All childhood cancers are considered rare; however, about 11% are very rare cancers—less than two cases per million people are seen each year. These very rare childhood cancers may be hard to diagnose and lack effective treatments and standardized care. There is also little information or understanding of how or why these cancers occur and how they behave in a young person’s body over time.
Through collection of standardized data and community collaboration, the CCDI Pediatric, Adolescent, and Young Adult Rare Cancer Study could advance our understanding of very rare childhood cancers and help improve outcomes for children, adolescents, and young adults (AYAs) with these cancers.
Which childhood cancers are very rare?
Very rare childhood cancers include many different histologies. Some examples of very rare cancers include adrenocortical, nasopharyngeal, thyroid, and any other (extra-cranial) carcinomas, as well as desmoplastic small round cell tumors, gastrointestinal or gonadal stromal tumors, neuroendocrine tumors, pancreatoblastoma, pleuropulmonary blastoma, and retinoblastoma.
What are the goals of this rare childhood cancer study?
The CCDI Pediatric and AYA Rare Cancer Study aims to:
- Build on knowledge and experience gained from other rare cancer programs.
- Establish the structure and resources needed to study rare childhood cancers and develop effective treatments.
What is this study?
The CCDI Pediatric and AYA Rare Cancer Study is a longitudinal, observational study that will follow children and AYAs with very rare cancers over time. Data from this study could help make meaningful comparisons across multiple cancer types and identify molecular targets. These data could also inform treatment and care recommendations.
Who can participate?
To join, a child or AYA must be diagnosed with a rare solid tumor. Enrollment will open to those with blood cancers in the future. To participate, a child or AYA must:
- Be 39 years old or younger
- Have been diagnosed less than one year ago
- Have been diagnosed with a rare solid tumor
- Be willing to provide tumor and saliva/cheek swab samples for testing
A child or AYA may enroll from the Children’s Oncology Group Project:EveryChild, which is open to young people with cancer who are 25 years old or younger. If they are not being seen at a Children’s Oncology Group–affiliated hospital, they can refer themselves to the study by talking with their doctor or contacting the study team at NCICCDIRCI@mail.nih.gov.
How long is this rare childhood cancer study and what is involved?
Follow-up of people with very rare cancers over time is a key part of this study, so we plan to stay in touch with participants for up to five years.
There will be no experimental treatments or in-person appointments—just surveys, data collection, and tumor, saliva, and cheek swab sample collection. Treatment will continue at participants’ home hospitals with their local oncologist.
What will happen to the data collected?
The primary goal of this study is to develop a registry containing a core data set on all rare cancer participants. It will include real-world data that can inform the design of rare childhood cancer clinical trials. For example, registry data for subsets of tumors could be used to create external control groups for trials.
Who can you access data from the study?
Data generated and collected through this study will be available through the CCDI Data Ecosystem. More information about these data will be shared as the initiative progresses.
Who can you contact with questions about the initiative?
For questions about the CCDI Pediatric and AYA Rare Cancer Study, NCICCDIRCI@mail.nih.gov.
For questions about CCDI, email NCIChildhoodCancerDataInitiative@mail.nih.gov.