干细胞移植治疗血栓闭塞性脉管炎的疗效判定

Curative efficacy judgement of the stem cell transplantation in treating thromboangiitis obliterans

目的探讨干细胞移植治疗血栓闭塞性脉管炎(TAO)的疗效判定。方法选取复旦大学附属中山医院2014年4月至2016年12月采用纯化自体CD34+细胞移植(ATPCC)治疗的23例TAO导致的重度肢体缺血(CLI)患者,观察并比较临床疗效[Wong-Baker疼痛评分(WBFPS)、无痛步行时间(PFWT)、溃疡愈合率、总无截肢生存率(TAFS)、主要无截肢生存率(MAFS)、踝肱指数(ABI)、趾肱指数(TBI)及经皮氧分压(TcPO2)]、复发/再发及卢瑟福分级、生活质量(QOL)和安全性。结果移植1、24和36个月WBFPS、PFWT及溃疡愈合率与移植前比较,差异有统计学意义;移植6个月卢瑟福分级与移植前比较,差异有统计学意义;移植24个月非CLI比例最高为78.26%;移植后无患者行踝上截肢,6例患者行截趾术;移植6个月的MAFS及TAFS分别为95.65%和69.57%,移植36个月为91.30%及69.57。移植12~36个月QOL与移植前比较,差异有统计学意义。随访期间,复发1例,再发3例,无不良事件发生。结论在ATPCC治疗TAO的过程中,WBFPS及PFWT在移植后早期表现出明显的差异,之后长时间保持在稳定的水平,可作为治疗TAO疗效的早期主要评价指标;卢瑟福分级的统计学差异出现较晚,可以作为中远期的评价指标;复发/再发率可以作为评估疗法的持久性的指标。

Objective To evaluate the efficacy of stem cell transplantation in the treatment of thromboangiitis obliterans(TAO).Method From April 2014 to December 2016,23 patients with critical limb ischemia(CLI)caused by Tao were selected from Zhongshan Hospital Affiliated to Fudan University and treated with autotransplantation of purified CD34+cells(ATPCC)to observe and compare the clinical efficacy[Wong-Baker FACES pain rating scale(WBFPS),pain free walking time(PFWT),ulcer healing rate,total-amputation-free survival rate(TAFS),major-amputation-free survival rate(MAFS),ankle brachial index(ABI),toe brachial index(TBI)and transcutaneous oxygen partial pressure(TcPO2)],recurrence/recurrence and rutherford classification,quality of life(QOL)and safety.Result The difference of WBFPS,PFWT and ulcer healing rate in 1,24 and 36 months after transplantation was statistically significant compared with that before transplantation;the difference of Rutherford’s classification in 6 months after transplantation was statistically significant compared with that before transplantation;the highest proportion of non-CLI in 24 months after transplantation was 78.26%;no patient underwent ankle amputation after transplantation,and 6 patients underwent toe amputation;the MAFS and TAFS in 6 months after transplantation were 95.65%and 69.57%,respectively;36 underwent transplantation 91.30%and 69.57%respectively.The difference of QOL between 12~36 months after transplantation and before transplantation was statistically significant.During the follow-up,1 case recurred,3 cases recurred,no adverse events occurred.Conclusion WBFPS and PFWT,which could be used as early indicators concerning the efficacy judgment of TAO,showed significant differences in the early stage,then plateaued and kept at a stable level.Likewise,Rutherford classification,which should be used as a mid-to-long term indicator;showed statistical differences at relative late stage.Recurrence/new lesion rate can be used as an indicator of the persistence of the treatment.