Division of Immunology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA.
Division of Rheumatology, Children's Hospital of Philadelphia and the University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Semin Arthritis Rheum. 2024 Oct;68:152516. doi: 10.1016/j.semarthrit.2024.152516. Epub 2024 Jul 15.
The pediatric Glucocorticoid Toxicity Index (pGTI) is a new, pediatric-specific tool to quantify glucocorticoid (GC)-related morbidity in children. We evaluated the feasibility and construct validity of retrospective pGTI scoring in patients with pediatric-onset systemic lupus erythematosus (pSLE) and identified risk factors for cumulative toxicity.
We conducted a retrospective cohort study of patients with pSLE treated with GCs at two pediatric centers (1999-2023). GC exposure was estimated using interval-averaged oral prednisone-equivalent dose and cumulative prednisone-equivalent dose. We scored change in GC toxicity every 6 months (±2) using a modified pGTI including 7 of 10 domains. We calculated the Cumulative Worsening Score (CWS), a continuous summation of toxicity accrued. Mixed effects linear regression was used to identify factors associated with CWS.
There were 126 patients with pSLE, including 88 with nephritis, with a median of 6 visits/patient. Nearly half (47 %) experienced toxicity in the Blood Pressure domain. Other common toxicities were mood disturbance (25 %), followed by increased body mass index (BMI), striae, and sleep disturbance (21 % each). Decreased growth velocity was observed in 18 %. There was modest correlation between cumulative GC dose and CWS (rho 0.3; p < 0.01). Greater cumulative toxicity was associated with younger age, elevated BMI, and rituximab use at the time of GC initiation, albeit indications for the latter were not captured.
Patients with pSLE experience a high burden of GC toxicity, particularly related to blood pressure, BMI, sleep, and growth. Standardized, pediatric-specific GC toxicity assessment is feasible in real-world settings and can facilitate evaluation of strategies to reduce morbidity in children requiring chronic GC treatment.
儿科糖皮质激素毒性指数（pGTI）是一种新的、专门针对儿科的工具，用于量化儿童糖皮质激素（GC）相关发病率。我们评估了在儿科发病的系统性红斑狼疮（pSLE）患者中回顾性 pGTI 评分的可行性和构建效度，并确定了累积毒性的危险因素。
我们对在两个儿科中心（1999-2023 年）接受 GC 治疗的 pSLE 患者进行了回顾性队列研究。GC 暴露量通过间隔平均口服泼尼松等效剂量和累积泼尼松等效剂量来估计。我们使用包括 10 个领域中的 7 个领域的改良 pGTI，每 6 个月（±2）对 GC 毒性变化进行评分。我们计算了累积恶化评分（CWS），这是毒性累积的连续总和。使用混合效应线性回归来确定与 CWS 相关的因素。
共有 126 名 pSLE 患者，其中 88 名患有肾炎，每名患者平均就诊 6 次。近一半（47%）患者在血压领域出现毒性。其他常见毒性包括情绪障碍（25%），其次是体重指数增加、条纹和睡眠障碍（各占 21%）。18%的患者出现生长速度下降。累积 GC 剂量与 CWS 之间存在适度相关性（rho 0.3；p<0.01）。更大的累积毒性与年龄较小、BMI 升高以及 GC 起始时使用利妥昔单抗有关，尽管后者的适应证并未被捕获。
pSLE 患者 GC 毒性负担高，特别是与血压、BMI、睡眠和生长有关。在真实环境中，标准化的、专门针对儿科的 GC 毒性评估是可行的，并且可以促进评估减少需要慢性 GC 治疗的儿童发病率的策略。
