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Patterned macular dystrophy 3(MDPT3)

MedGen UID:
934680
Concept ID:
C4310713
Disease or Syndrome
Synonym: MARTINIQUE CRINKLED RETINAL PIGMENT EPITHELIOPATHY
SNOMED CT: Martinique crinkled retinal pigment epitheliopathy (1187639002); MCRPE - Martinique crinkled retinal pigment epitheliopathy (1187639002)
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): MAPKAPK3 (3p21.2)
 
Monarch Initiative: MONDO:0014920
OMIM®: 617111
Orphanet: ORPHA466718

Definition

Patterned macular dystrophy-3 (MDPT3), also called Martinique crinkled retinal pigment epitheliopathy, appears in the fourth or fifth decade of life and is characterized by a 'dry desert land' pattern of the fundus, involving the posterior pole initially and progressing from the temporal fovea to the periphery of the retina. Polypoid choroidal vasculopathy, choroidal neovascularization, or atrophic fibrous macular scarring can cause reduced visual acuity after age 50. Late-stage MDPT3 consists of a retinitis pigmentosa (RP; see 268000)-like phenotype due to death of retinal pigment epithelium (RPE) and photoreceptor cells. The dry desert land pattern observed on fundus examination corresponds to an irregular thickness of the Bruch membrane and the RPE, with a scalloped elevation ('crinkling') of the RPE observed on optical coherence tomography (OCT). Full-field electroretinography may be normal at preclinical and early stages of the dystrophy, but later cone and rod responses are severely reduced, consistent with progressive photoreceptor cell dysfunction and death at the final state (summary by Meunier et al., 2016). For a general phenotypic description and discussion of genetic heterogeneity of patterned macular dystrophy, see MDPT1 (169150). [from OMIM]

Clinical features

From HPO
Reduced visual acuity
MedGen UID:
65889
Concept ID:
C0234632
Finding
Diminished clarity of vision.
Macular atrophy
MedGen UID:
140841
Concept ID:
C0423421
Finding
Well-demarcated area(s) of partial or complete depigmentation in the macula, reflecting atrophy of the retinal pigment epithelium with associated retinal photoreceptor loss.
Choroidal neovascularization
MedGen UID:
154726
Concept ID:
C0600518
Pathologic Function
Choroidal neovascularization (CNV) is the creation of new blood vessels in the choroid layer of the eye.
Rod-cone dystrophy
MedGen UID:
1632921
Concept ID:
C4551714
Disease or Syndrome
An inherited retinal disease subtype in which the rod photoreceptors appear to be more severely affected than the cone photoreceptors. Typical presentation is with nyctalopia (due to rod dysfunction) followed by loss of mid-peripheral field of vision, which gradually extends and leaves many patients with a small central island of vision due to the preservation of macular cones.

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
Skelly A, Taylor N, Fasser C, Malkowski JP, Goswami P, Downey L
Adv Ther 2022 Oct;39(10):4808-4820. Epub 2022 Aug 22 doi: 10.1007/s12325-022-02248-5. PMID: 35995894Free PMC Article
Cornelis SS, Runhart EH, Bauwens M, Corradi Z, De Baere E, Roosing S, Haer-Wigman L, Dhaenens CM, Vulto-van Silfhout AT, Cremers FPM
Am J Hum Genet 2022 Mar 3;109(3):498-507. Epub 2022 Feb 3 doi: 10.1016/j.ajhg.2022.01.008. PMID: 35120629Free PMC Article

