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Bartter disease type 1(BARTS1)

MedGen UID:
355727
Concept ID:
C1866495
Disease or Syndrome
Synonyms: Bartter syndrome, type 1, antenatal; Hyperprostaglandin E syndrome 1; HYPOKALEMIC ALKALOSIS WITH HYPERCALCIURIA 1, ANTENATAL
SNOMED CT: Hypokalemic alkalosis with hypercalciuria antenatal type 1 (700107006); Bartter syndrome antenatal type 1 (700107006); Hyperprostaglandin E syndrome type 1 (700107006); Bartter's syndrome type 1 (700107006)
 
Gene (location): SLC12A1 (15q21.1)
 
Monarch Initiative: MONDO:0100344
OMIM®: 601678
Orphanet: ORPHA620217

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 forms of Bartter syndrome 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 (see BARTS3, 607364) present later in life and may be sporadically asymptomatic or mildly symptomatic (summary by Simon et al., 1996 and Fremont and Chan, 2012). For a discussion of genetic heterogeneity of Bartter syndrome, see 607364. [from OMIM]

Clinical features

From HPO
Hypercalciuria
MedGen UID:
43775
Concept ID:
C0020438
Finding
Abnormally high level of calcium 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.
Hyposthenuria
MedGen UID:
68565
Concept ID:
C0232831
Finding
An abnormally low urinary specific gravity, i.e., reduced concentration of solutes in the urine.
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.
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.
Renal juxtaglomerular cell hypertrophy/hyperplasia
MedGen UID:
356547
Concept ID:
C1866496
Finding
Increased number and size of the juxtaglomerular cells.
Hyperprostaglandinuria
MedGen UID:
401015
Concept ID:
C1866498
Finding
An increased concentration of prostaglandin in the urine.
Low-to-normal blood pressure
MedGen UID:
356549
Concept ID:
C1866500
Finding
Small for gestational age
MedGen UID:
65920
Concept ID:
C0235991
Finding
Smaller than normal size according to sex and gestational age related norms, defined as a weight below the 10th percentile for the gestational age.
Short stature
MedGen UID:
87607
Concept ID:
C0349588
Finding
A height below that which is expected according to age and gender norms. Although there is no universally accepted definition of short stature, many refer to "short stature" as height more than 2 standard deviations below the mean for age and gender (or below the 3rd percentile for age and gender dependent norms).
Failure to thrive
MedGen UID:
746019
Concept ID:
C2315100
Disease or Syndrome
Failure to thrive (FTT) refers to a child whose physical growth is substantially below the norm.
Constipation
MedGen UID:
1101
Concept ID:
C0009806
Sign or Symptom
Infrequent or difficult evacuation of feces.
Diarrhea
MedGen UID:
8360
Concept ID:
C0011991
Sign or Symptom
Abnormally increased frequency (usually defined as three or more) loose or watery bowel movements a day.
Vomiting
MedGen UID:
12124
Concept ID:
C0042963
Sign or Symptom
Forceful ejection of the contents of the stomach through the mouth by means of a series of involuntary spasmic contractions.
Paresthesia
MedGen UID:
14619
Concept ID:
C0030554
Disease or Syndrome
Abnormal sensations such as tingling, pricking, or numbness of the skin with no apparent physical cause.
Seizure
MedGen UID:
20693
Concept ID:
C0036572
Sign or Symptom
A seizure is an intermittent abnormality of nervous system physiology characterized by a transient occurrence of signs and/or symptoms due to abnormal excessive or synchronous neuronal activity in the brain.
Global developmental delay
MedGen UID:
107838
Concept ID:
C0557874
Finding
A delay in the achievement of motor or mental milestones in the domains of development of a child, including motor skills, speech and language, cognitive skills, and social and emotional skills. This term should only be used to describe children younger than five years of age.
Intellectual disability
MedGen UID:
811461
Concept ID:
C3714756
Mental or Behavioral Dysfunction
Intellectual disability, previously referred to as mental retardation, is characterized by subnormal intellectual functioning that occurs during the developmental period. It is defined by an IQ score below 70.
Osteopenia
MedGen UID:
18222
Concept ID:
C0029453
Disease or Syndrome
Osteopenia is a term to define bone density that is not normal but also not as low as osteoporosis. By definition from the World Health Organization osteopenia is defined by bone densitometry as a T score -1 to -2.5.
Muscle spasm
MedGen UID:
52431
Concept ID:
C0037763
Sign or Symptom
Sudden and involuntary contractions of one or more muscles.
Tetany
MedGen UID:
11748
Concept ID:
C0039621
Finding
A condition characterized by intermittent involuntary contraction of muscles (spasms) related to hypocalcemia or occasionally magnesium deficiency.
Chondrocalcinosis
MedGen UID:
154303
Concept ID:
C0553730
Disease or Syndrome
Radiographic evidence of articular calcification that represent calcium pyrophosphate depositions in soft tissue surrounding joints and at the insertions of tendons near joints (Entheses/Sharpey fibers) .
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.
Fever
MedGen UID:
5169
Concept ID:
C0015967
Sign or Symptom
Body temperature elevated above the normal range.
Hypercalcemia
MedGen UID:
5686
Concept ID:
C0020437
Disease or Syndrome
An abnormally increased calcium concentration in the blood.
Hypokalemia
MedGen UID:
5712
Concept ID:
C0020621
Finding
An abnormally decreased potassium concentration in the blood.
Hypochloremia
MedGen UID:
39088
Concept ID:
C0085680
Disease or Syndrome
An abnormally decreased chloride concentration in the blood.
Hypomagnesemia
MedGen UID:
57481
Concept ID:
C0151723
Disease or Syndrome
An abnormally decreased magnesium concentration in the blood.
Hypokalemic metabolic alkalosis
MedGen UID:
152852
Concept ID:
C0740898
Disease or Syndrome
Increased serum prostaglandin E2
MedGen UID:
461708
Concept ID:
C3150358
Finding
An increased concentration of prostaglandin E2 in the blood.
Polyhydramnios
MedGen UID:
6936
Concept ID:
C0020224
Pathologic Function
The presence of excess amniotic fluid in the uterus during pregnancy.
Premature birth
MedGen UID:
57721
Concept ID:
C0151526
Pathologic Function
The birth of a baby of less than 37 weeks of gestational age.
Fetal polyuria
MedGen UID:
355948
Concept ID:
C1865279
Finding
Abnormally increased production of urine by the fetus resulting in polyhydramnios.
Increased circulating aldosterone concentration
MedGen UID:
6960
Concept ID:
C0020428
Disease or Syndrome
Overproduction of the mineralocorticoid aldosterone by the adrenal cortex.
Hyperparathyroidism
MedGen UID:
6967
Concept ID:
C0020502
Disease or Syndrome
Excessive production of parathyroid hormone (PTH) by the parathyroid glands.
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.

