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CC BY 4.0 · TH Open 2019; 03(03): e286-e294
DOI: 10.1055/s-0039-1696657
Original Article
Georg Thieme Verlag KG Stuttgart · New York

The International Prospective Glanzmann Thrombasthenia Registry: Pediatric Treatment and Outcomes

Rainer B. Zotz
1   Institute for Laboratory Medicine, Blood Coagulation and Transfusion Medicine (LBT), Düsseldorf, Germany
2   Department of Hemostasis, Hemotherapy and Transfusion Medicine, Heinrich Heine University Medical Centre, Düsseldorf, Germany
,
Man-Chiu Poon
3   Departments of Medicine, Pediatrics and Oncology, University of Calgary, Calgary, Alberta, Canada
4   Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Foothills Medical Centre, Calgary, Alberta, Canada
,
Giovanni Di Minno
5   Department of Clinical Medicine and Surgery, Regional Reference Center for Coagulation Disorders, Federico II University, Naples, Italy
,
Roseline D'Oiron
6   Center for Hemophilia and Rare Congenital Bleeding Disorders, University Hospitals Paris-Sud, AP-HP, Bicêtre Hospital, Le Kremlin-Bicêtre, France
,
for the Glanzmann Thrombasthenia Registry Investigators› Author Affiliations
Further Information

Publication History

04 February 2019

25 July 2019

Publication Date:
12 September 2019 (online)

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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

recombinant activated factor VII - Glanzmann thrombasthenia - autosomal dominant - pediatric - registry - observational study

Authors' Contributions

R. B. Zotz drafted, reviewed, and approved the article. M.-C. Poon, G. Di Minno, and R. d'Oiron reviewed and approved the article content.


Data Sharing Statement

The trial sponsor's policy on data sharing may be found at https://www.novonordisk-trials.com/how-access-clinical-trial-datasets.


Supplementary Material