Prognostic scoring indices in Wilson disease: a case series and cautionary tale

In children with acute presentations of Wilson disease (WD), liver transplantation may be the only effective therapy. The Wilson Index is a prognostic index used to determine the risk of death without transplant in WD. We sought to determine the accuracy of this system in our own patient population.

The clinical course of patients diagnosed as having acute WD seen at the Children's Hospital of Pittsburgh of the University of Pittsburgh Medical Center between 2003 and 2008 was reviewed.

Six patients were identified; their index scores ranged from 7 to 13, with 3 patients receiving a score predictive of death without transplantation (≥ 10). Of those 3, 1 underwent transplantation and 2 survived without transplant. The latter 2 have been removed from the transplant waitlist. In all, 5 patients were listed for transplantation, and 2 of the 5 received prioritized status 1A listing. Only 2 of the 5 patients went to transplantation, and neither was status 1A at the time of transplant.

Prognostic scoring systems, although useful, may not be entirely accurate. Likewise, aggressive utilization of status 1 prioritization may result in unnecessary transplants and misallocation of a rare resource. However, deferring status 1 prioritization may yield an incomplete response to therapy and preclude lifesaving transplantation. Continued investigation of predictors of outcome in WD is necessary.