Department of Cardiology, Cardiologicum Hamburg, Hamburg, Germany.
Advanced Heart Failure and Transplantation Center, University Medical Centre Ljubljana, Ljubljana, Slovenia.
Eur J Heart Fail. 2019 Aug;21(8):1032-1041. doi: 10.1002/ejhf.1412. Epub 2019 Feb 20.
Ischaemic heart failure (IHF) patients have a poor prognosis even with current guideline-derived therapy. Intramyocardial injections of autologous or allogeneic mesenchymal stromal cells might improve cardiac function leading to better clinical outcome.
The SCIENCE (Stem Cell therapy in IschEmic Non-treatable Cardiac diseasE) consortium has initiated a Horizon 2020 funded multicentre phase II study in six European countries. It is a double-blind, placebo-controlled trial testing the safety and efficacy of allogeneic Cardiology Stem Cell Centre Adipose-derived Stromal Cells (CSCC_ASC) from healthy donors or placebo in 138 symptomatic IHF patients. Main inclusion criteria are New York Heart Association class II-III, left ventricular ejection fraction < 45% and N-terminal pro-B-type natriuretic peptide levels > 300 pg/mL. Patients are randomized in a 2:1 pattern to receive intramyocardial injections of either CSCC_ASC or placebo. CSCC_ASC and placebo treatments are prepared centralized at Rigshospitalet in 5 mL vials as an off-the-shelf product. Vials are distributed to all clinical partners and stored in nitrogen vapour tanks ready to be used directly after thawing. A total of 100 × 10 CSCC_ASC or placebo are injected directly into viable myocardium in the infarct border zone using the NOGA XP system (BDS, Cordis, Johnson & Johnson, USA). Primary endpoint is a centralized core-laboratory assessed change in left ventricular end-systolic volume at 6-month follow-up measured by echocardiography. The trial started in January 2017, 58 patients were included and treated until July 2018.
The SCIENCE trial will provide clinical data on efficacy and safety of intramyocardial cell therapy of allogeneic adipose-derived stromal cells from healthy donors in patients with IHF.
即使采用当前指南指导的治疗方法，缺血性心力衰竭（IHF）患者的预后仍然很差。自体或同种异体间充质基质细胞的心肌内注射可能改善心脏功能，从而带来更好的临床结局。
SCIENCE（Stem Cell therapy in IschEmic Non-treatable Cardiac diseasE）研究联盟已在六个欧洲国家启动了一项由 Horizon 2020 资助的多中心 2 期研究。这是一项双盲、安慰剂对照试验，旨在测试来自健康供体的同种异体心脏干细胞治疗中心脂肪来源的基质细胞（CSCC_ASC）或安慰剂在 138 例有症状的 IHF 患者中的安全性和疗效。主要纳入标准为纽约心脏协会（NYHA）心功能分级 II-III 级、左心室射血分数（LVEF）<45%和 N 末端 pro-B 型利钠肽（NT-proBNP）水平>300pg/mL。患者以 2:1 的比例随机接受 CSCC_ASC 或安慰剂的心肌内注射。CSCC_ASC 和安慰剂治疗均在 Rigshospitalet 以 5mL 小瓶的形式集中制备，作为即用型产品。小瓶分发给所有临床合作单位，并储存在液氮罐中，解冻后可直接使用。共注射 100×10 的 CSCC_ASC 或安慰剂到梗死交界区的存活心肌中，使用 NOGA XP 系统（BDS，Cordis，Johnson & Johnson，USA）。主要终点是通过超声心动图在 6 个月随访时中心实验室评估的左心室收缩末期容积的变化。该试验于 2017 年 1 月开始，共纳入 58 例患者，并于 2018 年 7 月前完成治疗。
SCIENCE 试验将提供关于同种异体健康供体脂肪来源基质细胞的心肌内细胞治疗对 IHF 患者的疗效和安全性的临床数据。
