Treatment combined with bone marrow transplantation for advanced neuroblastoma: An analysis of patients who were pretreated intensively with the protocol of the study group of Japan

Abstract One hundred and ten patients with advanced neuroblastoma were treated with the protocol of the Study Group of Japan between January 1985 and March 1991. Patients received six cyclic courses of regimen A 1 , consisting of cyclophosphamide (1,200 mg/m 2 ), vincristine (1.5 mg/m 2 ), tetrahydropyranyl adriamycin (40 mg/m 2 ), and cisplatin (90 mg/m 2 ). Primary tumors and regional lymph node metastases were removed some time during the first six cycles of regimen A 1 . After six cycles of A 1 , the patients were divided into three groups. Patients in group 1 received alternating treatment with regimen B (cyclophosphamide and ACNU) and intensified A 1 , and those in group 2 were treated with alternating administration of regimen C (cyclophosphamide and DTIC) and intensified A 1 . Patients in group 3 were treated with supralethal therapy and bone marrow transplantation (BMT). Event‐free survival rates at five years were 38.8% in the chemotherapy group (groups 1 and 2) and 50.0% in the transplant group (group 3). Because of the study design that was not in truly randomized fashion and because of the small number of patients in each risk group, it is indicated, though not concluded, that the transplant group had a better prognosis than the chemotherapy group in the cases with stage III disease or with amplified N‐myc oncogene, based on the statistical calculations. Differences in survival rates for patients who underwent BMT when complete remission (CR) was achieved and for those who achieved CR but who did not undergo marrow transplant were statistically insignificant. BMT‐related death occurred in 3 of 31 cases (9.7%) undergoing marrow transplant, and the causes of the death included hemorrhagic pneumonia, myocardial disturbance and hemorrhagic uremia. © 1995 Wi1ey‐Liss Inc.