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The problem
Intervention: Early molecular testing (BRAF mutation analysis) followed by vemurafenib (BRAF-inhibitor) targeted therapy instead of or before surgery
Who it was tested with: Two children with low-grade gliomas: a 12-year-old girl and a 3-year-old boy, both with BRAF V600E mutations.
Experience from people who deliver programmes: stories, case studies and lessons learned. Valuable for understanding how and why, and for spotting what to test next, but not a measure of effect.
"After one month 45% reduction of the mass according to RANO criteria was found, as well as better balance control and strong reduction of the right arm paresis; five months later, a 70% shrinkage was detected, stabilized to 76% after a year" and "After only three days, visual acuity and muscle tone improved; brain MRI showed a 34% reduction of the mass after one week, increased up to 65% after six months." These case descriptions show rapid clinical and radiological responses to targeted therapy, demonstrating the intervention produced measurable improvements in both children.
Only two cases reported; no comparison group; unclear generalisability to other children with low-grade gliomas; long-term outcomes and potential adverse effects not detailed; both cases selected retrospectively, creating selection bias.
Across countries and designs
1 study · 0 countries
