Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases

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
import.

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.

- INCLUSION CRITERIA:

- A POB, CCR investigator decides that it is in the best interest of the patient and the
POB, CCR for the patient to receive treatment and follow-up at the NCI/NIH.

- Children and adults with cancer (or a pre-cancer syndrome or a rare disease), between
the age (s) of 2 years - 40 years (the NIH CC is not well equipped to provide optimal
care to patients < 2 years of age). Note: Patients greater than or equal to 18 and
less than or equal to 40 years may be evaluated on this protocol if their cancer (or
precancer syndrome or rare disease) is of specific interest to the POB, CCR, NCI.

- Patients with cancer (or a pre-cancer syndrome or rare disease), who present with
disease manifestations of special interest to Pediatric Oncology Branch investigators,
including but not limited to, lymphoma, brain tumors, Ewing's sarcoma, leukemia,
neuroectodermal tumors, osteosarcoma, rhabdomyosarcoma,RAS-related syndromes and
inherited immune disorders, amongst others.

- Patient must weigh greater than or equal to 12 kg (the NIH CC is not well equipped to
provide optimal care to patients < 12 kg)

- The patient, parent, guardian or their Legally Authorized Representative (LAR) is able
and willing to provide informed consent.

- Patients, and when indicated, parent, guardian or LAR who are deemed sufficiently
reliable to return for recommended follow-up visits.

EXCLUSION CRITERIA:

- Patients younger than 2 years of age.

- Patients weighing less than or equal to 12 kg.

- Pregnant women are excluded from enrollment onto this study because the invasive
procedures and/or sedation needed to perform these may cause unnecessary harm to the
unborn fetus. In the event a woman becomes pregnant while on study, she will not be
removed from the study; however, no invasive clinical or research procedures will be
done or cancer treatment provided during pregnancy that include unacceptable risk to
the patient and/or to the unborn fetus.