High-dose chemotherapy with autologous stem-cell rescue in children and adults with newly diagnosed pineoblastomas.
OBJECTIVE
We evaluated the usefulness of a treatment regimen that included high-dose chemotherapy (HDC) with autologous stem-cell rescue (ASCR) in patients with newly diagnosed pineoblastoma (PBL).


METHODS
Twelve patients with PBL were initially treated with surgery and induction chemotherapy. All but two patients underwent radiotherapy. Subsequently, all patients received HDC using cyclophosphamide (CTX) + melphalan (MEL) or busulfan (Bu) + MEL regimens and ASCR.


RESULTS
A total of six children and six adults with median ages of 4.2 (range, 0.3 to 19.8 years) and 23 years (range, 23 to 43.7 years), respectively, were treated according to this strategy. Four patients had metastatic disease confined to the neuraxis. Five of 12 patients (42%) had a complete tumor resection at diagnosis. Ten patients received radiotherapy at median doses of 36.0 and 59.4 Gy to the neuraxis and pineal region, respectively. Eleven patients received HDC with CTX + MEL, and one patient received BU + MEL followed by ASCR. Nine patients are alive with no evidence of disease recurrence at a median of 62 months from diagnosis (range, 28 to 125 months), including three patients with metastatic disease and two infants who did not receive any radiotherapy. Three patients have died of progressive disease at 19, 32, and 37 months from diagnosis, respectively. The actuarial 4-year progression-free and overall survivals are 69% (95% confidence interval [CI], 39% to 99%) and 71% (95% CI, 43% to 99%), respectively.


CONCLUSIONS
The use of HDC in addition to radiotherapy seems to be an effective treatment for patients with newly diagnosed pineoblastoma.
Gururangan
Sridharan
S
Brain Tumor Center at Duke and the Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA. gurur002@mc.duke.edu
McLaughlin
Colleen
C
Quinn
Jennifer
J
Rich
Jeremy
J
Reardon
David
D
Halperin
Edward C
EC
Herndon
James
J
Fuchs
Herbert
H
George
Timothy
T
Provenzale
James
J
Watral
Melody
M
McLendon
Roger E
RE
Friedman
Allan
A
Friedman
Henry S
HS
Kurtzberg
Joanne
J
Vredenbergh
James
J
Martin
Paul L
PL
D000293:Adolescent; D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D002066:Busulfan / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D009657:North Carolina / Q000453:epidemiology; D010870:Pineal Gland; D010871:Pinealoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D033581:Stem Cell Transplantation; D015996:Survival Rate; D014182:Transplantation, Autologous
D003520:Cyclophosphamide; D002066:Busulfan; D008558:Melphalan