Gaggiano Carla, de-la-Torre Alejandra, Guerriero Silvana, Costi Stefania, Ragab Gaafar, Paroli Maria Pia, Del Giudice Emanuela, Breda Luciana, La Bella Saverio, Cattalini Marco, Maggio Maria Cristina, Fonollosa Alex, Carreño Ester, Tarsia Maria, Dammacco Rosanna, Hashad Soad, Vitale Antonio, Fotis Lampros, Gentileschi Stefano, Minoia Francesca, Mejía-Salgado Germán, Etayari Halah, Saboya-Galindo Paola, Bernal-Valencia María Andrea, Civino Adele, Chimenti Maria Sole, Caggiano Valeria, Gicchino Maria Francesca, Sota Jurgen, Mauro Angela, Lopalco Giuseppe, Murthy Kalpana Babu, Hegazy Mohamed Tharwat, Gupta Vishali, La Torre Francesco, Amin Rana Hussein, Frediani Bruno, Tosi Gian Marco, Cantarini Luca, Fabiani Claudia
Rheumatology Unit, Department of Medical Sciences, Surgery and Neuroscience, University of Siena, Siena University Hospital [European Reference Network (ERN) for Rare Immunodeficiency, Autoinflammatory and Autoimmune Diseases (RITA) Center], Siena, Italy.
Neuroscience Research Group (NEUROS), Neurovitae Center for Neuroscience, Institute of Translational Medicine (IMT), School of Medicine and Health Sciences, Universidad del Rosario, Bogotá, Colombia.
Clin Exp Ophthalmol. 2025 Aug;53(6):619-626. doi: 10.1111/ceo.14534. Epub 2025 Apr 4.
The study objective was to compare the effectiveness of adalimumab (ADA) in monotherapy and in combination with methotrexate (MTX) for paediatric noninfectious uveitis (NIU).
Registry-based observational study. Children receiving ADA for active uveitis were divided into the ADA monotherapy group (group 1) and the ADA plus MTX combination group (group 2).
Eighty four children were enrolled (146 eyes): 22 in group 1 (26.2%) and 62 in group 2 (73.8%). ADA effectiveness was complete in 48 children (57.1%), partial in 23 (27.4%) and absent in 4 (5.3%), without any differences across the groups (p = 0.89). Fewer relapses per 100 PY occurred after ADA treatment both in group 1 (280.0 vs. 23.0, p = 0.005) and in group 2 (297.9 vs. 86.0, p < 0.001). The final BCVA was similar between groups 1 and 2 [median 1.0 (IQR 0.3) and 1.0 (IQR 0.3), respectively, p = 0.55]. A statistically significant steroid-sparing effect was observed in the entire cohort and in group 2 at the 6-month (p = 0.01 and p = 0.01), 12-month (p = 0.02 and p = 0.02), and last follow-up (p = 0.045 and p = 0.045). The estimated ADA retention rate was 97.1% at 12 months, 87.7% at 24 months, and 82.6% at 36 months, without a statistically significant difference among the groups (p = 0.77).
ADA monotherapy could be equally effective as its combination with MTX in both preventing uveitis relapses and preserving visual acuity in paediatric NIU, with comparable retention rates over 36 months of treatment. The steroid-sparing effect of ADA monotherapy warrants further extensive evaluation to define its optimal placement in the therapeutic strategy for paediatric NIU.
本研究的目的是比较阿达木单抗（ADA）单药治疗及联合甲氨蝶呤（MTX）治疗儿童非感染性葡萄膜炎（NIU）的有效性。
基于登记处的观察性研究。将接受ADA治疗活动性葡萄膜炎的儿童分为ADA单药治疗组（第1组）和ADA联合MTX治疗组（第2组）。
共纳入84名儿童（146只眼）：第1组22名（26.2%），第2组62名（73.8%）。48名儿童（57.1%）的ADA治疗效果完全，23名（27.4%）部分有效，4名（5.3%）无效，两组间无差异（p = 0.89）。第1组（280.0对23.0，p = 0.005）和第2组（297.9对86.0，p < 0.001）在ADA治疗后每100人年的复发次数均减少。第1组和第2组的最终最佳矫正视力相似[中位数分别为1.0（四分位距0.3）和1.0（四分位距0.3），p = 0.55]。在整个队列以及第2组中，在6个月（p = 0.01和p = 0.01）、12个月（p = 0.02和p = 0.02）和末次随访时（p = 0.045和p = 0.045）观察到有统计学意义的激素节省效应。ADA的估计保留率在12个月时为97.1%，24个月时为87.7%，36个月时为82.6%，组间无统计学差异（p = 0.77）。
在预防儿童NIU葡萄膜炎复发和保留视力方面，ADA单药治疗与联合MTX治疗同样有效，在36个月的治疗期内保留率相当。ADA单药治疗的激素节省效应值得进一步广泛评估，以确定其在儿童NIU治疗策略中的最佳位置。
Zotero 插件