移植改良骨髓间充质干细胞治疗中风的临床结果：一项1/2a期研究。
Clinical Outcomes of Transplanted Modified Bone Marrow-Derived Mesenchymal Stem Cells in Stroke: A Phase 1/2a Study.
作者信息
Steinberg Gary K, Kondziolka Douglas, Wechsler Lawrence R, Lunsford L Dade, Coburn Maria L, Billigen Julia B, Kim Anthony S, Johnson Jeremiah N, Bates Damien, King Bill, Case Casey, McGrogan Michael, Yankee Ernest W, Schwartz Neil E
机构信息
From the Department of Neurosurgery (G.K.S., M.L.C., J.N.J.) and Department of Neurology and Neurological Sciences (G.K.S., N.E.S.), Stanford University School of Medicine and Stanford Health Care, CA; Department of Neurosurgery, New York University and NYU Langone Medical Center, NY (D.K.); Department of Neurosurgery (L.D.L.) and Department of Neurology (L.R.W., J.B.B.), University of Pittsburgh Medical School and University of Pittsburgh Medical Center, PA; Department of Neurology, University of California, San Francisco (A.S.K.); SanBio, Inc, Mountain View, CA (D.B., C.C., M.M., E.W.Y.); and Western Statistical Consulting, LLC, Phoenix, AZ (B.K.).
出版信息
Stroke. 2016 Jul;47(7):1817-24. doi: 10.1161/STROKEAHA.116.012995. Epub 2016 Jun 2.
BACKGROUND AND PURPOSE
Preclinical data suggest that cell-based therapies have the potential to improve stroke outcomes.
METHODS
Eighteen patients with stable, chronic stroke were enrolled in a 2-year, open-label, single-arm study to evaluate the safety and clinical outcomes of surgical transplantation of modified bone marrow-derived mesenchymal stem cells (SB623).
RESULTS
All patients in the safety population (N=18) experienced at least 1 treatment-emergent adverse event. Six patients experienced 6 serious treatment-emergent adverse events; 2 were probably or definitely related to surgical procedure; none were related to cell treatment. All serious treatment-emergent adverse events resolved without sequelae. There were no dose-limiting toxicities or deaths. Sixteen patients completed 12 months of follow-up at the time of this analysis. Significant improvement from baseline (mean) was reported for: (1) European Stroke Scale: mean increase 6.88 (95% confidence interval, 3.5-10.3; P<0.001), (2) National Institutes of Health Stroke Scale: mean decrease 2.00 (95% confidence interval, -2.7 to -1.3; P<0.001), (3) Fugl-Meyer total score: mean increase 19.20 (95% confidence interval, 11.4-27.0; P<0.001), and (4) Fugl-Meyer motor function total score: mean increase 11.40 (95% confidence interval, 4.6-18.2; P<0.001). No changes were observed in modified Rankin Scale. The area of magnetic resonance T2 fluid-attenuated inversion recovery signal change in the ipsilateral cortex 1 week after implantation significantly correlated with clinical improvement at 12 months (P<0.001 for European Stroke Scale).
CONCLUSIONS
In this interim report, SB623 cells were safe and associated with improvement in clinical outcome end points at 12 months.
CLINICAL TRIAL REGISTRATION
URL: https://www.clinicaltrials.gov. Unique identifier: NCT01287936.
背景与目的
临床前数据表明，基于细胞的疗法有可能改善中风预后。
方法
18例稳定的慢性中风患者参与了一项为期2年的开放标签单臂研究，以评估经修饰的骨髓源性间充质干细胞（SB623）手术移植的安全性和临床结局。
结果
安全性人群中的所有患者（N = 18）均经历了至少1次治疗中出现的不良事件。6例患者经历了6次严重的治疗中出现的不良事件；2例可能或肯定与手术操作有关；均与细胞治疗无关。所有严重的治疗中出现的不良事件均已解决，无后遗症。没有剂量限制性毒性反应或死亡。在本次分析时，16例患者完成了12个月的随访。报告了相对于基线（平均值）的显著改善：（1）欧洲卒中量表：平均增加6.88（95%置信区间，3.5 - 10.3；P < 0.001），（2）美国国立卫生研究院卒中量表：平均降低2.00（95%置信区间，-2.7至-1.3；P < 0.001），（3）Fugl - Meyer总分：平均增加19.20（95%置信区间，11.4 - 27.0；P < 0.001），以及（4）Fugl - Meyer运动功能总分：平均增加11.40（95%置信区间，4.6 - 18.2；P < 0.001）。改良Rankin量表未观察到变化。植入后1周同侧皮质磁共振T2液体衰减反转恢复信号变化区域与12个月时的临床改善显著相关（欧洲卒中量表，P < 0.001）。
结论
在这份中期报告中，SB623细胞是安全的，并且与12个月时临床结局终点的改善相关。
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