Trial reportJournal of clinical oncology : official journal of the American Society of Clinical Oncology2020
Nonmetastatic Medulloblastoma of Early Childhood: Results From the Prospective Clinical Trial HIT-2000 and An Extended Validation Cohort.
Trial report in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 58 papers.
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Who cites it
58 citing papers in PubMed, 98 citations in OpenAlex.
- High-dose chemotherapy with autologous stem cell rescue in children under 5 years of age with central nervous system embryonal tumors: results from a prospective cohort in an upper-middle-income country.Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery · 2026Article
- Therapy and biomarker dependent progression-free survival in infant sonic hedgehog medulloblastoma: a multi-national retrospective cohort study.EClinicalMedicine · 2026Article
- Hetairos is a histology-based artificial intelligence model for predicting central nervous system tumor methylation subtypes.Nature cancer · 2026Article
- Neuropsychological late effects in young children with medulloblastoma: A longitudinal analysis after 5 and 15 years from the HIT-SKK-'87/'92 studies.Neuro-oncology practice · 2026Article
- Medulloblastoma in Adolescents and Young Adults: Molecular Subgroups, Prognostic Biomarkers, and Age-Specific Therapeutic Challenges.Current issues in molecular biology · 2026Review
- Exploring the lactate-metabolism related characteristics during the development of medulloblastoma through single-cell and bulk RNA-seq.Translational cancer research · 2026Article
- Identification of Raptor and GLI1 as USP37 substrates highlight its context-specific function in medulloblastoma cells.Oncogene · 2026Article
- High-dose chemotherapy and craniospinal irradiation-sparing approach for WNT medulloblastoma of early childhood.Journal of neuro-oncology · 2026Article
- Therapeutic radiation drives leptomeningeal dissemination of medulloblastoma through an innate immune process.Developmental cell · 2025Article
- Clinical, toxicity and long-term neurocognitive outcomes of first-line intensive chemotherapy in infant medulloblastoma: a single-center cohort study following AIEOP recommendations.Journal of neuro-oncology · 2025Article
- Salvage therapies for first relapse of SHH medulloblastoma in early childhood.Neuro-oncology · 2025Article
- Outcomes of Infants and Young Children With CNS Embryonal Tumors Using Pre-Irradiation Chemotherapy: A Decade Long Experience.Cancer medicine · 2025Article
- Old Tools in a New Era: The Continued Relevance of Chemotherapy in Pediatric Neuro-Oncology.Current oncology (Toronto, Ont.) · 2025Review
- Advancing medulloblastoma therapy: strategies and survival insights.Clinical and experimental medicine · 2025Review
- Pediatric CNS Radiation Oncology: Recent Developments and Novel Techniques.Current oncology (Toronto, Ont.) · 2025Review
- Analysis of brain necrosis and secondary cancers after proton beam therapy for pediatric intracranial tumors: a single-center retrospective study.Frontiers in oncology · 2025Article
- CD4 T cells correlate with better prognosis in medulloblastoma.Frontiers in oncology · 2025Article
- Tumor-associated macrophages correlate with better outcome in SHH medulloblastoma.Frontiers in oncology · 2025Article
- Risk factors for domain-specific neurocognitive outcome in pediatric survivors of a brain tumor in the posterior fossa-Results of the HIT 2000 trial.Neuro-oncology · 2024Article
- Medulloblastoma in children with Fanconi anemia: Association with FA-D1/FA-N, SHH type and poor survival independent of treatment strategies.Neuro-oncology · 2024Article
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Authors and funding
49 authors at 20 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeThe HIT-2000-BIS4 trial aimed to avoid highly detrimental craniospinal irradiation (CSI) in children < 4 years of age with nonmetastatic medulloblastoma by systemic chemotherapy, intraventricular methotrexate, and risk-adapted local radiotherapy. PATIENTS AND
methodsFrom 2001-2011, 87 patients received systemic chemotherapy and intraventricular methotrexate. Until 2006, CSI was reserved for nonresponse or progression. After 2006, local radiotherapy was introduced for nonresponders or 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 in the HIT-2000-BIS4 cohort and a validation cohort (n = 71) from the HIT group and Russia.
resultsFive years after diagnosis, patients with desmoplastic medulloblastoma (DMB) or medulloblastoma with extensive nodularity (MBEN; n = 42) had 93% progression-free survival (5y-PFS), 100% overall survival (5y-OS), and 93% CSI-free (5y-CSI-free) survival. Patients with CMB/LCA (n = 45) had 37% 5y-PFS, 62% 5y-OS, and 39% 5y-CSI-free survival. 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. Group 3 patients (5y-PFS, 36%; n = 14) relapsed more frequently than the SHH-INF group (5y-PFS, 93%; n = 28) or group 4 patients (5y-PFS, 83%; n = 6;
conclusionSystemic chemotherapy and intraventricular methotrexate led to favorable survival in both iSHH subtypes of SHH-activated DMB/MBEN with acceptable neurotoxicity. Survival in patients with non-wingless (WNT)/non-SHH disease with CMB/LCA was not improved by local radiotherapy. Patients with group 4 disease had more favorable survival rates than those with group 3 medulloblastoma.
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