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51. Noninvasive respiratory support outside the intensive care unit for acute respiratory failure related to coronavirus-19 disease: a systematic review and meta-analysis

Author

Cammarota, Gianmaria, Esposito, Teresa, Azzolina, Danila, Cosentini, Roberto, Menzella, Francesco, Aliberti, Stefano, Coppadoro, Andrea, Bellani, Giacomo, Foti, Giuseppe, Grasselli, Giacomo, Cecconi, Maurizio, Pesenti, Antonio, Vitacca, Michele, Lawton, Tom, Ranieri, V. Marco, Di Domenico, Sandro Luigi, Resta, Onofrio, Gidaro, Antonio, Potalivo, Antonella, Nardi, Giuseppe, Brusasco, Claudia, Tesoro, Simonetta, Navalesi, Paolo, Vaschetto, Rosanna, and De Robertis, Edoardo

Published
2021
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55. The administration of methotrexate in patients with Still’s disease, “real-life” findings from AIDA Network Still Disease Registry

Author

362., Ruscitti P, Sota, J, Vitale, A, Lopalco, G, Iannone, F, Morrone, M, Giardini, Ham, D'Agostin, Ma, Antonelli, Ipb, Almaghlouth, I, Asfina, Kn, Khalil, N, Sfikakis, Pp, Laskari, K, Tektonidou, M, Ciccia, F, Iacono, D, Riccio, F, Ragab, G, Hussein, Ma, Govoni, M, Ruffilli, F, Direskeneli, H, Alibaz-Oner, F, Giacomelli, R, Navarini, L, Bartoloni, E, Riccucci, I, Martín-Nares, E, Torres-Ruiz, J, Cipriani, P, Di Cola, I, Hernández-Rodríguez, J, Gómez-Caverzaschi, V, Dagna, L, Tomelleri, A, Makowska, J, Brzezinska, O, Iagnocco, A, Bellis, E, Caggiano, V, Gaggiano, C, Tarsia, M, Mormile, I, Emmi, G, Sfriso, P, Monti, S, Erten, Ş, Del Giudice, E, Lubrano, R, Conti, G, Olivieri, An, Lo Gullo, A, Tharwat, S, Karamanakos, A, Gidaro, A, Maggio, Mc, La Torre, F, Cardinale, F, Ogunjimi, B, Maier, A, Sebastiani, Gd, Opris-Belinski, D, Frassi, M, Viapiana, O, Bizzi, E, Carubbi, F, Fotis, L, Tufan, A, Kardas, Rc, Więsik-Szewczyk, E, Jahnz-Różyk, K, Fabiani, C, Frediani, B, Balistreri, A, Rigante, Donato, Cantarini, L, 362. Ruscitti P, Sota J, Vitale A, Lopalco G, Iannone F, Morrone M, Giardini HAM, D'Agostin MA, Antonelli IPB, Almaghlouth I, Asfina KN, Khalil N, Sfikakis PP, Laskari K, Tektonidou M, Ciccia F, Iacono D, Riccio F, Ragab G, Hussein MA, Govoni M, Ruffilli F, Direskeneli H, Alibaz-Oner F, Giacomelli R, Navarini L, Bartoloni E, Riccucci I, Martín-Nares E, Torres-Ruiz J, Cipriani P, Di Cola I, Hernández-Rodríguez J, Gómez-Caverzaschi V, Dagna L, Tomelleri A, Makowska J, Brzezinska O, Iagnocco A, Bellis E, Caggiano V, Gaggiano C, Tarsia M, Mormile I, Emmi G, Sfriso P, Monti S, Erten Ş, Del Giudice E, Lubrano R, Conti G, Olivieri AN, Lo Gullo A, Tharwat S, Karamanakos A, Gidaro A, Maggio MC, La Torre F, Cardinale F, Ogunjimi B, Maier A, Sebastiani GD, Opris-Belinski D, Frassi M, Viapiana O, Bizzi E, Carubbi F, Fotis L, Tufan A, Kardas RC, Więsik-Szewczyk E, Jahnz-Różyk K, Fabiani C, Frediani B, Balistreri A, Rigante D (ORCID:0000-0001-7032-7779), Cantarini L, 362., Ruscitti P, Sota, J, Vitale, A, Lopalco, G, Iannone, F, Morrone, M, Giardini, Ham, D'Agostin, Ma, Antonelli, Ipb, Almaghlouth, I, Asfina, Kn, Khalil, N, Sfikakis, Pp, Laskari, K, Tektonidou, M, Ciccia, F, Iacono, D, Riccio, F, Ragab, G, Hussein, Ma, Govoni, M, Ruffilli, F, Direskeneli, H, Alibaz-Oner, F, Giacomelli, R, Navarini, L, Bartoloni, E, Riccucci, I, Martín-Nares, E, Torres-Ruiz, J, Cipriani, P, Di Cola, I, Hernández-Rodríguez, J, Gómez-Caverzaschi, V, Dagna, L, Tomelleri, A, Makowska, J, Brzezinska, O, Iagnocco, A, Bellis, E, Caggiano, V, Gaggiano, C, Tarsia, M, Mormile, I, Emmi, G, Sfriso, P, Monti, S, Erten, Ş, Del Giudice, E, Lubrano, R, Conti, G, Olivieri, An, Lo Gullo, A, Tharwat, S, Karamanakos, A, Gidaro, A, Maggio, Mc, La Torre, F, Cardinale, F, Ogunjimi, B, Maier, A, Sebastiani, Gd, Opris-Belinski, D, Frassi, M, Viapiana, O, Bizzi, E, Carubbi, F, Fotis, L, Tufan, A, Kardas, Rc, Więsik-Szewczyk, E, Jahnz-Różyk, K, Fabiani, C, Frediani, B, Balistreri, A, Rigante, Donato, Cantarini, L, 362. Ruscitti P, Sota J, Vitale A, Lopalco G, Iannone F, Morrone M, Giardini HAM, D'Agostin MA, Antonelli IPB, Almaghlouth I, Asfina KN, Khalil N, Sfikakis PP, Laskari K, Tektonidou M, Ciccia F, Iacono D, Riccio F, Ragab G, Hussein MA, Govoni M, Ruffilli F, Direskeneli H, Alibaz-Oner F, Giacomelli R, Navarini L, Bartoloni E, Riccucci I, Martín-Nares E, Torres-Ruiz J, Cipriani P, Di Cola I, Hernández-Rodríguez J, Gómez-Caverzaschi V, Dagna L, Tomelleri A, Makowska J, Brzezinska O, Iagnocco A, Bellis E, Caggiano V, Gaggiano C, Tarsia M, Mormile I, Emmi G, Sfriso P, Monti S, Erten Ş, Del Giudice E, Lubrano R, Conti G, Olivieri AN, Lo Gullo A, Tharwat S, Karamanakos A, Gidaro A, Maggio MC, La Torre F, Cardinale F, Ogunjimi B, Maier A, Sebastiani GD, Opris-Belinski D, Frassi M, Viapiana O, Bizzi E, Carubbi F, Fotis L, Tufan A, Kardas RC, Więsik-Szewczyk E, Jahnz-Różyk K, Fabiani C, Frediani B, Balistreri A, Rigante D (ORCID:0000-0001-7032-7779), and Cantarini L

Abstract

Objectives: To describe clinical characteristics of patients with Still's disease treated with methotrexate (MTX) and to assess drug effectiveness evaluating change in disease activity, reduction of inflammatory markers, and glucocorticoid (GC)-sparing effect. Methods: Patients with Still's disease treated with MTX were assessed among those included in AIDA Network Still Disease Registry. Results: In this registry, 171 patients with Still's disease were treated with MTX (males 43.3%, age 37.1 ± 16.0 years). They were mainly characterised by joint features and fever without a prominent multiorgan involvement. MTX was administered with GCs in 68.4% of patients, with other conventional synthetic DMARDs in 6.4%, and with biologic DMARDs in 25.1%. A significant reduction of the modified systemic score was observed, and 38.6% patients were codified as being in clinical remission at the end of follow-up. The concomitant administration of a biologic DMARD resulted a predictor of the clinical remission. Furthermore, a reduction of inflammatory markers and ferritin levels was observed following the administration of MTX. Additionally, a marked reduction of the dosage of concomitant GCs was identified, while 36.7% discontinued such drugs. Male gender appeared as a predictor of GC discontinuation. MTX was discontinued in 12.3% of patients because of adverse effects, and in 12.3% for lack of efficacy. Conclusions: Clinical characteristics of patients with Still's disease treated with MTX were described, mainly joint features and fever without a prominent multiorgan involvement. The clinical usefulness of MTX was reported in reducing the disease activity, decreasing the inflammatory markers, and as GC-sparing agent.

