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North Carolina macular dystrophy(MCDR1)

MedGen UID:
147590
Concept ID:
C0730294
Disease or Syndrome
Synonyms: Central areolar pigment epithelial dystrophy; Foveal dystrophy progressive; Macular dystrophy retinal 1 North Carolina type; MCDR1; Retinal pigment epithelial dystrophy central
SNOMED CT: North Carolina macular dystrophy (312925009)
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): LOC111365204 (6q16.2)
 
Monarch Initiative: MONDO:0007630
OMIM®: 136550
Orphanet: ORPHA75327

Definition

North Carolina macular dystrophy (NCMD, MCDR1) is a congenital autosomal dominant trait that appears to be completely penetrant. It is generally nonprogressive. The ophthalmoscopic findings are highly variable and are always much more dramatic than one would predict from the relatively good visual acuity level, which ranges from 20/20 to 20/400 (median, 20/60). Patients may have only a few drusen in the central macular region (grade I), confluent drusen confined to the central macular region (grade II), or a severe macular coloboma/staphyloma (grade III) involving 3 to 4 disc areas of the central macular region. Choroidal neovascular membranes develop in some patients. Color vision is normal. Electrophysiologic studies are also normal (summary by Small, 1998). Genetic Heterogeneity of Retinal Macular Dystrophy MCDR2 (608051) is caused by mutation in the PROM1 gene (604365) on chromosome 4p15. MCDR3 (608850) is caused by a duplication on chromosome 5p15. MCDR4 (619977) is caused by mutation in the CLEC3B gene (187520) on chromosome 3p21. MCDR5 (see 613660) is caused by mutation in the CDHR1 gene (609502) on chromosome 10q23. See MAPPING for possible additional loci for MCDR. [from OMIM]

Clinical features

From HPO
Central scotoma
MedGen UID:
57750
Concept ID:
C0152191
Finding
An area of depressed vision located at the point of fixation and that interferes with central vision.
Reduced visual acuity
MedGen UID:
65889
Concept ID:
C0234632
Finding
Diminished clarity of vision.
Macular dystrophy
MedGen UID:
196451
Concept ID:
C0730292
Disease or Syndrome
Macular dystrophy is a nonspecific term for premature retinal cell aging and cell death, generally confied to the macula in which no clear extrinsic cause is evident.
Dyschromatopsia
MedGen UID:
163559
Concept ID:
C0858618
Disease or Syndrome
A form of colorblindness in which only two of the three fundamental colors can be distinguished due to a lack of one of the retinal cone pigments.
Drusen
MedGen UID:
488956
Concept ID:
C1260959
Finding
Drusen (singular, 'druse') are tiny yellow or white accumulations of extracellular material (lipofuscin) that build up in Bruch's membrane of the eye.
Peripheral retinal atrophy
MedGen UID:
765930
Concept ID:
C3553016
Finding
Abnormality of macular pigmentation
MedGen UID:
892991
Concept ID:
C4024756
Anatomical Abnormality
Abnormality of macular or foveal pigmentation.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVNorth Carolina macular dystrophy
Follow this link to review classifications for North Carolina macular dystrophy in Orphanet.

Professional guidelines

PubMed

Kaiser PK, Karpecki PM, Regillo CD, Baumal C, Ryan EH, Ip MS, Modi Y, Yeu E, Nijm L, Farid M, Rebenitsch RL, Kim T, Shechtman DL, Nichols K, Schweitzer J, Dunbar MT, Rafieetary MR, Donnenfeld ED
BMJ Open Ophthalmol 2023 Oct;8(1) doi: 10.1136/bmjophth-2023-001395. PMID: 37857560Free PMC Article
Prince J, Kumar D, Ghosh A, Arevalo JF, Zhang AY
Curr Diab Rep 2023 Jun;23(6):119-125. Epub 2023 Apr 12 doi: 10.1007/s11892-023-01505-3. PMID: 37043090
Browning DJ, Stewart MW, Lee C
Indian J Ophthalmol 2018 Dec;66(12):1736-1750. doi: 10.4103/ijo.IJO_1240_18. PMID: 30451174Free PMC Article

Recent clinical studies

Etiology

Small KW, Wiggins R, Udar N, Silva-Garcia R, Avetisjan J, Vincent A, Shaya FS
Ophthalmol Retina 2022 Jun;6(6):512-519. Epub 2022 Feb 11 doi: 10.1016/j.oret.2022.02.003. PMID: 35151913
Birtel J, Gliem M, Herrmann P, Neuhaus C, Holz FG, MacLaren RE, Scholl HPN, Charbel Issa P
Br J Ophthalmol 2022 Sep;106(9):1269-1273. Epub 2021 Mar 30 doi: 10.1136/bjophthalmol-2021-318815. PMID: 33785507
Audere M, Rutka K, Inaskina I, Peculis R, Sepetiene S, Valeina S, Lāce B
Medicina (Kaunas) 2016;52(3):180-6. Epub 2016 Apr 19 doi: 10.1016/j.medici.2016.04.001. PMID: 27496188
Voo I, Glasgow BJ, Flannery J, Udar N, Small KW
Am J Ophthalmol 2001 Dec;132(6):933-5. PMID: 11730667
Small KW, Weber J, Roses A, Pericak-Vance P
Ophthalmic Paediatr Genet 1993 Dec;14(4):143-50. doi: 10.3109/13816819309042913. PMID: 8015785

