Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis
Autologous hematopoietic stem cell transplantation has better outcomes than conventional therapies in patients with rapidly progressive systemic sclerosis
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DOI：
10.1038/bmt.2016.211
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发表时间：
2017-01-01
影响因子：
4.8
通讯作者：
Cortelezzi, A.
中科院分区：
文献类型：
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作者：
Del Papa, N.;Onida, F.;Cortelezzi, A.
我们回顾性评估了自体造血干细胞移植（AHSCT）对18例快速进展性弥漫性皮肤系统性硬化症（rp-dcSSc）患者的疗效，并将他们的疾病结局与36例接受常规治疗的人口统计学和临床匹配的患者进行了比较。皮肤受累（通过改进罗南皮肤评分（mRss））、肺弥散能力（通过测量肺一氧化碳弥散能力（DLCO））和疾病活动性（通过应用欧洲硬皮病研究组（ESSG）评分系统）是基线时间测量的结果变量，然后每12个月在ahsct治疗的患者和对照组中测量60个月。在AHSCT组中，治疗相关死亡率为5.6%。在该组中，mRss和ESSG评分在AHSCT后1年均显着降低（P < 0.002）；这些结果一直保持到随访结束。相反，DLCO值在整个随访期间保持稳定。AHSCT组患者的生存率明显高于对照组（P = 0.0005）。与AHSCT组相比，整个对照组和环磷酰胺治疗的对照组患者亚组的ESSG评分和mRss维持在高水平，DLCO下降的概率显著高于AHSCT组。本研究证实，在rp-dcSSc患者中，AHSCT在延长生存期、诱导皮肤受损伤和疾病活动度的快速减少以及保持肺功能方面是有效的。
We retrospectively evaluated the efficacy of autologous hematopoietic stem cell transplantation (AHSCT) in 18 patients with rapidly progressive diffuse cutaneous systemic sclerosis (rp-dcSSc), and compared their disease outcomes with those of 36 demographically- and clinically-matched patients treated with conventional therapies. Cutaneous involvement, by performing modified Rodnan skin score (mRss), lung diffusion capacity, by measuring diffusing capacity of lung for carbon monoxide (DLCO), and disease activity, by applying the European Scleroderma Study Group (ESSG) scoring system, were the outcome variables measured at the baseline time and then every 12 months for the following 60 months in both the AHSCT-treated patients and the control group. In the AHSCT group, treatment-related mortality was 5.6%. In this group, both mRss and ESSG scores showed a significant reduction 1 year after AHSCT (P < 0.002); and these results were maintained until the end of follow-up. Conversely, DLCO values remained stable during the whole period of follow-up. Survival rate of AHSCT group was much higher than that observed in the whole control group (P = 0.0005). The probability that the ESSG score and mRss would remain at a high level, and DLCO could decrease, was significantly higher in the control group as a whole and in the subgroup of control patients treated with cyclophosphamide than in the AHSCT group. This study confirms that the AHSCT is effective in prolonging survival, as well as in inducing a rapid reduction of skin involvement and disease activity, and preserving lung function in patients with rp-dcSSc.