Treatment of Muckle-Wells syndrome: analysis of two IL-1-blocking regimens

Kuemmerle‐Deschner, Jasmin; Wittkowski, Helmut; Tyrrell, Pascal N.; Koetter, I.; Lohse, Peter; Ummenhofer, Katharina; Reess, Fabian; Hansmann, Sandra; Koitschev, Assen; Deuter, Christoph; Bialkowski, Anja; Foell, Dirk; Benseler, Susanne M.

doi:10.1186/ar4237

Cited by 62 publications

(76 citation statements)

References 35 publications

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“…In one of these patients, the dose was increased up to 500 mg/day. This finding supports experience of Kuemmerle-Deschner et al with CAPS patients failing to respond to anakinra but having a good response to canakinumab [20]. The disappearance of proteinuria and the normal kidney function in our only amyloidosis patient is promising.…”

Section: Discussionsupporting

confidence: 89%

Treatment of colchicine-resistant Familial Mediterranean fever in children and adolescents

et al. 2015

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Familial Mediterranean fever (FMF) is the most common autoinflammatory disease worldwide. Approximately 5-10 % of patients are unresponsive to colchicine. Aim of this study was to determine the short- and long-term efficacy and safety of anti-interleukin 1 (anti-IL1) and anti-tumor necrosis factor agents in colchicine-resistant FMF cases in Turkish children and adolescents. This is a single-center retrospective case series of colchicine-resistant FMF patients. The included patients were treated with biologics for either colchicine resistance or because of one of the following: (1) amyloidosis, (2) recurrent prolonged febrile myalgia and frequent need of steroid and (3) persistent arthritis. Colchicine resistance was defined as at least one attack per month for three consecutive months and elevated erythrocyte sedimentation rate or C-reactive protein or serum amyloid A in-between attacks despite taking adequate dose of colchicine. Response to biologicals was evaluated by the Autoinflammatory Diseases Activity Index (AIDAI) score sheet, patients/parents'/physicians' global assessment of disease severity and laboratory parameters every 3-6 months. Fourteen patients were included in the study. Three patients were treated with etanercept for median 7 months (range 3-11 months), and all patients had to be switched to anti-IL1 treatment because of adverse effects and/or partial response. Eleven patients were treated with anakinra with a median duration of 8 months (4-60 months). Nine patients responded to treatment at the third month, but four of them switched to canakinumab because of noncompliance, local side effects and active arthritis. Nine patients were treated with canakinumab, all responded. At follow-up, in two patients the dose had to be increased, and on the other hand, in three patients the interval was increased to every 12-16 weeks. In three patients, anti-IL1 treatment could be stopped and they are fine with colchicine. This case series describes the largest cohort of colchicine-resistant FMF patients in childhood and adolescence. Anti-IL1 treatment is a safe and effective therapy to control inflammation. The treatment should be modified and decided for each patient on an individual basis.

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Section: Discussionsupporting

confidence: 89%

Treatment of colchicine-resistant Familial Mediterranean fever in children and adolescents

et al. 2015

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“…Most adverse events were linked to injection-site reaction, gastrointestinal intolerance and mild URTI [39]. (14) has shown some differences in terms of efficacy: The short-term efficacy was very high and comparable in both groups, but at last follow-up (median 52 months), 75% of anakinra-treated and 93% of canakinumab-treated patients remained in complete remission [44]. Patients rolled over from anakinra to canakinumab achieved the same complete responses.…”

Section: An Analysis Of the Data Presented In The Reviewmentioning

confidence: 64%

“…However, CINCA cohort data have evidenced some effect of anakinra in ear inflammation on MRI, on CSF cellularity and in eye inflammation, suggesting a possible diffusion of anakinra in those fluids and tissues, while administered at high dosages reaching 8-10 mg/kg/day [37,38,39]. There are also some reports of hearing loss stabilization or improvement under canakinumab, but perhaps not described as thoroughly as with anakinra [43,44]. The safety of anakinra in patients with CAPS and FMF-related end-stage renal disease and amyloidosis appeared good in few case reports.…”

