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 ≤ 5% blasts in the bone marrow without leukemic blasts in the peripheral blood, neutrophil recovery to ≥ 1500/μL, hemoglobin ≥ 9 g/dL, and independence from red blood cell and platelet transfusions. Complete remission (CR) with platelet recovery (≥ 100,000/μL) or without full platelet recovery (< 100,000/μL) (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. Cancer 2005. © 2005 American Cancer Society.