The Weill Cornell Medicine Pediatric Nephrology team focuses on a wide range of research activities to expand the understanding of renal and other nephrology disorders in children and adolescents, and to develop new strategies for the prevention and management of renal disease and injury. We continue to break new ground in our understanding of conditions including chronic kidney disease, kidney transplant outcomes and hemolytic uremic syndrome.
Chronic Kidney Disease (CKD) in Children - Clinical Research
The Division of Pediatric Nephrology at Weill Cornell has a robust chronic kidney disease (CKD) research program. Our current CKD-related clinical studies are led by Oleh M. Akchurin, MD, PhD, and include:
- Clinical trial of oral iron therapy in children with CKD (ClinicalTrials.gov Identifier: NCT03991169. Primary investigator: O. Akchurin, M.D., Ph.D.) This is an investigator-initiated clinical trial that allows children with CKD to benefit from novel approaches to iron therapy which may reduce potential side effects of this treatment. The trial is sponsored by the NIH through the Children's Hospital of Philadelphia Pediatric Center of Excellence in Nephrology and Weill Cornell Medicine Clinical and Translational Science Center.
- Growth hormone therapy and patient-reported outcomes in children with CKD: Pediatric Nephrology Research Consortium Study (Primary investigator: O. Akchurin, M.D., Ph.D.). In this multicenter observational study, using Patient-Reported Outcomes Measurement Information System (PROMIS), and other investigational tools, we are evaluating the perception of children and their parents on the changes in quality of life and treatment satisfaction following growth hormone therapy.
- Growth, inflammation and anemia in children with CKD (Primary investigator: O. Akchurin, M.D., Ph.D.). In this study, we have established a biorepository of blood and urine samples generously provided by our patients with CKD and healthy children, as well as a comprehensive research database that includes their demographic and clinical characteristics.
In addition to these studies led by Weill Cornell investigators, we collaborate with the Chronic Kidney Disease in Children (CKiD) study, the North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) registry, and several industry-sponsored clinical trials.
Mineral Metabolism in Children with Chronic Kidney Disease (CKD)
Juhi Kumar, M.D., M.P.H. was the recipient of a career development award (K 23) from the NIDDK, to evaluate the prevalence and correlates of vitamin D deficiency in the CKiD cohort. She continues to utilize the CKiD database to evaluate the associations between vitamin D and growth, and vitamin D and blood pressure in children with CKD.
Kidney Transplant Outcomes and Non-Invasive Methods to Monitor Allograft Status
Juhi Kumar, M.D., M.P.H. is the recipient of a CTSC pilot grant to evaluate markers of allograft damage in blood and urine, as well as the gut microbiome, in pediatric kidney transplant recipients both with and without donor-specific antibodies. In addition, she will explore the relationship of these markers and the gut microbiome with vitamin D and Fibroblast Growth Factor 23 (FGF23).
Effects of Iron Therapy on Renal Fibrosis and Bone Health in Juvenile Chronic Kidney Disease (CKD) – Preclinical Research
With the support of his K08 career development award from the NIH NIDDK and Rohr family Clinical Scholar award, Dr. Oleh Akchurin is using several mouse models of juvenile Chronic Kidney Disease (CKD) to understand how iron supplementation affects renal fibrosis and bone health in CKD. Iron supplementation is currently the most common therapy to treat anemia in children with mild to moderate CKD. Dr. Akchurin’s preliminary animal data demonstrated that oral iron therapy, in the doses compatible with those used in children with CKD improved anemia, but aggravated renal fibrosis in mice with adenine-induced CKD and worsened bone disease in these mice. The mechanism of these pro-fibrotic and bone effects of oral iron therapy could be related to the alterations of hepcidin, the master regulator of iron metabolism, in juvenile CKD. Previous research of Dr. Akchurin demonstrated that genetic blockade of hepcidin gene transcription not only ameliorated anemia in juvenile mice with CKD without a need for exogenous iron therapy, but also improved skeletal growth of these mice [See: http://vivo.med.cornell.edu/display/pubid84994609041]. Similarly, Dr. Akchurin have shown that the blockade of interleukin 6, a known upstream activator of hepcidin, was also protective against anemia in juvenile CKD [See: http://vivo.med.cornell.edu/display/pubid85061988121]. All these data have direct translational significance with respect to human Chronic Kidney Disease (CKD).
Atypical Hemolytic Uremic Syndrome (HUS) Study and Registry
Eduardo Perelstein, M.D., M.P.H. is the site principal investigator for an observational, multicenter study analyzing patients with atypical Hemolytic Uremic Syndrome (HUS), sponsored by Alexion Pharmaceuticals.
Kidney Transplant Research: Validating Injury in the Renal Transplant Using Urinary Signatures (VIRTUUS study)
Juhi Kumar, M.D., M.P.H. along with colleagues at the Children’s Hospital of Philadelphia (Drs. Amaral and Keating), leads one of the largest transplant studies in pediatric kidney transplant recipients in North America funded by a R01 grant from the National Institute of Child Health and Human Development (NICHD). The VIRTUUS study is a multi-center cohort study designed to improve approaches to identifying and characterizing early allograft injury in children with kidney transplants. This study aims to validate in children, urinary messenger RNA signatures and metabolite profiles associated with acute cellular rejection, antibody mediated rejection and BK virus nephropathy in adult kidney transplant recipients. The VIRTUUS study is of great relevance for the pediatric transplant recipients as it aims to improve long-term allograft outcomes by shifting clinical paradigms toward proactive, individualized treatment based on urinary changes that can be seen before clinical manifestations arise.
Kidney Transplant Research: North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) Registry
Juhi Kumar, M.D., M.P.H. is the site Principal Investigator for North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS). Our site contributes valuable data from our kidney transplant patients to this registry. This database collects information about current practices and trends in management of kidney transplant recipients with the goal of improving the care of pediatric renal allograft recipients in North America.
Kidney Transplant Research: Urine mRNA Levels and Donor Specific Antibodies
Identifying Biomarkers of Kidney Injury in Neuroblastoma Survivors