背景:患有中度至重度特应性皮炎(AD)的儿科患者通常会经历很高的疾病负担,并且患有持续性疾病的风险很高。尽管对持续使用的次优安全性和停药后复发的风险存在担忧,但儿科患者已将标准的免疫抑制全身治疗用于AD。生物剂dupilumab是第一个被批准用于6个月儿童中重度AD的全身性治疗。需要该患者群体的长期安全性和有效性数据来告知持续AD管理。
目的:这项工作的目的是在一项开放标签扩展(OLE)研究[LIBERTYADPED-OLE(NCT02612454)]中确定dupilumab治疗长达1年的长期安全性和有效性,这些儿童患有中度至重度AD,先前参加了16周,双盲,3期LIBERTYADPRESCHOOL试验(NCT03346434B部分;家长研究),随后纳入PED-OLE.
方法:在PED-OLE中,根据体重分级方案(体重≥5kg至<15kg:200mg;≥15kg至<30kg:300mg),患者每4周接受dupilumab.
结果:分析了142例患者的数据,其中60人在数据库锁定时完成了为期52周的访问。基线时的平均年龄为4.1y[SD,1.13;范围,1.0-5.9年]。大多数(78.2%)的患者报告了≥1例治疗紧急不良事件(TEAE),其中大多数是轻度或中度和短暂的。最常报告的TEAE是鼻咽炎(19.7%),咳嗽(15.5%),和发热(14.1%)。一次TEAE导致治疗中断(严重荨麻疹,在1天内解决)。到第52周,36.2%的患者达到了研究者的全球评估评分0/1(透明/几乎透明的皮肤),96.6%,79.3%,58.6%的人至少有50%,75%,或90%的改善,分别,湿疹面积和严重程度指数评分。
结论:与成年人的结果一致,青少年,和年龄较大的儿童(6-11岁),在中度至重度AD控制不充分的6个月至5岁儿童中,使用dupilumab治疗长达1年,证明了可接受的长期安全性和持续疗效.这些结果支持在该患者群体中长期连续使用dupilumab。
背景:ClinicalTrials.gov标识符:NCT02612454和NCT03346434(B部分)。
特应性皮炎(AD)是一种慢性炎症性皮肤病,通常会导致幼儿及其家庭的高疾病负担。患者通常需要长期治疗以控制其疾病症状,包括痒和皮疹.Dupilumab治疗16周已显示对6个月至5岁患有中重度AD的儿童有益处。具有可接受的安全性。由于AD可能会从童年持续到青春期和成年期,有必要提供数据支持在幼儿中长期使用dupilumab.在这项研究中,完成16周研究的儿童继续dupilumab治疗长达1年,每4周接受200mg或300mgdupilumab(取决于孩子的体重)。经过一年的治疗,78.2%的患者报告至少有一个副作用,其中大多数是轻度或中度。只有一名患者因严重皮疹(荨麻疹)而中断治疗,在1天内解决。到了年底,36.2%的患者皮肤清澈或几乎清澈,几乎所有(96.6%)的疾病范围和严重程度至少改善了50%。此外,79.3%,58.6%的患者在疾病范围和严重程度方面至少有75%或90%的改善。总之,与成人的结果一致,青少年,和年长的孩子,这项研究表明,dupilumab治疗1年可持续获益,且安全性可接受.这些结果支持在6个月至5岁患有中度至重度AD的儿童中长期连续使用dupilumab。使用dupilumab治疗中重度特应性皮炎的幼儿的长期安全性和有效性如何?
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.
OBJECTIVE: 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.
BACKGROUND: ClinicalTrials.gov Identifiers: NCT02612454 and NCT03346434 (part B).
Atopic dermatitis (AD) is a chronic inflammatory skin disease that often results in a high disease burden in young children and their families. Patients often need long-term treatment to control their disease symptoms, including itch and rash. Dupilumab treatment for 16 weeks has shown benefits in children aged 6 months to 5 years with moderate-to-severe AD, with an acceptable safety profile. As AD is likely to continue from childhood into adolescence and adulthood, there is a need for data supporting long-term use of dupilumab in young children. In this study, children who completed the 16-week study continued dupilumab treatment for up to 1 year, receiving 200 mg or 300 mg of dupilumab (depending on the child’s bodyweight) every 4 weeks. Through the year of treatment, 78.2% of patients reported at least one side effect, most of which were mild or moderate. Only one patient interrupted treatment because of severe skin rash (hives), which was resolved in 1 day. At the end of the year, 36.2% of patients had clear or almost clear skin, and almost all (96.6%) achieved at least 50% improvement in their extent and severity of disease. Additionally, 79.3%, and 58.6% had at least 75% or 90% improvement in their extent and severity of disease. In summary, consistent with results seen in adults, adolescents, and older children, this study showed that 1-year dupilumab treatment provides continued benefits with an acceptable safety profile. These results support long-term continuous use of dupilumab in children aged 6 months to 5 years with moderate-to-severe AD. What is the long-term safety and efficacy profile in young children with moderate-to-severeatopic dermatitis treated with dupilumab?