NovoSeven (recombinant factor VIIa) for the treatment of bleeding episodes and perioperative management in patients with Glanzmann’s thrombasthenia

Abstract

Glanzmann’s thrombasthenia is a rare inherited autosomal recessive bleeding disorder caused by qualitative or quantitative defects of the platelet membrane glycoprotein IIb/IIIa. The ensuing lack of platelet aggregation is frequently associated with mucocutaneous bleeding that may be variable in both frequency and intensity, ranging from minimal bruising to severe and life-threatening hemorrhages. A number of treatment modalities have been proposed to manage the bleeding episodes, which include local measures, antifibrinolytic agents, platelet transfusions and recombinant activated factor VII. The role of this bypassing hemostatic agent for treatment or prevention of bleeding episodes in Glanzmann’s thrombasthenia patients is critically analyzed in this review.

Keywords::

• bleeding
• Glanzmann’s thrombasthenia
• NovoSeven
• recombinant activated factor VII
• surgery

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Glanzmann’s thrombasthenia is a rare autosomal recessive disorder characterized by qualitative or quantitative abnormalities of the platelet membrane glycoprotein IIb/IIIa.

• The clinical manifestation in Glanzmann’s thrombasthenia is variable, ranging from easy bruising to severe and potentially life-threatening hemorrhages.

• Local measures, antifibrinolytic agents and platelet transfusion are the standard treatment for bleeding episodes in Glanzmann’s thrombasthenia patients.

• A number of case reports and case series have documented the successful use of recombinant activated factor VII (rFVIIa) in the management of bleeding or to cover surgery in Glanzmann’s thrombasthenia patients.

• Recombinant FVIIa is currently licensed in Europe for Glanzmann’s thrombasthenia patients with platelet alloimmunization and past or present history of platelet refractoriness.

• The recommended rFVIIa bolus dose is of 90 μg/kg at 2 h intervals until hemostasis is achieved.

• The published literature data document the safety of rFVIIa in the Glanzmann’s thrombasthenia setting.

• Over the next 5 years, the publication of international survey will improve our knowledge on the clinical presentation and management of Glanzmann’s thrombasthenia.

Notes