Department of Neurology, The Johns Hopkins Multiple Sclerosis Center, Johns Hopkins University School of Medicine, USA.
Curr Opin Oncol. 2011 Mar;23(2):221-6. doi: 10.1097/CCO.0b013e328342c6b3.
Immunomodulatory medications for multiple sclerosis provide only modest control of this potentially debilitating auto-immune disease of the central nervous system. The immunosuppression provided by high-dose chemotherapy has been studied to address treatment-refractory disease. In this review, we discuss the recent significant work in this field and its associated controversies.
Conclusive evidence for the efficacy of high-dose chemotherapy with stem cell rescue is lacking given the lack of uniform patient populations and varying treatment protocols. Moreover, the significant toxicity associated with this procedure has dampened enthusiasm for its widespread use. High-dose chemotherapy without stem cell rescue has been trialed as a less toxic approach that eliminates the possibility of re-infusing autoreactive lymphocytes found in the stem cell product.
Before high-dose chemotherapy with or without stem cell rescue can be adopted for clinical practice, both approaches require testing in randomized clinical trials. Both procedures have the possibility of decreasing disease activity but high-dose chemotherapy without stem cell rescue having a more favorable safety profile, may prove a more significant advance in the field of high-dose therapy for multiple sclerosis.
免疫调节药物对多发性硬化症的控制作用有限，多发性硬化症是一种潜在使人虚弱的中枢神经系统自身免疫性疾病。大剂量化疗的免疫抑制作用已被研究用于治疗难治性疾病。在这篇综述中，我们讨论了这一领域的最新重要工作及其相关争议。
由于缺乏统一的患者群体和不同的治疗方案，高剂量化疗加干细胞挽救治疗的疗效尚无确凿证据。此外，该治疗方法相关的严重毒性降低了其广泛应用的积极性。高剂量化疗而不进行干细胞挽救已被尝试作为一种毒性较小的方法，以消除干细胞产品中发现的自身反应性淋巴细胞再输注的可能性。
在高剂量化疗加或不加干细胞挽救治疗可以应用于临床实践之前，这两种方法都需要在随机临床试验中进行测试。这两种方法都有可能降低疾病活动度，但高剂量化疗而不进行干细胞挽救治疗具有更好的安全性，可能会成为多发性硬化症高剂量治疗领域的一个更重大的进展。
