Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-centre, real-world experience

Russo, Ricardo; MeloGomes, S; Lachmann, HJ; Wynne, Karen; Rajput, Kaukab; Eleftheriou, Despina; Edelsten, Clive; Hawkins, Philip N.; Brogan, Paul A.

doi:10.1093/rheumatology/ket415

Cited by 40 publications

(32 citation statements)

References 16 publications

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“…Canakinumab, a selective, human anti–IL‐1β monoclonal antibody, has been proven to be efficacious in patients with CAPS ≥2 years of age but long‐term follow‐up data for this age group have not previously been published. Canakinumab has also been reported to not affect the induction or persistence of antibody responses after immunization with unadjuvanted influenza or alum‐adjuvanted meningococcal vaccines in healthy subjects ages 18–45 years .…”

Section: Introductionmentioning

confidence: 99%

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

Brogan

Hofer

Kuemmerle‐Deschner³

et al. 2019

Arthritis & Rheumatology

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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).Methods CAPS 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.

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Section: Introductionmentioning

confidence: 99%

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

Brogan

Hofer

Kuemmerle‐Deschner³

et al. 2019

Arthritis & Rheumatology

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“…опубликова-ли результаты многоцентрового 48-недельного двойно-го слепого рандомизированного исследования (III фаза) по оценке эффективности канакинумаба у пациентов с CAPS [19]. В 1-е сут у всех больных купировались основ-ные симптомы заболевания, у подавляющего большин-ства (97%) наблюдался полный ответ к 15-м сут после Необходимость увеличения дозы канакинумаба у паци-ентов с CINCA/NOMID была продемонстрирована и в ряде других исследований, в частности у 10 человек с CAPS с длительностью наблюдения в рутинной клинической практике от 1 до 3 лет [25]. В работе C. H. Sibley и соавт.…”

Section: Discussionunclassified

Administration of the Interleukin-1 Inhibitor Canakinumab in Children with Autoinflammatory Diseases and Systemic Juvenile Arthritis: the Report for Case Series

Салугина¹,

Каледа²,

Фёдоров³

et al. 2016

Vopr. sovr. pediatr.

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“…All three conditions can be managed using biological agents that inhibit IL‐1 . This includes anakinra, rilonacept and canakinumab …”

Section: Cryopyrin‐associated Periodic Syndromesmentioning

confidence: 99%

Periodic fevers and autoinflammatory syndromes in childhood

Ostring

Singh‐Grewal

2016

J Paediatrics Child Health

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Recurrent fever is a common presentation in paediatric practice and can be caused by a wide variety of diseases including autoinflammatory conditions. The innate immune system plays an essential role in the 'first line' response to infection through mediation of inflammatory responses. Inflammasomes are part of the regulatory process for this system and result in the production of the powerful pro-inflammatory cytokine interleukin-1B. Dysregulation of inflammasomes, and Interleukin 1 production, contributes to the pathogenesis of autoinflammatory diseases. This review focuses on described periodic fever syndromes (PFS) which are now collectively referred to as autoinflammatory syndromes. Conditions discussed include periodic fever aphthous stomatitis pharyngitis and cervical adenopathy, familial Mediterranean fever, tumour necrosis factor receptor-associated periodic syndromes, hyperimmunoglobulinaemia D and the cryopyrin-associated periodic syndromes. Presenting features, complications, diagnostic and treatment approaches for these conditions are discussed. Nonetheless, as most of these conditions are rare and may have significant long-term complications, it is recommended that they be managed in consultations with a physician experienced in managing PFS.

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Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-centre, real-world experience

Abstract: Canakinumab, although costly, is a safe and effective treatment for CAPS in children, leading to sustained improvement in disease activity, serological markers, functional ability and HRQoL.

Cited by 40 publications

References 16 publications

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

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

Administration of the Interleukin-1 Inhibitor Canakinumab in Children with Autoinflammatory Diseases and Systemic Juvenile Arthritis: the Report for Case Series

Periodic fevers and autoinflammatory syndromes in childhood

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