The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes

Rainer B Zotz, Man-Chiu Poon, Giovanni Di Minno, Roseline D'Oiron, Glanzmann Thrombasthenia Registry Investigators, Rainer B Zotz, Man-Chiu Poon, Giovanni Di Minno, Roseline D'Oiron, Glanzmann Thrombasthenia Registry Investigators

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 included in the registry. Results Between 2007 and 2011, 27 children were treated for 44 surgical procedures (minor: 36; major: 8); nonsurgical bleeds occurred in 104 patients (599 episodes: severe, 145; moderate, 454; spontaneous, 423; posttraumatic, 176). The effectiveness of treatment for minor procedures, major procedures, nonsurgical bleeds was 6/6, 1/1, and 75/84 for rFVIIa, 6/6, 2/2, and 64/76 for rFVIIa + antifibrinolytics (AF), 11/12, 1/1, and 162/214 for platelets ± AF, and 5/6, 0/3, and 33/45 for rFVIIa + platelets ± AF. In all, 25 adverse events were reported in children; no thromboembolic events were reported. Conclusion For all patients, regardless of platelet antibody or refractoriness status, rFVIIa, administered with or without platelets (± AF), provided effective hemostasis with a low frequency of adverse events in surgical, as well as nonsurgical, bleeding in patients with GT. clinicaltrials.gov identifier: NCT01476423.

Keywords: Glanzmann thrombasthenia; autosomal dominant; observational study; pediatric; recombinant activated factor VII; registry.

Conflict of interest statement

Conflict of Interest R. B. Zotz has been a speaker for AstraZeneca, Bayer, Biotest, Boehringer Ingelheim, CSL Behring, GlaxoSmithKline, LEO Pharma, MEDA, Novartis, Novo Nordisk, Octapharma, Pfizer, Sanofi Aventis, and Wyeth, and received fees from Biotest, CSL Behring, GlaxoSmithKline, Novartis, Sanofi Aventis, and Wyeth. He is a member of the advisory boards at Bayer, Novo Nordisk, and Pfizer. He received research funding from CSL Behring. M.-C. Poon was chair of Novo Nordisk's expert panel on the GTR, has been an ad hoc speaker for Bayer, Novo Nordisk, and Pfizer, and attended advisory board meetings of Bioverativ/Biogen Idec, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Roche and Shire/Baxter, and received grant funding from Bayer and CSL Behring. G. Di Minno has been a speaker or a member of a speakers' bureau for Bayer, Biotest, Boehringer Ingelheim, Grifols, Novo Nordisk, Pfizer, and Sanofi Aventis, and has also acted as a consultant or ad hoc speaker/consultant for Bayer, Biotest, Boehringer Ingelheim, CSL Behring, Eli Lilly, Grifols, Novo Nordisk, Pfizer, and Sanofi Aventis. R. d'Oiron received fees or honoraria for attending advisory boards or speaking at symposia for Baxter, Bayer, CSL Behring, Novo Nordisk, Pfizer, and SOBI.

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