Cardiomyopathy as indication for pediatric heart transplantation

Alireza Raissadati1, Drishti Tolani2, Elizabeth L Profita1

  • 1Department of Pediatrics (Cardiology), Stanford University, Palo Alto, California.

JHLT Open
|August 22, 2025
PubMed

Insights

Pediatric cardiomyopathy (CMP) is a serious heart condition in children, often leading to heart failure and the need for transplantation. Ventricular assist devices (VADs) improve survival rates for these young patients.

Area of Science:

  • Pediatric Cardiology
  • Cardiovascular Research
  • Transplantation Medicine

Background:

  • Pediatric cardiomyopathy (CMP) affects 1 in 100,000 children, frequently progressing to end-stage heart failure.
  • CMP is the leading cause for pediatric heart transplantation, necessitating careful management of etiologies and listing criteria.
  • Navigating waitlist complexities and understanding specific CMP subtypes are crucial for successful outcomes.

Purpose of the Study:

  • To provide a comprehensive review of pediatric cardiomyopathy progressing to end-stage heart failure requiring transplantation.
  • To synthesize current knowledge on CMP etiologies, clinical presentations, and transplant evaluation.
  • To highlight the impact of ventricular assist devices (VADs) and contemporary post-transplant management.

Main Methods:

  • Literature review synthesizing current knowledge on pediatric cardiomyopathy and heart transplantation.
  • Analysis of etiologies, clinical presentations, and transplant evaluation strategies.
  • Examination of the role of mechanical circulatory support, specifically VADs, in bridging to transplant.

Main Results:

  • Pediatric CMP is a critical challenge, often leading to heart failure and transplantation.
  • Ventricular assist devices (VADs) have significantly improved survival rates for pediatric heart transplant candidates.
  • Post-transplant outcomes show continuous improvement with refined long-term management strategies.

Conclusions:

  • This review offers an updated framework for clinicians managing pediatric cardiomyopathy and heart failure.
  • Optimizing decision-making for transplant evaluation and VAD use is essential for this high-risk population.
  • Continued advancements in post-transplant care are improving survival and quality of life.

Related Concept Videos

Cardiomyopathy V: Interprofessional Care01:29

Cardiomyopathy V: Interprofessional Care

Managing cardiomyopathy involves addressing underlying or precipitating causes, treating heart failure with medications, and implementing dietary changes and a balanced exercise and rest regimen.Lifestyle ModificationsCardiomyopathy patients should adopt a low-sodium diet to reduce fluid retention and manage heart failure. A personalized exercise and rest plan helps maintain physical fitness without overstraining the heart. Avoiding alcohol and tobacco is essential to prevent further damage to...
33
Cardiomyopathy II: Dilated Cardiomyopathy01:30

Cardiomyopathy II: Dilated Cardiomyopathy

Dilated cardiomyopathy, or DCM, is a progressive myocardial disorder characterized by ventricular chamber dilation and contractile dysfunction.EtiologyVarious factors can cause DCM, including hypertension and heavy alcohol intake, which contribute to the weakening and enlargement of the heart muscle. Viral infections, such as Coxsackievirus B, adenoviruses, and influenza, can lead to DCM by causing inflammation and damage to heart tissue. Certain chemotherapeutic agents, including daunorubicin,...
22
Cardiomyopathy III: Hypertrophic Cardiomyopathy01:29

Cardiomyopathy III: Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy, or HCM, is an autosomal dominant genetic disorder characterized by asymmetric left ventricular hypertrophy without ventricular dilation. It is more common in men and is typically diagnosed in young, athletic adults.EtiologyHCM is primarily genetic and is caused by mutations in genes encoding sarcomeric proteins. Researchers have identified over 1400 mutations across at least 11 different genes. Among these, the most frequently occurring mutations are found in the...
47
Cardiomyopathy I: Introduction and Classification01:25

Cardiomyopathy I: Introduction and Classification

Cardiomyopathy, or CMP, is a group of diseases affecting the myocardial structure, impairing its ability to pump blood effectively. This condition can lead to arrhythmias, heart failure, or sudden cardiac death.Cardiomyopathies are classified into primary and secondary categories:Primary Cardiomyopathy refers to conditions involving only the heart muscle that are often idiopathic (of unknown cause) or genetic. They primarily affect the myocardium without the involvement of other systemic...
51
Cardiomyopathy IV: Restrictive Cardiomyopathy01:29

Cardiomyopathy IV: Restrictive Cardiomyopathy

Restrictive cardiomyopathy (RCM) is a rare heart muscle disease characterized by impaired ventricular filling due to stiffened ventricular walls, leading to significant diastolic dysfunction.EtiologyRestrictive cardiomyopathy can arise from both inherited and acquired diseases, many of which are systemic. It is categorized into four main types: infiltrative, storage, non-infiltrative, and endomyocardial diseases.Infiltrative diseases, such as amyloidosis, lead to RCM by depositing amyloid...
28
Kidney Transplant I: Introduction01:28

Kidney Transplant I: Introduction

A kidney transplant is a surgical approach that involves replacing a non-functioning kidney with a healthy one from a donor. This procedure is often a treatment option for end-stage renal disease (ESRD) patients. The method requires careful recipient selection, including evaluating various medical and psychosocial factors. These criteria vary between transplant centers but generally include assessments of the patient's overall health, adherence to medical recommendations, and lifestyle...
60