TriangleHEALTH PROFESSIONAL · SOURCE READING
Nondesmoplastic, non-MBEN, and non-SHH signaling–driven medulloblastoma
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.
Compared with children with desmoplastic medulloblastoma or MBEN treated with current intensive chemotherapy regimens, children with other histological and/or molecular subtypes fare less well. One study suggested that patients with molecularly identified group 4 tumors did well with chemotherapy alone.[111]
Source links and citations
Evidence (chemotherapy):
In children with nondesmoplastic, non-MBEN, and/or non-SHH–signaling tumors, the EFS rates are less than 40% despite the use of intensive chemotherapy supplemented with methotrexate (intravenous and intraventricular) or the use of high-dose chemotherapy regimens supported with stem cell rescue.[68,103,111,113,115]
In children with nondesmoplastic, non-MBEN, and/or non-SHH–signaling tumors, the EFS rates are less than 40% despite the use of intensive chemotherapy supplemented with methotrexate (intravenous and intraventricular) or the use of high-dose chemotherapy regimens supported with stem cell rescue.[68,103,111,113,115]
Outcome is particularly poor when these patients have disseminated disease.
In some studies, radiation therapy to the primary tumor site and/or craniospinal axis has been added after chemotherapy, which makes the assessment of the efficacy of chemotherapy more difficult.[111,113,115]
There is no consensus on how much radiation therapy (dose and extent) should be given and at what age radiation therapy should be instituted in young patients with localized or disseminated disease.
In the expanded HIT 2000 study, the addition of focal radiation therapy to the primary tumor site in patients with localized disease after chemotherapy did not improve EFS or OS.[111]
Source links and citations
In the St. Jude Children's Research Hospital (SJCRH) SJYC07 (NCT00602667) study, focal radiation therapy also did not improve EFS in infants with medulloblastoma denoted as intermediate risk (5-year EFS rate, 25% ± 12%).[65]
Source links and citations
The COG P9934 (NCT00006461) study, which also employed focal radiation therapy, had a similar EFS (4-year EFS rate, 23% ± 12%) for patients with nondesmoplastic medulloblastoma.[116]
Source links and citations
In the SJCRH SJYC07 study, 29 of the 54 infants with medulloblastoma whose disease progressed received CSI (median dose, 36 Gy). Of the 29 patients, 18 (62%) survived, compared with 6 of 25 patients (24%) who did not receive CSI.[65]
Source links and citations
Studies cited in this source section
Source citation is not a determination that a study applies to you.
Publication references
Read the original reference and check its publication notices.
Preserved source evidence · Not medical advice