Children who develop acute myeloid leukemia (AML) are typically treated with chemotherapy, but more than half of patients eventually experience a relapse and go on to transplantation or second-line therapy. A phase 3 study shows Mylotarg (gemtuzumab ozogamicin), when given with standard chemotherapy, decreased the risk of relapse, especially in patients who later undergo a transplantation.
Patients in the study were given standard chemotherapy with or without Mylotarg. Those with intermediate-risk disease and a matched related donor or with high-risk disease moved on to transplantation. Patients with low-risk disease or those with intermediate-risk AML without a matched family donor had additional treatment that included chemotherapy with or without Mylotarg. At three years, the high-risk group saw an increase in survival from 49 percent to 68 percent.Mylotarg was originally approved in 2000 for older patients with relapsed AML, but the drug maker voluntarily withdrew it from the market a decade later based on a lack of effectiveness and safety concerns that emerged in subsequent clinical trials. Based on recent studies in newly diagnosed AML, however, researchers are hopeful the drug can be resurrected and re-examined in this patient population—both young and old.