Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue: Results of the Chicago Pilot II study

Morris Kletzel*, Howard M. Katzenstein, Paul R. Haut, Alice L. Yu, Elaine R Morgan, Marleta Reynolds, Grant Geissler, Maryanne H. Marymount, Dachao Liu, John A Kalapurakal, Richard Shore, Diana M.E. Bardo, Jennifer Schmoldt, Alfred W Rademaker, Susan L. Cohn

*Corresponding author for this work

Research output: Contribution to journalArticle

101 Citations (Scopus)

Abstract

Purpose: 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. Patients and 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. Conclusion: 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.

Original languageEnglish (US)
Pages (from-to)2284-2292
Number of pages9
JournalJournal of Clinical Oncology
Volume20
Issue number9
DOIs
StatePublished - May 1 2002

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Neuroblastoma
Stem Cells
Therapeutics
Disease-Free Survival
Poisons
Granulocyte Colony-Stimulating Factor
Treatment Failure
Cyclophosphamide
Peripheral Blood Stem Cells
Survival Rate
Radiation
Transplants
Drug Therapy
Survival

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Cite this

Kletzel, Morris ; Katzenstein, Howard M. ; Haut, Paul R. ; Yu, Alice L. ; Morgan, Elaine R ; Reynolds, Marleta ; Geissler, Grant ; Marymount, Maryanne H. ; Liu, Dachao ; Kalapurakal, John A ; Shore, Richard ; Bardo, Diana M.E. ; Schmoldt, Jennifer ; Rademaker, Alfred W ; Cohn, Susan L. / Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue : Results of the Chicago Pilot II study. In: Journal of Clinical Oncology. 2002 ; Vol. 20, No. 9. pp. 2284-2292.
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title = "Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue: Results of the Chicago Pilot II study",
abstract = "Purpose: 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. Patients and 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. Conclusion: 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.",
author = "Morris Kletzel and Katzenstein, {Howard M.} and Haut, {Paul R.} and Yu, {Alice L.} and Morgan, {Elaine R} and Marleta Reynolds and Grant Geissler and Marymount, {Maryanne H.} and Dachao Liu and Kalapurakal, {John A} and Richard Shore and Bardo, {Diana M.E.} and Jennifer Schmoldt and Rademaker, {Alfred W} and Cohn, {Susan L.}",
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Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue : Results of the Chicago Pilot II study. / Kletzel, Morris; Katzenstein, Howard M.; Haut, Paul R.; Yu, Alice L.; Morgan, Elaine R; Reynolds, Marleta; Geissler, Grant; Marymount, Maryanne H.; Liu, Dachao; Kalapurakal, John A; Shore, Richard; Bardo, Diana M.E.; Schmoldt, Jennifer; Rademaker, Alfred W; Cohn, Susan L.

In: Journal of Clinical Oncology, Vol. 20, No. 9, 01.05.2002, p. 2284-2292.

Research output: Contribution to journalArticle

TY - JOUR

T1 - Treatment of high-risk neuroblastoma with triple-tandem high-dose therapy and stem-cell rescue

T2 - Results of the Chicago Pilot II study

AU - Kletzel, Morris

AU - Katzenstein, Howard M.

AU - Haut, Paul R.

AU - Yu, Alice L.

AU - Morgan, Elaine R

AU - Reynolds, Marleta

AU - Geissler, Grant

AU - Marymount, Maryanne H.

AU - Liu, Dachao

AU - Kalapurakal, John A

AU - Shore, Richard

AU - Bardo, Diana M.E.

AU - Schmoldt, Jennifer

AU - Rademaker, Alfred W

AU - Cohn, Susan L.

PY - 2002/5/1

Y1 - 2002/5/1

N2 - Purpose: 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. Patients and 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. Conclusion: 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.

AB - Purpose: 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. Patients and 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. Conclusion: 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.

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