What is the role of gemtuzumab ozogamicin (Mylotarg) in the treatment of acute myeloid leukemia (AML)?

Updated: May 26, 2020
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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In 2017, the FDA approved gemtuzumab ozogamicin (Mylotarg) for the treatment of adults with newly diagnosed AML whose tumors express the CD33 antigen (CD33-positive AML). The FDA also approved gemtuzumab ozogamicin for the treatment of relapsed or refractory CD33-positive AML in patients aged 2 years and older. [54]

Gemtuzumab ozogamicin originally received accelerated approval in 2000 as a stand-alone treatment for relapsed CD33-positive AML in older patients, but was voluntarily withdrawn from the market after subsequent confirmatory trials failed to verify clinical benefit. The 2017 approval includes a lower recommended dose, a different treatment schedule, and a new patient population. [54]

The approval is based on data from several trials, including the ALFA-0701 and AML-19 trials. The multicenter, open-label phase III ALFA-0701 trial randomized 271 patients with newly-diagnosed AML to daunorubicin and cytarabine alone or combined with gemtuzumab ozogamicin. Gemtuzumab at 3 mg/m2 was administered on days 1, 4, and 7 during induction and day 1 of each of the 2 consolidation chemotherapy courses. The primary endpoint was event-free survival (EFS) with a secondary endpoint of overall survival (OS). Gemtuzumab ozogamicin was associated with a statistically significant improvement in EFS of 7.8 months. However, the drug was not associated with a significant improvement in OS. [55]

In the open-label phase III study AML-19, elderly patients who could not tolerate other AML treatments were randomized to gemtuzumab ozogamicin (n=118) or best supportive care (n=119). Gemtuzumab ozogamicin was initially administered at 6 mg/m2 on day 1 and 3 mg/m2 on day 8. Patients without evidence of disease progression then received the drug at 2 mg/m2 on day 1 every 4 weeks. Patients received a single course of gemtuzumab ozogamicin. The trial measured how many patients achieved a complete remission. Following treatment with gemtuzumab ozogamicin, 26% of patients achieved a complete remission that lasted a median 11.6 months. [56]

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