22 results on '"Martellosio, Jean-Philippe"'
Search Results
2. Nocardia Infection in Patients With Anti–Granulocyte-Macrophage Colony-Stimulating Factor Autoantibodies: A Prospective Multicenter French Study.
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Kerdiles, Thibault, Lejeune, Sophie, Portais, Antoine, Bourgeois, Gaelle, Lefevre, Benjamin, Charmillon, Alexandre, Sixt, Thibault, Moretto, Florian, Cornille, Cyril, Vidal, Magali, Coustillères, François, Martellosio, Jean-Philippe, Quenet, Marion, Belan, Martin, Andry, Fanny, Jaffal, Karim, Pinazo-Melia, Angela, Rondeau, Paul, Paz, David Luque, and Jouneau, Stephane
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NOCARDIOSIS ,PULMONARY alveolar proteinosis ,AUTOANTIBODIES ,CENTRAL nervous system infections ,OPPORTUNISTIC infections ,SYMPTOMS - Abstract
Background Nocardiosis, a bacterial opportunistic infection caused by Nocardia spp, has recently been reported in patients with anti–granulocyte-macrophage colony-stimulating factor (GM-CSF) autoantibodies, but insufficient data are available about disease presentation, outcomes, and occurrence of autoimmune pulmonary alveolar proteinosis (aPAP) in this population. Methods We performed a prospective, multicenter, nationwide study in France and included patients with a Nocardia infection who had anti-GM-CSF autoantibodies. We describe their clinical, microbiological, and radiological characteristics, and their outcome at 1 year of follow-up. Results Twenty patients (18 [90%] male) were included, with a median age of 69 (interquartile range, 44–75) years. The organs most frequently involved were the brain (14/20 [70%]) and the lung (12/20 [60%]). Half of the infections were disseminated (10/20 [50%]). Nocardia identification was predominantly made in abscess fluid (17/20 [85%]), among which 10 (59%) were brain abscesses. The 1-year all-cause mortality was 5% (1/20), and only 1 case of aPAP (1/20 [5%]) occurred during the follow-up period. Conclusions Nocardiosis with anti-GM-CSF autoantibodies is associated with a low mortality rate despite a high incidence of brain involvement. Although the occurrence of aPAP was infrequent during the 1-year follow-up period, long-term clinical data are needed to fully understand the potential relationship between nocardiosis, anti-GM-CSF autoantibodies, and aPAP. [ABSTRACT FROM AUTHOR]
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- 2024
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3. Serious infections in patients with VEXAS syndrome: data from the French VEXAS registry
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de Valence, Benjamin, primary, Delaune, Marion, additional, Nguyen, Yann, additional, Jachiet, Vincent, additional, Heiblig, Mael, additional, Jean, Alexis, additional, Riescher Tuczkiewicz, Stanislas, additional, Henneton, Pierrick, additional, Guilpain, Philippe, additional, Schleinitz, Nicolas, additional, Le Guenno, Guillaume, additional, Lobbes, Hervé, additional, Lacombe, Valentin, additional, Ardois, Samuel, additional, Lazaro, Estibaliz, additional, Langlois, Vincent, additional, Outh, Roderau, additional, Vinit, Julien, additional, Martellosio, Jean-Philippe, additional, Decker, Paul, additional, Moulinet, Thomas, additional, Dieudonné, Yannick, additional, Bigot, Adrien, additional, Terriou, Louis, additional, Vlakos, Alexandre, additional, de Maleprade, Baptiste, additional, Denis, Guillaume, additional, Broner, Jonathan, additional, Kostine, Marie, additional, Humbert, Sebastien, additional, Lifermann, Francois, additional, Samson, Maxime, additional, Pechuzal, Susann, additional, Aouba, Achille, additional, Kosmider, Olivier, additional, Dion, Jeremie, additional, Grosleron, Sylvie, additional, Bourguiba, Rim, additional, Terrier, Benjamin, additional, Georgin-Lavialle, Sophie, additional, Fain, Olivier, additional, Mekinian, Arsène, additional, Morgand, Marjolaine, additional, Comont, Thibault, additional, and Hadjadj, Jerome, additional
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- 2023
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4. Serious infections in patients with VEXAS syndrome: data from the French VEXAS registry.
