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

Harold M. Burkhart, Muhammad Yasir Qureshi, Joseph W. Rossano, Susana Cantero Peral, Patrick W. O'Leary, Matthew Hathcock, Walter Kremers, Timothy J. Nelson, Amanda Breuer, Karen Cavanaugh, Frank Cetta, Joseph A. Dearani, Allan Dietz, Brooks Edwards, Shauna Hirsch, Kimberly Holst, Karen Krucker, Kathryn Lenn, Sara Martineau, Christopher E. MascioAngela Majerus, Angela Miller, Jennifer Miller, Karen Miller, Arshid Mir, Timothy M. Olson, Darci Radel, Chelsea Reece, Lori Riess, Sameh M. Said, Juanita Taylor, Julia Thebiay, Jess L. Thompson, Mark Wentworth, Joan Wobig

Research output: Contribution to journalArticlepeer-review

11 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1614-1623
Number of pages10
JournalJournal of Thoracic and Cardiovascular Surgery
Volume158
Issue number6
DOIs
StatePublished - Dec 2019

Keywords

  • hypoplastic left heart syndrome
  • regenerative therapy
  • single ventricle

PubMed: MeSH publication types

  • Clinical Trial, Phase I
  • Journal Article
  • Multicenter Study

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