Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors

A pediatric brain tumor consortium study (PBTC-001)

Susan M. Blaney, Mehmet Kocak, Amar Gajjar, Murali Chintagumpala, Thomas Merchant, Mark Kieran, Ian F. Pollack, Gururangan Sri, Russ Geyer, Peter Phillips, Roger E. McLendon, Roger Packer, Stewart Goldman, Anu Banerjee, Richard Heideman, James M. Boyett, Larry Kun

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstruction (n = 71) received IT mafosfamide (14 mg) with chemotherapy. Localized (M0) patients with SD or better subsequently received RT followed by 20 additional weeks of chemotherapy. Second look surgery was encouraged prior to RT if there was an incomplete surgical resection at diagnosis. 71 evaluable patients with normal CSF flow received IT Mafosfamide with systemic chemotherapy; patients with M ? disease were removed from protocol therapy at 20 weeks and those with PD at the time of progression. One and 5-year progression free survival (PFS) and overall survival (OS) for the cohort of 71 evaluable patients were 52 ± 6.5 % and 33 ± 13 %, and 67 ± 6.2 % and 51 ± 11 %, respectively. The 1-year Progression Free Survival (PFS) for M0 patients with medulloblastoma (MB, n = 20), supratentorial primitive neuroectodermal tumor (PNET, n = 9), and atypical teratoid rhabdoid tumor (ATRT, n = 12) was 80 ± 7 %, 67 ± 15 % and 27 ± 13 % and 5-year PFS was 65 ± 19 %, 37 ± 29 %, and 0 ± 0 %, respectively. The addition of IT mafosfamide to systemic chemotherapy in infants with embryonal CNS tumors was feasible. The PFS for M0 patients appears comparable to or better than most prior historical comparisons and was excellent for those receiving conformal radiotherapy.

Original languageEnglish (US)
Pages (from-to)565-571
Number of pages7
JournalJournal of Neuro-Oncology
Volume109
Issue number3
DOIs
StatePublished - Sep 1 2012

Fingerprint

Central Nervous System Neoplasms
Brain Neoplasms
Radiation
Pediatrics
Drug Therapy
Disease-Free Survival
Primitive Neuroectodermal Tumors
Radiotherapy
Second-Look Surgery
Conformal Radiotherapy
Neoplasms
Medulloblastoma
mafosfamide
Ambulatory Surgical Procedures
Survival

All Science Journal Classification (ASJC) codes

  • Oncology
  • Neurology
  • Clinical Neurology
  • Cancer Research

Cite this

Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors : A pediatric brain tumor consortium study (PBTC-001). / Blaney, Susan M.; Kocak, Mehmet; Gajjar, Amar; Chintagumpala, Murali; Merchant, Thomas; Kieran, Mark; Pollack, Ian F.; Sri, Gururangan; Geyer, Russ; Phillips, Peter; McLendon, Roger E.; Packer, Roger; Goldman, Stewart; Banerjee, Anu; Heideman, Richard; Boyett, James M.; Kun, Larry.

In: Journal of Neuro-Oncology, Vol. 109, No. 3, 01.09.2012, p. 565-571.

Research output: Contribution to journalArticle

Blaney, SM, Kocak, M, Gajjar, A, Chintagumpala, M, Merchant, T, Kieran, M, Pollack, IF, Sri, G, Geyer, R, Phillips, P, McLendon, RE, Packer, R, Goldman, S, Banerjee, A, Heideman, R, Boyett, JM & Kun, L 2012, 'Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: A pediatric brain tumor consortium study (PBTC-001)', Journal of Neuro-Oncology, vol. 109, no. 3, pp. 565-571. https://doi.org/10.1007/s11060-012-0929-x
Blaney, Susan M. ; Kocak, Mehmet ; Gajjar, Amar ; Chintagumpala, Murali ; Merchant, Thomas ; Kieran, Mark ; Pollack, Ian F. ; Sri, Gururangan ; Geyer, Russ ; Phillips, Peter ; McLendon, Roger E. ; Packer, Roger ; Goldman, Stewart ; Banerjee, Anu ; Heideman, Richard ; Boyett, James M. ; Kun, Larry. / Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors : A pediatric brain tumor consortium study (PBTC-001). In: Journal of Neuro-Oncology. 2012 ; Vol. 109, No. 3. pp. 565-571.
@article{16d6c918443c479c82e8e0e2ae89b047,
title = "Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors: A pediatric brain tumor consortium study (PBTC-001)",
abstract = "A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstruction (n = 71) received IT mafosfamide (14 mg) with chemotherapy. Localized (M0) patients with SD or better subsequently received RT followed by 20 additional weeks of chemotherapy. Second look surgery was encouraged prior to RT if there was an incomplete surgical resection at diagnosis. 71 evaluable patients with normal CSF flow received IT Mafosfamide with systemic chemotherapy; patients with M ? disease were removed from protocol therapy at 20 weeks and those with PD at the time of progression. One and 5-year progression free survival (PFS) and overall survival (OS) for the cohort of 71 evaluable patients were 52 ± 6.5 {\%} and 33 ± 13 {\%}, and 67 ± 6.2 {\%} and 51 ± 11 {\%}, respectively. The 1-year Progression Free Survival (PFS) for M0 patients with medulloblastoma (MB, n = 20), supratentorial primitive neuroectodermal tumor (PNET, n = 9), and atypical teratoid rhabdoid tumor (ATRT, n = 12) was 80 ± 7 {\%}, 67 ± 15 {\%} and 27 ± 13 {\%} and 5-year PFS was 65 ± 19 {\%}, 37 ± 29 {\%}, and 0 ± 0 {\%}, respectively. The addition of IT mafosfamide to systemic chemotherapy in infants with embryonal CNS tumors was feasible. The PFS for M0 patients appears comparable to or better than most prior historical comparisons and was excellent for those receiving conformal radiotherapy.",
author = "Blaney, {Susan M.} and Mehmet Kocak and Amar Gajjar and Murali Chintagumpala and Thomas Merchant and Mark Kieran and Pollack, {Ian F.} and Gururangan Sri and Russ Geyer and Peter Phillips and McLendon, {Roger E.} and Roger Packer and Stewart Goldman and Anu Banerjee and Richard Heideman and Boyett, {James M.} and Larry Kun",
year = "2012",
month = "9",
day = "1",
doi = "10.1007/s11060-012-0929-x",
language = "English (US)",
volume = "109",
pages = "565--571",
journal = "Journal of Neuro-Oncology",
issn = "0167-594X",
publisher = "Kluwer Academic Publishers",
number = "3",

