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Dilated cardiomyopathy 1A(CMD1A)

MedGen UID:
258500
Concept ID:
C1449563
Disease or Syndrome
Synonyms: CARDIOMYOPATHY, CONGESTIVE; CARDIOMYOPATHY, DILATED, WITH CONDUCTION DEFECT 1; Cardiomyopathy, Familial Idiopathic; CMD1A; Dilated cardiomyopathy with conduction defect; Dilated Cardiomyopathy with Quadriceps Myopathy; Familial dilated cardiomyopathy with conduction defect due to LMNA mutation; Idiopathic dilated cardiomyopathy
SNOMED CT: Primary idiopathic dilated cardiomyopathy (53043001)
Modes of inheritance:
Autosomal dominant inheritance
MedGen UID:
141047
Concept ID:
C0443147
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 heterozygotes. In the context of medical genetics, an autosomal dominant disorder is caused when a single copy of the mutant allele is present. Males and females are affected equally, and can both transmit the disorder with a risk of 50% for each child of inheriting the mutant allele.
 
Gene (location): LMNA (1q22)
 
Monarch Initiative: MONDO:0007269
OMIM®: 115200
Orphanet: ORPHA300751

Definition

LMNA-related dilated cardiomyopathy (DCM) is characterized by left ventricular enlargement and/or reduced systolic function preceded (sometimes by many years) by or accompanied by conduction system disease and/or arrhythmias. LMNA-related DCM usually presents in early to mid-adulthood with symptomatic conduction system disease or arrhythmias, or with symptomatic DCM including heart failure or embolus from a left ventricular mural thrombus. Sudden cardiac death can occur, and in some instances is the presenting manifestation; sudden cardiac death may occur with minimal or no systolic dysfunction. [from GeneReviews]