Professional guidelines

PubMed

Forbes TA, Wallace J, Kumble S, Delatycki MB, Stark Z
J Paediatr Child Health 2022 May;58(5):758-761. Epub 2022 Mar 29 doi: 10.1111/jpc.15955. PMID: 35348259Free PMC Article
Honka H, Salehi M
Curr Opin Clin Nutr Metab Care 2019 Jul;22(4):295-302. doi: 10.1097/MCO.0000000000000574. PMID: 31082828Free PMC Article
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
Genome Med 2023 Aug 23;15(1):62. doi: 10.1186/s13073-023-01215-1. PMID: 37612755Free PMC Article
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
Mrad FCC, Soares SBM, de Menezes Silva LAW, Dos Anjos Menezes PV, Simões-E-Silva AC
World J Pediatr 2021 Feb;17(1):31-39. Epub 2020 Jun 1 doi: 10.1007/s12519-020-00370-4. PMID: 32488762
Lopez HU, Haverfield E, Chung WK
Pediatr Dev Pathol 2015 Jul-Aug;18(4):324-6. Epub 2015 Apr 29 doi: 10.2350/14-08-1543-CR.1. PMID: 25923035
Knoers NV, Levtchenko EN
Orphanet J Rare Dis 2008 Jul 30;3:22. doi: 10.1186/1750-1172-3-22. PMID: 18667063Free PMC Article

