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High-dose chemotherapy with stem cell rescue
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.
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Context: Treatment of Recurrent Childhood Medulloblastoma and Other CNS Embryonal Tumors / Treatment Options
For patients who have previously received radiation therapy, higher-dose chemotherapeutic regimens, supported with autologous bone marrow rescue or peripheral stem cell support, have been used with variable results.[10,11,33-36][Level of evidence B4]; [37-39][Level of evidence C1]
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With such regimens, objective response is frequent, occurring in 50% to 75% of patients. However, long-term disease control is obtained in fewer than 30% of patients and is seen primarily in patients in first relapse and those with only localized disease at the time of relapse.[11]; [36][Level of evidence B4]; [37][Level of evidence C1]
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Additionally, results from national trials for relapsed medulloblastoma that specified intent to transplant as part of their treatment plan showed that only approximately 5% of patients initiating retrieval therapy achieved long-term disease-free survival with this strategy.[36,40] Thus, studies that report from the time of transplant overestimate the benefit of transplant-based approaches for the total population of patients who have a relapse.
Long-term disease control for patients with disseminated disease is infrequent.[41][Level of evidence C1]
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Preserved source evidence · Not medical advice