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Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety

Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety
Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety
Introduction Nuwiq® [human cell line‐derived recombinant factor VIII (human‐cl rhFVIII)] is a new generation rFVIII protein, without chemical modification or fusion to any other protein, produced in a human cell line. Aim/methods This prospective, open‐label, multinational phase III study assessed the efficacy and safety of human‐cl rhFVIII in 32 adult previously treated patients (PTPs) with severe haemophilia A during standard prophylaxis for ≥6 months and ≥50 exposure days. Efficacy in treating bleeds and during surgical prophylaxis was also assessed. Results Prophylactic efficacy, based on mean monthly bleeding rate, was rated as ‘excellent’ or ‘good’ in 97% of patients for all bleeds and in 100% of patients for spontaneous bleeds. Mean (SD) annualized bleeding rate was 2.28 (3.73) [median = 0.9] for all bleeds, 1.16 (2.57) [median = 0] for spontaneous bleeds and 1.00 (1.79) [median = 0] for traumatic bleeds. There were no bleeds in 50% of patients and there were no major, life‐threatening bleeds. Efficacy was ‘excellent’ or ‘good’ in treating 28 (100%) of 28 bleeds. Overall efficacy was rated as ‘excellent’ during four surgical procedures (three major, one minor) and ‘moderate’ during one major surgery. Incremental in vivo recovery (IVR) data were comparable with the one‐stage and chromogenic assays. IVR was >2.0% per IU kg−1 for all measurements and stable over 6 months. No patients developed FVIII inhibitors and there were no treatment‐related serious or severe adverse events. Conclusion These results in adult PTPs indicate that human‐cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.
1351-8216
225-231
Lissitchkov, T.
8d7393d2-abb5-4214-a611-61c69433a115
Hampton, K.
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Von Depka, M.
a8010640-e6a1-4101-bd9a-97d5513289be
Hay, C.
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Rangarajan, S.
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Tuddenham, E.
26dc750e-ca8d-4214-be26-b3ef6d2d0589
Holstein, K.
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Huth-kühne, A.
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Pabinger, I.
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Knaub, S.
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Bichler, J.
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Oldenburg, J.
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Lissitchkov, T.
8d7393d2-abb5-4214-a611-61c69433a115
Hampton, K.
ee16ffe3-8e96-4b5e-9a86-5e42d9cd9bda
Von Depka, M.
a8010640-e6a1-4101-bd9a-97d5513289be
Hay, C.
0a0e4482-8ef0-4701-a2da-457157a65a95
Rangarajan, S.
9a5e4c7e-55ba-4a3a-b5f6-f1e269d927c3
Tuddenham, E.
26dc750e-ca8d-4214-be26-b3ef6d2d0589
Holstein, K.
25a83378-8fe8-44a0-a945-fe0c0e3619e6
Huth-kühne, A.
707fbdf3-a02d-4ea6-b0de-9ff654fb9913
Pabinger, I.
0091c1d4-d9c2-4281-935c-7704b31363f3
Knaub, S.
01e79fc9-fb41-4fb6-8498-534b56e06cb0
Bichler, J.
0859b950-74f4-4cf3-b7c9-850cfbae8d8a
Oldenburg, J.
79e6962f-3ba2-47ab-aabb-b3806ac25689

Lissitchkov, T., Hampton, K., Von Depka, M., Hay, C., Rangarajan, S., Tuddenham, E., Holstein, K., Huth-kühne, A., Pabinger, I., Knaub, S., Bichler, J. and Oldenburg, J. (2016) Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety. Haemophilia, 22 (2), 225-231. (doi:10.1111/hae.12793).

Record type: Article

Abstract

Introduction Nuwiq® [human cell line‐derived recombinant factor VIII (human‐cl rhFVIII)] is a new generation rFVIII protein, without chemical modification or fusion to any other protein, produced in a human cell line. Aim/methods This prospective, open‐label, multinational phase III study assessed the efficacy and safety of human‐cl rhFVIII in 32 adult previously treated patients (PTPs) with severe haemophilia A during standard prophylaxis for ≥6 months and ≥50 exposure days. Efficacy in treating bleeds and during surgical prophylaxis was also assessed. Results Prophylactic efficacy, based on mean monthly bleeding rate, was rated as ‘excellent’ or ‘good’ in 97% of patients for all bleeds and in 100% of patients for spontaneous bleeds. Mean (SD) annualized bleeding rate was 2.28 (3.73) [median = 0.9] for all bleeds, 1.16 (2.57) [median = 0] for spontaneous bleeds and 1.00 (1.79) [median = 0] for traumatic bleeds. There were no bleeds in 50% of patients and there were no major, life‐threatening bleeds. Efficacy was ‘excellent’ or ‘good’ in treating 28 (100%) of 28 bleeds. Overall efficacy was rated as ‘excellent’ during four surgical procedures (three major, one minor) and ‘moderate’ during one major surgery. Incremental in vivo recovery (IVR) data were comparable with the one‐stage and chromogenic assays. IVR was >2.0% per IU kg−1 for all measurements and stable over 6 months. No patients developed FVIII inhibitors and there were no treatment‐related serious or severe adverse events. Conclusion These results in adult PTPs indicate that human‐cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.

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Accepted/In Press date: 17 July 2015
e-pub ahead of print date: 28 August 2015
Published date: 1 March 2016

Identifiers

Local EPrints ID: 443335
URI: http://eprints.soton.ac.uk/id/eprint/443335
ISSN: 1351-8216
PURE UUID: 76ccb826-a739-40d5-ae93-ebe9b7d5e079
ORCID for S. Rangarajan: ORCID iD orcid.org/0000-0001-7367-133X

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Date deposited: 20 Aug 2020 16:34
Last modified: 09 Jan 2022 04:11

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Contributors

Author: T. Lissitchkov
Author: K. Hampton
Author: M. Von Depka
Author: C. Hay
Author: S. Rangarajan ORCID iD
Author: E. Tuddenham
Author: K. Holstein
Author: A. Huth-kühne
Author: I. Pabinger
Author: S. Knaub
Author: J. Bichler
Author: J. Oldenburg

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