Research Collaborative Improves Outcome Prediction in Children With Posterior Urethral Valves

The Pediatric Urology Midwest Alliance (PUMA) recently published the results of its first collaborative study in Pediatrics. Using data from five institutions, the team demonstrated that the risk of renal replacement therapy and recommendation for clean intermittent catheterization increased with a child’s age and that serum nadir creatinine level in the first year of life (SNC1) strongly predicted the need for renal replacement therapy.

“This paper is the first big accomplishment of our collaborative and provides a proof of principle that we can get data that has never been collected before by increasing the numbers through collaboration and showed that certain predictors of outcomes can be successfully tracked for rare diseases, in this case, posterior urethral valves,” says Daryl McLeod, MD, MPH, urologist at Nationwide Children’s Hospital and lead author of the manuscript.

PUMA includes investigators from the Riley Hospital for Children at Indiana University Health, the Ann and Robert H. Lurie Children’s Hospital of Chicago, the Cincinnati Children’s Hospital Medical Center, Mayo Clinic and Nationwide Children’s Hospital. The collaborative was initiated with the goal of improving the management and investigation of children with rare urologic diseases.

The retrospective cohort study assessed 274 records of male infants who had early (<90 days of life) diagnosis and treatment of posterior urethral valves at one of the five PUMA hospitals from 1995 to 2005 and appears to be the largest population  of infants with posterior urethral valves studied to date.

The investigators were able to estimate the risk of progressing to renal replacement therapy by 10 years of age as 0%, 2%, 27% and 100% for an SNC1 <0.4, an SNC1 of 0.4 to 0.69, an SNC1 of 0.7 to 0.99, and an SNC1 of >1.0 mg/dL, respectively. Approximately one-quarter (26%) of the patients were recommended clean intermittent catheterization by 10 years of age.

“There was a group of patients in the middle between 0.4 and 0.99 who had a much more variable course, and we believe that those are the patients that we may be able to change the natural course of disease through improved family counseling and more tailored screening and intervention strategies,” says Dr. McLeod.

The PUMA team is now working on the development of a Standardized Clinical Assessment and Management Plan (SCAMP) for all babies born with posterior urethral valves at the five centers. The infants will be immediately placed on the standardized care pathway, and outcomes of interest, such as progression to renal failure or infection occurrence, will be periodically analyzed by the investigators, who will then revise the SCAMP based on the latest findings and will repeatedly collect and analyze the patient data and revise the plan accordingly.

“It’s hard to have a successful collaboration like this. It is something that is time consuming. People have to take off of clinical duties to travel to meet with other professionals that are interested in the same topic,” says Dr. McLeod, “I think that one of the main reasons why PUMA has been so successful is that the individual members and the administrations have both been willing to invest and commit to the mission of the collaboration and to look at the greater good and forget about promoting only their own institution.”

Reference:

McLeod DJ, Szymanski KM, Gong E, Granberg C, Reddy P, Sebastião Y, Fuchs M, Gargollo P, Whittam B, VanderBrink BA, Pediatric Urology Midwest Alliance (PUMA). Renal Replacement Therapy and Intermittent Catheterization Risk in Posterior Urethral Valves. Pediatrics. 2019;143(3): e20182656.