Abstract：
OBJECTIVE: The aim of this study is to identify whether low lupus disease activity status (LLDAS) and clinical remission (CR) of belimumab plus standard of care (SoC) therapy are achievable goals in childhood-onset SLE (cSLE).
METHODS: This multicentre, one arm pre-post intervention study was conducted at 15 centres in China. The primary end point was to describe the proportion of patients who achieved LLDAS and CR after 3, 6 and 12 months after treatment with belimumab plus SoC therapy. A multiple regression model was used to impute missing data. A Poisson regression model was used to calculate the effect of belimumab treatment on the reduced risk of serious diseases and the incidence of new damage.
RESULTS: A total of 193 (92.2% female) with active cSLE from 15 centres were included. At 3, 6 and 12 months, the proportion of LLDAS (CR) was 12.4% (1.0%), 25.6% (4.5%) and 70.3% (29.7%), respectively. The mean SELENA-SLEDAI score decreased from 11.0 at baseline to 3.7, 2.9 and 1.7 at 3, 6 and 12 months. At baseline, all patients received steroids at a mean (s.d.) prednisone equivalent dose of 31.0 (18.2) mg/day, which decreased to 19.4 (10.8) mg/day at month 3, 12.6 (7.2) mg/day at month 6 and 6.7 (5.3) mg/day at month 12. The symptoms and immunological indicators were also significantly improved.
CONCLUSIONS: This is the first and largest sample size prospective clinical intervention study of cSLE patients treated with belimumab in China. LLDAS and CR were attainable treat-to-target of belimumab plus SoC therapy in cSLE.
METHODS: This multicentre, one arm pre-post intervention study was conducted at 15 centres in China. The primary end point was to describe the proportion of patients who achieved LLDAS and CR after 3, 6 and 12 months after treatment with belimumab plus SoC therapy. A multiple regression model was used to impute missing data. A Poisson regression model was used to calculate the effect of belimumab treatment on the reduced risk of serious diseases and the incidence of new damage.
RESULTS: A total of 193 (92.2% female) with active cSLE from 15 centres were included. At 3, 6 and 12 months, the proportion of LLDAS (CR) was 12.4% (1.0%), 25.6% (4.5%) and 70.3% (29.7%), respectively. The mean SELENA-SLEDAI score decreased from 11.0 at baseline to 3.7, 2.9 and 1.7 at 3, 6 and 12 months. At baseline, all patients received steroids at a mean (s.d.) prednisone equivalent dose of 31.0 (18.2) mg/day, which decreased to 19.4 (10.8) mg/day at month 3, 12.6 (7.2) mg/day at month 6 and 6.7 (5.3) mg/day at month 12. The symptoms and immunological indicators were also significantly improved.
CONCLUSIONS: This is the first and largest sample size prospective clinical intervention study of cSLE patients treated with belimumab in China. LLDAS and CR were attainable treat-to-target of belimumab plus SoC therapy in cSLE.
摘要：
目的：这项研究的目的是确定低狼疮疾病活动度（LLDAS）和临床缓解（CR）的belimumab联合标准治疗（SoC）治疗是否是儿童期发病的SLE（cSLE）可实现的目标。
方法：这个多中心，一项干预前后研究在中国15个中心进行.主要终点是描述使用贝利木单抗加SoC治疗后3、6和12个月达到LLDAS和CR的患者比例。使用多元回归模型来估算缺失数据。使用泊松回归模型来计算贝利木单抗治疗对降低严重疾病风险和新损伤发生率的影响。
结果：来自15个中心的193例（92.2％为女性）患有活动性cSLE。在3、6和12个月时，LLDAS（CR）的比例为12.4%（1.0%），25.6%(4.5%)和70.3%(29.7%)，分别。平均SELENA-SLEDAI评分从基线时的11.0下降到第3、6和12个月时的3.7、2.9和1.7。在基线,所有患者均接受类固醇治疗，平均（SD）泼尼松等效剂量为31.0（18.2）mg/天，在第3个月降至19.4（10.8）mg/天，在第6个月降至12.6（7.2）mg/天，在第12个月降至6.7（5.3）mg/天。症状和免疫学指标也明显改善。
结论：这是中国第一个也是最大样本量的对接受贝利木单抗治疗的cSLE患者的前瞻性临床干预研究。LLDAS和CR是cSLE中belimumab加SoC治疗的目标。
方法：这个多中心，一项干预前后研究在中国15个中心进行.主要终点是描述使用贝利木单抗加SoC治疗后3、6和12个月达到LLDAS和CR的患者比例。使用多元回归模型来估算缺失数据。使用泊松回归模型来计算贝利木单抗治疗对降低严重疾病风险和新损伤发生率的影响。
结果：来自15个中心的193例（92.2％为女性）患有活动性cSLE。在3、6和12个月时，LLDAS（CR）的比例为12.4%（1.0%），25.6%(4.5%)和70.3%(29.7%)，分别。平均SELENA-SLEDAI评分从基线时的11.0下降到第3、6和12个月时的3.7、2.9和1.7。在基线,所有患者均接受类固醇治疗，平均（SD）泼尼松等效剂量为31.0（18.2）mg/天，在第3个月降至19.4（10.8）mg/天，在第6个月降至12.6（7.2）mg/天，在第12个月降至6.7（5.3）mg/天。症状和免疫学指标也明显改善。
结论：这是中国第一个也是最大样本量的对接受贝利木单抗治疗的cSLE患者的前瞻性临床干预研究。LLDAS和CR是cSLE中belimumab加SoC治疗的目标。
