自体外周干细胞移植治疗系统性硬化症:向前迈出了重要一步,但我们必须缓和我们的热情!]

L Mouthon
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引用次数: 0

摘要

三项前瞻性随机研究证明了自体造血干细胞(HSC)移植对系统性硬化症(SSc)患者生存的影响。这些结果鼓励我们为那些疾病进展迅速和有早期症状但没有晚期内脏受累的患者提供这种治疗。因此,在弥漫性皮肤SSc患者中,如果疾病持续时间自首次内脏表现(皮肤、心脏、消化、肺部或肾脏)起,排除雷诺现象少于5年,则可以讨论自体HSC移植。然而,在欧洲水平和国家诊断和护理方案(PNDS)中验证的SSc中自体HSC移植的适应症更广泛,其中一些适应症存在争议,特别是在弥漫性间质性肺疾病恶化的患者中。考虑到证据水平和自体造血干细胞移植的毒性,以合理的方式讨论了这些适应症。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[Autologous peripheral stem cell transplantation in systemic sclerosis: An important step forward, but we must temper our enthusiasm!]

Three prospective randomized studies have demonstrated the efficacy of autologous hematopoietic stem cell (HSC) transplantation in systemic sclerosis (SSc) on survival. These results encourage us to offer this therapy to patients who have a rapidly progressive disease and who have early symptoms but no advanced visceral involvement. HSC autograft can thus be discussed in patients with diffuse cutaneous SSc with a duration of the disease since the first visceral manifestations (cutaneous, cardiac, digestive, pulmonary, or renal) excluding Raynaud's phenomenon of less than 5 years. However, the indications for HSC autograft in SSc validated at European level and in the national diagnostic and care protocol (PNDS) are broader and some of these indications are debatable, in particular in patients with worsening diffuse interstitial lung disease. These indications are discussed in a reasoned way, taking into account the level of evidence and the toxicity of the HSC autograft.

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