A Snapshot of Pediatric Cancers
Incidence and Mortality
Cancer is the leading cause of death by disease past infancy among U.S. children. An estimated 10,450 new cases of cancer are expected to be diagnosed in children ages 0 to 14 years in 2014. The major types of cancers in children ages 0 to14 years are acute lymphocytic leukemia (ALL), brain and other central nervous system (CNS) tumors, and neuroblastoma, which are expected to account for more than half of new cases in 2014. White and Hispanic children have higher incidence rates of childhood cancer than children from other racial/ethnic groups.
Although the overall incidence rate of cancer in children has increased 0.6% annually over the past 35 years, mortality rates for some types of childhood cancer have declined by more than 50 percent.1 The 5-year survival rate for all childhood cancers combined has increased from 60 percent in the mid-1970s to 86 percent in recent years. The improvement in survival is largely attributable to better treatments and to the high proportion of pediatric patients participating in clinical trials. Although outcomes have improved for many childhood cancers, progress in others has been limited. Brain and central nervous system tumors remain the leading cause of cancer-related death in children.
The causes of most childhood cancers are unknown, and for the most part, these cancers cannot be prevented. Depending on the type of cancer, children with cancer may receive different standard treatments, including surgery, chemotherapy, cerebrospinal fluid diversion, radiation therapy, watchful waiting, chemotherapy with stem cell transplant, biological therapy, or targeted therapy.
Examples of NCI Activities Relevant to Pediatric Cancers
- The Therapeutically Applicable Research To Generate Effective Treatments (TARGET) initiative is identifying prognostic markers and therapeutic targets to develop new, more effective treatments for children with cancer.
- The Biomarker, Imaging and Quality of Life Studies Funding Program (BIQSFP) supports biomarker, imaging, and quality-of-life studies, with or without cost-effectiveness analysis, as part of randomized cancer clinical trials. Nine BIQSFP studies are focusing on biomarkers or quality of life in six childhood cancers.
- The NCI-supported Childhood Cancer Survivor Study is following more than 14,000 childhood cancer survivors and approximately 4,000 of their siblings to assess their mortality rates, determine their risks of developing subsequent cancers, and better understand the long-term effects of cancer treatments on the heart, the lungs, and fertility.
- The Center for Cancer Research’s Pediatric Oncology Branch conducts basic science research and clinical trials to improve outcomes for children with cancer or genetic tumor predisposition syndromes.
- The Epidemiology and Genomics Research Program supports research and resources focusing on interdisciplinary and translational cancer research, including three Childhood Cancer Epidemiology Consortia that conduct multidisciplinary research on clinical, infectious, environmental, and genetic risk factors in the etiology of childhood cancer.
- The Tumor Microenvironment Network (TMEN) is exploring the role of the microenvironment in tumor initiation and progression. One TMEN center is conducting research to identify inhibitors of environment-mediated drug resistance pathways and test these in clinical trials in children with ALL and neuroblastoma.
Selected Advances in Pediatric Cancers Research
- A comprehensive genetic analysis of paired samples of tumor and normal tissue from 147 children with the pediatric soft-tissue tumor rhabdomyosarcoma revealed two distinct genetic types of this tumor based on the presence or absence of a specific fusion gene and the recurrent mutation of multiple other genes. Published January 2014. [PubMed Abstract]
- Genetic analysis of patients with treatment-naive high-grade astrocytomas, a rare and incurable group of pediatric brain tumors, identified recurrent mutations specific to tumor location within the brain. Some of these mutations are potentially susceptible to blocking by existing drugs, and others are potential targets for drug development. Published April 2014. [PubMed Abstract]
- Genetic analysis of inflammatory myofibroblastic tumor, a rare form of childhood soft tissue sarcoma, identified gene fusions that result in a therapeutically targetable kinase protein in 85% of the 37 tumor samples examined, suggesting that routine molecular profiling of these cancers could inform treatment for individual patients. Published May 2014. [PubMed Abstract]
- Overexpression of LIN28B, an RNA binding protein that is often dysregulated in pediatric hepatoblastoma, is sufficient to initiate liver cancer and necessary for liver cancer maintenance in mouse models. Published August 2014. [PubMed Abstract]
Trends in NCI Funding for Pediatric Cancers Research
NCI’s investment2 in pediatric cancers research was $185.1 million in fiscal year (FY) 2013. In addition to the funding described in the graph, NCI supported $60.4 million in pediatric cancers research in FYs 2009 and 2010 using funding from the American Recovery and Reinvestment Act.
Additional Resources for Pediatric Cancers
- NCI Childhood Cancers Home Page
NCI's gateway for information about pediatric cancers.
- Cancer in Children and Adolescents
A fact sheet about statistics, types, causes, and treatment of cancers in children and adolescents in the United States.
- Young People with Cancer: A Handbook for Parents
Overview on the diagnosis and treatment of childhood cancers, including common medical procedures and side effects. Suggests ways to talk with children about cancer, support family life, and manage practical issues.
- PDQ® Cancer Information Summaries: Pediatric Treatment
Treatment options for childhood cancers.
- Late Effects of Treatment for Childhood Cancer (PDQ®)
Expert-reviewed information summary about the health problems that continue or appear after cancer treatment has ended.
- 1 Includes cancers in children 0-18 years of age.
- 2 The estimated NCI investment is based on funding associated with a broad range of peer-reviewed scientific activities. For additional information on research planning and budgeting at the National Institutes of Health (NIH), see About NIH.