Article
Anti-Il 1 Therapy in patients with FMF-related amyloidosis living in Germany
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Published: | August 29, 2016 |
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Background: Familial Mediterranean fever (FMF) is an autosomal recessive autoinflammatory disease characterized by recurrent, self-limiting attacks of fever, and serositis. Renal amyloidosis is a serious complication of FMF that determines the prognosis. Patients with AA-Amyloidosis can be treated with Interleukin-1 (IL-1)-inhibiting drugs.We report our single center experience in adult patients with colchicine-resistant FMF-related amyloidosis who were treated with colchicine and anakinra.
Methods: Demographic data, clinical and laboratory parameters, MEFV mutations, patient reported outcomes and physician global health were extracted from our GARROD (German Anti-IL1beta medication RegistRy in Orphan Diseases) registry.
Results: Within our cohort of 205 adult patients with FMF, we identified 36 patients (17.6%) with FMF-related amyloidosis. All patients received colchicine treatment. A subgroup of these patients continued to have colchicine-resistant FMF attacks and elevated acute phase reactants in the serum. Nine patients (2 female and 7 male) were treated with a combination of colchicine (1.5 +/- 0.65 mg per day) and Anakinra. All patients were of turkish-armenian ancestry. Homozygosity for high penetrance mutation was present in 7 patients, heterozygosity in 2 patients. Three patients (33%) presented with nephrotic syndrome, 3 patients (33%) with chronic kidney disease (CKD), one with end-stage renal disease (ESRD), and 2 patients were 5 and 7 years after renal transplantation.
Before initiation of anakinra the 24h urine protein excretion (mean +/-SD) was 5.0g +/-3.5g /24h. The proteinuria decreased with anakinra to 0.4g +/-1g /24h in the last visit after 24 +/- 18 months. The creatinine serum levels of all patients with CKD decreased or remained stable.
The patient reported health (VAS 70 +/- 21mm) and the physician reported global health (VAS 80 +/- 16mm) both improved significantly (VAS 10 +/- 20mm, VAS 7.5 +/- 20mm) (p 0.001, p 0.0009) after 6 months of treatment.
Conclusion: Anakinra is well tolerated and effective in patients with FMF-related amyloidosis.