title: Follow-up of pediatric patients treated by IVIG for Langerhans cell histiocytosis (LCH)-related neurodegenerative CNS disease.
abstract: The follow-up of eight Japanese children with Langerhans cell histiocytosis (LCH)-related neurodegenerative central nervous system (ND-CNS) disease who were treated with intravenous immunoglobulin (IVIG) for >3 years is described. The patients developed ND-CNS disease at a median age of 5.2 (range 3.5-10.0) years and received IVIG treatment for a median duration of 6.5 + (range 3.7 to 10+) years. After a median follow-up period of 11.6 + (8.3+ to 13.9+) years after ND-CNS disease diagnosis, the median Expanded Disability Status Scale (EDSS) score of the eight patients was 4.0 (range 2.0-9.5). At the last follow-up as of March 2014, three patients have low EDSS scores (<3.0) and can walk without any assistance. Another three patients have EDSS scores of 3.5-4.5 and can walk by themselves, albeit occasionally with supports. However, the remaining two patients are wheelchair bound or bed ridden. The school performance of seven of the eight patients was below average. IVIG appeared to be most beneficial when it was administered soon after ND-CNS disease diagnosis when the EDSS scores were low (1.0-2.5). The patients who began receiving IVIG when their high EDSS scores were higher (4.5-7.0) appeared to obtain less benefit. To prevent progression of ND-CNS disease in patients with LCH, it is recommended to introduce IVIG early and to continue this therapy for >3 years.
authors.affiliation: Department of Laboratory Medicine, Uji-Tokushukai Medical Center, Uji, Kyoto, shinim95@mbox.kyoto-inet.or.jp.
authors.forename: Shinsaku
authors.initials: S
authors.lastname: Imashuku
authors.forename: Naoto
authors.initials: N
authors.lastname: Fujita
authors.forename: Yoko
authors.initials: Y
authors.lastname: Shioda
authors.forename: Haruyoshi
authors.initials: H
authors.lastname: Noma
authors.forename: Shiro
authors.initials: S
authors.lastname: Seto
authors.forename: Toshinori
authors.initials: T
authors.lastname: Minato
authors.forename: Kazuo
authors.initials: K
authors.lastname: Sakashita
authors.forename: Nobuhiro
authors.initials: N
authors.lastname: Ito
authors.forename: Ryoji
authors.initials: R
authors.lastname: Kobayashi
authors.forename: Akira
authors.initials: A
authors.lastname: Morimoto
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006646:Histiocytosis, Langerhans-Cell / Q000150:complications*; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use*; D008279:Magnetic Resonance Imaging; D008297:Male; D019636:Neurodegenerative Diseases / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology*; D016896:Treatment Outcome
chemical_list: D016756:Immunoglobulins, Intravenous