HLHS Consortium Gives Hope to Babies with Rare Congenital Heart Defect

By Jen Schutz

Hypoplastic Left Heart Syndrome (HLHS) is a rare disease that affects approximately 1 of every 4,300 babies every year. HLHS is a congenital heart defect in which the left side of the heart is underdeveloped. It occurs during fetal growth when the baby's heart is developing. Without immediate intervention after birth, 95% of infants with HLHS will die within a few weeks.[i]

TheTodd and Karen Wanek Family Program for HLHS at Mayo Clinic is the catalyst that brings together hospitals to address this serious health condition. Together they’ve formed a national consortium to give patients more options when it comes to participating in innovative clinical trials and other HLHS research. This consortium, consisting of eight members, aligns regional centers and an advocacy group into a collaboration to accelerate innovation and discovery sciences, as well as bring clinical trials and expertise to patients across the country. The Ochsner Hospital for Children in New Orleans, Louisiana, is the latest to join the HLHS Consortium.

All consortium members are participating in a phase II clinical trial using stem cells from a baby's own umbilical cord blood in regenerative therapy. During the second of three surgeries to repair the heart, stem cells are injected into the heart muscle to help it grow stronger, with the goal to delay or prevent the need for transplant. The trial is open at all consortium sites, and Ochsner Hospital for Children already has collected cord blood with stem cells waiting to be used for a patient when the trial opens there later this year.