vemu {basket}R Documentation

Summary Data from the Vemurafenib Study


The 'vemu' and 'vemu_wide' data sets provides response information taken from the “Vemurafenib in multiple nonmelanoma cancers with BRAF v600 mutations” study where, in total, 18 responders were observed among the 84 patients contributing evaluable outcomes for statistical estimation. Observed response rates varied from $42%$ and $43%$ for baskets of NSCLC and ECD or LCH to 0 and $4%,$ for CRC with vemurafenib mono and combination therapies, respectively. Two responders of seven patients, ATC was associated with a $29%$ response rate, while one responder of eight patients was observed in the cholangiocarcinoma basket. Contrasting favorable results for preliminary vemurafenib activity among NSCLC and ECD or LCH patients with less favorable results for CRC patients, the authors concluded that nonmelanoma tumor types harboring $BRAF^V600$ mutations failed to respond uniformly to BRAF-targeted therapy giving credence to more conventional organ-specific nosology when compared to molecular tumor nosology.

Later, in the “Statistical challenges posed by basket trials: sensitivity analysis of the Vemurafenib study” it was shown that patient-enrollment types we likely drove the negative results for several targets, rather than Vemurafenib itself.


Hyman DM, Puzanov I, Subbiah V, Faris JE, Chau I, Blay JY, Wolf J, Raje NS, Diamond EL, Hollebecque A, et al. Vemurafenib in multiple nonmelanoma cancers with braf v600 mutations. New England Journal of Medicine 2015; 373(8):726

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