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News and Features Children's Mercy Oncology Leader and Team Help Bring New Insights to Rare Cancer

Experts at Children's Mercy Hospital (CMH), led by Alan Gamis, MD, CMH's chief of the section of oncology, participated in a national study on a rare childhood cancer and its even rarer offshoot: its presence in children with Down syndrome (DS). The results were published in the March 5, 2012 issue of one of the leading international oncology journals, Cancer.

Gamis is a specialist in the childhood cancer on which the study focused, acute myeloid leukemia (AML), and his expertise has made CMH one of the more well-known centers in the NIH's Children's Oncology Group (COG). He is also chair of the AML Disease Scientific Committee, which oversees all COG Research in AML.

"We know that the survival rate in AML children in general is 60-65%, and rises to 80-90% in kids with DS. It's due to a specific mutation that occurs in DS kids' AML cells called GATA1. This mutation causes AML in DS kids, but it also confers sensitivity to chemotherapy," says Gamis.

The question was: could this increased sensitivity mean less chemo could be used-and still provide the same benefits? The answers came from this phase-3 trial on DS children with AML, known as COG A2971, which compared results from the last such trial, known as CCG2891.

The A2971 trial eliminated two induction drugs and also eliminated three months of maintenance therapy from the drug combination known as DCTER. Specifically the follow-up (around 4.8 years of age) showed that in addition to similar remission rates, the 5-year event-free survival (EFS) rate in A2971 patients was about 79% compared to 77% in 2891 patients and the disease-free survival rate (DFS) was 89% vs. 85%.

Age was a key factor. "We found DS children who developed AML at age 4 or younger had the mutation that made it easier to treat. Those who developed AML at age 4 or older tended to develop the tougher to treat variation, requiring the more intense therapy of other AML patients," says Gamis. He adds that a follow-up to this study, presented in May 2012, noted that the faster the child goes into remission (by day 14 of treatment or sooner), the better the odds of overall survival.

"This was the largest trial for Down syndrome kids with AML to be performed in the world. It proved that we could continue to cure almost all of the DS children with AML, while reducing the intensity of treatment," says Gamis. He concludes: "At CMH, We help design future AML trials and we have insights into the design of what's coming down the pike five years from now. Through our knowledge and oversight of these national trials, we have a distinctive perspective on taking care of all AML children, including those with such unique mutations."

NOTE: Citation from CANCER:

Sorrell, A. D., Alonzo, T. A., Hilden, J. M., Gerbing, R. B., Loew, T. W., Hathaway, L., Barnard, D., Taub, J. W., Ravindranath, Yaddanapudi., Smith, F. O., Arceci, R. J., Woods, W. G. and Gamis, A. S. (2012), Favorable survival maintained in children who have myeloid leukemia associated with Down syndrome using reduced-dose chemotherapy on Children's Oncology Group trial A2971. Cancer. doi: 10.1002/cncr.27484



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