Background: In neuroblastomas (NBs), high mitotic-karyorrhectic index (High-MKI) defined by the International Neuroblastoma Pathology Classification is often associated with MYCN genomic amplification. However, little is known about MYCN and/or MYC protein expression by High-MKI NBs especially when MYCN oncogene is not amplified.
Materials and Methods: A total of 241 High-MKI NBs [120 MYCN amplified (28 undifferentiated, 92 poorly differentiated subtype) and 121 MYCN non-amplified (4 undifferentiated, 117 poorly differentiated subtype)] available at the Children’s Oncology Groups Neuroblastoma Pathology Reference Laboratory were studied immunohistochemically for expression of MYCN and MYC protein. Prognostic effects by these protein expressions [(+): high, (+/-): low, (-): none] were analyzed with conventional prognostic factors and prominent nucleolar (PN) formation.
Results: MYCN amplified High-MKI NBs were predominantly composed of tumors overexpressing MYCN protein: they were 101(84.2%) MYCN protein (+), one (0.8%) MYC protein (+), 2(1.7%) both proteins (+), and 16(13.3%) both proteins (-)/(+/-). In contrast, MYCN non-amplified High-MKI NBs were heterogeneous and included tumors of 7(5.8%) MYCN protein (+), 36(29.8%) MYC protein (+), 3(2.5%) both proteins (+), and 75(62.0%) both proteins (-)/(+/-). MYC-family-driven NBs defined by augmented expression of MYCN/MYC protein were more likely to show PN formation (putative site of RNA synthesis and accumulation). High-MKI NBs linked to a poor prognosis (4-year EFS 56.2+/-5.5%, 4-year OS 64.5+/-5.4%) irrespective of MYCN genomic status (amplified or non-amplified). However, patients with MYC-family-driven High-MKI NBs had significantly lower survivals (4-year EFS 50.2+/-6.2%, 4-year OS 56.1+/-6.1%) than those with Non-MYC-family-driven High MKI NBs (4-year EFS 66.3+/-11.1%, 4-year OS 80.1+/-9.5%) (p=0.0131 and p=0.0064, respectively). MYCN protein (+) and PN (+), in addition to clinical stage, were independently predictive of poor survivals in this cohort of patients.
Conclusions: Among High-MKI NBs, MYC-family-driven tumors with augmented MYCN/MYC protein expression were associated with PN formation and behaved significantly more aggressively than Non-MYC-family-driven tumors.