Real-life effectiveness and safety of systemic treatments and phototherapy in pediatric patients with moderate-to-severe atopic dermatitis: Observational study

Background and Design: Systemic therapies are frequently used off-label for the treatment of atopic dermatitis (AD); however, real-world data are limited.
Materials and Methods: This study aimed to evaluate the efficacy and safety of phototherapy and systemic treatments in patients with severe AD aged <18 years using real-world data. The study, designed as a single-center retrospective observational study, evaluated patients who received at least one systemic treatment or phototherapy between 2019 and 2022. Treatment response was assessed using the Physician Global Assessment (PGA) ordinal scale.
Results: This study evaluated 30 patients and 81 treatment courses. The most common treatment was cyclosporine (CSA), administered to 25 (83.3%) patients, followed by systemic corticosteroids (SCS), administered to 18 (60%) patients (treatment course: 21 patients). Azathioprine (AZA) was used in 12 (40%) patients, methotrexate (MTX) in 7 (23.3%) patients, phototherapy in 5 (16.6%, 6 treatment courses), intravenous immunoglobulin (IVIG) in 5 (16.6%), omalizumab in three (10%), dupilumab in one (3.3%), and mycophenolate mofetil in one (3.3%). While all courses of SCS and IVIG resulted in at least a good response (PGA1 or PGA2), the rates of patients with at least a good response to CSA, AZA, MTX, and phototherapy were 92%, 66.7%, 57.1%, and 50%, respectively (p=0.001). The percentage of patients experiencing side effects was 32% (n=8) for CSA, 23.8% (n=5) for SCS, 33.3% (n=4) for AZA, 14.3% (n=1) for MTX, and 33.3% (n=2) for phototherapy. Two patients discontinued CSA treatment due to side effects: one due to nausea and the other due to impetigo and febrile seizure. None of the patients discontinued other treatments because of side effects.
Conclusion: This observational study indicates that classical immunosuppressive treatments are effective alternatives for children with severe AD without serious side effects. However, comprehensive studies involving novel treatment modalities, such as biologics and small molecules, are warranted.

Keywords: pediatric dermatology, atopic dermatitis, therapy

Orta ve þiddetli atopik dermatitli pediatrik hastalarda sistemik tedavilerin ve fototerapinin gerçek yaþam etkinliði ve güvenliði: Gözlemsel çalýþma

Amaç: Sistemik tedaviler atopik dermatit (AD) tedavisinde lisanssýz olarak sýklýkla kullanýlmalarýna raðmen gerçek yaþam verileri kýsýtlýdýr. Þiddetli AD tanýlý 18 yaþ altý hastalarda fototerapi ve sistemik tedavilerin etkinlik ve güvenilirliklerini gerçek yaþam verileri üzerinden deðerlendirmek.
Gereç ve Yöntem: Tek merkezli retrospektif gözlemsel bir çalýþma olarak tasarlanan çalýþmada, 2019 ile 2022 yýllarý arasýnda en az bir sistemik tedavi veya fototerapi almýþ hastalar deðerlendirildi. Tedavi yanýtý, Hekimin Global Deðerlendirmesi (HGD 0-2) sýralý ölçeði kullanýlarak deðerlendirildi.
Bulgular: Toplam 30 hasta ve 81 tedavi kürü deðerlendirmeye alýndý. En sýk tedavi, 25 (%83,3) hastada kullanýlan siklosporin (CSA) olup bunu 18 hastada (60%) sistemik kortikosteroid (SKS) (21 tedavi kürü) takip etti. Azatioprin (AZA) 12 (%40), metotreksat (MTX) 7 (%23,3), fototerapi 5 (%16,6; 6 tedavi kürü), intravenöz immünglobulin (IVIG) 5 (%16,6), omalizumab 3 (%10), dupilumab ve mikofenolat mofetil birer (%3,3) hastada kullanýlmýþtý. SCS ve IVIG’in tüm kürleri en az "iyi düzeyde yanýtla (HGD1 veya HGD2) sonuçlanýrken, CSA, AZA, MTX ve fototerapi için en az "iyi" düzeyde yanýt alan hasta oranlarý sýrasýyla %92, %66,7, %57,1 ve %50 olarak saptandý (p=0,001). Yan etki gözlenen hasta oranlarý sýrasýyla CSA için %32 (n=8), SKS için %23,8 (n=5), AZA için %33,3 (n=4), MTX için %14,3 (n=1), fototerapi için %33,3 (n=2) olarak saptandý. Ýki hastada CSA tedavisi yan etki nedeniyle sonlandýrýldý: bir hastada bulantý, bir hastada impetigo ve febril nöbet. Diðer tedavi gruplarýnda yan etki nedeniyle tedavisi sonlandýrýlan hasta olmadý.
Sonuç: Çocukluk çaðý þiddetli AD tedavisinde konvansiyonel sistemik tedaviler, güvenli ve etkili tedavi seçenekleri olarak görünmektedir ancak biyolojikler ve küçük moleküller gibi yeni tedavi yöntemlerini de içeren kapsamlý çalýþmalara ihtiyaç vardýr.

Anahtar Kelimeler: pediatrik dermatoloji, atopik dermatit, tedavi