Kuipers Maria Theresa, Kersten Marie José
Amsterdam UMC Locatie VUmc, Amsterdam, The Netherlands
Amsterdam UMC Locatie VUmc, Amsterdam, The Netherlands.
Lupus Sci Med. 2025 Jan 19;12(1):e001157. doi: 10.1136/lupus-2024-001157.
CD19-directed chimeric antigen receptor (CAR) T-cell therapy, originally developed for haematological malignancies, has recently emerged as a promising therapy for patients with autoimmune diseases. By selectively depleting CD19-positive B-cells, this therapy brings a new approach in resetting immune dysregulation and potentially providing long-term remission for patients with a refractory disease. Recent reports have highlighted its effectiveness in conditions such as SLE, systemic sclerosis and myositis. However, while these early results are encouraging, questions remain regarding strategies for optimal patient selection and minimising toxicity on the short and long term. The experiences with CD19 CAR T-cell therapy in haematology may offer valuable insights for immunologists and rheumatologists. This article reviews the key principles learnt in haematology, the results and the mechanisms behind its efficacy, toxicities, and the challenges that need to be addressed for its broader application in clinical practice.
靶向CD19的嵌合抗原受体(CAR)T细胞疗法最初是为血液系统恶性肿瘤开发的,最近已成为治疗自身免疫性疾病患者的一种有前景的疗法。通过选择性清除CD19阳性B细胞,这种疗法为重置免疫失调和潜在地为患有难治性疾病的患者提供长期缓解带来了一种新方法。最近的报告强调了其在系统性红斑狼疮、系统性硬化症和肌炎等病症中的有效性。然而,尽管这些早期结果令人鼓舞,但关于最佳患者选择策略以及短期和长期将毒性降至最低的问题仍然存在。CD19 CAR T细胞疗法在血液学方面的经验可能为免疫学家和风湿病学家提供有价值的见解。本文回顾了在血液学中获得的关键原则、其疗效背后的结果和机制、毒性以及在临床实践中更广泛应用需要解决的挑战。