What drives the decision to optimise biological treatment in children and youngsters with juvenile idiopathic arthritis? A discrete-choice experiment.

OBJECTIVE: To analyse factors involved in the decision to optimise biologics in juvenile idiopathic arthritis. METHODS: A "discrete-choice" methodology was used. In a nominal group meeting, factors which may influence physicians' decisions to optimise biological dose were identified, together with decision nodes. 1000Minds® was used to create multiple fictitious clinical scenarios based on the factors identified, and to deploy surveys that were sent to a panel of experts. These experts decided for each item which of two clinical scenarios prompted them to optimise the dose of biologic. A conjoint analysis was carried out, and the partial-value functions and the weights of relative importance calculated. RESULTS: In the nominal group, three decision nodes were identified: (1) time to decide; (2) to maintain/reduce or prolong interval; (3) what drug to reduce. The factors elicited were different for each node and included patient and drug attributes. The presence of macrophage activation syndrome (MAS), systemic involvement, or subclinical inflammation made the decision easier (highest weights). The presence of joints of difficult control and year of debut influenced the decision in some but not all, and in different directions. Immunogenicity, adherence, and concomitant treatments were also aspects taken into account. CONCLUSIONS: The decision to optimise the dose of biological therapy in children and youngster can be divided into several nodes, and the factors, both patient and therapy-related, leading to the decision can be detailed. These decisions taken by experts may be transported to practice, study designs, and guidelines.

Risk scores for Kawasaki disease, a management tool developed by the KAWA-RACE cohort.

INTRODUCTION/OBJECTIVES: Asian scores developed to predict unresponsiveness to intravenous immunoglobulin (IVIG) or development of coronary artery aneurysms (CAA) in patients with Kawasaki disease (KD) are not appropriate in Western populations. The purpose of this study is to develop 2 scores, to predict unresponsiveness to IVIG and development of CAA, appropriate for Spanish population. METHOD: Data of 625 Spanish children with KD collected retrospectively (2011-2016) were used to identify variables to develop the 2 scores of interest: unresponsiveness to IVIG and development of CAA. A statistical model selected best variables to create the scores, and scores were validated with data from 98 patients collected prospectively. RESULTS: From 625 patients of the retrospective cohort, final analysis was performed in 439 subjects: 37 developed CAA, and 212 were unresponsive to IVIG. For the score to predict CAA, a cutoff ≥ 8 was considered for high risk, considering a score system with a different weight for each of the eight variables. External validation showed a sensitivity of 22% and a specificity of 75%. The score to predict unresponsiveness to IVIG established a cutoff ≥ 8 for high risk, considering a score system with a different weight for each of the nine variables. External validation showed a sensitivity of 78% and a specificity of 50%. CONCLUSIONS: Two risk scores for KD were developed from Spanish population, to predict development of CAA and unresponsiveness to IVIG; validation in other cohorts could help to implement these tools in the management of KD in other Western populations.

Consensus-based recommendations for the management of juvenile localised scleroderma.

In 2012, a European initiative called Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) was launched to optimise and disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic diseases. Juvenile localised scleroderma (JLS) is a rare disease within the group of paediatric rheumatic diseases (PRD) and can lead to significant morbidity. Evidence-based guidelines are sparse and management is mostly based on physicians' experience. This study aims to provide recommendations for assessment and treatment of JLS. Recommendations were developed by an evidence-informed consensus process using the European League Against Rheumatism standard operating procedures. A committee was formed, mainly from Europe, and consisted of 15 experienced paediatric rheumatologists and two young fellows. Recommendations derived from a validated systematic literature review were evaluated by an online survey and subsequently discussed at two consensus meetings using a nominal group technique. Recommendations were accepted if ≥80% agreement was reached. In total, 1 overarching principle, 10 recommendations on assessment and 6 recommendations on therapy were accepted with ≥80% agreement among experts. Topics covered include assessment of skin and extracutaneous involvement and suggested treatment pathways. The SHARE initiative aims to identify best practices for treatment of patients suffering from PRDs. Within this remit, recommendations for the assessment and treatment of JLS have been formulated by an evidence-informed consensus process to produce a standard of care for patients with JLS throughout Europe.

Enfermedad de Kawasaki / Malaltia de Kawasaki / Kawasaki´s disease

La malaltia de Kawasaki (MK) és una vasculitis sistèmica aguda d'etiologia desconeguda. El diagnòstic es basa en criteris clínics que inclouen febre, exantema, conjuntivitis, canvis en les extremitats, eritema de la mucosa oral i llavis, i adenopaties cervicals. No obstant això, aquests criteris tenen una sensibilitat i una especificitat baixes i, per tant, altres característiques clíniques i de laboratori poden ser útils per establir el diagnòstic, sobretot en els casos d'MK atípica o incompleta. El pronòstic depèn de l'extensió de l'afectació cardíaca; els aneurismes coronaris, que es de-sen volupen en el 20-25% dels pacients no tractats, poden provocar infart de miocardi o mort sobtada en l'edat adulta. El tractament amb altes dosis d'immunoglobulina intrave-nosa és eficaç per reduir el risc d'aneurismes coronaris en la majoria dels casos i és el tractament d'elecció. En aquesta revisió analitzem la clínica, l'epidemiologia i el tractament d'aquesta malaltia típica de l'edat pediàtrica

La enfermedad de Kawasaki (EK) es una vasculitis sistémica aguda de etiología desconocida. El diagnóstico se basa en criterios clínicos que incluyen fiebre, exantema, conjuntivitis, cambios en las extremidades, eritema de la mucosa oral y labios, y adenopatías cervicales. Sin embargo, estos criterios tienen una sensibilidad y una especificidad bajas y, por tanto, otras características clínicas y de laboratorio pueden ser útiles para establecer el diagnóstico, sobre todo en los casos de MK atípica o incompleta. El pronóstico depende de la extensión de la afectación cardiaca; los aneurismas coronarios, que se desarrollan en el 20-25% de los pacientes no tratados, pueden provocar infarto de miocardio o muerte súbita en la edad adulta. El tratamiento con altas dosis de inmunoglobulina intravenosa es eficaz para reducir el riesgo de aneurismas coronarios en la mayoría de los casos y es el tratamiento de elección. En esta revisión analizamos la clínica, la epidemiología y el tratamiento de esta enfermedad típica de la edad pediátrica (AU)

Kawasaki disease (MK) is an acute systemic vasculitis of unknown etiology. The diagnosis is based on clinical criteria that includes fever, rash, conjunctivitis, changes in the limbs, erythema of the oral mucosa and lips, and cervical lymphadenopathy. However, these criteria have a low sensitivity and specificity and, therefore, other clinical and laboratory features may be helpful in establishing the diagnosis, especially in cases of atypical or incomplete MK. The prognosis depends on the extent of heart involvement; coronary aneurysms, which develop in 20-25% of untreated patients can cause a heart attack or sudden death in adulthood. Treatment with high doses of intravenous immunoglobulin is effective to reduce the risk of coronary aneurysms in most cases and is the treatment of choice. In this review we analyze the symptoms, epidemiology and treatment of this disease, typical of paediatric patients (AU)