With current treatments, patients with low and intermediate risk disease have an excellent prognosis with cure rates above 90%. There are no FDA approved drugs for neuroblastoma and therefore this regimen and the individual drugs are considered off-label but it is medically necessary based on phase II studies. The most common combination of drugs to treat neuroblastoma consists of carboplatin (or cisplatin), cyclophosphamide, doxorubicin, and etoposide. Chemotherapy is given for four to eight cycles (12 to 24 weeks) and 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. Radiation therapy is reserved for patients with symptomatic life-threatening or organ-threatening tumor that does not respond rapidly enough to chemotherapy and/or surgery.
Phase III studies are difficult to perform in this disease because of the wide use of bone marrow transplatation. Although it is being delivered on a clincal trial, pediatric cancer is exceptional in that 95% of children with cancer are treated on a trail and within a trial framework, in the Children Oncology Group, and mere includion in a trial should not be conisdered to render it experimental. However, this combination is novel in that it doeas not include cyclophopshamide or ifosfamide and I had not been able to find this specific combinaton or regimen reported previously. For this reason I consider it investigational, especially on trial.
Baker DL, Schmidt M, Cohn S, et al.: A phase III trial of biologically-based therapy reduction for intermediate risk neuroblastoma. [Abstract] J Clin Oncol 25 (Suppl 18): A-9504, 2007.
Publisher Springer Berlin Heidelberg
ISBN 978-3-540-40841-3 (Print) 978-3-540-26616-7 (Online) , p. 199