2025-06-03 浙江大学 (ZJU)
Figure 1. Protocol of Ru-D regimen
<関連情報>
- https://www.zju.edu.cn/english/2025/0618/c19573a3062592/page.psp
- https://ashpublications.org/blood/article/doi/10.1182/blood.2024026139/536099/Ruxolitinib-combined-with-dexamethasone-for-adult
中国で新たに血球貪食性リンパ組織球症と診断された成人患者に対するルキソリチニブとデキサメタゾンの併用療法 Ruxolitinib combined with dexamethasone for adult patients with newly diagnosed hemophagocytic lymphohistiocytosis in China
De Zhou,Xianbo Huang,Lixia Zhu,Xuelian Hu,Xiudi Yang,Mixue Xie,Xin Huang,Fang Yu,Juying Wei,Liya Ma,Jingjing Zhu,Shuqi Zhao,Wanzhuo Xie,Hongyan Tong,Jie Jin,Xiujin Ye
Blood Published:March 16, 2025
DOI:https://doi.org/10.1182/blood.2024026139
Key Points
- The Ru-D regimen was well tolerated, resulting in OS rates of 85.7%, 67.9%, and 53.6% at 2 months, 6 months, and 2 years, respectively.
- Chinese patients with lymphoma-associated HLH had poorer outcomes than those with HLH that developed in the absence of underlying lymphoma.
Hemophagocytic lymphohistiocytosis (HLH) is a severe hyperinflammatory syndrome, and the overall survival of adult patients is poor. Ruxolitinib, a Janus kinase (JAK) 1/2 inhibitor, has shown promise in treating HLH and exerts synergistic effects when combined with dexamethasone. Our pilot study preliminarily demonstrated that the combination of ruxolitinib and dexamethasone (the Ru-D regimen) had a high response rate and led to favorable short-term survival outcomes in adult HLH patients. In this prospective phase 2 clinical trial, we propose the Ru-D regimen as a first-line treatment for adults newly diagnosed with HLH with unknown triggers (chictr.org.cn identifier: ChiCTR2100049996). A total of 28 Chinese patients were enrolled, and the median follow-up time was 25.1 months (range, 0.87-34.0). The 2-month OS rate (the primary endpoint) was 85.7%, which exceeded our expected 2-month OS rate of 75%. The 6-month and 2-year OS rates were 67.9% (19/28) and 53.6% (15/28), respectively. The median OS of lymphoma-associated HLH (LAHS) patients was 5.8 months, and most of these patients had NK/T-cell lymphoma. In contrast, the 2-year OS rate of non-LAHS patients was 75%. The overall response rate (ORR) was 85.7% (24/28); 17.9% (5/28) of patients achieved a complete response (CR) during the Ru-D regimen. Overall, the Ru-D regimen was well tolerated in HLH patients. This study demonstrates the efficacy and safety of the Ru-D regimen in adults newly diagnosed with HLH with unknown triggers and warrants a phase 3 randomized controlled study.
