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Long-term Transplant-free Survival Is Improved In Hypoplastic Left Heart Syndrome With Cell-based Therapy
Sunjay Kaushal, MD,PhD1, Kevin Ramdas
2, Rick Ohye
3, Narutoshi Hibino
4, Linda Lambert
5, Adil Husain
5, Barry Deatrick
6, Josh Hare
7.
1Sunrise Hospital, Las Vegas, NV, USA,
2Longeveron, Miami, FL, USA,
3Mott Childrens Hospital, Ann Arbor, MI, USA,
4Advocate Childrens Hospital, Chicago, IL, USA,
5Primary Childrens Hospital, Salt Lake City, UT, USA,
6University of Maryland Medical Center, Baltimore, MD, USA,
7Longeveron, miami, FL, USA.
Hypoplastic left heart syndrome (HLHS) has a high mortality due partly to RV dysfunction. We conducted a phase I clinical trial (ELPIS) of allogeneic bone-marrow derived, mesenchymal stem cells designed to improve cardiovascular performance, delivered as a one-time treatment during the Stage II surgery at approximately 4 months after birth. This trial met its primary endpoint (safety through 1-year post-treatment). To assess survival benefits, all ELPIS patients (
n=10, 7 males, and 3 females) were enrolled in a multi-year follow-on study and compared to a retrospective control group which was identified by a relevant clinical HLHS database. Patients in both studies were assessed for up to 5 years (range 3.5 - 5.0 years post-treatment for ELPIS) for mortality, heart transplants, and stage III (Fontan) surgery. Outcomes were compared with long-term historical data from patients in the Single Ventricle Reconstruction (SVR) Trial receiving the same shunt type at Stage I (Norwood) operation. 5-year Kaplan-Meier survival was 100% in ELPIS patients with none requiring heart transplant. This compared to 81.6% (95% CI= [76.5, 87.0]) transplant-free survival in the SVR trial through 5 years post-Glenn surgery, and a 5.2% (95% CI= [2.0, 8.3]) heart transplantation rate. No stem cell related safety issues were reported. These findings support cell based therapy as a potential adjunct to HLHS reconstruction surgery to improve clinical benefits and reduce the need for subsequent heart transplantation. Further long-term follow-up and controlled trials are both warranted and underway.
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