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Clinical and diagnostical problems of neuroblastoma

Treatment with polychemotherapy provokes a good initial response of NB. However on the one hand, disseminated stage 4 tumors frequently relapse due to minimal residual disease with a few resistant tumor cells, resulting in poor overall survival rates (< 35%). On the other hand, overtreatment of favorable, MYCN-negative stage 2 or 3 tumors is a major problem. Most surviving patients will have suffered from significant organ toxicity or development of secondary malignancies. Therefore, novel strategies to treat NB are urgently needed.

A wide range of diagnostic procedures is available to characterize NB. The prognostic predictive value is, however, uncertain, as shown by the heterogeneous treatment outcome even within the same defined risk groups. The combination of cytogenetic and molecular characterization with current technologies in expression profiling and proteomics will provide new diagnostic panels. This detailed information should allow intensity of therapy to be tailored to the needs of each individual NB patient. The new diagnostic panels may not only be utilized for precise diagnosis and classification, but also to identify the genetic and functional basis for intrinsic treatment resistance. Functional understanding of NB pathogenesis, regression and treatment resistance will provide new targets for intervention in certain biological tumor subtypes.

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