Liver transplantation in patients with neurological Wilson disease: What can a five-decade systematic literature review teach us?

Background

Neurological worsening occurs in up to 20 % of patients with Wilson disease and neurological involvement (neuroWD) despite optimal anti‑copper therapy. This study aimed to analyze the neurological outcomes of patients with neuroWD who underwent liver transplantation (LT) as a rescue therapy for neurological deterioration (Brain group), and to compare them with those who underwent LT for end-stage liver disease (ESLD) (Liver group).

Methods

A systematic PubMed search identified studies on neuroWD and LT published from January 1973 to January 2024.

Results

A total of 368 patients with neuroWD were identified, including 89 and 279 in the Brain and Liver groups, respectively. Post-LT survival rates were similar between groups (82 % vs. 86.6 %). Sepsis was the primary cause of death in both groups (68.7 % in the Brain group vs. 57.1 % in the Liver group). Among survivors beyond 1 year, 86.3 % in the Brain group and 79.7 % in the Liver group showed improvement or complete recovery from neuroWD. De novo post-LT neurological complications and calcineurin inhibitors-induced neurotoxicity were reported exclusively in the Liver group (p < 0.05).

Conclusion

LT is a viable option for neuroWD unresponsive to medical therapy. Delayed introduction or early minimization of calcineurin inhibitors is recommended for ESLD patients to reduce neurotoxicity. Further studies are needed to confirm these observations, as there are currently no evidence-based medicine criteria or standardized inclusion scales for LT in cases of neuro-WD.