Treatment of Intermediate-Risk Neuroblastoma
A small single institution study suggested that all MYCN-nonamplified INSS stage 3 tumors may be treated with surgical resection followed by observation without chemotherapy.[Level of evidence: 3iiDi]
Treatment Options Under Clinical Evaluation
The following are examples of national and/or institutional clinical trials that are currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.
- COG-ANBL0531: This COG trial is a response- and biology-based trial for intermediate-risk neuroblastoma. The COG is studying a reduction of chemotherapy duration for most intermediate risk group patients, based in part on a reduction of the degree of tumor response required to stop chemotherapy.
A new risk/treatment group classification system has been developed for this study and is described in Table 2 in the Treatment of Low-Risk Neuroblastoma section of this summary. Patients previously classified as intermediate risk are now in risk Groups 2, 3, or 4. Some children aged 8 to 12 months previously classified as high risk are now in intermediate-risk Group 4.
- The risk/treatment Groups 2 and 3 are upstaged by one risk/treatment group if the tumor cannot be studied for loss of heterozygosity (LOH) or if LOH of 1p or unbalanced LOH of 11q is found.
- The treatment for intermediate-risk patients involves surgery and the chemotherapy treatment regimen described below. Radiation therapy is reserved for patients with symptomatic life-threatening or organ-threatening tumor that does not respond rapidly to chemotherapy and/or surgery.
- Treatment Group 2: Two cycles of chemotherapy-stop chemotherapy when a partial response is achieved.
- Treatment Group 3: Four cycles of chemotherapy-stop chemotherapy when a partial response is achieved.
- Treatment Group 4: Eight cycles of chemotherapy-stop chemotherapy when a good partial response is achieved.
For all Groups noted above, the chemotherapy consists of moderate doses of carboplatin, cyclophosphamide, doxorubicin, and etoposide. The cumulative dose of each agent is kept low to minimize permanent injury from the chemotherapy regimen. If needed, additional cycles of this chemotherapy regimen, up to eight total, are given until partial response is obtained. If the required response has not been obtained with eight cycles of the afore-mentioned chemotherapy regimen, an additional one to six cycles of topotecan and cyclophosphamide chemotherapy is given.
(Refer to the International Neuroblastoma Response Criteria in the Treatment Option Overview section of this summary for more information.)
Current Clinical Trials
Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with neuroblastoma. The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.
General information about clinical trials is also available from the NCI Web site.
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