Varnier Giulia Camilla, Consolaro Alessandro, Cheng Iek Leng, Silva Riveiro Alicia, Pilkington Clarissa, Ravelli Angelo
Università degli Studi di Genova, Genoa, Italy.
Paediatric Rheumatology, Royal Manchester Children's Hospital, Manchester, UK.
Rheumatology (Oxford). 2023 Feb 23;62(SI2):SI163-SI169. doi: 10.1093/rheumatology/keac404.
The objective of this study was to evaluate the efficacy and safety of MMF in juvenile idiopathic inflammatory myopathies (JIIMs).
Patients diagnosed with JIIM and treated with MMF enrolled in the Juvenile Dermatomyositis Research Group (JDRG) in the UK or followed at the Giannina Gaslini Institute in Genoa, Italy, were included. The following information was collected retrospectively at MMF initiation, at 3, 6 and 12 months after treatment start, and at last follow-up visit: clinical manifestations, laboratory data, physicians' subjective assessment of disease activity, standardized outcome measures of muscle strength/endurance, cutaneous disease activity, physical function, global disease activity, cumulative damage, and ongoing treatment.
Of the 29 patients included, 23 had juvenile DM and 6 had overlap myositis. During administration of MMF, improvement in measures of muscle strength, skin disease activity, and overall disease activity was seen, with an increase in the frequency of normal scores for Manual Muscle Test-8 from 50.0% to 83.3%, Childhood Myositis Activity Score from 53.5% to 88.9%, muscle component of DAS from 55.2% to 84.2%, skin component of DAS from 31.0% to 42.1%, visual analogue scale for skin disease activity from 25.0% to 47.4%, and visual analogue scale for overall disease activity from 7.1% to 42.1%. The number of patients with inactive disease increased from 10.3% at baseline to 68.5% at last follow-up. CS dose was significantly reduced, from 0.3 to 0.1 mg/kg/day. No relevant side effects were reported.
Our experience suggests that MMF is a valuable therapeutic option for the management of JIIM.
本研究旨在评估霉酚酸酯（MMF）治疗儿童特发性炎性肌病（JIIMs）的疗效和安全性。
纳入在英国儿童皮肌炎研究组（JDRG）登记或在意大利热那亚的吉安尼娜·加斯利尼研究所接受随访、诊断为JIIM并接受MMF治疗的患者。在开始使用MMF时、治疗开始后3、6和12个月以及最后一次随访时回顾性收集以下信息：临床表现、实验室数据、医生对疾病活动的主观评估、肌肉力量/耐力的标准化结局指标、皮肤疾病活动度、身体功能、整体疾病活动度、累积损伤以及正在进行的治疗。
纳入的29例患者中，23例为儿童皮肌炎，6例为重叠性肌炎。在使用MMF期间，肌肉力量、皮肤疾病活动度和整体疾病活动度指标均有改善，徒手肌力测试-8正常评分的频率从50.0%增至83.3%，儿童肌炎活动评分从53.5%增至88.9%，疾病活动评分（DAS）的肌肉部分从55.2%增至84.2%，DAS的皮肤部分从31.0%增至42.1%，皮肤疾病活动度视觉模拟评分从25.0%增至47.4%，整体疾病活动度视觉模拟评分从7.1%增至42.1%。疾病无活动的患者数量从基线时的10.3%增至最后一次随访时的68.5%。糖皮质激素（CS）剂量从0.3mg/kg/天显著降至0.1mg/kg/天。未报告相关副作用。
我们的经验表明，MMF是治疗JIIM的一种有价值的治疗选择。
