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Meta-Analysis
. 2019 Nov;39(11):2136-2152.
doi: 10.1111/liv.14179. Epub 2019 Jul 10.

Comparative effectiveness of common therapies for Wilson disease: A systematic review and meta-analysis of controlled studies

Christian Appenzeller-Herzog  1 , Tim Mathes  2 , Marlies L S Heeres  3 , Karl Heinz Weiss  4 , Roderick H J Houwen  3 , Hannah Ewald  1   5
Affiliations
Meta-Analysis

Comparative effectiveness of common therapies for Wilson disease: A systematic review and meta-analysis of controlled studies

Christian Appenzeller-Herzog et al. Liver Int. 2019 Nov.

Abstract

Background & aims: Wilson disease (WD) is a rare disorder of copper metabolism. The objective of this systematic review was to determine the comparative effectiveness and safety of common treatments of WD.

Methods: We included WD patients of any age or stage and the study drugs D-penicillamine, zinc salts, trientine and tetrathiomolybdate. The control could be placebo, no treatment or any other treatment. We included prospective, retrospective, randomized and non-randomized studies. We searched Medline and Embase via Ovid, the Cochrane Central Register of Controlled Trials, and screened reference lists of included articles. Where possible, we applied random-effects meta-analyses.

Results: The 23 included studies reported on 2055 patients and mostly compared D-penicillamine to no treatment, zinc, trientine or succimer. One study compared tetrathiomolybdate and trientine. Post-decoppering maintenance therapy was addressed in one study only. Eleven of 23 studies were of low quality. When compared to no treatment, D-penicillamine was associated with a lower mortality (odds ratio 0.013; 95% CI 0.0010 to 0.17). When compared to zinc, there was no association with mortality (odds ratio 0.73; 95% CI 0.16 to 3.40) and prevention or amelioration of clinical symptoms (odds ratio 0.84; 95% CI 0.48 to 1.48). Conversely, D-penicillamine may have a greater impact on side effects and treatment discontinuations than zinc.

Conclusions: There are some indications that zinc is safer than D-penicillamine therapy while being similarly effective in preventing or reducing hepatic or neurological WD symptoms. Study quality was low warranting cautious interpretation of our findings.

Keywords: Wilson disease; hepatolenticular degeneration; meta-analysis; systematic review.

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References

REFERENCES

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