Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases
Condition: Ewing's Sarcoma; Leukemia; Lymphoma; Brain Tumors; Rhabdomyosarcoma
MeSH terms: Lymphoma; Sarcoma; Brain Neoplasms; Rhabdomyosarcoma; Sarcoma, Ewing
Study type: Observational

Summary: 
      This protocol is designed to evaluate children with cancer who appear to be probable
      candidates for future protocol entry or have disease manifestations that are of unique
      scientific importance or educational value.
    

Detailed description: 
      Background:

      Children and adults are referred to the Pediatric Oncology Branch (POB) for possible
      enrollment in clinical protocols for the treatment of cancer or other rare diseases. While
      some children are not eligible for a specific protocol, they may present with disease
      manifestations that offer the potential for important new insights into the pathogenesis or
      clinical behavior of their underlying disease. In addition, children who have completed
      participation in a clinical protocol but do not currently have therapeutic protocol
      alternatives may continue to provide POB with important information. Diseases of interest to
      the POB include, but are not limited to lymphoma, brain tumors, Ewing's sarcoma, leukemia,
      neuroectodermal tumors, osteosarcoma, rhabdomyosarcoma, RAS-related syndromes and inherited
      immune disorders, amongst others. Hence, serial clinical evaluation of such patients,
      including the performance of clinical, laboratory, and diagnostic studies to help elucidate
      longitudinally the underlying disease mechanisms, and when clinically indicated standard care
      therapies, will assist POB meet its overall mission.

      Objectives:

      To be able to follow and evaluate children and adults with cancer or pre-cancer syndromes and
      other rare diseases referred to the Pediatric Oncology Branch who present with disease
      manifestations that lend themselves to clinical evaluation and are of unique scientific
      importance.

      Eligibility:

      Patients who are evaluated by the Pediatric Oncology Branch and are:

        -  Children and adults with cancer (or a pre-cancer syndrome or rare disease), with disease
           manifestations of special interest to Pediatric Oncology Branch investigators.

        -  Patients must be greater than or equal to 2 years and less than or equal to 40 years of
           age at the time of study enrollment.

      Design:

      The medical procedures or tests will be selected for each patient on the basis of his/her
      individual diagnosis (or presumed diagnosis). When clinically indicated, standard therapy
      will be administered and patients will be followed according to standard medical practice.
      Specimens may be collected for clinical care purposes only to evaluate the patient s status.
      No investigational tests, drugs or therapies will be administered in this protocol.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Children and adults with cancer (or a pre-cancer syndrome/rare disease), between the age(s)
        of 2 years - 40 years, who present with disease manifestations of special interest to POB
        investigators.
      
Keywords: Leukemia; Ewing's Sarcoma; Neuroectodermal Tumors; Lymphoma, Osteosarcoma; Brain Tumors, Rhabdomyosarcoma; Lymphoma; Neoplasm; Sarcoma