Vasquez-Canizares Natalia, Pain Clare E, Zulian Francesco, Yildiz Amra Adrovic, Appenzeller Simone, Marrani Edoardo, Del Giudice Emanuela, Petaccia Antonella, Tirelli Francesca, Simonini Gabriele, Çakan Mustafa, Cattalini Marco, Julio Paulo Rogerio, Torok Kathryn, Khubchandani Raju, Kasapcopur Ozgur, Robinson Lauren, Al-Abadi Eslam, Gunalp Aybuke, Lu Meiping, Lythgoe Hanna, Robinson Amanda, Sozeri Betul, Shenoi Susan, Willis Emily, Clarke Katherine, Zheng Rongjun, Castaldi Biagio, Leone Valentina, Maniscalco Valerio, Stead Lucy, Ambartsumyan Lusine, Rosser Franziska, Duong Phuoc, Minuti Aurelia, Li Suzanne C, Twilt Marinka
Department of Pediatrics, Division of Pediatric Rheumatology, Children's Hospital at Montefiore/ Albert Einstein College of Medicine, 3415 Bainbridge Ave, Bronx, NY, 10467, USA.
Department of Rheumatology, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK.
Pediatr Rheumatol Online J. 2025 May 2;23(1):46. doi: 10.1186/s12969-025-01100-8.
Juvenile systemic sclerosis (JSSc) is a rare multisystemic disease with high morbidity and mortality rates. Treatment options remain limited, and there is a significant unmet need for effective therapies. This study aims to address this gap by investigating current JSSc management practices and identifying key outcome measures that can be used to inform the development of standardized assessment tools for future clinical trials.
A web-based survey was distributed to pediatric rheumatologists to assess cardiopulmonary assessment standard of care practices and immunosuppressive treatment use in JSSc. Respondents were categorized by region (North America, Europe, Latin America, and Asia/Africa), and country income level. A scoping literature review was conducted using the PRISMA-SCR framework to identify outcome measures for six domains in SSc.
One hundred forty-one pediatric rheumatologists from 26 countries completed the survey. Significant variations in JSSc cardiopulmonary assessment practices across regions and income levels were noted. Respondents in North America and Europe reported using pulmonary function tests (PFTs) with diffusing capacity of the lungs for carbon monoxide (DLCO) more frequently than those in Latin America, or Asia/Africa (p < 0.001). The 6-min walk test (6MWT) was used less frequently by respondents in North America than other regions (p = 0.004). Use of oral corticosteroid and cyclophosphamide for treatment of JSSc varies significantly based on country income level, with higher usage in low- and middle-income nations. The scoping review identified 848 relevant articles for data extraction (ranging from 36 to 156 per domain) from a pool of 31,825 records, which were screened in multiple stages by 39 investigators.
We found significant variability in JSSc assessment and treatment preferences, influenced by geography and income. This highlights the urgent need for international collaboration and standardized approaches in JSSc care.
青少年系统性硬化症（JSSc）是一种罕见的多系统疾病，发病率和死亡率都很高。治疗选择仍然有限，对有效疗法存在重大未满足需求。本研究旨在通过调查当前JSSc的管理实践并确定关键结局指标来填补这一空白，这些指标可用于为未来临床试验开发标准化评估工具提供依据。
向儿科风湿病学家进行了一项基于网络的调查，以评估JSSc中心肺评估的护理标准实践和免疫抑制治疗的使用情况。受访者按地区（北美、欧洲、拉丁美洲和亚洲/非洲）以及国家收入水平进行分类。使用PRISMA-SCR框架进行了一项范围界定文献综述，以确定系统性硬化症六个领域的结局指标。
来自26个国家的141名儿科风湿病学家完成了调查。注意到不同地区和收入水平的JSSc心肺评估实践存在显著差异。北美和欧洲的受访者报告使用一氧化碳弥散量（DLCO）的肺功能测试（PFTs）比拉丁美洲或亚洲/非洲的受访者更频繁（p<0.001）。北美受访者使用6分钟步行试验（6MWT）的频率低于其他地区（p=0.004）。口服糖皮质激素和环磷酰胺用于治疗JSSc的情况因国家收入水平而异，低收入和中等收入国家的使用率更高。范围界定综述从31,825条记录中确定了848篇相关文章进行数据提取（每个领域从36篇到156篇不等），由39名研究人员分多个阶段进行筛选。
我们发现JSSc评估和治疗偏好存在显著差异，受地理位置和收入影响。这凸显了在JSSc护理中进行国际合作和采用标准化方法的迫切需求。