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de Valence, Benjamin, Delaune, Marion, Yann Nguyen, Jachiet, Vincent, Heiblig, Mael, Jean, Alexis, Tuczkiewicz, Stanislas Riescher, Henneton, Pierrick, Guilpain, Philippe, Schleinitz, Nicolas, Le Guenno, Guillaume, Lobbes, Hervé, Lacombe, Valentin, Ardois, Samuel, Lazaro, Estibaliz, Langlois, Vincent, Outh, Roderau, Vinit, Julien, Martellosio, Jean-Philippe, and Decker, Paul
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- 2024
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5. Benralizumab for eosinophilic granulomatosis with polyangiitis
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Cottu, Adrien, primary, Groh, Matthieu, additional, Desaintjean, Charlene, additional, Marchand-Adam, Sylvain, additional, Guillevin, Loïc, additional, Puechal, Xavier, additional, Beaumesnil, Stacy, additional, Lazaro, Estibaliz, additional, Samson, Maxime, additional, Taille, Camille, additional, Durel, Cécile-Audrey, additional, Diot, Elizabeth, additional, Nicolas, Sarah, additional, Guilleminault, Laurent, additional, Ebbo, Mikael, additional, Cathebras, Pascal, additional, Dupin, Clairelyne, additional, Yildiz, Halil, additional, Belfeki, Nabil, additional, Pugnet, Grégory, additional, Chauvin, Pierre, additional, Jouneau, Stephane, additional, Lifermann, Francois, additional, Martellosio, Jean-Philippe, additional, Cottin, Vincent, additional, and Terrier, Benjamin, additional
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- 2023
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6. Staphylococcus aureus Bacteremia: Towards Oral Step-Down Therapy in Selected Cases
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Martellosio, Jean-Philippe, primary, Lemaigre, Clément, additional, and Moal, Gwenaël Le, additional
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- 2023
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7. Time to negative PCR conversion amongst high-risk patients with mild-to-moderate Omicron BA.1 and BA.2 COVID-19 treated with sotrovimab or nirmatrelvir
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Martin-Blondel, Guillaume, primary, Marcelin, Anne-Geneviève, additional, Soulié, Cathia, additional, Kaisaridi, Sofia, additional, Lusivika-Nzinga, Clovis, additional, Zafilaza, Karen, additional, Dorival, Céline, additional, Nailler, Laura, additional, Boston, Anaïs, additional, Ronchetti, Anne-Marie, additional, Melenotte, Cléa, additional, Cabié, André, additional, Choquet, Christophe, additional, Trinh-Duc, Albert, additional, Lacombe, Karine, additional, Gaube, Géraldine, additional, Coustillères, François, additional, Pourcher, Valérie, additional, Martellosio, Jean-Philippe, additional, Peiffer-Smadja, Nathan, additional, Chauveau, Marie, additional, Housset, Pierre, additional, Piroth, Lionel, additional, Devaux, Mathilde, additional, Pialoux, Gilles, additional, Martin, Aurélie, additional, Dubee, Vincent, additional, Frey, Jérôme, additional, Le Bot, Audrey, additional, Cazanave, Charles, additional, Petua, Philippe, additional, Liblau, Roland, additional, Carrat, Fabrice, additional, and Yordanov, Youri, additional
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- 2023
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8. Hypervirulent Klebsiella Pneumoniae, an Emerging Cause of Endogenous Endophthalmitis in A French Center: A Comparative Cohort Study.
