Medulloblastoma: intraventricular methotrexate is linked to improved survival

  • Mynarek M & al.
  • J Clin Oncol
  • 20 Jun 2020

  • curated by Deepa Koli
  • Univadis Clinical Summaries
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  • Adding intraventricular methotrexate to systemic chemotherapy improved survival outcomes in patients with desmoplastic medulloblastoma (DMB) and medulloblastoma with extensive nodularity (MBEN).

Why this matters

  • Findings suggest that poor outcomes for patients treated with systemic chemotherapy alone can be improved by the addition of intraventricular methotrexate.

Study design

  • 87 children with nonmetastatic medulloblastoma received systemic chemotherapy and intraventricular methotrexate.
  • After 2006, local radiotherapy was introduced for nonresponsive disease or for patients with classic medulloblastoma (CMB) or large-cell/anaplastic medulloblastoma (LCA).
  • DNA methylation profiles of infantile Sonic Hedgehog-activated medulloblastoma (SHH-INF) were subdivided into iSHH-I and iSHH-II subtypes.
  • Funding: German Childhood Cancer Foundation.

Key results

  • At 5 years, 42 patients with DMB or MBEN showed:
    • PFS: 93%;
    • OS: 100%; and
    • Craniospinal irradiation (CSI)-free survival: 93%.
  • At 5 years, 45 patients with CMB/LCA showed:
    • PFS: 37%;
    • OS: 62%; and
    • CSI-free survival: 39%.
  • Local radiotherapy did not improve survival in patients with CMB/LCA.
  • All DMB/MBEN assessed by DNA methylation profiling belonged to the SHH-INF subgroup.
  • 5-year relapse rate was higher in group 3 patients vs SHH-INF group (36% vs 93%) or group 4 patients (83%; n=6; P<.001>
  • In validation cohort subtypes, iSHH-I and iSHH-II subtypes were reproduced.
  • PFS and OS were similar between the 2 subtypes (P=.25 and .099, respectively).
  • Intelligence quotient was significantly lower in patients after CSI vs radiotherapy.


  • Small study.