Dupilumab可减少Sjögren-Larsson综合征双胞胎的瘙痒。

IF 1.2 4区医学 Q3 DERMATOLOGY

Pediatric Dermatology Pub Date : 2025-10-05 DOI:10.1111/pde.70073

Kennedy Gallagher, Rame Yousif, Kathryn Hummel, Amy S Paller

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引用次数: 0

摘要

Sjögren-Larsson综合征（SLS），现称为aldh3a2综合征型表皮分化障碍（sEDD），是一种罕见的遗传性疾病，以皮肤增厚、痉挛和智力残疾为特征。顽固性瘙痒几乎是SLS的普遍和衰弱特征，目前的治疗方法仍然管理不善。我们描述了患有遗传性SLS的4岁双胞胎女孩，她们在接受靶向白介素-4受体信号的生物制剂dupilumab治疗后，瘙痒症状得到了显着和持久的改善。6个月内，瘙痒严重程度评分明显下降，改善了睡眠和生活质量，无不良反应，支持进一步研究杜匹单抗治疗sls相关瘙痒。

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Dupilumab Reduces Pruritus in Twins With Sjögren-Larsson Syndrome.

Sjögren-Larsson Syndrome (SLS), now termed ALDH3A2-syndromic epidermal differentiation disorder (sEDD), is a rare genetic disorder marked by thickened skin, spasticity, and intellectual disability. Intractable pruritus is a nearly universal and debilitating feature of SLS that remains poorly managed by current therapies. We describe 4-year-old twin girls with genetically confirmed SLS who showed significant and lasting improvement in itch following treatment with dupilumab, a biologic targeting interleukin-4 receptor signaling. Within 6 months, pruritus severity scores declined markedly, improving sleep and quality of life without adverse effects, supporting further investigation of dupilumab for SLS-associated itch.

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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.