Clinical relevance of loss heterozygosity of the short arm of chromosome 1 in neuroblastoma: a single-institution study.

Neuroblastoma is characterized by a wide variability of its clinical course, and considerable effort has been made to identify factors determining outcome in this disease. In a series of 82 patients from a single institution, we have investigated the prognostic impact of multiple clinical, biological and genetic parameters. Univariate testing showed that advanced stage of disease, abdominal localization of the primary tumor, elevated urinary dopamine levels, N-myc amplification (NMA) and loss of heterozygosity of chromosome lp (LOH lp) were related to a poor outcome. Most of these parameters were strong indicators of treatment failure in children younger than 12 months of age but none of them, apart from stage, had a significant prognostic impact in patients older than 12 months at diagnosis. Interestingly, the shorter survival time associated with the presence of lp deletions or NMA appears to be more strongly linked to a poorer outcome after relapse or progression than to a shorter progression-free interval. Although different types of LOH lp have been described in neuroblastoma and may be associated with different biological features, as suggested by a different pattern of catecholamine secretion, tumors with LOH lp present an aggressive clinical behavior, regardless of the type of LOH lp. In this study, LOH lp is an indicator of poor prognosis and identifies a larger population at risk than NMA alone.