0.75, 95% CI 0.70-0.80; P < 0.0001), and patients residing farther away from a specialty care center with a pediatric rheumatologist (OR 0.57, 95% CI 0.51-0.63; P < 0.0001). CONCLUSION: Nonclinical factors play an increasingly important role in the hospitalization patterns of pediatric rheumatology patients in California. Understanding these factors is crucial if we are to ensure that the variation in access to care reflects clinical need.