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9 results on '"Pere Soler-Palacin"'

1. Multicenter Prospective Study of Biomarkers for Diagnosis of Invasive Candidiasis in Children and Adolescents

Author

Brian T Fisher, Craig L K Boge, Rui Xiao, Sydney Shuster, Dawn Chin-Quee, John Allen, Shareef Shaheen, Randall Hayden, Sri Suganda, Theoklis E Zaoutis, Yeh Chung Chang, Dwight E Yin, Anna R Huppler, Lara Danziger-Isakov, William J Muller, Emmanuel Roilides, José Romero, Paul K Sue, David Berman, Rachel L Wattier, Natasha Halasa, Daniel E Dulek, Alice Pong, Gabriela Maron, Pere Soler-Palacin, Susan C Hutto, Blanca E Gonzalez, Christine M Salvatore, Sujatha Rajan, Michael Green, Elizabeth Doby Knackstedt, Sarmistha B Hauger, and William J Steinbach

Subjects
Microbiology (medical), Antigens, Fungal, Invasive, Adolescent, Clinical Trials and Supportive Activities, Pediatrics, Medical and Health Sciences, Microbiology, Sensitivity and Specificity, Clinical Research, parasitic diseases, Major Article, Humans, Candidiasis, Invasive, Prospective Studies, Antigens, Child, Candida, screening and diagnosis, Candidiasis, biomarkers, invasive candidiasis, Biological Sciences, 4.1 Discovery and preclinical testing of markers and technologies, Detection, Fungal, Infectious Diseases, Biomarkers, 4.2 Evaluation of markers and technologies
Abstract

Background Diagnosis of invasive candidiasis (IC) relies on insensitive cultures; the relative utility of fungal biomarkers in children is unclear. Methods This multinational observational cohort study enrolled patients aged >120 days and Results Five hundred participants were enrolled at 22 centers in 3 countries, and IC was diagnosed in 13 (2.6%). Thirteen additional blood specimens were collected and successfully spiked with Candida species, to achieve a 5.0% event rate. Valid T2Candida, Fungitell, Platelia Candida Ag Plus, and Platelia Candida Ab Plus assay results were available for 438, 467, 473, and 473 specimens, respectively. Operating characteristics for T2Candida were most optimal for detecting IC due to any Candida species, with results as follows: sensitivity, 80.0% (95% confidence interval, 59.3%–93.2%), specificity 97.1% (95.0%–98.5%), positive predictive value, 62.5% (43.7%–78.9%), and negative predictive value, 98.8% (97.2%–99.6%). Only T2Candida and Platelia Candida Ag Plus assays met the threshold for combination testing. Positive result for either yielded the following results: sensitivity, 86.4% (95% confidence interval, 65.1%– 97.1%); specificity, 94.7% (92.0%–96.7%); positive predictive value, 47.5% (31.5%–63.9%); and negative predictive value, 99.2% (97.7%–99.8%). Conclusions T2Candida alone or in combination with Platelia Candida Ag Plus may be beneficial for rapid detection of Candida species in children with concern for IC. Clinical Trials Registration NCT02220790.

Published
2022
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2. Epstein–Barr virus‐associated risk factors for post‐transplant lymphoproliferative disease in pediatric liver transplant recipients

Author

Jesus Quintero Bernabeu, Javier Juamperez, Maria Mercadal‐Hally, Mauricio Larrarte King, Soledad Gallego Melcon, Luis Gros Subias, Constantino Sábado Álvarez, Pere Soler‐Palacin, Susana Melendo Pérez, Juliana Esperalba, Alexandra Navarro Jiménez, Marta Garrido Pontnou, Jessica Camacho Soriano, Ernest Hidalgo Llompart, Itxarone Bilbao Aguirre, and Ramón Charco Torra

Subjects
Epstein-Barr Virus Infections, Herpesvirus 4, Human, Transplantation, Postoperative Complications, Risk Factors, DNA, Viral, Pediatrics, Perinatology and Child Health, Humans, Viral Load, Child, Lymphoproliferative Disorders, Liver Transplantation
Abstract

