Study results show a combination of preoperative chemotherapy, quicker surgical removal of tumors, and tailored postoperative therapy improved survival rates and preservation of kidney functions in children with bilateral Wilms tumors.

A new approach to treating children diagnosed with bilateral Wilms tumors (BWT) significantly improved event-free survival (EFS), or the length of time a patient does not experience complications or recurrence of cancer following treatment, and overall survival (OS) rates after four years when compared to historical rates, according to a Children’s Oncology Group study published in the September issue of the journal Annals of Surgery. Jeffrey Dome, MD, PhD, Vice President of the Center for Cancer and Blood Disorders at Children’s National Health System was co-senior author of this first-ever, multi-institutional prospective study of children with BWT.

Also known as nephroblastoma, Wilms tumor is the most common pediatric kidney cancer, typically seen in children ages three to four. Compared to patients with unilateral Wilms tumors, children with BWT have poorer EFS and are at higher risk for later effects such as renal failure. The treatment of BWT is challenging because it involves surgical removal of the cancer, while preserving as much healthy kidney tissue as possible to avoid the need for an organ transplant.

Historically, patients with BWT have had poor outcomes especially if they have tumors with unfavorable histology, which is defined by specific Wilms tumor cell types seen under the microscope. In this study, Dr. Dome and 18 other clinical researchers followed a new treatment approach consisting of three chemotherapy drugs before surgery rather than the standard two drug regimen, surgical removal of cancerous tissue within 12 weeks of diagnosis, and postoperative chemotherapy that was adjusted based on histology.

The study found that preoperative chemotherapy expedited surgical treatment, with 84 percent of patients having surgery within 12 weeks of diagnosis. The new treatment approach also vastly improved EFS and OS rates for patients participating in the study. EFS rates were 82.1 percent, compared to 56 percent in a 2002 study, an improvement of 46.6 percent. OS rates were 94.9 percent, compared to 80.8 percent in the 2002 study, an improvement of 16.6 percent.

“The data show that event-free and overall survival rates can be significantly improved using an approach to enhance tumor response, expedite surgical resection and adjust therapy based on histology,” says Dr. Dome. “I am very encouraged by these results, which I believe will serve as a benchmark for future studies and lead to additional treatment improvements, giving more children the chance to overcome this diagnosis and lead healthy lives.”

A total of 189 patients at children’s hospitals, universities, and cancer centers in the United States and Canada participated in this study. These patients will continue to be followed for 10 years to track kidney failure rates.