Long-term safety and efficacy results from the phase 3b, open-label, multicentre Continuation study of rurioctocog alfa pegol for prophylaxis in previously treated patients with severe haemophilia A
© 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd..
INTRODUCTION: Previous studies reported the efficacy and safety profile of extended half-life PEGylated recombinant factor VIII (FVIII) rurioctocog alfa pegol (TAK-660, SHP660, BAX 855) in preventing bleeding in haemophilia A patients.
AIM: This study evaluated long-term safety and efficacy of rurioctocog alfa pegol for prophylaxis and treatment of bleeding in previously treated children and adults.
METHODS: In this phase 3b, prospective, open-label, multicentre study (NCT01945593), eligible patients ≤ 75 years with severe haemophilia A (FVIII < 1%) received prophylactic rurioctocog alfa pegol in a fixed dose (FD, twice-weekly or less frequent) or pharmacokinetic (PK)-tailored dose regimen. Co-primary endpoints were incidence of confirmed FVIII inhibitory antibody development and spontaneous annualized bleed rate (ABR), analysed using a generalised linear model. Secondary endpoints included overall haemostatic efficacy, occurrence of adverse events and health-related quality of life (HRQoL).
RESULTS: Overall, 216 patients were included; mean (SD) age at enrolment was 22.8 (15.7) years. No patients developed confirmed FVIII inhibitors. The point estimate (95% CI) of mean spontaneous ABR was 1.20 (0.92-1.56) among 186 patients receiving twice-weekly FD prophylaxis and 0.96 (0.54-1.71) among 25 patients receiving PK-tailored prophylaxis. Overall haemostatic efficacy was rated good or excellent in 88.6% of all bleeds. No new safety signals were observed. Patients reported improvements in HRQoL measures of pain, and physical and mental well-being.
CONCLUSION: These results highlight the long-term safety and efficacy of rurioctocog alfa pegol prophylaxis in previously treated children and adults with severe haemophilia A, with a safety profile similar to previous studies and continuing ABR reduction.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2020 |
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| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:26 |
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| Enthalten in: |
Haemophilia : the official journal of the World Federation of Hemophilia - 26(2020), 4 vom: 01. Juli, Seite e168-e178 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Chowdary, Pratima [VerfasserIn] |
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| Links: |
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| Anmerkungen: |
Date Completed 19.05.2021 Date Revised 19.05.2021 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1111/hae.14052 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM311751512 |
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| 100 | 1 | |a Chowdary, Pratima |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Long-term safety and efficacy results from the phase 3b, open-label, multicentre Continuation study of rurioctocog alfa pegol for prophylaxis in previously treated patients with severe haemophilia A |
| 264 | 1 | |c 2020 | |
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| 500 | |a Date Completed 19.05.2021 | ||
| 500 | |a Date Revised 19.05.2021 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2020 The Authors. Haemophilia published by John Wiley & Sons Ltd. | ||
| 520 | |a INTRODUCTION: Previous studies reported the efficacy and safety profile of extended half-life PEGylated recombinant factor VIII (FVIII) rurioctocog alfa pegol (TAK-660, SHP660, BAX 855) in preventing bleeding in haemophilia A patients | ||
| 520 | |a AIM: This study evaluated long-term safety and efficacy of rurioctocog alfa pegol for prophylaxis and treatment of bleeding in previously treated children and adults | ||
| 520 | |a METHODS: In this phase 3b, prospective, open-label, multicentre study (NCT01945593), eligible patients ≤ 75 years with severe haemophilia A (FVIII < 1%) received prophylactic rurioctocog alfa pegol in a fixed dose (FD, twice-weekly or less frequent) or pharmacokinetic (PK)-tailored dose regimen. Co-primary endpoints were incidence of confirmed FVIII inhibitory antibody development and spontaneous annualized bleed rate (ABR), analysed using a generalised linear model. Secondary endpoints included overall haemostatic efficacy, occurrence of adverse events and health-related quality of life (HRQoL) | ||
| 520 | |a RESULTS: Overall, 216 patients were included; mean (SD) age at enrolment was 22.8 (15.7) years. No patients developed confirmed FVIII inhibitors. The point estimate (95% CI) of mean spontaneous ABR was 1.20 (0.92-1.56) among 186 patients receiving twice-weekly FD prophylaxis and 0.96 (0.54-1.71) among 25 patients receiving PK-tailored prophylaxis. Overall haemostatic efficacy was rated good or excellent in 88.6% of all bleeds. No new safety signals were observed. Patients reported improvements in HRQoL measures of pain, and physical and mental well-being | ||
| 520 | |a CONCLUSION: These results highlight the long-term safety and efficacy of rurioctocog alfa pegol prophylaxis in previously treated children and adults with severe haemophilia A, with a safety profile similar to previous studies and continuing ABR reduction | ||
| 650 | 4 | |a Clinical Trial, Phase III | |
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| 650 | 4 | |a rurioctocog alfa pegol | |
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| 700 | 1 | |a Mullins, Eric S |e verfasserin |4 aut | |
| 700 | 1 | |a Konkle, Barbara A |e verfasserin |4 aut | |
| 700 | 1 | |a McGuinn, Catherine |e verfasserin |4 aut | |
| 700 | 1 | |a Park, Young Shil |e verfasserin |4 aut | |
| 700 | 1 | |a Stasyshyn, Oleksandra |e verfasserin |4 aut | |
| 700 | 1 | |a Zulfikar, Bülent |e verfasserin |4 aut | |
| 700 | 1 | |a Engl, Werner |e verfasserin |4 aut | |
| 700 | 1 | |a Tangada, Srilatha |e verfasserin |4 aut | |
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