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Bartter disease type 3(BARTS3)

MedGen UID:
335399
Concept ID:
C1846343
Disease or Syndrome
Synonyms: Bartter syndrome classic; Bartter syndrome type 3
SNOMED CT: Bartter syndrome type 3 (700111000); Bartter's syndrome type 3 (700111000); Classic Bartter syndrome (700111000)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Gene (location): CLCNKB (1p36.13)
 
Monarch Initiative: MONDO:0011822
OMIM®: 607364
Orphanet: ORPHA93605

Definition

Bartter syndrome refers to a group of disorders that are unified by autosomal recessive transmission of impaired salt reabsorption in the thick ascending loop of Henle with pronounced salt wasting, hypokalemic metabolic alkalosis, and hypercalciuria. Clinical disease results from defective renal reabsorption of sodium chloride in the thick ascending limb (TAL) of the Henle loop, where 30% of filtered salt is normally reabsorbed (Simon et al., 1997). Patients with antenatal (or neonatal) forms of Bartter syndrome (e.g., BARTS1, 601678) typically present with premature birth associated with polyhydramnios and low birth weight and may develop life-threatening dehydration in the neonatal period. Patients with classic Bartter syndrome present later in life and may be sporadically asymptomatic or mildly symptomatic (summary by Simon et al., 1996 and Fremont and Chan, 2012). Genetic Heterogeneity of Bartter Syndrome Antenatal Bartter syndrome type 1 (601678) is caused by loss-of-function mutations in the butmetanide-sensitive Na-K-2Cl cotransporter NKCC2 (SLC12A1; 600839). Antenatal Bartter syndrome type 2 (241200) is caused by loss-of-function mutations in the ATP-sensitive potassium channel ROMK (KCNJ1; 600359). One form of neonatal Bartter syndrome with sensorineural deafness, Bartter syndrome type 4A (602522), is caused by mutation in the BSND gene (606412). Another form of neonatal Bartter syndrome with sensorineural deafness, Bartter syndrome type 4B (613090), is caused by simultaneous mutation in both the CLCNKA (602024) and CLCNKB (602023) genes. Also see autosomal dominant hypocalcemia-1 with Bartter syndrome (601198), which is sometimes referred to as Bartter syndrome type 5 (Fremont and Chan, 2012), caused by mutation in the CASR gene (601199). See Gitelman syndrome (GTLMN; 263800), which is often referred to as a mild variant of Bartter syndrome, caused by mutation in the thiazide-sensitive sodium-chloride cotransporter SLC12A3 (600968). [from OMIM]

Additional description

From MedlinePlus Genetics
Beginning in infancy, affected individuals often fail to grow and gain weight at the expected rate (failure to thrive). They lose excess amounts of salt (sodium chloride) in their urine, which leads to dehydration, constipation, and increased urine production (polyuria). In addition, large amounts of calcium are lost through the urine (hypercalciuria), which can cause weakening of the bones (osteopenia). Some of the calcium is deposited in the kidneys as they are concentrating urine, leading to hardening of the kidney tissue (nephrocalcinosis). Bartter syndrome is also characterized by low levels of potassium in the blood (hypokalemia), which can result in muscle weakness, cramping, and fatigue. Rarely, affected children develop hearing loss caused by abnormalities in the inner ear (sensorineural deafness).

Two major forms of Bartter syndrome are distinguished by their age of onset and severity. One form begins before birth (antenatal) and is often life-threatening. The other form, often called the classical form, begins in early childhood and tends to be less severe. Once the genetic causes of Bartter syndrome were identified, researchers also split the disorder into different types based on the genes involved. Types I, II, and IV have the features of antenatal Bartter syndrome. Because type IV is also associated with hearing loss, it is sometimes called antenatal Bartter syndrome with sensorineural deafness. Type III usually has the features of classical Bartter syndrome.

In some cases, Bartter syndrome becomes apparent before birth. The disorder can cause polyhydramnios, which is an increased volume of fluid surrounding the fetus (amniotic fluid). Polyhydramnios increases the risk of premature birth.

