title: Retinoic acid therapy in "degenerative-like" neuro-langerhans cell histiocytosis: a prospective pilot study.
abstract: BACKGROUND
  Degenerative-like neuro-Langerhans cell histiocytosis (DN-LCH) is a rare complication of LCH marked by progressive cerebellar ataxia. No treatment has so far been shown to slow this progression.
  
  
  METHODS
  All-trans retinoic acid (ATRA) was administered orally at a dose of 45 mg/m(2) daily for 6 weeks and then 2 weeks every month for 1 year. The endpoints were clinical status at 1 year (assessed with rating scales for ataxia and disability), adverse effects, and changes in neurological abnormalities on MRI.
  
  
  RESULTS
  Ten patients were studied. The treatment was well tolerated. All the patients were clinically stable at the end of the study. No MRI changes were noted.
  
  
  CONCLUSIONS
  DN-LCH appeared to be stable during ATRA therapy, but further studies are required to appreciate the possible long-term benefits of ATRA.
authors.affiliation: Fédération Neurologique Mazarin, Groupe Hospitalier Pitié-Salpêtrière, Paris, France.
authors.forename: Ahmed
authors.initials: A
authors.lastname: Idbaih
authors.forename: J
authors.initials: J
authors.lastname: Donadieu
authors.forename: M A
authors.initials: MA
authors.lastname: Barthez
authors.forename: F
authors.initials: F
authors.lastname: Geissmann
authors.forename: Y
authors.initials: Y
authors.lastname: Bertrand
authors.forename: O
authors.initials: O
authors.lastname: Hermine
authors.forename: L
authors.initials: L
authors.lastname: Brugières
authors.forename: T
authors.initials: T
authors.lastname: Genereau
authors.forename: C
authors.initials: C
authors.lastname: Thomas
authors.forename: K
authors.initials: K
authors.lastname: Hoang-Xuan
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002524:Cerebellar Ataxia / Q000188:drug therapy* / Q000209:etiology; D002648:Child; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D014212:Tretinoin / Q000627:therapeutic use*
chemical_list: D000970:Antineoplastic Agents; D014212:Tretinoin