Division of Hematologic Malignancies and Cellular Therapy, Duke University School of Medicine, Durham, NC, USA.
Division of Rheumatology and Immunology, Duke University School of Medicine, Box 3874, Durham, NC, 27710, USA.
Curr Allergy Asthma Rep. 2021 Mar 24;21(3):22. doi: 10.1007/s11882-021-00996-y.
Evidence for hematopoietic stem cell transplantation (HCT) in autoimmune disease has been building since the 1990s; however, many clinicians may not yet be aware of its applications to autoimmune disease. We review the basic tenets of HCT and evidence for autologous HCT in multiple sclerosis (MS), systemic sclerosis (SSc), and lupus with an emphasis on recent advanced phase trials.
In MS, the phase 3 randomized MIST trial and the phase 2 randomized ASTIMS trial demonstrated the efficacy of autologous HCT in refractory MS over disease-modifying therapies and mitoxantrone, respectively. In SSc, the phase 3 randomized ASTIS trial and the phase 2 randomized SCOT trial demonstrated the efficacy of autologous HCT in advanced SSc compared to cyclophosphamide. The evidence for HCT in autoimmune diseases continues to grow, particularly in MS and SSc. In lupus, large, comparative trials are still needed. Across autoimmune diseases, questions that still remain to be answered include optimizing patient selection to limit TRM, the appropriate use of MAC, and the necessity for graft manipulation. Furthermore, collaboration between disease-specific and transplant physicians is imperative to expand the appropriate use of HCT in routine clinical practice.
自 20 世纪 90 年代以来，造血干细胞移植（HCT）在自身免疫性疾病中的应用证据不断增加；然而，许多临床医生可能还不知道它在自身免疫性疾病中的应用。我们回顾了 HCT 的基本原理和自体 HCT 在多发性硬化症（MS）、系统性硬化症（SSc）和狼疮中的应用证据，重点介绍了最近的高级临床试验。
在 MS 中，III 期随机 MIST 试验和 II 期随机 ASTIMS 试验分别证明了自体 HCT 在难治性 MS 中的疗效优于疾病修饰疗法和米托蒽醌。在 SSc 中，III 期随机 ASTIS 试验和 II 期随机 SCOT 试验证明了自体 HCT 在晚期 SSc 中的疗效优于环磷酰胺。HCT 在自身免疫性疾病中的证据不断增加，尤其是在 MS 和 SSc 中。在狼疮中，仍需要进行大型比较试验。在自身免疫性疾病中，仍有一些问题需要回答，包括优化患者选择以限制 TRM、MAC 的适当使用以及移植物处理的必要性。此外，疾病特异性和移植医生之间的合作对于扩大 HCT 在常规临床实践中的适当应用至关重要。
