Identifying Risk and Improving Access to Transplant in Children With End-Stage Heart Failure

Outcomes research designed to identify risk and disparities in pediatric heart transplant patients

By Shahnawaz Amdani, MD

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Children with end-stage heart failure often need advanced heart failure therapies such as the use of a ventricular assist device (VAD) and heart transplantation. Because the risk of mortality in this patient population is extremely high, a dedicated team of caregivers with experience and expertise is necessary to appropriately manage their care.  

At Cleveland Clinic Children’s, these children are routinely cared for with excellent outcomes, both while they are awaiting a heart transplant and once they have received a transplant. However, significant knowledge gaps in the literature remain with a need to better understand risk factors and transplant outcomes in children with cardiomyopathy and congenital heart disease presenting with advanced heart failure.

Over the last year, my research collaborators and I have published over a dozen studies designed to stratify high-risk groups, improve access to transplants, and examine the implications of transplant center evaluations. The following is a snapshot of this work:

Nomogram to predict mortality after transplant in Fontan patients

Using the Pediatric Heart Transplant Society database, we found that an easily calculated marker of liver and renal dysfunction, the Model for End-stage Liver Disease eXcluding INR (MELD-XI), can help identify Fontan patients who are at increased risk for post-heart transplant mortality. We derived a nomogram to predict one-year post-transplant mortality in Fontan patients based on the MELD-XI score at transplant. This score can be utilized by clinicians caring for such children to have informed conversations with other providers and families.

Read more here.

Risk stratification and management in children with end-stage heart failure undergoing VAD implantation

Leveraging data from the Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) registry, we found that using the MELD-XI score can help risk-stratify children with end-stage heart failure undergoing VAD implantation.

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I was also part of an international group of investigators from the International Society for Heart and Lung Transplantation who helped craft the consensus statement for the selection and management of pediatric patients on VADs.

Additionally, in collaboration with a national team of investigators, we analyzed rates of cerebrovascular accidents (CVA) in pediatric patients undergoing VAD implantation. We showed that rates have significantly decreased; and also identified risk factors that lead to CVA after VAD implantation.

Access to transplant for patients with non-dilated cardiomyopathy

Using data from the Scientific Registry for Transplant Recipients, we found that children with non-dilated cardiomyopathy (hypertrophic and restrictive cardiomyopathy), a group historically at increased risk for worse transplant outcomes, have similar a waitlist and post-heart transplant outcomes compared to children with dilated cardiomyopathy in the current era. This work also helped identify children with non-dilated cardiomyopathy who are at increased risk for waitlist mortality. This work will assist with prioritizing these higher risk non-dilated cardiomyopathy patients who are awaiting heart transplant.

Read more here.

Identifying racial disparities in access to heart transplant

We have also been involved in identifying racial disparities in pediatric heart transplantation. In a study that utilized the national transplant database, we found that African American children have a 25% increased risk for waitlist mortality compared to White children.

Read more here.

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I am also grateful to be a recipient of the Enduring Hearts: Pediatric Heart Transplant Society Health Disparities Research Award, which has enabled a new study: “The Impact of Community Risk Factors on Outcomes for Children Following Heart Transplantation.” This work is currently underway.  

Unintended consequences of flagging low-performance centers

Finally, in a multi-center evaluation, we showed the unintended consequences of publically flagging centers for low center performance and made recommendations for how to improve our existing processes. We found that “flagging” centers leads to risk-averse behavior when listing high-risk children and decreases listing and transplant volumes at such centers. This has the potential to reduce access to lifesaving therapy.

Read more here.

In short, my research goal is to uncover scientific evidence to guide care for some of the sickest children. In addition to the work mentioned above, I am the principal investigator for many national studies—all designed to improve outcomes for children with end-stage heart failure and reduce disparities in health outcomes.

About the Author. Dr. Amdani is Associate Staff in the Department of Pediatric Cardiology. He specializes in the management of pediatric cardiomyopathy, heart failure, mechanical circulatory support devices and heart transplantation.