- Adding intraventricular methotrexate to systemic chemotherapy improved survival outcomes in patients with desmoplastic medulloblastoma (DMB) and medulloblastoma with extensive nodularity (MBEN).
Why this matters
- Findings suggest that poor outcomes for patients treated with systemic chemotherapy alone can be improved by the addition of intraventricular methotrexate.
- 87 children with nonmetastatic medulloblastoma received systemic chemotherapy and intraventricular methotrexate.
- After 2006, local radiotherapy was introduced for nonresponsive disease or for patients with classic medulloblastoma (CMB) or large-cell/anaplastic medulloblastoma (LCA).
- DNA methylation profiles of infantile Sonic Hedgehog-activated medulloblastoma (SHH-INF) were subdivided into iSHH-I and iSHH-II subtypes.
- Funding: German Childhood Cancer Foundation.
- At 5 years, 42 patients with DMB or MBEN showed:
- PFS: 93%;
- OS: 100%; and
- Craniospinal irradiation (CSI)-free survival: 93%.
- At 5 years, 45 patients with CMB/LCA showed:
- PFS: 37%;
- OS: 62%; and
- CSI-free survival: 39%.
- Local radiotherapy did not improve survival in patients with CMB/LCA.
- All DMB/MBEN assessed by DNA methylation profiling belonged to the SHH-INF subgroup.
- 5-year relapse rate was higher in group 3 patients vs SHH-INF group (36% vs 93%) or group 4 patients (83%; n=6; P<.001>
- In validation cohort subtypes, iSHH-I and iSHH-II subtypes were reproduced.
- PFS and OS were similar between the 2 subtypes (P=.25 and .099, respectively).
- Intelligence quotient was significantly lower in patients after CSI vs radiotherapy.
- Small study.