My Pediatric and Adult Rare Tumor (MyPART) Natural History Study of Rare Solid Tumors

The goal of this study is to comprehensively and longitudinally evaluate the natural history of patients with rare solid tumors or tumor predisposition syndromes, estimating and defining their clinical spectrum (e.g. disease course and survival). Clinical data, patient reported outcomes (PROs), and biospecimen associated data are collected. Clinical data includes response to treatment, tumor pathology, and imaging. PROs include measures of pain, depression, anxiety, mobility, and cognitive functioning. Biospecimen data includes next-generation sequencing [RNAseq, whole exome sequencing (WES), whole genome sequencing (WGS), EPIC methylation array data, and TruSight Oncology 500 gene panel sequencing], cytokine analysis of plasma, immune phenotyping of circulating peripheral blood mononuclear cells.]]>
NCI/CCR/POB/Rare Tumor Protocol - Family History QuestionnaireNCI/CCR/POB/Rare Tumor Protocol Individual Information QuestionnaireNCI/CCR/POB/Rare Tumor Protocol Follow-up Individual Information QuestionnairePhysical ExamPROMIS Behavioral HealthReview of SystemsTreatment By Event FormTumor Evaluation FormTumor Pathology Report, Tumor Evaluation FormTumor Presentation FormInclusion CriteriaCohort 1: Subjects with a diagnosis of rare tumor (fewer than 15 cases in 100,000 people per year)Cohort 2: Relatives of subjects with a rare tumor who have a germline genetic variant that predisposes to a rare solid tumor or a subject who has a germline genetic variant that predisposes to a rare solid tumor Cohort 3: Relatives of subjects with a diagnosis of rare tumor that do NOT have known germline genetic variants that predispose to a rare solid tumor Cohort 4: Parents/guardians of children with a diagnosis of rare tumor participating in focus groups (if not enrolled in Cohorts 1, 2, or 3) No exclusion criteria ]]>
January 2018 – first participant enrolled March 2020 – COVID pandemic limits enrollment to the Clinic Group January 2021 – first follow-up forms sent to participants ]]>