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Martellosio, Jean-Philippe, Gastli, Nabil, Farhat, Rebecca, Tazi, Asmaa, Duraffour, Pierre, Rossi, Benjamin, Canouï, Etienne, Morbieu, Caroline, Billoët, Annick, Mouthon, Luc, Poyart, Claire, Brézin, Antoine, and Legendre, Paul
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KLEBSIELLA pneumoniae , *ENDOPHTHALMITIS , *COHORT analysis , *COMPARATIVE studies - Abstract
Klebsiella pneumoniae (KP) is the most common cause of endogenous endophthalmitis (EE) in Asia, but data in Europe are scarce. We describe eight cases of KP EE compared to a cohort of EE in a French center. EE cases were retrospectively studied between January 2014 and January 2021. KP EE cases were analyzed to assess clinical, microbiological features, and outcome. Among the 33 EE cases identified, the first causative agent (24%, n = 8) was KP, mainly (7/8) with hypervirulent phenotype (hvKP). All but one of these cases occurred from December 2019 to January 2021. Contrary to non-KP patients, KP patients had multiple extraocular infective foci (p =.006), all presented with liver abscesses (p <.001), 50% had cerebral involvement (p =.13). Visual outcome was poor in both groups. KP is an emerging cause of EE in a French center, consistently associated with liver abscesses, frequent cerebral involvement, and predominance of hvKP strains. [ABSTRACT FROM AUTHOR]
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- 2023
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9. Efficacy and Safety of Doravirine-Based Regimens in Real Life: A Prospective Monocentric French Study
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Garcia, Magali, primary, Martellosio, Jean Philippe, additional, Giraud, Valentin, additional, Béraud, Guillaume, additional, Catroux, Mélanie, additional, Roblot, France, additional, and Le Moal, Gwénael, additional
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- 2022
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10. Sotrovimab to prevent severe COVID-19 in high-risk patients infected with Omicron BA.2
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Martin-Blondel, Guillaume, primary, Marcelin, Anne-Genevieve, additional, Soulié, Cathia, additional, Kaisaridi, Sofia, additional, Lusivika-Nzinga, Clovis, additional, Dorival, Céline, additional, Nailler, Laura, additional, Boston, Anaïs, additional, Melenotte, Cléa, additional, Cabié, André, additional, Choquet, Christophe, additional, Coustillères, François, additional, Martellosio, Jean-Philippe, additional, Gaube, Géraldine, additional, Trinh-Duc, Albert, additional, Ronchetti, Anne-Marie, additional, Pourcher, Valerie, additional, Chauveau, Marie, additional, Lacombe, Karine, additional, Peiffer-Smadja, Nathan, additional, Housset, Pierre, additional, Perrot, Aurore, additional, Pialoux, Gilles, additional, Martin, Aurélie, additional, Dubee, Vincent, additional, Devaux, Mathilde, additional, Frey, Jérôme, additional, Cazanave, Charles, additional, Liblau, Roland, additional, Carrat, Fabrice, additional, and Yordanov, Youri, additional
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- 2022
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11. Nebulized Liposomal Amphotericin B for Treatment of Pulmonary Infection Caused by Hormographiella aspergillata: Case Report and Literature Review
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Godet, Cendrine, Cateau, Estelle, Rammaert, Blandine, Grosset, Marine, Le Moal, Gwenaël, Béraud, Guillaume, Martellosio, Jean Philippe, Iriart, Xavier, Cadranel, Jacques, and Roblot, France
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- 2017
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12. Hypervirulent Klebsiella Pneumoniae, an Emerging Cause of Endogenous Endophthalmitis in A French Center: A Comparative Cohort Study
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Martellosio, Jean-Philippe, primary, Gastli, Nabil, additional, Farhat, Rebecca, additional, Tazi, Asmaa, additional, Duraffour, Pierre, additional, Rossi, Benjamin, additional, Canouï, Etienne, additional, Morbieu, Caroline, additional, Billoët, Annick, additional, Mouthon, Luc, additional, Poyart, Claire, additional, Brézin, Antoine, additional, and Legendre, Paul, additional
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- 2022
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13. Inhaled Antifungal Agents for Treatment and Prophylaxis of Bronchopulmonary Invasive Mold Infections
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Brunet, Kévin, primary, Martellosio, Jean-Philippe, additional, Tewes, Frédéric, additional, Marchand, Sandrine, additional, and Rammaert, Blandine, additional
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- 2022
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14. Late Onset of Chronic Granulomatous Disease Revealed by Paecilomyces lilacinus Cutaneous Infection
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Lemaigre, Clément, primary, Suarez, Felipe, additional, Martellosio, Jean-Philippe, additional, Barbarin, Cindy, additional, Brunet, Kévin, additional, Chomel, Jean Claude, additional, Hainaut, Ewa, additional, Rammaert, Blandine, additional, Roblot, France, additional, and Torregrosa-Diaz, José Miguel, additional
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- 2021
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15. Corticosteroids alter alveolar macrophage control of Lichtheimia corymbifera spores in an ex vivo mouse model
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Brunet, Kévin, primary, Arrivé, François, additional, Martellosio, Jean-Philippe, additional, Lamarche, Isabelle, additional, Marchand, Sandrine, additional, and Rammaert, Blandine, additional
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- 2020
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16. Bone marrow biopsy diagnostic yield in internal medicine
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Martellosio, Jean-Philippe, primary, Puyade, Mathieu, additional, Debiais, Céline, additional, Elsendoorn, Antoine, additional, Souchaud-Debouverie, Odile, additional, Landron, Cédric, additional, Luca, Luminita, additional, Roy-Peaud, Frédérique, additional, Milin, Serge, additional, Roblot, Pascal, additional, and Martin, Mickaël, additional
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- 2020
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17. Corticosteroids alter alveolar macrophage control of Lichtheimia corymbifera spores in an ex vivo mouse model.
