Medulloblastoma in children 0-3 years old: forty years' experience in slovenia

Stefan Kopac1, Berta Jereb

  • 1Department of Neurology, University Children Hospital Medical Center, Zaloska, Slovenia.

Insights

For young children with medulloblastoma (MB), preradiation chemotherapy with high-dose cyclophosphamide, etoposide, and vincristine (HDCy) combined with reduced radiation doses shows promising long-term survival outcomes.

Area of Science:

  • Pediatric Oncology
  • Radiation Oncology
  • Medical History

Background:

  • Medulloblastoma (MB) is a common malignant brain tumor in young children.
  • Treatment strategies for MB have evolved significantly over time, impacting survival rates.
  • Early treatment protocols had high mortality rates, necessitating the exploration of alternative approaches.

Purpose of the Study:

  • To evaluate the long-term outcomes of medulloblastoma treatment in children aged 0-3 years.
  • To compare survival rates and developmental outcomes between different treatment eras and modalities.
  • To assess the efficacy and safety of preradiation chemotherapy with high-dose cyclophosphamide, etoposide, and vincristine (HDCy) and reduced radiation doses.

Main Methods:

  • Retrospective analysis of 21 children (0-3 years) diagnosed with MB between 1960 and 1996.
  • Categorization of patients based on treatment received: no treatment, postoperative radiotherapy (RT), iodine-125 interstitial therapy (IT), and preradiation chemotherapy (Cht) with HDCy.
  • Comparison of survival rates, including early mortality and long-term survival, and assessment of developmental outcomes in survivors.

Main Results:

  • Of 14 children treated before 1984, 12 died within one year; one survived 42 years with a secondary hypopharyngeal cancer, and one had a normal outcome.
  • Among 7 children treated from 1986-1996 with preradiation Cht (5 with HDCy), 3 with disseminated disease died.
  • Four children achieved long-term survival, with two treated with reduced RT doses exhibiting normal development.

Conclusions:

  • Preradiation chemotherapy with HDCy followed by reduced doses of RT appears to be a viable and effective treatment strategy for young children with medulloblastoma.
  • This approach may improve long-term survival and reduce treatment-related toxicities compared to older protocols.
  • Further research into optimized chemotherapy and radiotherapy regimens is warranted for pediatric medulloblastoma.

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