Yi Belina Y, Wahezi Dawn M, Covert Lauren, Ardalan Kaveh, Hui-Yuen Joyce, Vasquez-Canizares Natalia, Mosa Doaa Mosad, Jones Madison, Correll Colleen, Begezda Alexis, Shenoi Susan, Wu Eveline Y, Kovalick Leonard K, Lapin W Blaine, Tarvin Stacey E, Oliver Melissa S, Rodriguez Martha M, Marmor Itay, Baszis Kevin W, Taxter Alysha J, Hanson Andrew C, Crowson Cynthia S, Orandi Amir B
Department of Paediatrics, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Paediatric Rheumatology, The Children's Hospital at Montefiore, Bronx, New York, USA.
Clin Exp Rheumatol. 2025 Jul 25. doi: 10.55563/clinexprheumatol/35pbbq.
We performed a multi-institutional retrospective review of patients treated for juvenile dermatomyositis (JDM)-associated calcinosis to analyse the association between treatment outcomes and patient, disease, and treatment characteristics.
Childhood Arthritis and Rheumatology Research Alliance investigators searched their electronic health records for patients with JDM and calcinosis treated between 2003 and 2019 and analysed data at JDM diagnosis, calcinosis diagnosis, and calcinosis treatment. Statistical methods included univariable and multivariable analyses, Kaplan-Meier estimates, and multivariable Cox models.
Data were collected for 63 patients from 11 institutions. Median (IQR) age was 7.8 (4.1-≠11.1) years at JDM diagnosis and 9.4 (5.7-13.3) years at calcinosis diagnosis. Calcinosis was present at JDM diagnosis in 32% of patients (n=20). JDM was active in 76% of patients (47/62) at calcinosis diagnosis. Anti-nuclear matrix protein 2 (anti-NXP2) antibody was the most commonly detected myositis autoantibody (38%, 12/32). The presence of anti-NXP2 or anti-melanoma differentiation-associated gene 5 autoantibody did not significantly affect the probability of any calcinosis improvement (p=0.30). Patients received 103 unique treatment regimens of immunomodulatory agents with or without calcium-modifying agents, but those who received both had the greatest probability of improvement. Intravenous immunoglobulin (IVIG) was associated with a significantly higher probability of calcinosis improvement (p=0.02) than treatments without IVIG. Overall, 79% of patients (n=50) showed improved calcinosis.
Despite wide variations in treatment, many patients showed calcinosis improvement over time, especially those treated with IVIG. Studies using validated outcomes assessments may be needed to develop effective treatment plans for JDM-associated calcinosis.
我们对接受青少年皮肌炎（JDM）相关钙质沉着症治疗的患者进行了多机构回顾性研究，以分析治疗结果与患者、疾病及治疗特征之间的关联。
儿童关节炎与风湿病研究联盟的研究人员在其电子健康记录中搜索2003年至2019年间接受治疗的JDM合并钙质沉着症患者，并分析了JDM诊断、钙质沉着症诊断及钙质沉着症治疗时的数据。统计方法包括单变量和多变量分析、Kaplan-Meier估计以及多变量Cox模型。
收集了来自11个机构的63例患者的数据。JDM诊断时的中位（IQR）年龄为7.8（4.1 - 11.1）岁，钙质沉着症诊断时为9.4（5.7 - 13.3）岁。32%（n = 20）的患者在JDM诊断时即存在钙质沉着症。钙质沉着症诊断时，76%（47/62）的患者JDM处于活动期。抗核基质蛋白2（anti-NXP2）抗体是最常检测到的肌炎自身抗体（38%，12/32）。anti-NXP2或抗黑色素瘤分化相关基因5自身抗体的存在对任何钙质沉着症改善的概率无显著影响（p = 0.30）。患者接受了103种独特的免疫调节药物治疗方案，有或没有钙调节药物，但同时接受两者治疗的患者改善概率最大。静脉注射免疫球蛋白（IVIG）与无IVIG治疗相比，钙质沉着症改善的概率显著更高（p = 0.02）。总体而言，79%（n = 50）的患者钙质沉着症有所改善。
尽管治疗方法差异很大，但许多患者的钙质沉着症随时间推移有所改善，尤其是接受IVIG治疗的患者。可能需要使用经过验证的结局评估的研究来制定JDM相关钙质沉着症的有效治疗方案。
