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Fanconi renotubular syndrome 3(FRTS3)

MedGen UID:
816430
Concept ID:
C3810100
Disease or Syndrome
Synonym: FRTS3
 
Gene (location): EHHADH (3q27.2)
 
Monarch Initiative: MONDO:0014275
OMIM®: 615605

Definition

Fanconi renotubular syndrome-3 (FRTS3) is an autosomal dominant disorder characterized by rickets, impaired growth, glucosuria, generalized aminoaciduria, phosphaturia, metabolic acidosis, and low molecular weight proteinuria (summary by Klootwijk et al., 2014). For a general phenotypic description and a discussion of genetic heterogeneity of Fanconi renotubular syndrome, see FRTS1 (134600). [from OMIM]

Clinical features

From HPO
Glycosuria
MedGen UID:
42267
Concept ID:
C0017979
Finding
An increased concentration of glucose in the urine.
Aminoaciduria
MedGen UID:
116067
Concept ID:
C0238621
Disease or Syndrome
An increased concentration of an amino acid in the urine.
Hyperphosphaturia
MedGen UID:
78638
Concept ID:
C0268079
Disease or Syndrome
An increased excretion of phosphates in the urine.
Renal insufficiency
MedGen UID:
332529
Concept ID:
C1565489
Disease or Syndrome
A reduction in the level of performance of the kidneys in areas of function comprising the concentration of urine, removal of wastes, the maintenance of electrolyte balance, homeostasis of blood pressure, and calcium metabolism.
Low-molecular-weight proteinuria
MedGen UID:
333360
Concept ID:
C1839606
Finding
Excretion in urine of proteins of a size smaller than albumin (molecular weight 69 kD).
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.
Rickets
MedGen UID:
48470
Concept ID:
C0035579
Disease or Syndrome
Rickets is divided into two major categories including calcipenic and phosphopenic. Hypophosphatemia is described as a common manifestation of both categories. Hypophosphatemic rickets is the most common type of rickets that is characterized by low levels of serum phosphate, resistance to ultraviolet radiation or vitamin D intake. There are several issues involved in hypophosphatemic rickets such as calcium, vitamin D, phosphorus deficiencies. Moreover, other disorder can be associated with its occurrence such as absorption defects due to pancreatic, intestinal, gastric, and renal disorders and hepatobiliary disease. Symptoms are usually seen in childhood and can be varied in severity. Severe forms may be linked to bowing of the legs, poor bone growth, and short stature as well as joint and bone pain. Hypophosphatemic rickets are associated with renal excretion of phosphate, hypophosphatemia, and mineral defects in bones. The familial type of the disease is the most common type of rickets.
Bowing of the legs
MedGen UID:
1807399
Concept ID:
C5574706
Finding
A bending or abnormal curvature affecting a long bone of the leg.
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.
Elevated circulating creatinine concentration
MedGen UID:
148579
Concept ID:
C0700225
Finding
An increased amount of creatinine in the blood.

Professional guidelines

PubMed

Wlodarski MW, Vlachos A, Farrar JE, Da Costa LM, Kattamis A, Dianzani I, Belendez C, Unal S, Tamary H, Pasauliene R, Pospisilova D, de la Fuente J, Iskander D, Wolfe L, Liu JM, Shimamura A, Albrecht K, Lausen B, Bechensteen AG, Tedgard U, Puzik A, Quarello P, Ramenghi U, Bartels M, Hengartner H, Farah RA, Al Saleh M, Hamidieh AA, Yang W, Ito E, Kook H, Ovsyannikova G, Kager L, Gleizes PE, Dalle JH, Strahm B, Niemeyer CM, Lipton JM, Leblanc TM; international Diamond-Blackfan anaemia syndrome guideline panel
Lancet Haematol 2024 May;11(5):e368-e382. doi: 10.1016/S2352-3026(24)00063-2. PMID: 38697731
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
Haffner D, Leifheit-Nestler M, Grund A, Schnabel D
Pediatr Nephrol 2022 Oct;37(10):2289-2302. Epub 2022 Mar 29 doi: 10.1007/s00467-022-05505-5. PMID: 35352187Free PMC Article

