The Rational Use of Complement Inhibitors in Kidney Diseases.

However, the magnitude of the clinical improvement was highly significant as compared with historical controls and thus proved sufficient for the approval of this innovative drug for an ultrarare disease.

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To date, aHUS is the only kidney disease in which an enrichment of complement gene variants is clearly significant (30%–60%). 44 Second, the assessment of the pathogenicity of a complement variant is paramount for the interpretation of complement genetics, and only variants with demonstrated or likely pathogenic effect on the function of the encoded proteins are to be taken into account for clinical decision, including the use of complement inhibitors. 44 Finally, constitutional complement dysregulation is only a risk factor for some kidney diseases.

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