Published
2023

56. Can the length of a catheter change the time to bubble at the tip performing the 'Bubble Test'? A bench study

Author

Giustivi, D, Elli, S, Airoldi, C, Lo Izzo, F, Rossini, M, Gidaro, A, Lucchini, A, Privitera, D, Giustivi, Davide, Elli, Stefano, Airoldi, Chiara, Lo Izzo, Federica, Rossini, Michela, Gidaro, Antonio, Lucchini, Alberto, Privitera, Daniele, Giustivi, D, Elli, S, Airoldi, C, Lo Izzo, F, Rossini, M, Gidaro, A, Lucchini, A, Privitera, D, Giustivi, Davide, Elli, Stefano, Airoldi, Chiara, Lo Izzo, Federica, Rossini, Michela, Gidaro, Antonio, Lucchini, Alberto, and Privitera, Daniele

Abstract

Introduction: Intraprocedural tip control techniques are critical during central venous catheter placement. According to international guidelines (INS 2021), intracavitary electrocardiography is the first method of choice to verify it; when this technique is not feasible, it is considered acceptable to use a contrast-enhanced ultrasound-based tip location method, commonly known as "bubble-test" as an effective alternative.Objective: To assess whether the length of the vascular catheter can alter the time between the injection of the contrast media and its appearance at the catheter tip and the injection duration. Differences between operators stratified according to experience were evaluated as secondary endpoints.Methods: A bench study was conducted using an extracorporeal circuit. For each catheter length (60, 40, and 20 cm), three injections were obtained by each of the five operators with different levels of experience for a total of 45 measurements. Differences among operators were evaluated using ANOVA, and the impact of catheter length and operator expertise on times was assessed using repeated measurement models.Results: Hub-to-tip times of 247.33 ms (SD 168.82), 166 ms (SD 95.46), 138 ms (SD 54.48), and injection duration of 1620 ms (SD 748.58), 1614 ms (SD 570.95), 1566 ms (SD 302.83) were observed for 60, 40, 20 cm catheter length, respectively. A significant time variability between operators was observed. Moreover, moving from 60 to 20 cm, hubto-tip time was significantly longer for 60 cm devices (p = 0.0124), while little differences were observed for injection duration.Conclusions: Catheter length can change both the time between the injection of the contrast media and its appearance at the catheter tip and the injection duration. Hub-to-tip times obtained with 20 and 40 cm and overall injection duration did not differ significantly; skilled personnel could substantially reduce both values analyzed in this study.

Published
2023

59. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Author

E. Mercuri, A.M. Seferian, L. Servais, N. Deconinck, H. Stevenson, X. Ni, W. Zhang, L. East, S. Yonren, F. Muntoni, Nicolas Deconinck, Rudy Van Coster, Arnaud Vanlander, Andreea Seferian, Silvana De Lucia, Teresa Gidaro, Laura Vanden Brande, Laurent Servais, Janbernd Kirschner, Sabine Borell, Eugenio Mercuri, Claudia Brogna, Marika Pane, Lavinia Fanelli, Giulia Norcia, Francesco Muntoni, Chiara Brusa, Mary Chesshyre, Kate Maresh, Jaqueline Pitchforth, Lucia Schottlaender, Mariacristina Scoto, Arpana Silwal, and Fedrica Trucco

Subjects
Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)
Published
2023

61. A Diagnostic of Acquired Hemophilia Following PD1/PDL1 Inhibitors in Advanced Melanoma: The Experience of Two Patients and a Literature Review

Author

Antonio Gidaro, Giuseppe Palmieri, Mattia Donadoni, Lucia A. Mameli, Leyla La Cava, Giuseppe Sanna, Dante Castro, Alessandro P. Delitala, Roberto Manetti, and Roberto Castelli

Subjects
acquired hemophilia A (AHA), immune checkpoint inhibitors (ICIs), nivolumab, pembrolizumab, melanoma, Novoseven®, Medicine (General), R5-920
Abstract

Acquired hemophilia A (AHA) is a rare bleeding disorder caused by the development of specific autoantibodies against factor VIII (FVIII). Immunotherapy is a recent therapeutic option that targets the patient’s self-tolerance against tumor cells. Because therapeutic effects of the immune checkpoint inhibitors (ICIs) are mediated by enhancing the immune response to restore antitumor immunity, autoimmune-related adverse effects can be seen in up to 80% of patients during treatment and after treatment. A rare hematologic ICIs-related adverse event is AHA. Hereafter we report two cases of AHA developed during anti-PD-1 immunotherapy for advanced melanoma: one secondary to treatment with nivolumab and one secondary to pembrolizumab. Both patients were treated with activated FVII (Novoseven®, Novo Nordisk, Bagsværd, Denmark) as hemostatic treatment combined with the eradication of antibodies anti-FVIII obtained with rituximab. In the last few years these drugs have significantly improved the therapeutic armamentarium for the management of AHA. Indeed, while FVIIa has proven to be an effective and safe tool for the treatment of acute bleeding related to FVIII autoantibodies, rituximab is a promising alternative for the autoantibodies’ elimination and the restoration of normal hemostasis. Our finding supports the use of this combination even in AHA secondary to ICIs treatment.

Published
2022
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62. Immunological Profiles in Parry–Romberg Syndrome: A Case–Control Study.

Author

Saulle, Irma, Gidaro, Antonio, Donadoni, Mattia, Vanetti, Claudia, Mutti, Alessandra, Romano, Maria Eva, Clerici, Mario, Cogliati, Chiara, and Biasin, Mara

Subjects
*MONONUCLEAR leukocytes, *T cells, *B cells, *CASE-control method, *T helper cells, *SCLERODERMA (Disease)
Abstract

Background: Parry–Romberg syndrome (PRS) is a rare craniofacial disorder. The aim of this study is to provide information on the immunological profile of this pathology. Since PRS can be included in a wider spectrum of sclerodermic diseases, we propose a case–control study comparing a patient affected by PRS with one with a diagnosis of scleroderma, herein used as control (CTR). Methods: B lymphocyte, T lymphocyte, and monocyte phenotypes and functions were assessed by flow cytometry in influenza (Flu)- or anti cluster differentiation (CD)3/CD28-stimulated peripheral blood mononuclear cells (PBMCs). Cytokine concentration was evaluated as well in PBMC supernatants, plasma, and saliva by Luminex assay. Results: T and B lymphocytes were similarly activated in unstimulated PRS and CTR cells but differed following antigen stimulation. T helper (Th)17 lymphocytes were expanded in PRS compared to CTR; this increase correlated with higher interleukin (IL)-17 concentration. Conclusions: Our case–control study is the first to compare the immunological profiles of PRS and scleroderma patients. The higher percentage of Th17 cells in PRS suggests the use of anti-IL17 receptor monoclonal antibody in this rare disease; however, further studies with larger numbers of patients are needed to confirm our findings. [ABSTRACT FROM AUTHOR]

Published
2024
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64. Clinical and genetic features of patients suffering from CMT4J

Author

Beloribi-Djefaflia, Sadia, primary, Morales, Raul Juntas, additional, Fatehi, Farzad, additional, Isapof, Arnaud, additional, Servais, Laurent, additional, Leonard-Louis, Sarah, additional, Michaud, Maud, additional, Verdure, Pierre, additional, Gidaro, Teresa, additional, Pouget, Jean, additional, Poinsignon, Vianney, additional, Bonello-Palot, Nathalie, additional, and Attarian, Shahram, additional

