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首页> 外文期刊>Annals of the Rheumatic Diseases: A Journal of Clinical Rheumatology and Connective Tissue Research >Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis.
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Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis.

机译：自体造血干细胞移植治疗严重系统性硬化后的长期随访结果。

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OBJECTIVE: Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc. PATIENTS AND METHODS: A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used. RESULTS: After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%)and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years. CONCLUSION: This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.
机译：目的：全身性硬化症（SSc）是一种普遍的自身免疫性疾病，会导致发病和缩短预期寿命，尤其是对于快速进行性弥漫性皮肤SSc的患者。由于尚无经过证实的治疗方法，自体造血干细胞移植（HSCT）被用于治疗预后较差的患者。这项研究报告了重度弥漫性皮肤SSc患者对HSCT的存活，皮肤和主要器官功能的影响。患者与方法：共评估26例患者。使用环磷酰胺（4 g / m2）和rHu G-CSF（5至10 microg / kg /天）收集外周血干细胞，并在阳性CD34 +选择后重新输注。为了进行调节，使用了200mg / kg的环磷酰胺。结果：在中位随访5.3（1-7.5）年之后，有81％（n = 21/26）的患者表现出临床有益的反应。 Kaplan-Meier估计的5年生存率为96.2％（95％CI 89-100％）和7年为84.8％（95％CI 70.2-100％）和无事件生存，定义为无死亡率，复发或复发的生存。 5年时SSc的进展，导致主要器官功能障碍的发生率为64.3％（95％CI 47.9-86％），而7年时为57.1％（95％CI 39.3-83％）。结论：这项研究证实，在移植后严重的皮肤SSc严重患者中，自体HSCT可持续改善皮肤增厚，并在移植后长达7年内稳定器官功能。

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