title: AAML03P1, a pilot study of the safety of gemtuzumab ozogamicin in combination with chemotherapy for newly diagnosed childhood acute myeloid leukemia: a report from the Children's Oncology Group.
abstract: BACKGROUND
  The development of antigen-targeted therapies may provide additional options to improve outcomes in children with acute myeloid leukemia (AML). The Children's Oncology Group AAML03P1 trial sought to determine the safety of adding 2 doses of gemtuzumab ozogamicin, a humanized anti-CD33 antibody-targeted agent, to intensive chemotherapy during remission induction and postremission intensification for children with de novo AML.
  
  
  METHODS
  AAML03P1 enrolled 350 children with previously untreated AML. Patients with a matched family donor received 3 courses of chemotherapy followed by hematopoietic stem cell transplantation; those without a matched family donor received 5 courses of chemotherapy. Gemtuzumab ozogamicin 3 mg/m(2)/dose was administered on Day 6 of Course 1 and Day 7 of Course 4.
  
  
  RESULTS
  Toxicities observed in all courses of therapy were typical of AML chemotherapy regimens, with infection being most common. Patients achieved a complete remission rate of 83% after 1 course and 87% after 2 courses. The mortality rate was 1.5% after the first gemtuzumab ozogamicin-containing induction course and 2.6% after 2 induction courses. The 3-year event-free survival and overall survival rates were 53 ± 6% and 66 ± 5%, respectively.
  
  
  CONCLUSIONS
  This trial determined that it is safe and feasible to include gemtuzumab ozogamicin in combination with intensive chemotherapy. The survival rates compare favorably with the recently published results of clinical trials worldwide.
authors.affiliation: Aflac Cancer Center and Blood Disorders Service/Children's Healthcare of Atlanta/Emory University, Atlanta, Georgia 30322, USA. todd.cooper@choa.org
authors.forename: Todd M
authors.initials: TM
authors.lastname: Cooper
authors.forename: Janet
authors.initials: J
authors.lastname: Franklin
authors.forename: Robert B
authors.initials: RB
authors.lastname: Gerbing
authors.forename: Todd A
authors.initials: TA
authors.lastname: Alonzo
authors.forename: Craig
authors.initials: C
authors.lastname: Hurwitz
authors.forename: Susana C
authors.initials: SC
authors.lastname: Raimondi
authors.forename: Betsy
authors.initials: B
authors.lastname: Hirsch
authors.forename: Franklin O
authors.initials: FO
authors.lastname: Smith
authors.forename: Prasad
authors.initials: P
authors.lastname: Mathew
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Arceci
authors.forename: James
authors.initials: J
authors.lastname: Feusner
authors.forename: Robert
authors.initials: R
authors.lastname: Iannone
authors.forename: Robert S
authors.initials: RS
authors.lastname: Lavey
authors.forename: Soheil
authors.initials: S
authors.lastname: Meshinchi
authors.forename: Alan
authors.initials: A
authors.lastname: Gamis
mesh_terms: D000293:Adolescent; D000328:Adult; D000617:Aminoglycosides / Q000008:administration & dosage; D000677:Amsacrine / Q000008:administration & dosage; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage; D003630:Daunorubicin / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D000079982:Gemtuzumab; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D015470:Leukemia, Myeloid, Acute / Q000188:drug therapy* / Q000401:mortality*; D008297:Male; D009367:Neoplasm Staging; D010865:Pilot Projects; D011379:Prognosis; D015996:Survival Rate; D055815:Young Adult
chemical_list: D000617:Aminoglycosides; D061067:Antibodies, Monoclonal, Humanized; D000677:Amsacrine; D003561:Cytarabine; D005047:Etoposide; D000079982:Gemtuzumab; D003630:Daunorubicin