Pediatric Liver Transplant Resea​rch

The Siragusa Transplant Center is a globally recognized pediatric research center, demonstrating Lurie Children’s decades long dedication to transplantation research in pediatrics. The physician-researchers on our staff have pioneered influential new methods of studying pediatric liver disease and improving transplant outcomes and receive substantial funding from the National Institutes of Health. 

We are enrolling patients in research protocols and clinical trials every day of the year, providing substantial opportunities for your child and family to participate in ground-breaking research that increases the knowledge base of the medical community and improves pediatric care standards. 

And most importantly, at Lurie Children’s, the doctors, nurses and clinical staff members caring for your child are the very same researchers at the forefront of their respective fields. Therefore, you can be assured that your child is getting the most cutting-edge care available. 

Clinical research includes the following:

• Safe minimization and elimination of immunosuppression medications after liver transplantation.
• Health outcomes evaluations for pediatric recipients of liver transplants, including minimization of chronic health problems following transplantation and maximization of quality of life.
• Studies relating to the cause and treatments of biliary atresia.
• Studies relating to the causes and treatment of genetic and metabolic causes of neonatal cholestasis including PFIC, Alagille Syndrome and Alpha one anti trypsin deficiency.
• Studies on mitochondrial liver diseases.
• Epidemiology and treatment of acute liver failure.
• Cause and treatment of gestational alloimmune liver disease (GALD) formerly known as neonatal hemochromatosis.

Laboratory research is a vital component of our strategy to improve the care opportunities and successes of our patients. Ongoing laboratory research includes:

• Studying mechanisms involved in the progression of non-alcoholic fatty liver disease (NAFLD)
• Identifying the target of the immune attack responsible for GALD
• Studying the gene expression profiles of patients with progressive familial intrahepatic cholestasis (PFIC)
• Studying the role of macrophages in cholestatic liver disease, particularly biliary atresia
• The effect of the Unfolded Protein Response in cholestatic liver disease and injury

Our significant investment in, and commitment to clinical and laboratory research allows the Siragusa Transplant Center to lead the way in improving the health of children with liver disease. But we know our ability to succeed is enhanced when we engage in collaborative research with other respected transplant centers and organizations worldwide. Sharing information and keeping apprised of what others are doing to advance the health and outcomes of patients across the country is critical to our mission. Our investment in research, therefore, includes participation in national research studies and collaboration with other pediatric transplant centers. Examples of our efforts to reach out to the research community include:

1. SPLIT: Founding Membership in the Studies of Pediatric Liver Transplant (SPLIT) Registry Network, now renamed Society of Pediatric Liver Transplantation and a chapter of The Transplantation Society, the largest cooperative effort between transplant centers globally designed to prospectively collect and analyze information necessary for a full understanding of the problems and solutions surrounding liver transplantation in children. 
   
   Learn More About SPLIT
   
   
2. ChiLDREN: Participation as a clinical research center in Childhood Liver Disease Research and Education Network (ChiLDREN), a NIH-funded network of 16 clinical sites designed to propose, develop and undertake trials related to biliary atresia; currently the leading indicator for pediatric liver transplants in the United States, and genetically acquired cholestatic diseases such as PFIC. ChiLDREN is a collaborative team of doctors, nurses, research coordinators, and patient support organizations throughout the US and the United Kingdom, working together to improve the lives of children and families dealing with rare cholestatic liver diseases by providing ways for patients to join with doctors and researchers through participation in research studies. The greater the collaboration between doctors and patients and their families, the more we can learn about cholestatic liver diseases. There are several studies within ChiLDREN:
   • PROBE: A prospective study of infants and children with cholestasis
   • BASIC: A prospective database study of older children with biliary atresia
   • LOGIC: A longitudinal study of genetic causes of intrahepatic cholestasis including Alagille Syndrome, alpha-1 antitrypsin deficiency, progressive familial intrahepatic cholestasis, and bile acid synthesis defects both before and after liver transplantation
   • MITOHEP: A longitudinal study of mitochondrial hepatopathies
   Learn more about ChiLDREN
   
   
3. iWITH: Key site member of the NIH sponsored “Immunosuppression Withdrawal for Stable Pediatric Liver Transplant Recipients (iWITH)” study. With the information learned from this study, transplant doctors may be able to identify markers that can be used in the future to identify transplant recipients who may be taken off of their anti-rejection medicine.
   
   
4. PALF: Lead center for NIH sponsored treatment trial for Pediatric Acute Liver Failure (PALF) Treatment For Immune Mediated Pathophysiology (TRIUMPH) This is the first multi-center, multi-national collaborative study aimed at treating PALF
   
   Learn More About PALF
   
   
5. iMALT: Site member of the NIH-funded study titled Improving Medication Adherence in Children Who Had a Liver Transplant (iMALT). Non-adherence is the most important reason for organ rejection in long term survivors of pediatric liver transplantation. In order to address this important risk-factor effectively, the iMALT study will test a tailored telemetric intervention to reduce rejection rates by improving erratic medication adherence (nonadherene) to medications in pediatric liver transplant recipients.
   
   Learn more about iMALT
   
   
6. NASH: Site member of the NIH sponsored Non-alcoholic Steatohepatitis Clinical Research Network (NASH CRN), and includes the collaboration of 9 clinical centers around the country each with a pediatric and adult center. The NASH CRN is a cooperative network designed to propose, develop and undertake trials related to nonalcoholic fatty liver disease (NAFLD).
   
   Learn more about the NASH CRN
   
   
7. Cystic Fibrosis Liver Disease Research (CFF and NIH): A multicenter international study to investigate the risk factors for cirrhosis in cystic fibrosis. This includes:
   1. Prospective Study of Ultrasound to Predict the Development of Cirrhosis in CF (PUSH): A prospective multicenter international study to determine if US can identify children with CF at risk for the development of cirrhosis (CFF and NIH funded). 
   2. PUSH Cohort Analysis using MRI to Characterize and Predict CF Liver Disease: A prospective nested study of a subset of the PUSH cohort seeks to determine the utility of MRI with elastography in the prediction of the risk of children with CF to develop cirrhosis.
8. STARZL: Founding member of STARZL Network of Excellence in Pediatric Transplantation (SNEPT) which unites the top transplant centers in North America in an effort to bring new and lifesaving advances to children needing a transplant by bringing the latest technology, and best minds in the field together with patient advocacy. 
   
   Learn more about STARZL

In addition to external collaboration, the Siragusa Transplant Center collaborates with other divisions within the Lurie Children’s family and across Northwestern Medicine. For example, we partner with the kidney division in improving post transplant communication with families, and with Northwestern Medicine in transitioning patients to the adult hospital.

At the Siragusa Transplant Center, we will continue to pursue research directly related to the improvement of the health of your child. We will do so by continuing our ongoing commitment to institutional clinical and laboratory research coupled with important collaborative efforts in the field. We are relentless in our pursuit of new avenues of research with our own faculty, colleagues around the country, and with parents and caregivers.

Learn more about research studies available nationwide.