Post-transplant lymphoproliferative disorder (PTLD) are the most common de novo malignancies after liver transplantation (LT) in children. The aim of our study was to assess the role of pre-LT EBV status and post-LT EBV viral load as risk factors for developing PTLD in a cohort of pediatric LT recipients.Data of all children who underwent LT between January 2002 and December 2019 were collected. Two cohorts were built EBV pre-LT primary infected cohort and EBV post-LT primary infected cohort. Moreover, using the maximal EBV viral load, a ROC curve was constructed to find a cutoff point for the diagnosis of PTLD.Among the 251 patients included in the study, fifteen PTLD episodes in 14 LT recipients were detected (2 plasmacytic hyperplasia, 10 polymorphic PTLD, 2 monomorphic PTLD, and 1 Classical-Hodgkin's lymphoma). Patients of the EBV post-LT primary infected cohort were 17.1 times more likely to develop a PTLD than patients of the EBV pre-LT primary infected cohort (2.2-133.5). The EBV viral load value to predict PTLD was set at 211 000 UI/mL (93.3% sensitivity and 77.1% specificity; AUC 93.8%; IC 0.89-0.98). In EBV post-LT primary infected cohort, patients with a viral load above 211 000 were 30 times more likely to develop PTLD than patients with a viral load below this value (OR 29.8; 3.7-241.1; p 0.001).The combination of pretransplant EBV serological status with EBV post-transplant viral load could be a powerful tool to stratify the risk of PTLD in pediatric LT patients.

Published
2022
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3. Study protocol for monitoring SARS-CoV-2 infection and its determinants in Catalonia (Spain): an observational and participatory research approach in a Sentinel Network of Schools

Author

Jordi Sunyer, Alexis Sentís, Maria Mendoza, Antoni Soriano-Arandes, Juliana Esperalba, Tomas Pumarola, Jordi Casabona, Andreu Colom-Cadena, Isabel Martinez, Cinta Folch, Xavier Duran, Albert Blanco, Juliana Maria Reyes-Urueña, Ignacio Blanco, Belén Pérez, Josep Vidal, Pere Soler-Palacin, Mireia Gascón, Carmen Cabezas, Jacobo Mendioroz, Pere-Joan Cardona, Yesika Díaz, Juliana Reyes-Urueña, Anna Bordas, Maria Subirana, Rosina Malagrida, Andres Anton, Jessica Fernández-Morales, Josep Basora, Pol Romano, Esteve Muntada, Jessica Fernández, Antonio Soriano, Cristina Andrés, Maria Victoria González, Gema Fernández, Cristina Esteban, Lucia Alonso, Jordi Aceiton, Marcos Montoro, Marina Herrero, Alba García, Juan Rus, Esperança Macià i Silvia Burgaya, Mª Teresa Riera-Bosch, Elisabet Sola, Lidia Aulet, Lidia Busquets, Xavier Perramon, Júlia Sebastià, Ana Moreno, Anna Gatell, Maria Coma, Ariadna Mas, Maria Antònia Llopis, and Sandra Pequeño

Subjects
Community-Based Participatory Research, Schools, Sociodemographic Factors, Adolescent, SARS-CoV-2, education, public health, COVID-19, General Medicine, Observational Studies as Topic, Cross-Sectional Studies, Seroepidemiologic Studies, Spain, Humans, Medicine, epidemiology, Public Health, Child, Pandemics, community child health
Abstract

IntroductionSince the SARS-CoV-2 became of concern in January 2020, many preventive measures have been adopted in educational settings to ensure the control of COVID-19 pandemic among children and staff in schools. This study aims to set up a school sentinel surveillance network with the purpose of monitoring SARS-CoV-2 infection, seroprevalence as well as to analyse the impact of preventive interventions of SARS-CoV-2 in school settings. Additionally, we will assess diverse screening strategies in a cohort of students and school staff to monitor the screening acceptance and its potential impact. Altogether, we hope this study will enable the design of more effective strategies for the prevention of COVID-19 spread.Methods and analysisThe sentinel schools’ study is a cross-sectional, school-based project including 26 participating sentinel schools in Catalonia (Spain). Children, adolescents and staff at the schools will be invited to participate. This project will be carried out from January 2021 to June 2022 as follows: (1) twice yearly serological testing and molecular SARS-CoV-2 detection and questionnaires covering SARS-CoV-2 symptoms, tests, health, knowledge, attitudes and behaviours; (2) an environmental evaluation carried out in different classrooms; (3) SARS-CoV-2 transmission dynamics and the impact of different variants among confirmed cases and classmates; (4) a participatory process by which the participants are invited to act as coinvestigators to evaluate prevention strategies and provide recommendations to improve COVID-19 prevention in schools. Descriptive analysis will be performed for the main variables collected. The incidence and seroprevalence will be calculated and the association with sociodemographic factors and school characteristics will be determined using multivariate logistic regression.Ethics and disseminationEthical approval was obtained from the IDIAPJGol and the Hospital Universitari Vall d’Hebron ethics committees. A report will be generated quarterly. Findings will be disseminated at national and international conferences and published in peer-reviewed journals.

