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Molecular and Cellular Pediatrics

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Open Access 01-12-2016 | Case Study

The experience of canakinumab in renal amyloidosis secondary to Familial Mediterranean fever

Authors: Betul Sozeri, Nesrin Gulez, Malik Ergin, Erkin Serdaroglu

Published in: Molecular and Cellular Pediatrics | Issue 1/2016

Abstract

Introduction

Familial Mediterranean fever (FMF) is an autosomal recessive disease characterized by self-limited recurrent attacks of fever and serositis. Patients may develop renal amyloidosis. Colchicine prevents attacks and renal amyloidosis. Five to 10 % of the patients with FMF are resistant or intolerant to colchicine.

Case description

Herein, we reported our experience with clinical-laboratory features and treatment responses of a pediatric FMF patient with amyloidosis treated with canakinumab. We observed a significant decrease in proteinuria and increase growth in the patient.

Discussion and evaluation

The most serious complication of FMF is the development of AA type amyloidosis which is characterized by proteinuria. Colchicine is the prototype drug that decreases production of amyloidogenic precursor protein. Occasionally, colchicine inadequate patient is observed, as in our case. Canakinumab is a human anti-IL-1β monoclonal antibody. Previously, canakinumab efficacy were shown in a limited number of studies.

Conclusions

Our data, though limited to only one patient, emphasize that therapeutic intervention with canakinumab seems to be improve kidney function in colchicine-resistant FMF with renal amyloidosis.

Ben-Zvi I, Livneh A (2011) Chronic inflammation in FMF: markers, risk factors, outcomes and therapy. Nat Rev Rheumatol 7:105–112CrossRef

The International FMF Consortium (1997) Ancient missense mutations in a new member of the RoRet gene family are likely to cause familial Mediterranean fever. Cell 90:797CrossRef

French FMF Consortium (1997) A candidate gene for familial Mediterranean fever. Nat Genet 17:25CrossRef

Grattagliano I, Bonfrate L, Ruggiero V, Scaccianoce G, Palasciano G, Portincasa P (2014) Novel therapeutics for the treatment of familial Mediterranean fever: from colchicine to biologics. Clin Pharmacol Ther 95:89–97CrossRefPubMed

Ozen S, Bilginer Y (2014) A clinical guide to autoinflammatory diseases: Familial Mediterranean fever and next-of-kin. Nat Rev Rheumatol 10:135–147CrossRefPubMed

Merlini G, Bellotti V (2003) Molecular mechanisms of amyloidosis. N Engl J Med 349:583–596CrossRefPubMed

Hentgen V, Grateau G, Kone-Paut I, Livneh A, Padeh S, Rozenbaum M, Amselem S, Gershoni-Baruch R, Touitou I, Ben-Chetrit E (2013) Evidence-based recommendations for the practical management of Familial Mediterranean fever. Semin Arthritis Rheum 43:387–391CrossRefPubMed

Ter Haar N, Lachmann H, Ozen S, Woo P, Uziel Y, Modesto C, Koné-Paut I, Cantarini L, Insalaco A, Neven B, Hofer M, Rigante D, Al-Mayouf S, Touitou I, Gallizzi R, Papadopoulou-Alataki E, Martino S, Kuemmerle-Deschner J, Obici L, Iagaru N, Simon A, Nielsen S, Martini A, Ruperto N, Gattorno M, Frenkel J, Paediatric Rheumatology International Trials Organisation (PRINTO) and the Eurofever/Eurotraps Projects (2013) Treatment of autoinflammatory diseases: results from the Eurofever Registry and a literature review. Ann Rheum Dis 72:678–685CrossRef

Lidar M, Scherrmann JM, Shinar Y, Chetrit A, Niel E, Gershoni-Baruch R, Langevitz P, Livneh A (2004) Colchicine nonresponsiveness in familial Mediterranean fever: clinical, genetic, pharmacokinetic, and socioeconomic characterization. Semin Arthritis Rheum 33:273–282CrossRefPubMed

10.

Mitroulis I, Skendros P, Ritis K (2010) Targeting IL-1beta in disease; the expanding role of NLRP3 inflammasome. Eur J Intern Med 21:157–163CrossRefPubMed

11.

Moser C, Pohl G, Haslinger I, Knapp S, Rowczenio D, Russel T, Lachmann HJ, Lang U, Kovarik J (2009) Successful treatment of familial Mediterranean fever with Anakinra and outcome after renal transplantation. Nephrol Dial Transplant 24:676–678CrossRefPubMed

12.

Cetin P, Sari I, Sozeri B, Cam O, Birlik M, Akkoc N, Onen F, Akar S (2015) Efficacy of interleukin-1 targeting treatments in patients with familial Mediterranean fever. Inflammation 38:27–31CrossRefPubMed

13.

Hashkes P, Butbul Aviel Y, Lubin S, Ben-Dayan E, Tseng L, Brik R (2014) A76: long-term efficacy of canakinumab in childhood colchicine resistant familial mediterranean Fever. Arthritis Rheumatol 66:108CrossRef

14.

