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Abstract：
Chronic non-bacterial osteomyelitis (CNO) is an autoinflammatory bone-disease of unknown origin. The National Pediatric Rheumatologic Database (NPRD) collects long-term data of children and adolescents with rheumatic diseases including CNO.
To assess characteristics, courses, and outcomes of CNO with onset in childhood and adolescence and to identify outcome predictors.
From 2015 to 2021 patients with a confirmed diagnosis of CNO, who were registered in the NPRD during their first year of disease and at least one follow-up visit, were included in this analysis and observed for up to 4 years.
Four hundred patients with recent diagnosis of CNO were enrolled in the NRPD during the study period. After 4 years, patient data documentation was sufficient to be analyzed in 81 patients. A significant decline of clinical and radiological lesions is reported: at inclusion in the registry, the mean number of clinical lesions was 2.0 and 3.0 MRI lesions per patient. A significant decrease of manifestations during 4 years of follow-up (mean clinical lesions 0.5, p < 0.001; mean MRI lesions 0.9 (p < 0.001)) was documented. A significant improvement of physician global disease activity (PGDA), patient-reported overall well-being, and childhood health assessment questionnaire (C-HAQ) was documented. Therapeutically, an increase of disease-modifying anti-rheumatic drugs over the years can be stated, while bisphosphonates rather seem to be considered as a therapeutic DMARD option in the first years of disease. Only 5-7% of the patients had a severe disease course as defined by a PGDA > = 4. Predictors associated with a severe disease course include the site of inflammation (pelvis, lower extremity, clavicle), increased erythrocyte sedimentation rate, and multifocal disease at first documentation. The previously published composite PedCNO disease activity score was analyzed revealing a PedCNO70 in 55% of the patients at 4YFU.
An improvement of physician global disease activity (PGDA), patient reported overall well-being and imaging-defined disease activity measures was documented, suggesting that inactivity of CNO disease can be reached. PedCNO score and especially PGDA, MRI-defined lesions and in a number of patients also the C-HAQ seem to be reliable parameters for describing disease activity. The identification of risk factors at the beginning of the disease might influence treatment decision in the future.
To assess characteristics, courses, and outcomes of CNO with onset in childhood and adolescence and to identify outcome predictors.
From 2015 to 2021 patients with a confirmed diagnosis of CNO, who were registered in the NPRD during their first year of disease and at least one follow-up visit, were included in this analysis and observed for up to 4 years.
Four hundred patients with recent diagnosis of CNO were enrolled in the NRPD during the study period. After 4 years, patient data documentation was sufficient to be analyzed in 81 patients. A significant decline of clinical and radiological lesions is reported: at inclusion in the registry, the mean number of clinical lesions was 2.0 and 3.0 MRI lesions per patient. A significant decrease of manifestations during 4 years of follow-up (mean clinical lesions 0.5, p < 0.001; mean MRI lesions 0.9 (p < 0.001)) was documented. A significant improvement of physician global disease activity (PGDA), patient-reported overall well-being, and childhood health assessment questionnaire (C-HAQ) was documented. Therapeutically, an increase of disease-modifying anti-rheumatic drugs over the years can be stated, while bisphosphonates rather seem to be considered as a therapeutic DMARD option in the first years of disease. Only 5-7% of the patients had a severe disease course as defined by a PGDA > = 4. Predictors associated with a severe disease course include the site of inflammation (pelvis, lower extremity, clavicle), increased erythrocyte sedimentation rate, and multifocal disease at first documentation. The previously published composite PedCNO disease activity score was analyzed revealing a PedCNO70 in 55% of the patients at 4YFU.
An improvement of physician global disease activity (PGDA), patient reported overall well-being and imaging-defined disease activity measures was documented, suggesting that inactivity of CNO disease can be reached. PedCNO score and especially PGDA, MRI-defined lesions and in a number of patients also the C-HAQ seem to be reliable parameters for describing disease activity. The identification of risk factors at the beginning of the disease might influence treatment decision in the future.
摘要：
背景：慢性非细菌性骨髓炎（CNO）是一种不明原因的自身炎症性骨病。国家小儿风湿病数据库（NPRD）收集了包括CNO在内的风湿性疾病儿童和青少年的长期数据。
目的：为了评估特征，课程,以及儿童期和青春期发病的CNO结局，并确定结局预测因子。
方法：从2015年到2021年确诊为CNO的患者，他们在疾病的第一年和至少一次随访期间在NPRD登记，被纳入本分析，并观察了长达4年。
结果：在研究期间，有四百名最近诊断为CNO的患者被纳入NRPD。四年后,患者数据文档足以对81例患者进行分析.据报道，临床和放射学病变的显着下降：纳入注册表时，每个患者的平均临床病变数为2.0和3.0MRI病变.在4年的随访期间，表现显着减少（平均临床病变0.5，p<0.001；平均MRI病变0.9（p<0.001））。医生全球疾病活动（PGDA）的显着改善，患者报告的总体幸福感，记录儿童健康评估问卷（C-HAQ）。治疗学上,可以说，多年来改善疾病的抗风湿药物的增加，而在疾病的最初几年，双膦酸盐似乎被认为是一种治疗性DMARD的选择。只有5-7%的患者具有由PGDA>=4定义的严重病程。与严重病程相关的预测因素包括炎症部位(骨盆，下肢,锁骨)，红细胞沉降率增加，和多灶性疾病在第一次记录。分析了先前发表的复合PedCNO疾病活动评分，发现在4YFU的55％患者中PedCNO70。
结论：医师全球疾病活动（PGDA）的改善，记录患者报告的总体健康状况和影像学定义的疾病活动指标,表明可以达到CNO疾病的不活动性。PedCNO得分，尤其是PGDA，MRI定义的病变以及许多患者的C-HAQ似乎也是描述疾病活动的可靠参数。疾病开始时对危险因素的识别可能会影响将来的治疗决策。
目的：为了评估特征，课程,以及儿童期和青春期发病的CNO结局，并确定结局预测因子。
方法：从2015年到2021年确诊为CNO的患者，他们在疾病的第一年和至少一次随访期间在NPRD登记，被纳入本分析，并观察了长达4年。
结果：在研究期间，有四百名最近诊断为CNO的患者被纳入NRPD。四年后,患者数据文档足以对81例患者进行分析.据报道，临床和放射学病变的显着下降：纳入注册表时，每个患者的平均临床病变数为2.0和3.0MRI病变.在4年的随访期间，表现显着减少（平均临床病变0.5，p<0.001；平均MRI病变0.9（p<0.001））。医生全球疾病活动（PGDA）的显着改善，患者报告的总体幸福感，记录儿童健康评估问卷（C-HAQ）。治疗学上,可以说，多年来改善疾病的抗风湿药物的增加，而在疾病的最初几年，双膦酸盐似乎被认为是一种治疗性DMARD的选择。只有5-7%的患者具有由PGDA>=4定义的严重病程。与严重病程相关的预测因素包括炎症部位(骨盆，下肢,锁骨)，红细胞沉降率增加，和多灶性疾病在第一次记录。分析了先前发表的复合PedCNO疾病活动评分，发现在4YFU的55％患者中PedCNO70。
结论：医师全球疾病活动（PGDA）的改善，记录患者报告的总体健康状况和影像学定义的疾病活动指标,表明可以达到CNO疾病的不活动性。PedCNO得分，尤其是PGDA，MRI定义的病变以及许多患者的C-HAQ似乎也是描述疾病活动的可靠参数。疾病开始时对危险因素的识别可能会影响将来的治疗决策。
