Efficacy and safety of belimumab for the treatment of refractory childhood-onset systemic lupus erythematosus: A single-center, real-world, retrospective study

Abstract

ObjectiveThis study aimed to investigate the efficacy and safety of belimumab for treating children with refractory childhood-onset systemic lupus erythematosus (cSLE).MethodsTwenty-six cSLE patients who received belimumab treatment in our hospital from January 2020 to September 2021 (23 of them for more than 52 weeks) were enrolled in this study. Their clinical and laboratory data, assessment of disease activity, glucocorticoid dosage, and treatment-emergent adverse events (TEAEs) were retrieved for analysis. The paired samples t-test and the nonparametric test were used to compare the baseline and post-treatment data.ResultsThe mean age of onset was 10.3 ± 2.4 years old; the mean disease duration was 41.6 ± 37.4 months; the median Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score was 10 (P25, P75: 3, 17); and the mean Physician’s Global Assessment (PGA) score at baseline was 1.9 ± 1.0. Compared with the baseline values, there was a significant decrease in the 24-h urine protein quantifications at 24 and 52 weeks of treatment (P<0.05) as well as an elevated complement (C) 3 and C4 levels at 4, 12, 24, and 52 weeks of treatment. In addition, the SLEDAI-2K and PGA scores as well as the percentage of CD19+ B cells were significantly decreased at 12, 24, and 52 weeks of treatment compared with the baseline values (P<0.05). The dosage of glucocorticoid at 4, 12, 24, and 52 weeks of treatment was significantly less than that at baseline or the previous follow-up (P<0.05). At 52 weeks, 14 subjects (53.8%) achieved Lupus Low Disease Activity State (LLDAS), and 4 subjects (15.4%) reached clinical remission (CR). At the last follow-up, 16 subjects (61.5%) achieved LLDAS, and 10 subjects (38.5%) reached CR.ConclusionsBelimumab treatment can significantly improve laboratory indicators, reduce disease activity, and decrease the dosage of glucocorticoid required in children with cSLE. Moreover, it has a good safety profile.

Belimumab in a Patient with Systemic Lupus Erythematosus with Juvenile Onset and Steroid-induced Diabetes: Clinical Case

“…Препарат разрешен для лечения детей с СКВ в 2019 г. на основании результатов международного рандомизированного плацебо-контролируемого исследования PLUTO (The Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy) [11]. Показана высокая эффективность препарата в отношении купирования слизисто-кожных проявлений СКВ, суставного синдрома, тромбоцитопении, а также при необходимости быстрого снижения дозы пероральных глюкокортикоидов [11,14], что было актуальным и для нашей пациентки. По данным J.S.…”

“…Белимумаб хорошо переносится, инфузионные и серьезные постинфузионные реакции гиперчувствительности не зафиксированы [11,14]. Частота нежелательных реакций в исследовании PLUTO в группе белимумаба была сопоставима с таковой в группе плацебо (79,2 и 82,5% соответственно) [11]; большинство нежелательных реакций были легкими и средней тяжести [11].…”

“…Опыт применения белимумаба у детей с СКВ в реальной клинической практике описан в двух клинических наблюдениях [12,13] и одном ретроспективном наблюдательном исследовании [14]. Вместе с тем не сообщалось о применении белимумаба у пациентов с юСКВ и стероидным диабетом.…”

B cell metabolism in autoimmune diseases: signaling pathways and interventions

“…A retrospective, single-center study revealed that belimumab administration in pediatric patients with childhood-onset SLE has demonstrated positive effects on laboratory parameters, disease activity reduction, and a potential decrease in glucocorticoid dosage requirements. Furthermore, belimumab has exhibited a favorable safety record ( 54 ). Povetacicept, an enhanced dual APRIL/BAFF antagonist, demonstrates remarkable improvements in multiple disease indicators in a murine lupus model ( 55 ).…”

Therapierefraktäre Verläufe beim juvenilen systemischen Lupus erythematodes

“…B. für den ZNS-Lupus und -wie erwähnt -die LN nicht zugelassen. Es gibt inzwischen einige Fallserien für den Einsatz von Belimumab auch beim refraktären Lupus [4]. In einer chinesischen Studie wurden 26 Patienten mit refraktärem Lupus über einen Zeitraum von 6-20 Monate mit Belimumab therapiert.…”