Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain

Yıl: 2021 Cilt: 8 Sayı: 2 Sayfa Aralığı: 73 - 78 Metin Dili: İngilizce DOI: 10.5152/eurjrheum.2020.20102 İndeks Tarihi: 07-10-2021

Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain

Öz:
Objective: To evaluate the efficacy of colchicine therapy in pediatric patients with PFAPA syndromewho present with an incomplete response to the standard treatment or with frequent episodes (aninterval of less than 14 days between two disease flares).Methods: A multicenter cohort study of children diagnosed with PFAPA syndrome and treated withcolchicine was performed in three separate hospitals located in Spain. The patients clinical and laboratory data were reviewed by accessing their medical records. Response to colchicine was evaluatedafter 12 months of treatment for frequency, duration, and intensity of PFAPA episodes.Results: A total of 13 children were included in our study, 43% of whom were boys. Median age of thecolchicine therapy initiation was 6 years (interquartile range (IQR)=3-9.5). Following a 12-month period of colchicine therapy (median dosage of 0.02 mg/kg/day; IQR=0.02-0.03), a significant decrease inthe median number of flares (median 8; IQR=7-14 vs 3; IQR=2-4; p=0.005) and the duration of diseaseepisodes (median 4 days; IQR=3.25-5.125 vs 1 day; IQR=1-2; p=0.003) was observed. Furthermore, thehighest degree of fever during disease flares was reduced from median 40ºC (IQR=39.5-40) to 38.5ºC(IQR=37.7-38.9) (p=0.002).Conclusion: Colchicine therapy decreased the frequency and intensity of PFAPA. The use of colchicinecould be an effective treatment in pediatric patients with PFAPA syndrome who present with frequentor severe relapses.
Anahtar Kelime:

Belge Türü: Makale Makale Türü: Araştırma Makalesi Erişim Türü: Erişime Açık
  • 1. Rigante D. Autoinflammatory syndromes behind the scenes of recurrent fevers in children. Med Sci Monit 2009; 15: RA179-RA187.
  • 2. Adrovic A, Sahin S, Barut K, Kasapcopur O. Familial Mediterranean fever and periodic fever, aphthous stomatitis, pharyngitis, and adenitis (PFAPA) syndrome: Shared features and main differences. Rheumatol Int 2019; 39: 29-36.
  • 3. Cantarini L, Vitale A, Bartolomei B, Galeazzi M, Rigante D. Diagnosis of PFAPA syndrome applied to a cohort of 17 adults with unexplained recurrent fevers. Clin Exp Rheumatol 2012; 30: 269-71.
  • 4. Gentileschi S, Vitale A, Frediani B, Galeazzi M, Rigante D, Cantarini L. Challenges and new horizons in the periodic fever, aphthous stomatitis, pharingitis and adenitis (PFAPA) syndrome. Expert Opin Orphan Drugs 2017: 5: 165-71.
  • 5. Esposito S, Bianchini S, Fattizzo M, Baggi E, Marchisio P, Rigante D. The enigma of periodic fever, aphthous stomatitis, pharyngitis and adenitis syndrome. Pediatr Infect Dis J 2014; 33: 650-2.
  • 6. Kolly L, Busso N, von Scheven-Gete A, Bagnoud N, Moix I, Holzinger D, et al. Periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis syndrome is linked to dysregulated monocyte IL-1β production. J Allergy Clin Immunol 2013; 131: 1635-43.
  • 7. Cattalini M, Soliani M, Rigante D, Lopalco G, Iannone F, Galeazzi M, et al. Basic characteristics of adults with periodic fever, aphthous stomatitis, pharyngitis, and adenopathy syndrome in comparison with the typical pediatric expression of disease. Mediators Inflamm 2015; 2015: 570418.
  • 8. Gaggiano C, Rigante D, Sota J, Grosso S, Cantarini L. Treatment options for periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome in children and adults: A narrative review. Clin Rheumatol 2019; 38: 11-7.
