Recurrent medulloblastomas are associated with survival rates <10%. Adequate multimodal therapy is being discussed as having a major impact on survival. In this study, 93 patients with recurrent medulloblastoma treated in the German P-HIT-REZ 2005 Study were analyzed for survival (PFS, OS) dependent on patient, disease, and treatment characteristics. The median age at the first recurrence was 10.1 years (IQR: 6.9-16.1). Median PFS and OS, at first recurrence, were 7.9 months (CI: 5.7-10.0) and 18.5 months (CI: 13.6-23.5), respectively. Early relapses/progressions (<18 months, = 30/93) found mainly in molecular subgroup 3 were associated with markedly worse median PFS (HR: 2.34) and OS (HR: 3.26) in regression analyses. A significant survival advantage was found for the use of volume-reducing surgery as well as radiotherapy. Intravenous chemotherapy with carboplatin and etoposide (ivCHT, = 28/93) showed improved PFS and OS data and the best objective response rate (ORR) was 66.7% compared to oral temozolomide (oCHT, = 47/93) which was 34.8%. Intraventricular ( = 43) as well as high-dose chemotherapy ( = 17) at first relapse was not related to a significant survival benefit. Although the results are limited due to a non-randomized study design, they may serve as a basis for future treatment decisions in order to improve the patients' survival.
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http://dx.doi.org/10.3390/cancers14030471 | DOI Listing |
Cell Rep Med
January 2025
Beijing Neurosurgical Institute, Beijing 100070, China; China National Clinical Research Center for Neurological Diseases, Beijing Tiantan Hospital, Capital Medical University, Beijing 100070, China; Department of Neurosurgery, Beijing Tiantan Hospital, Capital Medical University, Beijing 100070, China. Electronic address:
Medulloblastoma (MB), a heterogeneous pediatric brain tumor, poses challenges in the treatment of tumor recurrence and dissemination. To characterize cellular diversity and genetic features, we comprehensively analyzed single-cell/nucleus RNA sequencing (sc/snRNA-seq), single-nucleus assay for transposase-accessible chromatin sequencing (snATAC-seq), and spatial transcriptomics profiles and identified distinct cellular populations in SHH (sonic hedgehog) and Group_3 subgroups, with varying proportions in local recurrence or dissemination. Local recurrence showed higher cycling tumor cell enrichment, whereas disseminated lesions had a relatively notable presence of differentiated subsets.
View Article and Find Full Text PDFCancers (Basel)
December 2024
Department of Neurosurgery, University of Oklahoma Health Sciences Center, 1000 N. Lincoln Ave. #4000, Oklahoma City, OK 73104, USA.
Objective: The aim of this study was to systematically review the existing individual patient data in the literature on adult cerebellopontine angle (CPA) medulloblastoma (MB) and characterize the patient presentation, management strategies used, and oncological outcomes of this rare entity to guide future clinical practice.
Methods: Following PRISMA guidelines, a systematic review was conducted by searching PubMed, EMBASE, Web of Science, and Cochrane databases from inception to 19 June 2024. Studies regarding adult patients with histologically confirmed MB radiographically confirmed to be located in the CPA were included.
J Neurosurg Case Lessons
January 2025
Department of Neurosurgery, Children's Hospital of Nanjing Medical University, Nanjing, Jiangsu, China.
Background: The diagnosis of intracranial extraosseous Ewing's sarcoma (EES) poses challenges due to the absence of specific clinical and imaging features prior to surgery. It is crucial to differentiate the tumor from other small round cell malignancies postoperatively.
Observations: A 7-year-old patient was admitted to the authors' hospital due to the in situ recurrence of a posterior fossa tumor more than 1 month after the initial surgery for headache.
Nat Genet
January 2025
Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, Ontario, Canada.
Transcription factors are frequent cancer driver genes, exhibiting noted specificity based on the precise cell of origin. We demonstrate that ZIC1 exhibits loss-of-function (LOF) somatic events in group 4 (G4) medulloblastoma through recurrent point mutations, subchromosomal deletions and mono-allelic epigenetic repression (60% of G4 medulloblastoma). In contrast, highly similar SHH medulloblastoma exhibits distinct and diametrically opposed gain-of-function mutations and copy number gains (20% of SHH medulloblastoma).
View Article and Find Full Text PDFRes Sq
December 2024
Department of Pediatrics, University of Colorado Anschutz Medical Campus, Aurora, CO, USA.
Medulloblastoma (MB) is the most prevalent malignant brain tumor in children, exhibiting clinical and genomic heterogeneity. Of the four major subgroups, Group 3 tumors (MYC-MB), display high levels of MYC and metastasis rates. Despite treatment with surgery, radiation, and chemotherapy, patients with Group 3 MB are more likely to develop aggressive recurrent tumors with poor survival.
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