Recent clinical studies

Etiology

El Younsi M, Trabelsi M, Ben Youssef S, Ouertani I, Hammi Y, Achour A, Maazoul F, Kharrat M, Gargah T, M'rad R
Pediatr Nephrol 2023 Jan;38(1):119-129. Epub 2022 Apr 20 doi: 10.1007/s00467-022-05525-1. PMID: 35445972
Emma F, Salviati L
Nephrol Ther 2017 Apr;13 Suppl 1:S23-S28. doi: 10.1016/j.nephro.2017.01.014. PMID: 28577739
Yurgelun MB, Kulke MH, Fuchs CS, Allen BA, Uno H, Hornick JL, Ukaegbu CI, Brais LK, McNamara PG, Mayer RJ, Schrag D, Meyerhardt JA, Ng K, Kidd J, Singh N, Hartman AR, Wenstrup RJ, Syngal S
J Clin Oncol 2017 Apr 1;35(10):1086-1095. Epub 2017 Jan 30 doi: 10.1200/JCO.2016.71.0012. PMID: 28135145Free PMC Article
Mak LY, Seto WK, Lai CL, Yuen MF
Expert Opin Drug Saf 2016;15(3):383-92. Epub 2016 Feb 1 doi: 10.1517/14740338.2016.1139573. PMID: 26752687
Nowell PC
Cancer Genet Cytogenet 1982 Mar;5(3):265-78. doi: 10.1016/0165-4608(82)90034-6. PMID: 7039817

Diagnosis

Kunchur MG, Mauch TJ, Parkanzky M, Rahilly LJ
J Vet Emerg Crit Care (San Antonio) 2024 Jul-Aug;34(4):325-355. Epub 2024 Jul 18 doi: 10.1111/vec.13407. PMID: 39023331
Wlodarski MW, Vlachos A, Farrar JE, Da Costa LM, Kattamis A, Dianzani I, Belendez C, Unal S, Tamary H, Pasauliene R, Pospisilova D, de la Fuente J, Iskander D, Wolfe L, Liu JM, Shimamura A, Albrecht K, Lausen B, Bechensteen AG, Tedgard U, Puzik A, Quarello P, Ramenghi U, Bartels M, Hengartner H, Farah RA, Al Saleh M, Hamidieh AA, Yang W, Ito E, Kook H, Ovsyannikova G, Kager L, Gleizes PE, Dalle JH, Strahm B, Niemeyer CM, Lipton JM, Leblanc TM; international Diamond-Blackfan anaemia syndrome guideline panel
Lancet Haematol 2024 May;11(5):e368-e382. doi: 10.1016/S2352-3026(24)00063-2. PMID: 38697731
Lemaire M
Am J Physiol Renal Physiol 2021 Feb 1;320(2):F145-F160. Epub 2020 Dec 7 doi: 10.1152/ajprenal.00214.2020. PMID: 33283647
Yurgelun MB, Kulke MH, Fuchs CS, Allen BA, Uno H, Hornick JL, Ukaegbu CI, Brais LK, McNamara PG, Mayer RJ, Schrag D, Meyerhardt JA, Ng K, Kidd J, Singh N, Hartman AR, Wenstrup RJ, Syngal S
J Clin Oncol 2017 Apr 1;35(10):1086-1095. Epub 2017 Jan 30 doi: 10.1200/JCO.2016.71.0012. PMID: 28135145Free PMC Article
Karande S, Kumbhare N, Kulkarni M
Indian Pediatr 2007 Mar;44(3):223-5. PMID: 17413201

Therapy

Webster ALH, Sanders MA, Patel K, Dietrich R, Noonan RJ, Lach FP, White RR, Goldfarb A, Hadi K, Edwards MM, Donovan FX, Hoogenboezem RM, Jung M, Sridhar S, Wiley TF, Fedrigo O, Tian H, Rosiene J, Heineman T, Kennedy JA, Bean L, Rosti RO, Tryon R, Gonzalez AM, Rosenberg A, Luo JD, Carroll TS, Shroff S, Beaumont M, Velleuer E, Rastatter JC, Wells SI, Surrallés J, Bagby G, MacMillan ML, Wagner JE, Cancio M, Boulad F, Scognamiglio T, Vaughan R, Beaumont KG, Koren A, Imielinski M, Chandrasekharappa SC, Auerbach AD, Singh B, Kutler DI, Campbell PJ, Smogorzewska A
Nature 2022 Dec;612(7940):495-502. Epub 2022 Nov 30 doi: 10.1038/s41586-022-05253-4. PMID: 36450981Free PMC Article
Haffner D, Leifheit-Nestler M, Grund A, Schnabel D
Pediatr Nephrol 2022 Oct;37(10):2289-2302. Epub 2022 Mar 29 doi: 10.1007/s00467-022-05505-5. PMID: 35352187Free PMC Article
Kumar B, Prabhakar R, Thangavelu S
J R Coll Physicians Edinb 2020 Sep;50(3):291-294. doi: 10.4997/JRCPE.2020.316. PMID: 32936106
Mak LY, Seto WK, Lai CL, Yuen MF
Expert Opin Drug Saf 2016;15(3):383-92. Epub 2016 Feb 1 doi: 10.1517/14740338.2016.1139573. PMID: 26752687
Tsimihodimos V, Psychogios N, Kakaidi V, Bairaktari E, Elisaf M
Am J Kidney Dis 2007 Sep;50(3):463-7. doi: 10.1053/j.ajkd.2007.03.021. PMID: 17720526

