Abstract: Objective To evaluate chemotherapy-related toxicity and the short-term efficacy of topotecan and cyclophosphamide
as maintenance chemotherapy for stage IV neuroblastoma in complete remission. Methods The 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. Results The 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). Conclusion
Topotecan 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.