Partial Heart Transplantation for Pediatric Heart Valve Dysfunction: A Clinical Trial Protocol

Taufiek Konrad Rajab, Brielle Ochoa, Kasparas Zilinskas, Jennie Kwon, Carolyn L. Taylor, Heather T. Henderson, Andrew J. Savage, Minoo Kavarana, Joseph W. Turek, John M. Costello

Congenital heart defects are the most common type of birth defects in humans and frequently involve heart valve dysfunction. The current treatment for unrepairable heart valves involves valve replacement with an implant, Ross pulmonary autotransplantation, or conventional orthotopic heart transplantation. Although these treatments are appropriate for older children and adults, they do not result in the same efficacy and durability in infants and young children for several reasons.

Congenital heart defects are the most common type of birth defects in humans. In North America, 7 in 1000 live born children are affected. Worldwide, this causes over 180,000 infant deaths per year. Treatment of congenital heart defects frequently involves heart valve replacement. However, infants and young children who undergo valve replacements uniformly outgrow their valve implants, which always become dysfunctional over time due to the development of stenosis, regurgitation, or a combination of the two.

Materials and Methods:

A prospective nonrandomized, single-center, single arm pilot trial will be performed in infants and young children who require semilunar heart valve replacement. The study received approval from the Medical University of South Carolina (MUSC) Institutional Review Board Protocol Record 00114653 and the protocol was registered and approved on the ClinicalTrials.gov platform, trial number NCT05372757.

Partial heart transplantation for valve disorders is innovative because it is new and substantially different from current approaches and opens new horizons for the management of infants and young children who require heart valve replacements. This contribution is significant because infants and young children with irreparable valves will be spared morbidity from successive implant exchanges and, in the case of mechanical valve replacement, from therapeutic anticoagulation.

Citation: Rajab TK, Ochoa B, Zilinskas K, Kwon J, Taylor CL, Henderson HT, et al. (2023) Partial heart transplantation for pediatric heart valve dysfunction: A clinical trial protocol. PLoS ONE 18(2): e0280163. https://doi.org/10.1371/journal.pone.0280163

Editor: Jaimin R. Trivedi, University of Louisville, UNITED STATES

Received: September 2, 2022; Accepted: December 20, 2022; Published: February 7, 2023.

Copyright: © 2023 Rajab et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Data Availability: No datasets were generated or analysed during the current study. All relevant data from this study will be made available upon study completion.

Funding: This research was supported by grants to TKR from the American Association of Thoracic Surgery, the Brett Boyer Foundation, the Children’s Excellence Fund held by the Department of Pediatrics at the Medical University of South Carolina, the Saving tiny Hearts Society, the Emerson Rose Heart Foundation, the South Carolina Clinical & Translational Research Institute (NIH/NCATS Grant Number UL1 TR001450), and Philanthropy by Senator Paul Campbell. The funders did not and will not have a role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.

Competing interests: The authors have declared that no competing interests exist.