Therapeutic interventions for childhood cancer: An umbrella review of randomized evidence

Area of Science:

• Pediatric Oncology
• Evidence-Based Medicine
• Systematic Reviews

Background:

• Pediatric cancer treatment advancements have improved outcomes.
• The strength of evidence supporting these advancements requires critical evaluation.

Purpose of the Study:

• To conduct an umbrella review of meta-analyses of randomized controlled trials (RCTs) on pediatric cancer interventions.
• To critically appraise the evidence for efficacy and safety of pediatric cancer treatments.

Main Methods:

• Searched for meta-analyses of RCTs evaluating pediatric cancer therapies.
• Included 14 publications comprising 68 meta-analyses with 31,496 participants.
• Assessed heterogeneity, small-study effects, and excess-significance bias.

Main Results:

• Acute lymphoblastic leukemia (ALL) was the most studied malignancy.
• Methotrexate and vincristine-prednisone pulses showed the strongest evidence for improving event-free survival in ALL.
• Evidence for other pediatric cancers was generally weaker, with substantial heterogeneity noted in some associations.

Conclusions:

• The current evidence base for pediatric cancer treatments is limited, with a focus on ALL.
• Adult trial data may serve as indirect evidence, but more pediatric-specific RCTs and individual patient data meta-analyses are necessary.
• Increased high-quality evidence is crucial for precise and certain care of pediatric cancer patients.