Amy S. Paller, Elaine C. Siegfried, Eric L. Simpson, Michael J. Cork, Robert Sidbury, Iris H. Chen, Faisal A. Khokhar, Jing Xiao, Ariane Dubost-Brama, Ashish Bansal
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Long-term safety and efficacy data in this patient population are needed to inform continuous AD management.</p><h3>Objectives</h3><p>The purpose of this work was to determine the long-term safety and efficacy of dupilumab treatment up to 1 year in an open-label extension (OLE) study [LIBERTY AD PED-OLE (NCT02612454)] in children aged 6 months to 5 years with moderate-to-severe AD who previously participated in the 16-week, double-blind, phase 3 LIBERTY AD PRESCHOOL trial (NCT03346434 part B; parent study) and were subsequently enrolled in PED-OLE.</p><h3>Methods</h3><p>In PED-OLE, patients received dupilumab every 4 weeks according to a weight-tiered regimen (body weight ≥ 5 kg to < 15 kg: 200 mg; ≥ 15 kg to < 30 kg: 300 mg).</p><h3>Results</h3><p>Data for 142 patients were analyzed, 60 of whom had completed the 52-week visit at time of database lock. Mean age at baseline was 4.1 y [SD, 1.13; range, 1.0–5.9 years]. A majority (78.2%) of patients reported ≥ 1 treatment-emergent adverse event (TEAE), most of which were mild or moderate and transient. The most frequently reported TEAEs were nasopharyngitis (19.7%), cough (15.5%), and pyrexia (14.1%). One TEAE led to treatment discontinuation (severe urticaria, which resolved in 1 day). By week 52, 36.2% of patients had achieved an Investigator’s Global Assessment score of 0/1 (clear/almost clear skin), and 96.6%, 79.3%, and 58.6% had at least 50%, 75%, or 90% improvement, respectively, in Eczema Area and Severity Index scores.</p><h3>Conclusions</h3><p>Consistent with results seen in adults, adolescents, and older children (aged 6–11 years), treatment with dupilumab for up to 1 year in children aged 6 months to 5 years with inadequately controlled moderate-to-severe AD demonstrated an acceptable long-term safety profile and sustained efficacy. 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引用次数: 0
摘要
背景:患有中度至重度特应性皮炎(AD)的儿童患者通常疾病负担较重,且病情持续存在的风险较高。尽管存在持续使用安全性不佳和停药后复发风险的问题,但标准的免疫抑制系统治疗一直被用于儿童特应性皮炎的标示外治疗。生物制剂 dupilumab 是首个获准用于 6 个月大儿童中度至重度 AD 的全身治疗药物。我们需要这一患者群体的长期安全性和疗效数据,以便为持续性 AD 管理提供依据:这项工作的目的是在一项开放标签扩展(OLE)研究[LIBERTY AD PED-OLE(NCT02612454)]中,确定6个月至5岁中重度AD儿童接受长达1年的dupilumab治疗的长期安全性和疗效,这些儿童之前参加了为期16周的双盲3期LIBERTY AD PRESCHOOL试验(NCT03346434 B部分;母研究),随后被纳入PED-OLE:在PED-OLE中,患者按照体重阶梯疗法(体重≥5 kg至<15 kg:200 mg;≥15 kg至<30 kg:300 mg)每4周接受一次dupilumab治疗:对142名患者的数据进行了分析,其中60名患者在数据库锁定时已完成52周的访问。基线时的平均年龄为 4.1 岁 [SD, 1.13; range, 1.0-5.9 years]。大多数患者(78.2%)报告了≥1次治疗突发不良事件(TEAE),其中大多数为轻度或中度,且为一过性。最常报告的 TEAE 为鼻咽炎(19.7%)、咳嗽(15.5%)和发热(14.1%)。有 1 例 TEAE 导致停药(严重荨麻疹,1 天后缓解)。到第52周时,36.2%的患者的研究者总体评估得分达到0/1(皮肤清澈/几乎清澈),96.6%、79.3%和58.6%的患者的湿疹面积和严重程度指数得分分别至少改善了50%、75%或90%:与在成人、青少年和年龄较大的儿童(6-11 岁)身上看到的结果一致,对于 6 个月至 5 岁中度至重度 AD 控制不佳的儿童,使用杜比单抗长达 1 年的治疗显示出了可接受的长期安全性和持续疗效。这些结果支持在这一患者群体中长期持续使用dupilumab:试验注册:ClinicalTrials.gov Identifiers:试验注册:ClinicalTrials.gov Identifiers:NCT02612454 和 NCT03346434(B 部分)。
Dupilumab Safety and Efficacy up to 1 Year in Children Aged 6 Months to 5 Years with Atopic Dermatitis: Results from a Phase 3 Open-Label Extension Study
Background
Pediatric patients with moderate-to-severe atopic dermatitis (AD) often experience a high disease burden and have a high risk of persistent disease. Standard-of-care immunosuppressive systemic treatments have been used off-label for AD in pediatric patients despite concerns for suboptimal safety with continuous use and risk of relapse upon discontinuation. The biologic agent dupilumab is the first systemic treatment approved for moderate-to-severe AD in children as young as 6 months. Long-term safety and efficacy data in this patient population are needed to inform continuous AD management.
Objectives
The purpose of this work was to determine the long-term safety and efficacy of dupilumab treatment up to 1 year in an open-label extension (OLE) study [LIBERTY AD PED-OLE (NCT02612454)] in children aged 6 months to 5 years with moderate-to-severe AD who previously participated in the 16-week, double-blind, phase 3 LIBERTY AD PRESCHOOL trial (NCT03346434 part B; parent study) and were subsequently enrolled in PED-OLE.
Methods
In PED-OLE, patients received dupilumab every 4 weeks according to a weight-tiered regimen (body weight ≥ 5 kg to < 15 kg: 200 mg; ≥ 15 kg to < 30 kg: 300 mg).
Results
Data for 142 patients were analyzed, 60 of whom had completed the 52-week visit at time of database lock. Mean age at baseline was 4.1 y [SD, 1.13; range, 1.0–5.9 years]. A majority (78.2%) of patients reported ≥ 1 treatment-emergent adverse event (TEAE), most of which were mild or moderate and transient. The most frequently reported TEAEs were nasopharyngitis (19.7%), cough (15.5%), and pyrexia (14.1%). One TEAE led to treatment discontinuation (severe urticaria, which resolved in 1 day). By week 52, 36.2% of patients had achieved an Investigator’s Global Assessment score of 0/1 (clear/almost clear skin), and 96.6%, 79.3%, and 58.6% had at least 50%, 75%, or 90% improvement, respectively, in Eczema Area and Severity Index scores.
Conclusions
Consistent with results seen in adults, adolescents, and older children (aged 6–11 years), treatment with dupilumab for up to 1 year in children aged 6 months to 5 years with inadequately controlled moderate-to-severe AD demonstrated an acceptable long-term safety profile and sustained efficacy. These results support the long-term continuous use of dupilumab in this patient population.
Trial Registration
ClinicalTrials.gov Identifiers: NCT02612454 and NCT03346434 (part B).
期刊介绍:
The American Journal of Clinical Dermatology is dedicated to evidence-based therapy and effective patient management in dermatology. It publishes critical review articles and clinically focused original research covering comprehensive aspects of dermatological conditions. The journal enhances visibility and educational value through features like Key Points summaries, plain language summaries, and various digital elements, ensuring accessibility and depth for a diverse readership.