High-risk neuroblastoma treated with tandem autologous peripheral-blood stem cell-supported transplantation: long-term survival update.
OBJECTIVE
To provide an update on long-term survival of patients with high-risk neuroblastoma treated with tandem cycles of myeloablative therapy and peripheral-blood stem-cell rescue (PBSCR).


METHODS
Ninety-seven patients with high-risk neuroblastoma were treated between 1994 and 2002. Patients underwent induction therapy with five cycles of standard agents, resection of the primary tumor and local radiation, and two consecutive courses of myeloablative therapy (including total-body irradiation) with PBSCR.


RESULTS
Fifty-one patients have experienced relapse or died. Median follow-up time among the 46 patients who remain alive without progression is 5.6 years (range, 15.1 months to 9.9 years). Progression-free survival (PFS) rate at 5 years from diagnosis was 47% (95% CI, 36% to 56%), and PFS rate at 7 years was 45% (95% CI, 34% to 55%). Overall survival rate was 60% (95% CI, 48% to 69%) and 53% (95% CI, 40% to 64%) at 5 and 7 years, respectively. The 5- and 7- year PFS rates from time of first transplantation for 82 patients who completed both transplants were 54% (95% CI, 42% to 64%) and 52% (95% CI, 40% to 63%), respectively. Five patients died from treatment-related toxicity after tandem transplantation. Relapse occurred in 37 (42%) of 89 patients, mainly within 3 years of transplantation and primarily in diffuse osseous sites. No primary CNS relapse or secondary leukemia was seen. One patient developed synovial cell sarcoma 8 years after therapy.


CONCLUSIONS
High-dose therapy with tandem autologous stem-cell rescue is effective for treating high-risk neuroblastoma, with encouraging long-term survival. CNS relapse and secondary malignancies are rare after this therapy.
George
Rani E
RE
Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA 02115, USA. rani_george@dfci.harvard.edu
Li
Shuli
S
Medeiros-Nancarrow
Cheryl
C
Neuberg
Donna
D
Marcus
Karen
K
Shamberger
Robert C
RC
Pulsipher
Michael
M
Grupp
Stephan A
SA
Diller
Lisa
L
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D006801:Humans; D007223:Infant; D016609:Neoplasms, Second Primary; D009447:Neuroblastoma / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D016019:Survival Analysis; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014916:Whole-Body Irradiation