Clinical Trials : Pediatric Cancer

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I) Introduction 1. Pediatric cancer 1) Approximately 70% of children diagnosed with cancer in the United States are cured, with a 5-year event-free survival rate of 80%1 2) Pediatric cancer is relatively rare - around 12,000 new cases diagnosed each year in the United States1 3) Pediatric oncology trials typically have high rates of accrual2 1. High accrual must be balanced with ethical treatment2 2. Overall goal is “optimal recruitment”, which focuses on allowing families to make informed decisions2 4) Drugs are typically tested and approved in the adult population before being introduced to children3 1. Children are pharmacologically different than adults, thus a need exists for pediatric clinical trials – possibility of different adverse event profile, etc.3 2. Challenges involving pediatric cancer trials 1) Ethics of phase I trials in children 1. Definition of phase I trial (i) Purpose is to determine the safety (maximum tolerated dose), toxicity, and pharmacokinetics4 (ii) Phase I trials do not assess efficacy of the drug4 2. Safety of phase I trials (i) Treatment-related mortality rate of 0.5%4 (ii) Overall well-tolerated – only 25% of patients typically experience a grade 3 or higher toxicity4 3. Understanding of risks and benefits of phase I trial (i) Phase I trials can involve toxicity and only provide a minimal chance of direct benefit to the child – 6.8% for single novel agent and 20.1% for novel agent/known anti-cancer drug combination4 (ii) Potential benefits
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