title: Guidance on the use of canakinumab in patients with cryopyrin-associated periodic syndrome in Japan.
abstract: Cryopyrin-associated periodic syndrome (CAPS) is an orphan disease with incidence of about one in 1,000,000 persons. This autoinflammatory disease develops in the neonatal period or early childhood, with various inflammatory symptoms occurring repeatedly throughout the patient's lifetime. It is caused by abnormality of the NLRP3 protein which mediates the intracellular signal transduction mechanism of inflammatory processes, resulting in continuous overproduction of interleukin (IL)-1β, which induces chronic inflammation and progressive tissue damage. Definitive diagnosis of CAPS is difficult, and treatment has also been difficult because of a lack of effective medications in Japan. Clinical studies of human anti-human IL-1β monoclonal antibody (canakinumab) treatment were conducted in Japan, and approval was granted for therapeutic use of canakinumab for CAPS in September 2011. Similar to other biological drugs, canakinumab is clinically highly effective. However, sufficient attention to the method of use and adverse drug reactions is necessary. This guidance describes the use of canakinumab in Japan for CAPS in relation to exclusion criteria, method of use, evaluation criteria, and adverse drug reactions.
authors.affiliation: Department of Pediatrics, Yokohama City University School of Medicine, 3-9, Fukuura, Kanazawa-ku, Yokohama 236-0004, Japan. syokota@med.yokohama-cu.ac.jp
authors.forename: Shumpei
authors.initials: S
authors.lastname: Yokota
authors.forename: Ryuta
authors.initials: R
authors.lastname: Nishikomori
authors.forename: Hidetoshi
authors.initials: H
authors.lastname: Takada
authors.forename: Masako
authors.initials: M
authors.lastname: Kikuchi
authors.forename: Tomo
authors.initials: T
authors.lastname: Nozawa
authors.forename: Taichi
authors.initials: T
authors.lastname: Kanetaka
authors.forename: Toshitaka
authors.initials: T
authors.lastname: Kizawa
authors.forename: Takako
authors.initials: T
authors.lastname: Miyamae
authors.forename: Masaaki
authors.initials: M
authors.lastname: Mori
authors.forename: Toshio
authors.initials: T
authors.lastname: Heike
authors.forename: Toshiro
authors.initials: T
authors.lastname: Hara
authors.forename: Tomoyuki
authors.initials: T
authors.lastname: Imagawa
mesh_terms: D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D056587:Cryopyrin-Associated Periodic Syndromes / Q000188:drug therapy*; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors*; D007564:Japan
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D007155:Immunologic Factors; D053583:Interleukin-1beta; C541220:canakinumab