Diagnosis

Stavniichuk A, Pyrshev K, Tomilin VN, Kordysh M, Zaika O, Pochynyuk O
Biomolecules 2023 Jan 14;13(1) doi: 10.3390/biom13010177. PMID: 36671562Free PMC Article
Palazzo V, Raglianti V, Landini S, Cirillo L, Errichiello C, Buti E, Artuso R, Tiberi L, Vergani D, Dirupo E, Romagnani P, Mazzinghi B, Becherucci F
Int J Mol Sci 2022 May 18;23(10) doi: 10.3390/ijms23105641. PMID: 35628451Free PMC Article
Mrad FCC, Soares SBM, de Menezes Silva LAW, Dos Anjos Menezes PV, Simões-E-Silva AC
World J Pediatr 2021 Feb;17(1):31-39. Epub 2020 Jun 1 doi: 10.1007/s12519-020-00370-4. PMID: 32488762
Assadi F
Iran J Kidney Dis 2008 Jul;2(3):115-22. PMID: 19377223
Bockenhauer D, Cruwys M, Kleta R, Halperin LF, Wildgoose P, Souma T, Nukiwa N, Cheema-Dhadli S, Chong CK, Kamel KS, Davids MR, Halperin ML
QJM 2008 Dec;101(12):927-42. Epub 2008 Oct 1 doi: 10.1093/qjmed/hcn119. PMID: 18829713

Therapy

Umene R, Kitamura M, Arai H, Matsumura K, Ishimaru Y, Maeda K, Uramatsu T, Obata Y, Mori T, Sohara E, Uchida S, Nishino T
CEN Case Rep 2020 Nov;9(4):375-379. Epub 2020 Jun 6 doi: 10.1007/s13730-020-00489-3. PMID: 32506365Free PMC Article
Carmosino M, Procino G, Svelto M
Biol Cell 2012 Apr;104(4):201-12. Epub 2012 Jan 26 doi: 10.1111/boc.201100049. PMID: 22211456
Assadi F
Iran J Kidney Dis 2008 Jul;2(3):115-22. PMID: 19377223
Bockenhauer D, Cruwys M, Kleta R, Halperin LF, Wildgoose P, Souma T, Nukiwa N, Cheema-Dhadli S, Chong CK, Kamel KS, Davids MR, Halperin ML
QJM 2008 Dec;101(12):927-42. Epub 2008 Oct 1 doi: 10.1093/qjmed/hcn119. PMID: 18829713
Iba K, Morii H, Wada M, Yasumoto R, Kishimoto T, Mitsuhashi T, Hojo K, Okahara T
Endocrinol Jpn 1981 Oct;28(5):595-604. doi: 10.1507/endocrj1954.28.595. PMID: 7047153

Prognosis

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
Palazzo V, Raglianti V, Landini S, Cirillo L, Errichiello C, Buti E, Artuso R, Tiberi L, Vergani D, Dirupo E, Romagnani P, Mazzinghi B, Becherucci F
Int J Mol Sci 2022 May 18;23(10) doi: 10.3390/ijms23105641. PMID: 35628451Free 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
Lopez HU, Haverfield E, Chung WK
Pediatr Dev Pathol 2015 Jul-Aug;18(4):324-6. Epub 2015 Apr 29 doi: 10.2350/14-08-1543-CR.1. PMID: 25923035
Knoers NV, Levtchenko EN
Orphanet J Rare Dis 2008 Jul 30;3:22. doi: 10.1186/1750-1172-3-22. PMID: 18667063Free PMC Article

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
Lopez HU, Haverfield E, Chung WK
Pediatr Dev Pathol 2015 Jul-Aug;18(4):324-6. Epub 2015 Apr 29 doi: 10.2350/14-08-1543-CR.1. PMID: 25923035
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

Recent systematic reviews

Qasba RK, Bucharles ACF, Piccoli MVF, Sharma P, Banga A, Kamaraj B, Nawaz FA, Kumar HJ, Happy MA, Qasba RK, Kogilathota Jagirdhar GS, Essar MY, Garg P, Reddy ST, Rama K, Surani S, Kashyap R
Medicina (Kaunas) 2023 Sep 11;59(9) doi: 10.3390/medicina59091638. PMID: 37763757Free PMC Article
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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