title: Clofarabine salvage therapy for refractory high-risk langerhans cell histiocytosis.
abstract: Pediatric patients with refractory multisystem Langerhans cell histiocytosis (LCH) have a poor prognosis despite aggressive chemotherapy. Salvage therapy with cytarabine and cladribine has shown promise as an effective treatment but is associated with significant toxicity. A previous report described two patients with refractory LCH who had a rapid response to single-agent clofarabine with minimal toxicity. In this report, we describe four children with refractory, risk-organ-positive LCH who were treated with clofarabine and provide follow-up for the two previously reported cases. The results support development of a formal trial evaluating clofarabine as front-line salvage for refractory LCH.
authors.affiliation: Department of Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee.
authors.forename: Allistair
authors.initials: A
authors.lastname: Abraham
authors.forename: Abdulrahman
authors.initials: A
authors.lastname: Alsultan
authors.forename: Michael
authors.initials: M
authors.lastname: Jeng
authors.forename: Carlos
authors.initials: C
authors.lastname: Rodriguez-Galindo
authors.forename: Patrick K
authors.initials: PK
authors.lastname: Campbell
mesh_terms: D000227:Adenine Nucleotides / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001087:Arabinonucleosides / Q000627:therapeutic use*; D002675:Child, Preschool; D000077866:Clofarabine; D005260:Female; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D008297:Male; D016879:Salvage Therapy / Q000379:methods*; D016896:Treatment Outcome
chemical_list: D000227:Adenine Nucleotides; D000970:Antineoplastic Agents; D001087:Arabinonucleosides; D000077866:Clofarabine