title: Response to mercaptopurine for refractory autoimmune cytopenias in children.
abstract: BACKGROUND
  Several treatment strategies are available for children with severe immune thrombocytopenic purpura (ITP) and other immune cytopenias refractory to initial therapies. 6-Mercaptopurine (6MP) is one option, however it has not been well studied in children, especially as a single agent, and no pediatric case series have been reported since 1970.
  
  
  METHODS
  We reviewed the experience at our institution over 8 years, using 6MP as a steroid sparing treatment for children with ITP, auto-immune hemolytic anemia (AIHA) or Evans syndrome. A total of 29 pediatric patients were treated with 6MP from 2000 to 2007.
  
  
  RESULTS
  Response was defined as a rise in hemoglobin by at least 1.5 g/dl and to a level of 10 g/dl or greater in patients treated for anemia, or a platelet count >or=50 x 10(9)/L in patients treated for thrombocytopenia. We found an overall response rate of 83% among all patients. Fourteen percent of patients stopped drug because of side effects.
  
  
  CONCLUSIONS
  These results suggest that 6MP can be an effective single-agent treatment for refractory immune cytopenias in children. Prospective studies are warranted to determine long-term efficacy and toxicity and to more clearly define patient populations most likely to respond.
authors: Division of Hematology/Oncology, Children's Hospital Boston, Boston, Massachusetts 02115, USA.
authors: Amy
authors: A
authors: Sobota
authors: Ellis J
authors: EJ
authors: Neufeld
authors: Sameer
authors: S
authors: Lapsia
authors: Carolyn M
authors: CM
authors: Bennett
mesh_terms: D002648:Child; D002908:Chronic Disease; D005260:Female; D006454:Hemoglobins / Q000032:analysis / Q000187:drug effects; D006801:Humans; D008297:Male; D015122:Mercaptopurine / Q000627:therapeutic use*; D019384:Nucleic Acid Synthesis Inhibitors / Q000627:therapeutic use; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D012189:Retrospective Studies; D016879:Salvage Therapy; D016896:Treatment Outcome
chemical_list: D006454:Hemoglobins; D019384:Nucleic Acid Synthesis Inhibitors; D015122:Mercaptopurine