Comparing Rituximab and Cyclophosphamide in Induction Therapy for Childhood-Onset ANCA-Associated Vasculitis: An ARChiVe registry-cohort study.

IF 3.7 2区 医学 Q1 RHEUMATOLOGY Arthritis Care & Research Pub Date : 2024-10-28 DOI:10.1002/acr.25455
Samuel J Gagne, Vidya Sivaraman, Else S Bosman, Brett Klamer, Kimberly A Morishita, Adam Huber, Alvaro Orjuela, Barbara Eberhard, Charlotte Myrup, Dana Gerstbacher, Dirk Foell, Eslam Al-Abadi, Flora McErlane, Kathryn Cook, Linda Wagner-Weiner, Melissa Elder, L Nandini Moorthy, Paul Dancey, Rae Yeung, Raju Khubchandani, Samundeeswari Deepak, Sirirat Charuvanij, Stacey Tarvin, Susan Shenoi, Tamara Tanner, Kelly Brown, David A Cabral
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Abstract

Objective: Granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) are chronic life-threatening vasculitides requiring substantial immunotherapy. Adult trials identified rituximab (RTX) as an alternative to cyclophosphamide (CYC) for remission-induction of GPA/MPA. Disease rarity has limited feasibility of similar trials in pediatrics. We aim to evaluate the relative efficacy and toxicity of CYC and RTX for childhood GPA/MPA through registry-based comparative evaluation.

Methods: From A Registry of Childhood Vasculitis we identified GPA/MPA patients who received induction with RTX or CYC. Pediatric vasculitis activity score (PVAS) and pediatric vasculitis damage index (pVDI) evaluated disease activity and damage. Descriptive statistics summarized patient characteristics. RTX/CYC comparisons used logistic regression for primary outcomes of post-induction remission (PVAS=0) or low disease activity (PVAS<2). Hospital admission for adverse events and pVDI were compared using logistic regression and ordinal regression, respectively.

Results: Among 104 patients, 43% received RTX, 46% CYC, 11% both. Treatment groups did not significantly differ for diagnosis PVAS and onset age. There was no difference in remission between groups (63% overall; OR 1.07, 95% CI: 0.45, 2.52). Hospitalizations occurred in 22% of RTX patients versus 10% on CYC (OR 2.27, 95% CI: 0.73, 7.05). The median 12-month pVDI was one in both groups (OR 0.98, 95% CI 0.43, 2.22).

Conclusion: This is the first study comparing CYC and RTX for induction in pediatric GPA/MPA. No significant differences were shown in rates of remission, severe adverse events, or organ damage. Limitations included lack of standardized treatment regimens, retrospectivity, and lack of longitudinal adverse drug-related event data.

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比较利妥昔单抗和环磷酰胺在儿童期发病的 ANCA 相关性血管炎诱导疗法中的应用:ARChiVe 登记队列研究。
目的:肉芽肿伴多血管炎(GPA)和显微镜下多血管炎(MPA)是危及生命的慢性血管炎,需要大量的免疫治疗。成人试验发现,利妥昔单抗(RTX)可替代环磷酰胺(CYC)用于诱导 GPA/MPA 的缓解。疾病的罕见性限制了在儿科进行类似试验的可行性。我们的目标是通过基于登记的比较评估来评价 CYC 和 RTX 治疗儿童 GPA/MPA 的相对疗效和毒性:方法:我们从儿童血管炎登记处确定了接受 RTX 或 CYC 诱导治疗的 GPA/MPA 患者。小儿血管炎活动评分(PVAS)和小儿血管炎损伤指数(pVDI)评估了疾病的活动性和损伤情况。描述性统计总结了患者的特征。RTX/CYC比较采用逻辑回归法,主要结果为诱导后缓解(PVAS=0)或低疾病活动性(PVASResults):104名患者中,43%接受了RTX治疗,46%接受了CYC治疗,11%同时接受两种治疗。治疗组在诊断PVAS和发病年龄上没有明显差异。组间缓解率无差异(总体缓解率为 63%;OR 1.07,95% CI:0.45,2.52)。22% 的 RTX 患者住院,而 10% 的 CYC 患者住院(OR 2.27,95% CI:0.73,7.05)。两组患者12个月的中位pVDI均为1(OR为0.98,95% CI为0.43,2.22):这是第一项比较 CYC 和 RTX 用于小儿 GPA/MPA 诱导的研究。结论:这是第一项比较 CYC 和 RTX 诱导治疗小儿 GPA/MPA 的研究,两者在缓解率、严重不良事件或器官损伤方面无明显差异。不足之处包括缺乏标准化治疗方案、回顾性以及缺乏纵向药物相关不良事件数据。
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来源期刊
CiteScore
9.40
自引率
6.40%
发文量
368
审稿时长
3-6 weeks
期刊介绍: Arthritis Care & Research, an official journal of the American College of Rheumatology and the Association of Rheumatology Health Professionals (a division of the College), is a peer-reviewed publication that publishes original research, review articles, and editorials that promote excellence in the clinical practice of rheumatology. Relevant to the care of individuals with rheumatic diseases, major topics are evidence-based practice studies, clinical problems, practice guidelines, educational, social, and public health issues, health economics, health care policy, and future trends in rheumatology practice.
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