Evidence map›Paper›PMID 31161734›Full record

Trial reportArthritis & rheumatology (Hoboken, N.J.)2019

Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger.

Paul A Brogan, Michael Hofer, Jasmin B Kuemmerle-Deschner, Isabelle Koné-Paut, Joachim Roesler, Tilmann Kallinich, Gerd Horneff, Inmaculada Calvo Penadés, Belén Sevilla-Perez, Laurence Goffin and 5 more

Open access · hybridAbstract readClinical Trial, Phase III
In one paragraph

Trial report in Arthritis & rheumatology (Hoboken, N.J.), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 3 pooled it
2.7field-weighted citation impact, top 9% 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

27 citing papers in PubMed, 3 syntheses or guidelines pooled it, 50 citations in OpenAlex.

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  14. NLRP3 inflammasome in neurodegenerative disease.Translational research : the journal of laboratory and clinical medicine · 2023
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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

15 authors at 14 institutions in 9 countries.

Paul A BroganUCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Foundation Trust, London, UK.
Michael HoferCentre Hospitalier Universitaire Vaudois, University of Lausanne, Lausanne, Switzerland, and Hôpitaux Universitaires de Genève, Geneva, Switzerland.
Jasmin B Kuemmerle-DeschnerUniversity Hospital Tuebingen, Tuebingen, Germany.
Isabelle Koné-PautCeRéMAIA, AP-HP, Bicêtre Hospital, University of Paris Sud, Le Kremlin-Bicêtre, France.
Joachim RoeslerUniversitätsklinikum Carl Gustav Carus Dresden, Dresden, Germany.
Tilmann KallinichCharité University Berlin, Berlin, Germany.
Gerd HorneffAsklepios Klinik Sankt Augustin GmbH, St. Augustin, Germany, and University of Cologne, Cologne, Germany.
Inmaculada Calvo PenadésHospital Universitario y Politécnico La Fe, Valencia, Spain.
Belén Sevilla-PerezClinico San Cecilio, Granada, Spain.
Laurence GoffinHôpital Universitaire Des Enfants Reine Fabiola, Université Libre de Bruxelles, Brussels, Belgium.
Bernard R LauwerysCliniques Universitaires Saint-Luc and Université Catholique de Louvain, Brussels, Belgium.
Helen J LachmannUniversity College London Royal Free Campus, London, UK.
Yosef UzielMeir Medical Center, Sackler School of Medicine, Tel Aviv University, Tel Aviv, Israel.
Xiaoling WeiChina Novartis Institutes for Biomedical Research Company, Ltd, Beijing, China.
Ronald M LaxerUniversity of Toronto and the Hospital for Sick Children, Toronto, Ontario, Canada.
Asklepios Klinik Sankt Augustin · DECharité - Universitätsmedizin Berlin · DECliniques Universitaires Saint-Luc · BEGreat Ormond Street Hospital for Children NHS Foundation Trust · GBHospital Universitari i Politècnic La Fe · ESInstituto de Investigación Biosanitaria de Granada · ESNovartis (China) · CNQueen Fabiola Children's University Hospital · BERoyal Free London NHS Foundation Trust · GBTel Aviv University · ILUniversité Paris-Sud · FRUniversity Hospital Carl Gustav Carus · DEUniversity Hospital of Lausanne · CHUniversity of Toronto · CA

Funding

NIHR Great Ormond Street Hospital Biomedical Research CentreNovartis Pharma AG, Switzerland
6 · The paper itself

Abstract

objectiveTo assess long-term efficacy and safety of canakinumab and the response to vaccination in children ages ≤5 years with cryopyrin-associated periodic syndrome (CAPS).

methodsCAPS patients (ages ≤5 years) received 2 mg/kg canakinumab subcutaneously every 8 weeks; patients with neonatal-onset multisystem inflammatory disease (NOMID) received a starting dose of 4 mg/kg in this open-label trial. Efficacy was evaluated using physician global assessment of disease activity and serum levels of C-reactive protein (CRP) and amyloid A (SAA). Adverse events (AEs) were recorded. Vaccination response was evaluated using postvaccination antibody titers at 4 and 8 weeks after immunization.

resultsOf the 17 patients enrolled, 12 (71%) had Muckle-Wells syndrome, 4 (24%) had NOMID, and 1 (6%) had familial cold autoinflammatory syndrome. All 17 patients had a complete response to canakinumab. Disease activity improved according to the physician global assessment, and for 65% of the patients autoinflammatory disease was characterized as "absent" at the end of the study. Median CRP levels decreased over time. No such change was evident in SAA levels. During the extension study, postvaccination antibody titers increased above protective levels in 16 (94%) of 17 assessable vaccinations. Ten of the patients (59%) had AEs suspected to be related to canakinumab; 8 (47%) experienced at least 1 serious AE (SAE). None of the AEs or SAEs required interruption of canakinumab therapy.

conclusionOur findings indicate that canakinumab effectively maintains efficacy through 152 weeks and appears to have no effect on the ability to produce antibodies against standard childhood non-live vaccines. The safety profile of canakinumab was consistent with previous studies, supporting long-term use of canakinumab for CAPS in children ≤5 years of age.

Indexed as

Antibodies, Monoclonal, HumanizedAntibody FormationChild, PreschoolC-Reactive ProteinCryopyrin-Associated Periodic SyndromesDiarrheaFemaleFeverHumansInfantInfant, NewbornMaleNasopharyngitisRespiratory Tract InfectionsSerum Amyloid A ProteinTreatment OutcomeAntibodies, Monoclonal, HumanizedcanakinumabC-Reactive ProteinSerum Amyloid A ProteinVaccines

Identifiers

PMID31161734
PMCPMC6899890
OpenAlexW2948040333

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.