}

TY - JOUR

T1 - Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with intracranial central nervous system tumors

T2 - A pediatric brain tumor consortium study (PBTC-001)

AU - Blaney, Susan M.

AU - Kocak, Mehmet

AU - Gajjar, Amar

AU - Chintagumpala, Murali

AU - Merchant, Thomas

AU - Kieran, Mark

AU - Pollack, Ian F.

AU - Sri, Gururangan

AU - Geyer, Russ

AU - Phillips, Peter

AU - McLendon, Roger E.

AU - Packer, Roger

AU - Goldman, Stewart

AU - Banerjee, Anu

AU - Heideman, Richard

AU - Boyett, James M.

AU - Kun, Larry

PY - 2012/9/1

Y1 - 2012/9/1

N2 - A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstruction (n = 71) received IT mafosfamide (14 mg) with chemotherapy. Localized (M0) patients with SD or better subsequently received RT followed by 20 additional weeks of chemotherapy. Second look surgery was encouraged prior to RT if there was an incomplete surgical resection at diagnosis. 71 evaluable patients with normal CSF flow received IT Mafosfamide with systemic chemotherapy; patients with M ? disease were removed from protocol therapy at 20 weeks and those with PD at the time of progression. One and 5-year progression free survival (PFS) and overall survival (OS) for the cohort of 71 evaluable patients were 52 ± 6.5 % and 33 ± 13 %, and 67 ± 6.2 % and 51 ± 11 %, respectively. The 1-year Progression Free Survival (PFS) for M0 patients with medulloblastoma (MB, n = 20), supratentorial primitive neuroectodermal tumor (PNET, n = 9), and atypical teratoid rhabdoid tumor (ATRT, n = 12) was 80 ± 7 %, 67 ± 15 % and 27 ± 13 % and 5-year PFS was 65 ± 19 %, 37 ± 29 %, and 0 ± 0 %, respectively. The addition of IT mafosfamide to systemic chemotherapy in infants with embryonal CNS tumors was feasible. The PFS for M0 patients appears comparable to or better than most prior historical comparisons and was excellent for those receiving conformal radiotherapy.

AB - A pilot study to investigate the feasibility of the addition of intrathecal (IT) mafosfamide to a regimen of concomitant multi-agent systemic chemotherapy followed by conformal radiation therapy (RT) for children <3 years with newly diagnosed embryonal CNS tumors was performed. Ninety-three newly diagnosed infants and children (<3 years) with embryonal CNS tumors were enrolled. Twenty weeks of systemic multi-agent chemotherapy commenced within 35 days of surgery. Patients without CSF flow obstruction (n = 71) received IT mafosfamide (14 mg) with chemotherapy. Localized (M0) patients with SD or better subsequently received RT followed by 20 additional weeks of chemotherapy. Second look surgery was encouraged prior to RT if there was an incomplete surgical resection at diagnosis. 71 evaluable patients with normal CSF flow received IT Mafosfamide with systemic chemotherapy; patients with M ? disease were removed from protocol therapy at 20 weeks and those with PD at the time of progression. One and 5-year progression free survival (PFS) and overall survival (OS) for the cohort of 71 evaluable patients were 52 ± 6.5 % and 33 ± 13 %, and 67 ± 6.2 % and 51 ± 11 %, respectively. The 1-year Progression Free Survival (PFS) for M0 patients with medulloblastoma (MB, n = 20), supratentorial primitive neuroectodermal tumor (PNET, n = 9), and atypical teratoid rhabdoid tumor (ATRT, n = 12) was 80 ± 7 %, 67 ± 15 % and 27 ± 13 % and 5-year PFS was 65 ± 19 %, 37 ± 29 %, and 0 ± 0 %, respectively. The addition of IT mafosfamide to systemic chemotherapy in infants with embryonal CNS tumors was feasible. The PFS for M0 patients appears comparable to or better than most prior historical comparisons and was excellent for those receiving conformal radiotherapy.

UR - http://www.scopus.com/inward/record.url?scp=84866034368&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84866034368&partnerID=8YFLogxK

U2 - 10.1007/s11060-012-0929-x

DO - 10.1007/s11060-012-0929-x

M3 - Article

VL - 109

SP - 565

EP - 571

JO - Journal of Neuro-Oncology

JF - Journal of Neuro-Oncology

SN - 0167-594X

IS - 3

ER -