Bartter syndrome is a group of very similar kidney disorders that cause an imbalance of potassium, sodium, chloride, and related molecules in the body.  https://medlineplus.gov/genetics/condition/bartter-syndrome

Clinical features

From HPO
Hypocalciuria
MedGen UID:
9381
Concept ID:
C0020599
Disease or Syndrome
An abnormally decreased calcium concentration in the urine.
Nephrocalcinosis
MedGen UID:
10222
Concept ID:
C0027709
Disease or Syndrome
Nephrocalcinosis is the deposition of calcium salts in renal parenchyma.
Polyuria
MedGen UID:
19404
Concept ID:
C0032617
Sign or Symptom
An increased rate of urine production.
Renal salt wasting
MedGen UID:
375868
Concept ID:
C1846347
Finding
A high concentration of one or more electrolytes in the urine in the presence of low serum concentrations of the electrolyte(s).
Renal potassium wasting
MedGen UID:
339499
Concept ID:
C1846348
Finding
High urine potassium in the presence of hypokalemia.
Impaired renal tubular reabsorption of chloride
MedGen UID:
375869
Concept ID:
C1846349
Finding
Any impairment of reabsorption of chloride by the kidney.
Increased urinary potassium
MedGen UID:
337562
Concept ID:
C1846351
Finding
An increased concentration of potassium(1+) in the urine.
Hyperchloriduria
MedGen UID:
337563
Concept ID:
C1846352
Finding
An increased concentration of chloride in the urine.
Hypotension
MedGen UID:
5715
Concept ID:
C0020649
Finding
Low Blood Pressure, vascular hypotension.
Generalized muscle weakness
MedGen UID:
155433
Concept ID:
C0746674
Sign or Symptom
Generalized weakness or decreased strength of the muscles, affecting both distal and proximal musculature.
Dehydration
MedGen UID:
8273
Concept ID:
C0011175
Disease or Syndrome
A condition resulting from the excessive loss of water from the body. It is usually caused by severe diarrhea, vomiting or diaphoresis.
Hypokalemia
MedGen UID:
5712
Concept ID:
C0020621
Finding
An abnormally decreased potassium concentration in the blood.
Hypokalemic metabolic alkalosis
MedGen UID:
152852
Concept ID:
C0740898
Disease or Syndrome
Increased circulating aldosterone concentration
MedGen UID:
6960
Concept ID:
C0020428
Disease or Syndrome
Overproduction of the mineralocorticoid aldosterone by the adrenal cortex.
Increased circulating renin concentration
MedGen UID:
66818
Concept ID:
C0240783
Finding
An increased level of renin in the blood.
Hyperactive renin-angiotensin system
MedGen UID:
335401
Concept ID:
C1846345
Finding
An abnormally increased activity of the renin-angiotensin system, causing hypertension by a combination of volume excess and vasoconstrictor mechanisms.
Abnormal retinal vascular morphology
MedGen UID:
870311
Concept ID:
C4024753
Anatomical Abnormality
A structural abnormality of retinal vasculature.
Abnormal choroid morphology
MedGen UID:
892839
Concept ID:
C4025836
Anatomical Abnormality
Any structural abnormality of the choroid.
Abnormal sclera morphology
MedGen UID:
871347
Concept ID:
C4025840
Anatomical Abnormality
An abnormality of the sclera.

Professional guidelines

PubMed

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Genet Med 2016 Feb;18(2):180-8. Epub 2015 Apr 16 doi: 10.1038/gim.2015.56. PMID: 25880437
Assadi F
Iran J Kidney Dis 2008 Jul;2(3):115-22. PMID: 19377223

Recent clinical studies

Etiology

Tschernoster N, Erger F, Kohl S, Reusch B, Wenzel A, Walsh S, Thiele H, Becker C, Franitza M, Bartram MP, Kömhoff M, Schumacher L, Kukat C, Borodina T, Quedenau C, Nürnberg P, Rinschen MM, Driller JH, Pedersen BP, Schlingmann KP, Hüttel B, Bockenhauer D, Beck B, Altmüller J
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J Am Soc Nephrol 2017 Aug;28(8):2540-2552. Epub 2017 Apr 5 doi: 10.1681/ASN.2016101057. PMID: 28381550Free PMC Article
Fremont OT, Chan JC
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Orphanet J Rare Dis 2008 Jul 30;3:22. doi: 10.1186/1750-1172-3-22. PMID: 18667063Free PMC Article

