Current Status of Pediatric Rheumatic Disease Care in Japan

Hidehiko Narazaki
2022 Nihon Ika Daigaku Igakkai Zasshi  
The rarity of pediatric rheumatic disease and scarcity of pediatric rheumatologists can lead to delayed diagnosis and initiation of treatment, which for patients with this chronic disease can have a significant impact on prognosis. On the other hand, recent advances in molecular targeted therapies with biologics and small molecule drugs have contributed to dramatic improvements in controlling the disease. Furthermore, treatment efficacy is now based not only on the scoring of symptoms and
more » ... al examination findings, but also on quality of life (QoL) and patient-reported outcomes (PRO). Various QoL/PRO assessment questionnaires have been developed in Europe and the US, and translated versions are now in clinical use for adult patients in Japan. However, self-assessment is difficult for children, and their understanding and perception of health, illness, and treatment differ from those of adults. Therefore, age-appropriate questionnaires have been developed for children and their guardians, but they have not yet been fully validated for use in Japan. Thanks to improvements in diagnosis and treatment, more patients are now living with the disease into adulthood, focusing attention on the issue of how to support patients through this transition to adulthood and help them understand their own abilities and limitations so that they can become more independent. To improve care for patients with pediatric rheumatic disease and their families, the Pediatric Rheumatology Association of Japan, with the support of a Health and Labor Sciences Research Grant, is conducting registry surveys, developing appropriate pediatric clinical outcome assessments (COAs), seeking approval of drugs and examinations not yet permitted in Japan and increased medical subsidies, and developing systems to support patients in the transition to adulthood.
doi:10.1272/manms.18.274 fatcat:ot3oyheuibcobbycx7ulfmpsda