PMID- 10519894
OWN - NLM
STAT- MEDLINE
DCOM- 19991014
LR  - 20240103
IS  - 0028-4793 (Print)
IS  - 0028-4793 (Linking)
VI  - 341
IP  - 16
DP  - 1999 Oct 14
TI  - Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, 
      autologous bone marrow transplantation, and 13-cis-retinoic acid. Children's 
      Cancer Group.
PG  - 1165-73
AB  - BACKGROUND: Children with high-risk neuroblastoma have a poor outcome. In this 
      study, we assessed whether myeloablative therapy in conjunction with 
      transplantation of autologous bone marrow improved event-free survival as 
      compared with chemotherapy alone, and whether subsequent treatment with 
      13-cis-retinoic acid (isotretinoin) further improves event-free survival. 
      METHODS: All patients were treated with the same initial regimen of chemotherapy, 
      and those without disease progression were then randomly assigned to receive 
      continued treatment with myeloablative chemotherapy, total-body irradiation, and 
      transplantation of autologous bone marrow purged of neuroblastoma cells or to 
      receive three cycles of intensive chemotherapy alone. All patients who completed 
      cytotoxic therapy without disease progression were then randomly assigned to 
      receive no further therapy or treatment with 13-cis-retinoic acid for six months. 
      RESULTS: The mean (+/-SE) event-free survival rate three years after the first 
      randomization was significantly better among the 189 patients who were assigned 
      to undergo transplantation than among the 190 patients assigned to receive 
      continuation chemotherapy (34+/-4 percent vs. 22+/-4 percent, P=0.034). The 
      event-free survival rate three years after the second randomization was 
      significantly better among the 130 patients who were assigned to receive 
      13-cis-retinoic acid than among the 128 patients assigned to receive no further 
      therapy (46+/-6 percent vs. 29+/-5 percent, P=0.027). CONCLUSIONS: Treatment with 
      myeloablative therapy and autologous bone marrow transplantation improved 
      event-free survival among children with high-risk neuroblastoma. In addition, 
      treatment with 13-cis-retinoic acid was beneficial for patients without 
      progressive disease when it was administered after chemotherapy or 
      transplantation.
FAU - Matthay, K K
AU  - Matthay KK
AD  - Department of Pediatrics, University of California School of Medicine, San 
      Francisco, USA.
FAU - Villablanca, J G
AU  - Villablanca JG
FAU - Seeger, R C
AU  - Seeger RC
FAU - Stram, D O
AU  - Stram DO
FAU - Harris, R E
AU  - Harris RE
FAU - Ramsay, N K
AU  - Ramsay NK
FAU - Swift, P
AU  - Swift P
FAU - Shimada, H
AU  - Shimada H
FAU - Black, C T
AU  - Black CT
FAU - Brodeur, G M
AU  - Brodeur GM
FAU - Gerbing, R B
AU  - Gerbing RB
FAU - Reynolds, C P
AU  - Reynolds CP
LA  - eng
GR  - CA02649/CA/NCI NIH HHS/United States
GR  - CA13539/CA/NCI NIH HHS/United States
GR  - CA22794/CA/NCI NIH HHS/United States
GR  - etc.
PT  - Clinical Trial
PT  - Comparative Study
PT  - Journal Article
PT  - Multicenter Study
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
PT  - Research Support, U.S. Gov't, P.H.S.
PL  - United States
TA  - N Engl J Med
JT  - The New England journal of medicine
JID - 0255562
RN  - EH28UP18IF (Isotretinoin)
SB  - IM
MH  - Adolescent
MH  - Antineoplastic Combined Chemotherapy Protocols/adverse effects/*therapeutic use
MH  - *Bone Marrow Transplantation/adverse effects
MH  - Child
MH  - Child, Preschool
MH  - Combined Modality Therapy
MH  - Disease-Free Survival
MH  - Humans
MH  - Infant
MH  - Isotretinoin/adverse effects/*therapeutic use
MH  - Life Tables
MH  - Neuroblastoma/*drug therapy/mortality/radiotherapy/*therapy
MH  - Prospective Studies
MH  - Risk
MH  - Transplantation Conditioning
MH  - Whole-Body Irradiation
EDAT- 1999/10/16 00:00
MHDA- 1999/10/16 00:01
CRDT- 1999/10/16 00:00
PHST- 1999/10/16 00:00 [pubmed]
PHST- 1999/10/16 00:01 [medline]
PHST- 1999/10/16 00:00 [entrez]
AID - 10.1056/NEJM199910143411601 [doi]
PST - ppublish
SO  - N Engl J Med. 1999 Oct 14;341(16):1165-73. doi: 10.1056/NEJM199910143411601.