Published
2023
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65. Derivation and validation of four patient clusters in Still’s disease, results from GIRRCS AOSD-study group and AIDA Network Still Disease Registry

Author

Ruscitti, Piero, primary, Masedu, Francesco, additional, Vitale, Antonio, additional, Di Cola, Ilenia, additional, Caggiano, Valeria, additional, Di Muzio, Claudia, additional, Cipriani, Paola, additional, Valenti, Marco, additional, Berardicurti, Onorina, additional, Navarini, Luca, additional, Iacono, Daniela, additional, Pantano, Ilenia, additional, Mauro, Daniele, additional, Ciccia, Francesco, additional, Rossi, Silvia, additional, De Stefano, Ludovico, additional, Monti, Sara, additional, Bugatti, Serena, additional, Montecucco, Carlomaurizio, additional, Caso, Francesco, additional, Costa, Luisa, additional, Prete, Marcella, additional, Perosa, Federico, additional, Iagnocco, Annamaria, additional, Atzeni, Fabiola, additional, Guggino, Giuliana, additional, Giardini, Henrique, additional, Antonelli, Isabele Parente de Brito, additional, Almaghlouth, Ibrahim A, additional, Asfina, Kazi, additional, Direskeneli, Haner, additional, Alibaz-Oner, Fatma, additional, Sevik, Gizem, additional, Tufan, Abdurrahman, additional, Sfikakis, Petros P, additional, La Torre, Francesco, additional, Hinojosa-Azaola, Andrea, additional, Martín-Nares, Eduardo, additional, Torres-Ruiz, Jiram, additional, Ragab, Gafaar, additional, Maggio, Maria Cristina, additional, Makowska, Joanna, additional, Del Giudice, Emanuela, additional, Bartoloni, Elena, additional, Emmi, Giacomo, additional, Govoni, Marcello, additional, Lo Gullo, Alberto, additional, Lopalco, Giuseppe, additional, Simonini, Gabriele, additional, Fotis, Lampros, additional, Ogunjimi, Benson, additional, Tharwat, Samar, additional, Frediani, Bruno, additional, Maier, Armin, additional, Carubbi, Francesco, additional, Dagna, Lorenzo, additional, Erten, Sukran, additional, Gidaro, Antonio, additional, Hernández-Rodríguez, José, additional, Sfriso, Paolo, additional, Fabiani, Claudia, additional, Giacomelli, Roberto, additional, and Cantarini, Luca, additional

Published
2023
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68. Physicochemical Characteristics of Antimicrobials and Practical Recommendations for Intravenous Administration: A Systematic Review

Author

Borgonovo, Fabio, primary, Quici, Massimiliano, additional, Gidaro, Antonio, additional, Giustivi, Davide, additional, Cattaneo, Dario, additional, Gervasoni, Cristina, additional, Calloni, Maria, additional, Martini, Elena, additional, La Cava, Leyla, additional, Antinori, Spinello, additional, Cogliati, Chiara, additional, Gori, Andrea, additional, and Foschi, Antonella, additional

Published
2023
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71. Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy

Author

Bernert, G., Knipp, F., Buyse, G.M., Goemans, N., Van den Hauwe, M., Voit, T., Doppler, V., Gidaro, T., Cuisset, J.-M., Coopman, S., Schara, U., Lutz, S., Kirschner, J., Borell, S., Will, M., D'Angelo, M.G., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D'Ambrosio, P., Taglia, A., Verschuuren, J.J.G.M., Straathof, C.S.M., Vílchez Padilla, J.J., Muelas Gómez, N., Sejersen, T., Hovmöller, M., Jeannet, P.-Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Finkel, R., Bönnemann, C., McDonald, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R.C., Meier, Thomas, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, Mayer, Oscar H., and Buyse, Gunnar M.

Published
2017
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72. Pathogenic DPAGT1 variants in limb‐girdle congenital myasthenic syndrome (LG‐CMS) associated with tubular aggregates and ORAI1 hypoglycosylation.

Author

vanden Brande, Laura, Bauché, Stéphanie, Pérez‐Guàrdia, Laura, Sternberg, Damien, Seferian, Andreea M., Malfatti, Edoardo, Silva‐Rojas, Roberto, Labasse, Clémence, Chevessier, Frédéric, Carlier, Pierre, Eymard, Bruno, Romero, Norma B., Laporte, Jocelyn, Servais, Laurent, Gidaro, Teresa, and Böhm, Johann

Subjects
CONGENITAL myasthenic syndromes, CALCIUM channels, RYANODINE receptors, MUSCLE weakness, POST-translational modification, NEUROMUSCULAR transmission, CHOLINERGIC receptors, HYBRID rice
Abstract

Aims: Limb‐girdle congenital myasthenic syndrome (LG‐CMS) is a genetically heterogeneous disorder characterised by muscle weakness and fatigability. The LG‐CMS gene DPAGT1 codes for an essential enzyme of the glycosylation pathway, a posttranslational modification mechanism shaping the structure and function of proteins. In DPAGT1‐related LG‐CMS, decreased glycosylation of the acetylcholine receptor (AChR) reduces its localization at the neuromuscular junction (NMJ) and results in diminished neuromuscular transmission. LG‐CMS patients also show tubular aggregates on muscle biopsy, but the origin and potential contribution of the aggregates to disease development are not understood. Here, we describe two LG‐CMS patients with the aim of providing a molecular diagnosis and to shed light on the pathways implicated in tubular aggregate formation. Methods: Following clinical examination of the patients, we performed next‐generation sequencing (NGS) to identify the genetic causes, analysed the biopsies at the histological and ultrastructural levels, investigated the composition of the tubular aggregates and performed experiments on protein glycosylation. Results: We identified novel pathogenic DPAGT1 variants in both patients and pyridostigmine treatment quantitatively improved muscle force and function. The tubular aggregates contained proteins of the sarcoplasmic reticulum (SR) and structurally conformed to the aggregates observed in tubular aggregate myopathy (TAM). TAM arises from overactivation of the plasma membrane calcium channel ORAI1, and functional studies on muscle extracts from our LG‐CMS patients evidenced abnormal ORAI1 glycosylation. Conclusions: We expand the genetic variant spectrum of LG‐CMS and provide a genotype/phenotype correlation for pathogenic DPAGT1 variants. The discovery of ORAI1 hypoglycosylation in our patients highlights a physiopathological link between LG‐CMS and TAM. [ABSTRACT FROM AUTHOR]

Published
2024
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74. X-linked myotubular myopathy: A prospective international natural history study

Author

Annoussamy, Mélanie, Lilien, Charlotte, Gidaro, Teresa, Gargaun, Elena, Chê, Virginie, Schara, Ulrike, Gangfuß, Andrea, DʼAmico, Adele, Dowling, James J., Darras, Basil T., Daron, Aurore, Hernandez, Arturo, de Lattre, Capucine, Arnal, Jean-Michel, Mayer, Michèle, Cuisset, Jean-Marie, Vuillerot, Carole, Fontaine, Stéphanie, Bellance, Rémi, Biancalana, Valérie, Buj-Bello, Ana, Hogrel, Jean-Yves., Landy, Hal, and Servais, Laurent

Published
2019
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75. Use and Prescription of Direct Oral Anticoagulants in Older and Frail Patients with Atrial Fibrillation: A Multidisciplinary Consensus Document

