A Patient with Glanzmann Thrombasthenia and Epistaxis Successfully Treated with Recombinant Factor Vila

Tengborn, Lilian; Petruson, Björn

doi:10.1055/s-0038-1650412

Cited by 75 publications

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“…Repeated platelet transfusion may, however, result in the development of antibodies [to human leukocyte antigen (HLA) or to GPIIb/IIIa] which may render future platelet transfusions ineffective. 2,[12][13][14] Following the successful treatment of epistaxis in a GT patient, 15 several studies suggested that recombinant activated factor VII (rFVIIa; NovoSeven ® ) may be effective in the treatment of bleeding and the perioperative management of surgery in GT. [16][17][18] Notably, an international survey in GT patients treated for bleeds and surgical/invasive procedures showed good effectiveness and tolerability for rFVIIa given as bolus injections; the findings also allowed for a preliminary suggestion of an optimal dosing regimen (≥3 doses of ≥80 mg/kg given at intervals of ≤2.5 h) for the treatment of moderate-to-severe bleeding episodes.…”

Section: Introductionmentioning

confidence: 99%

The international prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients

Minno

Zotz²,

d’Oiron

et al. 2015

Haematologica

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© F e r r a t a S t o r t i F o u n d a t i o nMedicines Agency (EMA) for the treatment and prevention of bleeding episodes and in surgery or invasive procedures in GT patients with antibodies to GPIIb/IIIa and/or HLA, and a past or present history of platelet refractoriness to platelet transfusions. 20 To date, for GT patients who are not refractory, platelets are used as the first-line treatment.As part of the approval, the EMA required the collection of post-marketing pharmacovigilance data on rFVIIa in GT. The observational, international, multicenter, webbased Glanzmann's Thrombasthenia Registry (GTR) was, therefore, launched to prospectively gather and evaluate information on the different treatment modalities and their outcomes in "real-world" clinical practice. 21 Collection of such registry data is key to improving the understanding and management of rare diseases such as GT, for which randomized clinical trials are difficult to perform.This paper reports new, clinically relevant effectiveness and safety data on rFVIIa, as well as the other therapeutic options available (antifibrinolytics and platelets), for the treatment of non-surgical bleeds in patients with GT, using data obtained from the largest observational study of GT patients, the GTR. Methods PatientsFrom May 10, 2007, to December 16, 2011, prospectively collected online data were entered into the GTR on the effectiveness and safety (including thromboembolic events) of systemic hemostatic treatments used in clinics in patients with GT, including rFVIIa, platelet transfusions (P), and antifibrinolytics (AF) alone or in combination with other agents. All patients were treated according to local practices in an open-label manner, and no drugs were supplied for this registry. The main features of the GTR include: (i) standardized data collection using a customized webbased tool; (ii) a protocol-based registry conducted in accordance with general data protection laws and any local country requirements for conducting observational studies; and (iii) centralized data management overseen by an external expert panel composed of four physicians and by the international medical director of Novo Nordisk (the study sponsor). Protocol definitions and outcomes specified by the study sponsor, and post hoc classifications of bleed severity employed by the external expert panel (in response to requests by the EMA), are reported in Online Supplementary Table S1.Only patients with a diagnosis of congenital GT were included in the GTR (see Online Supplementary Table S1). Patients with acquired thrombasthenic states caused by autoimmune disorders or medications were excluded. Refractoriness and the presence of antibodies were coded initially and assessed periodically as deemed important by the investigator. As tests for antibodies may not have been available at all centers, antibodies may also have been present in some patients classified as having refractoriness only. Data handlingAs this was an observational study, treatment was based on local practice ...

