RESUMO
Clinical trials of targeted therapy (TT) and immunotherapy (IT) for highly aggressive advanced melanoma have shown marked improvements in response and survival rates. However, real-world data on treatment patterns and clinical outcomes for patients with advanced BRAF V600 mutant melanoma are ultimately scarce. The study was designed as an observational retrospective chart review study, which included 382 patients with advanced BRAF V600 mutant melanoma, who received TT in a real-world setting and were not involved in clinical trials. The data were collected from twelve medical centers in Russia. The objective response rates (ORRs) to combined BRAFi plus MEKi and to BRAFi mono-therapy were 57.4% and 39.8%, respectively. The median progression-free survival (PFS) and median overall survival (OS) were 9.2 months and 22.6 months, respectively, for the combined first-line therapy; 9.4 months and 16.1 months, respectively, for the combined second-line therapy; and 7.4 months and 17.1 months, respectively, for the combined third- or higher-line therapy. Analysis of treatment patterns demonstrated the effectiveness of the combined TT with BRAF plus MEK inhibitors in patients with brain metastases, rare types of BRAF mutations, and across lines of therapy, as well as a well-tolerated and manageable safety profile.
RESUMO
Mutations of the epidermal growth factor receptor (EGFR) gene are known to be associated with the pronounced tumor response to EGFR tyrosine kinase inhibitors (TKI). Unfortunately, these TKI-sensitizing alterations have been detected almost exclusively in lung adenocarcinomas; indeed, their occurrence in tumors of other histologic types or other organs is exceptionally rare. Here we report a case of intragenic EGFR microdeletion in renal cell carcinoma (RCC) associated with the effect of treatment by gefitinib. Patient G., 60 years old, underwent radical surgery for clear cell RCC, however local relapse and metastatic lesions in the lungs were revealed 33 months later. The patient was successfully treated by interleukin-2 (first line therapy), tamoxifen (second line), and interferon-alpha (third line). After disease progression, EGFR mutation test was performed as a compassionate attempt. It revealed "classical" 15 base pair deletion, so gefitinib was administered. This treatment led to a dramatic symptomatic response within the first week of therapy; reduction of dyspnea was so evident that it allowed the patient to return to work. Clinical examination demonstrated significant improvement with respect to pleuritis and pericarditis. The duration of tumor response, which was classified as disease stabilization, was equal to 4 months. This case report suggests that kidney tumors have to be investigated more closely with respect to the occurrence of TKI-sensitizing EGFR mutations.
