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Fanconi syndrome

MedGen UID:
4653
Concept ID:
C0015624
Disease or Syndrome
Synonyms: Fanconi renotubular syndrome; Fanconi syndrome without cystinosis; Idiopathic De Toni-Debre-Fanconi syndrome; Toni-Fanconi syndrome
SNOMED CT: Fanconi syndrome (40488004)
 
Monarch Initiative: MONDO:0001083

Definition

Fanconi renotubular syndrome is an autosomal dominant renal disorder resulting from decreased solute and water reabsorption in the proximal tubule of the kidney. Patients have polydipsia and polyuria with phosphaturia, glycosuria, and aminoaciduria. They may develop hypophosphatemic rickets or osteomalacia, renal acidosis, and a tendency toward dehydration. Common laboratory abnormalities include glucosuria with a normal serum glucose, hyperaminoaciduria, hypophosphatemia, progressive renal insufficiency, renal sodium and potassium wasting, acidosis, uricosuria, and low molecular weight proteinuria. The disorder is progressive, and some patients will eventually develop renal insufficiency (summary by Lichter-Konecki et al., 2001). Genetic Heterogeneity of Fanconi Renotubular Syndrome See also FRTS2 (613388), caused by mutation in the SLC34A1 gene (182309) on chromosome 5q35; FRTS3 (615605), caused by mutation in the EHHADH gene (607037) on chromosome 3q27; FRTS4 (616026), which is associated with maturity-onset diabetes of the young (MODY), caused by mutation in the HNF4A gene (600281) on chromosome 20q13; and FRTS5 (618913), caused by mutation in the NDUFAF6 gene (612392) on chromosome 8q22. [from OMIM]

Term Hierarchy

Professional guidelines

PubMed

Haffner D, Leifheit-Nestler M, Grund A, Schnabel D
Pediatr Nephrol 2022 Oct;37(10):2289-2302. Epub 2022 Mar 29 doi: 10.1007/s00467-022-05505-5. PMID: 35352187Free PMC Article
Jamalpoor A, Othman A, Levtchenko EN, Masereeuw R, Janssen MJ
Trends Mol Med 2021 Jul;27(7):673-686. Epub 2021 May 8 doi: 10.1016/j.molmed.2021.04.004. PMID: 33975805
Ivanova E, De Leo MG, De Matteis MA, Levtchenko E
Pediatr Endocrinol Rev 2014 Sep;12 Suppl 1:176-84. PMID: 25345100

Recent clinical studies

Etiology

Bagga A, Sinha A
Indian J Pediatr 2020 Sep;87(9):733-744. Epub 2020 Jun 26 doi: 10.1007/s12098-020-03318-8. PMID: 32591997
Hogan JJ, Alexander MP, Leung N
Am J Kidney Dis 2019 Dec;74(6):822-836. Epub 2019 Jul 19 doi: 10.1053/j.ajkd.2019.04.029. PMID: 31331759
Alexander RT, Bitzan M
Pediatr Clin North Am 2019 Feb;66(1):135-157. doi: 10.1016/j.pcl.2018.08.011. PMID: 30454739
Yang B, Xie Y, Guo M, Rosner MH, Yang H, Ronco C
Clin J Am Soc Nephrol 2018 Oct 8;13(10):1605-1611. Epub 2018 Apr 3 doi: 10.2215/CJN.11571017. PMID: 29615394Free PMC Article
François H, Mariette X
Nat Rev Nephrol 2016 Feb;12(2):82-93. Epub 2015 Nov 16 doi: 10.1038/nrneph.2015.174. PMID: 26568188

Diagnosis

Albuquerque ALB, Dos Santos Borges R, Conegundes AF, Dos Santos EE, Fu FMM, Araujo CT, Vaz de Castro PAS, Simões E Silva AC
World J Pediatr 2023 Jul;19(7):619-634. Epub 2023 Feb 2 doi: 10.1007/s12519-023-00685-y. PMID: 36729281
Bagga A, Sinha A
Indian J Pediatr 2020 Sep;87(9):733-744. Epub 2020 Jun 26 doi: 10.1007/s12098-020-03318-8. PMID: 32591997
Hogan JJ, Alexander MP, Leung N
Am J Kidney Dis 2019 Dec;74(6):822-836. Epub 2019 Jul 19 doi: 10.1053/j.ajkd.2019.04.029. PMID: 31331759
Foreman JW
Pediatr Clin North Am 2019 Feb;66(1):159-167. doi: 10.1016/j.pcl.2018.09.002. PMID: 30454741
Alexander RT, Bitzan M
Pediatr Clin North Am 2019 Feb;66(1):135-157. doi: 10.1016/j.pcl.2018.08.011. PMID: 30454739

