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Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study

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Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study. / Escuriola Ettingshausen, Carmen; Katsarou, Olga; Kotnik, Barbara Faganel; Borel Derlon, Annie; Schwarz, Rudolf; Ypma, Paula F; Matytsina, Irina; Dey, Sohan; Schutgens, Roger E G.

In: HAEMOPHILIA, Vol. 28, No. 1, 01.2022, p. 46-54.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Escuriola Ettingshausen, C, Katsarou, O, Kotnik, BF, Borel Derlon, A, Schwarz, R, Ypma, PF, Matytsina, I, Dey, S & Schutgens, REG 2022, 'Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study', HAEMOPHILIA, vol. 28, no. 1, pp. 46-54. https://doi.org/10.1111/hae.14454

APA

Escuriola Ettingshausen, C., Katsarou, O., Kotnik, B. F., Borel Derlon, A., Schwarz, R., Ypma, P. F., Matytsina, I., Dey, S., & Schutgens, R. E. G. (2022). Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study. HAEMOPHILIA, 28(1), 46-54. https://doi.org/10.1111/hae.14454

Vancouver

Escuriola Ettingshausen C, Katsarou O, Kotnik BF, Borel Derlon A, Schwarz R, Ypma PF et al. Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study. HAEMOPHILIA. 2022 Jan;28(1):46-54. https://doi.org/10.1111/hae.14454

Bibtex

@article{2893e145f3ed46c9859a70d7a6d72b72,
title = "Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study",
abstract = "INTRODUCTION: Turoctocog alfa (NovoEight{\textregistered} ) is a B-domain-truncated recombinant factor VIII (FVIII) approved for patients with haemophilia A.AIM: To investigate the long-term safety and efficacy of turoctocog alfa in routine clinical practice.METHODS: Guardian 5 was a prospective, multinational, non-interventional, post-authorisation safety study. Male previously treated patients (> 150 exposure days [EDs]) of any age with severe/moderately severe haemophilia A (FVIII ≤ 2%) and a negative inhibitor test prior to first dosing (independent of FVIII-inhibitor history) were included to receive prophylaxis or on-demand treatment. The primary endpoint was the proportion of patients developing FVIII inhibitors (≥.6 Bethesda Units [BU]) after baseline visit, measured as per routine practice of each study site during clinic visits. Secondary endpoints included haemostatic effect, annualised bleeding rate (ABR), and adverse reactions assessment. The study concluded when 50 patients reached 100 EDs/patient minimum.RESULTS: Seventy patients were screened and 68 exposed to turoctocog alfa; 63 (92.6%) were on prophylaxis and five received on-demand treatment. Six (8.8%) patients reported a history of positive inhibitors. During the study, patients were exposed to turoctocog alfa for a mean (standard deviation) of 131.9 (99.0) days/patient. Fifty-five of 58 patients who completed the study were tested for FVIII inhibitors; no positive tests were reported. Overall success rate of turoctocog alfa for treatment of bleeds was 87.3%. Among patients receiving prophylaxis, median (range) ABR was 1.97 (.0-25.5) bleeds/year; estimated ABR (negative binomial model) was 3.65 (95% confidence interval: 2.53-5.25).CONCLUSION: Turoctocog alfa was safe and efficacious for haemophilia A treatment in routine clinical practice.",
keywords = "Drug Substitution, Factor VIII/therapeutic use, Hemophilia A/drug therapy, Hemostatics, Humans, Incidence, Male, Prospective Studies",
author = "{Escuriola Ettingshausen}, Carmen and Olga Katsarou and Kotnik, {Barbara Faganel} and {Borel Derlon}, Annie and Rudolf Schwarz and Ypma, {Paula F} and Irina Matytsina and Sohan Dey and Schutgens, {Roger E G}",
note = "{\textcopyright} 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.",
year = "2022",
month = jan,
doi = "10.1111/hae.14454",
language = "English",
volume = "28",
pages = "46--54",
journal = "HAEMOPHILIA",
issn = "1351-8216",
publisher = "Wiley-Blackwell",
number = "1",

}

RIS

TY - JOUR

T1 - Zero incidence of factor VIII inhibitors and successful haemostatic response in previously factor VIII-treated patients with haemophilia A switching to turoctocog alfa in a noninterventional study

AU - Escuriola Ettingshausen, Carmen

AU - Katsarou, Olga

AU - Kotnik, Barbara Faganel

AU - Borel Derlon, Annie

AU - Schwarz, Rudolf

AU - Ypma, Paula F

AU - Matytsina, Irina

AU - Dey, Sohan

AU - Schutgens, Roger E G

N1 - © 2021 The Authors. Haemophilia published by John Wiley & Sons Ltd.

