Experience of recombinant-activated factor VII to control bleeding in children with Glanzmann’s thrombasthenia

2007 ◽  
Vol 16 (3) ◽  
pp. 167-172 ◽  
Author(s):  
Kate Khair ◽  
Pavel Svirin
Keyword(s):  
Factor Vii ◽  
Recombinant Activated Factor Vii ◽  
Glanzmann’S Thrombasthenia ◽  
Activated Factor Vii ◽  
Glanzmann's Thrombasthenia

scholarly journals The Use of Recombinant Activated Factor VII in Patients with Glanzmann's Thrombasthenia

2020 ◽  
Author(s):  
Man-Chiu Poon
Keyword(s):  
Unmet Need ◽  
Factor Vii ◽  
European Medicines Agency ◽  
Recombinant Activated Factor Vii ◽  
Glanzmann’S Thrombasthenia ◽  
Activated Factor Vii ◽  
Safety Surveillance ◽  
Glanzmann's Thrombasthenia ◽  
Bleeding Episodes ◽  
Surveillance Database

AbstractPlatelet transfusion is the standard treatment to control or prevent bleeding in patients with Glanzmann's thrombasthenia (GT), but platelets are often unavailable. Recombinant activated factor VII (rFVIIa) is an effective alternative to platelets in patients with GT with past/present refractoriness to platelet transfusions and antibodies to platelets. However, there is an unmet need for an alternative to platelets in patients without antibodies. This report summarizes evidence of efficacy and safety of rFVIIa in patients with GT without refractoriness or antibodies to platelets from three different sources: the Glanzmann's Thrombasthenia Registry (GTR), published literature (January 01, 1999 to December 01, 2017), and the Novo Nordisk safety surveillance database. In the GTR, 133 patients received rFVIIa for the treatment of 333 bleeding episodes and prevention of bleeding in 157 surgical procedures. Overall efficacy rates were 79 and 88%, respectively, in patients treated for bleeding episodes or for the prevention of bleeding during surgery; effectiveness was generally similar across refractoriness/antibody status categories. Median dose per infusion of rFVIIa was close to that recommended for patients with GT (90 µg/kg). Data from 14 published case reports also demonstrated that rFVIIa is effective with an acceptable safety profile in patients with GT without antibodies to platelets. Analysis of adverse events reported in GTR and in Novo Nordisk safety surveillance database did not raise any new safety concerns. These data supported the label extension of rFVIIa to include cases where platelets are not readily available, which was approved by the European Medicines Agency in December 2018.

Alloimmunization in Congenital Deficiencies of Platelet Surface Glycoproteins: Focus on Glanzmann's Thrombasthenia and Bernard–Soulier's Syndrome

2018 ◽  
Vol 44 (06) ◽  
pp. 604-614 ◽  
Author(s):  
Roseline d'Oiron ◽  
Man-Chiu Poon
Keyword(s):  
Platelet Transfusion ◽  
Clinical Laboratory ◽  
Human Leukocyte ◽  
Factor Vii ◽  
Platelet Glycoprotein ◽  
Recombinant Activated Factor Vii ◽  
Platelet Surface ◽  
Glanzmann’S Thrombasthenia ◽  
Platelet Antibodies ◽  
Glanzmann's Thrombasthenia

AbstractGlanzmann's thrombasthenia (GT) and Bernard–Soulier's syndrome (BSS) are well-understood congenital bleeding disorders, showing defect/deficiency of platelet glycoprotein (GP) IIb/IIIa (integrin αIIbβ3) and GPIb-IX-V complexes respectively, with relevant clinical, laboratory, biochemical, and genetic features. Following platelet transfusion, affected patients may develop antiplatelet antibodies (to human leukocyte antigen [HLA], and/or αIIbβ3 in GT or GPIb-IX in BSS), which may render future platelet transfusion ineffective. Anti-αIIbβ3 and anti-GPIb-IX may also cross the placenta during pregnancy to cause thrombocytopenia and bleeding in the fetus/neonate. This review will focus particularly on the better studied GT to illustrate the natural history and complications of platelet alloimmunization. BSS will be more briefly discussed. Platelet transfusion, if unavoidable, should be given judiciously with good indications. Patients following platelet transfusion, and women during and after pregnancy, should be monitored for the development of platelet antibodies. There is now a collection of data suggesting the safety and effectiveness of recombinant activated factor VII in the management of affected patients with platelet antibodies.

