Department of Rheumatology, Leiden University Medical Center, Leiden, The Netherlands.
Medicine and Rheumatology Unit, IRCCS Istituto Ortopedico Rizzoli, Bologna, Italy.
Clin Exp Rheumatol. 2022 Oct;40(10):1993-1998. doi: 10.55563/clinexprheumatol/5rjvkc. Epub 2022 Oct 17.
Haematopoietic stem cell transplantation (HSCT) is a treatment option for patients with severe systemic sclerosis (SSc), but the efficacy of the procedure in remodelling the nailfold microvascular array is largely unknown. Therefore, this study aimed to evaluate the effect of HSCT on microangiopathy assessed through nailfold capillaroscopy (NC) and to compare the results with findings in patients receiving conventional immunosuppression.
We included SSc patients with severe SSc and whose pre- and post-treatment NC images were available. Findings in patients treated with HSCT were compared with patients not treated with HSCT. Images were scored by two independent observers blinded for clinical data and treatment history. Capillary pattern was determined and semiquantitative scores from 0 (no changes) to 3 (>66% alterations per millimetre) were used to quantify the degree of specific microvascular characteristics. Changes in severity of microangiopathy between baseline and post-treatment were compared between groups.
Images of 18 HSCT patients and 21 controls were scored. From baseline to follow-up, 33% of HSCT patients showed improvement from scleroderma pattern to normal NC, compared to 6% of controls (p=0.15). Pre- to post-treatment differences in semiquantitative scores showed significant improvement in HSCT patients compared to controls regarding capillary loss (-0.5 vs. 0.0, p<0.05) and disorganisation (-0.8 vs. 0.0, p<0.05).
The degree of microangiopathy improved significantly in severe SSc patients treated with HSCT compared with patients receiving conventional immunosuppressive therapy.
造血干细胞移植（HSCT）是治疗严重系统性硬化症（SSc）患者的一种选择，但该方法在重塑甲襞微血管方面的疗效在很大程度上尚不清楚。因此，本研究旨在评估 HSCT 对通过甲襞毛细血管镜（NC）评估的微血管病变的影响，并将结果与接受常规免疫抑制治疗的患者进行比较。
我们纳入了 SSc 患者，这些患者患有严重的 SSc，并且有治疗前后的 NC 图像。将接受 HSCT 治疗的患者的发现与未接受 HSCT 治疗的患者进行比较。由两名独立的观察者对图像进行评分，他们对临床数据和治疗史均不知情。确定毛细血管模式，并使用 0（无变化）至 3（每毫米 >66%改变）的半定量评分来量化特定微血管特征的改变程度。比较两组之间基线和治疗后微血管病变严重程度的变化。
对 18 名 HSCT 患者和 21 名对照者的图像进行了评分。与对照组的 6%相比，从基线到随访，有 33%的 HSCT 患者的硬化症模式转变为正常 NC（p=0.15）。与对照组相比，HSCT 患者的半定量评分在治疗前后的差异显示出明显的改善，包括毛细血管缺失（-0.5 对 0.0，p<0.05）和结构紊乱（-0.8 对 0.0，p<0.05）。
与接受常规免疫抑制治疗的患者相比，接受 HSCT 治疗的严重 SSc 患者的微血管病变程度显著改善。
