背景:静脉免疫球蛋白(IVIG)是系统性硬化症SSc的一种新的治疗方法。已经假定了免疫调节和抗纤维化活性。IVIG通常耐受性良好,只有罕见的副作用。我们的回顾性研究将注意力集中在SSc上,一种自身免疫性结缔组织病,以多种并发症为特征,对患者的生活质量有显著影响。病理生理学包括纤维化,血管和免疫方面。
目的:本研究的目的是验证IVIG对SSc皮肤受累的有效性。此外,还对迄今为止在SSc中使用静脉免疫球蛋白(IVIG)的结果进行了文献系统评价(SLR).
方法:24例患者(21例女性,评估了3名男性)的难治性弥漫性SSc皮肤受累(平均年龄为52.13岁)。以2g/Kg体重的剂量连续4天/月输注IVIG,始于2002年至2019年。在治疗前用改良的Rodnan皮肤评分(mRSS)评估皮肤受累,然后在6和12个月后再次评估。要执行单反,PubMed,Medline,Embase,和WebofScience数据库从1990年到2020年进行了搜索(关键词:IVIG,系统性硬化症)。三名评估员(E.A.,C.B.&M.M.C)确定了扫描所有论文的标准。
结果:从总SLR(106个结果)中,在从研究中分离临床病例后,鉴定出17篇论文(治疗患者总数183)。根据每个研究中考虑的器官受累情况对研究进行分类,以及规定的剂量(高或低剂量),和治疗方案。在选定的论文中,主要涉及的器官是皮肤,胃肠,关节和心血管系统。只有在一种情况下,血浆置换与IVIG相关.所有论文都报道了皮肤受累的显著减少,尽管通常工作的强度有限,但缺乏对照病例或涉及的患者人数很少。从真实的生活经验来看,随访6个月时mRSS有统计学意义的降低(平均值-6.61±5.2,p<0.001),并且在12个月后进一步保持了显着的稳定(-11.45±9.63,p<0.002)。
结论:这项SLR和回顾性研究的数据表明,IVIG可以改善皮肤受累,降低mRSS,特别是在那些对其他标准治疗无效的患者中,并且代表了并发肌炎患者的治疗选择。文献综述揭示了使用这种疗法的令人鼓舞的观点,鉴于在选定的作品中发现的有效性。
BACKGROUND: Intravenous immunoglobulins (IVIG) are a new therapeutic approach in systemic sclerosis SSc. An immunomodulatory and antifibrotic activity has been postulated. IVIG are generally well tolerated and have only rare side effects. Our retrospective study focused its attention on SSc, an autoimmune connective tissue disease, characterized by several complications which has a significant impact on patient\'s quality of life. The pathophysiology comprises fibrotic, vascular and immunological aspects.
OBJECTIVE: The aim of this study was to verify the effectiveness of IVIG on SSc skin involvement. Moreover, a systematic
review of the literature (SLR) of the results obtained to date on the use of Intravenous immunoglobulin (IVIG) in SSc has been also performed.
METHODS: The data of 24 patients (21 women, 3 male) with refractory diffuse SSc skin involvement were evaluated (mean age was 52.13 years). IVIG infusion at a dosage of 2 g/Kg body weight for 4 consecutive days/month, was started between 2002 and 2019. Skin involvement was evaluated with the modified Rodnan Skin Score (mRSS) before therapy and then again after 6 and 12 months. To perform the SLR, the PubMed, Medline, Embase, and Web of Science database were searched from 1990 to 2020 (keywords: IVIG, systemic sclerosis). Three assessors (E.A., C.B. & M.M.C) identified the criteria to scan all papers.
RESULTS: From the total SLR (106 results), 17 papers were identified after the separation of the clinical cases from the studies (total number of treated patients 183). The studies were classified according to the organ involvement considered in each study, as well as the prescribed dose (high or low doses), and the therapeutic regimens. In the selected papers, the organs mainly involved were the skin, the gastrointestinal, the joint and the cardiovascular systems. Only in one case, plasmapheresis was associated to IVIG. All papers reported significant reduction of the skin involvement, although generally the strength of the works was limited the lack of control cases or by the low number of patients involved. From the real life experience, a statistically significant reduction of mRSS was obtained at 6 months follow-up (average value of -6.61 ± 5.2, p < 0.001), and it was further maintained with a significant stabilization after 12-months (-11.45 ± 9.63, p < 0.002).
CONCLUSIONS: This SLR and the data of the retrospective study suggest that IVIG may improve skin involvement reducing mRSS in particular in those patients that were refractory to other standard of care therapies and represents a therapeutic option in patients with concomitant myositis. The literature
review revealed encouraging perspectives on the use of this therapy, given the effectiveness found in the selected works.