Literature DB >> 27832731

Screening Fabry's disease in chronic kidney disease patients not on dialysis: a multicenter study.

Yavuz Yeniçerioğlu1, Hakan Akdam1, Belda Dursun2, Alper Alp1, Funda Sağlam Eyiler3, Davut Akın4, Yelda Gün3, Bülent Hüddam5, Mehmet Batmazoğlu4, Dilek Gibyeli Genek5, Serhat Pirinççi6, İsmail Rıfkı Ersoy7, Atilla Üzüm7, Zeki Soypaçacı7, Mehmet Tanrısev8, Hülya Çolak8, Sibel Demiral Sezer8, Gökay Bozkurt9, Utku Oğan Akyıldız10, Ayşe İpek Akyüz Ünsal11, Mustafa Ünübol12, Meltem Uslu13, Ufuk Eryılmaz14, Ceren Günel15, İbrahim Meteoğlu16, İrfan Yavaşoğlu17, Alparslan Ünsal18, Harun Akar8, Pınar Okyay6.   

Abstract

OBJECTIVES: Fabry's disease is an X-linked inherited, rare, progressive, lysosomal storage disorder, affecting multiple organs due to the deficient activity of α-galactosidase A (α-Gal A) enzyme. The prevalence has been reported to be 0.15-1% in hemodialysis patients; however, the information on the prevalence in chronic kidney disease not on dialysis is lacking. This study aimed to determine the prevalence of Fabry's disease in chronic kidney disease.
METHODS: The patients older than 18 years, enclosing KDIGO 2012 chronic kidney disease definitions, not on dialysis, were enrolled. Dried blood spots on Guthrie papers were used to analyze α-Gal A enzyme and genetic analysis was performed in individuals with enzyme activity ≤1.2 μmol/L/h.
RESULTS: A total of 1453 chronic kidney disease patients not on dialysis from seven clinics in Turkey were screened. The mean age of the study population was 59.3 ± 15.9 years. 45.6% of patients were female. The creatinine clearance of 77.3% of patients was below 60 mL/min/1.73 m2, 8.4% had proteinuria, and 2.5% had isolated microscopic hematuria. The mean value of patients' α-Gal A enzyme was detected as 2.93 ± 1.92 μmol/L/h. 152 patients had low levels of α-Gal A enzyme activity (≤1.2 μmol/L/h). In mutation analysis, A143T and D313Y variants were disclosed in three male patients. The prevalence of Fabry's disease in chronic kidney disease not on dialysis was found to be 0.2% (0.4% in male, 0.0% in female).
CONCLUSION: Fabry's disease should be considered in the differential diagnosis of chronic kidney disease with unknown etiology even in the absence of symptoms and signs suggestive of Fabry's disease.

Entities:  

Keywords:  Fabry’s disease; chronic kidney disease; globotriaosylceramide; lysosomal storage; α-galactosidase A

Mesh:

Substances:

Year:  2016        PMID: 27832731      PMCID: PMC6014365          DOI: 10.1080/0886022X.2016.1254656

Source DB:  PubMed          Journal:  Ren Fail        ISSN: 0886-022X            Impact factor:   2.606


  32 in total

1.  Fabry disease: enzymatic diagnosis in dried blood spots on filter paper.

Authors:  N A Chamoles; M Blanco; D Gaggioli
Journal:  Clin Chim Acta       Date:  2001-06       Impact factor: 3.786

2.  Fabry-database.org: database of the clinical phenotypes, genotypes and mutant α-galactosidase A structures in Fabry disease.

Authors:  Seiji Saito; Kazuki Ohno; Hitoshi Sakuraba
Journal:  J Hum Genet       Date:  2011-03-17       Impact factor: 3.172

Review 3.  Fabry disease: a review of current management strategies.

Authors:  A Mehta; M Beck; F Eyskens; C Feliciani; I Kantola; U Ramaswami; A Rolfs; A Rivera; S Waldek; D P Germain
Journal:  QJM       Date:  2010-07-21

4.  An atypical variant of Fabry's disease in men with left ventricular hypertrophy.

Authors:  S Nakao; T Takenaka; M Maeda; C Kodama; A Tanaka; M Tahara; A Yoshida; M Kuriyama; H Hayashibe; H Sakuraba
Journal:  N Engl J Med       Date:  1995-08-03       Impact factor: 91.245

5.  Later-onset Fabry disease: an adult variant presenting with the cramp-fasciculation syndrome.

Authors:  Christopher S Nance; Christopher J Klein; Maryam Banikazemi; Steven H Dikman; Robert G Phelps; Justin C McArthur; Moses Rodriguez; Robert J Desnick
Journal:  Arch Neurol       Date:  2006-03

Review 6.  A systematic review on screening for Fabry disease: prevalence of individuals with genetic variants of unknown significance.