Diagnosis

Tschernoster N, Erger F, Kohl S, Reusch B, Wenzel A, Walsh S, Thiele H, Becker C, Franitza M, Bartram MP, Kömhoff M, Schumacher L, Kukat C, Borodina T, Quedenau C, Nürnberg P, Rinschen MM, Driller JH, Pedersen BP, Schlingmann KP, Hüttel B, Bockenhauer D, Beck B, Altmüller J
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García-Castaño A, Gómez-Conde S, Gondra L, Herrero M, Aguirre M, de la Hoz AB, Castaño L; Renaltube group, Madariaga L
Sci Rep 2023 Aug 3;13(1):12587. doi: 10.1038/s41598-023-38179-6. PMID: 37537162Free PMC Article
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Assadi F
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Therapy

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Genet Med 2016 Feb;18(2):180-8. Epub 2015 Apr 16 doi: 10.1038/gim.2015.56. PMID: 25880437
Chiang WF, Lin SH, Chan JS, Lin SH
Clin Nephrol 2014 Feb;81(2):146-50. doi: 10.5414/CN107606. PMID: 22854165
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J Postgrad Med 2009 Jul-Sep;55(3):208-10. doi: 10.4103/0022-3859.57407. PMID: 19884751
Assadi F
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Prognosis

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Knoers NV, Levtchenko EN
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Clinical prediction guides

Tschernoster N, Erger F, Kohl S, Reusch B, Wenzel A, Walsh S, Thiele H, Becker C, Franitza M, Bartram MP, Kömhoff M, Schumacher L, Kukat C, Borodina T, Quedenau C, Nürnberg P, Rinschen MM, Driller JH, Pedersen BP, Schlingmann KP, Hüttel B, Bockenhauer D, Beck B, Altmüller J
Genome Med 2023 Aug 23;15(1):62. doi: 10.1186/s13073-023-01215-1. PMID: 37612755Free PMC Article
Koumangoye R, Bastarache L, Delpire E
Function (Oxf) 2021;2(1):zqaa028. Epub 2020 Nov 3 doi: 10.1093/function/zqaa028. PMID: 33345190Free PMC Article
Seys E, Andrini O, Keck M, Mansour-Hendili L, Courand PY, Simian C, Deschenes G, Kwon T, Bertholet-Thomas A, Bobrie G, Borde JS, Bourdat-Michel G, Decramer S, Cailliez M, Krug P, Cozette P, Delbet JD, Dubourg L, Chaveau D, Fila M, Jourde-Chiche N, Knebelmann B, Lavocat MP, Lemoine S, Djeddi D, Llanas B, Louillet F, Merieau E, Mileva M, Mota-Vieira L, Mousson C, Nobili F, Novo R, Roussey-Kesler G, Vrillon I, Walsh SB, Teulon J, Blanchard A, Vargas-Poussou R
J Am Soc Nephrol 2017 Aug;28(8):2540-2552. Epub 2017 Apr 5 doi: 10.1681/ASN.2016101057. PMID: 28381550Free PMC Article
Andrini O, Keck M, Briones R, Lourdel S, Vargas-Poussou R, Teulon J
Am J Physiol Renal Physiol 2015 Jun 15;308(12):F1324-34. Epub 2015 Mar 25 doi: 10.1152/ajprenal.00004.2015. PMID: 25810436
Iqbal H, Sarfaraz T, Anjum F, Anwar Z, Mir A
J Biomed Biotechnol 2011;2011:304612. Epub 2011 Apr 4 doi: 10.1155/2011/304612. PMID: 21541222Free PMC Article

Recent systematic reviews

Vaisbich MH, Messa ACHL, Rangel-Santos AC, Ferreira JCOA, Nunes FAMDF, Watanabe A
Nephron 2023;147(8):478-495. Epub 2023 Mar 7 doi: 10.1159/000528557. PMID: 36882007

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