Combining the monoclonal antibody gemtuzumab with standard chemotherapy significantly reduced the risk of relapse and increased rates of disease-free survival in pediatric patients with acute myeloid leukemia (AML). Posttreatment relapse rates are a major indicator of potential for long-term survival in children with the disease.

This study was presented at the 2013 American Society of Hematology (ASH) Annual Meeting, December 7-10, 2013, in New Orleans, Louisiana. It evaluated a total of 1,022 children (average age, 10 years) at trial sites nationwide.

“Chemotherapy has limits. Particularly in children, increasing doses further can become too toxic for the patient while still not achieving the desired effect on the cancer,” said Alan Gamis, MD, MPH, associate division director, Section of Oncology at Children’s Mercy Hospital in Kansas City, Missouri. “This study is significant because it shows, for the first time, that this targeted treatment can augment the effects of chemotherapy in children and effectively reduce their risk of relapse. We found it was most effective in the patients most at risk.”

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Gemtuzumab was removed from the US market in 2010 because the FDA determined the potential risks of the drug outweighed the potential benefits. Recent research with the compound has raised questions about whether that action may have been premature. This data supports recent findings in adults with AML that gemtuzumab reduced their risk of relapse when added to standard chemotherapy.

“This could be an important treatment option for critical patients,” said Gamis. “Gemtuzumab appears to have a real impact in increasing the likelihood of long-term survival in high-risk patients.”

In this study, patients were treated with gemtuzumab or a standard treatment regimen. Compared with standard regimens, the addition of gemtuzumab was associated with better disease-free survival (61% vs 55%) and reduced relapse risk (33% vs 41%). It did not significantly improve overall survival (74% vs 70%).