Yuan Xiangning, Li Tong, Liu Yudong, Wang Min, Zuo Xiaoxia, Jiang Ying, Zhang Xuan
Department of Nephrology, Xiangya Hospital Central South University, Changsha, Hunan, China.
Postdoctoral Station of Basic Medicine, Xiangya Hospital Central South University, Changsha, Hunan, China.
Lupus Sci Med. 2025 May 12;12(1):e001501. doi: 10.1136/lupus-2025-001501.
Immune thrombocytopenia (ITP) is a haematological manifestation secondary to connective tissue disease (CTD). Many patients with CTD-ITP are refractory to glucocorticoids (GCs) plus immunosuppressant agents (ISAs); rituximab (RTX) is the recommended second-line therapy. Belimumab (BLM) shows efficacy against CTD. We compared the efficacy and safety of RTX and BLM.
Data of patients with CTD-ITP refractory to GCs plus ISAs administered were collected. The data of 11 patients with refractory CTD-ITP who received BLM were compared with those of 15 patients treated with RTX.
At week 2, BLM resulted in a better overall response (OR) than RTX (72.7% vs 26.7%, p=0.045). The OR rate was 60.0% (9/15), 66.7% (10/15) and 73.3% (11/15) at week 4, 8 and 12, respectively, in the RTX group. It remained at 72.7% (8/11) during week 4-12 in the BLM group. Excluding the data of three deceased patients, the OR rate dropped at week 24 in both groups (RTX vs BLM, 61.5% (8/13) vs 70.0% (7/10), p=1.000). At week 24, four patients with OR in both groups successfully withdrew GCs to <15 mg prednisone (RTX vs BLM, 40% (4/10) vs 66.7% (4/6), p=0.608). The serum C3 level did not significantly change, whereas the serum immunoglobulin G level significantly decreased at week 4, 8 and 12 in both groups. There were three patients with serious adverse effects who died of severe pneumonia during weeks 12-24.
BLM may be a safe and effective alternative to RTX for CTD-ITP refractory to GCs plus ISAs.
免疫性血小板减少症（ITP）是结缔组织病（CTD）的一种血液学表现。许多CTD-ITP患者对糖皮质激素（GCs）加免疫抑制剂（ISAs）治疗无效；利妥昔单抗（RTX）是推荐的二线治疗药物。贝利尤单抗（BLM）对CTD有疗效。我们比较了RTX和BLM的疗效及安全性。
收集对GCs加ISAs治疗无效的CTD-ITP患者的数据。将11例接受BLM治疗的难治性CTD-ITP患者的数据与15例接受RTX治疗的患者的数据进行比较。
在第2周时，BLM的总体缓解率（OR）优于RTX（72.7%对26.7%，p=0.045）。RTX组在第4、8和12周时的OR率分别为60.0%（9/15）、66.7%（10/15）和73.3%（11/15）。BLM组在第4至12周期间维持在72.7%（8/11）。排除3例死亡患者的数据后，两组在第24周时的OR率均下降（RTX对BLM，61.5%（8/13）对70.0%（7/10），p=1.000）。在第24周时，两组中达到OR的4例患者成功停用GCs至泼尼松<15mg（RTX对BLM，40%（4/10）对66.7%（4/6），p=0.608）。血清C3水平无显著变化，而两组在第4、8和12周时血清免疫球蛋白G水平显著降低。有3例严重不良反应患者在第12至24周期间死于重症肺炎。
对于对GCs加ISAs治疗无效的CTD-ITP，BLM可能是RTX的一种安全有效的替代药物。
