NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors

Background:

• Rare cancers are defined by the NCI as fewer than 15 incident cases per 100,000 people per year. Overall, pediatric tumors account for less than 1% of all cancers diagnosed in the United States (US) per year.
• The European Cooperative Study Group for Pediatric Rare Tumors (EXPeRT) group has determined that the definition of very rare pediatric cancers is fewer than 2 cases per million per year and/or those not eligible for clinical trials.
• Frequently, for these very rare solid tumors and hematologic malignancies, little is known about their natural history, including clinical behavior, molecular/genetic characteristics, optimal management, drug response, and evidence-based standard therapy does not exist. Many patients endure the challenges of achieving an accurate and timely diagnosis, along with the difficulty of identifying a center with treatment expertise.
• Although several disease-specific pediatric and adult rare cancer efforts exist, they are frequently siloed without standardization across efforts; and, many rare cancers have not been systematically studied.

Objective:

-To establish a longitudinal observational study and registry for very rare pediatric and Adolescents and Young Adults (AYA) solid tumors

Eligibility:

-Pediatric, adolescent, or young adult participants (<= 39 years old at diagnosis) with very rare solid tumors

Design:

• This will be a longitudinal observational study and registry for children and AYAs with very rare cancers.
• Recruitment will be through 2 mechanisms, the Children s Oncology Group s (COG) Project EveryChild Protocol (APEC14B1, NCT02402244) and physician/self-referral.
• This study will be led by the NCI s CCDI and NCI s POB.
• Enrollment, including consent, distribution of family history/individual medical history/patient report outcome questionnaires, collection of medical records and tissue/saliva/buccal samples, and extraction of data, as well as longitudinal follow-up will be managed centrally.
• Tumor tissue and saliva/buccal samples will be run through the CCDI s Molecular Characterization Initiative (MCI) for comprehensive clinical molecular profiling. The remaining samples will be banked for research analyses. Research analysis will be per the CCDI research characterization pipeline.
• Since long-term follow-up of individuals with very rare cancers is a major feature of the study, we intend to maintain active contact with study participants for up to 5 years.
• Data will be deposited into the CCDI Data Ecosystem.

• INCLUSION CRITERIA:
• 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.

Exclusion criteria

• Diagnosis of any of the following at any time:

• Ewing Sarcoma
• Osteosarcoma
• Rhabdomyosarcoma
• Diffuse midline glioma (H3K27 altered)
• Atypical teratoid rhabdoid tumor
• Pleuropulmonary blastoma
• Common adult cancers that occur in pediatric/AYA populations (i.e., colorectal cancer, breast cancer)
• The participant is unlikely to comply with the terms of the protocol.