The primary specific objectives of this study include:
1) To prospectively analyze the factors that are currently used for risk-group assignment (MYCN copy
number by FISH, DNA content by flow cytometry, and tumor histology using the International
Neuroblastoma Pathologic Classification System) in neuroblastoma tumors at the time of diagnosis.
2) To maintain a reference bank containing clinically and genetically characterized frozen tumor
tissue, tumor DNA and RNA, histology slides and paraffin blocks, neuroblastoma-derived cell
lines, patient serum and paired normal DNA obtained at the time of diagnosis, at the time of
second-look surgery and relapse for future research studies.
3) To prospectively analyze 1p, 11q, 14q and 17q allelic status, MYCN copy number by quantitative
PCR; the expression pattern of neurotrophin-related genes; and telomerase activity in diagnostic
neuroblastoma tumors, and assay for the presence of rare tumor cells in biological specimens by
immunocytochemistry and RT-PCR. These biological variables will be analyzed for independent
clinical significance compared to MYCN amplification, INSS stage, age, ploidy, and histologic variables in
predicting either response to treatment or outcome.
4) To build a database of the known biologic prognostic factors for patients on therapeutic studies.
Adjustment for, or stratification by, these prognostic factors will be performed when testing for
treatment effect in Phase III trials.
5) To serve as a Registry for neuroblastoma patients whose tumors demonstrate clinical and genetic
features defined as "Low Risk" for treatment failure in the absence of adjuvant therapy.
A secondary objective of this study is to prospectively analyze the role of ferritin, LDH, and imaging-defined risk factors identified at the time of diagnosis in risk assessment. |