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Article

Genetic risk stratification to reduce inhibitor development in the early treatment of hemophilia A: a SIPPET analysis.

Blood - 12 Oct 2017

Rosendaal Frits R, Palla Roberta, Garagiola Isabella, Mannucci Pier M, Peyvandi Flora

Abstract excerpt

A recent randomized trial, the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET), showed a higher risk of inhibitor development with recombinant factor VIII (rFVIII) than plasma-derived concentrates (pdFVIII). We investigated whether risk stratification by F8 mutation identifies patients who do not suffer this deleterious effect of rFVIII. Among 235 randomized patients with severe hemophilia A...

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Identifiers

PMID: 28768627
DOI: 10.1182/blood-2017-06-791756

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