NEW YORK (Reuters Health) - Systematic local therapy may be selectively avoided in certain children with nonmetastatic rhabdomyosarcoma, according to findings of a multinational study seeking to improve treatment of the condition. A particular aim was to restrain use of radical surgery and radiation.

"The thrust of the approach taken in this study," lead investigator Dr. Michael C. G. Stevens told Reuters Health, "should encourage clinicians to be as critical about the consequences of cure as they are at measuring their ability to achieve cure."

"The choice of treatment," he added, "must always attempt a balance between achieving a net reduction of risk of late sequelae for the majority of patients, without compromising the overall chance of cure."

In the April 20th issue of the Journal of Clinical Oncology, Dr. Stevens of the University of Bristol, UK, and colleagues report on a study of 503 previously untreated patients (median age 4.7 years) who received one of six treatment schedules by site and stage.

The 5-year overall survival was 71% and the event-free survival was 57%. In total, 34 patients died of the disease without achieving control and 4 patients died of treatment-related toxicity.

Moreover, patients with stage III disease treated with a novel 6-drug combination showed improved survival (60%) than that seen in a previous international study (42%).

Overall survival was not significantly higher than that in the earlier study, but 49% of survivors were cured without significant local therapy.

In an accompanying editorial, Dr. Sarah S. Donaldson of Stanford University School of Medicine, California and Dr. James R. Anderson of the University Nebraska School of Medicine, Omaha, point out that patients who relapse may require very aggressive local therapy at the risk of unacceptable morbidity.

Understanding the total burden of therapy, they add, will require years of follow-up. Until then, it must be accepted that in some patients "success may be compromised by a treatment approach that is conservative with respect to local therapy."

J Clin Oncol 2005;23:2618-2828.

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