The evaluation of efficacy in clinical trials in pediatric rheumatology have been classically based on physician’s centered measures and on patient’s reported outcome (PRO) The efficacy measures could then be divided into those who could be applied across diseases such as the physician’s evaluation of disease activity usually on a 10 cm Visual Analogue Scale (VAS) or on a 21 circle VAS, the functional ability tools such as the Childhood Health Assessment Questionnaire (CHAQ) or the evaluation of health related quality of life (HRQOL) such as the Child Health Questionnaire (CHQ), and parent’s/patients global assessment of overall well-being and pain usually on VAS and more recent newer tools such as the Juvenile Arthritis Multidimensional Assessment Report (JAMAR). For the disease specific measures we have for juvenile idiopathic arthritis (JIA) the classic rheumatologic examination with the evaluation of the number of joints with swelling, pain and limitation on motion as well as the number of active joints, and for damage the Juvenile Arthritis Damage Index (JADI). For juvenile dermatomyositis (JDM) the evaluation of muscle strength through the Childhood Myositis Assessment Scale (CMAS) or the Manual Muscle Testing (MMT), the evaluation of disease activity through the Disease Activity Score (DAS) or the Myositis Disease Activity Assessment Tool (MDAA), and for damage the Myositis Damage Index (MDI). For juvenile systemic lupus erythematosus (JSLE) the evaluation of disease activity through, the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), The Systemic Lupus Activity Measure (SLAM), European Consensus Lupus Activity Measurement (ECLAM) and for damage the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Damage Index (SLICC/ACR DI). Finally there are objective measures or laboratory indicators such as the index of inflammation (ESR or CRP) for JIA, the muscle enzymes for JDM and proteinuria or urine protein/creatinine ration for JSLE.
Most of these measures have a correspondent tool to be applied when a child transition to adulthood (for the CHAQ the HAQ, for the CHQ the SF-36) and most are used in the same format for both children and adults.
This lecture will describe the current scenario of the measures that could facilitate transition from childhood to adulthood.
Disclosure of Interest None Declared
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