Evidence map›Paper›PMID 31431992›Full record

ArticleRheumatology advances in practice2019

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

Suchika Garg, Karen Wynne, Ebun Omoyinmi, Despina Eleftheriou, Paul Brogan

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Rheumatology advances in practice, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed, 4 pooled it
1.0field-weighted citation impact, top 25% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

19 citing papers in PubMed, 4 syntheses or guidelines pooled it, 24 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Pooled it
  4. Pooled it
  5. Inflammasomes in respiratory diseases: from disease pathogenesis to drug development and use of multi-omics.European respiratory review : an official journal of the European Respiratory Society · 2026
    Review
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  15. "Lessons from Rare Forms of Osteoarthritis".Calcified tissue international · 2021
    Review
  16. Review
  17. Disseminated Herpes Zoster During COVID-19.Infectious diseases in clinical practice (Baltimore, Md.) · 2021
    Article
  18. Review
  19. The off-label use of anakinra in pediatric systemic autoinflammatory diseases.Therapeutic advances in musculoskeletal disease · 2020
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 1 institution in 1 country.

Suchika GargInfection, Inflammation and Rheumatology Section, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Karen WynneDepartment of Paediatric Rheumatology, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Ebun OmoyinmiInfection, Inflammation and Rheumatology Section, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Despina EleftheriouInfection, Inflammation and Rheumatology Section, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Paul BroganInfection, Inflammation and Rheumatology Section, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Great Ormond Street Hospital for Children NHS Foundation Trust · GB

Funding

Versus Arthritis 21791
6 · The paper itself

Abstract

objectiveThe aim was to carry out a retrospective review of the efficacy and safety of anakinra in paediatric patients with undifferentiated autoinflammatory disease (uAID).

methodsWe 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.

resultsThis 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.

conclusionRetrospectively, 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.

Indexed as

anakinrachildIL-1 receptor antagonistunclassified autoinflammatory diseaseundifferentiated autoinflammatory disease

Identifiers

PMID31431992
PMCPMC6649913
OpenAlexW2914847852

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.