Neuroblastoma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment Option Overview
The treatments described in this summary are based on the Children's Oncology Group (COG) group assignment, which is described in the Stage Information section of this summary. Treatment information is presented in this format because most children with neuroblastoma in North America are treated according to the COG schema. The prior COG risk-based neuroblastoma studies established the standard of care. They assigned each patient to a low-, intermediate-, or high-risk group and the basis of the assignment is described in Table 1.
In patients without metastatic disease, the standard of care is to perform an initial surgery to establish the diagnosis, to resect as much of the primary tumor as is safely possible, to accurately stage disease through sampling of regional lymph nodes that are not adherent to the tumor, and to obtain adequate tissue for biological studies. Accurate determination of biological characteristics, such as INPC system, usually requires an open biopsy. The accuracy of diagnosis and staging is increased by performing a metaiodobenzylguanidine (MIBG) scan. Urinary excretion of the catecholamine metabolites vanillylmandelic acid (VMA) and homovanillic acid (HVA) per mg of excreted creatinine should be measured prior to therapy. If elevated, these markers can be used to determine the persistence of disease.
Major pharmaceutical companies continually research and develop new medications and treatments for brain cancer, which must be shown to be safe and effective before doctors can prescribe them to patients. Through clinical trials, researchers test the effects of new medications on a group of volunteers with brain cancer. Following a strict protocol and using carefully controlled conditions, researchers evaluate the investigational drugs under development and measure the ability of the new drug to...
There is controversy about the need for immediate diagnostic biopsy in infants aged 3 months and younger with suspected neuroblastoma tumors that are likely to spontaneously regress. Biopsy is not required for infants entered into a COG study of expectant observation of adrenal masses in neonates. In a German clinical trial, 25 infants aged 3 months and younger with presumed neuroblastoma were observed without biopsy for periods of 1 to 18 months prior to biopsy or resection. There were no apparent ill effects of the delay.
There is also controversy about the need for attempted resection, whether at the time of diagnosis or later, in asymptomatic infants aged 12 months or younger with apparent stage 2B and 3 MYCN-nonamplified disease. In a German clinical trial, some of these patients were observed after biopsy or partial resection without chemotherapy or radiation, and many did not progress locally and never received additional resection.
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.