Long-term Dupilumab promotes itch relief across DEB subtypes
Real-world study also found benefits in wound healing, quality of life
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Long-term treatment with dupilumab, an injection therapy approved for certain inflammatory conditions, reduces disease severity, promotes wound healing, and eases itch among people with different subtypes of dystrophic epidermolysis bullosa (DEB), including severe DEB.
In their real-world study, researchers also reported that DEB patients treated with dupilumab experienced sustained reductions of skin symptoms and better quality of life, with no serious safety concerns.
“This study suggests that dupilumab is a promising candidate in treatment of all subtypes of DEB due to its reputation for fast alleviating [itching] and long-term safety,” the researchers wrote.
The study, “The Clinical Efficacy and Safety of Dupilumab Monotherapy in Dystrophic Epidermolysis Bullosa: A Retrospective Real-World Study,” was published in Dermatologic Therapy.
Dupilumab approved for eczema, asthma
DEB is a type of epidermolysis bullosa (EB) caused by mutations in the COL7A1 gene, which disrupts the production of collagen type VII, a protein essential for skin strength and stability. A deficiency in collagen VII leads to fragile skin, blistering, chronic wounds, itching, and scarring, with severity varying widely among patients.
Dupilumab (marketed as Dupixent), given as a subcutaneous (under the skin) injection, works by blocking signaling mediated by interleukin-4 and interleukin-13, two molecules involved in immune and allergic responses. It’s currently approved for conditions such as eczema and asthma.
A small study found that dupilumab eased itching, reduced blisters, and improved quality of life in people with different types of EB, including DEB. Another recent study found that the medication helped ease itch and reduce disease severity across different subtypes of DEB.
Building on these findings, researchers in China assessed the long-term effectiveness and safety of dupilumab in treating various subtypes of DEB, including severe DEB.
The team reviewed medical records from 12 patients, ages 3 to 54 years, with five distinct DEB subtypes: intermediate-dominant DEB (DDEB), severe recessive DEB (RDEB), intermediate RDEB, DDEB pruriginosa, and RDEB pruriginosa. Dominant DEB results from one disease-causing mutation, while recessive DEB requires two inherited mutations, one from each biological parent.
Disease severity scores dropped steadily over course of a year
Blood tests showed normal immune eosinophil cell levels but elevated immunoglobulin E (IgE) in seven patients. Elevated IgE, a type of antibody often linked to allergic inflammation, was associated with more severe clinical symptoms.
Disease severity was measured using the Birmingham Epidermolysis Bullosa Severity (BEBS) score, which ranges from zero to 100, with higher scores indicating greater disease severity. The average BEBS score before treatment (baseline) was 23.9. All but one patient completed at least one year of therapy.
After dupilumab treatment began, mean BEBS scores fell to 16.4 at 16 weeks (31.5% reduction), 12.3 at 24 weeks (48.3% reduction), and 10.4 after one year (56.4% reduction).
Two patients with severe DEB showed robust responses, with BEBS scores dropping by up to 64.1%. The researchers reported improved wound healing in these patients, including in chronic open wounds that had persisted for more than six months. Patients with DEB pruriginosa also showed marked improvement, with BEBS reductions of up to 78.1%.
Pruritus (itching) was measured using a numerical rating scale (NRS) from 0 to 10. Mean scores fell from 7.25 at baseline to 3.83 after four weeks and to 1.82 after one year — a 74.9% reduction. One patient discontinued treatment due to a lack of improvement in itching and skin lesions.
This study suggests that dupilumab is a promising candidate in treatment of all subtypes of DEB due to its reputation for fast alleviating pruritus and long-term safety.
Dupilumab appeared more effective for itch control in DEB pruriginosa than in severe DEB. After one year, four pruriginosa patients had NRS scores of 1, compared with scores of 2 to 3 in patients with severe DEB.
Quality of life, measured by the Dermatology Life Quality Index, improved steadily. Mean scores improved from 19.1 at baseline to 10.36 at 16 weeks, 5.82 at 24 weeks, and 5 after one year.
No serious adverse events were reported. One patient developed mild conjunctivitis (pink eye) after four doses of dupilumab, which resolved with eye drops. Routine blood tests, including liver and kidney function, remained normal.
The team noted that several patients continued dupilumab for up to 3.5 years, with only occasional lesions at friction sites. Itch control and quality-of-life improvements remained stable in these patients, and no long-term safety issues were observed.
The findings support “larger randomized placebo-controlled trials to better elucidate the efficacy of dupilumab in management of DEB,” the researchers wrote.
