Background:
All childhood cancers are rare, but some are called very rare. Very rare cancers are diagnosed in 2 or fewer out of 1 million people each year. Researchers want to gather data so they can learn more about these very rare cancers. They hope to use the data to develop future treatments.
Objective:
To gather data for a registry of very rare cancers found in children, teens, and young adults.
Eligibility:
People aged 1 month to 39 years newly diagnosed (within the past year) with a very rare cancer.
Design:
Participation will be by phone or email. No clinic visits are required.
Researchers will look at the participant s medical records. They will ask for samples of tumor tissue that were already removed. They will use the samples for genetic testing. The results of these tests will be sent to the participant s own doctors.
Some participants will be asked for saliva or cheek swab samples. They will receive a kit in the mail. They will spit into a tube or swab the inside of their cheek. They will mail the sample back to the lab.
Participants will fill out questionnaires once a year for 5 years. They will answer questions about:
Family history, such as other cancers in the family and their income, work, and education.
Demographics, such as their gender, nationality, ethnicity, education, and work history.
Symptoms and treatment for their cancer. This may include level of pain, and emotional and physical well-being.
Participants data will be added to a secure database for other researchers. Their data will be anonymous.
History of newly diagnosed (within 1 year of diagnosis) very rare solid tumor (defined as an estimated 2 incident cases per million per year).
Age >= 1 month and <= 39 years at the time of diagnosis.
Participants must have established care with a local treating physician.
Ability of the participant, parent/guardian, or Legally Authorized Representative (LAR) to understand and the willingness to sign a written informed consent document.
Ewing Sarcoma
Osteosarcoma
Rhabdomyosarcoma
Diffuse midline glioma (H3K27 altered)
This is a longitudinal observational study and registry for children, adolescents and young adults (AYAs) with very rare cancers.
Case-only
Prospective
Natural history study of individuals with very rare tumors
Other interventionWe will collect information about the initial presentation and diagnosis of the disease, management, and tumor treatment history. Participants or parent/guardian will be asked to complete questionnaires and patient-reported outcome (PRO) instruments. Pathology materials (e.g., tissue samples, slides, or blocks) and saliva and/or buccal sample will be requested.
To establish a longitudinal observational study and registry for very rare pediatric and AYA solid tumors
Percentage of participants from identified recruitment sources will be tabulated and described. An analysis of the ability to adequately obtain medical records at initial evaluation and follow-up to perform medical data extraction, which is critical to establishing a registry and longitudinal observational study will be assessed.
To evaluate the feasibility of PRO using validated reporting platforms suitable for pediatric and AYA populations
The percentage of participants who successfully complete longitudinal data collection will be described.
To conduct comprehensive clinical molecular characterization, utilizing CCDI MCI
The percentage of participants who successfully provide required sample(s) and where correlative analyses are able to be performed will be described.
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