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Familial hypobetalipoproteinemia 2(FHBL2)

MedGen UID:
341895
Concept ID:
C1857970
Disease or Syndrome
Synonym: HYPOLIPIDEMIA, FAMILIAL, COMBINED
 
Gene (location): ANGPTL3 (1p31.3)
 
Monarch Initiative: MONDO:0011505
OMIM®: 605019

Disease characteristics

Excerpted from the GeneReview: Familial Combined Hypolipidemia
Familial combined hypolipidemia is not associated with any pathologic signs or symptoms; diagnosis is suggested by low plasma concentrations of lipids. The lipid profile is one of hypocholesterolemia with low plasma low-density lipoprotein (LDL) cholesterol, low plasma high-density lipoprotein (HDL) cholesterol, low plasma triglycerides, and low plasma apolipoprotein (apo) B and apo A-I levels. [from GeneReviews]
Authors:
John R Burnett  |  Amanda J Hooper  |  Robert A Hegele   view full author information

Additional descriptions

From OMIM
Hypobetalipoproteinemia (HBL) is defined as permanently low levels, below the 5th percentile of sex- and age-matched individuals in the population, of apolipoprotein B (apoB), total cholesterol, and low-density lipoprotein (LDL) cholesterol; the lipid profile in FHBL2 includes low HDL cholesterol as well. HBL can result from environmental factors such as a strict vegetarian diet, or can be secondary to certain diseases such as intestinal fat malabsorption, chronic pancreatitis, severe liver disease, malnutrition, or hyperthyroidism. Heritable primary causes of HBL include chylomicron retention disease (CMRD; 246700), abetalipoproteinemia (200100), and familial hypobetalipoproteinemia (FHBL) (summary by Martin-Campos et al., 2012). For a discussion of genetic heterogeneity of familial hypobetalipoproteinemia, see FHBL1 (615558).  http://www.omim.org/entry/605019
From MedlinePlus Genetics
Familial hypobetalipoproteinemia (FHBL) is a disorder that impairs the body's ability to absorb and transport fats. This condition is characterized by low levels of a fat-like substance called cholesterol in the blood. The severity of signs and symptoms experienced by people with FHBL vary widely. The most mildly affected individuals have few problems with absorbing fats from the diet and no related signs and symptoms. Many individuals with FHBL develop an abnormal buildup of fats in the liver called hepatic steatosis or fatty liver. In more severely affected individuals, fatty liver may progress to chronic liver disease (cirrhosis). Individuals with severe FHBL have greater difficulty absorbing fats as well as fat-soluble vitamins such as vitamin E and vitamin A. This difficulty in fat absorption leads to excess fat in the feces (steatorrhea). In childhood, these digestive problems can result in an inability to grow or gain weight at the expected rate (failure to thrive).  https://medlineplus.gov/genetics/condition/familial-hypobetalipoproteinemia

Clinical features

From HPO
Hypotriglyceridemia
MedGen UID:
488913
Concept ID:
C0542037
Disease or Syndrome
An decrease in the level of triglycerides in the blood.
Decreased LDL cholesterol concentration
MedGen UID:
776554
Concept ID:
C0853085
Finding
An decreased concentration of low-density lipoprotein cholesterol in the blood.

Professional guidelines

PubMed

Long MT, Noureddin M, Lim JK
Gastroenterology 2022 Sep;163(3):764-774.e1. Epub 2022 Jul 14 doi: 10.1053/j.gastro.2022.06.023. PMID: 35842345Free PMC Article
Bredefeld C, Peretti N, Hussain MM; Medical Advisory Panel
Gastroenterology 2021 May;160(6):1912-1916. Epub 2020 Dec 1 doi: 10.1053/j.gastro.2020.11.040. PMID: 33275938
Burnett JR, Bell DA, Hooper AJ, Hegele RA
Eur J Hum Genet 2015 Jun;23(6):890-. Epub 2014 Oct 22 doi: 10.1038/ejhg.2014.225. PMID: 25335495Free PMC Article

Recent clinical studies

Etiology

Long MT, Noureddin M, Lim JK
Gastroenterology 2022 Sep;163(3):764-774.e1. Epub 2022 Jul 14 doi: 10.1053/j.gastro.2022.06.023. PMID: 35842345Free PMC Article
Tikka A, Jauhiainen M
Endocrine 2016 May;52(2):187-93. Epub 2016 Jan 11 doi: 10.1007/s12020-015-0838-9. PMID: 26754661Free PMC Article
Jung HH, Danek A, Walker RH
Orphanet J Rare Dis 2011 Oct 25;6:68. doi: 10.1186/1750-1172-6-68. PMID: 22027213Free PMC Article
Moutzouri E, Elisaf M, Liberopoulos EN
Curr Vasc Pharmacol 2011 Mar;9(2):200-12. doi: 10.2174/157016111794519354. PMID: 20626336
Stevenson VL, Hardie RJ
J Neurol 2001 Feb;248(2):87-94. doi: 10.1007/s004150170241. PMID: 11284140

