Clinical practice report on Dupilumab, Methotrexate, and Cyclosporine A in children with atopic dermatitis 度普利尤单抗、甲氨蝶呤和环孢素A治疗儿童湿疹的临床实践报告

Dupilumab, Methotrexate (MTX), and Cyclosporine A (CsA) have proven effective in clinical trials and practice-based studies for treating children with atopic dermatitis (AD). However, real-world data on their long-term efficacy and safety remain limited, as clinical trials often fail to fully capture daily clinical practice. A study published in JAMA Dermatology [1,2] compared the drug survival rates of Dupilumab, MTX, and CsA, and examined the clinical outcomes of treating pediatric eczema with these therapies, evaluating factors associated with treatment discontinuation to reflect real-world usage.

This cohort study included data from patients aged 2 to 17 years who were treated with Dupilumab, Methotrexate (MTX), and/or Cyclosporine A (CsA) for moderate-to-severe atopic dermatitis (AD). The data were collected from five medical centers in the Netherlands, covering the period from January 1, 2013, to December 31, 2022. The database was locked on July 1, 2023. A total of 362 eczema patients were included in the study, comprising 502 treatment cycles: 192 cycles with Dupilumab, 94 cycles with MTX, and 216 cycles with CsA.

Research Results

  1. Discontinuation due to lack of efficacy: 9.9% in the Dupilumab group, 39.4% in the Methotrexate (MTX) group, and 56.5% in the Cyclosporine A (CsA) group.
  2. Discontinuation due to adverse events: 8.3% in the Dupilumab group, 29.8% in the MTX group, and 23.1% in the CsA group.
  3. Discontinuation due to effective eczema control: 3.1% in the Dupilumab group, 6.4% in the MTX group, and 10.2% in the CsA group.
  4. Concurrent use of oral immunosuppressants during treatment: 3.6% in the Dupilumab group, 4.3% in the MTX group, and 3.2% in the CsA group. It remains unclear how many cases of effective eczema control were due to Dupilumab alone, oral steroids alone, or a combination of Dupilumab and steroids.
  5. Ongoing treatment at the study endpoint: 69.8% in the Dupilumab group, 24.5% in the MTX group, and 10.6% in the CsA group.
  6. Among patients who discontinued due to adverse events, the most common side effects were conjunctivitis in the Dupilumab group, nausea in the MTX group, and headaches in the CsA group.

度普利尤单抗（Dupilumab）、甲氨蝶呤（MTX）和环孢素A（Cyclosporine A）已在基于临床实践的研究和临床试验中证明了其在治疗儿童特应性皮炎（AD）中的有效性。然而，有关其长期疗效和安全性的日常实践数据仍然有限，因为临床试验往往无法完全反映日常临床实践。《美国医学会皮肤病学杂志》（JAMA Dermatology）的一篇研究论文[1,2]对比了Dupilumab 度普利尤单抗、Methotrexate 甲氨蝶呤和 Cyclosporine A 环孢素A的药物生存率，同时公布了用三种药物临床治疗儿童湿疹的效果，通过评估治疗终止相关的因素或特征，反映实际临床使用情况。

该队列研究纳入了2至17岁接受度普利尤单抗（dupilumab）、甲氨蝶呤（MTX）和/或环孢素A（CsA）治疗中重度特应性皮炎（AD）的患者数据。这些数据来源于荷兰五家医疗中心，研究时间范围为2013年1月1日至2022年12月31日，数据库锁定日期为2023年7月1日。研究共纳入362名湿疹患者，总计502个治疗周期，其中包括192个度普利尤单抗（dupilumab）治疗周期、94个甲氨蝶呤（MTX）治疗周期，以及216个环孢素A（CsA）治疗周期。

研究结果

  1. 由于疗效不佳而终止治疗：度普利尤单抗（dupilumab）组中为9.9%，甲氨蝶呤（MTX）组为39.4%，环孢素A（CsA）组为56.5%。
  2. 由于出现不良反应而终止治疗：度普利尤单抗（dupilumab）组中为8.3%，甲氨蝶呤（MTX）组为29.8%，环孢素A（CsA）组为23.1%。
  3. 由于湿疹控制良好而终止治疗：在度普利尤单抗（dupilumab）组中为3.1%，甲氨蝶呤（MTX）组为6.4%，环孢素A（CsA）组为10.2%。
  4. 在治疗过程当中，同时口服免疫抑制剂的：度普利尤单抗（dupilumab）组中有3.6%，甲氨蝶呤（MTX）组为4.3%，环孢素A（CsA）组为3.2%。所以那些因湿疹控制良好的人数中，有多少是因为度普利尤单抗控制了湿疹，还是因为口服激素控制了湿疹，更或是因为度普利尤单抗（dupilumab）与激素共同作用控制了病情，无从知晓。
  5. 在研究截止日仍在继续治疗的：度普利尤单抗（dupilumab）组中为69.8%，甲氨蝶呤（MTX）组为24.5%，环孢素A（CsA）组为10.6%。
  6. 在因不良反应而停止治疗的患者中，最常见的副作用在度普利尤单抗dupilumab组是结膜炎，MTX是恶心，CsA的是头痛。

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Reference

  1. van der Rijst LP, Kamphuis E, Schuttelaar MLA, et al. Drug Survival of Dupilumab, Methotrexate, and Cyclosporine A in Children With Atopic Dermatitis. JAMA Dermatol. 2025;161(1):12–21. doi:10.1001/jamadermatol.2024.3717
  2. van der Rijst LP, Kamphuis E, Schuttelaar MLA, et al. Drug Survival of Dupilumab, Methotrexate, and Cyclosporine A in Children With Atopic Dermatitis. Supplemental Online Content. JAMA Dermatol. 2025;161(1):12–21. doi:10.1001/jamadermatol.2024.3717