Laryngeal lymphoma : the high and low grades of rare lymphoma involvement sites

The larynx is an extremely rare site of involvement by lymphomatous disease.We present two cases of isolated laryngeal high-grade and another low-grade lymphoma, together with a literature review of laryngeal lymphoma management.peer-reviewe

Hepatic Necrosis after Trans-Arterial Embolization of Metastatic Neuroendocrine Tumour

Liver metastases from neuroendocrine tumours (NETs) are usually seen on cross-sectional imaging at presentation. In such cases, curative surgical resection is usually not possible given that most patients have bilobar disease. Various locoregional approaches are now widely available that enable local control of disease and management of systemic symptoms. These include trans-arterial embolization (TAE), trans-arterial chemoembolization (TACE), selective internal radiotherapy and thermal ablation. We describe a rare case of hepatic necrosis after TAE in a 61-year-old woman with a metastatic NET. Cross-sectional imaging showed extensive necrosis affecting segments VII and VIII of the liver. This occurred secondary to thrombosis in the portal vein branches to these segments, confirmed on a Doppler ultrasound scan. The mechanism of portal vein thrombosis after TAE could be due to the presence of occult arterioportal anastomoses. These allow transit of the embolizing agents into the portal vein branches supplying the treated segments and subsequent thrombosis

Filière « Territoires, espaces, sociétés »

Participants : Isabelle Backouche, Juan Carlos Garavaglia, Nancy L. Green, Alexandra Laclau, Christian Lamouroux, Michèle de La Pradelle, Susanna Magri, Marie-Claude Maurel, Alain Musset, Daniel Nordman, Marie-Vic Ozouf-Marignier, Jacques Poloni-Simard, Paul-André Rosental, Annie Sevin, Annick Tanter-Toubon, Christian Topalov, Caroline Varlet, Nicolas Verdier, Françoise Vergneault-Belmont, François Weil, Pierre-Paul Zalio L’enseignement de la filière s’inscrit dans le cadre du séminaire bimen..

Filière « Territoires, espaces, sociétés »

Participants : Isabelle Backouche, Juan Carlos Garavaglia, Nancy L. Green, Alexandra Laclau, Christian Lamouroux, Michèle de La Pradelle, Susanna Magri, Marie-Claude Maurel, Alain Musset, Daniel Nordman, Marie-Vic Ozouf-Marignier, Jacques Poloni-Simard, Paul-André Rosental, Annie Sevin, Annick Tanter-Toubon, Christian Topalov, Caroline Varlet, Nicolas Verdier, Françoise Vergneault-Belmont, François Weil, Pierre-Paul Zalio L’enseignement de la filière s’inscrit dans le cadre du séminaire bimen..

Suitability of external controls for drug evaluation in Duchenne muscular dystrophy

OBJECTIVE: To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular dystrophy (DMD). METHODS: The consistency of changes in the 6-minute walk distance (Δ6MWD) was assessed across multiple clinical trial placebo arms and sources of NHD/RWD. Six placebo arms reporting 48-week Δ6MWD were identified via literature review and represented 4 sets of inclusion/exclusion criteria (n = 383 patients in total). Five sources of RWD/NHD were contributed by Universitaire Ziekenhuizen Leuven, DMD Italian Group, The Cooperative International Neuromuscular Research Group, ImagingDMD, and the PRO-DMD-01 study (n = 430 patients, in total). Mean Δ6MWD was compared between each placebo arm and RWD/NHD source after subjecting the latter to the inclusion/exclusion criteria of the trial for baseline age, ambulatory function, and steroid use. Baseline covariate adjustment was investigated in a subset of patients with available data. RESULTS: Analyses included ∼1,200 patient-years of follow-up. Differences in mean Δ6MWD between trial placebo arms and RWD/NHD cohorts ranged from -19.4 m (i.e., better outcomes in RWD/NHD) to 19.5 m (i.e., worse outcomes in RWD/NHD) and were not statistically significant before or after covariate adjustment. CONCLUSIONS: We found that Δ6MWD was consistent between placebo arms and RWD/NHD subjected to equivalent inclusion/exclusion criteria. No evidence for systematic bias was detected. These findings are encouraging for the use of RWD/NHD to augment, or possibly replace, placebo controls in DMD trials. Multi-institution collaboration through the Collaborative Trajectory Analysis Project rendered this study feasible

Real-World Safety and Efficacy of Nivolumab in Advanced Squamous and Nonsquamous Non-Small-Cell Lung Cancer: A Retrospective Cohort Study in Croatia, Hungary, and Malta

Background. There is a lack of real-world data on the safety and efficacy of nivolumab in patients with previously treated advanced non-small-cell lung cancer (NSCLC) especially in South East Europe, a region with particularly high incidence and an unfavorable mortality-to-incidence ratio for lung cancer. Objectives. To evaluate the real-world safety and efficacy of nivolumab in patients with previously treated advanced squamous and nonsquamous NSCLC in South East Europe. Methods. This is a multicenter, retrospective cohort study on patients with stage IIIB or IV disease with at least one previous systemic treatment who received nivolumab through an expanded-access program between 2015 and 2017 in Croatia, Malta, and Hungary. The primary endpoint was the proportion of patients whose therapy was discontinued because of toxicity. Secondary endpoints were the incidence of adverse events (AEs), objective response rate (ORR), disease control rate (DCR), time to response (TTR), duration of response (DOR), progression-free survival (PFS), and overall survival (OS). Results. We analyzed data on 239 patients with a median (IQR) age of 62 (57–68), and 33% of them were women. Treatment was discontinued because of toxicity in 11.6% (95% CI 7.8% to 16.5%) of patients. The PFS was 6.4 (95% CI 5.2 to 8.6) months, and the median OS was 14.1 (10.6 to 18.0) months. Conclusions. The safety and efficacy of nivolumab in previously treated patients with advanced NSCLC in the real-world South East Europe clinical settings were consistent with the results of randomized clinical trials and comparable to the results from other countries

Role of allelic variants of FK506-binding protein 51 (FKBP5) gene in the development of anxiety disorders

Background: Anxiety disorders exhibit remarkably high rates of comorbidity with major depressive disorder (MDD). Mood and anxiety disorders are considered stress-related diseases. Genetic variations in the co-chaperone FK506-binding protein 51, FKBP5, which modulates the function of glucocorticoid receptors, have been associated with an increased risk for the development of posttraumatic stress disorder, but data regarding its role in MDD are controversial. The aims of this study were to clarify the role of the FKBP5 gene in depression and anxiety disorders through a case–control study and an associationstudy with personality traits using the Temperament and Character Inventory (TCI) in healthy subjects. Methods: Six hundred fifty-sevenMDDpatients, with or without an anxiety disorder in comorbidity, and 462 healthy volunteers were enrolled in the study. Two hundred fifty-six controls agreed to fill out the TCI. Results: The results showed that the T allele of rs1360780 was more frequent among the patients affected by MDD with a comorbidity of anxiety disorders, compared to those without (P < .001). Among the controls, we found that the T allele more often exhibited personality traits associated with an increased vulnerability to anxiety. Conclusions: These results support the hypothesis that allelic variants of FKBP5 are a risk factor for anxiety disorders. The identification ofgenetic variants involved in anxiety may have implications for the optimization of therapeutic interventions