The importance of determination of serum
neuron-specific enolase (NSE) in patients with
neuroblastoma has been emphasized by several authors. However, the specificity and sensitivity of NSE have not yet been well studied in
tumors of infancy and childhood, nor is the role of serial determination of NSE in monitoring these patients fully understood. Concentrations of serum NSE were determined by a newly developed radioimmunoassay technique in 241 samples from 111 patients. NSE was also assayed in sera of nude mice bearing human pediatric
tumors (16 samples), as well as in 30
tumor specimens. Eighty-two serum samples from 19 patients with
neuroblastoma all showed NSE values (mean 120.2 ng/mL, range 16.2 to 722.0 ng/mL) elevated beyond the upper border of the normal range (14.6 ng/mL), even though four of the 19 patients had normal urinary excretion of
3-methoxy-4-hydroxymandelic acid (VMA) and 3-methoxy-4-hydroxy-phenylacetic
acid (HVA). Twelve of these patients were monitored with serial NSE determinations, and their serum NSE were found to correlate well with the
tumor burden, but were transiently modified by chemotherapeutically induced cell death. All 68 samples from nine patients, free of
neuroblastoma at assessment, showed NSE values within the normal range. Thirteen of 25 patients with
tumors other than
neuroblastoma, however, showed serum NSE values mildly elevated beyond the upper border of the normal range (mean of the 25 patients 36.7 ng/mL, range 5.0 to 234.0 ng/mL). Results from our nude mouse study and from NSE analysis of the
tumor extracts paralleled the clinical results.(ABSTRACT TRUNCATED AT 250 WORDS)