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Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Low-Risk Neuroblastoma

Low-risk neuroblastoma represents nearly one-half of all newly diagnosed patients. The success of prior Children's Oncology Group (COG) clinical trials has contributed to the continued reduction in therapy for select patients with neuroblastoma.

The COG low-risk group assignment criteria are described in Table 6.

Table 6. Children's Oncology Group (COG) Neuroblastoma Low-Risk Group Assignment Schema Used for COG Studiesa

INSS StageAgeMYCNStatusINPC ClassificationDNA Ploidyb
INPC = International Neuroblastoma Pathologic Classification; INSS = International Neuroblastoma Staging System.
a The COG-P9641 (low risk) and COG-A3961 (intermediate risk) trials established the current standard of care for neuroblastoma patients in terms of risk group assignment and treatment strategies.
b DNA Ploidy: DNA Index (DI) > 1 is favorable, = 1 is unfavorable; hypodiploid tumors (with DI < 1) will be treated as a tumor with a DI > 1 (DI < 1 [hypodiploid] to be considered favorable ploidy).
c INSS stage 2A/2B symptomatic patients with spinal cord compression, neurologic deficits, or other symptoms are treated with immediate chemotherapy for four cycles.
d INSS stage 4S infants with favorable biology and clinical symptoms are treated with immediate chemotherapy until asymptomatic (2-4 cycles). Clinical symptoms include the following: respiratory distress with or without hepatomegaly or cord compression and neurologic deficit or inferior vena cava compression and renal ischemia; or genitourinary obstruction; or gastrointestinal obstruction and vomiting; or coagulopathy with significant clinical hemorrhage unresponsive to replacement therapy.
10-21 yAnyAnyAny
2A/2Bc<365 dAnyAnyAny
≥365 d-21 yNonamplifiedAny-
≥365 d-21 yAmplifiedFavorable-
4Sd<365 dNonamplifiedFavorable>1

(Refer to the Treatment of Stage 4S Neuroblastoma section of this summary for more information about the treatment of stage 4S neuroblastoma.)

Treatment Options for Low-Risk Neuroblastoma

For patients with localized disease that appears to be resectable (either based on the absence of image-defined risk factors [L1] or on the surgeon's expertise), the tumor should be resected by an experienced surgeon. If the biology is confirmed to be favorable, residual disease is not considered a risk factor for relapse. Several studies have shown that patients with favorable biology and residual disease have excellent outcomes with event-free survival (EFS) in excess of 90% and overall survival (OS) of 99% to 100%.[1,2]

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