Many children with retinoblastoma can safely forego adjuvant chemotherapy

A prospective clinical trial conducted in France found that children with low-risk retinoblastoma do not need postoperative (adjuvant) chemotherapy to prevent disease recurrence or metastasis. Further, the results suggest that certain patients with intermediate-risk disease can receive less aggressive adjuvant treatment, or perhaps forego it altogether. Avoiding chemotherapy spares patients from treatment side effects and such long-term health risks as cardiovascular disease and development of a second cancer.

Retinoblastoma is a rare form of cancer, accounting for 3% of all cancers diagnosed in children younger than 15 years. An estimated 300 cases of retinoblastoma will be diagnosed in the United States this year, most before the age of 5 years. The tumor begins in the retina, a thin layer of nerve tissue that coats the back of the eye.

About two thirds of children with retinoblastoma have unilateral disease, meaning that only one eye is affected by the tumor. In developed countries, this type of disease is typically detected at approximately age 2 years. More than 95% of such patients are cured by surgical removal of the affected eyeball, with eyesight preserved in the other eye. However, some patients remain at risk of the disease spreading and of recurrence.

To estimate the risk of disease recurrence and metastasis, doctors examine the surgically removed eye under a microscope and classify the tumor as either low-, intermediate-, or high-risk. All patients whose tumors are considered high-risk receive intensive postoperative chemotherapy and radiation therapy. There is no general consensus, however, on the need for adjuvant therapy and the optimal treatment approach for patients in the other two risk categories. Therefore, protocols for adjuvant chemotherapy use in these patient groups have varied widely.

"Our study confirms that it is not necessary to give adjuvant treatment to many children with unilateral retinoblastoma," said lead author Isabelle Aerts, MD, a pediatric oncologist at Institut Curie in Paris, France. "I think that our results will help establish the standard of care for such patients, reducing variations in postoperative chemotherapy protocols."

In the present study, 123 patients with unilateral, nonhereditary retinoblastoma were assigned to one of three risk groups according to previously established criteria. The median age at the time of eye removal surgery was 23 months. The 70 patients with low-risk disease received no postoperative chemotherapy; the 52 patients with intermediate-risk disease received four courses of adjuvant chemotherapy (etoposide, carboplatin, vincristine, cyclophosphamide), and the only patient with high-risk disease received six courses of high-dose adjuvant therapy (etoposide, carboplatin, thiotepa, vincristine, and cyclophosphamide).

At a median follow-up period of 71 months, all patients were alive. None of the patients experienced worsening disease, relapse, distant metastasis, or secondary cancers. The adjuvant chemotherapy was safe overall. These data confirm that the specific regimens administered in this study, which are already being used around the world, are very effective and safe. This study was published in Journal of Clinical Oncology (2013; doi:10.1200/JCO.2012.42.3962).

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