PY - 2022/1

Y1 - 2022/1

N2 - INTRODUCTION: Turoctocog alfa (NovoEight® ) is a B-domain-truncated recombinant factor VIII (FVIII) approved for patients with haemophilia A.AIM: To investigate the long-term safety and efficacy of turoctocog alfa in routine clinical practice.METHODS: Guardian 5 was a prospective, multinational, non-interventional, post-authorisation safety study. Male previously treated patients (> 150 exposure days [EDs]) of any age with severe/moderately severe haemophilia A (FVIII ≤ 2%) and a negative inhibitor test prior to first dosing (independent of FVIII-inhibitor history) were included to receive prophylaxis or on-demand treatment. The primary endpoint was the proportion of patients developing FVIII inhibitors (≥.6 Bethesda Units [BU]) after baseline visit, measured as per routine practice of each study site during clinic visits. Secondary endpoints included haemostatic effect, annualised bleeding rate (ABR), and adverse reactions assessment. The study concluded when 50 patients reached 100 EDs/patient minimum.RESULTS: Seventy patients were screened and 68 exposed to turoctocog alfa; 63 (92.6%) were on prophylaxis and five received on-demand treatment. Six (8.8%) patients reported a history of positive inhibitors. During the study, patients were exposed to turoctocog alfa for a mean (standard deviation) of 131.9 (99.0) days/patient. Fifty-five of 58 patients who completed the study were tested for FVIII inhibitors; no positive tests were reported. Overall success rate of turoctocog alfa for treatment of bleeds was 87.3%. Among patients receiving prophylaxis, median (range) ABR was 1.97 (.0-25.5) bleeds/year; estimated ABR (negative binomial model) was 3.65 (95% confidence interval: 2.53-5.25).CONCLUSION: Turoctocog alfa was safe and efficacious for haemophilia A treatment in routine clinical practice.

AB - INTRODUCTION: Turoctocog alfa (NovoEight® ) is a B-domain-truncated recombinant factor VIII (FVIII) approved for patients with haemophilia A.AIM: To investigate the long-term safety and efficacy of turoctocog alfa in routine clinical practice.METHODS: Guardian 5 was a prospective, multinational, non-interventional, post-authorisation safety study. Male previously treated patients (> 150 exposure days [EDs]) of any age with severe/moderately severe haemophilia A (FVIII ≤ 2%) and a negative inhibitor test prior to first dosing (independent of FVIII-inhibitor history) were included to receive prophylaxis or on-demand treatment. The primary endpoint was the proportion of patients developing FVIII inhibitors (≥.6 Bethesda Units [BU]) after baseline visit, measured as per routine practice of each study site during clinic visits. Secondary endpoints included haemostatic effect, annualised bleeding rate (ABR), and adverse reactions assessment. The study concluded when 50 patients reached 100 EDs/patient minimum.RESULTS: Seventy patients were screened and 68 exposed to turoctocog alfa; 63 (92.6%) were on prophylaxis and five received on-demand treatment. Six (8.8%) patients reported a history of positive inhibitors. During the study, patients were exposed to turoctocog alfa for a mean (standard deviation) of 131.9 (99.0) days/patient. Fifty-five of 58 patients who completed the study were tested for FVIII inhibitors; no positive tests were reported. Overall success rate of turoctocog alfa for treatment of bleeds was 87.3%. Among patients receiving prophylaxis, median (range) ABR was 1.97 (.0-25.5) bleeds/year; estimated ABR (negative binomial model) was 3.65 (95% confidence interval: 2.53-5.25).CONCLUSION: Turoctocog alfa was safe and efficacious for haemophilia A treatment in routine clinical practice.

KW - Drug Substitution

KW - Factor VIII/therapeutic use

KW - Hemophilia A/drug therapy

KW - Hemostatics

KW - Humans

KW - Incidence

KW - Male

KW - Prospective Studies

U2 - 10.1111/hae.14454

DO - 10.1111/hae.14454

M3 - SCORING: Journal article

C2 - 34791736

VL - 28

SP - 46

EP - 54

JO - HAEMOPHILIA

JF - HAEMOPHILIA

SN - 1351-8216

IS - 1

ER -