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Brunet, Kévin, Arrivé, François, Martellosio, Jean-Philippe, Lamarche, Isabelle, Marchand, Sandrine, and Rammaert, Blandine
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Alveolar macrophages (AM) are the first-line lung defense against Mucorales in pulmonary mucormycosis. Since corticosteroid use is a known risk factor for mucormycosis, the aim of this study was to describe the role of corticosteroids on AM capacities to control Lichtheimia corymbifera spore growth using a new ex vivo model. An in vivo mouse model was developed to determine the acetate cortisone dose able to trigger pulmonary invasive infection. Then, in the ex vivo model, male BALB/c mice were pretreated with the corticosteroid regimen triggering invasive infection, before AM collection through bronchoalveolar lavage. AMs from corticosteroid-treated mice and untreated control AMs were then exposed to L. corymbifera spores in vitro (ratio 1:5). AM control of fungal growth, adherence/phagocytosis, and oxidative burst were assessed using optical densities by spectrophotometer, flow cytometry, and 2', 7'-dichlorofluoresceine diacetate fluorescence, respectively. Cortisone acetate at 500 mg/kg, at D-3 and at D0, led to pulmonary invasive infection at D3. Co-incubated spores and AMs from corticosteroid-treated mice had significantly higher absorbance (fungal growth) than co-incubated spores and control AMs, at 24 h (P = .025), 36 h (P = .004), and 48 h (P = .001). Colocalization of spores with AMs from corticosteroid-treated mice was significantly lower than for control AMs (7.6 ± 1.9% vs 22.3 ± 5.8%; P = .003), reflecting spore adherence and phagocytosis inhibition. Finally, oxidative burst was significantly increased when control AMs were incubated with spores (P = 0.029), while corticosteroids hampered oxidative burst from treated AMs (P = 0.321). Corticosteroids enhanced fungal growth of L. corymbifera through AM phagocytosis inhibition and burst oxidative decrease in our ex vivo model. Lay Summary The aim of this study was to describe the impact of corticosteroids on alveolar macrophage (AM) capacities to control Mucorales growth in a new murine ex vivo model. Corticosteroids enhanced fungal growth of L. corymbifera through AM phagocytosis inhibition and burst oxidative decrease. [ABSTRACT FROM AUTHOR]
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- 2021
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18. Bone marrow biopsy diagnostic yield in internal medicine.
