New Approaches to Help Treat Childhood Polycystic Kidney Disease

A new study has revealed positive insights into dealing with childhood polycystic kidney disease. A group of researchers from the Medical College of Wisconsin (MCW) and Children's Hospital of Wisconsin Research Institute have developed a new evidence-based, clinical algorithm to help physicians treat complex patients with autosomal recessive polycystic kidney disease.

Their invited manuscript, written by Grzegorz Telega, M.D., associate professor of pediatrics (gastroenterology and hepatology) at MCW and program director of hepatology at Children's Hospital of Wisconsin; David Cronin, II, M.D., Ph.D., professor of surgery and member of the new Transplantation Institute; and Ellis D. Avner, M.D. professor of pediatrics (nephrology) and physiology at MCW, and director of the Multidisciplinary Childhood PKD Program (MCPP) at Children's Hospital of Wisconsin Research Institute.

ARPKD is classified as a rare genetic disorder that can cause progressive diseases of the kidneys and liver. Many patients survive their first year of life, and more than 85 percent will reach their tenth birthday. But few advancements have been made for the disease, and nearly 50 percent of those survivors develop the end stage of kidney disease during that time.

Based on a comprehensive analysis of published medical literature, unique insights generated from the MCPP (established in 2005 and the only such program in the U.S.) and more than 50 years of clinical experience by the authors in treating complex problems in ARPKD patients, an algorithm was developed to guide patient therapy. Of particular note, the authors recommend an innovative approach for a subgroup of ARPKD patients with severe kidney and liver disease: simultaneous kidney and liver transplantation.

"We believe combined liver-kidney transplantation can potentially decrease overall mortality and morbidity in carefully selected ARPKD patients with end stage renal disease and clinically significant congenital hepatic fibrosis," Avner said, according to a press release. "This is a particularly attractive option at our center, given the rapid growth of the joint solid organ transplantation program between Children's Hospital of Wisconsin, Froedtert Hospital, the Medical College of Wisconsin, and the BloodCenter of Wisconsin, directed by Dr. Johnny Hong."

The findings for the study appear in the April 17 edition of Pediatric Transplantation.