SynthesisHaemophilia : the official journal of the World Federation of Hemophilia2025
Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.
Synthesis in Haemophilia : the official journal of the World Federation of Hemophilia, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
3 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Efficacy and safety of recombinant activated factor VII in Glanzmann thrombasthenia: A systematic literature review.Haemophilia : the official journal of the World Federation of Hemophilia · 2025Pooled it
- Efficacy and safety of recombinant activated factor VII for secondary prophylaxis in patients with Glanzmann thrombasthenia.Research and practice in thrombosis and haemostasis · 2026Observational
- Molecular Pathogenesis of Inherited Platelet Dysfunction.Biomolecules · 2025Review
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPlatelet transfusion is considered the standard treatment for preventing or controlling severe haemorrhage in Glanzmann thrombasthenia (GT). However, platelet transfusion can have detrimental effects, including the production of anti-GPIIb/IIIa isoantibodies or anti-HLA antibodies (Ab) and platelet transfusion refractoriness. Recombinant activated factor VII (rFVIIa) has been proposed as an alternative treatment to platelet transfusion.
methodsWe analyzed data from 77 case reports including 100 subjects to investigate the effectiveness and safety of rFVIIa in combination with platelets or antifibrinolytics for preventing or treating non-surgical bleeds, as well as surgical and obstetrical procedures in GT.
resultsThe dosage of rFVIIa was consistent with previous recommendations (90 µg/kg per infusion). In subjects without Ab/refractoriness (n = 56), rFVIIa was effective in managing 93% of non-surgical bleeds (n = 42), 91% of minor (n = 11) and 92% of major (n = 26) surgical procedures and 89% of obstetrical procedures (n = 9). In subjects with Ab/refractoriness (n = 44), rFVIIa was effective in managing 90% of non-surgical bleeds (n = 39), and 75% of minor (n = 12) and 100% of major (n = 17) surgical procedures. The use of rFVIIa was safe, with 4 (2.7%) serious adverse events associated with rFVIIa.
conclusionAlthough the use of rFVIIa is currently restricted to subjects with Ab/refractoriness or when platelets are not available, our findings suggest expanding the indications for rFVIIa to encompass GT without Ab/refractoriness. Frontline use of rFVIIa may be proposed when clinically possible to mitigate the risks associated with platelet transfusion.
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