Introduction:
Recent strategies aim to reduce cytotoxic treatment for patients with localized Neuroblastoma. This may lead to progressions manageable with limited treatment. On the other hand some patients experience multiple subsequent progressions with fatal outcome. We here aimed to determine risk factors for outcome of relapsed localized neuroblastoma.
Patients and methods:
Data of patient with localized non-amplified Neuroblastoma diagnosed between 1995 and 2004 were analysed. Events were classified as metastatic (to stage 4S or to stage 4) or locoregional, either with favorable course managed with one line of treatment or unfavorable course with multiple episodes of subsequent progressions.
Results:
Of 739 patients, 116 experienced relapse or progression (5-year-EFS 0.83±0.01; 5-year-OS 0.96±0.01). A metastatic pattern fitting the stage 4S definition was seen in 13 patients, progression to stage 4 in 17 patients. Of 86 patients with locoregional events, five patients died from complications or early progression, 53 patients were managed with one line of relapse treatment (surgery only in 22 patients), and 28 patients experienced multiple episodes of progression. Eight patients developed distant metastases in subsequent events, fifteen patients finally died.
Survival was excellent for patients with progression to stage 4S and with manageable locoregional events, but unsatisfactory for patients with multiple locoregional events (5-year-OS: 0.59±0.10) or progression to stage 4 (5-year-OS: 0.65±0.12). Risk factors for both subsequent progressions and for fatal outcome were age at diagnosis (p<0.001), INSS stage (p=0.02 resp. 0.001), histology (p<0.001), and chemotherapeutic treatment prior to relapse (p<0.001). Multivariable analysis revealed age (HR 15.2), histology (HR 3.02), and chemotherapeutic treatment (HR 2.43) as independent factors for subsequent progression and age (HR 8.07) as independent factor for survival.
Conclusion:
In a substantial portion of patients, locoregional progressions or progressions to stage 4S were manageable assuring an excellent prognosis. Established risk factors helped to define patients with unfavorable outcome.