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Published on: May 4, 2017
Enrolling children with acute lymphoblastic leukaemia on a clinical trial improves event-free survival: a
Caron Strahlendorf1, Jason D Pole2, Randy Barber3
1British Columbia Children's Hospital, 4480 Oak St, Vancouver, BC V6H 3N1, Canada.
Insights
Children with acute lymphoblastic leukemia (ALL) enrolled in clinical trials at diagnosis had significantly better event-free survival. Further research is needed to understand barriers to trial enrollment in pediatric ALL patients.
Area of Science:
- Pediatric Oncology
- Clinical Trials
- Epidemiology
Background:
- Investigating the impact of clinical trial enrollment on survival outcomes in pediatric acute lymphoblastic leukemia (ALL).
- Utilizing a population-based approach to analyze survival data for children diagnosed with ALL.
Purpose of the Study:
- To determine if enrollment in a clinical trial at the time of diagnosis affects event-free survival (EFS) and overall survival (OS) in children with ALL.
- To analyze survival data using a population-based approach.
Main Methods:
- Retrospective cohort study of children aged 1-14 years newly diagnosed with ALL.
- Data sourced from the national Cancer in Young People in Canada (CYP-C) database.
- Univariate and multiple Cox proportional hazards models were employed for analysis.
Main Results:
- Out of 2569 children with ALL, 1408 (54.8%) were enrolled in a clinical trial at diagnosis.
- Five-year event-free survival was higher for those enrolled (89.8%±0.9) versus not enrolled (84.1%±1.2; P<0.0001).
- Five-year overall survival was also higher for enrolled children (94.1%±0.7) versus not enrolled (90.5%±1.0; P=0.001).
- Adjusted models showed trial enrollment significantly associated with better EFS (HR 0.67, P=0.023) but not OS (HR 0.69, P=0.102).
Conclusions:
- Event-free survival is significantly improved in pediatric ALL patients enrolled in clinical trials.
- Identifying barriers to clinical trial enrollment is crucial for future research and improving outcomes in pediatric ALL.
Background:
The objectives of this study were to describe the impact of trial enrollment at diagnosis on event-free and overall survival in paediatric acute lymphoblastic leukaemic (ALL) using a population-based approach.
Methods:
We conducted a retrospective cohort study that included children newly diagnosed with ALL between 1 and 14 years of age. The data source was the Cancer in Young People in Canada (CYP-C) national paediatric cancer population-based database. We conducted univariate and multiple Cox proportional hazards models.
Results:
There were 2569 children with ALL; 1408 (54.8%) were enrolled on a clinical trial at initial diagnosis. Event-free survival at 5 years was 89.8%±0.9 vs 84.1%±1.2. (P<0.0001) for those enrolled and not enrolled on a clinical trial, respectively. Overall survival at 5 years was higher for those enrolled (94.1%±0.7) vs not enrolled (90.5%±1.0; P=0.001). In a model that adjusted for demographic, leukaemic and socioeconomic factors, enrollment on trials was significantly associated with better event-free survival (hazard ratio (HR) 0.67, 95% confidence interval (CI) 0.47-0.95; P=0.023), but not overall survival (HR 0.69, 95% CI 0.44-1.08; P=0.102).
Conclusions:
Event-free survival was significantly better in children with ALL enrolled on a clinical trial. Future research should identify barriers to clinical trial enrollment for children with ALL.
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