The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes.

ABSTRACT: Background  Standard treatment for Glanzmann thrombasthenia (GT), a severe inherited bleeding disorder, is platelet transfusion. Recombinant activated factor VII (rFVIIa) is reported to be effective in GT with platelet antibodies and/or refractoriness to platelet transfusions. Methods  We evaluated rFVIIa effectiveness and safety for the treatment and prevention of surgical and nonsurgical bleeding in children <18 years old, with or without platelet antibodies and/or refractoriness, as reported in the GT Registry (GTR). Data were used from the GTR, an international, multicenter, observational, postmarketing study of rFVIIa that prospectively collected data on the treatment and outcomes of bleeds in patients with GT. Only patients with a diagnosis of congenital GT were include

SUBMITTER: Zotz RB 

PROVIDER: S-EPMC6742499 | biostudies-literature | 2019 Jul

REPOSITORIES: biostudies-literature