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Autosomal recessive proximal renal tubular acidosis

MedGen UID:
370883
Concept ID:
C1970309
Disease or Syndrome
Synonyms: Proximal Renal Tubular Acidosis with Ocular Abnormalities; RENAL TUBULAR ACIDOSIS, PROXIMAL, WITH OCULAR ABNORMALITIES AND IMPAIRED INTELLECTUAL DEVELOPMENT; RTA, PROXIMAL, AUTOSOMAL RECESSIVE
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): SLC4A4 (4q13.3)
 
Monarch Initiative: MONDO:0011422
OMIM®: 604278
Orphanet: ORPHA93607

Definition

A rare autosomal recessive form of proximal renal tubular acidosis characterized by an isolated defect in the proximal tubule leading to the decreased reabsorption of bicarbonate and consequentially to urinary bicarbonate wastage. Presentation is typically with hyperchloremic acidosis, usually occurring in childhood. Extrarenal manifestations include ocular abnormalities (band keratopathy, glaucoma, and cataracts), intellectual disability and severe growth retardation. Other features like dental enamel defects, basal ganglia calcification and pancreatitis are sometimes present. [from ORDO]

Clinical features

From HPO
Impaired renal tubular reabsorption of bicarbonate
MedGen UID:
1783147
Concept ID:
C5539860
Finding
Decreased renal tubular reabsorption of bicarbonate.
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).
Growth delay
MedGen UID:
99124
Concept ID:
C0456070
Pathologic Function
A deficiency or slowing down of growth pre- and postnatally.
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.
Increased red cell osmotic resistance
MedGen UID:
346950
Concept ID:
C1858628
Finding
Cerebral calcification
MedGen UID:
124360
Concept ID:
C0270685
Finding
The presence of calcium deposition within the cerebrum.
Hypokalemia
MedGen UID:
5712
Concept ID:
C0020621
Finding
An abnormally decreased potassium concentration in the blood.
Hyperchloremic acidosis
MedGen UID:
43207
Concept ID:
C0085569
Disease or Syndrome
Acidosis (pH less than 7.35) that develops with an increase in ionic chloride.
Metabolic acidosis
MedGen UID:
65117
Concept ID:
C0220981
Pathologic Function
Metabolic acidosis (MA) is characterized by a fall in blood pH due to a reduction of serum bicarbonate concentration. This can occur as a result of either the accumulation of acids (high anion gap MA) or the loss of bicarbonate from the gastrointestinal tract or the kidney (hyperchloremic MA). By definition, MA is not due to a respirary cause.
Proximal renal tubular acidosis
MedGen UID:
82804
Concept ID:
C0268435
Disease or Syndrome
A type of renal tubular acidosis characterized by a failure of the proximal tubular cells to reabsorb bicarbonate, leading to urinary bicarbonate wasting and subsequent acidemia.
Hyperamylasemia
MedGen UID:
105401
Concept ID:
C0476327
Finding
Increased level of amylase in the blood, an enzyme which helps digest glycogen and starch. It is produced mainly in the pancreas and salivary glands.
Bicarbonate-wasting renal tubular acidosis
MedGen UID:
347681
Concept ID:
C1858626
Disease or Syndrome
Decreased serum bicarbonate concentration
MedGen UID:
1670967
Concept ID:
C4732833
Finding
An abnormal reduction of the concentration of bicarbonate, HCO3[-], in the circulation.
Glaucoma
MedGen UID:
42224
Concept ID:
C0017601
Disease or Syndrome
Glaucoma refers loss of retinal ganglion cells in a characteristic pattern of optic neuropathy usually associated with increased intraocular pressure.
Cataract
MedGen UID:
39462
Concept ID:
C0086543
Disease or Syndrome
A cataract is an opacity or clouding that develops in the crystalline lens of the eye or in its capsule.
Band-shaped keratopathy
MedGen UID:
56354
Concept ID:
C0155120
Disease or Syndrome
An abnormality of the cornea characterized by the deposition of calcium in a band across the central cornea, leading to decreased vision, foreign body sensation, and ocular irritation.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVAutosomal recessive proximal renal tubular acidosis
Follow this link to review classifications for Autosomal recessive proximal renal tubular acidosis in Orphanet.

Professional guidelines

PubMed

Du T, Xia Y, Sun C, Gong Z, Liang L, Gong Z, Wang R, Lu D, Zhang K, Yang Y, Sun Y, Sun M, Sun Y, Xiao B, Qiu W
Orphanet J Rare Dis 2024 Feb 16;19(1):75. doi: 10.1186/s13023-024-03070-8. PMID: 38365697Free PMC Article
Hammi Y, Charfi H, Ferjani M, Sayari T, Mrad R, Gargah T
Tunis Med 2023 Aug-Sep;101(8-9):704-708. PMID: 38445406
Sundaram V, Rumbolo P, Grubb J, Strisciuglio P, Sly WS
Am J Hum Genet 1986 Feb;38(2):125-36. PMID: 3080873Free PMC Article

Recent clinical studies

Therapy

Azimov R, Abuladze N, Sassani P, Newman D, Kao L, Liu W, Orozco N, Ruchala P, Pushkin A, Kurtz I
Am J Physiol Renal Physiol 2008 Sep;295(3):F633-41. Epub 2008 Jul 9 doi: 10.1152/ajprenal.00015.2008. PMID: 18614622Free PMC Article

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