Publications by authors named "Caroline Gaudy-Marqueste"

Metastatic uveal melanoma is a rare disease with a poor prognosis. Usual treatments have not proven effective. Tebentafusp, a bispecific protein targeting melanoma cells and T lymphocytes, is the first approved treatment with a proven survival benefit in a randomized clinical.

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Background: Nivolumab obtained approval in advanced melanoma (AM) with weight-adjusted dose (WAD) administration (3 mg/kg/2 weeks). In 2018, the dosage regimen was changed to flat dose (FD) administration (240 mg/2 weeks or 480 mg/4 weeks) based on a modeling study, without clinical data.

Methods: AM patients have been prospectively included in the French national multicenter MelBase database since 2013.

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Background: The Checkmate 067 randomized controlled trial, published in 2015, demonstrated improved progression-free survival and numerically, although not statistically, superior overall survival for ipilimumab + nivolumab. The objective of this study was to compare the efficacy and safety of nivolumab to ipilimumab + nivolumab as first-line treatment for metastatic melanoma in a real-world setting.

Methods: Patients were prospectively included in the French Melbase cohort from 2013 to 2022.

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Article Synopsis
  • Advanced systemic mastocytosis (AdvSM) is a complex condition linked to poor outcomes, and while midostaurin is the first approved treatment, its long-lasting effectiveness is limited.
  • Various prognostic scoring systems like MARS, IPSM, and GPSM have been developed to assess patients' outcomes, but it's essential to tailor these scores to specific AdvSM subtypes for better accuracy.
  • A study of patients treated with midostaurin revealed that MARS and AdvSM subtype significantly predict overall survival, identifying five distinct patient subgroups with varying median survival times, highlighting the need for personalized management strategies.
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Background: Previous results from this trial showed longer overall survival after treatment with nivolumab plus ipilimumab or with nivolumab monotherapy than with ipilimumab monotherapy in patients with advanced melanoma. Given that patients with advanced melanoma are living longer than 7.5 years, longer-term data were needed to address new clinically relevant questions.

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Background: Currently, treatment options for patients with advanced melanoma who experience failed immunotherapy or targeted therapy are lacking. Recent studies suggest the antitumor activity of combined pembrolizumab and lenvatinib in patients with advanced melanoma progressing on immunotherapy. Herein, we report the clinical outcomes of combined lenvatinib and a programmed cell death protein-1 inhibitor (PD-1) in this population.

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  • Some patients with metastatic melanoma (a type of skin cancer) don't get better from usual treatments. Scientists are looking at personalized medicine, which means tailoring treatment to the patient’s specific cancer.
  • In a study, 26 patients who found specific genetic changes in their cancer received personalized treatments, with some seeing good results.
  • The study showed that using advanced testing followed by targeted therapy might help patients whose melanoma didn't respond to regular treatments.
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Background And Aims: Systemic mastocytosis (SM) is characterized by the accumulation of atypical mast cells (MCs) in organs. Liver histology of SM has been marginally described and accurate histological classification is critical, given the consequences of aggressive SM diagnosis. We aimed to describe the histological features associated with liver SM using updated tools.

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Melanoma is still a major health problem worldwide. Early diagnosis is the first step toward reducing its mortality, but it remains a challenge even for experienced dermatologists. Although computer-aided systems have been developed to help diagnosis, the lack of insight into their predictions is still a significant limitation toward acceptance by the medical community.

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Background: Mastocytosis and monoclonal mast cell (MC) activation syndrome (MMAS) are heterogeneous conditions characterized by the accumulation of atypical MCs. Despite the recurrent involvement of KIT mutations, the pathophysiologic origin of mastocytosis and MMAS is unclear. Although hereditary α-tryptasemia (HαT, related to TPSAB1 gene duplication) is abnormally frequent in these diseases, it is not known whether the association is coincidental or causal.

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  • Basal cell carcinoma (BCC) is the most prevalent skin cancer in white populations, and updated guidelines for its diagnosis and treatment have been established by a collaboration of various European medical organizations.
  • BCCs are classified into 'easy-to-treat' and 'difficult-to-treat' types, with complete surgical removal as the primary treatment method; alternative treatments include topical therapies, radiotherapy, and immunotherapy for more advanced cases.
  • Regular skin examinations and long-term follow-ups are crucial for patients at higher risk for BCC, including those with Gorlin syndrome or multiple lesions.
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Introduction: Metastatic melanoma is an aggressive tumor and can constitute a real therapeutic challenge despite the significant progress achieved with targeted therapies and immunotherapies, thus highlighting the need for the identification of new therapeutic targets. Adrenomedullin (AM) is a peptide with significant expression in multiple types of tumors and is multifunctional. AM impacts angiogenesis and tumor growth and binds to calcitonin receptor-like receptor/receptor activity-modifying protein 2 or 3 (CLR/RAMP2; CLR/RAMP3).

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Background: Anti-PD1 agents are currently recommended as first-line treatment in advanced cutaneous squamous cell carcinoma (acSCC) by updated European guidelines. Although acSCC frequently affects elderly patients with multiple comorbidities, this subset of patients is often excluded of registration clinical trials.

Purpose: To assess anti-PD-1 efficacy and safety in elderly acSCC patients in real-life conditions and describe this specific population with oncogeriatric evaluation tools.

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Background: Cancer therapies targeting actionable molecular alterations (AMA) have developed, but the clinical routine impact of high-throughput molecular profiling remains unclear. We present a monocentric experience of molecular profiling based on liquid biopsy in patients with cancer.

Methods: Patients included had solid cancer and underwent cfDNA genomic profiling with FoudationOne Liquid CDx (F1LCDx) test, analyzing 324 genes.

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Although cemiplimab has been approved for locally advanced (la) and metastatic (m) cutaneous squamous-cell carcinomas (CSCCs), its real-life value has not yet been demonstrated. An early-access program enrolled patients with la/mCSCCs to receive cemiplimab. Endpoints were best overall response rate (BOR), progression-free survival (PFS), overall survival (OS), duration of response (DOR) and safety.

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Article Synopsis
  • The study investigates the growth rates of primary cutaneous melanomas (≥1 mm thickness) and their link to aggressiveness, focusing on fast-growing melanomas (FGMM) versus slower ones.
  • Researchers evaluated the mutational profiles of these melanomas, finding that FGMM displayed a higher number of harmful mutations and poorer relapse-free survival rates.
  • Key factors associated with FGMM include ulceration, increased thickness, lower sun exposure, and specific mutations in the FGFR2 gene, suggesting that testing for FGFR2 could help identify patients at higher risk for aggressive disease.
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