Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue: results of the Chicago Pilot II Study.
OBJECTIVE
To investigate whether intensive induction therapy followed by triple-tandem cycles of high-dose therapy with peripheral-blood stem-cell rescue and local irradiation will improve event-free survival for patients with high-risk neuroblastoma.


METHODS
From August 1995 to January 2000, 25 consecutive newly diagnosed high-risk neuroblastoma patients and one child with recurrent MYCN-amplified disease were enrolled onto the Chicago Pilot II Protocol. After induction therapy and surgery, peripheral-blood stem cells were mobilized with three cycles of high-dose cyclophosphamide and granulocyte colony-stimulating factor. Patients then underwent triple-tandem cycles of high-dose therapy with peripheral-blood stem-cell rescue followed by radiation to the primary site.


RESULTS
Twenty-two of the 26 patients successfully completed induction therapy and were eligible for the triple-tandem consolidation high-dose therapy. Sufficient numbers of peripheral-blood stem cells were collected in all but one patient. Seventeen patients were able to complete all three cycles of high-dose therapy and peripheral-blood stem-cell rescue, two patients completed two cycles, and three patients completed one cycle. There was one toxic death, and one patient died from complications of treatment for graft failure. With a median follow-up of 38 months, the 3-year event-free survival and survival rates are 57% +/- 11% and 79% +/- 10%, respectively.


CONCLUSIONS
The results of this pilot study demonstrate that it is feasible to intensify consolidation with triple-tandem high-dose chemotherapy and peripheral-blood stem-cell rescue and local irradiation, and suggest that this treatment strategy may lead to improved survival for patients with high-risk neuroblastoma.
Kletzel
Morris
M
Department of Pediatrics, Northwestern University, Feinberg School of Medicine, Chicago, IL, USA. mkletzel@northwestern.edu
Katzenstein
Howard M
HM
Haut
Paul R
PR
Yu
Alice L
AL
Morgan
Elaine
E
Reynolds
Marleta
M
Geissler
Grant
G
Marymount
Maryanne H
MH
Liu
Dachao
D
Kalapurakal
John A
JA
Shore
Richard M
RM
Bardo
Diana M E
DM
Schmoldt
Jennifer
J
Rademaker
Alfred W
AW
Cohn
Susan L
SL
D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D009447:Neuroblastoma / Q000628:therapy*; D010865:Pilot Projects; D016019:Survival Analysis; D014182:Transplantation, Autologous; D016896:Treatment Outcome