Neuroblastoma in Infants: Long Term Survival from INES Protocols - A
SIOPEN Study
Abstract
Purpose: Neuroblastoma is the most frequent extracranial solid
tumors in infants, with a characteristic presentation and a possibility
of spontaneous regression even in disseminated disease. Despite an
overall good prognosis, relapse can occur in some patients, leading to a
poorer outcome in a subgroup of patients. A long-term analysis of these
patients assessing survival at 10 years from diagnosis is relevant in
estimating the risks of late relapse, progression or long-term toxicity
due to very young exposure to treatment. Methods: Estimation of
the 10-year event-free and overall survivals in 750 infants under 12
months of age with Neuroblastoma, enrolled in the prospective INES
protocols between 1999 and 2004. Follow-up data from INES patients were
updated, and survival analyses were performed in order to determine
prognostic factors such as: age, stage, genomic profile or MYCN
amplification. Results: Overall, 10-year OS was 91.1% ± 1.0%,
and 10-year EFS was 82.4% ±1.4%, with significantly better outcomes in
infants under 6 months compared to those aged 6 to 12 months, even
considering the MYCN-amplified tumors only. MYCN amplification was the
strongest prognostic factor and was correlated with substantially lower
survival rates among patients with metastatic disease.
Discussion: Survival in infancy remains excellent and stable
even at long term, as a 10-year follow-up did not change the number of
events. However, survival in MYCN-amplified tumors remained poor, this
population needs to be treated more aggressively. Patients with
metastatic tumors require accurate risk stratification, especially for
younger infants under 6 months. For each treatment group, there was no
significant difference in long-term outcomes compared to previous
publications from INES. No lethal toxicity affecting long-term survival
occurred.