BACKGROUND: Immunotherapy and targeted therapy are currently two alternative backbones in the therapy of BRAF-mutated malignant melanoma. However, predictive biomarkers that would help with treatment selection are lacking. METHODS: This retrospective study investigated outcomes of anti-programmed death receptor-1 monotherapy and targeted therapy in the first-line setting in patients with metastatic BRAF-mutated melanoma, focusing on clinical and laboratory parameters associated with treatment outcome. RESULTS: Data from 174 patients were analysed. The median progression-free survival (PFS) was 17.0 months (95% CI; 8-39) and 12.5 months (95% CI; 9-14.2) for immunotherapy and targeted therapy, respectively. The 3-year PFS rate was 39% for immunotherapy and 25% for targeted therapy. The objective response rate was 72% and 51% for targeted therapy and immunotherapy. The median overall (OS) survival for immunotherapy has not been reached and was 23.6 months (95% CI; 16.1-38.2) for targeted therapy, with a 3-year survival rate of 63% and 40%, respectively. In a univariate analysis, age < 70 years, a higher number of metastatic sites, elevated serum LDH and a neutrophil-lymphocyte ratio above the cut-off value were associated with inferior PFS regardless of the therapy received, but only serum LDH level and the presence of lung metastases remained significant predictors of PFS in a multivariate analysis. CONCLUSIONS: Present real-world data document the high effectiveness of immunotherapy and targeted therapy. Although targeted therapy had higher response rates, immunotherapy improved PFS and OS. While the prognostic value of LDH was confirmed, the potential use of blood cell count-derived parameters to predict outcomes needs further investigation.
Spinocelulární karcinom anu představuje vzácné onemocnění gastrointestinálního traktu. Díky přítomnosti časných příznaků, mezi které patří krvácení, bolestivost a pocit rezistence v konečníku, bývá toto onemocnění diagnostikováno v časných stadiích. V současné době je standardním léčebným postupem multimodální přístup, který kombinuje radioterapii a chemoterapii na bázi 5-fluorouracilu s mytomicinem nebo cisplatinou. Prognóza je u většiny pacientů s onemocněním lokalizovaným pouze na anus velmi příznivá, přesto je léčba spjata s jistými riziky. Tato kazuistika popisuje managment širokého spektra komplikací včetně kardiotoxicity spojené s 5-fluorouracilem, rozvoje heparinem indukované trombocytopenie a trombotických komplikací u jinak standardně léčené pacientky pro spinocelulární karcinom anu.
Squamous cell carcinoma of the anus is a rare gastrointestinal tract disease. Due to the early presence of symptoms, which include bleeding, soreness and a feeling of resistance in the anus, this disease is usually diagnosed in its initial stages. The standard treatment consists of a multimodal approach combining radiotherapy and 5-fluorouracil-based chemotherapy with mitomycin or cisplatin. The prognosis is very favorable for most patients with the disease localized only to the anus, yet there are some risks associated with the treatment. This case report describes the management of a wide range of complications, including 5-fluorouracil-associated cardiotoxicity, development of heparin-induced thrombocytopenia, and thrombotic complications in a patient otherwise standardly treated for squamous cell carcinoma of the anus.
- MeSH
- antitumorózní látky aplikace a dávkování škodlivé účinky MeSH
- fluorouracil škodlivé účinky terapeutické užití MeSH
- heparin nízkomolekulární aplikace a dávkování škodlivé účinky MeSH
- kardiotoxicita patofyziologie patologie terapie MeSH
- lidé MeSH
- nádory anu * komplikace terapie MeSH
- nežádoucí účinky léčiv * patofyziologie patologie terapie MeSH
- riziko MeSH
- senioři MeSH
- trombocytopenie chemicky indukované patofyziologie terapie MeSH
- Check Tag
- lidé MeSH
- senioři MeSH
- ženské pohlaví MeSH
- Publikační typ
- kazuistiky MeSH
