Deuxième analyse intermédiaire d’une étude en ouvert du Leniolisib dans le syndrome PI3Kδ activée : efficacité et tolérance long terme

Autor: Rao, V.K., Sediva, A., Dalm, V., Alessandro, P., Catharina, S., Shcherbina, A., Trizzino, A., Kulm, E., Zharankova, Y., Körholz, J., Lougaris, V., Rodina, J., Bradt, J., Webster, S., Alanvin, O., Vandier, D., Amores, X., Relan, A., Gulbu, U.

Publikováno v: In La revue de médecine interne June 2024 45 Supplement 1:A135-A135

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A randomised, placebo-controlled, phase III trial of leniolisib in activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS): Adolescent and adult subgroup analysis.

Autor: Rao VK; National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States. Electronic address: korao@niaid.nih.gov., Šedivá A; Department of Immunology, Motol University Hospital, 2nd Faculty of Medicine, Charles University, Prague, Czech Republic., Dalm VASH; Department of Internal Medicine, Division of Allergy and Clinical Immunology; Department of Immunology, Erasmus University Medical Center, Rotterdam, the Netherlands., Plebani A; Pediatrics Clinic, Department of Clinical and Experimental Sciences, University of Brescia, Azienda Socio Sanitaria Territoriale Spedali Civili di Brescia, Brescia, Italy., Schuetz C; Pediatric Immunology, Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany., Shcherbina A; Department of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Trizzino A; Department of Pediatric Hematology and Oncology, ARNAS Ospedali Civico Di Cristina Benfratelli Hospital, Palermo, Italy., Zharankova Y; Belarusian Research Center for Pediatric Oncology, Hematology and Immunology, Minsk, Belarus., Orpia A; National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States., Kulm E; Clinical Research Directorate, Frederick National Laboratory for Cancer Research, Bethesda, MD, United States., Webster S; National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States; Pharming Healthcare Inc, Warren, NJ, United States., Körholz J; Pediatric Immunology, Department of Pediatrics, Faculty of Medicine and University Hospital Carl Gustav Carus, Technische Universität Dresden, Dresden, Germany., Lougaris V; Pediatrics Clinic, Department of Clinical and Experimental Sciences, University of Brescia, Azienda Socio Sanitaria Territoriale Spedali Civili di Brescia, Brescia, Italy., Rodina Y; Department of Immunology, Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia., Conlon N; Wellcome Trust Clinical Research Facility, St. James's Hospital and School of Medicine, Trinity College Dublin, Ireland., Coulter T; Regional Immunology Services of Northern Ireland, Belfast Health and Social Care Trust, United Kingdom., Bradt J; Pharming Healthcare Inc, Warren, NJ, United States., Relan A; Pharming Healthcare Inc, Warren, NJ, United States., Uzel G; National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, United States.

Publikováno v: Clinical immunology (Orlando, Fla.) [Clin Immunol] 2024 Nov 17, pp. 110400. Date of Electronic Publication: 2024 Nov 17.