Rebecca Kruse-Jarres1, Johannes Oldenburg2, Elena Santagostino3, Midori Shima4, Christine L Kempton5, Craig M Kessler6, Michaela Lehle7, Sammy Chebon7, Nives Selak Bienz7, Elina Asikanius7, Johnny Mahlangu8. 1. Washington Center for Bleeding Disorders at Bloodworks Northwest, Seattle, Washington. 2. Universitätsklinikum Bonn, Bonn, Germany. 3. Centro Emofilia e Trombosi A. Bianchi Bonomi, IRCCS Fondazione Ca' Granda, Milano, Italy. 4. Department of Pediatrics, Nara Medical University, Kashihara, Japan. 5. Department of Hematology and Medical Oncology, Emory School of Medicine, Atlanta, Georgia. 6. Lombardi Comprehensive Cancer Center Georgetown University Medical Center, Washington, District of Columbia. 7. F. Hoffmann-La Roche Ltd, Basel, Switzerland. 8. Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Parktown, Johannesburg, South Africa.
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.
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.
Authors: K John Pasi; Michael Laffan; Savita Rangarajan; Tara M Robinson; Nina Mitchell; Will Lester; Emily Symington; Bella Madan; Xinqun Yang; Benjamin Kim; Glenn F Pierce; Wing Yen Wong Journal: Haemophilia Date: 2021-08-11 Impact factor: 4.263
Authors: Barbara A Konkle; Doris V Quon; Leslie Raffini; Michael Recht; Vlad C Radulescu; Shannon L Carpenter; Amy L Dunn; Mei Lu; Maureen Watt Journal: J Blood Med Date: 2021-10-14
Authors: Michael U Callaghan; Elina Asikanius; Michaela Lehle; Johannes Oldenburg; Johnny Mahlangu; Marianne Uguen; Sammy Chebon; Rebecca Kruse-Jarres; Víctor Jiménez-Yuste; Midori Shima; Peter Trask; Christine L Kempton; Craig M Kessler; Gallia G Levy; Flora Peyvandi Journal: Res Pract Thromb Haemost Date: 2022-09-13
Authors: Gili Kenet; Yeu-Chin Chen; Gillian Lowe; Charles Percy; Huyen Tran; Annette von Drygalski; Marc Trossaërt; Mark Reding; Johannes Oldenburg; Maria Eva Mingot-Castellano; Young-Shil Park; Flora Peyvandi; Margareth C Ozelo; Johnny Mahlangu; Jennifer Quinn; Mei Huang; Divya B Reddy; Benjamin Kim Journal: J Clin Med Date: 2021-12-18 Impact factor: 4.241