Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols

Girish Dhall, Howard Grodman, Lingyun Ji, Stephen Sands, Sharon Gardner, Ira J. Dunkel, Geoffrey B. McCowage, Bianca Diez, Jeffrey C. Allen, Anjali Gopalan, Albert S. Cornelius, Amanda Termuhlen, Minnie Abromowitch, Richard Sposto, Jonathan L. Finlay

Research output: Contribution to journalArticle

120 Citations (Scopus)

Abstract

Purpose. To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods. Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, "Head Start" I and "Head Start" II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. Results. The 5-year event-free (EFS) and overall survival (OS) rates (±SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 ± 11% and 70 ± 10%, 64 ± 13% and 79 ± 11%, and 29 ± 17% and 57 ± 19%, respectively. The 5-year EFS and OS (± SE) for patients with desmoplastic and classical medulloblastoma were 67 ± 16% and 78 ± 14%, and 42 ± 14 and 67 ± 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. Conclusion. This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.

Original languageEnglish (US)
Pages (from-to)1169-1175
Number of pages7
JournalPediatric Blood and Cancer
Volume50
Issue number6
DOIs
StatePublished - Jun 1 2008

Fingerprint

Medulloblastoma
Drug Therapy
Etoposide
Hematopoietic Stem Cells
Survivors
Survival Rate
Craniospinal Irradiation
Quality of Life
Thiotepa
Induction Chemotherapy
Survival
Poisons
Carboplatin
Residual Neoplasm
Vincristine
Cyclophosphamide
Cisplatin
Recurrence
Mortality

Keywords

  • Infants
  • Medulloblastoma
  • Myeloablative chemotherapy and late effects

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

Cite this

Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols. / Dhall, Girish; Grodman, Howard; Ji, Lingyun; Sands, Stephen; Gardner, Sharon; Dunkel, Ira J.; McCowage, Geoffrey B.; Diez, Bianca; Allen, Jeffrey C.; Gopalan, Anjali; Cornelius, Albert S.; Termuhlen, Amanda; Abromowitch, Minnie; Sposto, Richard; Finlay, Jonathan L.

In: Pediatric Blood and Cancer, Vol. 50, No. 6, 01.06.2008, p. 1169-1175.

Research output: Contribution to journalArticle

Dhall, G, Grodman, H, Ji, L, Sands, S, Gardner, S, Dunkel, IJ, McCowage, GB, Diez, B, Allen, JC, Gopalan, A, Cornelius, AS, Termuhlen, A, Abromowitch, M, Sposto, R & Finlay, JL 2008, 'Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols', Pediatric Blood and Cancer, vol. 50, no. 6, pp. 1169-1175. https://doi.org/10.1002/pbc.21525
Dhall, Girish ; Grodman, Howard ; Ji, Lingyun ; Sands, Stephen ; Gardner, Sharon ; Dunkel, Ira J. ; McCowage, Geoffrey B. ; Diez, Bianca ; Allen, Jeffrey C. ; Gopalan, Anjali ; Cornelius, Albert S. ; Termuhlen, Amanda ; Abromowitch, Minnie ; Sposto, Richard ; Finlay, Jonathan L. / Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols. In: Pediatric Blood and Cancer. 2008 ; Vol. 50, No. 6. pp. 1169-1175.
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abstract = "Purpose. To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods. Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, {"}Head Start{"} I and {"}Head Start{"} II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. Results. The 5-year event-free (EFS) and overall survival (OS) rates (±SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 ± 11{\%} and 70 ± 10{\%}, 64 ± 13{\%} and 79 ± 11{\%}, and 29 ± 17{\%} and 57 ± 19{\%}, respectively. The 5-year EFS and OS (± SE) for patients with desmoplastic and classical medulloblastoma were 67 ± 16{\%} and 78 ± 14{\%}, and 42 ± 14 and 67 ± 14{\%}, respectively. There were four treatment related deaths. The majority of survivors (71{\%}) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. Conclusion. This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52{\%} of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.",
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T1 - Outcome of children less than three years old at diagnosis with non-metastatic medulloblastoma treated with chemotherapy on the "Head Start" I and II protocols

AU - Dhall, Girish

AU - Grodman, Howard

AU - Ji, Lingyun

AU - Sands, Stephen

AU - Gardner, Sharon

AU - Dunkel, Ira J.

AU - McCowage, Geoffrey B.

AU - Diez, Bianca

AU - Allen, Jeffrey C.

AU - Gopalan, Anjali

AU - Cornelius, Albert S.

AU - Termuhlen, Amanda

AU - Abromowitch, Minnie

AU - Sposto, Richard

AU - Finlay, Jonathan L.

PY - 2008/6/1

Y1 - 2008/6/1

N2 - Purpose. To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods. Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, "Head Start" I and "Head Start" II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. Results. The 5-year event-free (EFS) and overall survival (OS) rates (±SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 ± 11% and 70 ± 10%, 64 ± 13% and 79 ± 11%, and 29 ± 17% and 57 ± 19%, respectively. The 5-year EFS and OS (± SE) for patients with desmoplastic and classical medulloblastoma were 67 ± 16% and 78 ± 14%, and 42 ± 14 and 67 ± 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. Conclusion. This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.

AB - Purpose. To determine the survival of infants and young children with non-metastatic medulloblastoma using intensive myeloablative chemotherapy and autologous hematopoietic progenitor cell rescue (AuHCR). Methods. Twenty-one children less than 3 years old at diagnosis with non-metastatic medulloblastoma were enrolled on two identical serial studies, "Head Start" I and "Head Start" II. After surgery, patients received five cycles of induction chemotherapy consisting of vincristine, cisplatin, cyclophosphamide and etoposide. Following induction, all patients underwent myeloablative chemotherapy using carboplatin, thiotepa and etoposide with AuHCR. Irradiation was used only at relapse. Results. The 5-year event-free (EFS) and overall survival (OS) rates (±SE) for all patients, patients with gross total resection, and patients with residual tumor were 52 ± 11% and 70 ± 10%, 64 ± 13% and 79 ± 11%, and 29 ± 17% and 57 ± 19%, respectively. The 5-year EFS and OS (± SE) for patients with desmoplastic and classical medulloblastoma were 67 ± 16% and 78 ± 14%, and 42 ± 14 and 67 ± 14%, respectively. There were four treatment related deaths. The majority of survivors (71%) avoided irradiation completely. Mean intellectual functioning and quality of life (QoL) for children surviving without irradiation was within average range for a majority of survivors tested. Conclusion. This strategy of brief intensive chemotherapy for young children with non-metastatic medulloblastoma eliminated the need for craniospinal irradiation 52% of the patients, and may preserve QoL and intellectual functioning. The excellent survival rates are somewhat dampened by high toxic mortality.

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