Clinical trial participation alone may not be associated with improved outcomes for childhood cancer patients

Carl Koschmann, M.D., and colleagues at the University of Washington and Seattle Children's Hospital studied 322 patients with newly diagnosed acute lymphoblastic leukemia (a cancer involving the white blood cells), approximately half of whom participated in one of several available clinical trial protocols for the disease.

Overall, 79 percent of the patients survived five years without recurrence of their leukemia, with no significant difference between study participants and non-participants (80 percent vs. 77 percent). "Clinical trial participation does not, by itself, lead to improved outcome for pediatric patients with acute lymphoblastic leukemia in the current era," the authors conclude. "Discussions about participation in a clinical trial should focus on improvement of future therapy, not the direct benefit of the research participant."

"Considerable evidence indicates that clinical trials are associated with substantial benefits as measured by both public health and economic gains," writes Steven Joffe, M.D., M.P.H., of the Dana-Farber Cancer Institute, Boston, in an accompanying editorial. "In the modern era, five-year survival rates for children with acute lymphoblastic leukemia are 80 percent to 90 percent. It is likely that these remarkable outcomes owe much not only to the legacy of treatment insights derived from past trials, but also to the happy side effect of improved quality attributable to the existence of a cooperative clinical trials program and infrastructure in the settings in which most children with cancer receive their care."

The research appears in Archives of Pediatrics & Adolescent Medicine.