Purpose: 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 pr
ogram of induction chemotherapy followed by tandem high-dose, myeloablative
treatments (high-dose therapy) with stem-cell rescue (HDT/SCR) in rapid se
quence.
Patients and Methods: Patients underwent induction chemotherapy during whic
h peripheral-blood stem and progenitor cells were collected and local contr
ol 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 wets 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 su
pport each cycle. Engraftment was rapid; median time to neutrophil engraftm
ent was 11 days. Four patients who completed the first HDT course did not c
omplete the second, and there were three deaths due to toxicity. With a med
ian follow-up of 22 months (from diagnosis), 26 of 39 patients remained eve
nt-free. The 3-year event-free survival rate for these patients was 58%.
Conclusion: A tandem HDT/SCR regimen for highrisk neuroblastoma is a feasib
le treatment strategy for children and may improve disease-free survival. J
Clin Oncol 18:2567-2575. (C) 2000 by American Society of Clinical Oncology
.