Table 1.
Proposed consensus for the design of the next generation of clinical trials
1. Subgrouping: All patients should be treated on a molecularly informed clinical trial. All tumors subgrouped by genome wide methylation array or other validated methods using at least 2 techniques as part of initial clinical workup |
2. Tissue collection: Snap frozen and paraffin embedded tumor tissue, blood and CSF should be collected on all patients |
3. Central review: Real-time neuroimaging, neuropathological diagnostics and radiotherapy planning for clinical trial or registry |
4. Treatment-related side effects: Quality of life measures and neuropsychological outcomes including short, medium and long term is a high priority and should be evaluated in all patients |
5. TP53 mutations in SHH medulloblastoma: TP53 mutated SHH patients have a very poor prognosis and new treatment options are needed especially if germline TP53 mutation |
6. Genetic predisposition: All families with pediatric patients carrying SHH tumors should be offered genetic counseling. Tumors should be sequenced for somatic and germline mutations of TP53, PTCH, and SUFU as part of the diagnostic process in accredited laboratories. |
7. Need to re-biopsy: Recurrent tumors should be re-biopsied before using targeted therapy or 2 years beyond initial diagnosis or diagnosis is in doubt |
8. Extent of resection: Neurosurgeons should aim for maximal safe removal: NTR (to be defined) is acceptable and prognostically equivalent to GTR for staging |
9. Non-metastatic WNT medulloblastoma: All WNT properly subgrouped < 16 years old have excellent prognosis and should be treated with reduced radiation/chemotherapy |