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Amyloidosis

MedGen UID:
272
Concept ID:
C0002726
Disease or Syndrome
Synonym: Amyloidoses
SNOMED CT: Amyloidosis (17602002)
 
HPO: HP:0011034
Monarch Initiative: MONDO:0019065
Orphanet: ORPHA69

Definition

The presence of amyloid deposition in one or more tissues. Amyloidosis may be defined as the extracellular deposition of amyloid in one or more sites of the body. [from HPO]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • Amyloidosis
Follow this link to review classifications for Amyloidosis in Orphanet.

Conditions with this feature

Multiple myeloma
MedGen UID:
10122
Concept ID:
C0026764
Neoplastic Process
Multiple myeloma is a neoplastic plasma cell disorder characterized by clonal proliferation of malignant plasma cells in the bone marrow microenvironment, monoclonal protein in the blood or urine, and associated organ dysfunction (Palumbo and Anderson, 2011).
Familial Mediterranean fever
MedGen UID:
45811
Concept ID:
C0031069
Disease or Syndrome
Familial Mediterranean fever (FMF) is divided into two phenotypes: type 1 and type 2. FMF type 1 is characterized by recurrent short episodes of inflammation and serositis including fever, peritonitis, synovitis, pleuritis, and, rarely, pericarditis and meningitis. The symptoms and severity vary among affected individuals, sometimes even among members of the same family. Amyloidosis, which can lead to renal failure, is the most severe complication, if untreated. FMF type 2 is characterized by amyloidosis as the first clinical manifestation of FMF in an otherwise asymptomatic individual.
Bullous cutaneous amyloidosis
MedGen UID:
78672
Concept ID:
C0268399
Disease or Syndrome
Amyloidosis of gingiva and conjunctiva, with intellectual disability
MedGen UID:
347240
Concept ID:
C1859815
Disease or Syndrome
Amyloidosis, hereditary systemic 1
MedGen UID:
414031
Concept ID:
C2751492
Disease or Syndrome
Hereditary transthyretin (ATTR) amyloidosis is characterized by a slowly progressive peripheral sensorimotor and/or autonomic neuropathy as well as non-neuropathic changes of cardiomyopathy, nephropathy, vitreous opacities, and CNS amyloidosis. The disease usually begins in the third to fifth decade in persons from endemic foci in Portugal and Japan; onset is later in persons from other areas. Typically, sensory neuropathy starts in the lower extremities with paresthesias and hypesthesias of the feet, followed within a few years by motor neuropathy. In some persons, particularly those with early-onset disease, autonomic neuropathy is the first manifestation of the condition; findings can include: orthostatic hypotension, constipation alternating with diarrhea, attacks of nausea and vomiting, delayed gastric emptying, sexual impotence, anhidrosis, and urinary retention or incontinence. Cardiac amyloidosis is mainly characterized by progressive cardiomyopathy. Individuals with leptomeningeal amyloidosis may have the following CNS findings: dementia, psychosis, visual impairment, headache, seizures, motor paresis, ataxia, myelopathy, hydrocephalus, or intracranial hemorrhage.
Corneal intraepithelial dyskeratosis-palmoplantar hyperkeratosis-laryngeal dyskeratosis syndrome
MedGen UID:
815206
Concept ID:
C3808876
Neoplastic Process
Multiple self-healing palmoplantar carcinoma (MSPC) is characterized by recurrent keratoacanthomas in palmoplantar skin as well as in conjunctival and corneal epithelia. In addition, patients experience a high susceptibility to malignant squamous cell carcinoma (summary by Zhong et al., 2016).

