{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T09:57:47Z","timestamp":1790243867843,"version":"4.1.0"},"reference-count":25,"publisher":"American Society of Hematology","issue":"16","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2024,10,17]]},"abstract":"<jats:sec>\n                  <jats:title>Abstract<\/jats:title>\n                  <jats:p>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\u2013IL-1 agents in these patients. We conducted a multicenter retrospective analysis of 9 patients with WAS treated with anti\u2013IL-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\u2013IL-1 agents. Our experience demonstrates that some inflammatory manifestations of WAS are dependent on IL-1 and respond well to its pharmacologic blockade.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1182\/blood.2024024524","type":"journal-article","created":{"date-parts":[[2024,7,24]],"date-time":"2024-07-24T17:10:18Z","timestamp":1721841018000},"page":"1699-1704","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":9,"title":["Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series"],"prefix":"10.1182","volume":"144","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1915-9965","authenticated-orcid":false,"given":"Samuele","family":"Naviglio","sequence":"first","affiliation":[{"name":"1Institute for Maternal and Child Health IRCCS \u201cBurlo Garofolo,\u201d Trieste, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0371-3279","authenticated-orcid":false,"given":"Maria Pia","family":"Cicalese","sequence":"additional","affiliation":[{"name":"2Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy"},{"name":"3Universit\u00e0 Vita-Salute San Raffaele, Milan, Italy"},{"name":"4San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elizabeth","family":"Rivers","sequence":"additional","affiliation":[{"name":"5Great Ormond Street Institute of Child Health, University College London, London, United 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Brazil"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1413-4371","authenticated-orcid":false,"given":"Sabina","family":"Cenciarelli","sequence":"additional","affiliation":[{"name":"2Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy"},{"name":"4San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0684-264X","authenticated-orcid":false,"given":"Kai-Ning","family":"Cheong","sequence":"additional","affiliation":[{"name":"8Hong Kong Children's Hospital, Hong Kong, China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5715-501X","authenticated-orcid":false,"given":"Maura","family":"Faraci","sequence":"additional","affiliation":[{"name":"9Hematopoietic Stem Cell Transplantation Unit, Department of Hematology-Oncology, IRCCS Istituto Giannina Gaslini, Genoa, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7788-6407","authenticated-orcid":false,"given":"Stefano","family":"Giardino","sequence":"additional","affiliation":[{"name":"9Hematopoietic Stem Cell Transplantation Unit, Department of Hematology-Oncology, IRCCS Istituto Giannina Gaslini, Genoa, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sujal","family":"Ghosh","sequence":"additional","affiliation":[{"name":"10Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical Faculty, Heinrich Heine University\u2013D\u00fcsseldorf University Hospital, D\u00fcsseldorf, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5934-7338","authenticated-orcid":false,"given":"Pamela P.","family":"Lee","sequence":"additional","affiliation":[{"name":"11Department of Paediatrics and 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Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valentina","family":"Sofia","sequence":"additional","affiliation":[{"name":"3Universit\u00e0 Vita-Salute San Raffaele, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1865-7700","authenticated-orcid":false,"given":"Antimo","family":"Tessitore","sequence":"additional","affiliation":[{"name":"14Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Tommasini","sequence":"additional","affiliation":[{"name":"1Institute for Maternal and Child Health IRCCS \u201cBurlo Garofolo,\u201d Trieste, Italy"},{"name":"14Department of Medicine, Surgery and Health Sciences, University of Trieste, Trieste, 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