心肌病患者细胞治疗后的长期无事件生存率：HYPERION观察队列。

IF 5.4 2区医学 Q1 CELL & TISSUE ENGINEERING

Stem Cells Translational Medicine Pub Date : 2025-05-19 DOI:10.1093/stcltm/szaf010

Russell G Saltzman, Andrew Sundin, Lina V Caceres, Jairo A Tovar, Ana Maria Garzon, Maria A Cabreja, Hossein Shayestehyekta, Jeanette Soto, Dushyantha Jayaweera, Aisha Khan, Ivonne H Schulman, Raul D Mitrani, Joshua M Hare

{"title":"心肌病患者细胞治疗后的长期无事件生存率：HYPERION观察队列。","authors":"Russell G Saltzman, Andrew Sundin, Lina V Caceres, Jairo A Tovar, Ana Maria Garzon, Maria A Cabreja, Hossein Shayestehyekta, Jeanette Soto, Dushyantha Jayaweera, Aisha Khan, Ivonne H Schulman, Raul D Mitrani, Joshua M Hare","doi":"10.1093/stcltm/szaf010","DOIUrl":null,"url":null,"abstract":"Introduction: There is limited long-term clinical outcome data supporting the use of cell-based therapy to treat heart failure. The HYPERION study (NCT03071835) followed long-term outcomes of patients with ischemic cardiomyopathy (ICM) and non-ischemic cardiomyopathy (NIDCM) who received mesenchymal stromal cells (MSC). We hypothesized that improved cardiac parameters predict longer event-free survival.Methods: We performed a Kaplan-Meier analysis to examine event-free survival as the primary outcome. Time-to-event information was captured from all eligible participants. Endpoint events were defined as death (all-cause), Left Ventricular Assist Device (LVAD) placement, or Heart Transplant. Subjects were categorized based on increase in Left Ventricular Ejection Fraction (LVEF) or decrease in Left Ventricular End Diastolic Volume (LVEDV) for comparisons within disease etiologies.Results: There were 134 men and 21 women, with mean age 60.0 ± 11.0 years. There were 121 (78%) with ICM and 34 (22%) with NIDCM. By the end of long-term follow-up (~13 years), 38 (24.5%) subjects had deceased, 5 (3.2%) received LVAD, and 8 (5.2%) underwent heart transplantation. Post-therapy increase of ≥5% LVEF was associated with longer event-free survival in NIDCM (HR:0.31; 95%CI, 0.11,0.86; P = .025), but not ICM (HR:1.14; 95%CI, 0.47,2.72; P = .776). Conversely, reduction in left ventricular end-diastolic volume (LVEDV) was associated with longer event-free survival in ICM (HR:0.16; 95%CI, 0.05, 0.55; P = .008) but not NIDCM (HR:0.35; 95%CI, 0.1,1.2; P = .098). ICM improvers had LVEDV of 225.7 ± 95.9 mL at baseline and 209.0 ± 100.6 mL by year 5 (P = .046). NIDCM improvers had LVEF of 27.2 ± 8.9% at baseline and 36.1 ± 11.6% by year 5 (P = .018).Conclusion: In this long-term observational cohort analysis, improvement of LVEF and/or reduction in LVEDV was associated with survival benefits among subjects with NIDCM and ICM, respectively. In both etiologies the respective improvements are sustained for up to 5 years, providing evidence that cell-based therapy may be a promising and durable treatment option for patients with heart failure.","PeriodicalId":21986,"journal":{"name":"Stem Cells Translational Medicine","volume":"14 5","pages":""},"PeriodicalIF":5.4000,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12105610/pdf/","citationCount":"0","resultStr":"{\"title\":\"Long term event-free survival following cell-based therapy in patients with cardiomyopathy: the HYPERION observational cohort.\",\"authors\":\"Russell G Saltzman, Andrew Sundin, Lina V Caceres, Jairo A Tovar, Ana Maria Garzon, Maria A Cabreja, Hossein Shayestehyekta, Jeanette Soto, Dushyantha Jayaweera, Aisha Khan, Ivonne H Schulman, Raul D Mitrani, Joshua M Hare\",\"doi\":\"10.1093/stcltm/szaf010\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Introduction: There is limited long-term clinical outcome data supporting the use of cell-based therapy to treat heart failure. The HYPERION study (NCT03071835) followed long-term outcomes of patients with ischemic cardiomyopathy (ICM) and non-ischemic cardiomyopathy (NIDCM) who received mesenchymal stromal cells (MSC). We hypothesized that improved cardiac parameters predict longer event-free survival.Methods: We performed a Kaplan-Meier analysis to examine event-free survival as the primary outcome. Time-to-event information was captured from all eligible participants. Endpoint events were defined as death (all-cause), Left Ventricular Assist Device (LVAD) placement, or Heart Transplant. Subjects were categorized based on increase in Left Ventricular Ejection Fraction (LVEF) or decrease in Left Ventricular End Diastolic Volume (LVEDV) for comparisons within disease etiologies.Results: There were 134 men and 21 women, with mean age 60.0 ± 11.0 years. There were 121 (78%) with ICM and 34 (22%) with NIDCM. By the end of long-term follow-up (~13 years), 38 (24.5%) subjects had deceased, 5 (3.2%) received LVAD, and 8 (5.2%) underwent heart transplantation. Post-therapy increase of ≥5% LVEF was associated with longer event-free survival in NIDCM (HR:0.31; 95%CI, 0.11,0.86; P = .025), but not ICM (HR:1.14; 95%CI, 0.47,2.72; P = .776). Conversely, reduction in left ventricular end-diastolic volume (LVEDV) was associated with longer event-free survival in ICM (HR:0.16; 95%CI, 0.05, 0.55; P = .008) but not NIDCM (HR:0.35; 95%CI, 0.1,1.2; P = .098). ICM improvers had LVEDV of 225.7 ± 95.9 mL at baseline and 209.0 ± 100.6 mL by year 5 (P = .046). NIDCM improvers had LVEF of 27.2 ± 8.9% at baseline and 36.1 ± 11.6% by year 5 (P = .018).Conclusion: In this long-term observational cohort analysis, improvement of LVEF and/or reduction in LVEDV was associated with survival benefits among subjects with NIDCM and ICM, respectively. In both etiologies the respective improvements are sustained for up to 5 years, providing evidence that cell-based therapy may be a promising and durable treatment option for patients with heart failure.\",\"PeriodicalId\":21986,\"journal\":{\"name\":\"Stem Cells Translational Medicine\",\"volume\":\"14 5\",\"pages\":\"\"},\"PeriodicalIF\":5.4000,\"publicationDate\":\"2025-05-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12105610/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Stem Cells Translational Medicine\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1093/stcltm/szaf010\",\"RegionNum\":2,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"CELL & TISSUE ENGINEERING\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Stem Cells Translational Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1093/stcltm/szaf010","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CELL & TISSUE ENGINEERING","Score":null,"Total":0}

