Treatment

The stepwise approach to management is to stage the disease and treat it with surgery, radiation, and chemotherapy.​​​ All patients require surgery, with the age of the patient and staging determining appropriate further treatment. (1)

Typical treatment of medulloblastoma is maximal surgical excision with external beam irradiation to the posterior fossa and craniospinal axis. Pre-operative ventriculo-peritoneal shunts may be employed but provide a potential route for tumour seeding, especially to bone.

Pre-symptomatic craniospinal irradiation may be practised to reduce the incidence of tumour recurrence distant to the primary site, but it is associated with neurocognitive dysfunction, growth retardation and myelosuppression, and endocrine disorders. It is excluded from children less than 18 months of age since the immature infant is very intolerant of radiation. (2)

Chemotherapy regimens for both average and high-risk groups include cisplatin, cyclophosphamide, lomustine, and vincristine administered during and after radiation therapy. (1)

For recurrent or progressive medulloblastoma, treatment options include repeated operation followed by adjuvant temozolomide/irinotecan with bevacizumab, temozolomide/topotecan (TOTEM), metronomic antiangiogenic therapy (MEMMAT regimen), or carboplatin/etoposide. (3)

Prognosis

Five-year progression-free survival for average-risk patients is approximately 71% to 83% but only 50% to 66% for high-risk patients. (4)

Poor prognostic factors include age <3 years, the presence of metastases, and at least 1.5 cm² of residual tumour after surgery. (5) It is unclear whether time to diagnosis is a prognostic factor, although one study suggested no significant association between the pre-diagnostic symptom interval and the risk of metastatic disease and survival. (6)

Recurrent medulloblastoma in patients who have already had radiotherapy has a very poor outcome with a salvage rate of <10%, irrespective of the treatment modality used. (7)

Reference:

1. Franceschi E, Hofer S, Brandes AA, et al. EANO-EURACAN clinical practice guideline for diagnosis, treatment, and follow-up of post-pubertal and adult patients with medulloblastoma. Lancet Oncol. 2019 Dec;20(12):e715-28.
2. Padovani L, Horan G, Ajithkumar T. Radiotherapy advances in paediatric medulloblastoma treatment. Clin Oncol (R Coll Radiol). 2019 Mar;31(3):171-81.
3. Gajjar A et al. Pediatric Central Nervous System Cancers, Version 2.2025, NCCN Clinical Practice Guidelines In Oncology. J Natl Compr Canc Netw. 2025 Mar;23(3):113-130
4. Michalski JM, Janss AJ, Vezina LG, et al. Children's oncology group phase III trial of reduced-dose and reduced-volume radiotherapy with chemotherapy for newly diagnosed average-risk medulloblastoma. J Clin Oncol. 2021 Aug 20;39(24):2685-97.
5. Rutkowski S, von Hoff K, Emser A, et al. Survival and prognostic factors of early childhood medulloblastoma: an international meta-analysis. J Clin Oncol. 2010 Nov 20;28(33):4961-8.
6. Gerber NU, von Hoff K, von Bueren AO, et al. A long duration of the prediagnostic symptomatic interval is not associated with an unfavourable prognosis in childhood medulloblastoma. Eur J Cancer. 2012 Sep;48(13):2028-36.
7. O'Halloran K, Phadnis S, Friedman GK, et al. Effective re-induction regimen for children with recurrent medulloblastoma. Neurooncol Adv. 2024 May 10;6(1):vdae070.