  • 9. Pruzanski W, Kennedy BP, van den Bosch H, Stefanski E, Vadas P. Microtubule depolymerization selectively down-regulates the synthesis of proinflammatory secretory nonpancreatic phospholipase A2. Lab Invest 1997; 76: 171-8.
  • 10. Tasher D, Stein M, Dalal I, Somekh E. Colchicine prophylaxis for frequent periodic fever, aphthous stomatitis, pharyngitis and adenitis episodes. Acta Paediatr 2008; 97: 1090-2.
  • 11. Thomas KT, Feder HM Jr, Lawton AR, Edwards KM. Periodic fever syndrome in children. J Pediatr 1999; 135: 15-21.
  • 12. Gattorno M, Hofer M, Federici S, Vanoni F, Bovis F, Aksentijevich I, et al. Classification criteria for autoinflammatory recurrent fevers. Ann Rheum Dis 2019; 78: 1025-32.
  • 13. Federici S, Gattorno M. A practical approach to the diagnosis of autoinflammatory diseases in childhood. Best Pract Res Clin Rheumatol 2014; 28: 263-76.
  • 14. Gattorno M, Sormani MP, D'Osualdo A, Pelagatti MA, Caroli F, Federici S, et al. A diagnostic score for molecular analysis of hereditary autoinflammatory syndromes with periodic fever in children. Arthritis Rheum 2008; 58: 1823-32.
  • 15. Lachmann HJ. Periodic fever syndromes. Best Pract Res Clin Rheumatol 2017; 31: 596-609.
  • 16. Butbul Aviel Y, Tatour S, Gershoni Baruch R, Brik R. Colchicine as a therapeutic option in periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome. Semin Arthritis Rheum 2016; 45: 471-4.
  • 17. Dagan E, Gershoni-Baruch R, Khatib I, Mori A, Brik R. MEFV, TNF1rA, CARD15 and NLRP3 mutation analysis in PFAPA. Rheumatol Int 2010; 30: 633-6.
  • 18. Perko D, Debeljak M, Toplak N, Avcin T. Clinical features and genetic background of the periodic Fever syndrome with aphthous stomatitis, pharyngitis, and adenitis: A single center longitudinal study of 81 patients. Mediators Inflamm 2015; 2015: 293417.
  • 19. Taniuchi S, Nishikomori R, Iharada A, Tuji S, Heike T, Kaneko K. MEFV variants in patients with PFAPA syndrome in Japan. Open Rheumatol J 2013; 7: 22-5.
  • 20. Pehlivan E, Adrovic A, Sahin S, Barut K, Kul Cınar O, Kasapcopur O. PFAPA syndrome in a population with endemic familial Mediterranean fever. J Pediatr 2018; 192: 253-5.
  • 21. Gunes M, Cekic S, Kilic SS. Is colchicine more effective to prevent periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis episodes in Mediterranean fever gene variants?. Pediatr Int 2017; 59: 655-60.
  • 22. Dusser P, Hentgen V, Neven B, Koné-Paut I. Is colchicine an effective treatment in periodic fever, aphtous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome?. Joint Bone Spine 2016; 83: 406-11.
  • 23. Haytoglu Z, Gundeslioglu OO. Mediterranean fever gene variants and colchicine therapy in periodic fever, aphthous stomatitis pharyngitis, adenitis syndrome in a Mediterranean region. Expert Rev Clin Immunol 2019; 15: 571-5. [Retraction in: Haytoglu Z, Gundeslioglu OO. Expert Rev Clin Immunol 2020; 16: 229].
  • 24. Harel L, Hashkes PJ, Lapidus S, Edwards KM, Padeh S, Gattorno M, et al. The first international conference on periodic fever, aphthous stomatitis, pharyngitis, adenitis syndrome. J Pediatr 2018; 193: 265-74.e3.
  • 25. Manthiram K, Nesbitt E, Morgan T, Edwards KM. Family history in periodic fever, aphthous stomatitis, pharyngitis, adenitis (PFAPA) syndrome. Pediatrics 2016; 138: e20154572.