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Martellosio, Jean-Philippe, Puyade, Mathieu, Debiais, Céline, Elsendoorn, Antoine, Souchaud-Debouverie, Odile, Landron, Cédric, Luca, Luminita, Roy-Peaud, Frédérique, Milin, Serge, Roblot, Pascal, and Martin, Mickaël
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INTERNAL medicine ,BONE marrow ,DIAGNOSIS ,MYELODYSPLASTIC syndromes ,BIOPSY ,GOLD standard ,RICE quality - Abstract
Trephine bone marrow biopsy (BMB) in internal medicine has only been studied in fever of unknown origin and inflammation of unknown origin. The aim was to assess BMB diagnostic yield according to main indications and patient characteristics in internal medicine. Quality of BMB and contribution of bone marrow aspiration (BMA) to BMB were also analyzed. BMB performed in the internal medicine department of Poitiers university hospital between January 2000 and December 2015 were retrospectively analyzed. Patient characteristics, BMB indications, quality parameters, and results were collected from medical records. Contributive BMB was BMB allowing accurate final diagnosis. Diagnostic yield was the proportion of contributive BMB among total BMB performed. A total of 468 BMBs conducted for primary diagnostic purpose from 468 patients were analyzed. Cytopenia(s) and the indication 'adenopathy and/or splenomegaly and/or hepatomegaly' represented 70% of the indications. Overall BMB diagnostic yield was 32.7%, lymphoma being the main histologic finding (31%). Among indications, cytopenia(s) had the highest diagnostic yield (49.1%). Isolated fever of unknown origin had low diagnostic yield (5.6%). Factors independently associated with contributive BMB were: anemia, neutropenia, circulating immature granulocytes or blasts, monoclonal gammopathy, period of BMB processing, quality of BMB, and immunohistochemestry (IHC) analysis. Concomitant BMA improved diagnostic yield by 5.5%, mostly for myelodysplastic syndromes. Cytopenia(s), blood cythemias and monoclonal gammopathy are indications with the highest diagnostic yield. Concomitant BMA and IHC analysis should be systematically performed to increase BMB diagnostic yield in internal medicine. [ABSTRACT FROM AUTHOR]
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- 2021
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19. Nebulized Liposomal Amphotericin B for Treatment of Pulmonary Infection Caused by Hormographiella aspergillata: Case Report and Literature Review
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Godet, France, Cateau, France, Rammaert, France, Grosset, France, Le Moal, France, Béraud, France, Martellosio, France, Iriart, France, Cadranel, France, Roblot, France, Godet, Cendrine, Cateau, Estelle, Rammaert, Blandine, Grosset, Marine, Le Moal, Gwenaël, Béraud, Guillaume, Martellosio, Jean Philippe, Iriart, Xavier, Cadranel, Jacques, Médecine Interne et Maladies Infectieuses, CHU de Poitiers, Poitiers, France, Centre hospitalier universitaire de Poitiers (CHU Poitiers), Université de Poitiers - Faculté de Médecine et de Pharmacie, Université de Poitiers, Laboratoire de Parasitologie-Mycologie (CHU de Poitiers), Pharmacologie des anti-infectieux (PHAR), Université de Poitiers-Institut National de la Santé et de la Recherche Médicale (INSERM), Evaluation des technologies de santé et des pratiques médicales - ULR 2694 (METRICS), Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille), Hasselt University (UHasselt), CHU Toulouse [Toulouse], Centre de Physiopathologie Toulouse Purpan (CPTP), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Service de Pneumologie - Oncologie Thoracique - Maladies Pulmonaires Rares [CHU Tenon], CHU Tenon [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Sorbonne Université (SU), Univ Hosp Poitiers, Dept Infect Dis, Service des Maladies Infectieuses, Centre hospitalier universitaire de Poitiers (CHU Poitiers)-Hôpital de La Milétrie, Microbiologie de l'Eau (MDE), Ecologie et biologie des interactions (EBI), Université de Poitiers-Centre National de la Recherche Scientifique (CNRS)-Université de Poitiers-Centre National de la Recherche Scientifique (CNRS), Université de Poitiers-Centre National de la Recherche Scientifique (CNRS), Institut Pasteur du Cambodge, Réseau International des Instituts Pasteur (RIIP), CMES, Laboratoire Microorganismes : Génome et Environnement (LMGE), Université Blaise Pascal - Clermont-Ferrand 2 (UBP)-Université d'Auvergne - Clermont-Ferrand I (UdA)-Centre National de la Recherche Scientifique (CNRS)-Université Blaise Pascal - Clermont-Ferrand 2 (UBP)-Université d'Auvergne - Clermont-Ferrand I (UdA)-Centre National de la Recherche Scientifique (CNRS), Santé publique : épidémiologie et qualité des soins-EA 2694 (CERIM), Centre de Physiopathologie Toulouse Purpan ex IFR 30 et IFR 150 (CPTP), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-Centre National de la Recherche Scientifique (CNRS), Theranoscan, Université Pierre et Marie Curie - Paris 6 (UPMC), Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), and Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)
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0301 basic medicine ,Adult ,Male ,medicine.medical_specialty ,Antifungal Agents ,Veterinary (miscellaneous) ,medicine.medical_treatment ,[SDV]Life Sciences [q-bio] ,030106 microbiology ,Hormographiella aspergillata ,Hematopoietic stem cell transplantation ,Applied Microbiology and Biotechnology ,Microbiology ,03 medical and health sciences ,0302 clinical medicine ,Medical microbiology ,Refractory ,[SDV.MHEP.MI]Life Sciences [q-bio]/Human health and pathology/Infectious diseases ,Amphotericin B ,Administration, Inhalation ,Medicine ,Humans ,Transplantation, Homologous ,030212 general & internal medicine ,Lung ,Aerosols ,Lung Diseases, Fungal ,business.industry ,Mortality rate ,Standard treatment ,Hematopoietic Stem Cell Transplantation ,[SDV.SP]Life Sciences [q-bio]/Pharmaceutical sciences ,3. Good health ,medicine.anatomical_structure ,[SDV.MP]Life Sciences [q-bio]/Microbiology and Parasitology ,Treatment Outcome ,Anesthesia ,Administration, Intravenous ,Nebulized liposomal amphotericin B ,business ,Complication ,Agaricales ,Agronomy and Crop Science - Abstract
International audience; Invasive fungal infection is a serious complication following allogeneic hematopoietic stem cell transplantation. Pulmonary infection due to Hormographiella aspergillata is an uncommon condition associated with a high mortality rate. The susceptibility of H. aspergillata to available antifungal agents is not well established. We report for the first time a case of H. aspergillata lung infection that responded poorly to conventional treatment with liposomal amphotericin B (LAmB; 3 mg kg-1 of body weight per day) with renal damage at higher posology (5 mg kg-1 of body weight per day), but improved rapidly after addition of nebulized LAmB to intravenous LAmB (3 mg kg-1 of body weight per day). Successful treatment of our patient using nebulized LAmB would be worth evaluating in cases refractory to standard treatment or when the reference treatment may not be extended due to interaction or side effects.
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- 2016
20. Multicentric Hyaline-Vascular Castleman Disease: The Missing Link?
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Beydon, Maxime, Dieudonné, Yannick, Meignin, Veronique, Kaphan, Eléonore, Terriou, Louis, Martellosio, Jean Philippe, Viallard, Jean-Francois, Ballot-Schmit, Claire, Dourthe, Marie Emilie, Mallebranche, Coralie, Meyran, Deborah, Maisonobe, Lucas, Galicier, Lionel, Oksenhendler, Eric, and Boutboul, David
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Introduction:Castleman disease (CD) is a rare lymphoproliferative disorder that encompasses distinct clinicopathological entities. Unicentric form of the disease (UCD) usually involves a single lymph node station, exhibits hyaline vascular (HV) pathological changes with follicular dendritic cell expansion, and may associate with specific and life-threatening complications including paraneoplastic pemphigus (PNP) and follicular dendritic cell sarcoma (FDCS). Plasma cell interfollicular infiltration is absent in the HV subtype as opposed to the Plasma-Cell (PC) or Mixed subtypes. PC subtype has been associated with inflammatory symptoms thought to be secondary to plasma cell infiltration and represents the majority of HHV8-negative “idiopathic” multicentric forms of the disease. Interestingly, patients with PC-UCD behave as PC-MCD, suggesting a stronger role of histology rather than Unicentric vs Multicentric phenotype in disease expression. We here studied the phenotype of HV-MCD, a rare subtype of CD characterized by hyaline-vascular pathological changes involving 2 or more lymph node stations.
