Nationwide Experience With Off‐Label Use of Interleukin‐1 Targeting Treatment in Familial Mediterranean Fever Patients

Akar, Servet; Cetin, P.; Kalyoncu, Umut; Karadağ, Ömer; Sarı, İ̇smail; Çınar, M.; Yılmaz, Sedat; Onat, Ahmet Mesut; Kısacık, Bünyamin; Erden, Abdülsamet; Balkarlı, Ayşe; Küçükşahin, Orhan; Öner, Setenay; Şenel, Soner; Tufan, Abdurrahman; Direşkeneli, Haner; Oksuz, Ferhat; Pehlıvan, Yavuz; Bayındır, Özün; Keser, Gökhan; Aksu, Kenan; Omma, Ahmet; Kaşifoğlu, Timuçin; Ünal, Ali; Yıldız, Fatih; Balcı, Mehmet; Yavuz, Şule; Erten, Şükran; Özgen, Metin; Sayarlıoğlu, Mehmet; Doğru, Atalay; Yildirim, Gozde; Öner, Fatma Alıbaz; Tezcan, Mehmet Engin; Pamuk, Ömer Nurı; Önen, Fatoş

doi:10.1002/acr.23446

Cited by 53 publications

(50 citation statements)

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“…Another Varan et al showed that anti-IL1 treatments significantly reduced the amount of proteinuria (from 1606 mg/day to 519 mg/day) [14]. Moreover, national data that was collected with 172 subjects demonstrated a significant decrease in proteinuria (5458.7 mg/24 h before and 3557.3 mg/24 h after) [9]. Instead, in these studies, patients were not sub-grouped, and their data was not analyzed regarding their GFR.…”

Section: Discussionmentioning

confidence: 95%

“…Some previous studies have demonstrated the efficiency of these treatments. Akar et al reported the response rate as 76.5% in the anakinra group and 67.5% in the canakinumab group in a total of 172 colchicine-resistant patients [9]. In the study by Kucuksahin et al, anti-IL-1 treatments significantly decreased the number of attacks in 26 colchicine-resistant patients with FMF.…”

Section: Discussionmentioning

confidence: 97%

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Assessment of effectiveness of anakinra and canakinumab in patients with colchicine-resistant/unresponsive familial Mediterranean fever

et al. 2020

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İntroduction: Familial Mediterranean fever (FMF) is a hereditary auto-inflammatory disease characterized by recurrent fever and serosal inflammation. Anti-interleukin-1 (Anti-IL-1) treatments are recommended in colchicine resistant and/or intolerant FMF patients. This study aims to evaluate the efficacy of anakinra and canakinumab in FMF patients that are resistant/intolareted to colchicine or complicated with amyloidosis. who were diagnosed with FMF according to the criteria of Tel-Hashomer were included in the study. The laboratory values and clinical features of patients and disease activities were recorded at least every 3 months, and these data were analyzed.Results: Forty-one (63.1%) patients used anakinra (100 mg/day) and 24 (36.9%) patients used canakinumab (150 mg/8 week). The median duration of anti-IL-1 agents use was 7 months (range, 3-30). Fifteen (23.1%) cases were complicated with amyloidosis. Seven (10.8%) patients had renal transplantation. Overall, the FMF 50 score response was 96.9%. In the group that had a glomerular filtration rate (GFR) ≥ 60 ml/min/m 2 , the median proteinuria decreased from 2390 mg/day (range, 1400-7200) to 890 mg/day (range, 120-2750) (p = 0.008). No serious infections were detected, except in one patient.Conclusions: Anti-IL-1 agents are effective and safe in the treatment of FMF patients. These agents are particularly effective at reducing proteinuria in patients with GFR ≥ 60 ml/min/m 2 , but less effective in cases with FMF associated with arthritis and sacroiliitis. Large and long follow-up studies are now needed to establish the longterm effects of these treatments.

