title: Final report of the efficacy and safety of gemtuzumab ozogamicin (Mylotarg) in patients with CD33-positive acute myeloid leukemia in first recurrence.
abstract: BACKGROUND
  In this study, the authors analyzed the efficacy and safety of gemtuzumab ozogamicin (GO) (Mylotarg), an antibody-targeted chemotherapy for CD33-positive acute myeloid leukemia (AML).
  
  
  METHODS
  Patients with CD33-positive AML in first recurrence were entered in 3 open-label, single-arm, Phase II studies. Patients received monotherapy with GO 9 mg/m(2) as a 2-hour intravenous infusion in 2 doses separated by 2 weeks. Patients were evaluated for remission, survival, and treatment-emergent adverse events.
  
  
  RESULTS
  Two hundred seventy-seven patients (median age, 61 yrs) were treated with GO, and 71 patients (26%) achieved remission, which was defined as < or = 5% blasts in the bone marrow without leukemic blasts in the peripheral blood, neutrophil recovery to > or = 1500/microL, hemoglobin > or = 9 g/dL, and independence from red blood cell and platelet transfusions. Complete remission (CR) with platelet recovery (> or = 100,000/microL) or without full platelet recovery (< 100,000/microL) (CRp) was observed in 35 patients (13%) and 36 patients (13%), respectively. The median recurrence-free survival was 6.4 months for patients who achieved CR and 4.5 months for patients who achieved CRp. Although expected incidences of Grade 3 or 4 neutropenia (98%) and thrombocytopenia (99%) were observed, the incidence of Grade 3 or 4 sepsis (17%) and pneumonia (8%) was relatively low. Grade 3 or 4 hyperbilirubinemia and hepatic aspartate aminotransferase and alanine aminotransferase elevations were reported in 29%, 18%, and 9% of patients, respectively; 0.9% of patients who did not undergo prior or subsequent hematopoietic stem cell transplantation developed hepatic venoocclusive disease after GO treatment.
  
  
  CONCLUSIONS
  When it was administered to patients with CD33-positive AML in first recurrence, single-agent GO induced a 26% remission rate with a generally acceptable safety profile.
authors.affiliation: Department of Medicine, University of Chicago, Chicago, Illinois, USA. rlarson@medicine.bsd.uchicago.edu
authors.forename: Richard A
authors.initials: RA
authors.lastname: Larson
authors.forename: Eric L
authors.initials: EL
authors.lastname: Sievers
authors.forename: Edward A
authors.initials: EA
authors.lastname: Stadtmauer
authors.forename: Bob
authors.initials: B
authors.lastname: Löwenberg
authors.forename: Elihu H
authors.initials: EH
authors.lastname: Estey
authors.forename: Hervé
authors.initials: H
authors.lastname: Dombret
authors.forename: Matthias
authors.initials: M
authors.lastname: Theobald
authors.forename: Dimitris
authors.initials: D
authors.lastname: Voliotis
authors.forename: John M
authors.initials: JM
authors.lastname: Bennett
authors.forename: Maria
authors.initials: M
authors.lastname: Richie
authors.forename: Lance H
authors.initials: LH
authors.lastname: Leopold
authors.forename: Mark S
authors.initials: MS
authors.lastname: Berger
authors.forename: Matthew L
authors.initials: ML
authors.lastname: Sherman
authors.forename: Michael R
authors.initials: MR
authors.lastname: Loken
authors.forename: Jacques J M
authors.initials: JJ
authors.lastname: van Dongen
authors.forename: Irwin D
authors.initials: ID
authors.lastname: Bernstein
authors.forename: Frederick R
authors.initials: FR
authors.lastname: Appelbaum
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000617:Aminoglycosides / Q000008:administration & dosage*; D000911:Antibodies, Monoclonal / Q000008:administration & dosage*; D061067:Antibodies, Monoclonal, Humanized; D015703:Antigens, CD / Q000276:immunology*; D015214:Antigens, Differentiation, Myelomonocytic / Q000276:immunology*; D017322:Clinical Trials, Phase II as Topic; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005069:Evaluation Studies as Topic; D005260:Female; D005500:Follow-Up Studies; D000079982:Gemtuzumab; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology / Q000401:mortality*; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D012008:Recurrence; D018570:Risk Assessment; D012720:Severity of Illness Index; D063268:Sialic Acid Binding Ig-like Lectin 3; D016037:Single-Blind Method; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D000617:Aminoglycosides; D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D015703:Antigens, CD; D015214:Antigens, Differentiation, Myelomonocytic; C571163:CD33 protein, human; D063268:Sialic Acid Binding Ig-like Lectin 3; D000079982:Gemtuzumab