Published
2022

4. Autoantibodies neutralizing type I IFNs are present in

Author

Paul, Bastard, Adrian, Gervais, Tom, Le Voyer, Jérémie, Rosain, Quentin, Philippot, Jérémy, Manry, Eleftherios, Michailidis, Hans-Heinrich, Hoffmann, Shohei, Eto, Marina, Garcia-Prat, Lucy, Bizien, Alba, Parra-Martínez, Rui, Yang, Liis, Haljasmägi, Mélanie, Migaud, Karita, Särekannu, Julia, Maslovskaja, Nicolas, de Prost, Yacine, Tandjaoui-Lambiotte, Charles-Edouard, Luyt, Blanca, Amador-Borrero, Alexandre, Gaudet, Julien, Poissy, Pascal, Morel, Pascale, Richard, Fabrice, Cognasse, Jesus, Troya, Sophie, Trouillet-Assant, Alexandre, Belot, Kahina, Saker, Pierre, Garçon, Jacques G, Rivière, Jean-Christophe, Lagier, Stéphanie, Gentile, Lindsey B, Rosen, Elana, Shaw, Tomohiro, Morio, Junko, Tanaka, David, Dalmau, Pierre-Louis, Tharaux, Damien, Sene, Alain, Stepanian, Bruno, Megarbane, Vasiliki, Triantafyllia, Arnaud, Fekkar, James R, Heath, José Luis, Franco, Juan-Manuel, Anaya, Jordi, Solé-Violán, Luisa, Imberti, Andrea, Biondi, Paolo, Bonfanti, Riccardo, Castagnoli, Ottavia M, Delmonte, Yu, Zhang, Andrew L, Snow, Steven M, Holland, Catherine, Biggs, Marcela, Moncada-Vélez, Andrés Augusto, Arias, Lazaro, Lorenzo, Soraya, Boucherit, Boubacar, Coulibaly, Dany, Anglicheau, Anna M, Planas, Filomeen, Haerynck, Sotirija, Duvlis, Robert L, Nussbaum, Tayfun, Ozcelik, Sevgi, Keles, Ahmed A, Bousfiha, Jalila, El Bakkouri, Carolina, Ramirez-Santana, Stéphane, Paul, Qiang, Pan-Hammarström, Lennart, Hammarström, Annabelle, Dupont, Alina, Kurolap, Christine N, Metz, Alessandro, Aiuti, Giorgio, Casari, Vito, Lampasona, Fabio, Ciceri, Lucila A, Barreiros, Elena, Dominguez-Garrido, Mateus, Vidigal, Mayana, Zatz, Diederik, van de Beek, Sabina, Sahanic, Ivan, Tancevski, Yurii, Stepanovskyy, Oksana, Boyarchuk, Yoko, Nukui, Miyuki, Tsumura, Loreto, Vidaur, Stuart G, Tangye, Sonia, Burrel, Darragh, Duffy, Lluis, Quintana-Murci, Adam, Klocperk, Nelli Y, Kann, Anna, Shcherbina, Yu-Lung, Lau, Daniel, Leung, Matthieu, Coulongeat, Julien, Marlet, Rutger, Koning, Luis Felipe, Reyes, Angélique, Chauvineau-Grenier, Fabienne, Venet, Guillaume, Monneret, Michel C, Nussenzweig, Romain, Arrestier, Idris, Boudhabhay, Hagit, Baris-Feldman, David, Hagin, Joost, Wauters, Isabelle, Meyts, Adam H, Dyer, Sean P, Kennelly, Nollaig M, Bourke, Rabih, Halwani, Narjes Saheb, Sharif-Askari, Karim, Dorgham, Jérome, Sallette, Souad Mehlal, Sedkaoui, Suzan, AlKhater, Raúl, Rigo-Bonnin, Francisco, Morandeira, Lucie, Roussel, Donald C, Vinh, Sisse Rye, Ostrowski, Antonio, Condino-Neto, Carolina, Prando, Anastasiia, Bonradenko, András N, Spaan, Laurent, Gilardin, Jacques, Fellay, Stanislas, Lyonnet, Kaya, Bilguvar, Richard P, Lifton, Shrikant, Mane, Mark S, Anderson, Bertrand, Boisson, Vivien, Béziat, Shen-Ying, Zhang, Evangelos, Vandreakos, Olivier, Hermine, Aurora, Pujol, Pärt, Peterson, Trine H, Mogensen, Lee, Rowen, James, Mond, Stéphanie, Debette, Xavier, de Lamballerie, Xavier, Duval, France, Mentré, Marie, Zins, Pere, Soler-Palacin, Roger, Colobran, Guy, Gorochov, Xavier, Solanich, Sophie, Susen, Javier, Martinez-Picado, Didier, Raoult, Marc, Vasse, Peter K, Gregersen, Lorenzo, Piemonti, Carlos, Rodríguez-Gallego, Luigi D, Notarangelo, Helen C, Su, Kai, Kisand, Satoshi, Okada, Anne, Puel, Emmanuelle, Jouanguy, Charles M, Rice, Pierre, Tiberghien, Qian, Zhang, Aurélie, Cobat, Laurent, Abel, and Hind, Hamzeh-Cognasse

