Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series.

Autor:	Naviglio S; Institute for Maternal and Child Health IRCCS 'Burlo Garofolo,' Trieste, Italy., Cicalese MP; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Università Vita-Salute San Raffaele, Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Rivers E; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom., Ferrua F; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Bonfim C; Bone Marrow Transplantation Unit, Complexo Hospital de Clínicas, Federal University of Paraná, Curitiba, Brazil.; Instituto de Pesquisa Pelé Pequeno Príncipe, Hospital Pequeno Príncipe, Curitiba, Brazil., Cenciarelli S; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy., Cheong KN; Hong Kong Children's Hospital, Hong Kong, China., Faraci M; Hematopoietic Stem Cell Transplantation Unit, Department of Hematology-Oncology, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Giardino S; Hematopoietic Stem Cell Transplantation Unit, Department of Hematology-Oncology, IRCCS Istituto Giannina Gaslini, Genoa, Italy., Ghosh S; Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich Heine University-Düsseldorf University Hospital, Düsseldorf, Germany., Lee PP; Department of Paediatrics and Adolescent Medicine, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China., Lyra PT; Department of Clinical Immunology, Instituto de Medicina Integral Professor Fernando Figueira, Recife, Brazil.; Children's Department of Infectious Disease, Oswaldo Cruz University Hospital, Universidade de Pernambuco, Recife, Brazil., Meisel R; Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich Heine University-Düsseldorf University Hospital, Düsseldorf, Germany., Sofia V; Università Vita-Salute San Raffaele, Milan, Italy., Tessitore A; Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy., Tommasini A; Institute for Maternal and Child Health IRCCS 'Burlo Garofolo,' Trieste, Italy.; Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy., Valencic E; Institute for Maternal and Child Health IRCCS 'Burlo Garofolo,' Trieste, Italy., Vallée TC; Dr. von Hauner University Children's Hospital, Ludwig-Maximilians-Universität, Munich, Germany., Volpi S; Unità Operativa Complessa Clinica Pediatrica e Reumatologia, Centro per le Malattie Autoinfiammatorie e Immunodeficienze, Istituto Giannina Gaslini, Genoa, Italy.; Dipartimento di Neuroscienze, riabilitazione, oftalmologia, genetica e scienze materno-infantili, Università degli Studi di Genova, Genoa, Italy., Worth AJ; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Rabusin M; Institute for Maternal and Child Health IRCCS 'Burlo Garofolo,' Trieste, Italy., Albert MH; Dr. von Hauner University Children's Hospital, Ludwig-Maximilians-Universität, Munich, Germany., Thrasher AJ; Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom., Aiuti A; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy.; Università Vita-Salute San Raffaele, Milan, Italy.; San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Jazyk:	angličtina
Zdroj:	Blood [Blood] 2024 Oct 17; Vol. 144 (16), pp. 1699-1704.
DOI:	10.1182/blood.2024024524
Abstrakt:	Abstract: Up to 70% of patients with Wiskott-Aldrich syndrome (WAS) develop autoimmune and inflammatory manifestations. Dysregulation of interleukin 1 (IL-1) may be involved in their pathogenesis, yet there is little evidence on treatment with anti-IL-1 agents in these patients. We conducted a multicenter retrospective analysis of 9 patients with WAS treated with anti-IL-1 agents (anakinra or canakinumab). All patients had prominent inflammatory manifestations, including systemic, cutaneous, articular, and intestinal symptoms; 3 patients presented with a severe systemic inflammatory syndrome since the first months of life. Corticosteroid therapy was associated with partial or no response, whereas treatment with anakinra or canakinumab resulted in prompt, often dramatic, responses in all patients, allowing bridging to gene therapy (4 patients) or hematopoietic stem cell transplantation (HSCT; 5 patients). Treatment was overall well tolerated. Low donor myeloid chimerism developed in 4 patients after HSCT and was associated with the appearance or the recurrence of inflammatory manifestations. A second HSCT was performed in 2 patients, achieving full-donor chimerism and resolution of inflammatory manifestation, whereas the other 2 patients were treated with prolonged therapy with anti-IL-1 agents. Our experience demonstrates that some inflammatory manifestations of WAS are dependent on IL-1 and respond well to its pharmacologic blockade. (© 2024 American Society of Hematology. Published by Elsevier Inc. All rights are reserved, including those for text and data mining, AI training, and similar technologies.)
Databáze:	MEDLINE
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