引用次数: 0

摘要

目前有限的长期临床结果数据支持使用细胞疗法治疗心力衰竭。HYPERION研究（NCT03071835）随访了接受间充质基质细胞（MSC）治疗的缺血性心肌病（ICM）和非缺血性心肌病（NIDCM）患者的长期预后。我们假设心脏参数的改善预示着更长的无事件生存期。方法：我们进行了Kaplan-Meier分析，以检查无事件生存作为主要终点。从所有符合条件的参与者处捕获到事件的时间信息。终点事件定义为死亡（全因）、左心室辅助装置（LVAD）放置或心脏移植。受试者根据左心室射血分数（LVEF）升高或左心室舒张末期容积（LVEDV）降低进行分类，以便在疾病病因中进行比较。结果：男性134例，女性21例，平均年龄60.0±11.0岁。ICM 121例（78%），NIDCM 34例（22%）。长期随访（~13年）结束时，38例（24.5%）受试者死亡，5例（3.2%）接受LVAD， 8例（5.2%）接受心脏移植。治疗后LVEF增加≥5%与NIDCM患者更长的无事件生存期相关(HR:0.31；95%可信区间,0.11,0.86;P = 0.025)，但ICM没有(HR:1.14；95%可信区间,0.47,2.72;p = .776)。相反，ICM患者左室舒张末期容积（LVEDV）的减少与更长的无事件生存相关(HR:0.16；95%ci, 0.05, 0.55；P = 0.008)，而非NIDCM (HR:0.35；95%可信区间,0.1,1.2;p = .098)。ICM改善者基线时LVEDV为225.7±95.9 mL， 5年后为209.0±100.6 mL （P = 0.046）。NIDCM改善者的LVEF基线时为27.2±8.9%，第5年为36.1±11.6% （P = 0.018）。结论：在这项长期观察队列分析中，NIDCM和ICM患者的LVEF改善和/或LVEDV降低分别与生存获益相关。在这两种病因中，各自的改善可持续长达5年，这表明细胞治疗可能是心力衰竭患者的一种有希望和持久的治疗选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Long term event-free survival following cell-based therapy in patients with cardiomyopathy: the HYPERION observational cohort.

