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New approaches in treating complicated childhood polycystic kidney disease

A collaborative team of physician-scientists at the Medical College of Wisconsin (MCW) and Children's Hospital of Wisconsin Research Institute has developed a new evidence-based, clinical algorithm to help physicians treat complex patients with autosomal recessive polycystic kidney disease (ARPKD).

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, appears in the April 17 edition of Pediatric Transplantation

ARPKD is a rare genetic disorder that causes progressive disease of the kidneys and liver. Of the patients with ARPKD who survive the first year of life, more than 85 percent will reach their tenth birthday. However, despite dramatic improvements in overall survival and quality of life, nearly 50 percent of those survivors develop end stage 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," said Dr. Avner. "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 authors also emphasize that this therapy only be considered in major pediatric organ transplant centers with experienced pediatric transplant surgeons, subspecialty physicians and nurses, and complete pediatric ancillary services. The commentary also includes a "tree" to help guide clinicians making decisions about the appropriate course of treatment for complications of ARPKD.

About the Medical College of Wisconsin
The Medical College of Wisconsin is the state's only private medical school and health sciences graduate school. Founded in 1893, it is dedicated to leadership and excellence in education, patient care, research and service. More than 1,200 students are enrolled in the Medical College's medical school and graduate school programs. A major national research center, it is the largest research institution in the Milwaukee metro area and second largest in Wisconsin. In FY 2011 – 12, faculty received more than $166 million in external support for research, teaching, training and related purposes, of which more than $152 million is for research. This total includes highly competitive research and training awards from the National Institutes of Health (NIH). Annually, College faculty direct or collaborate on more than 2,000 research studies, including clinical trials. Additionally, more than 1,350 physicians provide care in virtually every specialty of medicine for more than 425,000 patients annually.

About Children's Hospital of Wisconsin

Children's Hospital of Wisconsin is the region's only independent health care system dedicated solely to the health and well-being of children. The hospital, with locations in Milwaukee and Neenah, Wis., is recognized as one of the leading pediatric health care centers in the United States and is ranked No. 4 in the nation by Parents magazine. Children's Hospital provides primary care, specialty care, urgent care, emergency care, community health services, foster and adoption services, child and family counseling, child advocacy services and family resource centers. In 2011, Children's Hospital invested more than $100 million in the community to improve the health status of children through medical care, advocacy, education and pediatric medical research. Children's Hospital achieves its mission in part through donations from individuals, corporations and foundations and is proud to be a member of Children's Miracle Network Hospitals. For more information, visit the website at

Maureen Mack | EurekAlert!
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