Dystrophic Epidermolysis Bullosa is a subtype of Epidermolysis Bullosa.
The group of Epidermolysis Bullosa diseases includes several rare
genetic disorders characterized by skin fragility and blistering and
mutations in several genes, such as KRT5, KRT14, PLEC, TGM5, COL17A1,
ITGB4, COL7A1. Recessive Dystrophic Epidermolysis Bullosa is the most
severe form, and it is a very rare disease, with an incidence of
approximately 1/51,000 live births. We report a case of novel variant
c.7795-1G>A of COL7A1 Gene in a 12-month-old female child
with Recessive Dystrophic Epidermolysis Bullosa, reporting clinical
characteristics and signs. BACKGROUND:We report a case of a novel c.7795-1G>A variant of the
COL7A1 gene in a 12-month-old female child with recessive dystrophic
epidermolysis bullosa (RDEB) syndrome. Dystrophic Epidermolysis Bullosa
(DEB)1 is a subtype of Epidermolysis Bullosa
(EB)2. EB refers to a group of rare genetic skin
diseases characterized by skin fragility and blistering.
According to data from the National Epidermolysis Bullosa Registry, the
estimated prevalence of epidermolysis bullosa in the United States is
approximately 11.1 cases per one million live births. The incidence is
reported to be approximately 1 in every 51,000 live
births3. There are different classifications and
subtypes of epidermolysis bullosa, each with different clinical features
and mutations. The main genes associated with EB subtypes are KRT5,
KRT14, PLEC, TGM5, LAMA3, LAMB3, LAMC2, COL17A1, ITGB4, COL7A1 and
FERMT14. The patients who suffer from it have
extremely fragile skin that blisters and forms open sores in response to
minor trauma or friction. The severity of symptoms can vary widely,
ranging from mild blisters to life-threatening
complications5. Recessive DEB (RDEB) is the most
severe form, and it is a very rare disease6. The
incidence of Recessive Dystrophic Epidermolysis Bullosa (RDEB) is
reported ranging from 0.2 to 6.65 per million births7.
RDEB follows an autosomal recessive inheritance pattern, which means
that an affected individual inherits two copies of the mutated gene, one
from each parent who usually carries the condition. Both males and
females can be affected. RDEB is caused by mutations in the COL7A1 gene,
which codes for type VII collagen. Mutations in the COL7A1 gene result
in a deficiency or absence of functional type VII collagen, which leads
to the characteristic blistering and fragile skin in RDEB. The main
clinical feature of RDEB is the formation of blisters and erosions on
the skin and mucous membranes. These blisters can occur anywhere on the
body and in addition to the skin, the oral cavity and gastrointestinal
tract can be involved. Blisters are often painful and slow to heal,
causing scarring and the development of contractures, which can limit
joint mobility. Other complications may include dental abnormalities,
nail dystrophy and corneal erosions. Diagnosis of EBD usually involves a
combination of clinical examination, evaluation of family history, skin
biopsy and genetic testing. The clinical evaluation includes a thorough
examination of the skin and mucous membranes to identify characteristic
features such as blisters, scars, and nail dystrophy. A family history
of blistering disorders may also suggest a potential genetic cause.CASE REPORT:We report a case of a 12-month-old female child referred to our
Pediatric Dermatology Regional Center for the recurrent onset of
blisters and large boils localized mainly on the upper and lower limbs
(Figure 1 and Figure 2). No similar cases in the family were reported in
her medical history. Suspecting a genetic bullous disease, such as EB, a
skin biopsy and genetic testing were performed. Skin biopsy and genetic
testing reported the diagnosis of RDEB.
Genetic analysis revealed a novel mutation, the c.7795-1G>A
variant of the COL7A1 gene, to date, the c.7795-1G>A
variant was never described before in patients with RDEB
syndrome6 and is absent in the general population
(gnomAD). The patient is currently undergoing regular dermatological
follow-up where she is periodically re-evaluated and medicated if
necessary. The chronic nature of the condition, pain and physical
limitations can lead to emotional distress, social isolation, and
depression.CONCLUSION:The management of DEB focuses on symptom reduction, wound care, pain
management, nutritional support, and infection prevention. A
multidisciplinary approach involving dermatologists, geneticists, wound
care specialists, nutritionists and other health professionals is
essential. Specialized dressings and creams are used to protect fragile
skin and promote healing. nutritional support ensures adequate nutrition
and measures are taken to prevent infection. At present, there is no
cure for EBD, but future treatment prospects for DEB are promising,
including gene therapy or protein replacement
therapy8,9.
In all patients with suspected EB, clinicians should recommend genetic
examination to achieve the correct diagnosis and to identify the
specific syndrome among the heterogeneous group of EB.Figure
1Figure 2REFERENCES: