Survival analysis of clinical, pathologic, and genetic features in neuroblastoma presenting as locoregional disease

BACKGROUND, Locoregional neuroblastoma is a clinical subgroup characterized by the absence of distant metastasis (International Neuroblastoma Staging System Stages 1, 2, and 3). Although these patients generally have an excel lent survival with minimal therapy, some do experience recurrence with leth al consequences. METHODS. To identify risk factors for disease progression, the authors perf ormed a retrospective analysis of clinical (age and stage) and tumor biolog ic markers (histology, MYCN, DNA index, and allelic analysis of chromosomes 1p, 11q12-qter, and 14q12-q32) in 44 patients (10 Stage 1, 18 Stage 2, and 16 Stage 3). Allelic analysis was performed using polymorphic polymerase c hain reaction markers in a semiautomated, fluorescent detection system. RESULTS. Sixteen patients (38%) were younger than 365 days at diagnosis. Se venteen of 39 tumors (43%) had unfavorable histology, 6 (13%) were MYCN amp lified, 14 (31%) were diploid, 17 (38%) had 1p36 loss of heterozygosity (LO H), 11 (25%) had 1p22 LOH, 10 (22%) had 11q LOH,and 13 (29%) had 14q LOH. S eventeen patients (38%) progressed, including 6 who progressed to Stage 4 d isease (13%). Sixteen patients with progressive disease received cytotoxic therapy. Thirty-seven patients are alive (84%) with a median follow-up of 5 1 months. By permutation log rank test, both MYCN amplification and diploid y were associated with overall survival (OS), but only diploidy was associa ted with progression free survival (PFS) and progression to Stage 4 disease . LOH of 1p36, 1p22, 11q, or 14q did not show correlation with either OS or PFS. CONCLUSIONS. Locoregional neuroblastoma tumors with diploid DNA index, rega rdless of other biologic features, have increased risk of local recurrence and Stage 4 progression. (C) 2001 American Cancer Society.