Background: In severe haemophilia A with inhibitors, emicizumab has emerged as an effective prophylactic treatment for prevention of bleeding.

Meanwhile, patients with renal failure were excluded from the clinical trials, and there are limited experience with emicizumab in chronic renal failure and haemodialysis. Furthermore, it has been speculated that renal failure with proteinuria could affect emicizumab plasma levels.

Aims: We report emicizumab plasma levels and clinical efficacy of continued emicizumab prophylaxis in a case of severe haemophilia A with inhibitors and progressive renal failure with substantial proteinuria due to AA-amyloidosis , developing end-stage renal disease necessitating chronic intermittent haemodialysis.

Methods: Emicizumab plasma levels (Haemochrom Diagnostica), urinary protein and urine output were measured regularly in the two years leading up to haemodialysis. Emicizumab plasma levels were measured before and after the dialysis procedure on four occassions. Bleeding episodes and treatment with by-passing agents were recorded, as a measure of emicizumab efficacy.

Results: Emicizumab levels remained stable during progression of renal disease and haemodialysis, with mean level 70 mg/l (95% CI 64-76) versus 72 mg/l (95% CI 68-75) before and after initiation of dialysis. Marked proteinuria did not affect emicizumab levels in this case. Bleeding pattern did not change, and break through bleeds were succesfully treated with recombinant FVIIa in similiar doses as before haemodialysis.

Conclusion(s): Emicizumab in standard dosing remained stable and effective as prophylaxis during progressive renal failure and haemodialysis

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ISTH Congress Abstracts - https://abstracts.isth.org/abstract/emicizumab-in-haemophilia-a-with-inhibitors-and-end-stage-renal-disease/