Katherine K. Matthay1,2,3,4,5, C. Patrick Reynolds1,2,3,4,5, Robert C. Seeger1,2,3,4,5, Hiroyuki Shimada1,2,3,4,5, E. Stanton Adkins1,2,3,4,5, Daphne A. Haas‐Kogan1,2,3,4,5, Robert B. Gerbing1,2,3,4,5, Wendy B. London1,2,3,4,5, Judith G. Villablanca1,2,3,4,5
1From the University of California at San Francisco School of Medicine, San Francisco, CA; the
2From the University of California at San Francisco School of Medicine, San Francisco, CA; the Childrens Hospital Los Angeles and the University of Southern California Keck School of Medicine; and the Children's Oncology Group Statistics and Data Center, Los Angeles, CA; the South Carolina Cancer Center, Columbia, SC; and the University of Florida and Children's Oncology Group Statistics and Data Center, Gainesville, FL.
3South Carolina Cancer Center, Columbia, SC
4Univer-sity of Florida and Children's Oncology Group Statistics and Data Center, Gainesville, FL.
5University of California School of Medicine, 505 Parnassus Ave, Room M647, San Francisco, CA, 94143-0106;
Tóm tắt
Purpose We assessed the long-term outcome of patients enrolled on CCG-3891, a high-risk neuroblastoma study in which patients were randomly assigned to undergo autologous purged bone marrow transplantation (ABMT) or to receive chemotherapy, and subsequent treatment with 13-cis-retinoic acid (cis-RA). Patients and Methods Patients received the same induction chemotherapy, with random assignment (N = 379) to consolidation with myeloablative chemotherapy, total-body irradiation, and ABMT versus three cycles of intensive chemotherapy. Patients who completed consolidation without disease progression were randomly assigned to receive no further therapy or cis-RA for 6 months. Results The event-free survival (EFS) for patients randomly assigned to ABMT was significantly higher than those randomly assigned to chemotherapy; the 5-year EFS (mean ± SE) was 30% ± 4% versus 19% ± 3%, respectively (P = .04). The 5-year EFS (42% ± 5% v 31% ± 5%) from the time of second random assignment was higher for cis-RA than for no further therapy, though it was not significant. The 5-year overall survival (OS) from the second random assignment of patients who underwent both random assignments and who were assigned to ABMT/cis-RA was 59% ± 8%; for ABMT/no cis-RA, it was 41% ± 7%; for continuing chemotherapy/cis-RA, it was 38% ± 7%; and for chemotherapy/no cis-RA, it was 36% ± 7%. Conclusion Myeloablative therapy and autologous hematopoietic cell rescue result in significantly better 5-year EFS than nonmyeloablative chemotherapy; neither myeloablative therapy with autologous hematopoietic cell rescue nor cis-RA given after consolidation therapy significantly improved OS.
