Mancardi Gianluigi, Saccardi Riccardo
Department of Neuroscience, Ophthalmology, and Genetics, and Centre of Excellence for Biomedical Research, San Martino Hospital, University of Genoa, Genoa, Italy.
Lancet Neurol. 2008 Jul;7(7):626-36. doi: 10.1016/S1474-4422(08)70138-8.
Intense immunosuppression followed by autologous haematopoietic stem-cell transplantation has been assessed over the past few years as a possible new therapeutic strategy in severe forms of multiple sclerosis. Pioneering studies began in 1995, and since then, more than 400 patients worldwide have been treated with this procedure. Small uncontrolled studies show that about 60-70% of treated cases do not progress in the follow-up period of at least 3 years. Transplant-related mortality, which was 5-6% in the first reported series, has reduced in the past 5 years to 1-2%. Relapses dramatically decrease and inflammatory MRI activity is almost completely suppressed. Autologous haematopoietic stem-cell transplantation is associated with qualitative immunological changes in the blood, suggesting that, beyond its immunosuppressive potential, it could also have some beneficial effect for the resetting of the immune system. Patients with severe, rapidly worsening multiple sclerosis who are unresponsive to approved therapies could be candidates for this treatment, but its clinical efficacy has still to be shown in large, prospective, controlled studies.
在过去几年中，强烈免疫抑制后进行自体造血干细胞移植已被评估为重度多发性硬化症可能的新治疗策略。开创性研究始于1995年，自那时起，全球已有400多名患者接受了该治疗程序。小型非对照研究表明，在至少3年的随访期内，约60-70%的治疗病例病情未进展。移植相关死亡率在首个报道系列中为5-6%，在过去5年中已降至1-2%。复发显著减少，炎症性MRI活动几乎完全受到抑制。自体造血干细胞移植与血液中的定性免疫变化有关，这表明，除了其免疫抑制潜力外，它对免疫系统的重置也可能有一些有益作用。对已批准治疗无反应的重度、快速恶化的多发性硬化症患者可能是这种治疗的候选者，但其临床疗效仍有待大型、前瞻性、对照研究来证实。
