Neuro-Oncology: Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with

Thursday, July 19, 2012

Pilot study of systemic and intrathecal mafosfamide followed by conformal radiation for infants with

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.

Content Type Journal Article
Category Clinical Study - Patient Study
Pages 1-7
DOI 10.1007/s11060-012-0929-x
Authors
- Susan M. Blaney, Texas Children's Cancer Center, Baylor College of Medicine, 6621 Fannin; CC 1410.00, Houston, TX 77030, USA
- Mehmet Kocak, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
- Amar Gajjar, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
- Murali Chintagumpala, Texas Children's Cancer Center, Baylor College of Medicine, 6621 Fannin; CC 1410.00, Houston, TX 77030, USA
- Thomas Merchant, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
- Mark Kieran, Dana-Farber Cancer Institute and Children's Hospital Boston, Boston, MA 02115, USA
- Ian F. Pollack, Children's Hospital of Pittsburgh, Pittsburgh, PA 15213, USA
- Sri Gururangan, Duke University Medical Center, Durham, NC 27710, USA
- Russ Geyer, Children's Hospital and Medical Center, Seattle, WA 98105, USA
- Peter Phillips, Children's Hospital of Philadelphia, Philadelphia, PA 191044, USA
- Roger E. McLendon, Duke University Medical Center, Durham, NC 27710, USA
- Roger Packer, Children's National Medical Center, Washington, D.C., 20010 USA
- Stewart Goldman, Children's Memorial Hospital, Chicago, IL 60614, USA
- Anu Banerjee, University of California, San Francisco, CA 94143, USA
- Richard Heideman, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
- James M. Boyett, St. Jude Children's Research Hospital, Memphis, TN 38105, USA
- Larry Kun, St. Jude Children's Research Hospital, Memphis, TN 38105, USA