Additional description

From OMIM
Dilated cardiomyopathy (CMD) is characterized by cardiac dilatation and reduced systolic function. CMD is the most frequent form of cardiomyopathy and accounts for more than half of all cardiac transplantations performed in patients between 1 and 10 years of age. A heritable pattern is present in 20 to 30% of cases. Most familial CMD pedigrees show an autosomal dominant pattern of inheritance, usually presenting in the second or third decade of life (summary by Levitas et al., 2010). Olson and Keating (1996) noted that the causes of CMD include myocarditis, coronary artery disease, systemic diseases, and myocardial toxins; idiopathic dilated cardiomyopathy in which these causes are excluded represents approximately one-half of all cases. Idiopathic dilated cardiomyopathy occurs with a prevalence of about 36.5 per 100,000; it accounts for more than 10,000 deaths in the U.S. annually and is the primary indication for cardiac transplantation. Seidman and Seidman (2001) reviewed the clinical and genetic heterogeneity of dilated cardiomyopathy. Dilated cardiomyoopathy-1A (CMD1A) is an autosomal dominant disorder with typical onset between the ages of 30 and 40. In contrast to most other forms of familial CMD, sudden cardiac death may be the first manifestaton even in the absence of systolic dysfunction, owing to malignant arrhythmias such as ventricular tachycardia and fibrillation (summary by Lee et al., 2019). Genetic Heterogeneity of Dilated Cardiomyopathy Mutations in many other genes have been found to cause different forms of autosomal dominant dilated cardiomyopathy. These include CMD1C (601493), with or without left ventricular noncompaction, caused by mutation in the LDB3 gene (605906) on 10q23; CMD1D (601494), caused by mutation in the TNNT2 gene (191045) on 1q32; CMD1E (601154), caused by mutation in the SCN5A gene (600163) on 3p22; CMD1G (604145), caused by mutation in the TTN gene (188840) on 2q31; CMD1I (604765), caused by mutation in the DES gene (125660) on 2q35; CMD1J (605362), caused by mutation in the EYA4 gene (603550) on 6q23; CMD1L (606685), caused by mutation in the SGCD gene (601411) on 5q33; CMD1M (607482), caused by mutation in the CSRP3 gene (600824) on 11p15; CMD1O (608569), caused by mutation in the ABCC9 gene (601439) on 12p12; CMD1P (609909), caused by mutation in the PLN gene (172405) on 6q22; CMD1R (613424), caused by mutation in the ACTC gene (102540) on 15q14; CMD1S (613426), caused by mutation in the MYH7 gene (160760) on 14q12; CMD1U (613694), caused by mutation in the PSEN1 gene (104311) on 14q24; CMD1V (613697), caused by mutation in the PSEN2 gene (600759) on 1q42; CMD1W (611407), caused by mutation in the gene encoding metavinculin (VCL; 193065) on 10q22; CMD1X (611615), caused by mutation in the gene encoding fukutin (FKTN; 607440) on 9q31; CMD1Y (611878), caused by mutation in the TPM1 gene (191010) on 15q22; CMD1Z (611879), caused by mutation in the TNNC1 gene (191040) on 3p21; CMD1AA (612158), caused by mutation in the ACTN2 gene (102573) on 1q43; CMD1BB (612877), caused by mutation in the DSG2 gene (125671) on 18q12; CMD1CC (613122), caused by mutation in the NEXN gene (613121) on 1p31; CMD1DD (613172), caused by mutation in the RBM20 gene (613171) on 10q25; CMD1EE (613252), caused by mutation in the MYH6 gene (160710) on 14q12; CMD1FF (613286), caused by mutation in the TNNI3 gene (191044) on 19q13; CMD1GG (613642), caused by mutation in the SDHA gene (600857) on 5p15; CMD1HH (613881), caused by mutation in the BAG3 gene (603883) on 10q26; CMD1II (615184), caused by mutation in the CRYAB gene (123590) on 6q21; CMD1JJ (615235), caused by mutation in the LAMA4 gene (600133) on 6q21; CMD1KK (615248), caused by mutation in the MYPN gene (608517) on 10q21; CMD1LL (615373), caused by mutation in the PRDM16 gene (605557) on 1p36; CMD1MM (see 615396), caused by mutation in the MYBPC3 gene (600958) on 11p11; CMD1NN (615916), caused by mutation in the RAF1 gene (164760) on 3p25; CMD1OO (620247), caused by mutation in the VEZF1 gene (606747) on chromosome 17q22; and CMD1PP (see 617047), caused by mutation in the FLNC gene (102565) on chromosome 7q32. Several additional loci for autosomal dominant familial dilated cardiomyopathy have been mapped: CMD1B (600884) on 9q13; CMD1H (604288) on 2q14-q22; CMD1K (605582) on 6q12-q16; and CMD1Q (609915) on 7q22.3-q31.1. Autosomal recessive CMD includes CMD2A (611880), caused by mutation in the TNNI3 gene (191044) on 19q13; CMD2B (614672), caused by mutation in the GATAD1 gene (614518) on 7q21; CMD2C (618189), caused by mutation in the PPCS gene (609853) on 1p34; CMD2D (619371), caused by mutation in the RPL3L gene (617416) on 16p13; CMD2E (619492), caused by mutation in the JPH2 gene (605267) on chromosome 20q13; CMD2F (619747), caused by mutation in the BAG5 gene (603885) on chromosome 14q32; CMD2G (619897), caused by mutation in the LMOD2 gene (608006) on chromosome 7q31; CMD2H (620203), caused by mutation in the GET3 gene (601913) on chromosome 19p13; CMD2I (620462), caused by mutation in the CAP2 gene (618385) on chromosome 6p22; CMD2J (620635), caused by mutation in the FLII gene (600362) on chromosome 17p11; and CMD2K (620894), caused by mutation in the GCOM1 gene (see 614071) on chromosome 15q21. An X-linked form of CMD (CMD3B; 302045) is caused by mutation in the DMD gene (300377). An X-linked form previously designated CMD3A was found to be the same as Barth syndrome (302060). Reclassified Forms of CMD The symbol CMD1F was formerly used for a disorder later found to be the same as desmin-related myopathy (601419). The symbol CMD1N (see 607487) was previously used for a form of dilated cardiomyopathy reported to be caused by a mutation in the TCAP gene (604488.0003); this variant has been reclassified as a variant of unknown significance. The symbol CMD1T was previously used for a form of dilated cardiomyopathy reported to be caused by a mutation in the TMPO gene (188380.0001); this variant has been reclassified as a variant of unknown significance.  http://www.omim.org/entry/115200

