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Nephrotic syndrome, type 21(NPHS21)

MedGen UID:
1684676
Concept ID:
C5231498
Disease or Syndrome
Synonyms: NEPHROTIC SYNDROME, TYPE 21; NPHS21
 
Gene (location): AVIL (12q14.1)
 
Monarch Initiative: MONDO:0032826
OMIM®: 618594

Definition

Nephrotic syndrome type 21 (NPHS21) is an autosomal recessive renal disorder characterized by onset of kidney dysfunction in the first year of life. Laboratory studies show proteinuria and renal biopsy shows diffuse mesangial sclerosis. The disorder is rapidly progressive and ultimately results in end-stage renal disease. Some patients with variable extrarenal manifestations, such as microcephaly or impaired intellectual development, have been reported, but it is not clear whether these features are consistently part of the phenotype (summary by Rao et al., 2017). (Rao et al. (2017) designated the disorder NPHS25.) For a general phenotypic description and a discussion of genetic heterogeneity of nephrotic syndrome, see NPHS1 (256300). [from OMIM]

Clinical features

From HPO
Diffuse mesangial sclerosis
MedGen UID:
78698
Concept ID:
C0268747
Disease or Syndrome
Diffuse sclerosis of the mesangium, as manifestated by diffuse mesangial matrix expansion.
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.
Podocyte foot process effacement
MedGen UID:
481733
Concept ID:
C3280103
Finding
An anomaly of podocyte morphology characterized by the loss of the interdigitating foot process pattern (generally called foot process effacement; FPE). The term FPE designates the loss of the usual interdigitating pattern of foot processes of neighboring podocytes, leading to relatively broad expanses of podocyte processes covering the glomerular basement membrane (GBM). It is widely viewed as a pathological derangement that is associated with leakage of macromolecules such as albumin through the glomerular filtration barrier.

Professional guidelines

PubMed

Zhong F, Liu S, Li Y, Li G, Liu M, Wang J, Cui W, Suo Y, Gao X
Lipids Health Dis 2022 Apr 10;21(1):38. doi: 10.1186/s12944-022-01632-y. PMID: 35399079Free PMC Article
Hogan J, Radhakrishnan J
Adv Chronic Kidney Dis 2014 Sep;21(5):434-41. doi: 10.1053/j.ackd.2014.03.016. PMID: 25168833
Sethna CB, Gipson DS
Adv Chronic Kidney Dis 2014 Mar;21(2):194-9. doi: 10.1053/j.ackd.2014.01.010. PMID: 24602468

Recent clinical studies

Etiology

Kurasawa S, Kato S, Ozeki T, Akiyama S, Ishimoto T, Mizuno M, Tsuboi N, Kato N, Kosugi T, Maruyama S; J-MARINE collaborators
Clin Exp Nephrol 2024 May;28(5):431-439. Epub 2024 Jan 25 doi: 10.1007/s10157-023-02449-4. PMID: 38267800
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
Sethi S, Madden BJ, Debiec H, Charlesworth MC, Gross L, Ravindran A, Hummel AM, Specks U, Fervenza FC, Ronco P
J Am Soc Nephrol 2019 Jun;30(6):1123-1136. Epub 2019 May 6 doi: 10.1681/ASN.2018080852. PMID: 31061139Free 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
Sethna CB, Gipson DS
Adv Chronic Kidney Dis 2014 Mar;21(2):194-9. doi: 10.1053/j.ackd.2014.01.010. PMID: 24602468

Diagnosis

Kurasawa S, Kato S, Ozeki T, Akiyama S, Ishimoto T, Mizuno M, Tsuboi N, Kato N, Kosugi T, Maruyama S; J-MARINE collaborators
Clin Exp Nephrol 2024 May;28(5):431-439. Epub 2024 Jan 25 doi: 10.1007/s10157-023-02449-4. PMID: 38267800
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
AlYousef A, AlSahow A, AlHelal B, Alqallaf A, Abdallah E, Abdellatif M, Nawar H, Elmahalawy R
BMC Nephrol 2020 May 18;21(1):186. doi: 10.1186/s12882-020-01836-3. PMID: 32423387Free 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
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

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
Hamasaki Y, Hamada R, Muramatsu M, Matsumoto S, Aya K, Ishikura K, Kaneko T, Iijima K
BMC Nephrol 2020 Aug 24;21(1):363. doi: 10.1186/s12882-020-02010-5. PMID: 32838745Free 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
Hogan J, Radhakrishnan J
Adv Chronic Kidney Dis 2014 Sep;21(5):434-41. doi: 10.1053/j.ackd.2014.03.016. PMID: 25168833
Sethna CB, Gipson DS
Adv Chronic Kidney Dis 2014 Mar;21(2):194-9. doi: 10.1053/j.ackd.2014.01.010. PMID: 24602468

Prognosis

Kurasawa S, Kato S, Ozeki T, Akiyama S, Ishimoto T, Mizuno M, Tsuboi N, Kato N, Kosugi T, Maruyama S; J-MARINE collaborators
Clin Exp Nephrol 2024 May;28(5):431-439. Epub 2024 Jan 25 doi: 10.1007/s10157-023-02449-4. PMID: 38267800
AlYousef A, AlSahow A, AlHelal B, Alqallaf A, Abdallah E, Abdellatif M, Nawar H, Elmahalawy R
BMC Nephrol 2020 May 18;21(1):186. doi: 10.1186/s12882-020-01836-3. PMID: 32423387Free PMC Article
Sethi S, Madden BJ, Debiec H, Charlesworth MC, Gross L, Ravindran A, Hummel AM, Specks U, Fervenza FC, Ronco P
J Am Soc Nephrol 2019 Jun;30(6):1123-1136. Epub 2019 May 6 doi: 10.1681/ASN.2018080852. PMID: 31061139Free 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
Sethna CB, Gipson DS
Adv Chronic Kidney Dis 2014 Mar;21(2):194-9. doi: 10.1053/j.ackd.2014.01.010. PMID: 24602468

Clinical prediction guides

Kurasawa S, Kato S, Ozeki T, Akiyama S, Ishimoto T, Mizuno M, Tsuboi N, Kato N, Kosugi T, Maruyama S; J-MARINE collaborators
Clin Exp Nephrol 2024 May;28(5):431-439. Epub 2024 Jan 25 doi: 10.1007/s10157-023-02449-4. PMID: 38267800
AlYousef A, AlSahow A, AlHelal B, Alqallaf A, Abdallah E, Abdellatif M, Nawar H, Elmahalawy R
BMC Nephrol 2020 May 18;21(1):186. doi: 10.1186/s12882-020-01836-3. PMID: 32423387Free PMC Article
Li LZ, Hu Y, Ai SL, Cheng L, Liu J, Morris E, Li Y, Gou SJ, Fu P
Sci Rep 2019 Apr 23;9(1):6421. doi: 10.1038/s41598-019-42905-4. PMID: 31015507Free 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
Sethna CB, Gipson DS
Adv Chronic Kidney Dis 2014 Mar;21(2):194-9. doi: 10.1053/j.ackd.2014.01.010. PMID: 24602468

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
Wu S, Kim C, Baer L, Zhu X
J Am Soc Nephrol 2010 Aug;21(8):1381-9. Epub 2010 Jun 10 doi: 10.1681/ASN.2010020167. PMID: 20538785Free PMC Article

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