Naviglio, S;
Cicalese, MP;
Rivers, E;
Ferrua, F;
Bonfim, C;
Cenciarelli, S;
Cheong, KN;
... Aiuti, A; + view all
(2024)
Interleukin-1 blockade in patients with Wiskott-Aldrich Syndrome: a retrospective multinational case series.
Blood
10.1182/blood.2024024524.
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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.
| Type: | Article |
|---|---|
| Title: | Interleukin-1 blockade in patients with Wiskott-Aldrich Syndrome: a retrospective multinational case series |
| Location: | United States |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1182/blood.2024024524 |
| Publisher version: | http://dx.doi.org/10.1182/blood.2024024524 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10197184 |
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