Professional guidelines

PubMed

Heidenreich PA, Bozkurt B, Aguilar D, Allen LA, Byun JJ, Colvin MM, Deswal A, Drazner MH, Dunlay SM, Evers LR, Fang JC, Fedson SE, Fonarow GC, Hayek SS, Hernandez AF, Khazanie P, Kittleson MM, Lee CS, Link MS, Milano CA, Nnacheta LC, Sandhu AT, Stevenson LW, Vardeny O, Vest AR, Yancy CW
Circulation 2022 May 3;145(18):e876-e894. Epub 2022 Apr 1 doi: 10.1161/CIR.0000000000001062. PMID: 35363500
Heidenreich PA, Bozkurt B, Aguilar D, Allen LA, Byun JJ, Colvin MM, Deswal A, Drazner MH, Dunlay SM, Evers LR, Fang JC, Fedson SE, Fonarow GC, Hayek SS, Hernandez AF, Khazanie P, Kittleson MM, Lee CS, Link MS, Milano CA, Nnacheta LC, Sandhu AT, Stevenson LW, Vardeny O, Vest AR, Yancy CW
Circulation 2022 May 3;145(18):e895-e1032. Epub 2022 Apr 1 doi: 10.1161/CIR.0000000000001063. PMID: 35363499
Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, Burazor I, Caforio ALP, Damy T, Eriksson U, Fontana M, Gillmore JD, Gonzalez-Lopez E, Grogan M, Heymans S, Imazio M, Kindermann I, Kristen AV, Maurer MS, Merlini G, Pantazis A, Pankuweit S, Rigopoulos AG, Linhart A
Eur Heart J 2021 Apr 21;42(16):1554-1568. doi: 10.1093/eurheartj/ehab072. PMID: 33825853Free PMC Article

Recent clinical studies

Etiology

Nader R, Angel-Korman A, Havasi A
Semin Nephrol 2022 Nov;42(6):151343. Epub 2023 May 4 doi: 10.1016/j.semnephrol.2023.151343. PMID: 37148782
Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, Burazor I, Caforio ALP, Damy T, Eriksson U, Fontana M, Gillmore JD, Gonzalez-Lopez E, Grogan M, Heymans S, Imazio M, Kindermann I, Kristen AV, Maurer MS, Merlini G, Pantazis A, Pankuweit S, Rigopoulos AG, Linhart A
Eur Heart J 2021 Apr 21;42(16):1554-1568. doi: 10.1093/eurheartj/ehab072. PMID: 33825853Free PMC Article
Vaxman I, Gertz M
Acta Haematol 2020;143(4):304-311. Epub 2020 Apr 27 doi: 10.1159/000506617. PMID: 32340017
Martinez-Naharro A, Hawkins PN, Fontana M
Clin Med (Lond) 2018 Apr 1;18(Suppl 2):s30-s35. doi: 10.7861/clinmedicine.18-2-s30. PMID: 29700090Free PMC Article
Wechalekar AD, Gillmore JD, Hawkins PN
Lancet 2016 Jun 25;387(10038):2641-2654. Epub 2015 Dec 21 doi: 10.1016/S0140-6736(15)01274-X. PMID: 26719234

Diagnosis

Gertz MA
Heart Fail Clin 2022 Jul;18(3):479-488. doi: 10.1016/j.hfc.2022.02.005. PMID: 35718420Free PMC Article
Gupta N, Kaur H, Wajid S
Protoplasma 2020 Sep;257(5):1259-1276. Epub 2020 May 24 doi: 10.1007/s00709-020-01513-0. PMID: 32447467
Martinez-Naharro A, Hawkins PN, Fontana M
Clin Med (Lond) 2018 Apr 1;18(Suppl 2):s30-s35. doi: 10.7861/clinmedicine.18-2-s30. PMID: 29700090Free PMC Article
Falk RH, Alexander KM, Liao R, Dorbala S
J Am Coll Cardiol 2016 Sep 20;68(12):1323-41. doi: 10.1016/j.jacc.2016.06.053. PMID: 27634125
Wechalekar AD, Gillmore JD, Hawkins PN
Lancet 2016 Jun 25;387(10038):2641-2654. Epub 2015 Dec 21 doi: 10.1016/S0140-6736(15)01274-X. PMID: 26719234

