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Nephrotic syndrome, type 20(NPHS20)

MedGen UID:
1678854
Concept ID:
C5193011
Disease or Syndrome
Synonyms: NEPHROTIC SYNDROME, TYPE 20; NPHS20
 
Gene (location): TBC1D8B (Xq22.3)
 
Monarch Initiative: MONDO:0026726
OMIM®: 301028

Definition

Nephrotic syndrome type 20 (NPHS20) is an X-linked renal disorder characterized by onset of steroid-resistant nephrotic syndrome and proteinuria in the first decade of life in affected males. The course of the disorder is highly variable: some patients progress to end-stage kidney disease and may die in childhood without renal transplantation, whereas others have milder symptoms and maintain normal renal function. Carrier females may have a milder disorder with proteinuria or may be unaffected. Renal biopsy typically shows focal segmental glomerulosclerosis (FSGS) and effacement of podocyte foot processes (summary by Dorval et al., 2019 and Kampf et al., 2019). For a general phenotypic description and a discussion of genetic heterogeneity of nephrotic syndrome, see NPHS1 (256300). [from OMIM]

Clinical features

From HPO
Focal segmental glomerulosclerosis
MedGen UID:
4904
Concept ID:
C0017668
Disease or Syndrome
Segmental accumulation of scar tissue in individual (but not all) glomeruli.
Proteinuria
MedGen UID:
10976
Concept ID:
C0033687
Finding
Increased levels of protein in the urine.
Steroid-resistant nephrotic syndrome
MedGen UID:
588369
Concept ID:
C0403397
Disease or Syndrome
A form of nephrotic syndrome that does not respond to treatment with steroid medication, defined as persistent proteinuria despite 60mg/m2 or 2mg/kg for 8 weeks, after insuring no infection or non-adherence to medication.
Stage 5 chronic kidney disease
MedGen UID:
384526
Concept ID:
C2316810
Disease or Syndrome
A degree of kidney failure severe enough to require dialysis or kidney transplantation for survival characterized by a severe reduction in glomerular filtration rate (less than 15 ml/min/1.73 m2) and other manifestations including increased serum creatinine.

Professional guidelines

PubMed

Jia SL, Ni FF, Ma YJ, Wu YH, Ma WK, Gao XJ
Klin Padiatr 2021 Mar;233(2):63-68. Epub 2021 Mar 8 doi: 10.1055/a-1288-3670. PMID: 33684950
Taşdemir M, Canpolat N, Yıldız N, Özçelik G, Benzer M, Saygılı SK, Özkayin EN, Türkkan ÖN, Balat A, Candan C, Çelakıl M, Yavuz S, Akıncı N, Göknar N, Akgün C, Tülpar S, Alpay H, Sever FL, Bilge İ
Turk J Med Sci 2021 Aug 30;51(4):1781-1790. doi: 10.3906/sag-2012-297. PMID: 33581711Free PMC Article
Dufek S, Holtta T, Trautmann A, Ylinen E, Alpay H, Ariceta G, Aufricht C, Bacchetta J, Bakkaloglu SA, Bayazit A, Cicek RY, Dursun I, Duzova A, Ekim M, Iancu D, Jankauskiene A, Klaus G, Paglialonga F, Pasini A, Printza N, Said Conti V, do Sameiro Faria M, Schmitt CP, Stefanidis CJ, Verrina E, Vidal E, Vondrak K, Webb H, Zampetoglou A, Bockenhauer D, Edefonti A, Shroff R
Nephrol Dial Transplant 2019 Aug 1;34(8):1369-1377. doi: 10.1093/ndt/gfy165. PMID: 30215773

Recent clinical studies

Etiology

Hodson EM, Sinha A, Cooper TE
Cochrane Database Syst Rev 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3. PMID: 35224732Free PMC Article
Sanchez-Rodriguez E, Southard CT, Kiryluk K
Clin J Am Soc Nephrol 2021 Mar 8;16(3):458-466. Epub 2020 Jul 17 doi: 10.2215/CJN.14031119. PMID: 32680915Free PMC Article
Mayer U, Schmitz J, Bräsen JH, Pape L
Pediatr Nephrol 2020 May;35(5):829-842. Epub 2020 Feb 12 doi: 10.1007/s00467-019-04436-y. PMID: 32052153Free PMC Article
Ozkok A, Yildiz A
World J Gastroenterol 2014 Jun 28;20(24):7544-54. doi: 10.3748/wjg.v20.i24.7544. PMID: 24976695Free PMC Article
Feng M, Yuan W, Zhang R, Fu P, Wu T
Cochrane Database Syst Rev 2013 Jun 5;2013(6):CD006335. doi: 10.1002/14651858.CD006335.pub3. PMID: 23740567Free PMC Article

Diagnosis

Hua MR, Zhao YL, Yang JZ, Zou L, Zhao YY, Li X
Int Immunopharmacol 2023 Jul;120:110317. Epub 2023 May 17 doi: 10.1016/j.intimp.2023.110317. PMID: 37207447
Kostovska I, Trajkovska KT, Topuzovska S, Cekovska S, Labudovic D, Kostovski O, Spasovski G
Adv Clin Chem 2022;108:1-36. Epub 2021 Sep 10 doi: 10.1016/bs.acc.2021.08.001. PMID: 35659057
Sanchez-Rodriguez E, Southard CT, Kiryluk K
Clin J Am Soc Nephrol 2021 Mar 8;16(3):458-466. Epub 2020 Jul 17 doi: 10.2215/CJN.14031119. PMID: 32680915Free PMC Article
Olowu WA, Ademola A, Ajite AB, Saad YM
Paediatr Int Child Health 2017 Nov;37(4):259-268. Epub 2017 Sep 26 doi: 10.1080/20469047.2017.1374002. PMID: 28949280
Galla JH
Kidney Int 1995 Feb;47(2):377-87. doi: 10.1038/ki.1995.50. PMID: 7723227

