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Chemotherapy for Ependymomas in Children

This page was last updated on April 8th, 2024


The role of chemotherapy in treating ependymomas is not clear. Chemotherapy can induce a partial or complete response in some patients, and it may be helpful in facilitating second-look surgery. It has been successful in delaying radiotherapy in some infant protocols. Its use is presently being evaluated in residual and anaplastic disease following resection and radiation in the open COG protocol for patients who are 12 months of age and older.

Chemotherapy After Resection and Radiation

This regimen, proposed by Needle in 1997, consists of alternating courses of regimen A: carboplatin and vincristine, and regimen B: ifosfamide and etoposide. Each regimen was delivered for a total of four cycles (26) as long as counts recovered adequately.

  • Regimen A (28 day cycle): Weekly for first 3 weeks: carboplatin, 560 mg/ m² IV, and vincristine, 1.5 mg/ m² IV
  • Regimen B (28 day cycle): Day 1, ifosfamide, 1.8 g/ m² IV; Days 1-5, etoposide, 100 mg/ m² IV

Chemotherapy After Resection but Before Radiation

This regimen has been proposed by Garvin et al. to manage residual disease after surgery. It is delivered in four cycles, each lasting 21 days (17).

  • Days 1, 8, and 15 for first three cycles: Vincristine, 1.5 mg/m² IV (maximum, 2 mg)
  • Days 1, 2, 3: Etoposide, 100 mg/m² IV
  • Day 1: Cisplatin, 100 mg/m² IV
  • Days 2, 3: Cyclophosphamide, 1000 mg/m² IV

Chemotherapy to Delay Radiation in Infants

The aim of the Baby POG protocol (54) was 28-day cycles of chemotherapy for children younger than 36 months to delay radiation until child reached 36 months. Alternating AABAAB 28-day cycles as listed below:

  • Cycle A: Vincristine, 0.065 mg/kg IV (maximum, 1.5 g), on days 1 and 8; cyclophosphamide, 65 mg/kg IV, on day 1
  • Cycle B: Cisplatin, 4 mg/kg IV, on day 1; etoposide, 6.5 mg/kg IV, on days 3 and 4


Seven cycles, each of 56 days duration. Dose is by weight for children up to 10 kg and by m2 for children greater than 10 kg (19).

  • Day 1: Vincristine, 0.05 mg/kg IV; carboplatin, 20 mg/kg IV infusion over 4 hours
  • Day 15: Vincristine, 0.05 mg/kg IV; methotrexate, 250 mg/kg IV; leucovorin, 15 mg/kg
  • Day 29: Vincristine, 0.05 mg/kg IV; cyclophosphamide, 50 mg/kg IV; mesna, 60 mg/kg IV
  • Day 43: Cisplatin, 1.3 mg/kg, as continuous IV infusion for 48 hours

Head Start 2

Chemotherapy as used for medulloblastoma; methotrexate is added for metastatic disease (44). This regimen can be considered to assist in two-stage resection of large ependymomas that are difficult to resect completely.

Adjuvant Therapy

  • Radiation: Involved field in the absence of dissemination is the main adjuvant therapy. Children as young as 12 months of age are being considered for conformal or proton beam radiation in the setting of a clinical study.


  • Similar to those in medulloblastomas: The side effects of radiation are reduced, due to the reduced field of radiation to the tumor bed only for the majority of young children receiving irradiation.


Chemotherapy After Resection and Radiation

  • 74% 5-year PFS rate (26)

Chemotherapy After Resection but Before Radiation

  • 57% 5-year EFS rate (17)
  • Possible benefit if >90% resection: The report suggested a benefit from chemotherapy prior to radiation, but only in those patients with >90% resection. 14% of tumors progressed prior to radiation.

Chemotherapy to Delay Radiation in Infants

  • 5-year PFS rate: 55% if >24 months of age; 12.7% if < 24 months of age
  • 5-year overall survival rate: 63% if >24 months of age; 26% if <24 months of age


  • 5-year PFS rate: 49% if gross total resection vs. 26% if incomplete resection
  • 5-year overall survival rate: 68% if gross total resection vs. 52 % if incomplete resection

Head Start 2

  • 5-year EFS rate: 12% (44)
  • 5-year overall survival rate: 38% (44)
  • Methotrexate may increase survival: Most patients in this study had non-metastatic disease and did not receive methotrexate. Their cure rate was low (38% 5 yr overall survival rate). However, 4/5 (80%) of those with metastatic disease received IV methotrexate and were cured without radiation. The favorable outcome of those with metastatic disease in the Head Start protocol and the relatively favorable outcome in the UKCCSG/SIOP study suggest the probable importance of IV methotrexate as part of the chemotherapy regimen for ependymoma if it is planned to delay or avoid radiation in young children.