History and exam

Key diagnostic factors

common

positive family history of epidermolysis bullosa (EB)

The only known risk factor for EB is a positive family history. Most patients with confirmed autosomal dominant forms of EB have a family history of the disease occurring in a parent.

mechanical fragility of the skin

With the rarest of exceptions, patients with EB will have a history of mechanically fragile skin.

If minor traction is applied to normal-appearing skin, a fresh peel (Nikolsky sign) can be induced.

recurrent blisters and erosions

A characteristic feature of all forms of EB. [Figure caption and citation for the preceding image starts]: Extensive blistering on leg, foot, and hand of an infant with EBS, generalized severe (EBS-gen sev)From the collection of J.D. Fine, MD [Citation ends].com.bmj.content.model.Caption@407d32c5

poorly healing wounds

Complex, with multiple potential contributory factors in addition to underlying skin fragility, including: persistent inflammation; microbial colonization and infection; anemia; fibrosis; scarring; and altered protease activity.

onset of cutaneous signs at birth or early infancy

When widespread blistering occurs at this early age, one of the more generalized subtypes of EB is more likely.

Some of the extracutaneous complications may not occur until late childhood or adulthood.

resolution of blistering within the first 1-2 years of life

Characteristic of bullous dermolysis of the newborn, a rare subtype of both types of dystrophic EB.[34]

generalized or localized distribution of skin involvement

Most common pattern of distribution for EB subtypes is either generalized or localized (e.g., palms and soles in localized EB simplex [EBS-loc]).

combination of milia, scarring, and dystrophic nails

Although all of the major EB types and subtypes overlap considerably, this combination of signs is most suggestive of dystrophic EB (DEB).

absence of milia, scarring, and dystrophic nails

Most consistent with EB simplex (EBS).[39]

exuberant granulation tissue

Nearly pathognomonic of severe junctional EB (JEB).[39][Figure caption and citation for the preceding image starts]: Axillary contractions and granulation tissue in a child with junctional EB, generalized severe (JEB-gen sev)From the collection of J.D. Fine, MD [Citation ends].com.bmj.content.model.Caption@1ebe8b52

herpetiform blistering

Polycyclic or arcuate array of blisters or vesicles is pathognomonic of severe EBS.[39]

pseudosyndactyly

Most commonly occurs in recessive dystrophic EB (RDEB), especially severe RDEB. [Figure caption and citation for the preceding image starts]: Hand deformities, extensive scarring of wrists and legs in a child with recessive dystrophic EB, generalized severe (RDEB-gen sev)From the collection of J.D. Fine, MD [Citation ends].com.bmj.content.model.Caption@d5f7347

May also arise in all other RDEB subtypes, dominant dystrophic EB (DDEB), and rarely even intermediate junctional EB.[40][41]

enamel hypoplasia

Seen only in junctional EB (JEB) and occurs in all patients with JEB.[42]

reticulate hyperpigmentation

Characteristic of EB simplex with mottled pigmentation (EBS-MP).

muscular dystrophy

Associated with the subtype EB simplex with muscular dystrophy (EBS-MD). Of importance, this complication may not occur until young adulthood in some patients.

tracheolaryngeal stenosis or stricture

Occurs almost exclusively in junctional EB (JEB), most commonly severe JEB.[43]

severe upper airway disease

Associated with both generalized junctional EB (JEB) and JEB, laryngo-onycho-cutaneous syndrome (JEB-LOC).

uncommon

onset in mid or late childhood

More indicative of localized EB simplex (EBS-loc), or late-onset junctional EB (JEB-lo).

inverse (intertriginous), acral, or centripetal distribution of skin involvement

These distributions are rare, but when they are present they are pathognomonic for specific EB subtypes: recessive dystrophic EB, inversa (RDEB-I); junctional EB, inversa (JEB-I); and RDEB, centripetalis (RDEB-Ce).

These patterns usually only become more apparent after the first year of life.

Distribution types have specific prognostic implications.

severe cardiomyopathy

Reported in several patients with a rare intermediate subtype of EB simplex associated with KLHL24 mutations.[44][45]

Risk factors

strong

Family history of EB

Family history of EB is the only known risk factor for this disease.

Although spontaneous mutations do arise among autosomal dominant (AD) forms of EB, most patients have a positive family history of the disease occurring in a parent.

Of those who do not have a positive family history, particularly those with dystrophic EB, subsequent mutational analyses have demonstrated autosomal recessive (AR) rather than AD transmission in most.

Incomplete penetrance among AD forms of EB is extremely rare.

The risk to an affected parent heterozygous for AD EB of having an affected child is 50% with each pregnancy; all other children will be unaffected.

By definition, each parent of a child with AR EB is a silent carrier for the causative mutation. With each additional pregnancy, the risk of having an affected child, a silent carrier, or a genetically unaffected child is 25%, 50%, or 25%, respectively.

If a patient with an AR form of EB, who is, by definition, homozygous or compound heterozygous for the mutation, chooses to have children and has a genetically unaffected mate, then all of their offspring will be silent carriers for the disease.

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