Subjects
Adult, Aged, 80 and over, Adolescent, Critical Illness, Infant, Newborn, COVID-19, Infant, Interferon-alpha, Middle Aged, Antibodies, Neutralizing, Young Adult, Case-Control Studies, Child, Preschool, Immunoglobulin G, Interferon Type I, Humans, Child, Aged, Autoantibodies
Abstract

Circulating autoantibodies (auto-Abs) neutralizing high concentrations (10 ng/mL, in plasma diluted 1 to 10) of IFN-α and/or -ω are found in about 10% of patients with critical COVID-19 pneumonia, but not in subjects with asymptomatic infections. We detect auto-Abs neutralizing 100-fold lower, more physiological, concentrations of IFN-α and/or -ω (100 pg/mL, in 1/10 dilutions of plasma) in 13.6% of 3,595 patients with critical COVID-19, including 21% of 374 patients80 years, and 6.5% of 522 patients with severe COVID-19. These antibodies are also detected in 18% of the 1,124 deceased patients (aged 20 days-99 years; mean: 70 years). Moreover, another 1.3% of patients with critical COVID-19 and 0.9% of the deceased patients have auto-Abs neutralizing high concentrations of IFN-β. We also show, in a sample of 34,159 uninfected subjects from the general population, that auto-Abs neutralizing high concentrations of IFN-α and/or -ω are present in 0.18% of individuals between 18 and 69 years, 1.1% between 70 and 79 years, and 3.4%80 years. Moreover, the proportion of subjects carrying auto-Abs neutralizing lower concentrations is greater in a subsample of 10,778 uninfected individuals: 1% of individuals70 years, 2.3% between 70 and 80 years, and 6.3%80 years. By contrast, auto-Abs neutralizing IFN-β do not become more frequent with age. Auto-Abs neutralizing type I IFNs predate SARS-CoV-2 infection and sharply increase in prevalence after the age of 70 years. They account for about 20% of both critical COVID-19 cases in the over-80s, and total fatal COVID-19 cases.

Published
2021

5. Diagnostic Accuracy of the Panbio Severe Acute Respiratory Syndrome Coronavirus 2 Antigen Rapid Test Compared with Reverse-Transcriptase Polymerase Chain Reaction Testing of Nasopharyngeal Samples in the Pediatric Population

Author

Serena Villaverde, Sara Domínguez-Rodríguez, Gema Sabrido, Conchita Pérez-Jorge, Marta Plata, María Pilar Romero, Carlos Daniel Grasa, Ana Belén Jiménez, Elena Heras, Antonio Broncano, María del Mar Núñez, Marta Illán, Paloma Merino, Beatriz Soto, David Molina-Arana, Amanda Bermejo, Pablo Mendoza, Manuel Gijón, Begoña Pérez-Moneo, Cinta Moraleda, Alfredo Tagarro, Cristina Calvo, Ma José Mellado, Paula Rodríguez-Molino, Teresa del Rosal, Mar Santos, Marisa Navarro, Elena Rincón, Begoña Santiago, Jesús Saavedra-Lozano, David Aguilera-Alonso, Cristina Epalza, Daniel Blázquez-Gamero, Sara Villanueva, Pablo Rojo, Gero Calleja, J.A. Alonso, Mercedes de la Torre, F.J. Sanz-Santaeufemia, M.I. Iglesias, B. Herrero, M. Alonso, Toni Soriano-Arandes, J.M. Pujol, Susana Melendo, Pere Soler-Palacin, Silvia Simó, Victoria Fumadó, Miguel Lanaspa, M. Urretavizcaya, Mercedes Herranz, Marta Pareja, Fatima Ara, Santiago Cabañas, Rut del Valle, Ana Barrios, Enrique Otheo, José Luis Vázquez, Lola Falcón, Olaf Neth, Peter Olbrich, Walter Goicoechea, Laura Martín, Lucía Figueroa, María Llorente, María Penin, Claudia García, María García, Teresa Alvaredo, Ma Inmaculada Olmedo, Agustín López, Elvira Cobo, Mariam Tovizi, Pilar Galán, Sara Guillén, Adriana Navas, M. Luz García, Sara Pérez, María José Hernández, Arantxa Berzosa, Nerea Gallego, Ana López, Beatriz Ruiz, Santiago Alfayate, Ana Menasalvas, Eloísa Cervantes, María Méndez, Ángela Hurtado, Yolanda Ruiz, Cristina García, Inés Amich, Manuel Oltra, Álvaro Villaroya, Angustias Ocaña, Isabel Romero, María Fernanda Guzmán, M.J. Pascual, María Sánchez-Códez, Elena Montesinos, Julia Jensen, María Rodríguez, Gloria Caro, Neus Rius, Alba Gómez, Rafael Bretón, Margarita Rodríguez, Julio Romero, Ana Campos, Mercedes García, Rosa María Velasco, Zulema Lobato, Fernando Centeno, Elena Pérez, Paula Vidal, Corsino Rey, Ana Vivanco, Maruchi Alonso, Pedro Alcalá, Javier González de Dios, Eduard Solé, Laura Minguell, Itziar Astigarraga, Ma Ángeles Vázquez, Miguel Sánchez, Elena Díaz, Eduardo Consuegra, María Cabanillas, Luis Peña, Elisa Garrote, Maite Goicoechea, Irene Centelles, Santiago Lapeña, Sara Gutiérrez, Soraya Gutiérrez, Amparo Cavalle, José María Olmos, Alejandro Cobo, Sara Díaz, Beatriz Jiménez, Raúl González, Miguel Lafuente, Matilde Bustillo, Natividad Pons, Julia Morata, and Elsa Segura

Subjects
Male, medicine.medical_specialty, Adolescent, Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), Diagnostic accuracy, Disease, medicine.disease_cause, Gastroenterology, law.invention, antigen test, 03 medical and health sciences, 0302 clinical medicine, Antigen, law, 030225 pediatrics, Internal medicine, Nasopharynx, medicine, Humans, 030212 general & internal medicine, Pediatrics, Perinatology, and Child Health, Child, Antigens, Viral, Pandemics, Polymerase chain reaction, Coronavirus, business.industry, Reverse Transcriptase Polymerase Chain Reaction, SARS-CoV-2, Infant, Newborn, COVID-19, Infant, Reproducibility of Results, Reverse transcriptase, PCR, Child, Preschool, Pediatrics, Perinatology and Child Health, DNA, Viral, Brief Reports, Female, business, Pediatric population
Abstract

We conducted a multicenter clinical validity study of the Panbio coronavirus disease 2019 Antigen Rapid Test of nasopharyngeal samples in pediatric patients with coronavirus disease 2019-compatible symptoms of ≤5 days of evolution. Our study showed limited accuracy in nasopharyngeal antigen testing: overall sensitivity was 45.4%, and 99.8% of specificity, positive-predictive value was 92.5%.

Published
2020

6. Disease Evolution and Response to Rapamycin in Activated Phosphoinositide 3-Kinase δ Syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry

