Back ground
A high ALK protein expression, unfavorable histology (UH) according to the International Neuroblastoma Pathology Classification (INPC) and MYCN amplification are associated with a poor outcome in neuroblastoma (NB). Correlations between a high ALK expression and histology are inconclusive.
Method
Immunohistochemistry for ALK and INPC was evaluated in 195 NBs analyzed for MYCN amplification, 1p/11q LOH, 17q gain, and ALK mutations. Over 50% positivity of tumor cells indicated a high ALK expression. Correlations between the ALK expression and INPC and genomic alteration(s) were analyzed for prognostic significance.
Results
Eighty-six NBs with high ALK expression levels and 109 NBs with low ALK expression levels were identified. High ALK expressors significantly demonstrated UH (70/86, p<0.0001). MYCN amplification and 11q LOH were not associated with high ALK expression levels. High ALK expressors with a favorable histology (FH) were younger than those with UH; median age at diagnosis was 6.5 months and 36.5 months, respectively. Among ALK high expressors, ALK mutations were detected in 12/70 (17%) in the UH group, and 4/16 (25%) in the FH group. In the FH group, high ALK expressors showed a significantly inferior overall survival rate (OS)(58%) than low ALK expressors (100%) (p=0.001), while ALK expression was not associated with the prognosis in UH (OS was 41% and 50%, respectively). FH and MYCN nonamplified (NA) tumors showed less ALK high expression (14/65; 22%) and 11q LOH (9/65; 14%)(p<0.0001). UH and MYCN-NA tumors tended to exhibit 11q LOH (43/70; 61%)(p=0.056), but not a high ALK expression. UH and MYCN amplified tumors were associated with high ALK expression levels (36/56; 64%)(p=0.033) and less 11q LOH (9/56; 16%)(p<0.0001).
Conclusion
A high expression of ALK correlated with UH, but not MYCN amplification or 11q LOH. In the FH group, but not in UH, high ALK expressors had an inferior survival.