自体造血干细胞移植治疗弥漫性皮肤系统性硬化症:单中心经验。

IF 1 Q4 RHEUMATOLOGY
Reumatismo Pub Date : 2025-09-17 Epub Date: 2025-05-28 DOI:10.4081/reumatismo.2025.1822
Marko Barešić, Ljiljana Smiljanić Tomičević, Igor Aurer, Mislav Cerovec, Branimir Anić
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引用次数: 0

摘要

系统性硬化症(SSc)是一种以多系统累及为特征的慢性自身免疫性疾病。患者可分为惰性或快速进展的病程。一个渐进的过程需要早期和更积极的治疗,以防止不可逆的器官损害。治疗策略应根据症状和器官受累情况量身定制。自体造血干细胞移植(AHSCT)已被证明是治疗特定表型SSc的有效方法,特别是进行性弥漫性皮肤SSc。然而,移植的最佳时机仍然未知。我们报告了两例快速进展的弥漫性皮肤系统性硬化症(dcSSc)在对常规免疫抑制治疗反应不足后用AHSCT治疗。虽然两名患者在ahsct后皮肤均有明显改善,但其中一名患者的内脏器官受损伤进展,最终因严重败血症导致死亡,而另一名患者在长期随访期间保持临床稳定,未接受免疫抑制治疗。该报告为越来越多的证据支持AHSCT作为精心挑选的进展性dcSSc病例的治疗选择做出了贡献。据我们所知,我们的病例是克罗地亚和巴尔干半岛斯拉夫人口中采用这种治疗方式的第一次成功经验,促进了对病程进展的患者,特别是皮肤受损伤患者进行早期干预的必要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Autologous hematopoietic stem-cell transplantation in diffuse cutaneous systemic sclerosis: a single-center experience.

Systemic sclerosis (SSc) is a chronic autoimmune disorder characterized by multisystem involvement. Patients can be stratified into an indolent or rapidly progressive disease course. A progressive course warrants early and more aggressive treatment to prevent irreversible organ damage. Therapeutic strategies should be tailored to the presenting symptoms and organ involvement. Autologous hematopoietic stem cell transplantation (AHSCT) has proven to be an effective treatment modality for specific phenotypes of SSc, especially progressive diffuse cutaneous SSc. However, the optimal timing for the transplantation remains unknown. We present two cases of rapidly progressive diffuse cutaneous SSc (dcSSc) treated with AHSCT following inadequate response to conventional immunosuppressive therapy. While both patients experienced significant cutaneous improvement post-AHSCT, internal organ involvement progressed in one case, ultimately resulting in a fatal outcome due to severe sepsis, whereas the second patient remained clinically stable and without immunosuppressive therapy during long-term follow-up. This report contributes to the growing body of evidence supporting AHSCT as a therapeutic option in carefully selected cases of progressive dcSSc. To our knowledge, our cases are the first successful experiences with this treatment modality in Croatia and among the Slavic populations of the Balkan Peninsula, promoting the need for earlier interventions in patients who develop a progressive disease course, particularly with skin involvement.

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来源期刊
Reumatismo
Reumatismo RHEUMATOLOGY-
CiteScore
2.10
自引率
7.10%
发文量
20
审稿时长
10 weeks
期刊介绍: Reumatismo is the official Journal of the Italian Society of Rheumatology (SIR). It publishes Abstracts and Proceedings of Italian Congresses and original papers concerning rheumatology. Reumatismo is published quarterly and is sent free of charge to the Members of the SIR who regularly pay the annual fee. Those who are not Members of the SIR as well as Corporations and Institutions may also subscribe to the Journal.
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