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Doyne Honeycomb Macular Dystrophy

OMIM ID:

autosomal dominant

Doyne Honeycomb Macular Dystrophy

Alternate Names

DHRD
DHD
Doyne Honeycomb Degeneration of the Retina
Malattia Leventinese
MLVT
radial drusen

Defective Genes

EFEMP1

Clinical Characteristics

Ocular Features

Beginning usually in midlife, the retina has radially localized white, large drusen in the posterior pole.  These may begin as small drusen that later enlarge and become confluent creating a honeycomb pattern.  The disease begins as an accumulation of material between the Bruch membrane and the RPE.  Eventually drusen occupy the entire thickness of the Bruch membrane and are continuous with or internal to the RPE basement membrane.  Vision early is normal and a slow loss of vision occurs sometime after the drusen appear in most individuals.  In some patients geographic atrophy, pigmentary changes, and a subfoveal neovascular net develops with macular scarring, vitreous hemorrhage, and severe reduction of vision.

Systemic Features

No systemic disease is associated.

Genetics

Inheritance

Doyne honeycomb macular disease, or dominant drusen, is the result of mutations in the EFEMP1 gene at 2p16 in the majority of cases.  It is an autosomal dominant disorder. The mutant protein product (a member of the fibulin famiy) is folded abnormally and secreted inefficiently.  It is also resistant to degradation which may lead to receptor damage by limiting access to nutrients from the choriocapillaris.  Some genetic heterogeneity may exist since a few cases seem to be linked to a locus at 6q14.    Some have considered Malattia Leventinese and Doyne honeycomb retinal dystrophy as separate entities but mutations in the same gene seem to be responsible for both conditions suggesting they are clinical variations of the same disorder.

Pedigree

Autosomal dominant

Autosomal dominant disorders require only one mutation for the disease to be expressed.  Since an affected parent has two chromosomes, only one of which has the mutant gene, parents can expect that half (50%) of their children will receive that one and inherit the disease.  It is common for individuals that inherit the mutation, however, to not have evidence of the disease (nonpenetrance).

Image
Sample pedigree of autosomal dominant inheritance

Autosomal dominant inheritance leads to a vertical pattern of transmission

Treatment & Management

The subfoveal net responds to photodynamic therapy.

Selected Resources

Publications

Displaying 1 - 4 of 4

A single EFEMP1 mutation associated with both Malattia Leventinese and Doyne honeycomb retinal dystrophy

PubMedID: 10369267

Association of EFEMP1 with malattia leventinese and age-related macular degeneration: a mini-review

PubMedID: 15512998

Photodynamic therapy with verteporfin in mallatia leventinese1 1The authors have no proprietary interest in any aspect of this article.

PubMedID: 11825812

The R345W mutation in EFEMP1 is pathogenic and causes AMD-like deposits in mice

PubMedID: 17666404