Trial reportNeuro-oncology2021
Prognostic relevance of clinical and molecular risk factors in children with high-risk medulloblastoma treated in the phase II trial PNET HR+5.
Trial report in Neuro-oncology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
27 citing papers in PubMed, 1 synthesis or guideline pooled it, 54 citations in OpenAlex.
- Questions and answers in the management of children with medulloblastoma over the time. How did we get here? A systematic review.Frontiers in oncology · 2023Pooled it
- Age-Dependent Toxicity Patterns in Medulloblastoma: A Comparative Analysis of Treatment-Related Adverse Events Between Pediatric and Adult Populations.Journal of clinical medicine · 2026Article
- Bayesian reappraisal of ACNS0332 and ACNS0334 strengthens subgroup treatment effects in high-risk pediatric Group 3 medulloblastoma.Neuro-oncology · 2025Article
- Review
- Long-term outcome of the Milano-hyperfractionated accelerated radiotherapy strategy for high-risk medulloblastoma, including the impact of molecular subtype.Neuro-oncology · 2025Article
- Outcomes and Toxicity of Adult Medulloblastoma Treated with Pediatric Multimodal Protocols: A Single-Institution Experience.Oncology research · 2025Article
- SNO-EANO-EURACAN consensus on management of pineal parenchymal tumors.Neuro-oncology · 2024Review
- Article
- Clinical and translational advances in primary brain tumor therapy with a focus on glioblastoma-A comprehensive review of the literature.World neurosurgery: X · 2024Review
- Long-term Outcomes of Protocol-Based Treatment for Newly Diagnosed Medulloblastoma.Cancer research and treatment · 2024Article
- Clinico-Radiological Outcomes in WNT-Subgroup Medulloblastoma.Diagnostics (Basel, Switzerland) · 2024Article
- Evolution of Systemic Therapy in Medulloblastoma Including Irradiation-Sparing Approaches.Diagnostics (Basel, Switzerland) · 2023Review
- Clinical outcome of pediatric medulloblastoma patients with Li-Fraumeni syndrome.Neuro-oncology · 2023Article
- Recent Advances in Pediatric Medulloblastoma.Current neurology and neuroscience reports · 2023Review
- Optimizing reirradiation for relapsed medulloblastoma: identifying the ideal patient and tumor profiles.Journal of neuro-oncology · 2023Article
- Molecular characterisation defines clinically-actionable heterogeneity within Group 4 medulloblastoma and improves disease risk-stratification.Acta neuropathologica · 2023Article
- Medulloblastoma: Current Perspectives and Recent Advances.Brain tumor research and treatment · 2023Review
- Review
- Review
- Clinical Trials in High-Risk Medulloblastoma: Evolution of the SIOP-Europe HR-MB Trial.Cancers · 2022Review
Corrections and comments
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Authors and funding
24 authors at 16 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHigh-risk medulloblastoma is defined by the presence of metastatic disease and/or incomplete resection and/or unfavorable histopathology and/or tumors with MYC amplification. We aimed to assess the 3-year progression-free survival (PFS) and define the molecular characteristics associated with PFS in patients aged 5-19 years with newly diagnosed high-risk medulloblastoma treated according to the phase II trial PNET HR+5.
methodsAll children received postoperative induction chemotherapy (etoposide and carboplatin), followed by 2 high-dose thiotepa courses (600 mg/m2) with hematological stem cell support. At the latest 45 days after the last stem cell rescue, patients received risk-adapted craniospinal radiation therapy. Maintenance treatment with temozolomide was planned to start between 1-3 months after the end of radiotherapy. The primary endpoint was PFS. Outcome and safety analyses were per protocol (all patients who received at least one dose of induction chemotherapy).
resultsFifty-one patients (median age, 8 y; range, 5-19) were enrolled. The median follow-up was 7.1 years (range: 3.4-9.0). The 3 and 5-year PFS with their 95% confidence intervals (95% CI) were 78% (65-88) and 76% (63-86), and the 3 and 5-year OS were 84% (72-92) and 76% (63-86), respectively. Medulloblastoma subtype was a statistically significant prognostic factor (P-value = 0.039) with large-cell/anaplastic being of worse prognosis, as well as a molecular subgroup (P-value = 0.012) with sonic hedgehog (SHH) and group 3 being of worse prognosis than wingless (WNT) and group 4. Therapy was well tolerated.
conclusionsThis treatment based on high-dose chemotherapy and conventional radiotherapy resulted in a high survival rate in children with newly diagnosed high-risk medulloblastoma.
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