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Background <p>Hypoplastic Left Heart Syndrome (HLHS) is a severe congenital heart disease characterized by underdevelopment of the left heart structures, often requiring staged surgical palliation. Despite improvements in surgical management, HLHS is associated with high mortality and long-term morbidity. Stem cell-based therapies have emerged as a promising adjunct to improve cardiac function and outcomes in these patients.</p> Objectives <p>This systematic review evaluates the long-term safety and efficacy of stem cell therapy, particularly cardiosphere-derived cells (CDCs) and umbilical cord blood-derived mononuclear cells (UCB-MNCs), in the treatment of HLHS.</p> Methods <p>Following PRISMA guidelines, a comprehensive search was conducted across major databases. Seven studies met the inclusion criteria, comprising randomized controlled trials, observational cohorts, and in vitro analyses. Quality appraisal was performed using RoB2, Newcastle–Ottawa, and SYRCLE tools.</p> Results <p>Stem cell therapy was associated with improvements in right ventricular ejection fraction, somatic growth, heart failure status, and psychosocial quality of life. CDCs showed favorable outcomes in both early and long-term follow-up, with reductions in scar burden, ventricular volume, and adverse events. Importantly, no major adverse effects such as arrhythmias, systemic infections, or tumor formation were reported. The use of allogeneic cells could extend beyond HLHS to other forms of congenital heart disease with right ventricular dysfunction, including tetralogy of Fallot, pulmonary atresia, and other single-ventricle variants. In vitro studies confirmed the regenerative capacity of pediatric-derived stem cells, with the highest potential in the first year of life.</p> Conclusion <p>Stem cell therapy demonstrates safety, feasibility, and potential efficacy in improving cardiac outcomes in HLHS. Larger, multicenter trials such as the APOLLON trial are warranted to validate these findings and support integration into standard clinical care.</p>

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Stem cell therapy for hypoplastic left heart syndrome: a systematic review of efficacy, safety, and future directions

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年12月8日

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  • Kavya Thayappa,
  • Aman Bazarov,
  • Gresa Puwar,
  • Sri Divya Vasupalli,
  • Michael Joy Dara,
  • Jamil Nasrallah,
  • Talha Farooq,
  • Priyansh Patel

摘要

Background

Hypoplastic Left Heart Syndrome (HLHS) is a severe congenital heart disease characterized by underdevelopment of the left heart structures, often requiring staged surgical palliation. Despite improvements in surgical management, HLHS is associated with high mortality and long-term morbidity. Stem cell-based therapies have emerged as a promising adjunct to improve cardiac function and outcomes in these patients.

Objectives

This systematic review evaluates the long-term safety and efficacy of stem cell therapy, particularly cardiosphere-derived cells (CDCs) and umbilical cord blood-derived mononuclear cells (UCB-MNCs), in the treatment of HLHS.

Methods

Following PRISMA guidelines, a comprehensive search was conducted across major databases. Seven studies met the inclusion criteria, comprising randomized controlled trials, observational cohorts, and in vitro analyses. Quality appraisal was performed using RoB2, Newcastle–Ottawa, and SYRCLE tools.

Results

Stem cell therapy was associated with improvements in right ventricular ejection fraction, somatic growth, heart failure status, and psychosocial quality of life. CDCs showed favorable outcomes in both early and long-term follow-up, with reductions in scar burden, ventricular volume, and adverse events. Importantly, no major adverse effects such as arrhythmias, systemic infections, or tumor formation were reported. The use of allogeneic cells could extend beyond HLHS to other forms of congenital heart disease with right ventricular dysfunction, including tetralogy of Fallot, pulmonary atresia, and other single-ventricle variants. In vitro studies confirmed the regenerative capacity of pediatric-derived stem cells, with the highest potential in the first year of life.

Conclusion

Stem cell therapy demonstrates safety, feasibility, and potential efficacy in improving cardiac outcomes in HLHS. Larger, multicenter trials such as the APOLLON trial are warranted to validate these findings and support integration into standard clinical care.