超说明书使用双剂量Beremagene geperpaec （B-VEC）治疗隐性营养不良大疱性表皮松解症：一个有希望的临床病例。

IF 1.2 4区医学 Q3 DERMATOLOGY

Pediatric Dermatology Pub Date : 2025-09-30 DOI:10.1111/pde.70065

Melissa M Yamada, Liza H Siegel

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引用次数: 0

摘要

营养不良性大疱性表皮松解症（DEB）是一种罕见的遗传性起泡性皮肤病，治疗方案有限。Beremagene geperpavec （B-VEC）是一种基于1型单纯疱疹病毒的局部基因疗法，已被美国食品和药物管理局（FDA）批准用于治疗DEB，每周固定剂量。虽然B-VEC在治疗DEB方面显示出巨大的希望，但很少有研究检查B-VEC在标签外以更高剂量使用的情况。我们描述了在急性荨麻疹发作后大面积伤口的隐性DEB （RDEB）患者中暂时增加B-VEC剂量的成功适应症外使用，强调了高剂量B-VEC在管理大面积伤口负担患者中的潜在效用。

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Off-Label Use of Double-Dose Beremagene Geperpavec (B-VEC) in the Treatment of Recessive Dystrophic Epidermolysis Bullosa: A Promising Clinical Case.

Dystrophic epidermolysis bullosa (DEB) is a rare, inherited blistering skin disease with limited treatment options. Beremagene geperpavec (B-VEC) is a herpes simplex virus type 1-based topical gene therapy that is approved by the Food and Drug Administration (FDA) for the treatment of DEB at a fixed weekly dose. While B-VEC has shown significant promise in treating DEB, few studies have examined off-label use of B-VEC at higher dose volumes. We describe the successful off-label use of a temporarily increased B-VEC dose in a patient with recessive DEB (RDEB) with extensive wounds following an episode of acute urticaria, highlighting the potential utility of higher dose B-VEC in managing patients with extensive wound burden.

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来源期刊

Pediatric Dermatology 医学-皮肤病学

CiteScore

3.20

自引率

6.70%

发文量

269

审稿时长

1 months

期刊介绍： Pediatric Dermatology answers the need for new ideas and strategies for today''s pediatrician or dermatologist. As a teaching vehicle, the Journal is still unsurpassed and it will continue to present the latest on topics such as hemangiomas, atopic dermatitis, rare and unusual presentations of childhood diseases, neonatal medicine, and therapeutic advances. As important progress is made in any area involving infants and children, Pediatric Dermatology is there to publish the findings.