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- 2023
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21. Serious Infections in Patients with Vexas Syndrome: A Study from the French Vexas Group
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De Valence de Minardière, Benjamin, Delaune, Marion, Nguyen, Yann, Jachiet, Vincent, Heiblig, Mael, Jean, Alexis, Rieschert, Stanislas, Henneton, Pierrick, Guilpain, Philippe, Lobbes, Hervé, Le Guenno, Guillaume, Schleinitz, Nicolas, Lacombe, Valentin, Langlois, Vincent, Outh, Roderau, Decker, Paul, Moulinet, Thomas, Dieudonné, Yannick, Bigot, Adrien, Vlakos, Alexandre, Dion, Jeremie, Denis, Guillaume, Broner, Jonahan, Kostine, Marie, Humbert, Sebastien, Vinit, Julien, Martellosio, Jean Philippe, Terriou, Louis, De Maleprade, Baptiste, Fenaux, Pierre, Terrier, Benjamin, Georgin-Lavialle, Sophie, Fain, Olivier, Mekinian, Arsene, Morgand, Marjolaine, Hadjadj, Jerome, and Comont, Thibault
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Background
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- 2023
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22. Efficacy and safety of targeted therapies in VEXAS syndrome: retrospective study from the FRENVEX.
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Hadjadj J, Nguyen Y, Mouloudj D, Bourguiba R, Heiblig M, Aloui H, McAvoy C, Lacombe V, Ardois S, Campochiaro C, Maria A, Coustal C, Comont T, Lazaro E, Lifermann F, Le Guenno G, Lobbes H, Grobost V, Outh R, Campagne J, Dor-Etienne A, Garnier A, Jamilloux Y, Dossier A, Samson M, Audia S, Nicolas B, Mathian A, de Maleprade B, De Sainte-Marie B, Faucher B, Bouaziz JD, Broner J, Dumain C, Antoine C, Carpentier B, Castel B, Lartigau-Roussin C, Crickx E, Volle G, Fayard D, Decker P, Moulinet T, Dumont A, Nguyen A, Aouba A, Martellosio JP, Levavasseur M, Puigrenier S, Antoine P, Giraud JT, Hermine O, Lacout C, Martis N, Karam JD, Chasset F, Arnaud L, Marianetti P, Deligny C, Chazal T, Woaye-Hune P, Roux-Sauvat M, Meyer A, Sujobert P, Hirsch P, Abisror N, Fenaux P, Kosmider O, Jachiet V, Fain O, Terrier B, Mekinian A, and Georgin-Lavialle S
- Abstract
Objectives: Vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic (VEXAS) syndrome is an adult-onset autoinflammatory disease associated with somatic ubiquitin-like modifier-activating enzyme 1 (UBA1) mutations. We aimed to evaluate the efficacy and safety of targeted therapies., Methods: Multicentre retrospective study including patients with genetically proven VEXAS syndrome who had received at least one targeted therapy. Complete response (CR) was defined by a clinical remission, C-reactive protein (CRP) ≤10 mg/L and a ≤10 mg/day of prednisone-equivalent therapy, and partial response (PR) was defined by a clinical remission and a 50% reduction in CRP levels and glucocorticoid dose., Results: 110 patients (median age 71 (68-79) years) who received 194 targeted therapies were included: 78 (40%) received Janus kinase (JAK) inhibitors (JAKi), 51 (26%) interleukin (IL)-6 inhibitors, 33 (17%) IL-1 inhibitors, 20 (10%) tumour necrosis factor (TNFα) blockers and 12 (6%) other targeted therapies. At 3 months, the overall response (CR and PR) rate was 24% with JAKi, 32% with IL-6 inhibitors, 9% with anti-IL-1 and 0% with TNFα blockers or other targeted therapies. At 6 months, the overall response rate was 30% with JAKi and 26% with IL-6 inhibitors. Survival without treatment discontinuation was significantly longer with JAKi than with the other targeted therapies. Among patients who discontinued treatment, causes were primary failure, secondary failure, serious adverse event or death in 43%, 14%, 19% and 19%, respectively, with JAKi and 46%, 11%, 31% and 9%, respectively, with IL-6 inhibitors., Conclusions: This study shows the benefit of JAKi and IL-6 inhibitors, whereas other therapies have lower efficacy. These results need to be confirmed in prospective trials., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ on behalf of EULAR.)
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- 2024
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