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Efficacy and safety of anakinra for undifferentiated autoinflammatory diseases in children: a retrospective case review

Garg, S; Wynne, K; Omoyinmi, E; Eleftheriou, D; Brogan, P; (2019) Efficacy and safety of anakinra for undifferentiated autoinflammatory diseases in children: a retrospective case review. Rheumatology Advances in Practice , 3 (1) , Article rkz004. 10.1093/rap/rkz004. Green open access

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Abstract

Objective: The aim was to carry out a retrospective review of the efficacy and safety of anakinra in paediatric patients with undifferentiated autoinflammatory disease (uAID). Methods: We carried out a retrospective study of children with uAID at a single quaternary centre. The clinical efficacy of anakinra was evaluated using physician global assessment (PGA) and serological response assessed by levels of serum amyloid A and CRP. Safety was assessed by exploring adverse events, including infection and drug reactions. Results: This study included 22 patients, 64% females and 36% males of median age 7.1 years (range 0.13-14.11 years), with uAID. The median starting dose of anakinra was 2 mg/kg (range 2-6 mg/kg) and the median duration of treatment 19.6 months (range 0.8-100 months). Before anakinra treatment, the median PGA, on a three-point Likert scale, was 2 (range 1-2), which fell to 1 (range 0-2) within 3 months of treatment. Eight of 22 (36%) patients achieved complete clinical and serological remission; 8/22 (36%) achieved a partial response; and 6/22 (28%) had no response to anakinra. Adverse events included death (3/22, 14%) and allogeneic haematopoietic stem cell transplantation (1/22, 5%). There were no new safety signals, and anakinra was well tolerated overall. Conclusion: Retrospectively, 72% of children with uAID responded well to anakinra, with 36% achieving full clinical and serological remission within 3 months. This suggests that empirical trials of IL-1 blockade might be warranted in children with uAID. Clear stopping criteria based on predefined parameters should be considered, because non-responders required alternative therapies, facilitated by a definitive molecular diagnosis where possible.

Type: Article
Title: Efficacy and safety of anakinra for undifferentiated autoinflammatory diseases in children: a retrospective case review
Location: England
Open access status: An open access version is available from UCL Discovery
DOI: 10.1093/rap/rkz004
Publisher version: https://doi.org/10.1093/rap/rkz004
Language: English
Additional information: Copyright © The Author(s) 2019. Published by Oxford University Press on behalf of the British Society for Rheumatology. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted reuse, distribution, and reproduction in any medium, provided the original work is properly cited.
Keywords: IL-1 receptor antagonist, anakinra, child, unclassified autoinflammatory disease, undifferentiated autoinflammatory disease
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 Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Medical Sciences > Div of Medicine > Inflammation
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/10080596
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