Westwell-Roper Clara, Lubieniecka Joanna M, Brown Kelly L, Morishita Kimberly A, Mammen Cherry, Wagner-Weiner Linda, Yen Eric, Li Suzanne C, O'Neil Kathleen M, Lapidus Sivia K, Brogan Paul, Cimaz Rolando, Cabral David A
Clinical Professor, Division of Rheumatology, Department of Pediatrics, University of British Columbia, BC Children's Hospital, Room K4-119 4480 Oak Street Vancouver, Vancouver, BC, V6H 3V4, Canada.
Simon Fraser University, Burnaby, BC, Canada.
Pediatr Rheumatol Online J. 2017 Aug 7;15(1):61. doi: 10.1186/s12969-017-0191-z.
Because pediatric antineutrophil cytoplasmic antibody-associated vasculitis is rare, management generally relies on adult data. We assessed treatment practices, uptake of existing clinical assessment tools, and interest in pediatric treatment protocols among rheumatologists caring for children with granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA).
A needs-assessment survey developed by an international working group of pediatric rheumatologists and two nephrologists was circulated internationally. Data were summarized with descriptive statistics. Pearson's chi-square tests were used in inferential univariate analyses.
The 209 respondents from 36 countries had collectively seen ~1600 children with GPA/MPA; 144 had seen more than two in the preceding 5 years. Standardized and validated clinical assessment tools to score disease severity, activity, and damage were used by 59, 63, and 36%, respectively; barriers to use included lack of knowledge and limited perceived utility. Therapy varied significantly: use of rituximab rather than cyclophosphamide was more common among respondents from the USA (OR = 2.7 [1.3-5.5], p = 0.0190, n = 139), those with >5 years of independent practice experience (OR = 3.8 [1.3-12.5], p = 0.0279, n = 137), and those who had seen >10 children with GPA/MPA in their careers (OR = 4.39 [2.1-9.1], p = 0.0011, n = 133). Respondents who had treated >10 patients were also more likely to continue maintenance therapy for at least 24 months (OR = 3.0 [1.4-6.4], p = 0.0161, n = 127). Ninety six percent of respondents believed in a need for pediatric-specific treatment guidelines; 46% supported adaptation of adult guidelines while 69% favoured guidelines providing a limited range of treatment options to allow comparison of effectiveness through a registry.
These data provide a rationale for developing pediatric-specific consensus treatment guidelines for GPA/MPA. While pediatric rheumatologist uptake of existing clinical tools has been limited, guideline uptake may be enhanced if outcomes of consensus-derived treatment options are evaluated within the framework of an international registry.
由于儿童抗中性粒细胞胞浆抗体相关性血管炎较为罕见，其治疗通常依赖于成人数据。我们评估了治疗方法、现有临床评估工具的应用情况，以及在照顾患有肉芽肿性多血管炎（GPA）和显微镜下多血管炎（MPA）儿童的风湿病学家中对儿童治疗方案的兴趣。
由国际儿童风湿病学家工作组和两名肾脏病学家制定的需求评估调查问卷在国际范围内分发。数据采用描述性统计进行汇总。在单变量推断分析中使用Pearson卡方检验。
来自36个国家的209名受访者总共诊治了约1600例GPA/MPA患儿；其中144人在过去5年中诊治过超过2例。分别有59%、63%和36%的受访者使用标准化且经过验证的临床评估工具来评估疾病严重程度、活动度和损伤情况；使用的障碍包括知识缺乏和认为实用性有限。治疗方法差异显著：在美国受访者中，使用利妥昔单抗而非环磷酰胺更为常见（OR = 2.7 [1.3 - 5.5]，p = 0.0190，n = 139），独立执业经验超过5年的受访者（OR = 3.8 [1.3 - 12.5]，p = 0.0279，n = 137），以及职业生涯中诊治过超过10例GPA/MPA患儿的受访者（OR = 4.39 [2.1 - 9.1]，p = 0.0011，n = 133）。治疗过超过10例患者的受访者也更有可能持续进行至少24个月的维持治疗（OR = 3.0 [1.4 - 6.4]，p = 0.0161，n = 127）。96%的受访者认为需要针对儿童的治疗指南；46%的人支持改编成人指南，而69%的人倾向于指南提供有限的治疗选择范围，以便通过登记系统比较疗效。
这些数据为制定针对GPA/MPA的儿童特异性共识治疗指南提供了依据。虽然儿童风湿病学家对现有临床工具的应用有限，但如果在国际登记系统的框架内评估共识性治疗方案的结果，可能会提高指南的采用率。