Author

Proietti, M, Camera, M, Gallieni, M, Gianturco, L, Gidaro, A, Piemontese, C, Pizzetti, G, Redaelli, F, Scimeca, B, Tadeo, C, Cesari, M, Bellelli, G, Dalla Vecchia, L, Proietti, Marco, Camera, Marina, Gallieni, Maurizio, Gianturco, Luigi, Gidaro, Antonio, Piemontese, Carlo, Pizzetti, Giuseppe, Redaelli, Franco, Scimeca, Barbara, Tadeo, Carlo Sebastiano, Cesari, Matteo, Bellelli, Giuseppe, Dalla Vecchia, Laura Adelaide, Proietti, M, Camera, M, Gallieni, M, Gianturco, L, Gidaro, A, Piemontese, C, Pizzetti, G, Redaelli, F, Scimeca, B, Tadeo, C, Cesari, M, Bellelli, G, Dalla Vecchia, L, Proietti, Marco, Camera, Marina, Gallieni, Maurizio, Gianturco, Luigi, Gidaro, Antonio, Piemontese, Carlo, Pizzetti, Giuseppe, Redaelli, Franco, Scimeca, Barbara, Tadeo, Carlo Sebastiano, Cesari, Matteo, Bellelli, Giuseppe, and Dalla Vecchia, Laura Adelaide

Abstract

In the last twelve years the clinical management of patients with atrial fibrillation has been revolutionised by the introduction of direct oral anticoagulants. Despite the large amount of evidence produced, some populations remain relatively poorly explored regarding the effectiveness and safety of direct oral anticoagulants, such as the oldest and/or frailest individuals. Frailty is clinical syndrome characterized by a reduction of functions and physiological reserves which results in individuals having higher vulnerability. While current evidence underlines a relationship between atrial fibrillation and frailty, particularly in determining a higher risk of adverse outcomes, data regarding effectiveness and safety of direct oral anticoagulants in frailty atrial fibrillation patients are still lacking, leaving uncertainty about how to guide prescription in this specific subgroup. On these premises, this multidisciplinary consensus document explains why it would be useful to integrate the clinical evaluation performed through comprehensive geriatric assessment to gather further elements to guide prescription of direct oral anticoagulants in such a high-risk group of patients.

Published
2022

76. Retrospective survey from vascular access team Lombardy net in COVID-19 era

Author

Gidaro, A, Vailati, D, Gemma, M, Lugli, F, Casella, F, Cogliati, C, Canelli, A, Nadia, C, Monolo, D, Cordio, G, Frosi, C, Destefanis, R, Rossi, A, Alemanno, M, Valenza, F, Luisoni, M, Elli, S, Caldarini, A, Lucchini, A, Paglia, S, Baroni, M, Giustivi, D, Antonio, Gidaro, Davide, Vailati, Marco, Gemma, Francesca, Lugli, Francesco, Casella, Chiara, Cogliati, Antonio, Canelli, Cremonesi, Nadia, Davide, Monolo, Giuseppe, Cordio, Chiara, Frosi, Riccardo, Destefanis, Anna, Rossi, Maria Chiara, Alemanno, Franco, Valenza, Mara Dina, Luisoni, Stefano, Elli, Andrea, Caldarini, Alberto, Lucchini, Stefano, Paglia, Monica, Baroni, Davide, Giustivi, Gidaro, A, Vailati, D, Gemma, M, Lugli, F, Casella, F, Cogliati, C, Canelli, A, Nadia, C, Monolo, D, Cordio, G, Frosi, C, Destefanis, R, Rossi, A, Alemanno, M, Valenza, F, Luisoni, M, Elli, S, Caldarini, A, Lucchini, A, Paglia, S, Baroni, M, Giustivi, D, Antonio, Gidaro, Davide, Vailati, Marco, Gemma, Francesca, Lugli, Francesco, Casella, Chiara, Cogliati, Antonio, Canelli, Cremonesi, Nadia, Davide, Monolo, Giuseppe, Cordio, Chiara, Frosi, Riccardo, Destefanis, Anna, Rossi, Maria Chiara, Alemanno, Franco, Valenza, Mara Dina, Luisoni, Stefano, Elli, Andrea, Caldarini, Alberto, Lucchini, Stefano, Paglia, Monica, Baroni, and Davide, Giustivi

Abstract

BACKGROUND: Venous Access Devices (VADs) are the most used devices in COVID-19 patients.OBJECTIVE: Identify VADs implanted, catheter related thrombosis (CRT), catheter-related bloodstream infection (CRBSI), and accidental remove of VADs in both COVID-19 positive and COVID-19 free patients. Successive analysis was conducted comparing COVID-19 positive patients with COVID-19 free with inverse probability propensity score weights using simple regression to account for these two confounders (peripheral tip as central/peripheral and hospitalization as no/yes).METHODS: This multicenter, retrospective cohort study collected data from seven hospitals in Lombardy during the pandemic period from February 21st to May 31st 2020.RESULTS: A total of 2206 VADs were evaluated, 1107 (50.2%) of which were inserted in COVID-19 patients. In COVID-19 cohort the first choice was Long Peripheral Cannula in 388 patients (35.1%) followed by Midline Catheter in 385 (34.8%). The number of "central tip" VADs inserted in COVID-free inpatients and COVID-19 positive were similar (307 vs 334). We recorded 42 (1.9%) CRT; 32 (79.2%) were observed in COVID-19 patients. A total of 19 CRBSI were diagnosed; 15 (78.95%) were observed in COVID-19. Accidental removals were the more represented complication with 123 cases, 85 (69.1%) of them were in COVID-19. COVID-19 significantly predicted occurrence of CRT (OR=2.00(1.85-5.03); p<0.001), CRSB (OR=3.82(1.82-8.97); p<0.001), and Accidental Removal (OR=2.39(1.80-3.20); p<0.001) in our propensity score weighted models.CONCLUSIONS: CRT, CRBSI, and accidental removal are significantly more frequent in COVID-19 patients. Accidental removals are the principal complication, for this reason, the use of subcutaneously anchored securement is recommended for a shorter period than usual.

Published
2022

78. JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (P7-9.004)

Author

Chiriboga, Claudia, primary, Bruno, Claudio, additional, Duong, Tina, additional, Fischer, Dirk, additional, Kirschner, Janbernd, additional, Scoto, Mariacristina, additional, Mercuri, Eugenio, additional, Gerber, Marianne, additional, Gorni, Ksenija, additional, Kletzl, Heidemarie, additional, Carruthers, Imogen, additional, Martin, Carmen, additional, Gidaro, Teresa, additional, and Muntoni, Francesco, additional

Published
2023
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79. Aging of the Arterial System

Author

Castelli, Roberto, primary, Gidaro, Antonio, additional, Casu, Gavino, additional, Merella, Pierluigi, additional, Profili, Nicia I., additional, Donadoni, Mattia, additional, Maioli, Margherita, additional, and Delitala, Alessandro P., additional

Published
2023
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80. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Author

Mercuri, E., primary, Seferian, A.M., additional, Servais, L., additional, Deconinck, N., additional, Stevenson, H., additional, Ni, X., additional, Zhang, W., additional, East, L., additional, Yonren, S., additional, Muntoni, F., additional, Deconinck, Nicolas, additional, Van Coster, Rudy, additional, Vanlander, Arnaud, additional, Seferian, Andreea, additional, De Lucia, Silvana, additional, Gidaro, Teresa, additional, Brande, Laura Vanden, additional, Servais, Laurent, additional, Kirschner, Janbernd, additional, Borell, Sabine, additional, Mercuri, Eugenio, additional, Brogna, Claudia, additional, Pane, Marika, additional, Fanelli, Lavinia, additional, Norcia, Giulia, additional, Muntoni, Francesco, additional, Brusa, Chiara, additional, Chesshyre, Mary, additional, Maresh, Kate, additional, Pitchforth, Jaqueline, additional, Schottlaender, Lucia, additional, Scoto, Mariacristina, additional, Silwal, Arpana, additional, and Trucco, Fedrica, additional

Published
2023
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81. Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping

Author

Mercuri, Eugenio Maria, Seferian, A. M., Servais, L., Deconinck, N., Stevenson, H., Ni, X., Zhang, W., East, L., Yonren, S., Muntoni, F., Van Coster, R., Vanlander, A., Seferian, A., De Lucia, Sara Sofia, Gidaro, T., Brande, L. V., Kirschner, J., Borell, S., Brogna, Claudia, Pane, Marika, Fanelli, L., Norcia, G., Brusa, C., Chesshyre, M., Maresh, K., Pitchforth, J., Schottlaender, L., Scoto, M., Silwal, A., Trucco, F., Mercuri E. (ORCID:0000-0002-9851-5365), De Lucia S., Brogna C., Pane M. (ORCID:0000-0002-4851-6124), Mercuri, Eugenio Maria, Seferian, A. M., Servais, L., Deconinck, N., Stevenson, H., Ni, X., Zhang, W., East, L., Yonren, S., Muntoni, F., Van Coster, R., Vanlander, A., Seferian, A., De Lucia, Sara Sofia, Gidaro, T., Brande, L. V., Kirschner, J., Borell, S., Brogna, Claudia, Pane, Marika, Fanelli, L., Norcia, G., Brusa, C., Chesshyre, M., Maresh, K., Pitchforth, J., Schottlaender, L., Scoto, M., Silwal, A., Trucco, F., Mercuri E. (ORCID:0000-0002-9851-5365), De Lucia S., Brogna C., and Pane M. (ORCID:0000-0002-4851-6124)

Abstract

Eteplirsen is FDA-approved for the treatment of Duchenne muscular dystrophy (DMD) in exon 51 skip-amenable patients. Previous studies in boys > 4 years of age indicate eteplirsen is well tolerated and attenuates pulmonary and ambulatory decline compared with matched natural history cohorts. Here the safety, tolerability and pharmacokinetics of eteplirsen in boys aged 6–48 months is evaluated. In this open-label, multicenter, dose-escalation study (NCT03218995), boys with a confirmed mutation of the DMD gene amenable to exon 51 skipping (Cohort 1: aged 24–48 months, n = 9; Cohort 2: aged 6 to < 24 months, n = 6) received ascending doses (2, 4, 10, 20, 30 mg/kg) of once-weekly eteplirsen intravenously over 10 weeks, continuing at 30 mg/kg up to 96 weeks. Endpoints included safety (primary) and pharmacokinetics (secondary). All 15 participants completed the study. Eteplirsen was well tolerated with no treatment-related discontinuations, deaths or evidence of kidney toxicity. Most treatment-emergent adverse events were mild; most common were pyrexia, cough, nasopharyngitis, vomiting, and diarrhea. Eteplirsen pharmacokinetics were consistent between both cohorts and with previous clinical experience in boys with DMD > 4 years of age. These data support the safety and tolerability of eteplirsen at the approved 30-mg/kg dose in boys as young as 6 months old.

Published
2023

82. Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy

Author

Bernert, G., Knipp, F., Buyse, G.M., Goemans, N., van den Hauwe, M., Voit, T., Doppler, V., Gidaro, T., Cuisset, J.-M., Coopman, S., Schara, U., Lutz, S., Kirschner, J., Borell, S., Will, M., D'Angelo, M.G., Brighina, E., Gandossini, S., Gorni, K., Falcier, E., Politano, L., D'Ambrosio, P., Taglia, A., Verschuuren, J.J.G.M., Straathof, C.S.M., Vílchez Padilla, J.J., Muelas Gómez, N., Sejersen, T., Hovmöller, M., Jeannet, P.-Y., Bloetzer, C., Iannaccone, S., Castro, D., Tennekoon, G., Finkel, R., Bönnemann, C., McDonald, C., Henricson, E., Joyce, N., Apkon, S., Richardson, R.C., McDonald, Craig M., Meier, Thomas, Voit, Thomas, Schara, Ulrike, Straathof, Chiara S.M., D'Angelo, M. Grazia, Bernert, Günther, Cuisset, Jean-Marie, Finkel, Richard S., Goemans, Nathalie, Rummey, Christian, Leinonen, Mika, Spagnolo, Paolo, and Buyse, Gunnar M.

Published
2016
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86. A Prospective Observational Study of EHR-Based Versus Virtual Desktop-Based Access to Pediatric Anesthesia Emergency Algorithms.

Author

MARKS, Allyson M., JOHNSON, Greg, GIDARO, Umberto, SLOBERMAN, Larry, DRAKE, Francesca M., WEINTRAUB, Ari Y., NELSON, Olivia, Kha M. TRAN, and SIMPAO, Allan F.

Abstract

When pediatric anesthesia emergencies occur, situations can deteriorate rapidly. At our hospital, the Society for Pediatric Anesthesia's (SPA) emergency algorithms are used as cognitive aids during crises, and nurses are tasked with accessing the algorithms. Operating room nurses' typical workflow includes continuous display of the of the electronic health record (EHR) intraoperative navigator, which can delay navigating to the virtual desktop window and the algorithms' icon. Thus, we implemented a button in the intraoperative navigator's toolbar to access the algorithms with one click. We conducted an observational study of the time required to access and display overhead an algorithm using the new button and old method. We surveyed participants on usability. [ABSTRACT FROM AUTHOR]

Published
2024
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87. Midline peripheral catheters inserted in the superficial femoral vein at mid-thigh: Wise choice in COVID-19 acute hypoxemic respiratory failure patients with helmet continuous positive airway pressure.

Author

Gidaro, Antonio, Samartin, Federica, Salvi, Emanuele, Casella, Francesco, Cogliati, Chiara, Giustivi, Davide, Lugli, Francesca, Trione, Chiara, Melchionda, Chiara, Bartoli, Arianna, Foschi, Antonella, Schiavini, Monica, Schiuma, Marco, Castelli, Roberto, and Calloni, Maria

Published
2023
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88. Disinfectant caps in vitro effectiveness.

Author

Rimoldi, Sara Giordana, La Cava, Leyla, Palladino, Chiara, Piacenza, Mariagrazia, Vimercati, Stefania, Cristina, Pagani, Salari, Federica, Curreli, Daniele, Gismondo, Maria Rita, Foschi, Antonella, Giustivi, Davide, Diotto, Veronica, Bizzi, Emanuele, Calloni, Maria, Casella, Francesco, Martini, Elena, Donadoni, Mattia, Cogliati, Chiara, and Gidaro, Antonio

Published
2023
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89. A decision-making algorithm proposal for PICCs and midlines insertion in patients with advanced kidney disease: A pilot study

Author

Bartoli, Arianna, primary, Gallieni, Maurizio, additional, Cogliati, Chiara, additional, Casella, Francesco, additional, Calloni, Maria, additional, Melchionda, Chiara, additional, Heidempergher, Marco, additional, Foschi, Antonella, additional, Luca Brucato, Antonio, additional, Rizzi, Giulia, additional, Quici, Massimiliano, additional, and Gidaro, Antonio, additional

Published
2023
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91. The Administration of Methotrexate in Patients with Still's Disease, “Real-Life” Findings from Aida Network Still Disease Registry

