Promising Tomorrow: Transplant Research Translating into Hope
Kidney transplant recipient Jacob Bottone,
with his stuffed animal.
When a critically ill child faces the possibility of an organ transplant, their carefree world turns upside down - their childhood on hold as they wait for an organ donation, prepare for major surgery and face a stringent life-long medication regimen.
The heart, liver and kidney transplant programs at Children's Hospital Colorado, in partnership with the University of Colorado School of Medicine, give these patients hope and the courage to face an organ transplantation.
Pediatric patients requiring a liver transplant often suffer from biliary atresia - a bile duct blockage that causes a damaging build-up of bile in the liver.
For the past decade, Cara Mack, MD, associate professor at the school of medicine, has conducted National Institutes of Health-funded studies of the mechanisms of bile duct injury. Her basic research aims to define the immunologic pathways involved in bile duct and liver injury. Dr. Mack is also investigating how genetics influence a child's susceptibility to biliary atresia.
Dr. Mack and her colleagues conduct research within Children's Hospital Colorado Liver Center, the region's only provider of complete pediatric multi-specialty liver transplant services.
"We have a great collaborative program with open discussion and participation, including the patients and families," reflected Michael Narkewicz, medical director of Children's Liver Center and the Hewit/Andrews Chair in Pediatrics Liver Diseases. "They're one of the best teams I've worked with. Everyone is dedicated to achieving the best outcomes for patients and families."
Patients living with renal disease and end-stage renal failure depend on Children's Hospital Colorado Kidney Center- the region's only pediatric dialysis center - where patients receive artificial blood filtration, a process normally performed by healthy kidneys.
Children living with autosomal dominant polycystic kidney disease (ADPKD) - a very common disorder characterized by progressive kidney cyst development often resulting in kidney failure in adulthood - will benefit from the work of pediatric nephrology associate professor Melissa Cadnapaphornchai, MD.
As director of pediatric research at the University of Colorado's Polycystic Kidney Disease Research Group, Dr. Cadnapaphornchai spearheads the only clinical trial being performed in children with ADPKD in the United States.
Participants nation-wide come to Children's where Dr. Cadnapaphornchai's research has the potential to markedly impact outcomes in these patients. "We are looking for early pharmacologic interventions which might alter the course of the disease and slow the progression of renal failure," she explained.
Last year, 13 patients received new hearts and thus a renewed life, thanks to Children's Hospital Colorado Cardiac Transplant Program. Transplant coordinator Dee Dee Gilbert, CPNP, attributes the program's success to the full spectrum of care Children's offers cardiac patients.
"We have access to other great sub-specialties, and we can easily get hold of another care team who accesses the same electronic health record," Gilbert noted. "Our teams are cohesive, everyone knows each other, and we are working toward the same goal and the success of these patients."
Currently, Children's is a major participant in the North American FDA trial of the Berlin Heart - a cardiac device that uses compressed air to help pump blood while the patient's heart rests as he or she awaits a transplant.
"Currently, there are no FDA-approved, long-term cardiac assist devices capable of bridging children with severe heart failure to cardiac recovery or heart transplantation," explained cardiothoracic surgeon Max Mitchell, MD. "Participation allows us to offer the most advanced pediatric cardiac care available in this region, and provides hope to patients who otherwise have no options for survival."
Options including leading therapies, innovative techniques and access to leading experts give hope to transplant patients. And their families place trust in the hands of Children's renowned teams who are ready to make the miracle of a brighter tomorrow a reality today.