Medulloblastoma: intraventricular methotrexate is linked to improved survival

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

  • curated by Deepa Koli
  • Univadis Clinical Summaries
Access to the full content of this site is available only to registered healthcare professionals. Access to the full content of this site is available only to registered healthcare professionals.

Takeaway

  • 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.

Limitations

  • Small study.