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Topical Recombinant Alpha1-Antitrypsin: A Potential Treatment for Netherton Syndrome?

Juliette Mazereeuw-Hautier, MD, PhD; Judith Cope, BPharm, MSc, MRPharmS; Christina Ong, MBBS, MRCPCH; Adrian Green, RSCN, RGN, AdDip, PgDip; Alain Hovnanian, MD, PhD; John I. Harper, MD, FRCP, FRCPCH
Arch Dermatol. 2006;142(3):393-403. doi:10.1001/archderm.142.3.396.
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Netherton syndrome is a severe autosomal recessive disorder characterized by generalized scaly erythroderma, specific hair shaft defects, and atopic manifestations. Netherton syndrome is caused by mutations in SPINK5, which cause loss of expression of LEKTI (lymphoepithelial Kazal-type–related inhibitor), a serine protease inhibitor.1LEKTI could be involved in skin desquamation by its inhibitory effect on stratum corneum trypsin enzyme and stratum corneum chymotryptic enzyme. LEKTI itself would be the preferred option for replacement therapy, but it is not as yet available. Alpha1-antitrypsin (AAT) is an endogenous serine protease inhibitor used as replacement therapy in congenital AAT deficiency.2 It is a good candidate for LEKTI replacement because it inhibits pancreatic trypsin that demonstrates considerable similarities to stratum corneum trypsin enzyme and stratum corneum chymotryptic enzyme.3 To our knowledge, this is the first study to evaluate the use of topical recombinant human AAT (rAAT) gel for Netherton syndrome. The objective of the study was to evaluate the short-term efficacy and tolerability of this drug in patients with Netherton syndrome.

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Scoring of erythema and scaling for each patient, separately on each side and for each visit using 2 separate 10-point analogue scales (0 corresponded to the absence of lesions and 10 to the most severe skin involvement). AAT indicates alpha1-antitrypsin.

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