Author

Maria Elena Maccari, Hassan Abolhassani, Asghar Aghamohammadi, Alessandro Aiuti, Olga Aleinikova, Catherine Bangs, Safa Baris, Federica Barzaghi, Helen Baxendale, Matthew Buckland, Siobhan O. Burns, Caterina Cancrini, Andrew Cant, Pascal Cathébras, Marina Cavazzana, Anita Chandra, Francesca Conti, Tanya Coulter, Lisa A. Devlin, J. David M. Edgar, Saul Faust, Alain Fischer, Marina Garcia Prat, Lennart Hammarström, Maximilian Heeg, Stephen Jolles, Elif Karakoc-Aydiner, Gerhard Kindle, Ayca Kiykim, Dinakantha Kumararatne, Bodo Grimbacher, Hilary Longhurst, Nizar Mahlaoui, Tomas Milota, Fernando Moreira, Despina Moshous, Anna Mukhina, Olaf Neth, Benedicte Neven, Alexandra Nieters, Peter Olbrich, Ahmet Ozen, Jana Pachlopnik Schmid, Capucine Picard, Seraina Prader, William Rae, Janine Reichenbach, Stephan Rusch, Sinisa Savic, Alessia Scarselli, Raphael Scheible, Anna Sediva, Svetlana O. Sharapova, Anna Shcherbina, Mary Slatter, Pere Soler-Palacin, Aurelie Stanislas, Felipe Suarez, Francesca Tucci, Annette Uhlmann, Joris van Montfrans, Klaus Warnatz, Anthony Peter Williams, Phil Wood, Sven Kracker, Alison Mary Condliffe, Stephan Ehl, Federal Ministry of Education and Research (Germany), Novartis, GlaxoSmithKline, University of Tehran, Service de Médecine Interne, CHU Saint-Etienne-Hôpital Nord - Saint-Etienne, Département de Biothérapie [CHU Necker], CHU Necker - Enfants Malades [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris Descartes - Paris 5 (UPD5), Department of Medical Oncology, Regina Elena Cancer Institute, Developpement Normal et Pathologique du Système Immunitaire, Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Chaire Médecine expérimentale (A. Fischer), Collège de France (CdF (institution)), Istanbul University, Addenbrookes Hospital, Center for Chronic Immunodeficiency (CCI), University Medical Center Freiburg, Freiburg, Germany, IFR Necker-Enfants Malades (IRNEM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Service d'immuno-hématologie pédiatrique [CHU Necker], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-CHU Necker - Enfants Malades [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Unité d'Immunologie Hématologie et Rhumatologie Pédiatrique [Necker, Paris], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Division of Clinical Epidemiology, Deutches Krebsforschungszentrum, Faculty of Medicine, Division of Pediatric Allergy and Immunology, Marmara University, Génétique Humaine des Maladies Infectieuses (Inserm U980), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Dmitry Rogachev Federal Research and Clinical Center of Pediatric Hematology, Oncology and Immunology, Moscow, European Laboratory for Food Induced Diseases, Università degli studi di Napoli Federico II, Imagine - Institut des maladies génétiques (IMAGINE - U1163), CHU Necker - Enfants Malades [AP-HP]-Université Paris Descartes - Paris 5 (UPD5)-Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Assistance publique - Hôpitaux de Paris (AP-HP) (APHP)-CHU Necker - Enfants Malades [AP-HP], CHU Necker - Enfants Malades [AP-HP]-Assistance publique - Hôpitaux de Paris (AP-HP) (APHP), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Descartes - Paris 5 (UPD5), Centre National de la Recherche Scientifique (CNRS)-Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Maccari, Maria Elena, Abolhassani, Hassan, Aghamohammadi, Asghar, Aiuti, Alessandro, Aleinikova, Olga, Bangs, Catherine, Baris, Safa, Barzaghi, Federica, Baxendale, Helen, Buckland, Matthew, Burns, Siobhan O., Cancrini, Caterina, Cant, Andrew, Cathebras, Pascal, Cavazzana, Marina, Chandra, Anita, Conti, Francesca, Coulter, Tanya, Devlin, Lisa A., Edgar, J. David M., Faust, Saul, Fischer, Alain, Prat, Marina Garcia, Hammarstrom, Lennart, Heeg, Maximilian, Jolles, Stephen, Karakoc-Aydiner, Elif, Kindle, Gerhard, Kiykim, Ayca, Kumararatne, Dinakantha, Grimbacher, Bodo, Longhurst, Hilary, Mahlaoui, Nizar, Milota, Tomas, Moreira, Fernando, Moshous, Despina, Mukhina, Anna, Neth, Olaf, Neven, Benedicte, Nieters, Alexandra, Olbrich, Peter, Ozen, Ahmet, Schmid, Jana Pachlopnik, Picard, Capucine, Prader, Seraina, Rae, William, Reichenbach, Janine, Rusch, Stephan, Savic, Sinisa, Scarselli, Alessia, Scheible, Raphael, Sediva, Anna, Sharapova, Svetlana O., Shcherbina, Anna, Slatter, Mary, Soler-Palacin, Pere, Stanislas, Aurelie, Suarez, Felipe, Tucci, Francesca, Uhlmann, Annette, van Montfrans, Joris, Warnatz, Klaus, Williams, Anthony Peter, Wood, Phil, Kracker, Sven, Condliffe, Alison Mary, Ehl, Stephan, Cathébras, Pascal, Hammarström, Lennart, Milota, Toma, Montfrans, Joris van, and Warnatz, Klau

Subjects
0301 basic medicine, Oncology, activated phosphoinositide 3-kinase δ syndrome, [SDV]Life Sciences [q-bio], SIROLIMUS, registry, Activated phosphoinositide 3-kinase δ syndrome, Immunology and Allergy, Registries, Young adult, Non-U.S. Gov't, Child, MUTATION, Societies, Medical, ComputingMilieux_MISCELLANEOUS, Research Support, Non-U.S. Gov't, Middle Aged, PIK3R1, 3. Good health, Europe, Settore MED/02, natural history, Child, Preschool, Perspective, Cohort, Sirolimus/therapeutic use, [SDV.IMM]Life Sciences [q-bio]/Immunology, medicine.symptom, Immunosuppressive Agents, medicine.drug, Adult, lcsh:Immunologic diseases. Allergy, medicine.medical_specialty, Registry, activated phosphoinositide 3-kinase delta syndrome, Adolescent, Class I Phosphatidylinositol 3-Kinases, Primary Immunodeficiency Diseases, Immunology, Natural history, Research Support, Asymptomatic, 03 medical and health sciences, Young Adult, Medical, Internal medicine, Journal Article, medicine, Humans, Rapamycin, Preschool, Sirolimus, Immunologic Deficiency Syndromes/drug therapy, Cytopenia, [SDV.GEN]Life Sciences [q-bio]/Genetics, Bronchiectasis, business.industry, rapamycin, Immunologic Deficiency Syndromes, PIK3CD, medicine.disease, 030104 developmental biology, [SDV.GEN.GH]Life Sciences [q-bio]/Genetics/Human genetics, Autoimmune lymphoproliferative syndrome, Primary immunodeficiency, Immunosuppressive Agents/therapeutic use, [SDV.SPEE]Life Sciences [q-bio]/Santé publique et épidémiologie, Societies, business, lcsh:RC581-607
Abstract

Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS), caused by autosomal dominant mutations in PIK3CD (APDS1) or PIK3R1 (APDS2), is a heterogeneous primary immunodeficiency. While initial cohort-descriptions summarized the spectrum of clinical and immunological manifestations, questions about long-term disease evolution and response to therapy remain. The prospective European Society for Immunodeficiencies (ESID)-APDS registry aims to characterize the disease course, identify outcome predictors, and evaluate treatment responses. So far, 77 patients have been recruited (51 APDS1, 26 APDS2). Analysis of disease evolution in the first 68 patients pinpoints the early occurrence of recurrent respiratory infections followed by chronic lymphoproliferation, gastrointestinal manifestations, and cytopenias. Although most manifestations occur by age 15, adult-onset and asymptomatic courses were documented. Bronchiectasis was observed in 24/40 APDS1 patients who received a CT-scan compared with 4/15 APDS2 patients. By age 20, half of the patients had received at least one immunosuppressant, but 2–3 lines of immunosuppressive therapy were not unusual before age 10. Response to rapamycin was rated by physician visual analog scale as good in 10, moderate in 9, and poor in 7. Lymphoproliferation showed the best response (8 complete, 11 partial, 6 no remission), while bowel inflammation (3 complete, 3 partial, 9 no remission) and cytopenia (3 complete, 2 partial, 9 no remission) responded less well. Hence, non-lymphoproliferative manifestations should be a key target for novel therapies. This report from the ESID-APDS registry provides comprehensive baseline documentation for a growing cohort that will be followed prospectively to establish prognostic factors and identify patients for treatment studies., This study was supported by the German Federal Ministry of Education and Research (BMBF 01E01303). The ESID-APDS registry is supported by the pharmaceutical companies Novartis, GlaxoSmithKline, and UCB UK.

Published
2018
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7. Human immunoglobulin (KIOVIG®/GAMMAGARD LIQUID®) for immunodeficiency and autoimmune diseases: an observational cohort study

Author

Nikolai Nikolov, Pere Soler-Palacin, Siraj A Misbah, Werner Engl, Bohumír Blažek, Victoria G. Empson, Heinz Leibl, David Gelmont, and Barbara McCoy

Subjects
Adult, Male, medicine.medical_specialty, Adolescent, Immunology, Autoimmune Diseases, Human immunoglobulin, hemic and lymphatic diseases, Internal medicine, medicine, Humans, Immunology and Allergy, Child, Adverse effect, Immunodeficiency, Aged, Autoimmune disease, business.industry, Immunologic Deficiency Syndromes, Immunoglobulins, Intravenous, Infant, Middle Aged, medicine.disease, Oncology, Tolerability, Child, Preschool, Female, Observational study, business, Adverse drug reaction, Cohort study
Abstract