Author

Ruscitti, Piero, primary, Sota, jurgen, additional, Vitale, Antonio, additional, Lopalco, Giuseppe, additional, Iannone, Fiorenzo, additional, Morrone, Maria, additional, Mayrink Giardini, Henrique Ayres Ayres Mayrink, additional, Dagostin, Marilia A., additional, de Brito Antonelli, Isabelle Parente, additional, Almaghlouth, Ibrahim, additional, Asfina, Kazi Nur, additional, Khalil, Najma, additional, Sfikakis, Petros, additional, Laskari, Katerina, additional, Tektonidou, Maria, additional, Ciccia, Francesco, additional, Iacono, Daniela, additional, Riccio, Flavia, additional, Ragab, Gaafar, additional, Hussein, Mohamed A., additional, Govoni, Marcello, additional, Ruffilli, Francesca, additional, Direskeneli, Rafi Haner, additional, Alibaz-Oner, Fatma, additional, Giacomelli, Roberto, additional, Navarini, Luca, additional, Bartoloni, Elena, additional, Riccucci, Ilenia, additional, Martín-Nares, Eduardo, additional, Torres-Ruiz, Jiram, additional, Cipriani, Paola, additional, Di Cola, Ilenia, additional, Hernández-Rodríguez, José, additional, Gómez-Caverzaschi, Verónica, additional, Dagna, Lorenzo, additional, Tomelleri, Alessandro, additional, Makowska, Joanna, additional, Brzezinska, Olga, additional, Iagnocco, Annamaria, additional, Bellis, Elisa, additional, Caggiano, Valeria, additional, Gaggiano, Carla, additional, Tarsia, Maria, additional, Mormile, Ilaria, additional, Emmi, Giacomo, additional, Sfriso, Paolo, additional, Monti, Sara, additional, Erten, Şükran, additional, Del Giudice, Emanuela, additional, Lubrano, Riccardo, additional, Conti, Giovanni, additional, Olivieri, Alma Nunzia, additional, Lo Gullo, Alberto, additional, Tharwat, Samar, additional, Karamanakos, Anastasios, additional, Gidaro, Antonio, additional, Maggio, Maria Cristina, additional, La Torre, Francesco, additional, Cardinale, Fabio, additional, Ogunjimi, Benson, additional, Maier, Armin, additional, Sebastiani, Gian Domenico, additional, Opris-Belinski, Daniela, additional, Frassi, Micol, additional, Viapiana, Ombretta, additional, Bizzi, Emanuele, additional, Carubbi, Francesco, additional, Fotis, Lampros, additional, Tufan, Abdurrahman, additional, Kardas, Riza Can, additional, Więsik-Szewczyk, Ewa, additional, Jahnz-Różyk, Karina, additional, Fabiani, Claudia, additional, Frediani, Bruno, additional, Rigante, Donato, additional, Balistreri, Alberto, additional, and Cantarini, Luca, additional

Published
2023
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92. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial

Author

Eugenio Mercuri, Nicolas Deconinck, Elena S Mazzone, Andres Nascimento, Maryam Oskoui, Kayoko Saito, Carole Vuillerot, Giovanni Baranello, Odile Boespflug-Tanguy, Nathalie Goemans, Janbernd Kirschner, Anna Kostera-Pruszczyk, Laurent Servais, Marianne Gerber, Ksenija Gorni, Omar Khwaja, Heidemarie Kletzl, Renata S Scalco, Hannah Staunton, Wai Yin Yeung, Carmen Martin, Paulo Fontoura, John W Day, Joseph J. Volpe, John Posner, Ulrich Kellner, Rosaline Quinlivan, Aurore Daron, Stéphanie Delstanche, Romain Bruninx, Fabian Dal Farra, Olivier Schneider, Irina Balikova, Patricia Delbeke, Inge Joniau, Valentine Tahon, Sylvia Wittevrongel, Elke De Vos, Ingele Casteels, Liesbeth De Waele, Catherine Cassiman, Lies Prové, David Kinoo, Lisa Vancampenhout, Marleen Van Den Hauwe, Annelies Van Impe, Alexandra Prufer de Queiroz Campos Araujo, Aline Chacon Pereira, Flávia Nardes, Lorena Haefeli, Julia Rossetto, Marcos Ferreira Rebel, Jaqueline Almeida Pereira, Craig Campbell, Sapna Sharan, Wendy McDonald, Cheryl Scholtes, Jean Mah, Maria Sframeli, Angela Chiu, Jane Hagel, Raquel Beneish, Gaela Cariou-Palmer, Connie Pham, Daniela Toffoli, Stephanie Arpin, Sarah Turgeon Desilets, Yi Wang, Chaoping Hu, Jianfeng Huan, Chen Qian, Li Shen, Ying Xiao, Zhenxuan Zhou, Hui Li, Sujuan Wang, Hui Xiong, Xingzhi Chang, Hui Dong, Ying Liu, Tian Sang, Cuijie Wei, Jing Wen, Yiwen Cao, Xingyao Ly, Jingjing Zhao, Wenzhu Li, Lun Qin, Nina Barisic, Martina Galiot Delic, Petra Kristina Ivkic, Nenad Vukojevic, Ivana Kern, Boris Najdanovic, Marin Skugor, Teresa Gidaro, Andreea Seferian, Silvana De Lucia, Emmanuel Barreau, Nabila Mnafek, Marta Milkova Momtchilova, Helene Peche, Carole Valherie, Allison Grange, Charlotte Lilien, Darko Milascevic, Shotaro Tachibana, Claudia Ravelli, Ruxandra Cardas, Jessica Taytard, Guillaume Aubertin, Laure Vanden Brande, Jean-Baptiste Davion, Stephanie Coopman, Ikram Bouacha, Philippe Debruyne, Sabine Defoort, Gilles Derlyn, Florian Leroy, Loïc Danjoux, Julie Guilbaud, Isabelle Desguerre, Christine Barnérias, Michaela Semeraro, Dominique Bremond-Gignac, Lenaic Bruere, Maxence Rateaux, Élodie Deladrière, Virginie Germa, Yann Pereon, Sandra Mercie, Fanny Billaud, Lucie Le Goff, Guy Letellier, Aurélie Portefaix, Camille De-Montferrand, Laure Le-Goff, Stephanie Fontaine, Manel Saidi, Nabil Bouzid, Aurélie Barriere, Marie Tinat, Michelle Dreesbach, Wolf Lagréze, Bettina Michaelis, Fanni Molnar, Dorina Seger, Sibylle Vogt, Enrico Bertini, Adele D'Amico, Sergio Petroni, Anna Maria Bonetti, Adelina Carlesi, Irene Mizzoni, Claudio Bruno, Enrico Priolo, Giuseppe Rao, Simone Morando, Paola Tacchetti, Ambra Zuffi, Giacomo Pietro Comi, Roberta Brusa, Stefania Corti, Velardo Daniele, Alessandra Govoni, Francesca Magri, Valeria Minorini, Silvia Gabriella Osnaghi, Francesca Abbati, Federica Fassini, Michaela Foa, Amaqlia Lopopolo, Megi Meneri, Francesca Zoppas, Valeria Parente, Riccardo Masson, Stefania Bianchi Marzoli, Diletta Santarsiero, Myriam Garcia Sierra, Gemma Tremolada, Maria Teresa Arnoldi, Marta Vigano, Riccardo Zanin, Laura Antonaci, Roberto de Sanctis, Marika Pane, Maria Carmela Pera, Giulia Maria Amorelli, Costanza Barresi, Gugliemo D'Amico, Lorenzo Orazi, Giorgia Coratti, Kazuhiro Haginoya, Atsuko Kato, Yuko Morishita, Ryutaro Kira, Kiyomu Akiyama, Miwako Goto, Yujiro Mori, Misato Okamoto, Saki Tsutsui, Yuta Takatsuji, Aya Tanaka, Hirofumi Komaki, Miina Omori, Ippei Suzuki, Mizuki Takeuchi, Daisuke Todoroki, Seji Watanabe, Tomoko Matsubayashi, Emi Inakazu, Hiroe Nagura, Akira Suzuki, Manami Usui, Nobutsune Ishikawa, Yousuke Harada, Kenishi Fudeyasu, Kazuhiko Hirata, Kana Michiue, Kazuyuki Ueda, Junko Fujitani, Reiko Arakawa, Kozue Takano, Shigeko Yashiro, Maiko Seki, Nozomi Sano, Koji Fukuyama, Yuki Matsumoto, Hirofumi Miyazaki, Minoru Shibata, Kyoko Kobayashi, Yukie Nakamura, Yasuhiro Takeshima, Moe Kuma, Anna Fraczek, Maria Jedrzejowska, Anna Lusakowska, Agnieszka Czeszyk-Piotrowicz, Wojciech Hautz, Klaudia Rakusiewicz, Malgorzata Burlewicz, Zuzanna Gierlak-Wojcicka, Malwina Kepa, Adam Sikorski, Marcin Sobieraj, Maria Mazurkiewicz-Beldzinska, Anna Lemska, Sandra Modrzejewska, Mateusz Koberda, Urszula Stodolska-Koberda, Agnieszka Waskowska, Jagoda Kolendo, Agnieszka Sobierajska-Rek, Barbara Steinborn, Magdalena Dalz, Julia Grabowska, Wojciech Hajduk, Justyna Janasiewicz-Karachitos, Monika Klimas, Marcin Stopa, Ewa Gajewska, Beata Pusz, Dmitry Vlodavets, Evgenia Melnik, Natalya Leppenen, Nataliya Yupatova, Anastasya Monakhova, Yulia Papina, Olga Shidlovsckaia, Vedrana Milic Rasic, Vesna Brankovic, Ana Kosac, Olivera Djokic, Vesna Jakšic, Ana Pepic, Jelena Martinovic, Francina Munell Casadesus, Eduardo Tizzano, Nieves Martín Begué, Charlotte Wolley Dod, Olaia Subira, Bernat Planas Pascual, Esther Toro Tamargo, Marcos Madruga Garrido, José David Medina Romero, Marta Peña Salinas, Andrés Nascimento Osorio, Ana Díaz Cortés, Enrique Jiménez Gañan, Simone Dowon Suh, Julita Medina Cantillo, Obdulia Moya, Nuria Padros, Sandra Roca Urraca, Hugo Gonzalez Valdivia, Samuel Pascual Pascual, Sofía de Manuel, Susana Noval Martin, Paul Burnham, Sandra Espinosa, Mercedes Martinez Moreno, Haluk Topaloglu, Ibrahim Oncel, Nesibe Eroglu Ertugru, Bahadir Konuskan, Bora Eldem, Sibel Kadayifçilar, Ipek Alemdaroglu, Aynur Ayse Karaduman, Oznur Tunca Yilmaz, Neslihan Bilgin, Seher Sari, Claudia Chiriboga, John J. Lee, Donnielle Rome-Martin, John W. Day, Shannon Beres, Tina Duong, Richard Gee, Sally Dunaway Young, Sabine Fuerst-Recktenwald, Anne Marquet, Nicoletta Muelhardt, and Dylan Trundell