Diagnosis

Nekolova J, Stepanov A, Kousal B, Stredova M, Jiraskova N
Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub 2022 Dec;166(4):418-427. Epub 2021 Jun 22 doi: 10.5507/bp.2021.037. PMID: 34158671
Birtel J, Gliem M, Herrmann P, Neuhaus C, Holz FG, MacLaren RE, Scholl HPN, Charbel Issa P
Br J Ophthalmol 2022 Sep;106(9):1269-1273. Epub 2021 Mar 30 doi: 10.1136/bjophthalmol-2021-318815. PMID: 33785507
Green DJ, Lenassi E, Manning CS, McGaughey D, Sharma V, Black GC, Ellingford JM, Sergouniotis PI
Invest Ophthalmol Vis Sci 2021 Jun 1;62(7):16. doi: 10.1167/iovs.62.7.16. PMID: 34125159Free PMC Article
Pichi F, Abboud EB, Ghazi NG, Khan AO
Acta Ophthalmol 2018 Aug;96(5):e549-e561. Epub 2017 Nov 2 doi: 10.1111/aos.13602. PMID: 29098804
Bowne SJ, Sullivan LS, Wheaton DK, Locke KG, Jones KD, Koboldt DC, Fulton RS, Wilson RK, Blanton SH, Birch DG, Daiger SP
Mol Vis 2016;22:1239-1247. Epub 2016 Oct 17 PMID: 27777503Free PMC Article

Therapy

Bakall B, Bryan JS 3rd, Stone EM, Small KW
Retin Cases Brief Rep 2021 Sep 1;15(5):509-513. doi: 10.1097/ICB.0000000000000838. PMID: 30383557
Stepanov A, Středová M, Dusová J, Jirásková N, Studnička J
Cesk Slov Oftalmol 2019 Summer;75(3):138-144. doi: 10.31348/2019/3/4. PMID: 31779462

Prognosis

Small KW, Wiggins R, Udar N, Silva-Garcia R, Avetisjan J, Vincent A, Shaya FS
Ophthalmol Retina 2022 Jun;6(6):512-519. Epub 2022 Feb 11 doi: 10.1016/j.oret.2022.02.003. PMID: 35151913
Nekolova J, Stepanov A, Kousal B, Stredova M, Jiraskova N
Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub 2022 Dec;166(4):418-427. Epub 2021 Jun 22 doi: 10.5507/bp.2021.037. PMID: 34158671
Birtel J, Gliem M, Herrmann P, Neuhaus C, Holz FG, MacLaren RE, Scholl HPN, Charbel Issa P
Br J Ophthalmol 2022 Sep;106(9):1269-1273. Epub 2021 Mar 30 doi: 10.1136/bjophthalmol-2021-318815. PMID: 33785507
Green DJ, Lenassi E, Manning CS, McGaughey D, Sharma V, Black GC, Ellingford JM, Sergouniotis PI
Invest Ophthalmol Vis Sci 2021 Jun 1;62(7):16. doi: 10.1167/iovs.62.7.16. PMID: 34125159Free PMC Article
Small KW
Ophthalmology 1989 Dec;96(12):1747-54. doi: 10.1016/s0161-6420(89)32655-8. PMID: 2622620

Clinical prediction guides

Green DJ, Lenassi E, Manning CS, McGaughey D, Sharma V, Black GC, Ellingford JM, Sergouniotis PI
Invest Ophthalmol Vis Sci 2021 Jun 1;62(7):16. doi: 10.1167/iovs.62.7.16. PMID: 34125159Free PMC Article
Small KW, DeLuca AP, Whitmore SS, Rosenberg T, Silva-Garcia R, Udar N, Puech B, Garcia CA, Rice TA, Fishman GA, Héon E, Folk JC, Streb LM, Haas CM, Wiley LA, Scheetz TE, Fingert JH, Mullins RF, Tucker BA, Stone EM
Ophthalmology 2016 Jan;123(1):9-18. Epub 2015 Oct 24 doi: 10.1016/j.ophtha.2015.10.006. PMID: 26507665Free PMC Article
Voo I, Glasgow BJ, Flannery J, Udar N, Small KW
Am J Ophthalmol 2001 Dec;132(6):933-5. PMID: 11730667
Small KW, Weber J, Roses A, Pericak-Vance P
Ophthalmic Paediatr Genet 1993 Dec;14(4):143-50. doi: 10.3109/13816819309042913. PMID: 8015785
Small KW
Ophthalmology 1989 Dec;96(12):1747-54. doi: 10.1016/s0161-6420(89)32655-8. PMID: 2622620

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