Therapy

Hodson EM, Sinha A, Cooper TE
Cochrane Database Syst Rev 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3. PMID: 35224732Free PMC Article
Basu B, Angeletti A, Islam B, Ghiggeri GM
Front Immunol 2022;13:805697. Epub 2022 Feb 11 doi: 10.3389/fimmu.2022.805697. PMID: 35222385Free PMC Article
Sanchez-Rodriguez E, Southard CT, Kiryluk K
Clin J Am Soc Nephrol 2021 Mar 8;16(3):458-466. Epub 2020 Jul 17 doi: 10.2215/CJN.14031119. PMID: 32680915Free PMC Article
Mayer U, Schmitz J, Bräsen JH, Pape L
Pediatr Nephrol 2020 May;35(5):829-842. Epub 2020 Feb 12 doi: 10.1007/s00467-019-04436-y. PMID: 32052153Free PMC Article
Ozkok A, Yildiz A
World J Gastroenterol 2014 Jun 28;20(24):7544-54. doi: 10.3748/wjg.v20.i24.7544. PMID: 24976695Free PMC Article

Prognosis

Kostovska I, Trajkovska KT, Topuzovska S, Cekovska S, Labudovic D, Kostovski O, Spasovski G
Adv Clin Chem 2022;108:1-36. Epub 2021 Sep 10 doi: 10.1016/bs.acc.2021.08.001. PMID: 35659057
Sanchez-Rodriguez E, Southard CT, Kiryluk K
Clin J Am Soc Nephrol 2021 Mar 8;16(3):458-466. Epub 2020 Jul 17 doi: 10.2215/CJN.14031119. PMID: 32680915Free PMC Article
Mayer U, Schmitz J, Bräsen JH, Pape L
Pediatr Nephrol 2020 May;35(5):829-842. Epub 2020 Feb 12 doi: 10.1007/s00467-019-04436-y. PMID: 32052153Free PMC Article
Olowu WA, Ademola A, Ajite AB, Saad YM
Paediatr Int Child Health 2017 Nov;37(4):259-268. Epub 2017 Sep 26 doi: 10.1080/20469047.2017.1374002. PMID: 28949280
Galla JH
Kidney Int 1995 Feb;47(2):377-87. doi: 10.1038/ki.1995.50. PMID: 7723227

Clinical prediction guides

Qi Z, Yuan S, Wei J, Xia S, Huang Y, Chen X, Han Y, Li Z, Xiao Y, Peng F, Fu X, Sun L, Liu H, Zhu X
J Med Virol 2023 Oct;95(10):e29196. doi: 10.1002/jmv.29196. PMID: 37881096
Chen XJ, Huang Y, Yuan S, Han Y, Li Z, Xu X, Fu X, Peng F, Zhang S, Xiang L, Shi K, Cui X, Zhang Z, Wei J, Xia S, Xiao Y, Sun L, Liu H, Zhu X
Postgrad Med J 2023 Dec 21;100(1179):20-27. doi: 10.1093/postmj/qgad094. PMID: 37827532
Sanchez-Rodriguez E, Southard CT, Kiryluk K
Clin J Am Soc Nephrol 2021 Mar 8;16(3):458-466. Epub 2020 Jul 17 doi: 10.2215/CJN.14031119. PMID: 32680915Free PMC Article
Han SS, Xu YQ, Lu Y, Gu XC, Wang Y
Medicine (Baltimore) 2017 Jun;96(24):e7191. doi: 10.1097/MD.0000000000007191. PMID: 28614261Free PMC Article
Anglés-Cano E, de la Peña Díaz A, Loyau S
Ann N Y Acad Sci 2001;936:261-75. doi: 10.1111/j.1749-6632.2001.tb03514.x. PMID: 11460483

Recent systematic reviews

Pournasiri Z, Madani A, Nazarpack F, Sayer JA, Chavoshzadeh Z, Nili F, Tran P, Saba JD, Jamee M
World J Pediatr 2023 May;19(5):425-437. Epub 2022 Nov 12 doi: 10.1007/s12519-022-00615-4. PMID: 36371483
Hodson EM, Sinha A, Cooper TE
Cochrane Database Syst Rev 2022 Feb 28;2(2):CD003233. doi: 10.1002/14651858.CD003233.pub3. PMID: 35224732Free PMC Article
Liu ID, Willis NS, Craig JC, Hodson EM
Cochrane Database Syst Rev 2019 Nov 21;2019(11) doi: 10.1002/14651858.CD003594.pub6. PMID: 31749142Free PMC Article
Feng M, Yuan W, Zhang R, Fu P, Wu T
Cochrane Database Syst Rev 2013 Jun 5;2013(6):CD006335. doi: 10.1002/14651858.CD006335.pub3. PMID: 23740567Free PMC Article
Yuan W, Wang J, Wu T
Cochrane Database Syst Rev 2008 Apr 16;(2):CD006335. doi: 10.1002/14651858.CD006335.pub2. PMID: 18425948

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