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Rheumatic Disease
Published in John S. Axford, Chris A. O'Callaghan, Medicine for Finals and Beyond, 2023
There are a variety of different causes of childhood arthritis as listed in Table 4.11. JIA is the principal form of chronic idiopathic synovitis.
Autologous Stem Cell Transplantation for Refractory Juvenile Idiopathic Arthritis (JIA)
Published in Richard K. Burt, Alberto M. Marmont, Stem Cell Therapy for Autoimmune Disease, 2019
A growing number of functional outcome scales have become available for paediatric rheumatology research. Instruments to measure health status and function specifically developed for juvenile arthritis include the Childhood Arthritis Impact Measurement Scales (CHAIMS),18 the Childhood Health Assessment Questionnaire (CHAQ),19 the Juvenile Arthritis Functional Assessment Report (JAFAR),20 the Childhood Arthritis Health Profile (CAHP),21 the Juvenile Arthritis Quality of Life Questionnaire (JAQQ),22 and the Juvenile Arthritis Self Report Index (JASI).23 Of these, the instrument tested most widely is the CHAQ. For evaluation after HSCT, we used the core set of outcome variables for clinical trials in childhood arthritis as proposed by Giannini and the Pediatric Rheumatology International Trials Organisation group (PRINTO)24-27 (Table 3). PRINTO rheumatological outcome measures include a physician’s global assessment of disease activity, the child health assessment questionnaire, CHAQ (a parent/patient assessment of overall well-being, the functional ability and disease severity) (Table 4), the number of joints with active arthritis (Fuchs Swelling Index, FSI), the number of joints with limited range of motion, (EPM-ROM) (Table 5) and the erythrocyte sedimentation rate (ESR). Giannini proposed a 30% improvement from baseline of 3 out of 6 variables, with no more than one of the remaining variables worsening more than 30%.24 The evolution of the disease was followed at 3 month intervals.
Micronutrients in Prevention and Improvement of the Standard Therapy in Arthritis
Published in Kedar N. Prasad, Micronutrients in Health and Disease, 2019
JRA is the most common childhood arthritis. This is considered an autoimmune disease in which immune cells attack healthy cells of the joint causing inflammatory reactions that release toxic chemical species, which cause progressive damage to the joints and their surrounding tissues causing redness, swelling, pain, and stiffness. Some children with JRA outgrow the illness. As a matter of fact, the symptoms of JRA disappear in over about 50% of affected children. JRA may cause chronic fever and anemia and can affect the heart, lungs, eyes, and nervous system. These arthritic episodes may last for several weeks and then may recur with less severe symptoms. This disease can impair bone development and weaken fine motor skills. The incidence of JRA in girls is higher than in boys. Transient JRA may follow certain infections.
Classification and management strategies for paediatric chronic nonbacterial osteomyelitis and chronic recurrent multifocal osteomyelitis
Published in Expert Review of Clinical Immunology, 2023
Shabnam Singhal, Caren Landes, Rajeev Shukla, Liza J McCann, Christian M Hedrich
In the absence of comparative randomized clinical trials (RCTs), to generate evidence on the comparative effectiveness of available treatments, consensus treatment plans (CTPs) have been developed for use by the Childhood Arthritis and Rheumatology Research Alliance (CARRA). Notably, CTPs were developed based on expert consensus and are not intended as treatment recommendations. Patients with CNO/CRMO who are refractory to NSAIDs and/or have active spinal lesions can be enrolled in the CNO/CRMO international register (CHOIR) and treated following one of the three CTPs [88]: A) cDMARDs (methotrexate, sulfasalazine), B) TNFi (adalimumab, etanercept, infliximab) ± methotrexate C) Bisphosphonates (pamidronate, zoledronic acid). Use of glucocorticoids and NSAIDS may be included in each of the three regimens.
Emerging therapies for juvenile arthritis: agents in early clinical trials
Published in Expert Opinion on Investigational Drugs, 2022
Silvia Rosina, Gabriella Giancane, Nicolino Ruperto
Another promoting factor of remarkable improvement in the field has been the availability of excellent networks of pediatric rheumatology, such as the Pediatric Rheumatology Collaborative Study Group (PRCSG, founded in North America in 1973) [100], PRINTO (founded in Italy in 1996 and currently covering 93 countries worldwide), the Childhood Arthritis and Rheumatology Research Alliance (CARRA), the Understanding Childhood Arthritis Network and the CLUSTER consortium. The collaborative efforts among these networks need to be further strengthened to help the designing and implementation of clinical trials, the selection of participating centers as well as trial conduct and analysis, with the common purpose of providing a better future for children with JIA. For the same aim, the formal future involvement of families in prioritizing and planning of clinical trials and investigator-initiated studies by academia, pharmaceutical companies, and regulatory authorities, is also advisable.
Current status and prospects of IL-6–targeting therapy
Published in Expert Review of Clinical Pharmacology, 2022
Masashi Narazaki, Tadamitsu Kishimoto
After the efficacy of IL-6 inhibitory therapy was established for Castleman disease and RA, IL-6 inhibitory therapy was attempted for some refractory inflammatory diseases. sJIA is a chronic childhood arthritis with symptoms including fever, skin rash, pericarditis, hepatosplenomegaly, and growth retardation. It is often treated with glucocorticoids, but the long-term use of glucocorticoids can potentially cause growth impairment. IL-6 levels are correlated with the severity of disease manifestation of sJIA. A randomized phase 3 trial of TCZ in children with refractory sJIA showed drastic improvement in disease activity [95]. TCZ also restored body growth and improved bone homeostasis [96,97]. TCZ was approved for sJIA treatment in 2008 in Japan and in 2011 in the EU and the United States.