In very young children with surgically treated medulloblastoma with metastases, lengthy remissions can be obtained with intensive chemotherapy without radiation therapy, according to a report in the March 10th issue of The New England Journal of Medicine. Investigators also found that cognitive function during follow-up is not affected as drastically as it is when radiation therapy is applied.

The prognosis for young children with medulloblastoma is poor and radiotherapy-induced cognitive deficits are common, lead researcher Dr. Stefan Rutkowski, a pediatric oncologist at the University of Wurzburg in Germany, and his associates note.

In their study, they resected the tumors of 43 patients younger than 3 years old, and administered three 2-month cycles of chemotherapy (cyclophosphamide, methotrexate, vincristine, carboplatin and etoposide).

In addition, an intraventricular subcutaneous reservoir was implanted in the anterior horn of a lateral ventricle, “with the aim of prolonging cytotoxic methotrexate levels in the cerebrospinal fluid by giving repeated small single doses,” the investigators report.

Complete resection of the tumor was accomplished in 17 patients, while residual tumor remained in 14 and macroscopic metastases were evident in 12 patients. Residual disease was further treated with surgery, chemotherapy or radiation therapy.

Altogether, the 5-year progression-free and overall survival rates were 58% and 66%, the authors report.

The 5-year progression free survival rates were 82%, 50% and 33% for those with complete resection, residual tumor, and metastases, respectively, and overall survival rates were 93%, 56% and 38%.

Risk of relapse or death was significantly lower for children with desmoplastic medulloblastoma, no evidence of metastasis, and those older than 2 years. There were no signs of methotrexate-induced neurotoxicity.

Among 14 patients who underwent detailed neuropsychological testing at a median of 4.8 years, IQ was lower than the general population of the same age. Among those not receiving any radiation therapy, IQ was higher than documented in other studies of patients who underwent radiotherapy.

“Results are especially promising for patients without initial metastases, in whom radiotherapy should be reserved for salvage strategies at relapse,” Dr. Rutkowski’s group indicates.

This report “represents a substantial step forward in the treatment” of medulloblastoma, Dr. Lisa M. DeAngelis, at Memorial Sloan-Kettering Cancer Center in New York, notes. She agrees that “radiotherapy may be withheld in the very young without compromising disease control.”

(Source: N Engl J Med 2005;352:978-986,1036-1038: Reuters Health: Oncolink: March 2005.)

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