Novel Therapeutics for the Treatment of Familial Mediterranean Fever: From Colchicine to Biologics

Grattagliano, Ignazio; Bonfrate, Leonilde; Ruggiero, Valentina; Scaccianoce, Giuseppe; Palasciano, Giuseppe; Portincasa, Piero

doi:10.1038/clpt.2013.148

Cited by 67 publications

(53 citation statements)

References 69 publications

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“…However, it has been demonstrated that polymorphonuclear neutrophils are also a source of ChT (26). Colchicine inhibits the chemotaxis of neutrophils and the release of arachidonate and 5-lipoxygenase in macrophages (27). In our study, 76 patients were receiving colchicine.…”

Section: Discussionmentioning

confidence: 99%

Decreased Chitotriosidase Activity and Levels in Familial Mediterranean Fever

et al. 2016

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Different studies have demonstrated changes in chitotriosidase (ChT) activity and concentrations in multiple diseases. However, changes in ChT activity and concentrations have not been concurrently evaluated in patients with Familial Mediterranean Fever (FMF). In this study, we analyzed the changes in serum ChT activity and concentrations in patients with FMF. The study included a total of 80 patients with FMF and 80 healthy controls. ChT enzyme activity and concentrations were measured and then compared between the groups. ChT activity was measured by using fluorometric ELISA and ChT concentrations were measured by using colorimetric ELISA methods. The median ChT activity was 10.00 (6.00–15.00) nmol/mL/hr in the patients and 14.00 (6.25–20.75) nmol/mL/hr in the controls. There was a statistically significant difference in the ChT activity between the controls and patients (P = 0.027). The median ChT concentrations were 65.40 (46.20–84.92) pg/mL and 125.00 (75.72–143.95) pg/mL in the patients and controls, respectively (P < 0.001), which were expressed as median percentiles (25th–75th). Additionally, we found no correlation between C-reactive protein and ChT activity (P = 0.978, r = 0.003) and concentrations (P = 0.446, r = −0.87). Serum ChT enzyme activity and concentrations may not be considered as a biomarker in FMF patients taking colchicine. New studies are needed to evaluate the changes of enzyme activity and concentration in colchicine-negative patients.

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Section: Discussionmentioning

confidence: 99%

Decreased Chitotriosidase Activity and Levels in Familial Mediterranean Fever

et al. 2016

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“…In the later group, daily dose is decreased to alternate day or thrice a week after clinical remission is reached or rarely increased two-to threefold the recommended dose in case of relapse or partial response. The dose is also modifi ed in patients on hemodialysis [ 46 ]. Among the three anti-IL-1 agents, anakinra is the most extensively used one.…”

Section: Anakinramentioning

confidence: 99%

The Emerging Treatments in Familial Mediterranean Fever

Özdoğan

Uğurlu

2015

Rare Diseases of the Immune System

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9As described in detail by Ben-Chetrit in the previous chapter, colchicine is the mainstay of the treatment of familial Mediterranean fever (FMF) [ 1 , 2 ]. If used daily for lifelong, it controls the febrile attacks in the majority of the patients and prevents the development of amyloidosis, the most serious complication of the disease.Colchicine is very effective as shown in controlled trials [ 3 ] and within the therapeutic range. Side effects are rare and/or mild, so that it can be used even during pregnancy and lactation, plus it is very cheap; however, even colchicine has some limitations in the treatment of FMF:1. Response to therapy: A small proportion of patients with FMF continue to experience attacks despite proper dose and good compliance to colchicine. Overall, it has been accepted that colchicine prevents febrile attacks in more than 60 % of patients and signifi cantly reduces the number of attacks in another 20-30 %. Five to ten percent of patients do not respond to therapy [ 4 , 5 ].Compliance is a main issue among patients who report attacks under colchicine treatment. Approximately 40 % of the patients are not compliant and receive lower than the dose needed to control the infl ammatory attacks and thus prone to high risk of amyloidosis [ 6 ]. Therefore, it is very important to check the compliance and the daily dose before diagnosing the patient as unresponsive to colchicine therapy. Recently the data that comes from the Eurofever Registry which collects information on patients with various autoinfl ammatory diseases, including FMF, showed that among the 121 FMF patients on colchicine, 62 % reported complete, 36 % partial, and 2 % no response [ 7 ]. In another recent report, only 5 % of the compliant patients on proper dose of colchicine continued to experience frequent attacks [ 8 ].

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“…55 Anakinra is a response modifier that acts by antagonizing the biological effects of IL1, which is a proinflammatory cytokine. The drug is currently approved by the FDA for the treatment of rheumatoid arthritis.…”

Section: Anakinra and Biological Agentsmentioning

confidence: 99%

Recurrent pericarditis: new and emerging therapeutic options

et al. 2015

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Recurrent pericarditis is one of the most common and troublesome complications after an episode of pericarditis, and affects 20-50% of patients treated for pericarditis. In most of these patients, the pericarditis remains idiopathic, although an immune-mediated (either autoimmune or autoinflammatory) pathogenesis is often presumed. The mainstay of therapy for recurrences is aspirin or NSAIDs, with the adjunct of colchicine. Corticosteroids are a second-line option to be considered for specific indications, such as connective tissue disease or pregnancy; contraindications or intolerance to aspirin, NSAIDs, and/or colchicine; or insufficient response to these medications. Furthermore, corticosteroids can be added to NSAIDs and colchicine in patients with persistent symptoms. In patients who do not respond adequately to any of these conventional therapies, alternative treatment options include azathioprine, intravenous human immunoglobulins, and anakinra. An improved understanding of how recurrent pericarditis develops after an initiating event is critical to prevent this complication, and further research is needed into the pathogenesis of recurrences. We discuss the aetiology and diagnosis of recurrent pericarditis, and extensively review the treatment options for this condition.

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Novel Therapeutics for the Treatment of Familial Mediterranean Fever: From Colchicine to Biologics

Cited by 67 publications

References 69 publications

Decreased Chitotriosidase Activity and Levels in Familial Mediterranean Fever

Decreased Chitotriosidase Activity and Levels in Familial Mediterranean Fever

The Emerging Treatments in Familial Mediterranean Fever

Recurrent pericarditis: new and emerging therapeutic options

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