Prognosis

Kumar B, Prabhakar R, Thangavelu S
J R Coll Physicians Edinb 2020 Sep;50(3):291-294. doi: 10.4997/JRCPE.2020.316. PMID: 32936106
Yurgelun MB, Kulke MH, Fuchs CS, Allen BA, Uno H, Hornick JL, Ukaegbu CI, Brais LK, McNamara PG, Mayer RJ, Schrag D, Meyerhardt JA, Ng K, Kidd J, Singh N, Hartman AR, Wenstrup RJ, Syngal S
J Clin Oncol 2017 Apr 1;35(10):1086-1095. Epub 2017 Jan 30 doi: 10.1200/JCO.2016.71.0012. PMID: 28135145Free PMC Article
Mak LY, Seto WK, Lai CL, Yuen MF
Expert Opin Drug Saf 2016;15(3):383-92. Epub 2016 Feb 1 doi: 10.1517/14740338.2016.1139573. PMID: 26752687
Freedman MH
Baillieres Best Pract Res Clin Haematol 2000 Sep;13(3):391-406. doi: 10.1053/beha.2000.0084. PMID: 11030041
Nowell PC
Cancer Genet Cytogenet 1982 Mar;5(3):265-78. doi: 10.1016/0165-4608(82)90034-6. PMID: 7039817

Clinical prediction guides

Yurgelun MB, Kulke MH, Fuchs CS, Allen BA, Uno H, Hornick JL, Ukaegbu CI, Brais LK, McNamara PG, Mayer RJ, Schrag D, Meyerhardt JA, Ng K, Kidd J, Singh N, Hartman AR, Wenstrup RJ, Syngal S
J Clin Oncol 2017 Apr 1;35(10):1086-1095. Epub 2017 Jan 30 doi: 10.1200/JCO.2016.71.0012. PMID: 28135145Free PMC Article
Mak LY, Seto WK, Lai CL, Yuen MF
Expert Opin Drug Saf 2016;15(3):383-92. Epub 2016 Feb 1 doi: 10.1517/14740338.2016.1139573. PMID: 26752687
Poumpouridou N, Kroupis C
Clin Chem Lab Med 2011 Dec 23;50(3):423-34. doi: 10.1515/cclm-2011-0840. PMID: 22505525
Tsimihodimos V, Psychogios N, Kakaidi V, Bairaktari E, Elisaf M
Am J Kidney Dis 2007 Sep;50(3):463-7. doi: 10.1053/j.ajkd.2007.03.021. PMID: 17720526
Caskey CT
Perspect Nephrol Hypertens 1976;3:255-74. PMID: 765953

Recent systematic reviews

Dos Santos ES, Pérez-de-Oliveira ME, Normando AGC, Gueiros LAM, Rogatto SR, Vargas PA, Lopes MA, da Silva Guerra EN, Leme AFP, Santos-Silva AR
Head Neck 2022 Dec;44(12):2925-2937. Epub 2022 Sep 16 doi: 10.1002/hed.27193. PMID: 36114663
Medic G, van der Weijden M, Karabis A, Hemels M
Curr Med Res Opin 2017 Nov;33(11):2065-2076. Epub 2017 Aug 3 doi: 10.1080/03007995.2017.1354288. PMID: 28692321
Phillips RS, Tyerman K, Al-Kassim MI, Picton S
Pediatr Hematol Oncol 2008 Mar;25(2):107-13. doi: 10.1080/08880010701885276. PMID: 18363176

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