Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Recurrent Neuroblastoma
Age, International Neuroblastoma Staging System (INSS) stage, MYCN status, and time from diagnosis to first relapse are significant prognostic factors for postrelapse survival. The Children's Oncology Group (COG) experience with recurrence in intermediate-risk neuroblastoma is that the majority of recurrences can be salvaged, as demonstrated by a 3-year event free survival (EFS) of 88% and an overall survival (OS) of 96%. When neuroblastoma recurs in a child originally diagnosed with high-risk disease and is widespread, the prognosis is usually poor despite additional intensive therapy.[1,3,4,5]
In selected patients originally diagnosed with low- or intermediate-risk disease, recurrence may be treated successfully with limited intervention. The combination of cyclophosphamide plus topotecan has been active in patients with recurrent or refractory disease who have not received topotecan previously. Iodine I 131 metaiodobenzylguanidine (131 I-MIBG) therapy is also active in patients with recurrent or refractory neuroblastoma. Clinical trials are appropriate and should be considered. Information about ongoing clinical trials is available from the NCI Web site.
There is no defined staging system for childhood central nervous system atypical teratoid/rhabdoid tumor (AT/RT). Patients are classified as having newly diagnosed or recurrent disease with or without neuraxis dissemination.
Central nervous system (CNS) involvement, though rare at initial presentation, may occur in 5% to 10% of patients with recurrent neuroblastoma. Because upfront treatment for newly diagnosed patients does not adequately treat the CNS, the CNS has emerged as a sanctuary site leading to relapse.[8,9] CNS relapses have been almost always fatal with a median time to death of 6 months. Current treatment approaches generally include eradicating bulky and microscopic residual disease in the CNS as well as minimal residual systemic disease that may herald further relapses. Neurosurgical interventions serve to decrease edema, control hemorrhage, and remove bulky tumor prior to starting radiation therapy. Compartmental radioimmunotherapy using intrathecal radioiodinated monoclonal antibodies has been tested in patients with recurrent metastatic CNS neuroblastoma following surgery, craniospinal radiation therapy, and chemotherapy.
In North America, the COG investigated a risk-based neuroblastoma treatment plan that assigned all patients to a low-, intermediate-, or high-risk group based on age, INSS stage, and tumor biology (i.e., MYCN gene amplification, International Neuroblastoma Pathology Classification [INPC] system, and DNA ploidy). Treatment of recurrent disease was determined by risk group at the time of diagnosis (refer to Table 1), extent of disease at recurrence, patient age at recurrence, and the tumor biology. If tumor was unavailable for biological studies at recurrence, the biology of the tumor at time of diagnosis was used to help determine treatment.
Recurrent Neuroblastoma in Patients Initially Classified as Low Risk
(Risk categories are defined in Table 1 in the Stage Information section of this summary.)
Local regional recurrent cancer is resected if possible:
Those with favorable biology and regional recurrence more than 3 months after completion of planned treatment are observed if resection of the recurrence is total or near total (≥90% resection). Those with favorable biology and a less than near-total resection are treated with 12 weeks of chemotherapy.
Infants younger than 1 year at the time of local/regional recurrence whose tumors have any unfavorable biologic properties are observed if resection is total or near total. If the resection is less than near total, these same infants are treated with 24 weeks of chemotherapy.