Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non-interventional study in a real-world setting

Rebecca Kruse-Jarres, Johannes Oldenburg, Elena Santagostino, Midori Shima, Christine L. Kempton, Craig M. Kessler, Michaela Lehle, Sammy Chebon, Nives Selak Bienz, Elina Asikanius, Johnny Mahlangu

Research output: Contribution to journalArticle

Abstract

Introduction: Prospectively collected real-world data on bleeds, haemophilia treatment and safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited. A global, non-interventional study (NIS; NCT02476942) prospectively collected real-world data in PwHA who were treated per local routine clinical practice. Aim: Assess annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs) in adult/adolescent PwHA without inhibitors. Methods: Eligible participants aged ≥12 years with severe HA without history of inhibitors prospectively collected bleeding and treatment information. Results: Ninety-four participants were enrolled (median [range] age, 34 [12-76] years) and monitored for a median (range) of 29.8 (12.4-47.7) weeks. In the episodic (n = 45) and prophylactic (n = 49) treatment groups, respectively, 872/1066 (81.8%) and 151/189 (79.9%), bleeds were treated; ABRs (95% confidence interval) were 36.1 (30.8-42.3) and 5.0 (3.3-7.5), respectively, for treated bleeds and 43.1 (36.5-50.9) and 6.2 (4.2-9.2), respectively, for all bleeds, and median (interquartile range) ABRs were 31.1 (19.8-51.6) and 1.9 (0.0-8.2), respectively, for treated bleeds and 35.3 (21.7-62.9) and 2.7 (0.0-9.4), respectively, for all bleeds. Half of the participants on FVIII prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median doses/wk of standard and extended half-life FVIII, respectively. Serious AEs included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE was viral upper respiratory tract infection. Conclusion: PwHA without inhibitors continue to bleed on prophylaxis, consistent with the literature, and require treatment for breakthrough bleeds. This prospective NIS demonstrates the need for more efficacious haemostatic approaches.

Original languageEnglish
Pages (from-to)213-220
Number of pages8
JournalHaemophilia
Volume25
Issue number2
DOIs
Publication statusPublished - Mar 1 2019

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Hemophilia A
Hemorrhage
Safety
Factor VIII
Therapeutics
Hemarthrosis
Gastrointestinal Hemorrhage
Hemostatics
Polyps
Respiratory Tract Infections
Half-Life
Confidence Intervals

Keywords

  • blood coagulation factor inhibitors
  • factor VIII
  • haemophilia A
  • non-interventional study
  • observational study
  • prospective study

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Bleeding and safety outcomes in persons with haemophilia A without inhibitors : Results from a prospective non-interventional study in a real-world setting. / Kruse-Jarres, Rebecca; Oldenburg, Johannes; Santagostino, Elena; Shima, Midori; Kempton, Christine L.; Kessler, Craig M.; Lehle, Michaela; Chebon, Sammy; Selak Bienz, Nives; Asikanius, Elina; Mahlangu, Johnny.

In: Haemophilia, Vol. 25, No. 2, 01.03.2019, p. 213-220.

