Li Si-Yuan, Gao Yu-Han, Dang Yue, Chang Long, Shen Kai-Ni, Cai Hua-Cong, Zhao Dan-Qing, Wei Chong, Feng Jun, Zhang Lu, Li Jian
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, 100730, China.
Ann Hematol. 2025 Mar;104(3):1713-1720. doi: 10.1007/s00277-025-06329-7. Epub 2025 Apr 3.
Idiopathic multicentric Castleman disease (iMCD) is a rare disease characterized by polyclonal lymphoproliferation and systemic inflammation. Siltuximab, targeting interleukin-6 (IL-6), has been recommended as the first-line therapy for iMCD. However, substantial real-world data from China were still lacking, and treatment for patients with severe iMCD remained challenging. This single-center retrospective study investigated the real-world efficacy and safety of siltuximab-based therapy in 43 consecutive patients with iMCD in China from July 2022 to March 2024. The overall response rate (including symptomatic and biochemical response) was 59% at week 3 and increased to 91% at week 12, with complete and partial response rates of 54% and 37%, respectively. Patients who received siltuximab as a first-line treatment exhibited better treatment response (OR = 0·040, 95% CI, 0·004 - 0·390, p = 0·006). Inflammatory markers (such as interleukin-6 and high-sensitivity C-reactive protein [hsCRP]) and pathologic types showed no predictive role in the treatment responses. Eighteen patients, who were all classified as severe iMCD, received combined therapy of siltuximab and BCD regimen (bortezomib, cyclophosphamide and dexamethasone). Of them, the overall response rate was 50% at week 3, which increased to 100% at week 12. Our findings reinforced the existing evidence on the efficacy and safety of siltuximab and highlighted the potential benefits of combining siltuximab with BCD regimen in severe iMCD patients.
特发性多中心Castleman病（iMCD）是一种罕见疾病，其特征为多克隆淋巴细胞增殖和全身炎症。靶向白细胞介素-6（IL-6）的司妥昔单抗已被推荐作为iMCD的一线治疗药物。然而，中国仍缺乏大量真实世界数据，重症iMCD患者的治疗仍然具有挑战性。这项单中心回顾性研究调查了2022年7月至2024年3月期间中国43例连续iMCD患者接受司妥昔单抗治疗的真实世界疗效和安全性。第3周时总缓解率（包括症状和生化缓解）为59%，第12周时升至91%，完全缓解率和部分缓解率分别为54%和37%。接受司妥昔单抗作为一线治疗的患者表现出更好的治疗反应（OR = 0·040，95% CI，0·004 - 0·390，p = 0·006）。炎症标志物（如白细胞介素-6和高敏C反应蛋白[hsCRP]）和病理类型在治疗反应中未显示出预测作用。18例均被归类为重症iMCD的患者接受了司妥昔单抗与硼替佐米、环磷酰胺和地塞米松（BCD方案）的联合治疗。其中，第3周时总缓解率为50%，第12周时升至100%。我们的研究结果强化了关于司妥昔单抗疗效和安全性的现有证据，并突出了司妥昔单抗与BCD方案联合用于重症iMCD患者的潜在益处。