Aim: To document the therapeutic efficacy and safety of Human Normal Immunoglobulin 10% Liquid (KIOVIG®/GAMMAGARD LIQUID® [IVIG 10%]) under clinical routine conditions. Patients & methods: Subjects received IVIG 10% according to the prescribing information and were followed for 6 ± 1 weeks to 12 ± 2 months depending on indication. Efficacy, adverse events, infusion rates and duration and dose were recorded. Results: Overall efficacy of IVIG 10% was rated as good or very good by the investigator in 81.8% of subjects; overall tolerability was good or very good in 87.5%. One serious adverse drug reaction (ADR) occurred (urticaria); no severe ADRs occurred. Conclusion: In this observational study, the efficacy and safety of IVIG 10% in routine clinical practice was similar to that previously reported in clinical studies.

Published
2015
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8. [The Spanish Society of Paediatric Infectious Diseases (SEIP) recommendations on the diagnosis and management of invasive candidiasis]

Author

C, Figueras, C, Díaz de Heredia, J J, García, M, Navarro, J, Ruiz-Contreras, R, Rossich, J, Rumbao, M A, Frick, E M, Roselló, and Pere, Soler-Palacin

Subjects
Infant, Newborn, Humans, Infant, Candidiasis, Invasive
Abstract

Candida yeasts are ubiquitous commensals, which can cause opportunistic infection in any location of the body. The source of infection may be both endogenous and exogenous. Invasive candidiasis encompasses different entities ranging from invasive candidiasis to disseminated multiorgan infection. Invasive candidiasis is the third leading cause of nosocomial bloodstream infection and the fourth of all nosocomial infections. It is also the most common invasive fungal infection in non-neutropenic critically ill patients, with a remarkable increase in the last 20 years owing to the increased survival of these patients and to more complex diagnostic, therapeutic and surgical procedures. Its incidence in infants, according to recent reviews, stands at 38.8 cases/100,000 children younger than 1 year. Candida albicans remains the most frequent isolate in invasive infections, although infections caused by other species have risen in the last years, such as C. kruzsei, C. glabrata and C. parapsilosis; the latter causing invasive candidiasis mainly associated with central venous catheter management, especially in neonatal units. The overall mortality of invasive candidiasis is high, with 30-day mortality reaching 20-44% in some series involving paediatric patients. This report provides an update on incidence, epidemiology, clinical presentation, diagnosis, treatment and outcome of invasive infection by Candida spp. in the paediatric patient.

Published
2010

9. A new tool for the paediatric HIV research: general data from the Cohort of the Spanish Paediatric HIV Network (CoRISpe)

Author

José Luis Jiménez, Olaf Neth, JOAQUIN DUEÑAS MORALES, Raquel Lorente Rodriguez, Ana Menasalvas, Pere Soler-Palacin, Mª Ángeles Muñoz-Fernández, María Espiau, Miguel Angel Roa, Daniel Blázquez-Gamero, and César Gavilán

Subjects
Adult, Male, Pediatrics, medicine.medical_specialty, Biomedical Research, Databases, Factual, Pediatric hiv, MEDLINE, Prospective data, HIV Infections, Paediatric HIV infection, lcsh:Infectious and parasitic diseases, Cohort Studies, Correspondence, Electronic Health Records, Humans, Medicine, lcsh:RC109-216, Child, Prospective cohort study, Paediatric patients, HIV paediatric cohort, business.industry, Infant, Newborn, Spanish HIV HGM biobank, Infant, Viral Load, Infectious Disease Transmission, Vertical, Infectious Diseases, Spain, Child, Preschool, Family medicine, Cohort, Research studies, Female, business, Cohort study
Abstract

There are approximately from 1,100 to 1,200 HIV-infected children in a follow-up in Spain. In 2008 an open, multicentral, retrospective and prospective Cohort of the Spanish Paediatric HIV Network (CoRISpe) was founded. The CoRISpe is divided into the node 1 and node 2 representing geographically almost the whole territory of Spain. Since 2008 seventy-five hospitals have been participating in the CoRISpe. All the retrospective data of the HIV-infected children have been kept in the CoRISpe since 1995 and prospective data since 2008. In this article we are going to present the notion of CoRISpe, its role, the structure, how the CoRISpe works and the process how a child is transferred from Paediatric to Adults Units. The main objective of the CoRISpe is to contribute to furthering scientific knowledge on paediatric HIV infection by providing demographic, sociopsychological, clinical and laboratory data from HIV-infected paediatric patients. Its aim is to enable high-quality research studies on HIV-infected children.

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