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Original Article
Volume 341:1165-1173 October 14, 1999 Number 16
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Treatment of High-Risk Neuroblastoma with Intensive Chemotherapy, Radiotherapy, Autologous Bone Marrow Transplantation, and 13-cis-Retinoic Acid
Katherine K. Matthay, M.D., Judith G. Villablanca, M.D., Robert C. Seeger, M.D., Daniel O. Stram, Ph.D., Richard E. Harris, M.D., Norma K. Ramsay, M.D., Patrick Swift, M.D., Hiroyuki Shimada, M.D., C. Thomas Black, M.D., Garrett M. Brodeur, M.D., Robert B. Gerbing, M.A., C. Patrick Reynolds, M.D., Ph.D., for The Children's Cancer Group

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ABSTRACT

Background Children with high-risk neuroblastoma have a poor outcome. In this study, we assessed whether myeloablative therapy in conjunction with transplantation of autologous bone marrow improved event-free survival as compared with chemotherapy alone, and whether subsequent treatment with 13-cis-retinoic acid (isotretinoin) further improves event-free survival.

Methods All patients were treated with the same initial regimen of chemotherapy, and those without disease progression were then randomly assigned to receive continued treatment with myeloablative chemotherapy, total-body irradiation, and transplantation of autologous bone marrow purged of neuroblastoma cells or to receive three cycles of intensive chemotherapy alone. All patients who completed cytotoxic therapy without disease progression were then randomly assigned to receive no further therapy or treatment with 13-cis-retinoic acid for six months.

Results The mean (±SE) event-free survival rate three years after the first randomization was significantly better among the 189 patients who were assigned to undergo transplantation than among the 190 patients assigned to receive continuation chemotherapy (34±4 percent vs. 22±4 percent, P=0.034). The event-free survival rate three years after the second randomization was significantly better among the 130 patients who were assigned to receive 13-cis-retinoic acid than among the 128 patients assigned to receive no further therapy (46±6 percent vs. 29±5 percent, P=0.027).

Conclusions Treatment with myeloablative therapy and autologous bone marrow transplantation improved event-free survival among children with high-risk neuroblastoma. In addition, treatment with 13-cis-retinoic acid was beneficial for patients without progressive disease when it was administered after chemotherapy or transplantation.


Source Information

From the Departments of Pediatrics (K.K.M.) and Radiation Oncology (P.S.), University of California School of Medicine, San Francisco; the Departments of Pediatrics (J.G.V., R.C.S., C.P.R.) and Pathology (H.S., C.P.R.), University of Southern California School of Medicine and Children's Hospital, Los Angeles; the Department of Preventive Medicine, University of Southern California School of Medicine, Los Angeles (D.O.S.); the Department of Pediatrics, Children's Hospital Medical Center, Cincinnati (R.E.H.); the Department of Pediatrics, University of Minnesota School of Medicine, Minneapolis (N.K.R.); the Department of Surgery, M.D. Anderson Hospital, Houston (C.T.B.); the Children's Cancer Group, Arcadia, Calif. (D.O.S., R.B.G.); and the Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia (G.M.B.).

Address reprint requests to Dr. Matthay at the Children's Cancer Group, P.O. Box 60012, Arcadia, CA 91066-6012.

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