SUMMARY
Cheryl L. Tran, M.D., has a special research interest in nephrotic syndrome (NS), and is a co-investigator in a multicenter drug trial investigating adrenocorticotropic hormone (ACTH) therapy in pediatric patients with steroid-dependent and frequently relapsing NS. Dr. Tran is involved in various multicenter research studies including Nephrotic Syndrome Study Network (NEPTUNE) and Cure Glomerulonephropathy Network (CureGN), examining membranous nephropathy, minimal change disease and focal segmental glomerulosclerosis (FSGS/MCD) through clinical, biorepository and drug trial research.
Focus areas
- Immunization practices in children with NS. This survey study aims to determine the current immunization recommendations of pediatric nephrologists in the Midwest Pediatric Nephrology Consortium. Specifically, the proportion that routinely provides vaccination education to families with children who have NS and the proportion that modifies vaccine guidelines for patients with NS. The goal of the survey is to examine the immunization education and knowledge in families with NS.
- Atlantis: A trial of ACTH in NS. This industry-funded phase III trial examines the effectiveness of ACTH in the treatment of children with steroid-dependent NS or frequently relapsing NS.
- NEPTUNE. This study aims to determine the rates and predictors of clinical remission of FSGS/MCD and to identify gene expression profiles from renal biopsies of participants with FSGS/MCD that can be used to classify participants into distinct molecular subgroups.
- Evaluating childhood cardiovascular health outcomes in NEPTUNE (ECHO NEPTUNE). ECHO NEPTUNE will determine the prevalence of risk factors among children with NS enrolled in the NEPTUNE cohort using echocardiogram, 24-hour ambulatory blood pressure monitor and carotid intimal thickness.
- Cure Glomerulonephropathy Network (CureGN). This is a longitudinal database of patients with glomerular diseases, in which a standardized collection of biospecimens is performed at scheduled visits. The goal of CureGN is the development of translational and clinical ancillary studies that will advance the diagnosis and care of patients with glomerular diseases.
- Bovine serum albumin (BSA)-related Idiopathic membranous nephropathy. Dr. Tran is examining children with idiopathic membranous nephropathy and investigating the involvement of cationic BSA and its relationship to the child's clinical presentation and past medical history including dietary history, family history and clinical course.
- Assessment of risky behavior in teens with CKD. This assessment characterizes risky behavior in teens on dialysis or who have chronic kidney disease (CKD). The goal is to determine prevalence of risky behavior in teens on dialysis or who have CKD. Specific risky behaviors unique to this patient population are compared to their healthy peers.
- A pilot study to Assess the Efficacy of Rituximab Therapy in Patients with Treatment Resistant Idiopathic Focal Segmental Glomerulosclerosis (FSGS). This pilot trial assesses the safety, feasibility and efficacy of rituximab in adult and pediatric patients with either steroid or calcineurin inhibitor resistant FSGS or with an intolerance or contraindication to use of these agents.
- Utility of timely ADAMTS13 activity assay in management of thrombotic microangiopathy (TMA). Dr. Tran assesses the utility of timely availability of ADAMTS13 activity results and its clinical use in evaluation of patients with TMA and the effect on guiding initiation of appropriate therapy and interruption of TPE, which would presumably reduce mortality, morbidity and cost.
- Eculizumab use in pediatric practice. This retrospective review focuses on disease spectrum, dosing strategy, duration of treatment and outcomes. The goal is to describe the clinical indications for use of eculizumab and to describe patient characteristics of children and young adults currently being treated with eculizumab.
- Risk factors for complications in children hospitalized with NS. Dr. Tran hopes to determine the incidence and risk factors of acute kidney injury in children hospitalized with NS, and to identify the proportion of childhood NS-associated thromboembolisms that occurs in the presence and absence of co-morbid infections.
Significance to patient care
There is a continued need for large-scale pediatric studies in the field of pediatric nephrology. Participation in the multicenter NS research studies noted above will help pediatric nephrologists learn more about the disease, including gene expression profiles, risk factors, comorbidities, predictors of treatment response and outcomes. Dr. Tran hopes the data from her research will help pediatric nephrologists find novel therapies, including nonimmunomodulating therapies, to better treat NS and improve long-term outcomes. Participation in drug-trial studies, such as the ACTH trial in pediatric patients with steroid-dependent and frequently relapsing NS, offers patients additional treatment options that might otherwise be unavailable.
Professional highlights
- Ad hoc reviewer, JAMA Pediatrics, 2013 and 2015