Abstract
BackgroundInfants diagnosed with stage 4 s neuroblastoma commonly experience spontaneous disease regression, with few succumbing without response to therapy. We analyzed a large cohort of such infants enrolled in the Italian Neuroblastoma Registry to detect changes over time in presenting features, treatment and outcome.MethodsOf 3355 subjects aged 0–18 years with previously untreated neuroblastoma diagnosed between 1979 and 2013, a total of 280 infants (8.3%) had stage 4 s characteristics, 268 of whom were eligible for analyses. Three treatment eras were identified on the basis of based diagnostic and chemotherapy adopted. Group 1 patients received upfront chemotherapy; Group 2 and 3 patients underwent observation in the absence of life-threatening symptoms (LTS), except for Group 3 patients with amplified MYCN gene, who received more aggressive therapy.ResultsThe three groups were comparable, with few exceptions. Ten-year overall survival significantly increased from 76.9 to 89.7% and was worse for male gender, age 0–29 days and presence of selected LTS on diagnosis, elevated LDH, and abnormal biologic features. Infants who underwent primary resection ± chemotherapy did significantly better. On multivariate analysis, treatment eras and the association of hepatomegaly to dyspnea were independently associated with worse outcome.ConclusionsOur data confirm that stage 4 s neuroblastoma is curable in nearly 90% of cases. Hepatomegaly associated to dyspnea was the most important independent risk factor. The cure rate could be further increased through timely identification of patients at risk who might benefit from surgical techniques, such as intra-arterial chemoembolization and/or liver transplantation, which must be carried out in institutions with specific expertise.
Highlights
Infants diagnosed with stage 4 s neuroblastoma commonly experience spontaneous disease regression, with few succumbing without response to therapy
We aimed to describe the modifications in presenting features and survival probabilities that occurred over a 34-year period in a cohort of stage 4 s infants enrolled in the Italian Neuroblastoma Registry (RINB) [23], and to define the impact of presenting features on patient outcome
On reviewing the records of the 280 stage 4 s infants enrolled in the RINB, 12 were excluded because of insufficient data (n = 10) or unconfirmed stage (n = 2)
Summary
Infants diagnosed with stage 4 s neuroblastoma commonly experience spontaneous disease regression, with few succumbing without response to therapy. [4] In a minority of patients, stage 4 s disease progresses independently of any therapy, leading to death This outcome has Bernardi et al Italian Journal of Pediatrics (2019) 45:8 been recorded in several studies, which have reported survival rates ranging from 60 to 90% [5,6,7,8,9,10,11,12,13,14].The outcome of infants diagnosed with stage 4 s disease has been seen to be negatively affected by several factors: age < 2 months [9, 11], life-threatening symptoms (LTS) [15, 16] and some biologic features of tumor cells [11, 13, 17,18,19,20,21,22]. Several issues remain poorly defined, i.e., which patients may benefit from chemotherapy, the timing and effect of primary tumor resection, the management of patients with unfavorable biologic features, and the feasibility and benefit of some surgical procedures in the case of massive liver enlargement
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