  • 26. Di Gioia SA, Bedoni N, von Scheven-Gête A, Vanoni F, Superti-Furga A, Hofer M, et al. Analysis of the genetic basis of periodic fever with aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome. Sci Rep 2015; 5: 10200.
APA Quintana-Ortega C, Seoane Reula M, Fernandez-Silveira L, Camacho Lovillo M, Olbrich P, Neth O, Murias S, Udaondo C, Remesal A, Calvo C, Alcobendas R (2021). Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. , 73 - 78. 10.5152/eurjrheum.2020.20102
Chicago Quintana-Ortega Cristian,Seoane Reula M Elena,Fernandez-Silveira Laura,Camacho Lovillo Marisol,Olbrich Peter,Neth Olaf,Murias Sara,Udaondo Clara,Remesal Agustin,Calvo Cristina,Alcobendas Rosa Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. (2021): 73 - 78. 10.5152/eurjrheum.2020.20102
MLA Quintana-Ortega Cristian,Seoane Reula M Elena,Fernandez-Silveira Laura,Camacho Lovillo Marisol,Olbrich Peter,Neth Olaf,Murias Sara,Udaondo Clara,Remesal Agustin,Calvo Cristina,Alcobendas Rosa Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. , 2021, ss.73 - 78. 10.5152/eurjrheum.2020.20102
AMA Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. . 2021; 73 - 78. 10.5152/eurjrheum.2020.20102
Vancouver Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. . 2021; 73 - 78. 10.5152/eurjrheum.2020.20102
IEEE Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R "Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain." , ss.73 - 78, 2021. 10.5152/eurjrheum.2020.20102
ISNAD Quintana-Ortega, Cristian vd. "Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain". (2021), 73-78. https://doi.org/10.5152/eurjrheum.2020.20102
APA Quintana-Ortega C, Seoane Reula M, Fernandez-Silveira L, Camacho Lovillo M, Olbrich P, Neth O, Murias S, Udaondo C, Remesal A, Calvo C, Alcobendas R (2021). Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. European Journal of Rheumatology, 8(2), 73 - 78. 10.5152/eurjrheum.2020.20102
Chicago Quintana-Ortega Cristian,Seoane Reula M Elena,Fernandez-Silveira Laura,Camacho Lovillo Marisol,Olbrich Peter,Neth Olaf,Murias Sara,Udaondo Clara,Remesal Agustin,Calvo Cristina,Alcobendas Rosa Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. European Journal of Rheumatology 8, no.2 (2021): 73 - 78. 10.5152/eurjrheum.2020.20102
MLA Quintana-Ortega Cristian,Seoane Reula M Elena,Fernandez-Silveira Laura,Camacho Lovillo Marisol,Olbrich Peter,Neth Olaf,Murias Sara,Udaondo Clara,Remesal Agustin,Calvo Cristina,Alcobendas Rosa Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. European Journal of Rheumatology, vol.8, no.2, 2021, ss.73 - 78. 10.5152/eurjrheum.2020.20102
AMA Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. European Journal of Rheumatology. 2021; 8(2): 73 - 78. 10.5152/eurjrheum.2020.20102
Vancouver Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain. European Journal of Rheumatology. 2021; 8(2): 73 - 78. 10.5152/eurjrheum.2020.20102
IEEE Quintana-Ortega C,Seoane Reula M,Fernandez-Silveira L,Camacho Lovillo M,Olbrich P,Neth O,Murias S,Udaondo C,Remesal A,Calvo C,Alcobendas R "Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain." European Journal of Rheumatology, 8, ss.73 - 78, 2021. 10.5152/eurjrheum.2020.20102
ISNAD Quintana-Ortega, Cristian vd. "Colchicine treatment in children with periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) syndrome: A multicenter study in Spain". European Journal of Rheumatology 8/2 (2021), 73-78. https://doi.org/10.5152/eurjrheum.2020.20102