Section: An Analysis Of the Data Presented In The Reviewmentioning

confidence: 93%

“…One prefilled glass syringe contains: 0.67 ml (100 mg) of anakinra in a solution (pH 6.5) containing sodium citrate (1.29 mg), sodium chloride (5.48 mg), disodium EDTA (0.12 mg) and polysorbate 80 (0.70 mg) in Water for [25] Lepore et al [43,44] Olivecrona et al Injection, USP. The syringe is graduated to allow dosing in steps of 10 mg, starting at 20 mg.…”

Section: Drug Presentationmentioning

confidence: 99%

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Anakinra for cryopyrin-associated periodic syndrome

Koné‐Paut

Galeotti

2013

Expert Review of Clinical Immunology

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Cryopyrin-associated periodic syndrome (CAPS) is a very rare auto-inflammatory syndrome, which has recently served as a pure model of IL-1β-driven diseases. CAPS is caused by mutations into the NLRP3 gene that encodes crypoyrin, which serves as a receptor of the innate immunity that senses danger signals and pathogens. Constitutive activation of cryopyrin in CAPS leads to an excessive secretion of IL-1β. CAPS patients experience symptoms of systemic inflammation, intense fatigue and have poor quality of life. In the most severe forms, they may develop serious organ damage such as visual and hearing impairment, neurological deterioration and renal insufficiency. Anti-IL-1 drugs are effective in treating symptoms of almost all CAPS patients and have radically transformed their lives. We describe the history of the 'revival' of CAPS patients through anti-IL-1 treatments with a special focus on anakinra, the first drug used in cohorts with variable disease severity and number of patients.

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“…Starting dose of anakinra varied between 0.5 and 2 mg/kg/day (children) or 100 mg (adults) subcutaneously, but some patients, especially young children, required dose escalation up to 5 or 8 mg/kg/day to achieve sustained remission 31 42. A study on pharmacokinetics of anakinra supports this finding: in order to reach the same effective steady-state concentration, young children need higher doses of anakinra 44…”

Section: Resultsmentioning

confidence: 98%

Recommendations for the management of autoinflammatory diseases

et al. 2015

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: Autoinflammatory diseases are characterised by fever and systemic inflammation, with potentially serious complications. Owing to the rarity of these diseases, evidence-based guidelines are lacking. In 2012, the European project Single Hub and Access point for paediatric Rheumatology in Europe (SHARE) was launched to optimise and disseminate regimens for the management of children and young adults with rheumatic diseases, facilitating the clinical practice of paediatricians and (paediatric) rheumatologists. One of the aims of SHARE was to provide evidence-based recommendations for the management of the autoinflammatory diseases cryopyrin-associated periodic syndromes (CAPS), tumour necrosis factor (TNF) receptor-associated periodic syndrome (TRAPS) and mevalonate kinase deficiency (MKD). These recommendations were developed using the European League Against Rheumatism standard operating procedure. An expert committee of paediatric and adult rheumatologists was convened. Recommendations derived from the systematic literature review were evaluated by an online survey and subsequently discussed at a consensus meeting using Nominal Group Technique. Recommendations were accepted if more than 80% agreement was reached. In total, four overarching principles, 20 recommendations on therapy and 14 recommendations on monitoring were accepted with ≥80% agreement among the experts. Topics included (but were not limited to) validated disease activity scores, therapy and items to assess in monitoring of a patient. By developing these recommendations, we aim to optimise the management of patients with CAPS, TRAPS and MKD

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Treatment of Muckle-Wells syndrome: analysis of two IL-1-blocking regimens

Cited by 62 publications

References 35 publications

Treatment of colchicine-resistant Familial Mediterranean fever in children and adolescents

Treatment of colchicine-resistant Familial Mediterranean fever in children and adolescents

Anakinra for cryopyrin-associated periodic syndrome

Recommendations for the management of autoinflammatory diseases

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