Glanzmann’s thrombasthenia (defective platelet integrin αIIb-β3): proposals for management between evidence and open issues

2009 ◽  
Vol 102 (12) ◽  
pp. 1157-1164 ◽  
Author(s):  
Giovanni Di Minno ◽  
Matteo Di Minno ◽  
Man-Chiu Poon ◽  
Antonio Coppola
Keyword(s):  
Clinical Management ◽  
Factor Vii ◽  
Major Surgery ◽  
Clinical Settings ◽  
Minor Bleeding ◽  
Antifibrinolytic Agents ◽  
Recombinant Activated Factor Vii ◽  
Platelet Surface ◽  
Glanzmann’S Thrombasthenia ◽  
Glanzmann's Thrombasthenia

SummaryGlanzmann’s Thrombasthenia (GT) is a rare autosomal recessive bleeding disorder, characterized by a quantitative or qualitative defect of platelet surface αIIb-β3 integrin. Presently, no specific guideline/algorithm for clinical management for GT is available. Due to the rarity and heterogeneity of inherited platelet abnormalities, recommendations and guidelines are based on reports from opinions and clinical experience of panel of experts, and refer to the general management of platelet disorders. Based on the limited evidence in the area and on the strategies in clinical settings of inherited/acquired platelet defects, proposals for management of minor bleeding; moderate/major bleeding unresponsive to conservative management; major surgery; minor surgery and dental procedures for GT patients without, or with anti-platelet isoantibodies are reported. In addition to life-style advices and continuous patient education programs, when and how to employ/combine local measures, antifibrinolytic agents, hormone treatment, platelet transfusions and recombinant activated Factor VII is described. The prospective collection of treatments in GT patients recently established (Glanzmann’s Thrombasthenia Registry, GTR), based on a careful definition of clinical settings and outcomes, is likely to provide newer insight for optimising clinical management in GT.

scholarly journals Type-1 Glanzmann’s thrombasthenia: a rare cause of epistaxis in a child

2021 ◽  
Vol 8 (2) ◽  
pp. 374
Author(s):  
Saumil M. Patel ◽  
Rekha Thaddanee ◽  
Ajeet Kumar Khilnani ◽  
Gurudas Khilnani
Keyword(s):  
Adenosine Diphosphate ◽  
Membrane Receptors ◽  
Factor Vii ◽  
Definitive Treatment ◽  
Cell Transplant ◽  
Recombinant Activated Factor Vii ◽  
Platelet Surface ◽  
Glanzmann’S Thrombasthenia ◽  
Glanzmann's Thrombasthenia ◽  
Recurrent Epistaxis

Glanzmann’s thrombasthenia (GT) is a rare genetic platelet surface disorder of glycoprotein IIb/IIIa receptor presenting with muco-cutaneous bleeding of varying severity. We are reporting an unusual case of a child presenting with recurrent epistaxis with prolonged bleeding time, moderate thrombocytopenia and giant platelet size. GT was suspected because the platelet aggregation was abnormal with adenosine diphosphate, epinephrine, collagen, and thrombin; but normal with ristocetin. Diagnosis was confirmed by flow cytometry which showed deficiency of platelet membrane receptors CD 41 (Gp IIb) and CD 61 (GpIIIa) with normal expression of CD 42b (GpIb). Platelets transfusions and antifibrinolytics were given to manage bleeding. Due to repeat platelets transfusions patients with GT can develop anti-platelet antibodies for which rFVIIa (recombinant activated factor VII) is effective. Definitive treatment includes stem cell transplant or gene therapy.

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