Diagnosis

Long MT, Noureddin M, Lim JK
Gastroenterology 2022 Sep;163(3):764-774.e1. Epub 2022 Jul 14 doi: 10.1053/j.gastro.2022.06.023. PMID: 35842345Free PMC Article
Desaldeleer C, Henno S, Bruneau B, Dabadie A
Dig Liver Dis 2013 Feb;45(2):e3. Epub 2012 Sep 5 doi: 10.1016/j.dld.2012.08.003. PMID: 22959141
Jung HH, Danek A, Walker RH
Orphanet J Rare Dis 2011 Oct 25;6:68. doi: 10.1186/1750-1172-6-68. PMID: 22027213Free PMC Article
Moutzouri E, Elisaf M, Liberopoulos EN
Curr Vasc Pharmacol 2011 Mar;9(2):200-12. doi: 10.2174/157016111794519354. PMID: 20626336
Lloyd JK
Clin Endocrinol Metab 1973 Mar;2(1):127-47. doi: 10.1016/s0300-595x(73)80030-1. PMID: 4361962

Therapy

Legland Ép Dejean AM, Plantamura J, Arnoux I, Paleiron N, Loosveld M, Buono Ép Foucher B
Br J Haematol 2022 Apr;197(2):131. Epub 2022 Feb 7 doi: 10.1111/bjh.18020. PMID: 35128635
Welty FK
Curr Opin Lipidol 2020 Apr;31(2):49-55. doi: 10.1097/MOL.0000000000000663. PMID: 32039990
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Prognosis

Long MT, Noureddin M, Lim JK
Gastroenterology 2022 Sep;163(3):764-774.e1. Epub 2022 Jul 14 doi: 10.1053/j.gastro.2022.06.023. PMID: 35842345Free PMC Article
Takahashi M, Okazaki H, Ohashi K, Ogura M, Ishibashi S, Okazaki S, Hirayama S, Hori M, Matsuki K, Yokoyama S, Harada-Shiba M
J Atheroscler Thromb 2021 Oct 1;28(10):1009-1019. Epub 2021 May 16 doi: 10.5551/jat.RV17056. PMID: 33994405Free PMC Article
Simone ML, Rabacchi C, Kuloglu Z, Kansu A, Ensari A, Demir AM, Hizal G, Di Leo E, Bertolini S, Calandra S, Tarugi P
J Clin Lipidol 2019 Jul-Aug;13(4):554-562. Epub 2019 May 30 doi: 10.1016/j.jacl.2019.05.013. PMID: 31253576
Jung HH, Danek A, Walker RH
Orphanet J Rare Dis 2011 Oct 25;6:68. doi: 10.1186/1750-1172-6-68. PMID: 22027213Free PMC Article
Palau F, Espinós C
Orphanet J Rare Dis 2006 Nov 17;1:47. doi: 10.1186/1750-1172-1-47. PMID: 17112370Free PMC Article

Clinical prediction guides

Rimbert A, Yeung MW, Dalila N, Thio CHL, Yu H, Loaiza N, Oldoni F, van der Graaf A, Wang S, Said MA, Blauw LL, Girardeau A, Bray L, Caillaud A, Bloks VW, Marrec M, Moulin P, Rensen PCN, van de Sluis B, Snieder H, Di Filippo M, van der Harst P, Tybjaerg-Hansen A, Zimmerman P, Cariou B, Kuivenhoven JA
Arterioscler Thromb Vasc Biol 2022 Oct;42(10):1262-1271. Epub 2022 Sep 1 doi: 10.1161/ATVBAHA.122.317514. PMID: 36047410
Long MT, Noureddin M, Lim JK
Gastroenterology 2022 Sep;163(3):764-774.e1. Epub 2022 Jul 14 doi: 10.1053/j.gastro.2022.06.023. PMID: 35842345Free PMC Article
Simone ML, Rabacchi C, Kuloglu Z, Kansu A, Ensari A, Demir AM, Hizal G, Di Leo E, Bertolini S, Calandra S, Tarugi P
J Clin Lipidol 2019 Jul-Aug;13(4):554-562. Epub 2019 May 30 doi: 10.1016/j.jacl.2019.05.013. PMID: 31253576
Schonfeld G
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Recent systematic reviews

Noto D, Arca M, Tarugi P, Cefalù AB, Barbagallo CM, Averna MR
Acta Diabetol 2017 Feb;54(2):111-122. Epub 2016 Nov 2 doi: 10.1007/s00592-016-0931-4. PMID: 27804036

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