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Ref Type Journal Article
PMID (39234402)
Authors Kroll MR, Au C, Slostad J, Christ TN, Papas SG, Tan A
Title Case report: Metastatic BRAF V600E-mutated adult Wilms' tumor with robust response to BRAF/MEK inhibitor therapy.
Journal Vol Issue Date Pages Journal Short Title
Frontiers in oncology 14 2024 1376270 Front Oncol
URL
Abstract Text Nephroblastoma or Wilms' tumor (WT) is the most common pediatric renal malignancy but rare in adults. Treatment protocols for adults are typically extrapolated from pediatric guidelines, but there are no standard guidelines for adults due to the rarity of the disease. However, next-generation sequencing has led to new therapeutic options for adult WT patients. We present the first case to our knowledge of a recurrent adult WT treated with dual BRAF/MEK-targeted therapy, which showed initial robust clinical response and was well tolerated.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E renal Wilms' tumor predicted - sensitive Dabrafenib + Trametinib Case Reports/Case Series Actionable In a clinical study, combination treatment with Tafinlar (dabrafenib) and Mekinist (trametinib) resulted in regression in the metastatic disease of the liver, spleen, and peritoneum along with clinical symptom improvement in an adult patient with metastatic Wilms' tumor harboring BRAF V600E (PMID: 39234402). 39234402