Tandem high-dose therapy in rapid sequence for children with high-risk neuroblastoma.
OBJECTIVE
Advances in chemotherapy and supportive care have slowly improved survival rates for patients with high-risk neuroblastoma. The focus of many of these chemotherapeutic advances has been dose intensification. In this phase II trial involving children with advanced neuroblastoma, we used a program of induction chemotherapy followed by tandem high-dose, myeloablative treatments (high-dose therapy) with stem-cell rescue (HDT/SCR) in rapid sequence.


METHODS
Patients underwent induction chemotherapy during which peripheral-blood stem and progenitor cells were collected and local control measures undertaken. Patients then received tandem courses of HDT/SCR, 4 to 6 weeks apart. Thirty-nine patients (age 1 to 12 years) were assessable, and 70 cycles of HDT/SCR were completed.


RESULTS
Pheresis was possible in the case of all patients, despite their young ages, with an average of 7.2 x 10(6) CD34(+) cells/kg available to support each cycle. Engraftment was rapid; median time to neutrophil engraftment was 11 days. Four patients who completed the first HDT course did not complete the second, and there were three deaths due to toxicity. With a median follow-up of 22 months (from diagnosis), 26 of 39 patients remained event-free. The 3-year event-free survival rate for these patients was 58%.


CONCLUSIONS
A tandem HDT/SCR regimen for high-risk neuroblastoma is a feasible treatment strategy for children and may improve disease-free survival.
Grupp
S A
SA
Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania, School of Medicine, Philadelphia, PA 19104, USA. grupp@email.chop.edu
Stern
J W
JW
Bunin
N
N
Nancarrow
C
C
Ross
A A
AA
Mogul
M
M
Adams
R
R
Grier
H E
HE
Gorlin
J B
JB
Shamberger
R
R
Marcus
K
K
Neuberg
D
D
Weinstein
H J
HJ
Diller
L
L
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001781:Blood Component Removal; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D008297:Male; D009447:Neuroblastoma / Q000628:therapy*