Gül A, Ozdogan H, Erer B, Ugurlu S, Kasapcopur O, Davis N, Sevgi S (2015) Efficacy and safety of canakinumab in adolescents and adults with colchicine-resistant familial Mediterranean fever. Arthritis Res Ther 17:243CrossRefPubMedPubMedCentral

15.

Mamou H, Cattan R (1952) La maladie periodique sur 14 cas personnels dont 8 compliqués de nephropathies. Semaine hop Paris 28:1062PubMed

16.

Twig G, Livneh A, Vivante A, Afek A, Shamiss A, Derazne E, Tzur D, Ben-Zvi I, Tirosh A, Barchana M, Shohat T, Golan E, Amital H (2014) Mortality risk factors associated with Familial Mediterranean fever among a cohort of 1.25 million adolescents. Ann Rheum Dis 73:704–709CrossRefPubMed

17.

Tunca M, Akar S, Onen F, Ozdogan H, Kasapcopur O, Yalcinkaya F, Tutar E, Ozen S, Topaloglu R, Yilmaz E, Arici M, Bakkaloglu A, Besbas N, Akpolat T, Dinc A, Erken E, Turkish FMF Study Group (2005) Familial Mediterranean fever (FMF) in Turkey: results of a nationwide multicenter study. Medicine (Baltimore) 84:1–11CrossRef

18.

Lachmann HJ, Goodman HJ, Gilbertson JA, Gallimore JR, Sabin CA, Gillmore JD, Hawkins PN (2007) Natural history and outcome in systemic AA amyloidosis. N Engl J Med 356:2361–237CrossRefPubMed

19.

Bennhold H (1922) spezifische Amyloidfarbung mit Kongorot. Munch Med Wochenschr 69:1537–1538

20.

Sen S, Sarsik B (2010) A proposed histopathologic classification, scoring, and grading system for renal amyloidosis: standardization of renal amyloid biopsy report. Arch Pathol Lab Med 134:532–544PubMed

21.

Castano E, Palmer MB, Vigneault C, Luciano R, Wong S, Moeckel G (2015) Comparison of amyloid deposition in human kidney biopsies as predictor of poor patient outcome. BMC Nephrol 16:64CrossRefPubMedPubMedCentral

22.

Oner A, Erdogan O, Demircin G, Bulbul M, Memis L (2003) Efficacy of colchicine therapy in amyloid nephropathy of familial Mediterranean fever. Pediatr Nephrol 18:521–526PubMed

23.

MacRae VE, Wong SC, Farquharson C, Ahmed SF (2006) Cytokine actions in growth disorders associated with pediatric chronic inflammatory diseases (review). Int J Mol Med 18:1011–1018PubMed

24.

Zung A, Barash G, Zadik Z, Barash J (2006) Familial Mediterranean fever and growth: effect of disease severity and colchicine treatment. J Pediatr Endocrinol 19:15CrossRef

25.

Türkmen M, Soylu OB, Kasap B, Güneş S, Tüfekçi O, Soylu A, Erçal D, Kavukçu S (2008) Growth in Familial Mediterranean fever: effect of attack rate, genotype and colchicine treatment. J Pediatr Endocrinol Metab 21:789–792CrossRefPubMed

26.

Sozeri B, Yilmaz E, Mir S, Berdeli A (2011) Effect of colchicine-resistant Familial Mediterranean fever on growth parameters. Archives of Rheum 26:1–5

27.

Bilginer Y, Ayaz NA, Ozen S (2010) Anti-IL-1 treatment for secondary amyloidosis in an adolescent with FMF and Behçet’s disease. Clin Rheumatol 29:209–210CrossRefPubMed

28.

Ozçakar ZB, Ozdel S, Yılmaz S, Kurt-Şükür ED, Ekim M, Yalçınkaya F (2014) Anti-IL-1 treatment in Familial Mediterranean fever and related amyloidosis. Clin Rheumatol 35:441–446CrossRefPubMed

29.

Topaloglu R, Batu ED, Orhan D, Ozen S, Besbas N (2015) Anti-interleukin 1 treatment in secondary amyloidosis associated with autoinflammatory diseases. Pediatr Nephrol 31:633–640CrossRefPubMed

Title: The experience of canakinumab in renal amyloidosis secondary to Familial Mediterranean fever
Authors: Betul Sozeri
Nesrin Gulez
Malik Ergin
Erkin Serdaroglu
Publication date: 01-12-2016
Publisher: Springer Berlin Heidelberg
Published in: Molecular and Cellular Pediatrics / Issue 1/2016
Electronic ISSN: 2194-7791
DOI: https://doi.org/10.1186/s40348-016-0058-2

Keynote webinar | Spotlight on medication adherence

Springer Medicine

The experience of canakinumab in renal amyloidosis secondary to Familial Mediterranean fever

Abstract

Introduction

Case description

Discussion and evaluation

Conclusions

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Introduction

Case description

Discussion and evaluation

Conclusions

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