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Section: Introductionmentioning

confidence: 99%

The international prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients

Minno

Zotz²,

d’Oiron

et al. 2015

Haematologica

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“…Recent patient reports have suggested that recombinant factor VIIa (rVIIa) (NovoSeven Ò ; Novo Nordisk, Hillerød, Denmark) may be effective therapy for treatment of bleeding in patients with congenital platelet function defects and other acquired platelet disorders, including thrombocytopenia (Tengborn & Petruson, 1996;Peters & Heijboer, 1998;Ancliff et al, 1999;Chuansumrit et al, 1999;Poon et al, 1999Poon et al, , 2001d'Orion et al, 2000;Poon & d'Orion, 2000;Vidarsson & Onundarson 2000;Gerotziafas et al, 2002). The exact mechanism of how rVIIa improves haemostasis in platelet disorders is not fully understood but it is proposed that the increase in thrombin generated on the platelet surface as a result of direct activation of factors IX and X by rVIIa may result in faster platelet activation via this mechanism, compensating for either a lower number of platelets present or dysfunctional platelets (Monroe et al, 1997(Monroe et al, , 2002Kjalke et al, 2001).…”

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confidence: 99%

The use of recombinant factor VIIa in children with inherited platelet function disorders

et al. 2003

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Summary. Inherited deficiencies of platelet surface glycoproteins such as Glanzmann's thrombasthenia (GT) or Bernard–Soulier syndrome (BSS) can lead to a severe bleeding diathesis. In the past, bleeding episodes in these patients have often required platelet transfusion to secure haemostasis but recently a number of patient reports have suggested that recombinant factor VIIa (rVIIa) may also be effective. We have used rVIIa on 33 occasions in seven children with inherited platelet function disorders over a 2‐year period: five had GT, one had BSS and one had storage pool disease with a severe phenotype. Bleeding ceased with rVIIa alone in 10 of 28 acute bleeding episodes, but recurred in two of these. The two features that predicted response to rVIIa were the severity of the bleeding and the delay from the onset of bleeding to treatment. Five episodes of planned surgical intervention were treated successfully with rVIIa. Eighteen out of the 28 acute episodes and none of the planned surgical episodes required blood product support. We have found variable efficacy of rVIIa for acute bleeding episodes in this small series of children with inherited platelet function defects but larger studies are warranted, particularly as rVIIa is a relatively low‐risk treatment approach for these disorders.

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“…In a retrospective surgical case series reported by O'Connell et al 6 , a wide range of 15-180 µg.kg -1 rFVIIa is used for massive bleeding. In 1996, Tengborn and Petruson first reported the successful use of rFVIIa for treating severe epistaxis in a 2-year-old boy with GT who had not responded to conservative treatment 7 . After this report, rFVIIa was used by other investigators for bleeding episodes or surgery in patients with GT with or without alloantibodies to platelet Gp IIb/IIIa complex or HLA 1,3,4,8,9 .…”

Section: Discussionmentioning

confidence: 99%

Conduta no perioperatório de paciente pediátrico com trombastenia de glanzmann durante adenoidectomia

Duman

Saylan

Çekiç

2012

Rev. Bras. Anestesiol.

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Summary: Duman EN, Saylan S, Cekic B -Perioperative Management of a Pediatric Patient with Glanzmann's Thrombasthenia during Adenoidectomy. Background and objective:Glanzmann's thrombasthenia (GT) is an autosomal recessively inherited platelet disorder. There is not any specific treatment. Platelet transfusion is currently the standard treatment when bleeding does not respond to local measures and/or antifibrinolytic treatment, although it may result in alloimmunization. Recombinant activated factor VII (rFVIIa) might be used to avoid recurrent platelet transfusion.

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A Patient with Glanzmann Thrombasthenia and Epistaxis Successfully Treated with Recombinant Factor Vila

Cited by 75 publications

References 0 publications

The international prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients

The international prospective Glanzmann Thrombasthenia Registry: treatment modalities and outcomes in non-surgical bleeding episodes in Glanzmann thrombasthenia patients

The use of recombinant factor VIIa in children with inherited platelet function disorders

Conduta no perioperatório de paciente pediátrico com trombastenia de glanzmann durante adenoidectomia

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