Subjects
Adult, Risdiplam, spinal muscular atrophy, Adolescent, Spinal Muscular Atrophies of Childhood, Settore MED/26 - NEUROLOGIA, Young Adult, Pyrimidines, Settore MED/39 - NEUROPSICHIATRIA INFANTILE, Double-Blind Method, Child, Preschool, Humans, Neurology (clinical), Child, Preschool, Azo Compounds, Aged
Abstract

BACKGROUND: Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy, with approval for use in patients with type 2 and type 3 spinal muscular atrophy granted on the basis of unpublished data. The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN. We aimed to investigate the safety and efficacy of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy. METHODS: In this phase 3, randomised, double-blind, placebo-controlled study, patients aged 2-25 years with confirmed 5q autosomal recessive type 2 or type 3 spinal muscular atrophy were recruited from 42 hospitals in 14 countries across Europe, North America, South America, and Asia. Participants were eligible if they were non-ambulant, could sit independently, and had a score of at least 2 in entry item A of the Revised Upper Limb Module. Patients were stratified by age and randomly assigned (2:1) to receive either daily oral risdiplam, at a dose of 5·00 mg (for individuals weighing =20 kg) or 0·25 mg/kg (for individuals weighing

Published
2022

93. The Administration of Methotrexate in Patients with Still's Disease, 'Real-Life' Findings from Aida Network Still Disease Registry

Author

Piero Ruscitti, jurgen Sota, Antonio Vitale, Giuseppe Lopalco, Fiorenzo Iannone, Maria Morrone, Henrique Ayres Ayres Mayrink Mayrink Giardini, Marilia A. Dagostin, Isabelle Parente de Brito Antonelli, Ibrahim Almaghlouth, Kazi Nur Asfina, Najma Khalil, Petros Sfikakis, Katerina Laskari, Maria Tektonidou, Francesco Ciccia, Daniela Iacono, Flavia Riccio, Gaafar Ragab, Mohamed A. Hussein, Marcello Govoni, Francesca Ruffilli, Rafi Haner Direskeneli, Fatma Alibaz-Oner, Roberto Giacomelli, Luca Navarini, Elena Bartoloni, Ilenia Riccucci, Eduardo Martín-Nares, Jiram Torres-Ruiz, Paola Cipriani, Ilenia Di Cola, José Hernández-Rodríguez, Verónica Gómez-Caverzaschi, Lorenzo Dagna, Alessandro Tomelleri, Joanna Makowska, Olga Brzezinska, Annamaria Iagnocco, Elisa Bellis, Valeria Caggiano, Carla Gaggiano, Maria Tarsia, Ilaria Mormile, Giacomo Emmi, Paolo Sfriso, Sara Monti, Şükran Erten, Emanuela Del Giudice, Riccardo Lubrano, Giovanni Conti, Alma Nunzia Olivieri, Alberto Lo Gullo, Samar Tharwat, Anastasios Karamanakos, Antonio Gidaro, Maria Cristina Maggio, Francesco La Torre, Fabio Cardinale, Benson Ogunjimi, Armin Maier, Gian Domenico Sebastiani, Daniela Opris-Belinski, Micol Frassi, Ombretta Viapiana, Emanuele Bizzi, Francesco Carubbi, Lampros Fotis, Abdurrahman Tufan, Riza Can Kardas, Ewa Więsik-Szewczyk, Karina Jahnz-Różyk, Claudia Fabiani, Bruno Frediani, Donato Rigante, Alberto Balistreri, and Luca Cantarini

Published
2023

94. Concomitant use of Tyrosine-Kinase Inhibitor and Mepolizumab in Asthma Secondary to Chronic Myeloid Leukemia with Hypereosinophilia

Author

Roberto Castelli, Antonio Gidaro, Maria Cristina Carraro, Emanuele Salvi, and Roberta Simona Rossi

Subjects
Pharmacology, business.industry, medicine.drug_class, Immunology, Myeloid leukemia, Hypereosinophilia, Imatinib, General Medicine, medicine.disease, Tyrosine-kinase inhibitor, hemic and lymphatic diseases, Eosinophilic, medicine, Immunology and Allergy, medicine.symptom, business, Bosutinib, Mepolizumab, Asthma, medicine.drug
Abstract

Introduction: Asthma and hypereosinophilia have been treated with different therapeutics in the past. Some of them appear to be more effective in symptoms resolution and decreasing eosinophilic count. Case Presentation: We report here an unusual case of asthma with hypereosinophilia secondary to Chronic Myeloid Leukemia (CML) with high prevalence of eosinophilic infiltrate, treated simultaneously with an anti-IL-5 antibody (Mepolizumab) and Tyrosine-kinase Inhibitors (TKI: Imatinib and Bosutinib) for three years. The patient showed a promising reduction of pulmonary exacerbations and good control of CML without developing side effects. Conclusion: We hope that this finding could inspire further studies on the efficacy and safety of the concomitant use of anti-IL-5 and TKI.

Published
2021

95. Due casi di difficile rimozione del catetere venoso centrale permanente

Author

M. Cornacchiari, M. Zuccari, A.L. Neri, B. Gidaro, N. Bellotti, A. Stasi, L.F. Di Toma, and C. Guastoni

Subjects
Internal medicine, RC31-1245, Diseases of the genitourinary system. Urology, RC870-923
Abstract

I cateteri venosi centrali o permanenti sono sempre più utilizzati nei nostri Centri dialisi. Una delle problematiche emergenti, legata alla lunga permanenza dei CVC permanenti, è la difficoltà nel rimuoverli, a causa della formazione di tenaci aderenze tra tali dispositivi e la parete vasale. Attualmente per tali complicanze, non esistono linee guida definite. Riteniamo quindi importante condividere le singole esperienze e le procedure utilizzate per la loro rimozione anche per poter definire la reale incidenza del fenomeno, le eventuali complicanze e le migliori strategie da attuare. In questo articolo, descriviamo due casi clinici di difficile rimozione di un CVCp posizionato in vena giugulare interna sin ed i metodi utilizzati per la sua estrazione.

