Long-term survivors of advanced neuroblastoma with MYCN amplification: A report of 19 patients surviving disease-free for more than 66 months

Purpose: According to initial reports, stage 4 neuroblastoma patients with amplification of the MYCN proto-oncogene developed progressive disease with in 8 months. The prognosis for such patients, however, should now be reeval uated in light of recent results achieved with up-to-date combination chemo therapy Patients and Methods: Patients with stage 3, 4, and 4S neuroblastoma and mo re than 10 copies of MYCN received induction chemotherapy, which from Janua ry 1985 to February 1991 consisted of regimen A(1) (cyclophosphamide 1,200 mg/m(2) on day 1, vincristine 1.5 mg/m(2) on day 1, pirarubicin 40 mg/m(2) on day 3, and cisplatin 90 mg/m(2) on day 5) and from March 1991 to Septemb er 1993 consisted of regimen A(3) (cyclophosphamide 1,200 mg/m(2) on days 1 and 2, pirarubicin 40 mg/m(2) on day 3, etoposide 100 mg/m(2) on days 1 th rough 5, and continuous infusion cisplatin 25 mg/m(2) on days 1 through 5). Most of these patients underwent radical surgery to remove the original tu mor and local metastases, irradiation, and supralethal preconditioning regi mens, followed by blood stem-cell transplantation (SCT). Data on the patien ts were collected in December 1998, and the factors contributing to disease -free survival were analyzed. Results: During the study period, 66 patients with more than 10 copies of M YCN were treated. Five of nine patients with stage 3 disease, 13 of 55 with stage 4, and one of two with stage 4S survived for at least 66 months. If is interesting that ail but one patient who survived for more than 66 month s underwent SCT, in contrast with only five of 45 patients who died. Conclusion: Not all patients with advanced neuroblastoma who have more than 10 copies of MYCN will die. The requisites for survival in such patients s eem ta be intensive induction chemotherapy, effective surgery irradiation, and the use Of SCT. (C) 1999 by American Society of Clinical Oncology.