Cardiomyopathies pose a significant risk of morbidity and mortality worldwide, with dilated cardiomyopathy (DCM) recognized as the leading cause of pediatric heart transplantation. Understanding the unique presentation of DCM in the pediatric population remains challenging due to difficulties in classification and the variability in disease progression. Recent advancements in genetic analysis, advanced imaging, and electrophysiological assessments have played a crucial role in refining risk stratification, enabling more personalized treatment strategies. While existing therapies have improved outcomes, emerging treatments such as gene therapy and precision medicine in combination with the use of artificial intelligence show promising potential. Furthermore, transitional care, structured exercise recommendations, and early integration of palliative care are essential for optimizing survival and quality of life in these patients. By integrating these advancements, clinicians can more accurately predict disease progression, optimize individualized treatment plans, and ultimately improve long-term outcomes. This review summarises recent progress in the field offering clinicians valuable insights into how they can refine their risk assessment strategies and tailor treatment protocols to pediatric patients with DCM.<Table Float="No" ID="Taba"> <tgroup cols="2"> <colspec align="left" colname="c1" colnum="1" /> <colspec align="left" colname="c2" colnum="2" /> <tbody> <row> <entry align="left" nameend="c2" namest="c1"> What is Known: • Dilated cardiomyopathy (DCM) is the most common form of cardiomyopathy in children and the leading indication for pediatric heart transplantation. • Pediatric DCM presents with diverse etiologies and clinical trajectories, making diagnosis, risk stratification and management particularly challenging. </entry> </row> <row> <entry align="left" nameend="c2" namest="c1"> What is New: • Recent advancements in genetic testing, imaging technologies, and biomarker profiling are enhancing early diagnosis and personalized risk assessment in pediatric DCM. • Innovative strategies such as gene therapy, precision medicine, and structured transitional care, are enabling more proactive, tailored management strategies and improving long-term outcomes. </entry> </row> </tbody> </tgroup> </Table>

Advancements and challenges in pediatric dilated cardiomyopathy: a comprehensive review of current approaches and future directions

期刊论文
发表日期：

2025年8月12日

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José Luis López-Guillén,
Roger Esmel-Vilomara,
Iulia Rodoman,
Andreia Duarte Constante,
Scott Kendall,
Andrea Freixa-Benavente,
Daniel Diaz-Gil,
Nicoletta Cantarutti,
Sara Moscatelli,
Aamir Jeewa

摘要

Cardiomyopathies pose a significant risk of morbidity and mortality worldwide, with dilated cardiomyopathy (DCM) recognized as the leading cause of pediatric heart transplantation. Understanding the unique presentation of DCM in the pediatric population remains challenging due to difficulties in classification and the variability in disease progression. Recent advancements in genetic analysis, advanced imaging, and electrophysiological assessments have played a crucial role in refining risk stratification, enabling more personalized treatment strategies. While existing therapies have improved outcomes, emerging treatments such as gene therapy and precision medicine in combination with the use of artificial intelligence show promising potential. Furthermore, transitional care, structured exercise recommendations, and early integration of palliative care are essential for optimizing survival and quality of life in these patients. By integrating these advancements, clinicians can more accurately predict disease progression, optimize individualized treatment plans, and ultimately improve long-term outcomes. This review summarises recent progress in the field offering clinicians valuable insights into how they can refine their risk assessment strategies and tailor treatment protocols to pediatric patients with DCM.

What is Known:

• Dilated cardiomyopathy (DCM) is the most common form of cardiomyopathy in children and the leading indication for pediatric heart transplantation.

• Pediatric DCM presents with diverse etiologies and clinical trajectories, making diagnosis, risk stratification and management particularly challenging.

What is New:

• Recent advancements in genetic testing, imaging technologies, and biomarker profiling are enhancing early diagnosis and personalized risk assessment in pediatric DCM.

• Innovative strategies such as gene therapy, precision medicine, and structured transitional care, are enabling more proactive, tailored management strategies and improving long-term outcomes.