Published
2018
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96. La nefropatia da contrasto. Definizione, fattori di rischio, prevenzione

Author

C. Guastoni, B. Gidaro, and P. Covella

Subjects
Contrasto, N-acetilcisteina, Insufficienza renale acuta, Internal medicine, RC31-1245, Diseases of the genitourinary system. Urology, RC870-923
Abstract

La nefropatia da mezzo di contrasto (contrast induced nephropathy) (CIN) è una delle cause più frequenti di danno renale acuto nei pazienti ricoverati. L'incidenza di CIN dipende dalla presenza di fattori di rischio legati al paziente (insufficienza renale, diabete, malattie cardiovascolari, età avanzata) e da cause dipendenti dalla procedura (dose elevata di mezzo di contrasto, via di somministrazione intra-arteriosa). L'insufficienza renale rappresenta il maggiore fattore di rischio di CIN, in particolare quando è associata al diabete. L'idratazione pre- e post-somministrazione di MDC rappresenta la sola terapia di prevenzione ad essere strettamente raccomandata dalle Linee Guida nei pazienti a rischio. Gli studi sulla prevenzione della CIN hanno riguardato soprattutto casistiche cardiologiche di pazienti con moderato rischio renale (GFR 60– 40 mL/min) sottoposti a somministrazione intra-arteriosa di mezzo di contrasto. In molti trial clinici è stata valutata l'efficacia dell'idratazione con bicarbonato di sodio e della N-acetilcisteina (NAC) nella prevenzione della CIN. L'infusione con sodio bicarbonato ha dimostrato una maggiore efficacia rispetto alla fisiologica, in modo particolare quando l'idratazione necessita tempi rapidi come nelle procedure in emergenza. La NAC non ha dimostrato una chiara efficacia in quanto i risultati positivi osservati in alcuni studi non sono stati confermati in altri. Il problema aperto rimane la prevenzione della CIN nei soggetti con elevato rischio renale (e-GFR < 30 mL/min) nei quali la presenza di CIN può associarsi all'ingresso in dialisi cronica.

Published
2018
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97. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study.

Author

Aurélie Chabanon, Andreea Mihaela Seferian, Aurore Daron, Yann Péréon, Claude Cances, Carole Vuillerot, Liesbeth De Waele, Jean-Marie Cuisset, Vincent Laugel, Ulrike Schara, Teresa Gidaro, Stéphanie Gilabert, Jean-Yves Hogrel, Pierre-Yves Baudin, Pierre Carlier, Emmanuel Fournier, Linda Pax Lowes, Nicole Hellbach, Timothy Seabrook, Elie Toledano, Mélanie Annoussamy, Laurent Servais, and NatHis-SMA study group

Subjects
Medicine, Science
Abstract

Spinal muscular atrophy (SMA) is a monogenic disorder caused by loss of function mutations in the survival motor neuron 1 gene, which results in a broad range of disease severity, from neonatal to adult onset. There is currently a concerted effort to define the natural history of the disease and develop outcome measures that accurately capture its complexity. As several therapeutic strategies are currently under investigation and both the FDA and EMA have recently approved the first medical treatment for SMA, there is a critical need to identify the right association of responsive outcome measures and biomarkers for individual patient follow-up. As an approved treatment becomes available, untreated patients will soon become rare, further intensifying the need for a rapid, prospective and longitudinal study of the natural history of SMA Type 2 and 3. Here we present the baseline assessments of 81 patients aged 2 to 30 years of which 19 are non-sitter SMA Type 2, 34 are sitter SMA Type 2, 9 non-ambulant SMA Type 3 and 19 ambulant SMA Type 3. Collecting these data at nine sites in France, Germany and Belgium established the feasibility of gathering consistent data from numerous and demanding assessments in a multicenter SMA study. Most assessments discriminated between the four groups well. This included the Motor Function Measure (MFM), pulmonary function testing, strength, electroneuromyography, muscle imaging and workspace volume. Additionally, all of the assessments showed good correlation with the MFM score. As the untreated patient population decreases, having reliable and valid multi-site data will be imperative for recruitment in clinical trials. The pending two-year study results will evaluate the sensitivity of the studied outcomes and biomarkers to disease progression. TRIAL REGISTRATION:ClinicalTrials.gov (NCT02391831).

Published
2018
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98. Leveraging Natural History Data in One- and Two-Arm Hierarchical Bayesian Studies of Rare Disease Progression

Author

A. Daron, James J. Dowling, Carole Vuillerot, Severine Denis, Bruno Boulanger, Rémi Bellance, Jean-Michel Arnal, Carina Wallgren-Pettersson, Kimberly Amburgey, Etsuko Tsuchiya, A. Hernandez, Jean-Marie Cuisset, Bradley P. Carlin, Enrico Bertini, Andrea Gangfuß, Barbara Andres, Arnaud Monseur, E. Gargaun, Dominique Duchene, Ruxandra Cardas, Virginie Latournerie, Ana Buj-Bello, Ulrike Schara, Basil T. Darras, H. Landy, V. Chê, Chris Freitag, Laurent Servais, S. Fontaine, Adele D'Amico, Jean-Yves Hogrel, Teresa Gidaro, Nacera Reguiba, Andreea Mihaela Seferian, L. Thielemans, Valérie Biancalana, Michèle Mayer, and Capucine de Lattre

Subjects
Statistics and Probability, medicine.medical_specialty, Clinical study design, Bayesian probability, Context (language use), Disease, Placebo, Biochemistry, Genetics and Molecular Biology (miscellaneous), Natural history, Physical medicine and rehabilitation, medicine, Biostatistics, Psychology, Type I and type II errors
Abstract

The small sample sizes inherent in rare and pediatric disease settings offer significant challenges for clinical trial design. In such settings, Bayesian adaptive trial methods can often pay dividends, allowing the sensible incorporation of auxiliary data and other relevant information to bolster that collected by the trial itself. Previous work has also included the use of one-arm trials augmented by the participants’ own natural history data, from which the future course of the disease in the absence of intervention can be predicted. Patient response can then be defined by the degree to which post-intervention observations are inconsistent with the predicted “natural” trajectory. While such trials offer obvious advantages in efficiency and ethical hazard (since they expose no new patients to a placebo, anathema to patients or their parents and caregivers), they can offer no protection against bias arising from the presence of any “placebo effect,” the tendency of patients to improve merely by being in the trial. In this paper, we investigate the impact of both static and transient placebo effects on one-arm responder studies of this type, as well as two-arm versions that incorporate a small concurrent placebo group but still borrow strength from the natural history data. We also propose more traditional Bayesian changepoint models that specify a parametric functional form for the patient’s post-intervention trajectory, which in turn allow quantification of the treatment benefit in terms of the model parameters, rather than semi-parametrically in terms of a response relative to some “null” model. We compare the operating characteristics of our designs in the context of an ongoing investigation of centronuclear myopathies (CNMs), a group of congenital neuromuscular diseases whose most common and severe form is X-linked, affecting approximately 1 in 50,000 newborn boys. Our results indicate our two-arm responder and changepoint methods can offer protection against placebo effects, improving power while protecting the trial’s Type I error rate. However, further research into innovative trial designs as well as ongoing dialog with regulatory authorities remain critically important in rare disease research.

Published
2021

99. A Diagnostic of Acquired Hemophilia Following PD1/PDL1 Inhibitors in Advanced Melanoma: The Experience of Two Patients and a Literature Review

Author

Gidaro, Antonio, primary, Palmieri, Giuseppe, additional, Donadoni, Mattia, additional, Mameli, Lucia A., additional, La Cava, Leyla, additional, Sanna, Giuseppe, additional, Castro, Dante, additional, Delitala, Alessandro P., additional, Manetti, Roberto, additional, and Castelli, Roberto, additional

Published
2022
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100. P.110 JEWELFISH: 24-month safety and pharmacodynamic data in non-treatment-naïve patients with spinal muscular atrophy (SMA)

Author

Chiriboga, C., primary, Bruno, C., additional, Duong, T., additional, Fischer, D., additional, Kirschner, J., additional, Scoto, M., additional, Mercuri, E., additional, Gerber, M., additional, Gorni, K., additional, Kletzl, H., additional, Carruthers, I., additional, Martin, C., additional, Gidaro, T., additional, and Muntoni, F., additional

Published
2022
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