CHICAGO – A personalized treatment targeting a patient's individual genetic makeup helped cure more children with a rare form of kidney cancer, researchers said on Wednesday.
The findings, based on two studies led by Dr. David Dix of the British Columbia Children's Hospital in Vancouver, were released ahead of the American College of Clinical Oncology meeting later this month.
They were performed on young children with Wilms tumor, a rare kidney cancer that mostly affects children under age 5. About 500 cases of Wilms tumor are diagnosed in the United States each year. They make up 75 percent of all pediatric kidney cancers.
About 5 percent to 6 percent of children with this tumor have a genetic abnormality that puts them at higher risk for relapse when given standard treatment.
Dix and colleagues wanted to see if augmenting standard therapies for children with these high-risk tumors might improve their long-term survival rates.
In the studies, the teams identified 35 high-risk patients with early-stage tumors and 52 high-risk patients with late-stage disease.
The team found that augmenting standard chemotherapy treatments with an additional chemotherapy regimen helped more of these children survive for four years without a relapse, compared with standard therapy.
In patients with early-stage disease, four-year relapse-free rates were 83.9 percent, compared with the 75 percent typically seen in these high-risk children. In children with late-stage cancers, four-year relapse free survival increased to 91.5 percent, compared with 66 percent using standard treatments.
Dix said in a telephone briefing that toxicity levels among those who received the augmented therapy were expected and manageable.
He said the tailored approach based on the genetic profile of the tumor helps doctors give additional treatment only to children who have a higher risk of relapse, sparing those who can achieve a cure with less chemotherapy from the additional side effects.
"The ability to easily identify a small subset of patients with a poorer prognosis means these children can receive treatment that's right for them, while decreasing side effects for lower-risk patients," Dr. Julie Vose, president-elect of ASCO, told the briefing.