The FDA extended the indication for gemtuzumab ozogamicin (Mylotarg, Wyeth) to treat newly diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients aged 1 month and older.
The safety and efficacy of the agent in the pediatric population were supported by data from AAML0531, a multicenter, randomized study of 1,063 patients aged 0 to 29 years with newly diagnosed AML (ClinicalTrials.gov Identifier: NCT00372593).
In the study, patients were randomly assigned to receive five-cycle chemotherapy alone or with gemtuzumab ozogamicin (3 mg/m2), administered once on day 6 during the first cycle of induction and once on day 7 during the second cycle of intensification. The main efficacy outcome measure was event-free survival (EFS), measured from the date of trial entry until induction failure, relapse or death from any cause.
The EFS hazard ratio was 0.84 (95% CI, 0.71-0.99). The estimated percentage of patients free of induction failure, relapse or death at five years was 48% (95% CI, 43%-52%) in the gemtuzumab ozogamicin plus chemotherapy arm versus 40% (95% CI, 36% 45%) in the chemotherapy alone arm. No difference in overall survival was demonstrated between treatment arms.
The most common grade 3 and higher adverse reactions that occurred in at least 5% of patients who received gemtuzumab ozogamicin during induction 1 and intensification 2 were infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea and hypotension.
For more information, view full prescribing information for gemtuzumab ozogamicin.
—Clinical Oncology News Staff
Based on a press release from the FDA.