Introduction: There is limited long-term clinical outcome data supporting the use of cell-based therapy to treat heart failure. The HYPERION study (NCT03071835) followed long-term outcomes of patients with ischemic cardiomyopathy (ICM) and non-ischemic cardiomyopathy (NIDCM) who received mesenchymal stromal cells (MSC). We hypothesized that improved cardiac parameters predict longer event-free survival.

Methods: We performed a Kaplan-Meier analysis to examine event-free survival as the primary outcome. Time-to-event information was captured from all eligible participants. Endpoint events were defined as death (all-cause), Left Ventricular Assist Device (LVAD) placement, or Heart Transplant. Subjects were categorized based on increase in Left Ventricular Ejection Fraction (LVEF) or decrease in Left Ventricular End Diastolic Volume (LVEDV) for comparisons within disease etiologies.

Results: There were 134 men and 21 women, with mean age 60.0 ± 11.0 years. There were 121 (78%) with ICM and 34 (22%) with NIDCM. By the end of long-term follow-up (~13 years), 38 (24.5%) subjects had deceased, 5 (3.2%) received LVAD, and 8 (5.2%) underwent heart transplantation. Post-therapy increase of ≥5% LVEF was associated with longer event-free survival in NIDCM (HR:0.31; 95%CI, 0.11,0.86; P = .025), but not ICM (HR:1.14; 95%CI, 0.47,2.72; P = .776). Conversely, reduction in left ventricular end-diastolic volume (LVEDV) was associated with longer event-free survival in ICM (HR:0.16; 95%CI, 0.05, 0.55; P = .008) but not NIDCM (HR:0.35; 95%CI, 0.1,1.2; P = .098). ICM improvers had LVEDV of 225.7 ± 95.9 mL at baseline and 209.0 ± 100.6 mL by year 5 (P = .046). NIDCM improvers had LVEF of 27.2 ± 8.9% at baseline and 36.1 ± 11.6% by year 5 (P = .018).

Conclusion: In this long-term observational cohort analysis, improvement of LVEF and/or reduction in LVEDV was associated with survival benefits among subjects with NIDCM and ICM, respectively. In both etiologies the respective improvements are sustained for up to 5 years, providing evidence that cell-based therapy may be a promising and durable treatment option for patients with heart failure.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Stem Cells Translational Medicine CELL & TISSUE ENGINEERING-

CiteScore

12.90

自引率

3.30%

发文量

140

审稿时长

6-12 weeks

期刊介绍： STEM CELLS Translational Medicine is a monthly, peer-reviewed, largely online, open access journal. STEM CELLS Translational Medicine works to advance the utilization of cells for clinical therapy. By bridging stem cell molecular and biological research and helping speed translations of emerging lab discoveries into clinical trials, STEM CELLS Translational Medicine will help move applications of these critical investigations closer to accepted best patient practices and ultimately improve outcomes. The journal encourages original research articles and concise reviews describing laboratory investigations of stem cells, including their characterization and manipulation, and the translation of their clinical aspects of from the bench to patient care. STEM CELLS Translational Medicine covers all aspects of translational cell studies, including bench research, first-in-human case studies, and relevant clinical trials.