Seattle Children's Hospital, University of Washington, Seattle, WA, USA.
Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
Lupus. 2023 Aug;32(9):1111-1116. doi: 10.1177/09612033231187752. Epub 2023 Jul 6.
Studies of real-world effectiveness of belimumab in adults with systemic lupus erythematosus have shown improved disease control and decreased oral glucocorticoid use. However, belimumab use outside of clinical trial settings has not been well studied in childhood-onset systemic lupus erythematosus (cSLE). We aimed to characterize indications for belimumab use and evaluate oral glucocorticoid doses and disease activity scores in the year following belimumab initiation at a single, large pediatric rheumatology center.
We included children and young adults with cSLE who received ≥ 1 dose of belimumab. Repeated measures one-way ANOVA was used to compare SLEDAI-2K scores and prednisone-equivalent daily oral glucocorticoid doses at baseline, 6 months, and 12 months after belimumab initiation for those who continued therapy for a year.
We identified 21 patients with cSLE who received ≥ 1 dose of belimumab. The median disease duration at belimumab initiation was 30.8 months [IQR 21.0-79.1]. At the time of belimumab initiation, 100% of patients were taking an antimalarial, 81% were on oral glucocorticoids, and 91% were on at least one conventional DMARD. Thirteen patients (62%) continued belimumab for ≥6 months and 11 (52%) for ≥12 months. Among those continuing belimumab for ≥12 months, median [IQR] oral prednisone daily doses in milligrams at baseline, 6 months, and 12 months were 12.5 [7.5-17.5], 9 [6.25-10], and 5 [5-9.5], = 0.037, and median [IQR] SLEDAI-2K scores at baseline, 6 months, and 12 months were 8 [5.5-10.5], 6 [3.5-10], and 6 [6-8.5], = 0.548, respectively.
In our cohort of pediatric patients with lupus and moderate disease activity treated with belimumab for ≥12 months, daily oral glucocorticoid doses were significantly lower 6 and 12 months after belimumab initiation than baseline. Use in patients with active nephritis was uncommon. Further research is needed in a large, multicenter cohort to determine the real-world effectiveness of belimumab in children and develop guidelines for use.
对成人系统性红斑狼疮（SLE）患者真实世界疗效的研究表明，贝鲁单抗可改善疾病控制并减少口服糖皮质激素的使用。然而，贝鲁单抗在儿童发病的系统性红斑狼疮（cSLE）患者的临床试验以外的使用情况尚未得到很好的研究。我们旨在描述贝鲁单抗使用的指征，并在单一大型儿科风湿病中心评估贝鲁单抗治疗起始后 1 年内口服糖皮质激素剂量和疾病活动评分。
我们纳入了接受至少 1 剂贝鲁单抗的儿童和青年 cSLE 患者。对于接受贝鲁单抗治疗 1 年的患者，采用重复测量单向方差分析比较贝鲁单抗治疗起始时、6 个月和 12 个月时的 SLEDAI-2K 评分和泼尼松等效日口服糖皮质激素剂量。
我们确定了 21 例接受贝鲁单抗治疗的 cSLE 患者。贝鲁单抗治疗起始时的中位疾病病程为 30.8 个月[IQR 21.0-79.1]。贝鲁单抗治疗起始时，100%的患者正在服用抗疟药，81%正在服用口服糖皮质激素，91%正在服用至少一种常规 DMARD。13 例（62%）患者继续使用贝鲁单抗≥6 个月，11 例（52%）患者继续使用贝鲁单抗≥12 个月。在继续使用贝鲁单抗≥12 个月的患者中，基线、6 个月和 12 个月时的中位数[IQR]口服泼尼松每日剂量分别为 12.5[7.5-17.5]、9[6.25-10]和 5[5-9.5]， = 0.037，基线、6 个月和 12 个月时的中位数[IQR]SLEDAI-2K 评分分别为 8[5.5-10.5]、6[3.5-10]和 6[6-8.5]， = 0.548。
在我们接受贝鲁单抗治疗至少 12 个月的患有狼疮且疾病活动度中等的儿科患者队列中，贝鲁单抗治疗起始后 6 个月和 12 个月时的每日口服糖皮质激素剂量明显低于基线。在有活动肾炎的患者中，贝鲁单抗的使用并不常见。需要在大型多中心队列中进行进一步研究，以确定贝鲁单抗在儿童中的真实世界疗效，并制定使用指南。
