Evidence map›Paper›PMID 39724475›Full record

ArticleRheumatology and therapy2025

Long-Term Safety and Effectiveness of Canakinumab in Patients with MKD/HIDS: Interim Analysis of the RELIANCE Registry.

Prasad T Oommen, Tilmann Kallinich, Juergen Rech, Norbert Blank, Julia Weber-Arden, Jasmin B Kuemmerle-Deschner

Abstract read
In one paragraph

Article in Rheumatology and therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Review
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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

6 authors.

Prasad T OommenDivision of Paediatric Rheumatology, Department of Paediatric Oncology, Haematology and Clinical Immunology, Medical Faculty, Heinrich-Heine-University Duesseldorf, Duesseldorf, Germany. Prasad.Oommen@med.uni-duesseldorf.de.ORCID http://orcid.org/0000-0003-2893-7252
Tilmann KallinichDepartment of Paediatric Respiratory Medicine, Immunology and Critical Care Medicine, Charité Universitätsmedizin, Berlin, Germany.ORCID http://orcid.org/0000-0003-2404-9397
Juergen RechDepartment of Internal Medicine 3, Rheumatology and Immunology, Friedrich-Alexander University (FAU) Erlangen-Nürnberg and Universitätsklinikum Erlangen, Erlangen, Germany.ORCID http://orcid.org/0000-0002-2569-2029
Norbert BlankDivision of Rheumatology, Department of Internal Medicine, Heidelberg University Hospital, Heidelberg, Germany.ORCID http://orcid.org/0000-0002-9419-1690
Julia Weber-ArdenNovartis Pharma GmbH, Nuernberg, Germany.
Jasmin B Kuemmerle-DeschnerDivision of Paediatric Rheumatology and Autoinflammation Reference Centre Tübingen, Department of Paediatrics, University Hospital Tübingen; Member of ERN-RITA, Tübingen, Germany.ORCID http://orcid.org/0000-0002-6365-6598

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionInterim analysis of the long-term safety and effectiveness of canakinumab, at a patient level, in the mevalonate kinase deficiency/hyperimmunoglobulin-D syndrome (MKD/HIDS) cohort of the RELIANCE registry.

methodsFrom June 2018, the RELIANCE registry enrolled paediatric (aged ≥ 2 years) and adult patients (aged ≥ 18 years) with MKD/HIDS who were receiving canakinumab as part of their routine medical care. Safety, physician- and patient-reported measures of disease activity and dosing patterns were evaluated at baseline and every 6 months until end-of-study visit.

resultsAt the analysis cut-off date of December 2022, eight patients with MKD/HIDS were enrolled. Five (62.5%) were children (< 18 years) and five (62.5%) were female. The median patient age was 8.0 (range 2.0-39.0) years, and all patients were pre-treated with canakinumab prior to enrolment (median duration of canakinumab treatment: 3.8 years). Canakinumab was well tolerated, with seven (87.5%) patients reporting 48 adverse events (incidence rate/100 patient years: 218.1). No serious adverse drug reactions were reported. Patients continued to receive vaccinations during long-term treatment with canakinumab. Disease activity, evaluated by physician-reported (physician's global assessment, disease remission, C-reactive protein, serum amyloid A, erythrocyte sedimentation rate) and patient-reported (autoinflammatory disease activity index diary, disease activity, fatigue, impact on social life) measures, was generally well controlled throughout the study. Over 50.0% of patients maintained disease remission from baseline to month 24, and medians of all inflammatory markers remained within normal limits throughout the study. Most patients received higher than the recommended starting dose of canakinumab throughout the study.

conclusionData from this interim analysis of a unique registry of patients with a rare disease support the long-term safety and effectiveness of the IL-1-blocking agent canakinumab for the treatment of MKD/HIDS.

Indexed as

Autoinflammatory diseaseCanakinumabFever syndromesHyperimmunoglobulin-d syndromeMevalonate kinase deficiency

Identifiers

PMID39724475
PMCPMC11751260

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