ADVERTISEMENT
Vemurafenib Beneficial for Nonmelanoma Cancers With BRAF V600 Mutations
Study data presented by Pascale Tomasini, MD, and co-investigators at the 2020 ESMO Virtual Congress show that vemurafenib has an important role in the treatment of BRAF V600-mutated nonmelanoma cancers.
BRAF mutations have been identified and observed in several cancer histotypes at a low frequency of <5%. Vemurafenib, active in BRAF-V600- mutated melanoma, as well as non-melanoma BRAF-V600-mutated cancers were also reported to respond to BRAF inhibitors.
BRAF mutational status were measured based upon direct sequencing (or NGS) within the INCa molecular genetic platforms network.
Between October 2014 and May 2019, a total of 216 (median age, 66 years) patients were included in the study from 116 centers.
In addition, patients with various BRAF (being V600 or non V600) mutated cancers (lung, ovarian, bladder, thyroid, prostate cancers, cholangiocarcinoma, sarcoma/GIST, multiple myeloma, chronic lymphocytic leukemia [CLL], and hairy cell leukemia [HCL]) advancing after 1 or more standard treatment, were incorporated within dedicated cohorts to receive vemurafenib 960 mg twice daily.
The Bayesian approach permitted sequential analyses in each cohort and prompt hindering using an inefficacy boundary for objective response rate of 10%. The ORR was evaluated every 8 weeks using RECIST v1.1 criteria for solid tumors and specific criteria for myeloma, CLL and HCL.
“Antitumor activity of vemurafenib was important in V600-mutated NSCLC, HCL, sarcoma, ovarian cancer, brain tumors, histiocytosis and gangliogliomas. Importantly, Non-V600 mutated tumors derived no benefit,” Dr Tomasini and co-investigators concluded.—Alexis Hyams
Tomasini P, Mazieres J, Cropet C, et al. Vemurafenib in non-melanoma V600 and non-V600 BRAF mutated cancers: Results of the AcSé basket trial. Presented at: the ESMO Virtual Congress 2020; September 19-21, 2020; virtual. Abstract 538P.