Therapy

Topaloglu R
Pediatr Nephrol 2024 Aug;39(8):2283-2292. Epub 2023 Dec 21 doi: 10.1007/s00467-023-06225-0. PMID: 38127152
Trepiccione F, Iervolino A, D'Acierno M, Siccardi S, Costanzo V, Sardella D, De La Motte LR, D'Apolito L, Miele A, Perna AF, Capolongo G, Zacchia M, Frische S, Nielsen R, Staiano L, Sambri I, De Cegli R, Unwin R, Eladari D, Capasso G
Sci Transl Med 2023 Nov;15(720):eabn4214. Epub 2023 Nov 1 doi: 10.1126/scitranslmed.abn4214. PMID: 37910600
Haffner D, Leifheit-Nestler M, Grund A, Schnabel D
Pediatr Nephrol 2022 Oct;37(10):2289-2302. Epub 2022 Mar 29 doi: 10.1007/s00467-022-05505-5. PMID: 35352187Free PMC Article
Hogan JJ, Alexander MP, Leung N
Am J Kidney Dis 2019 Dec;74(6):822-836. Epub 2019 Jul 19 doi: 10.1053/j.ajkd.2019.04.029. PMID: 31331759
Yang B, Xie Y, Guo M, Rosner MH, Yang H, Ronco C
Clin J Am Soc Nephrol 2018 Oct 8;13(10):1605-1611. Epub 2018 Apr 3 doi: 10.2215/CJN.11571017. PMID: 29615394Free PMC Article

Prognosis

Nasr SH, Sirac C, Leung N, Bridoux F
Kidney Int 2024 Aug;106(2):201-213. Epub 2024 May 7 doi: 10.1016/j.kint.2024.02.027. PMID: 38723749
Albuquerque ALB, Dos Santos Borges R, Conegundes AF, Dos Santos EE, Fu FMM, Araujo CT, Vaz de Castro PAS, Simões E Silva AC
World J Pediatr 2023 Jul;19(7):619-634. Epub 2023 Feb 2 doi: 10.1007/s12519-023-00685-y. PMID: 36729281
Alexander RT, Bitzan M
Pediatr Clin North Am 2019 Feb;66(1):135-157. doi: 10.1016/j.pcl.2018.08.011. PMID: 30454739
François H, Mariette X
Nat Rev Nephrol 2016 Feb;12(2):82-93. Epub 2015 Nov 16 doi: 10.1038/nrneph.2015.174. PMID: 26568188
Veys KR, Besouw MT, Pinxten AM, Dyck MV, Casteels I, Levtchenko EN
Acta Clin Belg 2016 Jun;71(3):131-7. doi: 10.1179/2295333714Y.0000000113. PMID: 25560059

Clinical prediction guides

Viering DHHM, Vermeltfoort L, Bindels RJM, Deinum J, de Baaij JHF
J Am Soc Nephrol 2023 Nov 1;34(11):1875-1888. Epub 2023 Sep 6 doi: 10.1681/ASN.0000000000000224. PMID: 37678265Free PMC Article
Kleta R, Bockenhauer D
J Am Soc Nephrol 2018 Mar;29(3):727-739. Epub 2017 Dec 13 doi: 10.1681/ASN.2017060600. PMID: 29237739Free PMC Article
Nielsen R, Christensen EI, Birn H
Kidney Int 2016 Jan;89(1):58-67. doi: 10.1016/j.kint.2015.11.007. PMID: 26759048
Duran M, Loof NE, Ketting D, Dorland L
J Clin Chem Clin Biochem 1990 May;28(5):359-63. PMID: 2199597
Blainey JD, Adams RG, Brewer DB, Harvey TC
Br J Ind Med 1980 Aug;37(3):278-84. doi: 10.1136/oem.37.3.278. PMID: 7426480Free PMC Article

Recent systematic reviews

Joshi M, Clark B, Lee TA
Ann Pharmacother 2024 Aug;58(8):857-869. Epub 2023 Nov 6 doi: 10.1177/10600280231206703. PMID: 37932920
Viering DHHM, Vermeltfoort L, Bindels RJM, Deinum J, de Baaij JHF
J Am Soc Nephrol 2023 Nov 1;34(11):1875-1888. Epub 2023 Sep 6 doi: 10.1681/ASN.0000000000000224. PMID: 37678265Free PMC Article
Anguissola G, Leu D, Simonetti GD, Simonetti BG, Lava SAG, Milani GP, Bianchetti MG, Scoglio M
Pediatr Nephrol 2023 Jun;38(6):1725-1731. Epub 2023 Jan 16 doi: 10.1007/s00467-022-05869-8. PMID: 36645492Free PMC Article
Kasimer RN, Langman CB
Pediatr Nephrol 2021 Feb;36(2):223-236. Epub 2020 Feb 3 doi: 10.1007/s00467-020-04487-6. PMID: 32016627
Medic G, van der Weijden M, Karabis A, Hemels M
Curr Med Res Opin 2017 Nov;33(11):2065-2076. Epub 2017 Aug 3 doi: 10.1080/03007995.2017.1354288. PMID: 28692321

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