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Drug Offers Promising Approach to Improve Outcome for Children with High-risk Leukemia
Date:11/15/2013

ing antibody-based therapies, that target the same AML surface protein, said the study’s senior author Jeffrey Rubnitz, M.D., Ph.D., a member of the St. Jude Department of Oncology.

AML is diagnosed in about 500 children annually and accounts for about 20 percent of childhood leukemia. The disease affects white blood cells.

“Currently there are few options for AML patients who relapse or do not respond to conventional therapy,” said first author Carol O’Hear, M.D., Ph.D., a St. Jude postdoctoral oncology fellow. Added Rubnitz: “Without new agents, it is unlikely we will be able to improve pediatric AML survival beyond current levels of about 70 percent. The results of this and earlier studies make a strong case that some patients benefit from this targeted therapy, which has us looking for new ways to take aim at the same protein.”

This study involved a subset of patients enrolled in a clinical trial of pediatric AML called AML02. Altogether, 232 children with AML enrolled in the six-year, multicenter trial that ended in 2008. A total of 46 patients received GO alone or in combination with conventional chemotherapy.

For this analysis, researchers wanted to know if GO was associated with reduced MRD. Patients were considered free of MRD if less than one cancer cell could be detected in 1,000 normal bone marrow cells.

Seventeen patients received GO alone when MRD was detected after two rounds of chemotherapy with the drugs cytarabine, daunorubicin and etoposide. After GO treatment, leukemia declined in 14 of the 17 patients and fell to undetectable levels in 13 patients.

An additional 29 patients received GO as part of their second round of three-drug chemotherapy. All had levels of residual disease that indicated a poor initial response to chemotherapy alone, including nine with MRD levels of 25 percent or more. Residual disease fell to undetectable levels in four of the nine patients following combination therapy.


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