title: Acquisition of expanded indications for intravenous cyclophosphamide in the management of childhood rheumatic disease in general.
abstract: We created the final bill of "Intravenous cyclophosphamide (IVCY) for the treatment of rheumatic disease of children in general" in order to get approval for the off-label use of IVCY from the Study Group on Pediatric Drug Therapy. As a result, this study group approved IVCY's off-label use in children via public applications. We could create IVCY regimen specific for Japanese children independently of the efficacy, dosage, and administration applied in Germany, which at the time of application was the only country in Europe and the USA where IVCY was approved. Later, its use in adults was given approval through public applications after being fully reviewed by the Study Group on Unapproved and Off-label Drugs of High Medical Need. Consequently, IVCY is now available for the treatment of both childhood and adult rheumatic diseases in general.
authors.affiliation: Department of Pediatrics, Yokohama City University Medical Center, 4-57, Urafune-cho, Minami-ku, Yokohama, 232-0024, Japan. mmori@med.yokohama-cu.ac.jp
authors.forename: Masaaki
authors.initials: M
authors.lastname: Mori
authors.forename: Takuji
authors.initials: T
authors.lastname: Murata
authors.forename: Syuji
authors.initials: S
authors.lastname: Takei
authors.forename: Tomoyuki
authors.initials: T
authors.lastname: Imagawa
authors.forename: Shumpei
authors.initials: S
authors.lastname: Yokota
mesh_terms: D018501:Antirheumatic Agents / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage* / Q000009:adverse effects; D017277:Drug Approval; D006801:Humans; D007275:Injections, Intravenous; D007564:Japan; D056687:Off-Label Use; D012216:Rheumatic Diseases / Q000188:drug therapy*
chemical_list: D018501:Antirheumatic Agents; D003520:Cyclophosphamide