Journal of Neuro-Oncology

, Volume 103, Issue 3, pp 561–566 | Cite as

Liposomal cytarabine for central nervous system embryonal tumors in children and young adults

  • Sonia Partap
  • Patricia A. Murphy
  • Hannes Vogel
  • Patrick D. Barnes
  • Michael S. B. Edwards
  • Paul G. Fisher
Clinical Study – Patient Study


To assess the tolerability and efficacy of liposomal cytarabine (LC), an encapsulated, sustained-release, intrathecal (IT) formulation of cytosine arabinoside, in de novo and relapsed central nervous system (CNS) embryonal tumors in children and young adults. We studied retrospectively all patients less than age 30 at our institution treated consecutively with LC for medulloblastoma (MB), primitive neuroectodermal tumor (PNET), and atypical teratoid rhabdoid tumor (ATRT). Seventeen patients received LC (2 mg/kg up to 50 mg, every 2 weeks to monthly) at diagnosis of high-risk CNS embryonal tumor (2 PNET, 3 ATRT) or relapse of MB (12 MB; 9 had leptomeningeal metastases). Sixteen patients received concurrent systemic chemotherapy. A total of 108 doses were administered (IT 82, intraventricular 26) with a mean of six (range 1–16) treatments per patient. Only three administrations were associated with adverse effects of arachnoiditis or headache. None developed malignant cerebrospinal fluid (CSF) cytology while receiving LC. All the six evaluable patients with malignant CSF cytology and treated with at least two doses cleared their CSF (mean 3 doses, range 1–5). Median overall survival in relapse patients was 9.1 months. Five patients (4 de novo and 1 relapsed) remain alive in complete remission for a median 26.8 months from first LC. Liposomal cytarabine is an easily administered, well-tolerated, and active drug in patients with high-risk embryonal neoplasms. One-third of our cohort remains in remission from otherwise fatal diagnoses. Our findings warrant a phase II trial of LC in newly diagnosed or recurrent CNS embryonal tumors.


Brain tumor Liposomal cytarabine Embryonal tumor Childhood Medulloblastoma Central nervous system 


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Copyright information

© Springer Science+Business Media, LLC. 2010

Authors and Affiliations

  • Sonia Partap
    • 1
  • Patricia A. Murphy
    • 1
  • Hannes Vogel
    • 2
  • Patrick D. Barnes
    • 3
  • Michael S. B. Edwards
    • 4
  • Paul G. Fisher
    • 1
    • 4
    • 5
    • 6
  1. 1.Department of NeurologyStanford University Medical CenterStanfordUSA
  2. 2.Department of PathologyStanford UniversityStanfordUSA
  3. 3.Department of RadiologyStanford UniversityStanfordUSA
  4. 4.Department of NeurosurgeryStanford UniversityStanfordUSA
  5. 5.Department of PediatricsStanford UniversityStanfordUSA
  6. 6.Department of Human BiologyStanford UniversityStanfordUSA

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