No evidence of a trial effect in newly diagnosed pediatric acute lymphoblastic leukemia.

OBJECTIVE

To determine whether clinical trial enrollment by itself is associated with improved outcome.

DESIGN

Retrospective cohort study.

SETTING

Seattle Children's Hospital from 1997 to 2005.

PARTICIPANTS

Data were drawn from 322 patients with newly diagnosed acute lymphoblastic leukemia. Main Exposure Enrollment in a Children's Oncology Group or Children's Cancer Group clinical trial.

MAIN OUTCOME MEASURES

(1) Demographic variables associated with trial participation. (2) Event-free survival, which was defined as the time from initial diagnosis to either leukemia recurrence or death from any cause.

RESULTS

No outcome advantage was found for participants in a clinical trial compared with nonparticipants. Additionally, there were not demographic factors associated with increased clinical trial participation.

CONCLUSIONS

Clinical trial participation does not, by itself, lead to improved outcome for pediatric patients with acute lymphoblastic leukemia in the current era. Discussions about participation in a clinical trial should focus on improvement of future therapy, not the direct benefit of the research participant.