Authors:  L van der Tol; B E Smid; B J H M Poorthuis; M Biegstraaten; R H Lekanne Deprez; G E Linthorst; C E M Hollak
Journal:  J Med Genet       Date:  2013-08-06       Impact factor: 6.318

7.  Screening for Fabry disease in patients undergoing dialysis for chronic renal failure in Turkey: identification of new case with novel mutation.

Authors:  Ilyas Okur; Fatih Ezgu; Gursel Biberoglu; Leyla Tumer; Yasemin Erten; Muzeyyen Isitman; Fatma Tuba Eminoglu; Alev Hasanoglu
Journal:  Gene       Date:  2013-06-10       Impact factor: 3.688

8.  Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey.

Authors:  A Mehta; R Ricci; U Widmer; F Dehout; A Garcia de Lorenzo; C Kampmann; A Linhart; G Sunder-Plassmann; M Ries; M Beck
Journal:  Eur J Clin Invest       Date:  2004-03       Impact factor: 4.686

Review 9.  Fabry disease.

Authors:  Dominique P Germain
Journal:  Orphanet J Rare Dis       Date:  2010-11-22       Impact factor: 4.123

10.  Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.

Authors:  Dominique P Germain; Joel Charrow; Robert J Desnick; Nathalie Guffon; Judy Kempf; Robin H Lachmann; Roberta Lemay; Gabor E Linthorst; Seymour Packman; C Ronald Scott; Stephen Waldek; David G Warnock; Neal J Weinreb; William R Wilcox
Journal:  J Med Genet       Date:  2015-03-20       Impact factor: 6.318

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  5 in total

1.  Prevalence of Fabry disease in male dialysis patients: Argentinean screening study.

Authors:  Joaquín Frabasil; Consuelo Durand; Silvia Sokn; Daniela Gaggioli; Patricia Carozza; Ricardo Carabajal; Juan Politei; Andrea B Schenone
Journal:  JIMD Rep       Date:  2019-05-02

Review 2.  Deficiency in the Screening Process of Fabry Disease: Analysis of Chronic Kidney Patients Not on Dialysis.

Authors:  Yuri Battaglia; Fulvio Fiorini; Cristiano Azzini; Pasquale Esposito; Alessandro De Vito; Antonio Granata; Alda Storari; Renzo Mignani
Journal:  Front Med (Lausanne)       Date:  2021-02-09

3.  The prevalence of Fabry disease in a statewide chronic kidney disease cohort - Outcomes of the aCQuiRE (Ckd.Qld fabRy Epidemiology) study.

Authors:  Andrew Mallett; Phoebe Jane Kearey; Anne Cameron; Helen G Healy; Charles Denaro; Mark Thomas; Vincent W Lee; Samantha Louise Stark; Maria Fuller; Zaimin Wang; Wendy E Hoy
Journal:  BMC Nephrol       Date:  2022-05-04       Impact factor: 2.585

4.  The Ckd. Qld fabRy Epidemiology (aCQuiRE) study protocol: identifying the prevalence of Fabry disease amongst patients with kidney disease in Queensland, Australia.

Authors:  Andrew Mallett; Phoebe Kearey; Anne Cameron; Helen Healy; Charles Denaro; Mark Thomas; Vincent W Lee; Samantha Stark; Maria Fuller; Wendy E Hoy
Journal:  BMC Nephrol       Date:  2020-02-22       Impact factor: 2.388

5.  Sudden onset of nephrotic syndrome in an asymptomatic Fabry patient: a case report.

Authors:  Ruixiao Zhang; Zeqing Chen; Yanhua Lang; Shihong Shao; Yan Cai; Qingqing You; Yan Sun; Sai Wang; Xiaomeng Shi; Zhiying Liu; Wencong Guo; Yue Han; Leping Shao
Journal:  Ren Fail       Date:  2020-11       Impact factor: 2.606

  5 in total

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