Recent clinical studies

Etiology

Rämö JT, Abner E, van Dijk EHC, Wang X, Brinks J, Nikopensius T, Nõukas M, Marjonen H, Silander K, Jukarainen S, Kiiskinen T, Choi SH, Kajanne R, Mehtonen J, Palta P, Lubitz SA, Kaarniranta K, Sobrin L, Kurki M, Yzer S, Ellinor PT, Esko T, Daly MJ, den Hollander AI, Palotie A, Turunen JA, Boon CJF, Rossin EJ; FinnGen Study; Estonian Biobank Research Team
JAMA Ophthalmol 2023 May 1;141(5):449-457. doi: 10.1001/jamaophthalmol.2023.0706. PMID: 37079300Free PMC Article
Mulpuri L, Sridhar J, Goyal H, Tonk R
Curr Opin Ophthalmol 2023 May 1;34(3):189-194. Epub 2023 Mar 1 doi: 10.1097/ICU.0000000000000943. PMID: 36866844
Bacci T, Essilfie JO, Leong BCS, Freund KB
Graefes Arch Clin Exp Ophthalmol 2021 May;259(5):1123-1134. Epub 2020 Nov 26 doi: 10.1007/s00417-020-05021-y. PMID: 33242167
Waldstein SM, Vogl WD, Bogunovic H, Sadeghipour A, Riedl S, Schmidt-Erfurth U
JAMA Ophthalmol 2020 Jul 1;138(7):740-747. doi: 10.1001/jamaophthalmol.2020.1376. PMID: 32379287Free PMC Article
Annweiler C, Drouet M, Duval GT, Paré PY, Leruez S, Dinomais M, Milea D
Maturitas 2016 Jun;88:101-12. Epub 2016 Apr 2 doi: 10.1016/j.maturitas.2016.04.002. PMID: 27105707

Diagnosis

Gabrielle PH
Acta Ophthalmol 2022 Sep;100 Suppl 269:3-43. doi: 10.1111/aos.15226. PMID: 36117363
Bacci T, Essilfie JO, Leong BCS, Freund KB
Graefes Arch Clin Exp Ophthalmol 2021 May;259(5):1123-1134. Epub 2020 Nov 26 doi: 10.1007/s00417-020-05021-y. PMID: 33242167
Waldstein SM, Vogl WD, Bogunovic H, Sadeghipour A, Riedl S, Schmidt-Erfurth U
JAMA Ophthalmol 2020 Jul 1;138(7):740-747. doi: 10.1001/jamaophthalmol.2020.1376. PMID: 32379287Free PMC Article
Fleckenstein M, Mitchell P, Freund KB, Sadda S, Holz FG, Brittain C, Henry EC, Ferrara D
Ophthalmology 2018 Mar;125(3):369-390. Epub 2017 Oct 27 doi: 10.1016/j.ophtha.2017.08.038. PMID: 29110945
Stojanov O
Vojnosanit Pregl 2016 Sep;73(9):881-4. doi: 10.2298/VSP150321140S. PMID: 29320624

Therapy

Waldstein SM, Vogl WD, Bogunovic H, Sadeghipour A, Riedl S, Schmidt-Erfurth U
JAMA Ophthalmol 2020 Jul 1;138(7):740-747. doi: 10.1001/jamaophthalmol.2020.1376. PMID: 32379287Free PMC Article
van Nispen RM, Virgili G, Hoeben M, Langelaan M, Klevering J, Keunen JE, van Rens GH
Cochrane Database Syst Rev 2020 Jan 27;1(1):CD006543. doi: 10.1002/14651858.CD006543.pub2. PMID: 31985055Free PMC Article
Fleckenstein M, Mitchell P, Freund KB, Sadda S, Holz FG, Brittain C, Henry EC, Ferrara D
Ophthalmology 2018 Mar;125(3):369-390. Epub 2017 Oct 27 doi: 10.1016/j.ophtha.2017.08.038. PMID: 29110945
Wong IY, Koo SC, Chan CW
Int Ophthalmol 2011 Feb;31(1):73-82. Epub 2010 Sep 23 doi: 10.1007/s10792-010-9397-5. PMID: 20862519Free PMC Article
Bernhard D, Moser C, Backovic A, Wick G
Exp Gerontol 2007 Mar;42(3):160-5. Epub 2006 Nov 3 doi: 10.1016/j.exger.2006.09.016. PMID: 17084574