Therapy

Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators
Amyloid 2023 Mar;30(1):1-9. Epub 2022 Jul 23 doi: 10.1080/13506129.2022.2091985. PMID: 35875890
Palladini G, Merlini G
Blood 2022 May 12;139(19):2918-2930. doi: 10.1182/blood.2020008737. PMID: 34517412
Kastritis E, Palladini G, Minnema MC, Wechalekar AD, Jaccard A, Lee HC, Sanchorawala V, Gibbs S, Mollee P, Venner CP, Lu J, Schönland S, Gatt ME, Suzuki K, Kim K, Cibeira MT, Beksac M, Libby E, Valent J, Hungria V, Wong SW, Rosenzweig M, Bumma N, Huart A, Dimopoulos MA, Bhutani D, Waxman AJ, Goodman SA, Zonder JA, Lam S, Song K, Hansen T, Manier S, Roeloffzen W, Jamroziak K, Kwok F, Shimazaki C, Kim JS, Crusoe E, Ahmadi T, Tran N, Qin X, Vasey SY, Tromp B, Schecter JM, Weiss BM, Zhuang SH, Vermeulen J, Merlini G, Comenzo RL; ANDROMEDA Trial Investigators
N Engl J Med 2021 Jul 1;385(1):46-58. doi: 10.1056/NEJMoa2028631. PMID: 34192431
Gertz MA, Dispenzieri A
JAMA 2020 Jul 7;324(1):79-89. doi: 10.1001/jama.2020.5493. PMID: 32633805
Cullis PR, Hope MJ
Mol Ther 2017 Jul 5;25(7):1467-1475. Epub 2017 Apr 13 doi: 10.1016/j.ymthe.2017.03.013. PMID: 28412170Free PMC Article

Prognosis

Tam CS, Opat S, D'Sa S, Jurczak W, Lee HP, Cull G, Owen RG, Marlton P, Wahlin BE, Sanz RG, McCarthy H, Mulligan S, Tedeschi A, Castillo JJ, Czyz J, Fernández de Larrea C, Belada D, Libby E, Matous JV, Motta M, Siddiqi T, Tani M, Trneny M, Minnema MC, Buske C, Leblond V, Trotman J, Chan WY, Schneider J, Ro S, Cohen A, Huang J, Dimopoulos M
Blood 2020 Oct 29;136(18):2038-2050. doi: 10.1182/blood.2020006844. PMID: 32731259Free PMC Article
Lane T, Fontana M, Martinez-Naharro A, Quarta CC, Whelan CJ, Petrie A, Rowczenio DM, Gilbertson JA, Hutt DF, Rezk T, Strehina SG, Caringal-Galima J, Manwani R, Sharpley FA, Wechalekar AD, Lachmann HJ, Mahmood S, Sachchithanantham S, Drage EPS, Jenner HD, McDonald R, Bertolli O, Calleja A, Hawkins PN, Gillmore JD
Circulation 2019 Jul 2;140(1):16-26. Epub 2019 May 21 doi: 10.1161/CIRCULATIONAHA.118.038169. PMID: 31109193
Kaneko S, Yamagata K
Semin Dial 2018 Nov;31(6):612-618. Epub 2018 Jun 12 doi: 10.1111/sdi.12720. PMID: 29896815
Maurer MS, Hanna M, Grogan M, Dispenzieri A, Witteles R, Drachman B, Judge DP, Lenihan DJ, Gottlieb SS, Shah SJ, Steidley DE, Ventura H, Murali S, Silver MA, Jacoby D, Fedson S, Hummel SL, Kristen AV, Damy T, Planté-Bordeneuve V, Coelho T, Mundayat R, Suhr OB, Waddington Cruz M, Rapezzi C; THAOS Investigators
J Am Coll Cardiol 2016 Jul 12;68(2):161-72. doi: 10.1016/j.jacc.2016.03.596. PMID: 27386769Free PMC Article
Pepys MB
Clin Med (Lond) 2007 Dec;7(6):562-78. doi: 10.7861/clinmedicine.7-6-562. PMID: 18193704Free PMC Article

