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Studies Find Therapy Changes, Personalized Treatment Can Improve Outcomes for Childhood Kidney Cancer Patients

Washington, DC - A series of studies presented at the 51st meeting of the American Society of Clinical Oncology (ASCO) revealed changes in therapy and personalized treatment that could significantly improve outcomes for children with advanced stage Wilms tumors, the most common kidney cancer of childhood. Jeffrey Dome, MD, PhD, Chief of Children’s National Health System’s Division of Oncology and Hematology, supervised the clinical trials as part of the Children’s Oncology Group (COG).

The studies of the COG, which represents more than 200 children’s hospitals around the world, showed that therapy can be adapted according to clinical and biological predictors of outcome.

“With a combination of identifying novel clinical risk factors and biological markers, we are able to tailor therapy on the basis of a patient’s risk of tumor recurrence,” says Dr. Dome.

“The prognostic markers allow us to increase therapy to certain groups of patients who need it, or decrease it for those who don’t. It’s the ultimate example of personalized medicine, tailoring treatment to findings in each individual patient, and the results are outstanding outcomes,” Dr. Dome adds.

Children’s National is one of the leading centers for the treatment of pediatric kidney cancer. Wilms tumor mainly impacts children under the age of five, but accounts for 75 percent of all childhood renal cancers, and is the fifth most common cancer in children.  About 500 children with Wilms tumor are diagnosed in North America each year.

As chair of the Renal Tumor Committee for the COG, Dr. Dome has been involved in a leadership role in development and execution of the studies.

“Wilms tumor has been one of the great success stories in pediatric healthcare, with survival rates of approximately 90 percent,” Dr. Dome says. “However, there are subgroups that don’t have a great survival rate, and the trick has been how to identify them.”

Moreover, survival comes at a cost, he says, because a significant percentage of patients have severe chronic health conditions related to treatment. “It would be beneficial to increase therapy in the patients with a high risk of recurrence, while decreasing therapy and avoiding long-term side effects in patients with low risk of recurrence,” Dr. Dome says. “These studies have successfully put these principles into practice.”

Among the phase III COG study findings:

  • Augmenting therapy with additional drugs improves outcomes for children with stage III and IV Wilms tumor that contains a specific chromosomal abnormality associated with poorer prognosis. Patients who received additional therapy had four-year relapse free survival rates of 91.5 percent, as opposed to 65.9 percent based on research conducted several years ago.
  • Omission of lung radiation therapy was feasible without compromising overall survival in patients with stage IV Wilms tumor with a complete radiographic lung nodule response after 6 weeks of chemotherapy. Based on these findings, lung radiation therapy can potentially be avoided in 40 percent of patients with Wilms tumor and metastatic spread to the lungs.
  • Novel biological prognostic markers, or biomarkers, were effective in predicting a group of patients with a very low risk of recurrence following surgery without any chemotherapy.  

Contact: Emily Hartman or Caitlyn Camacho at 202-476-4500.


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