Prognosis

Iovino C, Ramtohul P, Au A, Romero-Morales V, Sadda S, Freund KB, Sarraf D
Surv Ophthalmol 2023 May-Jun;68(3):361-379. Epub 2023 Jan 30 doi: 10.1016/j.survophthal.2023.01.009. PMID: 36720370
Barth T, Helbig H
Klin Monbl Augenheilkd 2021 Sep;238(9):962-970. Epub 2021 Aug 20 doi: 10.1055/a-1402-7592. PMID: 34416787
Ma DJ, Lee HS, Kim K, Choi S, Jang I, Cho SH, Yoon CK, Lee EK, Yu HG
BMC Med Genomics 2021 Mar 10;14(1):74. doi: 10.1186/s12920-021-00874-6. PMID: 33691693Free PMC Article
Waldstein SM, Vogl WD, Bogunovic H, Sadeghipour A, Riedl S, Schmidt-Erfurth U
JAMA Ophthalmol 2020 Jul 1;138(7):740-747. doi: 10.1001/jamaophthalmol.2020.1376. PMID: 32379287Free PMC Article
Fleckenstein M, Mitchell P, Freund KB, Sadda S, Holz FG, Brittain C, Henry EC, Ferrara D
Ophthalmology 2018 Mar;125(3):369-390. Epub 2017 Oct 27 doi: 10.1016/j.ophtha.2017.08.038. PMID: 29110945

Clinical prediction guides

Cornelis SS, Runhart EH, Bauwens M, Corradi Z, De Baere E, Roosing S, Haer-Wigman L, Dhaenens CM, Vulto-van Silfhout AT, Cremers FPM
Am J Hum Genet 2022 Mar 3;109(3):498-507. Epub 2022 Feb 3 doi: 10.1016/j.ajhg.2022.01.008. PMID: 35120629Free PMC Article
Ma DJ, Lee HS, Kim K, Choi S, Jang I, Cho SH, Yoon CK, Lee EK, Yu HG
BMC Med Genomics 2021 Mar 10;14(1):74. doi: 10.1186/s12920-021-00874-6. PMID: 33691693Free PMC Article
Waldstein SM, Vogl WD, Bogunovic H, Sadeghipour A, Riedl S, Schmidt-Erfurth U
JAMA Ophthalmol 2020 Jul 1;138(7):740-747. doi: 10.1001/jamaophthalmol.2020.1376. PMID: 32379287Free PMC Article
Fleckenstein M, Mitchell P, Freund KB, Sadda S, Holz FG, Brittain C, Henry EC, Ferrara D
Ophthalmology 2018 Mar;125(3):369-390. Epub 2017 Oct 27 doi: 10.1016/j.ophtha.2017.08.038. PMID: 29110945
Gulshan V, Peng L, Coram M, Stumpe MC, Wu D, Narayanaswamy A, Venugopalan S, Widner K, Madams T, Cuadros J, Kim R, Raman R, Nelson PC, Mega JL, Webster DR
JAMA 2016 Dec 13;316(22):2402-2410. doi: 10.1001/jama.2016.17216. PMID: 27898976

Recent systematic reviews

Ma X, Wang Y, Wang N, Zhang R
J Neurol 2022 Mar;269(3):1272-1281. Epub 2021 Jul 10 doi: 10.1007/s00415-021-10703-6. PMID: 34245345
van Nispen RM, Virgili G, Hoeben M, Langelaan M, Klevering J, Keunen JE, van Rens GH
Cochrane Database Syst Rev 2020 Jan 27;1(1):CD006543. doi: 10.1002/14651858.CD006543.pub2. PMID: 31985055Free PMC Article
Zhu W, Wu Y, Meng YF, Xing Q, Tao JJ, Lu J
Nutrients 2016 Nov 22;8(11) doi: 10.3390/nu8110743. PMID: 27879656Free PMC Article
Annweiler C, Drouet M, Duval GT, Paré PY, Leruez S, Dinomais M, Milea D
Maturitas 2016 Jun;88:101-12. Epub 2016 Apr 2 doi: 10.1016/j.maturitas.2016.04.002. PMID: 27105707
Arbesman M, Mosley LJ
Am J Occup Ther 2012 May-Jun;66(3):277-83. doi: 10.5014/ajot.2012.003327. PMID: 22549592

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