Clinical prediction guides

Arana-Achaga X, Goena-Vives C, Villanueva-Benito I, Solla-Ruiz I, Rengel Jimenez A, Gaspar TI, Urreta-Barallobre I, Barge-Caballero G, Seijas-Marcos S, Cabrera E, Garcia-Pavía P, Basurte Elorz MT, Ayestarán NM, Sierra LT, Robledo Iñarritu M, Lozano-Bahamonde A, Escolar-Perez V, Gómez-Ramírez C, Alzola E, Andrés RN, Francisco Matias JL, Limeres Freire J, Armengou Arxe A, Negre Busó M, Piqueras-Flores J, Martínez-Del Río J, Onaindia Gandarias JJ, Rodriguez Sanchez I, Querejeta Iraola R
JACC Cardiovasc Imaging 2023 Dec;16(12):1567-1580. Epub 2023 Jun 28 doi: 10.1016/j.jcmg.2023.05.002. PMID: 37389511
Boldrini M, Cappelli F, Chacko L, Restrepo-Cordoba MA, Lopez-Sainz A, Giannoni A, Aimo A, Baggiano A, Martinez-Naharro A, Whelan C, Quarta C, Passino C, Castiglione V, Chubuchnyi V, Spini V, Taddei C, Vergaro G, Petrie A, Ruiz-Guerrero L, Moñivas V, Mingo-Santos S, Mirelis JG, Dominguez F, Gonzalez-Lopez E, Perlini S, Pontone G, Gillmore J, Hawkins PN, Garcia-Pavia P, Emdin M, Fontana M
JACC Cardiovasc Imaging 2020 Apr;13(4):909-920. Epub 2019 Dec 18 doi: 10.1016/j.jcmg.2019.10.011. PMID: 31864973
Dorbala S, Cuddy S, Falk RH
JACC Cardiovasc Imaging 2020 Jun;13(6):1368-1383. Epub 2019 Oct 11 doi: 10.1016/j.jcmg.2019.07.015. PMID: 31607664Free PMC Article
Siddiqi OK, Ruberg FL
Trends Cardiovasc Med 2018 Jan;28(1):10-21. Epub 2017 Jul 13 doi: 10.1016/j.tcm.2017.07.004. PMID: 28739313Free PMC Article
Muchtar E, Blauwet LA, Gertz MA
Circ Res 2017 Sep 15;121(7):819-837. doi: 10.1161/CIRCRESAHA.117.310982. PMID: 28912185

Recent systematic reviews

Antonopoulos AS, Panagiotopoulos I, Kouroutzoglou A, Koutsis G, Toskas P, Lazaros G, Toutouzas K, Tousoulis D, Tsioufis K, Vlachopoulos C
Eur J Heart Fail 2022 Sep;24(9):1677-1696. Epub 2022 Aug 2 doi: 10.1002/ejhf.2589. PMID: 35730461
Aimo A, Merlo M, Porcari A, Georgiopoulos G, Pagura L, Vergaro G, Sinagra G, Emdin M, Rapezzi C
Eur J Heart Fail 2022 Dec;24(12):2342-2351. Epub 2022 May 16 doi: 10.1002/ejhf.2532. PMID: 35509173Free PMC Article
Filippi M, Cecchetti G, Spinelli EG, Vezzulli P, Falini A, Agosta F
JAMA Neurol 2022 Mar 1;79(3):291-304. doi: 10.1001/jamaneurol.2021.5205. PMID: 35099507
Gertz MA, Dispenzieri A
JAMA 2020 Jul 7;324(1):79-89. doi: 10.1001/jama.2020.5493. PMID: 32633805
Weidner T, Illing T, Elsner P
Am J Clin Dermatol 2017 Oct;18(5):629-642. doi: 10.1007/s40257-017-0278-9. PMID: 28342017

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