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Treatment Option Overview

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    Low-Risk Neuroblastoma

    Treatment for patients categorized as low risk (refer to Table 1 in the Stage Information section of this summary) may be surgery alone, but surgery may be combined with chemotherapy in some cases. Chemotherapy is reserved for patients who are symptomatic, such as from spinal cord compression or, in stage 4S, respiratory compromise secondary to hepatic infiltration. The chemotherapy consists of carboplatin, cyclophosphamide, doxorubicin, and etoposide. The cumulative dose of each agent is kept low to minimize permanent injury from the chemotherapy regimen (COG-P9641).

    The COG study COG-P9641 demonstrated excellent survival in patients with asymptomatic, low-risk, stage 2A or 2B disease with favorable histology.[3]

    Observation without surgery for localized, suspected adrenal neuroblastoma in infants

    Studies suggest that selected presumed neuroblastomas detected in infants by screening or incidental ultrasound may safely be observed without obtaining a definitive histologic diagnosis and without surgical intervention, thus avoiding potential complications of surgery in the newborn.[4,5,6] The experience with tumors detected by mass urinary catecholamine metabolite screening in Japan appears to be applicable to tumors detected by prenatal or perinatal ultrasound in the United States.[4] The COG is investigating systematic observation without surgery for infants with presumed small Evans stage I adrenal neuroblastoma detected by prenatal or perinatal ultrasound.

    Intermediate-Risk Neuroblastoma

    Patients categorized as intermediate risk (refer to Table 1 in the Stage Information section of this summary) have been successfully treated with surgery and 12 to 24 weeks of the same chemotherapy regimen described above (COG-A3961). As a rule, patients whose tumors have unfavorable biology receive twice as many cycles of chemotherapy as those with favorable biology.

    Whether initial chemotherapy is indicated for all intermediate-risk infants with localized neuroblastoma is controversial. A German prospective clinical trial enrolled 340 infants aged 1 year or younger whose tumors were stage 1, 2, or 3, histologically verified, and lacked MYCN amplification. Forty-four of 93 infants with unresected tumors experienced spontaneous regression (17 were complete regressions) and 39 infants experienced progression. The 3-year overall survival (OS) rate was 99%, and the metastases-free survival rate was 94% for infants with unresected tumors and was not different from infants treated with surgery or chemotherapy (median follow-up, 58 months). The investigators suggested that a wait-and-see strategy is appropriate for infants with localized neuroblastoma because regressions have been observed after the first year of life.[2]

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