Zhang Huayong, Liang Jun, Tang Xiaojun, Wang Dandan, Feng Xuebing, Wang Fan, Hua Bingzhu, Wang Hong, Sun Lingyun
Department of Rheumatology and Immunology, The Affiliated Drum Tower Hospital of Nanjing University Medical School, 321 Zhongshan Road, Nanjing, 210008, China.
Arthritis Res Ther. 2017 Jul 19;19(1):165. doi: 10.1186/s13075-017-1373-2.
Systemic sclerosis (SSc) is an autoimmune disease involving the skin and several internal organs. Most therapies available for this disease are symptomatic. Given the difficulty in treating SSc, we conducted this study to investigate the effect of combined plasmapheresis (PE) and allogeneic mesenchymal stem cells transplantation (MSCT) therapy on SSc.
Fourteen patients underwent three repeated PE treatments with subsequent pulse cyclophosphamide on days 1, 3 and 5. Patients received a single MSCT (1 × 10 cells/kg of body weight) on day 8. During follow up, evaluations performed included complete physical examination, serologic testing, and organ function.
The mean modified Rodnan skin score (MRSS) improved from 20.1 ± 3.1 to 13.8 ± 10.2 (P < 0.001) at 12 months of follow up. Three patients had interstitial lung disease, all had improvement of lung function and improved computed tomography (CT) images after 12 months of combined therapy. This combined treatment also significantly decreased the anti-Scl70 autoantibody titer and serum transforming growth factor-β and vascular endothelial growth factor levels during follow up.
The results indicate that PE combined with MSCT is a feasible treatment associated with possible clinical benefit for SSc patients.
ClinicalTrials.gov, NCT00962923 . Registered on 19 August 2009.
系统性硬化症（SSc）是一种累及皮肤和多个内脏器官的自身免疫性疾病。目前针对该疾病的大多数疗法都是对症治疗。鉴于系统性硬化症治疗困难，我们开展了本研究，以探讨血浆置换（PE）联合异基因间充质干细胞移植（MSCT）疗法对系统性硬化症的疗效。
14例患者接受了3次重复血浆置换治疗，随后在第1、3和5天接受脉冲环磷酰胺治疗。患者在第8天接受单次异基因间充质干细胞移植（1×10⁶细胞/kg体重）。在随访期间，进行的评估包括全面体格检查、血清学检测和器官功能检查。
随访12个月时，平均改良Rodnan皮肤评分（MRSS）从20.1±3.1改善至13.8±10.2（P<0.001）。3例患者患有间质性肺病，联合治疗12个月后，所有患者的肺功能均有改善，计算机断层扫描（CT）图像也有所改善。这种联合治疗在随访期间还显著降低了抗Scl70自身抗体滴度以及血清转化生长因子-β和血管内皮生长因子水平。
结果表明，血浆置换联合异基因间充质干细胞移植对系统性硬化症患者是一种可行的治疗方法，可能具有临床益处。
ClinicalTrials.gov，NCT00962923。于2009年8月19日注册。
