Efficacy of Intrathecal Methotrexate in Children with High-risk
Medulloblastoma over 3 years: A retrospective study from a single center
Abstract
Background To evaluate the therapeutic benefits and side
effects (especially leukoencephalopathy) of intrathecal methotrexate
(MTX) in children aged over three years old with high-risk
medulloblastoma (MB). Methods In the present retrospective
study, patients who received intrathecal MTX during chemotherapy were
classified as MTX group, while those receiving cerebrospinal fluid (CSF)
cytology analysis only were recruited in control group. Results
Among the 46 MB patients, 32 were classified in MTX group, whereas 14 in
control group. For those 32 patients in MTX group, 27 (84.38%) had
metastatic disease, 3 (9.38%) had diffuse anaplasia, and 3 (9.38%) had
residual disease greater than 1.5 cm 2. Molecular
subgroup classification was available in 28 (87.5%) patients. Of those
14 patients in control group, 8 (57.14%) had metastatic disease, 3
(27.27%) had diffuse anaplasia, and 6 (42.86%) had residual disease
greater than 1.5 cm 2. Molecular subgroup
classification was available in 6 (42.86%) patients. The 5-year
progression-free survival (PFS) was 70.99% (95% CI, 58.22%-90.42%)
and 5-year overall survival (OS) was 72.99% (95% CI, 60.41%-93.06%)
for MTX group, while those were 41.67% (95% CI, 17.93%-66.92%) and
50% (95% CI, 27.88%-77.14%) for control group, separately. Among the
32 patients in MTX group, 6 (18.75%) with group 4 disease developed
MTX-related acute leukoencephalopathy and one of them died.
Conclusions Intrathecal MTX improves the 5-year PFS and OS for
children with high-risk MB. However, intrathecal MTX is not recommended
for patients with group 4 MB due to the high risk of acute
leukoencephalopathy.