title: Efficacy of vinblastine in central nervous system Langerhans cell histiocytosis: a nationwide retrospective study.
abstract: BACKGROUND
  Vinblastine (VBL) is the standard treatment for systemic Langerhans cell histiocytosis (LCH), but little is known about its efficacy in central nervous system (CNS) mass lesions.
  
  
  METHODS
  A retrospective chart review was conducted. Twenty patients from the French LCH Study Group register met the inclusion criteria. In brief, they had CNS mass lesions, had been treated with VBL, and were evaluable for radiologic response.
  
  
  RESULTS
  The median age at diagnosis of LCH was 11.5 years (range: 1-50). Intravenous VBL 6 mg/m2 was given in a 6-week induction treatment, followed by a maintenance treatment. The median total duration was 12 months (range: 3-30). Eleven patients received steroids concomitantly. Fifteen patients achieved an objective response; five had a complete response (CR: 25%), ten had a partial response (PR: 50%), four had stable disease (SD: 20%) and one patient progressed (PD: 5%). Of interest, four out of the six patients who received VBL without concomitant steroids achieved an objective response. With a median follow-up of 6.8 years, the 5-year event-free and overall survival was 61% and 84%, respectively. VBL was well-tolerated and there were no patient withdrawals due to adverse events.
  
  
  CONCLUSIONS
  VBL, with or without steroids, could potentially be a useful therapeutic option in LCH with CNS mass lesions, especially for those with inoperable lesions or multiple lesions. Prospective clinical trials are warranted for the evaluation of VBL in this indication.
authors.affiliation: APHP-UPMC, Service de neurologie 2-Mazarin, Groupe Hospitalier Pitié-Salpêtrière, Paris, France. khe.hoang-xuan@psl.aphp.fr
authors.forename: Sophie
authors.initials: S
authors.lastname: Ng Wing Tin
authors.forename: Nadine
authors.initials: N
authors.lastname: Martin-Duverneuil
authors.forename: Ahmed
authors.initials: A
authors.lastname: Idbaih
authors.forename: Catherine
authors.initials: C
authors.lastname: Garel
authors.forename: Maria
authors.initials: M
authors.lastname: Ribeiro
authors.forename: Judith Landman
authors.initials: JL
authors.lastname: Parker
authors.forename: Anne-Sophie
authors.initials: AS
authors.lastname: Defachelles
authors.forename: Anne
authors.initials: A
authors.lastname: Lambilliotte
authors.forename: Mohamed
authors.initials: M
authors.lastname: Barkaoui
authors.forename: Martine
authors.initials: M
authors.lastname: Munzer
authors.forename: Martine
authors.initials: M
authors.lastname: Gardembas
authors.forename: Jean
authors.initials: J
authors.lastname: Sibilia
authors.forename: Patrick
authors.initials: P
authors.lastname: Lutz
authors.forename: Renato
authors.initials: R
authors.lastname: Fior
authors.forename: Michel
authors.initials: M
authors.lastname: Polak
authors.forename: Alain
authors.initials: A
authors.lastname: Robert
authors.forename: Olivier
authors.initials: O
authors.lastname: Aumaitre
authors.forename: Dominique
authors.initials: D
authors.lastname: Plantaz
authors.forename: Corinne
authors.initials: C
authors.lastname: Armari-Alla
authors.forename: Thierry
authors.initials: T
authors.lastname: Genereau
authors.forename: Perrine Marec
authors.initials: PM
authors.lastname: Berard
authors.forename: Ghislain Nokam
authors.initials: GN
authors.lastname: Talom
authors.forename: Jean-Loup
authors.initials: JL
authors.lastname: Pennaforte
authors.forename: Hubert Ducou
authors.initials: HD
authors.lastname: Le Pointe
authors.forename: Marie-Anne
authors.initials: MA
authors.lastname: Barthez
authors.forename: Gérard
authors.initials: G
authors.lastname: Couillault
authors.forename: Julien
authors.initials: J
authors.lastname: Haroche
authors.forename: Karima
authors.initials: K
authors.lastname: Mokhtari
authors.forename: Jean
authors.initials: J
authors.lastname: Donadieu
authors.forename: Khê
authors.initials: K
authors.lastname: Hoang-Xuan
mesh_terms: D000293:Adolescent; D000328:Adult; D000972:Antineoplastic Agents, Phytogenic / Q000627:therapeutic use*; D002490:Central Nervous System / Q000187:drug effects / Q000473:pathology*; D002493:Central Nervous System Diseases / Q000188:drug therapy* / Q000401:mortality; D002648:Child; D002675:Child, Preschool; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D013256:Steroids / Q000627:therapeutic use; D016896:Treatment Outcome; D014747:Vinblastine / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D000972:Antineoplastic Agents, Phytogenic; D013256:Steroids; D014747:Vinblastine