Title: HR-MB: An international High-Risk Medulloblastoma trial
Funder: The Brain Tumour Charity

Abstract: Medulloblastoma is a type of cancer found in and around the balance centre in the brain (cerebellum). It affects children and young adults. Typically, standard treatment involves surgery, radiotherapy and chemotherapy. At diagnosis, patients are placed in to ‘risk’ groups based on whether the cancer has spread to other parts of the body, how much tumour is remaining after surgery and the biological features of the tumour. The long-term survival outcome of those patients with standard and low-risk disease is approximately 80%. However, those patients with high-risk disease have a poorer outcome and the HR-MB clinical trial will be looking to see if the outcome in this patient group can be improved by comparing different radiotherapy and chemotherapy treatment regimens. There have been very few clinical trials which have studied high-risk disease and the HR-MB trial will one of the first trials to determine whether different ways of giving radiotherapy and chemotherapy affect patient outcome, as well as looking in to the how the biology of the disease may have an impact. The treatment regimens to be evaluated: The trial will test whether either high dose chemotherapy followed by conventional radiotherapy (once a day for 6 weeks) or more intensive radiotherapy (twice a day for 3 weeks) leads to improved outcome, with acceptable side effects, compared to conventional radiotherapy. A second question is whether oral chemotherapy for maintenance treatment (after radiotherapy) is as effective as conventional intravenous maintenance chemotherapy. Quality of survival The treatment for medulloblastoma also unfortunately has many side effects that can have a significant impact on a patient’s quality of life and, so the trial will also be evaluating how the different treatments affect patients’ quality of survival. Biological research Tumour samples from the trial will be analysed to identify any biological characteristics that predict which patients will do well or less well. This information will be used to potentially alter and personalise treatment in the future. Hopefully, this will help doctors in the future to identify patients who may be able to receive less treatment and therefore experience less side-effects and/or identify patients who may benefit from more intensive therapy.

Lay abstract: Medulloblastoma is a type of cancer found in and around the balance centre in the brain (cerebellum). It affects children and young adults. Typically, standard treatment involves surgery, radiotherapy and chemotherapy. At diagnosis, patients are placed in to ‘risk’ groups based on whether the cancer has spread to other parts of the body, how much tumour is remaining after surgery and the biological features of the tumour. The long-term survival outcome of those patients with standard and low-risk disease is approximately 80%. However, those patients with high-risk disease have a poorer outcome and the HR-MB clinical trial will be looking to see if the outcome in this patient group can be improved by comparing different radiotherapy and chemotherapy treatment regimens. There have been very few clinical trials which have studied high-risk disease and the HR-MB trial will one of the first trials to determine whether different ways of giving radiotherapy and chemotherapy affect patient outcome, as well as looking in to the how the biology of the disease may have an impact. The treatment regimens to be evaluated: The trial will test whether either high dose chemotherapy followed by conventional radiotherapy (once a day for 6 weeks) or more intensive radiotherapy (twice a day for 3 weeks) leads to improved outcome, with acceptable side effects, compared to conventional radiotherapy. A second question is whether oral chemotherapy for maintenance treatment (after radiotherapy) is as effective as conventional intravenous maintenance chemotherapy. Quality of survival The treatment for medulloblastoma also unfortunately has many side effects that can have a significant impact on a patient’s quality of life and, so the trial will also be evaluating how the different treatments affect patients’ quality of survival. Biological research Tumour samples from the trial will be analysed to identify any biological characteristics that predict which patients will do well or less well. This information will be used to potentially alter and personalise treatment in the future. Hopefully, this will help doctors in the future to identify patients who may be able to receive less treatment and therefore experience less side-effects and/or identify patients who may benefit from more intensive therapy.

Research activity codes: 6.5 RADIOTHERAPY AND OTHER NON-INVASIVE THERAPIES
Health categories: CANCER AND NEOPLASMS