Living with recurrent medulloblastoma can be an incredibly challenging journey, particularly when facing limited treatment options. However, a recent breakthrough offers hope for patients in this difficult situation. The Medulloblastoma European Multitarget Metronomic Anti-Angiogenic Trial (MEMMAT) has explored a unique treatment approach, and the results are encouraging.
About the study
The study, conducted between April 2014 and March 2021, involved 40 paediatric patients under 20 years old at the time of their original diagnosis. These patients faced relapsed or refractory medulloblastoma after previous radiation therapy. The innovative treatment plan included a combination regimen used to treat recurrent medulloblastoma. The combination of these drugs, administered in a metronomic fashion (more frequent with lower doses), aims to provide sustained therapeutic effects while minimizing toxicity.
The drugs used included daily oral thalidomide, fenofibrate, celecoxib, and alternating cycles of low-dose oral etoposide and cyclophosphamide. Additionally, intravenous bevacizumab and intraventricular therapy were administered.
Were there positive outcomes?
After six months of this therapy, 57.5% of patients achieved disease control, with a notable response in 45% of cases. The median overall survival (OS) reached 25.5 months, offering a sustained survival benefit for those facing recurrent medulloblastoma. The study also assessed progression-free survival (PFS), which was 8.5 months on average. These findings are particularly promising, considering the challenging nature of recurrent medulloblastoma.
What is the long-term impact?
Looking at the broader picture, the study observed a mean progression-free survival (PFS) of 24.6% at both 3 and 5 years, with overall survival (OS) at 43.6% and 22.6%, respectively. The treatment showed consistency across molecular subgroups and the number of prior recurrences. Notably, patients who responded well to the treatment demonstrated a 5-year PFS of 49.7%, and those who remained progression-free for the first 12 months saw an impressive 66.7% 5-year PFS.
Equally important is the fact that the MEMMAT combination regimen was generally well-tolerated by patients. Although there were some grade 3 to 4 treatment-related adverse events, such as myelosuppression (where the bone marrow’s ability to produce blood cells is inhibited), infections, seizures, and headaches, the overall safety profile suggests that this predominantly oral and outpatient treatment could be a feasible option for recurrent medulloblastoma.
In conclusion, the MEMMAT combination regimen offers hope for those grappling with recurrent medulloblastoma. The study’s results, showcasing a well-tolerated and promising treatment approach, call for further evaluation and potential integration into standard care protocols. This breakthrough opens new doors in the quest to improve outcomes and quality of life for individuals facing the challenges of recurrent medulloblastoma.
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