Abstract | INTRODUCTION: MATERIAL AND METHODS: Nine patients with severe haemophilia A with inhibitors who are on emicizumab were recruited to participate. Patients were infused with varying doses of aPCC in vivo. Samples were tested with thrombin generation (TG) assay. RESULTS: In the current in vivo arm of the study four out of nine patients reached the highest dose, 75 U/kg of aPCC and six out of nine patients were actually eligible for the highest dose. In the previous in vitro arm of the study seven out of eight patients reached the normal plasma with spiking aPCC at a very low concentration equivalent to 5 U/kg. CONCLUSION: The in vitro portion of the study demonstrated that clinically relevant concentrations of aPCC resulted in excessive TG, however, in vivo administration of aPCC to the same patients demonstrated that most of the patients had normal TG at the approved doses of aPCC. In the management of breakthrough bleeding clinicians should heed the boxed warning for concomitant use of emicizumab and aPCC, however, should also be aware that low doses of aPCC may not result in sufficient TG.
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Authors | Hande Kizilocak, Elizabeth Marquez-Casas, Jemily Malvar, Guy Young |
Journal | Haemophilia : the official journal of the World Federation of Hemophilia
(Haemophilia)
Vol. 29
Issue 1
Pg. 100-105
(Jan 2023)
ISSN: 1365-2516 [Electronic] England |
PMID | 36287631
(Publication Type: Clinical Trial, Phase I, Journal Article)
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Copyright | © 2022 John Wiley & Sons Ltd. |
Chemical References |
- prothrombin complex concentrates
- anti-inhibitor coagulant complex
- emicizumab
- Blood Coagulation Factors
- Factor VIII
- Factor IX
- Thrombin
- Recombinant Proteins
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Topics |
- Humans
- Female
- Hemophilia A
(complications)
- Metrorrhagia
(complications)
- Blood Coagulation Factors
(pharmacology, therapeutic use)
- Factor VIII
- Factor IX
- Thrombin
- Recombinant Proteins
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