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Section: Discussionmentioning

confidence: 95%

Section: Discussionmentioning

confidence: 97%

Assessment of effectiveness of anakinra and canakinumab in patients with colchicine-resistant/unresponsive familial Mediterranean fever

et al. 2020

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“…Biological therapies to treat FMF have been recently reviewed and include anakinra, an IL-1 receptor antagonist; rilonacept, a dimeric fusion protein that blocks the IL-1 receptor; and canakinumab, a human monoclonal anti-IL-1β antibody [55]. Recent studies in FMF patients with colchicine resistance have shown a significant reduction in attack frequency with anakinra or canakinumab [56,57], with a complete response up to 16 weeks of~70% for canakinumab [58]. Other agents that have been considered but with limited evidence to date include etanercept, a fusion protein targeting the tumour necrosis factor-α receptor; infliximab, a chimeric monoclonal antibody to TNF-α [59]; tocilizumab, a humanized monoclonal antibody targeting the IL-6 receptor [60]; and tofacitinib, a Janus kinase inhibitor [61].…”

Section: An Argument For Biologically-informed Treatmentmentioning

confidence: 99%

Periodic fever syndromes: beyond the single gene paradigm

et al. 2019

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Familial Mediterranean fever (FMF) is the most common monogenic autoinflammatory disease in Canada and is characterized by a clinical syndrome of episodic inflammatory symptoms. Traditionally, the disease is defined by autosomal recessive inheritance of MEFV gene variants, yet FMF also not uncommonly manifests in individuals with only one identified disease-associated allele. Increasing availability and affordability of gene sequencing has led to the identification of multiple MEFV variants; however, they are often of unknown clinical significance. Variants in other genes affecting overlapping or distinct inflammatory signaling pathwaystogether with gene-environment interactions including epigenetic modulationlikely underlie the significant genetic and phenotypic heterogeneity seen among patients with this disease. We review recent evidence of the expanding spectrum of FMF genotype and phenotype and suggest that current drug funding schemes restricting biologic agents to patients with homozygous mutations have not kept pace with our biological understanding of the disease.

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“…Подобные случаи -не редкость, у пациентов с FMF при-ступы лихорадки при приеме колхицина сохраняются в 30-45% случаев. В то же время назначение ингибиторов ИЛ1 у 42% пациентов с FMF приводит к полному контролю забо-левания, а в остальных случаях примерно в семь раз снижает частоту приступов лихорадки и более чем в полтора раза про-теинурию [40]. Основные принципы лечения АВЗ, сформи-рованные на основании анализа данных литературы в рамках инициативы EUROFEVER, предполагают применение у па-циентов с плохо контролируемыми MVKD, TRAPS, PAPA и FMF в качестве прпаратов выбора ингибиторов ИЛ1 или ФНОα [41].…”

Section: криопирин-ассоциированные периодические синдромыunclassified

Hereditary periodic fever syndromes in adult rheumatology practice

Желябина¹,

Елисеев²,

Чикина³

2018

RJTAO

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Аутовоспалительные заболевания (АВЗ) -гетероген-ная группа редких генетически детерминированных, на-следственно обусловленных состояний, характеризую-щихся непровоцируемыми приступами воспаления, ма-нифестирующими рецидивирующей лихорадкой и кли-нической симптоматикой, напоминающей ревматиче-ские проявления, при отсутствии аутоиммунных или ин-фекционных причин. По данным эпидемиологических исследований последних лет, распространенность АВЗ растет во всем мире, что обусловлено улучшением их ди-агностики и лечения. Так, если раньше исход многих за-болеваний был предрешен и курация таких пациентов сводилась к симптоматическому лечению и наблюдению, то в настоящее время возможности генетической диагно-стики и патогенетической терапии значительно расши-рились [1,2].Термин «аутовоспалительные заболевания» появился недавно. В конце ХХ столетия D. Kastner и J. O`Shea пред-ложили термин «аутовоспаление» [1]. Первое описание АВЗ принадлежит выдающемуся английскому клиницисту У. Ге-бердену и датировано 1802 г., но тогда оно осталось без вни-мания [3].

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Nationwide Experience With Off‐Label Use of Interleukin‐1 Targeting Treatment in Familial Mediterranean Fever Patients

Abstract: Anti-IL-1 treatment is an effective alternative for controlling attacks and decreasing proteinuria in colchicine-resistant FMF patients.

Cited by 53 publications

References 20 publications

Assessment of effectiveness of anakinra and canakinumab in patients with colchicine-resistant/unresponsive familial Mediterranean fever

Assessment of effectiveness of anakinra and canakinumab in patients with colchicine-resistant/unresponsive familial Mediterranean fever

Periodic fever syndromes: beyond the single gene paradigm

Hereditary periodic fever syndromes in adult rheumatology practice

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