Outcome and prognostic factors of children with bone marrow or bone metastatic neuroblastoma

Objective The presence of metastases is the risk factors in patients with neuroblastoma (NB). The aim of this study was to evaluate the clinical features and outcomes of metastatic NB treated in Xinhua hospital. Methods We analyzed 75 NB patients with metastases from January 2007 to December 2014 retrospectively in our hospital, including clinical manifestations, laboratory tests, stage, risk groups, pathology subtype, treatment protocol, outcome and complications were analyzed. Median follow-up time was 56.0 months with a minimum of 1.1 months and a maximum of 133.3 months. Results In all 75 NB patients with metastasis, fifty-two were male (69.3%) and twenty-three female (30.7%), middle age at diagnosis was 39.1 months. Seventeen cases (22.7%) were intermediate risk group, fifty-eight (77.3%) were in very high risk group. Fifty-two cases (69.3%) had metastasis with bone marrow at diagnosis, forty-five (60.0%) with bone and thirty (40.0%) with lymph nodes. The 5-year overall survival (OS) and event-free survival (EFS) were (45.3±5.7)% and (31.1±5.6)% respectively. The 5 year OS in intermediate risk and very high risk group were (80.7±12.7)% and (43.0±6.5)%(P<0.001). The 5-year OS in early and late bone marrow response groups were (64.3±9.1)% and (40.0±11.0)%(P=0.017), the 5-year OS in single bone and multiple bone metastases were (66.7±11.1)% and (33.3±9.1)% respectively (P=0.045). The multivariate analysis revealed that patients with bone metastasis (HR: 2.2, 95%CI 1.1-4.8, P=0.028) could be regarded as independent poor prognostic factors. Conclusions The most common metastatic sites of NB were bone marrow and bone, children with complete remission of bone marrow less than 2 courses or with single bone metastasis had a better prognosis. N-MYC gene amplification reduced the survival rate of the children younger than 18 months, the parameters such as serum lactate dehydrogenase, neuron-specific enolase, ferritin, and urine VMA at the time of diagnosis had no independent prognostic value. Key words: Neuroblastoma; Metastasis; Children; Clinical features; Prognosis