Research output: Contribution to journalArticle

Kruse-Jarres, R, Oldenburg, J, Santagostino, E, Shima, M, Kempton, CL, Kessler, CM, Lehle, M, Chebon, S, Selak Bienz, N, Asikanius, E & Mahlangu, J 2019, 'Bleeding and safety outcomes in persons with haemophilia A without inhibitors: Results from a prospective non-interventional study in a real-world setting', Haemophilia, vol. 25, no. 2, pp. 213-220. https://doi.org/10.1111/hae.13655
Kruse-Jarres, Rebecca ; Oldenburg, Johannes ; Santagostino, Elena ; Shima, Midori ; Kempton, Christine L. ; Kessler, Craig M. ; Lehle, Michaela ; Chebon, Sammy ; Selak Bienz, Nives ; Asikanius, Elina ; Mahlangu, Johnny. / Bleeding and safety outcomes in persons with haemophilia A without inhibitors : Results from a prospective non-interventional study in a real-world setting. In: Haemophilia. 2019 ; Vol. 25, No. 2. pp. 213-220.
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abstract = "Introduction: Prospectively collected real-world data on bleeds, haemophilia treatment and safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited. A global, non-interventional study (NIS; NCT02476942) prospectively collected real-world data in PwHA who were treated per local routine clinical practice. Aim: Assess annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs) in adult/adolescent PwHA without inhibitors. Methods: Eligible participants aged ≥12 years with severe HA without history of inhibitors prospectively collected bleeding and treatment information. Results: Ninety-four participants were enrolled (median [range] age, 34 [12-76] years) and monitored for a median (range) of 29.8 (12.4-47.7) weeks. In the episodic (n = 45) and prophylactic (n = 49) treatment groups, respectively, 872/1066 (81.8{\%}) and 151/189 (79.9{\%}), bleeds were treated; ABRs (95{\%} confidence interval) were 36.1 (30.8-42.3) and 5.0 (3.3-7.5), respectively, for treated bleeds and 43.1 (36.5-50.9) and 6.2 (4.2-9.2), respectively, for all bleeds, and median (interquartile range) ABRs were 31.1 (19.8-51.6) and 1.9 (0.0-8.2), respectively, for treated bleeds and 35.3 (21.7-62.9) and 2.7 (0.0-9.4), respectively, for all bleeds. Half of the participants on FVIII prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median doses/wk of standard and extended half-life FVIII, respectively. Serious AEs included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE was viral upper respiratory tract infection. Conclusion: PwHA without inhibitors continue to bleed on prophylaxis, consistent with the literature, and require treatment for breakthrough bleeds. This prospective NIS demonstrates the need for more efficacious haemostatic approaches.",
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AU - Santagostino, Elena

AU - Shima, Midori

AU - Kempton, Christine L.

AU - Kessler, Craig M.

AU - Lehle, Michaela

AU - Chebon, Sammy

AU - Selak Bienz, Nives

AU - Asikanius, Elina

AU - Mahlangu, Johnny

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N2 - Introduction: Prospectively collected real-world data on bleeds, haemophilia treatment and safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited. A global, non-interventional study (NIS; NCT02476942) prospectively collected real-world data in PwHA who were treated per local routine clinical practice. Aim: Assess annualized bleeding rate (ABR), haemophilia treatment practices and adverse events (AEs) in adult/adolescent PwHA without inhibitors. Methods: Eligible participants aged ≥12 years with severe HA without history of inhibitors prospectively collected bleeding and treatment information. Results: Ninety-four participants were enrolled (median [range] age, 34 [12-76] years) and monitored for a median (range) of 29.8 (12.4-47.7) weeks. In the episodic (n = 45) and prophylactic (n = 49) treatment groups, respectively, 872/1066 (81.8%) and 151/189 (79.9%), bleeds were treated; ABRs (95% confidence interval) were 36.1 (30.8-42.3) and 5.0 (3.3-7.5), respectively, for treated bleeds and 43.1 (36.5-50.9) and 6.2 (4.2-9.2), respectively, for all bleeds, and median (interquartile range) ABRs were 31.1 (19.8-51.6) and 1.9 (0.0-8.2), respectively, for treated bleeds and 35.3 (21.7-62.9) and 2.7 (0.0-9.4), respectively, for all bleeds. Half of the participants on FVIII prophylaxis had relatively high adherence to treatment, using 2.9 and 2.1 median doses/wk of standard and extended half-life FVIII, respectively. Serious AEs included gastrointestinal polyp haemorrhage and haemarthrosis; the most common AE was viral upper respiratory tract infection. Conclusion: PwHA without inhibitors continue to bleed on prophylaxis, consistent with the literature, and require treatment for breakthrough bleeds. This prospective NIS demonstrates the need for more efficacious haemostatic approaches.

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