Autologous stem cell therapy for hypoplastic left heart syndrome: Safety and feasibility of intraoperative intramyocardial injections.

TitleAutologous stem cell therapy for hypoplastic left heart syndrome: Safety and feasibility of intraoperative intramyocardial injections.
Publication TypeJournal Article
Year of Publication2019
AuthorsBurkhart HM, Qureshi MYasir, Rossano JW, Peral SCantero, O'Leary PW, Hathcock M, Kremers W, Nelson TJ
Corporate AuthorsWanek HLHS Consortium Clinical Pipeline
JournalJ Thorac Cardiovasc Surg
Date Published2019 Jun 07
ISSN1097-685X
Abstract

OBJECTIVES: Staged surgical palliation for hypoplastic left heart syndrome results in an increased workload on the right ventricle serving as the systemic ventricle. Concerns for cardiac dysfunction and long-term heart failure have generated interest in first-in-infant, cell-based therapies as an additional surgical treatment modality.

METHODS: A phase 1 clinical trial was conducted to evaluate the safety and feasibility of direct intramyocardial injection of autologous umbilical cord blood-derived mononuclear cells in 10 infants with hypoplastic left heart syndrome at the time of stage II palliation.

RESULTS: All 10 patients underwent successful stage II palliation and intramyocardial injection of umbilical cord blood-derived mononuclear cells. Operative mortality was 0%. There was a single adverse event related to cell delivery: An injection site epicardial bleed that required simple oversew. The cohort did not demonstrate any significant safety concerns over 6 months. Additionally, the treatment group did not demonstrate any reduction in cardiac function in the context of the study related intramyocardial injections of autologous cells.

CONCLUSIONS: This phase 1 clinical trial showed that delivering autologous umbilical cord blood-derived mononuclear cells directly into the right ventricular myocardium during planned stage II surgical palliation for hypoplastic left heart syndrome was safe and feasible. Secondary findings of preservation of baseline right ventricular function throughout follow-up and normalized growth rates support the design of a phase 2b follow-up trial.

DOI10.1016/j.jtcvs.2019.06.001
Alternate JournalJ. Thorac. Cardiovasc. Surg.
PubMed ID31345560