Clinical features

From HPO
Sudden cardiac death
MedGen UID:
38841
Concept ID:
C0085298
Pathologic Function
The heart suddenly and unexpectedly stops beating resulting in death within a short time period (generally within 1 h of symptom onset).
Atrial fibrillation
MedGen UID:
445
Concept ID:
C0004238
Finding
An atrial arrhythmia characterized by disorganized atrial activity without discrete P waves on the surface EKG, but instead by an undulating baseline or more sharply circumscribed atrial deflections of varying amplitude an frequency ranging from 350 to 600 per minute.
Atrial flutter
MedGen UID:
13955
Concept ID:
C0004239
Pathologic Function
A type of atrial arrhythmia characterized by atrial rates of between 240 and 400 beats per minute and some degree of atrioventricular node conduction block. Typically, the ventricular rate is half the atrial rate. In the EKG; atrial flutter waves are observed as sawtooth-like atrial activity. Pathophysiologically, atrial flutter is a form of atrial reentry in which there is a premature electrical impulse creates a self-propagating circuit.
Primary dilated cardiomyopathy
MedGen UID:
2880
Concept ID:
C0007193
Disease or Syndrome
Familial dilated cardiomyopathy is a genetic form of heart disease. It occurs when heart (cardiac) muscle becomes thin and weakened in at least one chamber of the heart, causing the open area of the chamber to become enlarged (dilated). As a result, the heart is unable to pump blood as efficiently as usual. To compensate, the heart attempts to increase the amount of blood being pumped through the heart, leading to further thinning and weakening of the cardiac muscle. Over time, this condition results in heart failure.\n\nIt usually takes many years for symptoms of familial dilated cardiomyopathy to cause health problems. They typically begin in mid-adulthood, but can occur at any time from infancy to late adulthood. Signs and symptoms of familial dilated cardiomyopathy can include an irregular heartbeat (arrhythmia), shortness of breath (dyspnea), extreme tiredness (fatigue), fainting episodes (syncope), and swelling of the legs and feet. In some cases, the first sign of the disorder is sudden cardiac death. The severity of the condition varies among affected individuals, even in members of the same family.
Congestive heart failure
MedGen UID:
9169
Concept ID:
C0018802
Disease or Syndrome
The presence of an abnormality of cardiac function that is responsible for the failure of the heart to pump blood at a rate that is commensurate with the needs of the tissues or a state in which abnormally elevated filling pressures are required for the heart to do so. Heart failure is frequently related to a defect in myocardial contraction.
Pericardial effusion
MedGen UID:
10653
Concept ID:
C0031039
Disease or Syndrome
Accumulation of fluid within the pericardium.
Sinus bradycardia
MedGen UID:
39316
Concept ID:
C0085610
Pathologic Function
Bradycardia related to a mean resting sinus rate of less than 50 beats per minute.
Ventricular arrhythmia
MedGen UID:
39082
Concept ID:
C0085612
Disease or Syndrome
A disorder characterized by an electrocardiographic finding of an atypical cardiac rhythm resulting from a pathologic process in the cardiac ventricles.
First degree atrioventricular block
MedGen UID:
43215
Concept ID:
C0085614
Disease or Syndrome
Delay of conduction through the atrioventricular node, which is manifested as prolongation of the PR interval in the electrocardiogram (EKG). All atrial impulses reach the ventricles.
Third degree atrioventricular block
MedGen UID:
56230
Concept ID:
C0151517
Disease or Syndrome
Third-degree atrioventricular (AV) block (also referred to as complete heart block) is the complete dissociation of the atria and the ventricles. Third-degree AV block exists when more P waves than QRS complexes exist and no relationship (no conduction) exists between them.
Second degree atrioventricular block
MedGen UID:
75546
Concept ID:
C0264906
Disease or Syndrome
An intermittent atrioventricular block with failure of some atrial impulses to conduct to the ventricles, i.e., some but not all atrial impulses are conducted through the atrioventricular node and trigger ventricular contraction.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
Follow this link to review classifications for Dilated cardiomyopathy 1A in Orphanet.

Professional guidelines

PubMed

Heymans S, Lakdawala NK, Tschöpe C, Klingel K
Lancet 2023 Sep 16;402(10406):998-1011. doi: 10.1016/S0140-6736(23)01241-2. PMID: 37716772
Arbelo E, Protonotarios A, Gimeno JR, Arbustini E, Barriales-Villa R, Basso C, Bezzina CR, Biagini E, Blom NA, de Boer RA, De Winter T, Elliott PM, Flather M, Garcia-Pavia P, Haugaa KH, Ingles J, Jurcut RO, Klaassen S, Limongelli G, Loeys B, Mogensen J, Olivotto I, Pantazis A, Sharma S, Van Tintelen JP, Ware JS, Kaski JP; ESC Scientific Document Group
Eur Heart J 2023 Oct 1;44(37):3503-3626. doi: 10.1093/eurheartj/ehad194. PMID: 37622657
Lampejo T, Durkin SM, Bhatt N, Guttmann O
Clin Med (Lond) 2021 Sep;21(5):e505-e510. doi: 10.7861/clinmed.2021-0121. PMID: 34507935Free PMC Article

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