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← Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors

HEALTH PROFESSIONAL · SOURCE READING

Adjuvant chemotherapy

Source: Childhood Medulloblastoma and Other Central Nervous System Embryonal Tumors Treatment (PDQ®)–Health Professional Version, National Cancer Institute.

Source updated: April 11, 2025 · Captured 2026-09-09.

Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.

Context: Childhood Medulloblastoma / Treatment of Childhood Medulloblastoma / Treatment of children older than 3 years with high-risk medulloblastoma

Evidence (adjuvant chemotherapy):

The drugs that are useful in children with average-risk disease are the same drugs that have been used extensively in children with high-risk disease, including cisplatin, lomustine, cyclophosphamide, etoposide, and vincristine.[139]

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The drugs that are useful in children with average-risk disease are the same drugs that have been used extensively in children with high-risk disease, including cisplatin, lomustine, cyclophosphamide, etoposide, and vincristine.[139]

These therapies have increased 5-year and 10-year EFS and OS rates and have likely decreased the incidence of late relapse.

The drugs that are useful in children with average-risk disease are the same drugs that have been used extensively in children with high-risk disease, including cisplatin, lomustine, cyclophosphamide, etoposide, and vincristine.[139]

However, long-term survivors treated with multimodality therapy are at a high risk of late effects such as hearing loss, cardiac complications, and secondary neoplasms.[133]

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Postradiation high-dose nonmyeloablative chemotherapy supported by peripheral stem cell rescue, but with reduced cumulative doses of vincristine and cisplatin, has also been used and has resulted in 5-year PFS rates of approximately 60%.[58]

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The COG ACNS0332 (NCT00392327) study randomly assigned patients to receive daily carboplatin compared with weekly vincristine during radiation therapy (36 Gy craniospinal plus a posterior fossa boost) followed by six cycles of adjuvant treatment with cisplatin, cyclophosphamide, and vincristine.[135]

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The COG ACNS0332 (NCT00392327) study randomly assigned patients to receive daily carboplatin compared with weekly vincristine during radiation therapy (36 Gy craniospinal plus a posterior fossa boost) followed by six cycles of adjuvant treatment with cisplatin, cyclophosphamide, and vincristine.[135]

Of the 261 evaluable patients, the 5-year EFS rate was 62.9%, and the OS rate was 73.4%.

The COG ACNS0332 (NCT00392327) study randomly assigned patients to receive daily carboplatin compared with weekly vincristine during radiation therapy (36 Gy craniospinal plus a posterior fossa boost) followed by six cycles of adjuvant treatment with cisplatin, cyclophosphamide, and vincristine.[135]

For the entire cohort, there was no difference in EFS rate between patients who were treated with carboplatin (66.4%) and patients who were not treated with carboplatin (59.2%).

The COG ACNS0332 (NCT00392327) study randomly assigned patients to receive daily carboplatin compared with weekly vincristine during radiation therapy (36 Gy craniospinal plus a posterior fossa boost) followed by six cycles of adjuvant treatment with cisplatin, cyclophosphamide, and vincristine.[135]

In a subset analysis based on molecular subgrouping, patients with group 3 medulloblastoma appeared to benefit from the use of daily carboplatin during radiation therapy, with a 5-year EFS rate of 73.2% for patients who received carboplatin, compared with 53.7% for those who did not.

The COG ACNS0332 (NCT00392327) study randomly assigned patients to receive daily carboplatin compared with weekly vincristine during radiation therapy (36 Gy craniospinal plus a posterior fossa boost) followed by six cycles of adjuvant treatment with cisplatin, cyclophosphamide, and vincristine.[135]

A second randomization testing the utility of isotretinoin maintenance therapy was closed at the time of a planned interim analysis for futility.

In a trial of 51 patients with newly diagnosed high-risk disease, patients were treated with postoperative induction chemotherapy (etoposide and carboplatin), followed by two high-dose thiotepa courses with peripheral stem cell rescue and risk-adapted craniospinal radiation therapy.[136]

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In a trial of 51 patients with newly diagnosed high-risk disease, patients were treated with postoperative induction chemotherapy (etoposide and carboplatin), followed by two high-dose thiotepa courses with peripheral stem cell rescue and risk-adapted craniospinal radiation therapy.[136]

The 5-year PFS and OS rates were 76%.

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Preserved source evidence · Not medical advice