title: Treatment of children with Langerhans cell histiocytosis with 2-chlorodeoxyadenosine.
abstract: Langerhans cell histiocytosis (LCH) is a disorder characterized by proliferation of activated Langerhans cells. Immune dysregulation is believed to be part of the pathogenesis. Although current therapies are very effective at inducing remission, multiple recurrences and long-term sequelae are common for patients with low-risk disease, and a significant proportion of young patients die of their disease. More effective therapies based on the pathogenesis of LCH are needed. We investigated the use of 2-chloro-deoxyadenosine (2-CdA), a purine analogue with an antiproliferative effect on histiocytes and lymphocytes, in patients with recurrent or high-risk LCH. Six patients with recurrent LCH received 2-CdA (5-7 mg/m(2)/day for 5 days, repeated every 21-28 days). All patients achieved remission. With a median follow-up of 15 months (range, 3-25 months), 5 patients remain in remission. A patient with multisystem disease who recurred after 13 months, achieved a second remission with 2-CdA. Hematologic toxicity was minimal, and no infectious complications were documented. 2-CdA is among the most effective drugs for the treatment of LCH, and this is probably due to both its anti-proliferative and immunomodulatory effects. 2-CdA needs to be considered for the treatment of recurrent LCH. However, its incorporation into front-line treatment of patients with multi-system LCH needs further study.
authors.affiliation: Department of Hematology-Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA. Carlos.Rodriguez-Galindo@stjude.org
authors.forename: Carlos
authors.initials: C
authors.lastname: Rodriguez-Galindo
authors.forename: Patrick
authors.initials: P
authors.lastname: Kelly
authors.forename: Michael
authors.initials: M
authors.lastname: Jeng
authors.forename: Gerald G
authors.initials: GG
authors.lastname: Presbury
authors.forename: Martha
authors.initials: M
authors.lastname: Rieman
authors.forename: Winfred
authors.initials: W
authors.lastname: Wang
mesh_terms: D002648:Child; D002675:Child, Preschool; D017338:Cladribine / Q000008:administration & dosage* / Q000592:standards; D015331:Cohort Studies; D018572:Disease-Free Survival; D006646:Histiocytosis, Langerhans-Cell / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000592:standards; D007223:Infant; D012008:Recurrence; D012074:Remission Induction; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D017338:Cladribine