OBJECTIVETo evaluate chemotherapy-related toxicity and the short-term efficacy of topotecan and cyclophosphamide as maintenance chemotherapy for stage IV neuroblastoma in complete remission.

METHODSThe clinical data of 16 children with stage IV neuroblastoma received 3 cycles of maintenance chemotherapy with topotecan (0.75 mg·m(-2)·day(-1), infused on days 0-4) and cyclophosphamide 250 mg·m(-2)·day(-1), infused on days 0-4). The two-year event-free survival after complete remission was recorded and the chemotherapy-related toxicities were evaluated according to the Common Terminology Criteria for Adverse Events of the National Cancer Institute.

RESULTSThe most common chemotherapy-related toxicity was bone marrow suppression and suppressions of neutrophils, hemoglobin and platelets, which occurred in all the patients mostly of grade III and IV. All the patients experienced episodes of infections, which were controlled effectively with antibiotics. Impairment of gastrointestinal and liver functions in these cases was mostly mild (grade I and II) and recovered after corresponding treatments. None of the patients exhibited damages in the nervous system or the renal or cardiac functions. After complete remission, the two-year event-free survival rate of these patients was 68.75% (11/16).

CONCLUSIONTopotecan plus cyclophosphamide for maintenance chemotherapy can be effective and relative safe for stage IV neuroblastoma in complete remission, thus giving a chance to those patients who choose not to have stem cell transplantation.