Johannes Oldenburg1, Johnny N Mahlangu2, Willem Bujan3, Peter Trask4, Michael U Callaghan5, Guy Young6, Elina Asikanius7, Flora Peyvandi8, Elena Santagostino8, Rebecca Kruse-Jarres9, Claude Negrier10, Craig Kessler11, Jin Xu4, Jerzy Windyga12, Midori Shima13, Sylvia von Mackensen14. 1. Department of Experimental Haematology and Transfusion Medicine, Universitätsklinikum University Clinic Bonn, Bonn, Germany. 2. Haemophilia Comprehensive Care Centre, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. 3. Instituto de Costarricense de Investigaciones Científicas, San Jose, Costa Rica. 4. Genentech, Inc., South San Francisco, California. 5. Children's Hospital of Michigan, Detroit Medical Center, Detroit, Michigan. 6. Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California. 7. F. Hoffmann-La Roche Ltd, Basel, Switzerland. 8. IRCCS Ca' Granda Foundation, Ospedale Maggiore Policlinico, Milan, Italy. 9. Washington Center for Bleeding Disorders at BloodWorks NW, University of Washington, Seattle, Washington. 10. Louis Pradel University Hospital, Lyon, France. 11. Georgetown University Medical Center, N.W., Washington, District of Columbia. 12. Department of Disorders of Hemostasis and Internal Medicine, Institute of Hematology and Transfusion Medicine, Warsaw, Poland. 13. Department of Pediatrics, Nara Medical University, Kashihara, Japan. 14. University Medical Centre Hamburg-Eppendorf, Hamburg, Germany.
Abstract
INTRODUCTION:Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. AIM: Describe health-related outcomes in PwHA with inhibitors in HAVEN 1. METHODS:PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis receivedemicizumab prophylaxis (Arm C; n = 49). Health-related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS) and work/school days. Days hospitalized also recorded. RESULTS: At week 25, differences (ANCOVA) in adjusted mean scores (95% confidence interval) favoured Arm A versus B for Haem-A-QoL "Total" score (14.0 [5.6, 22.5]; P = 0.002) and "Physical Health" (21.6 [7.9, 35.2]; P = 0.003); EQ-VAS (-9.7 [-17.6, -1.82]; P = 0.017); and IUS (-0.16 [-0.25, -0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire-specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. CONCLUSIONS: In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health-related outcomes.
RCT Entities:
INTRODUCTION:Persons with haemophilia A (PwHA) with inhibitors to factor VIII often experience decreased health-related outcomes. In HAVEN 1 (NCT02622321), there was a statistically significant reduction in bleeding with emicizumab prophylaxis versus no prophylaxis. AIM: Describe health-related outcomes in PwHA with inhibitors in HAVEN 1. METHODS: PwHA with inhibitors aged ≥12 years previously on episodic bypassing agents (BPAs) were randomized to emicizumab prophylaxis (Arm A; n = 35) or no prophylaxis (Arm B; n = 18); participants previously on BPA prophylaxis received emicizumab prophylaxis (Arm C; n = 49). Health-related outcomes assessed at baseline and monthly thereafter: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL), Haemophilia-specific Quality of Life Questionnaire for Children Short Form (Haemo-QoL SF), EuroQol 5-Dimensions 5-Levels (EQ-5D-5L) index utility score (IUS) and visual analogue scale (EQ-VAS) and work/school days. Days hospitalized also recorded. RESULTS: At week 25, differences (ANCOVA) in adjusted mean scores (95% confidence interval) favoured Arm A versus B for Haem-A-QoL "Total" score (14.0 [5.6, 22.5]; P = 0.002) and "Physical Health" (21.6 [7.9, 35.2]; P = 0.003); EQ-VAS (-9.7 [-17.6, -1.82]; P = 0.017); and IUS (-0.16 [-0.25, -0.07]; P = 0.001); mean scores are comparable in Arms A and C. Throughout the study, a greater proportion of participants on emicizumab prophylaxis than no prophylaxis exceeded questionnaire-specific responder thresholds. Mean proportion of missed work days and number of days hospitalized were lower with emicizumab prophylaxis than no prophylaxis. CONCLUSIONS: In PwHA with inhibitors, emicizumab prophylaxis was associated with substantial and meaningful improvements in health-related outcomes.
Authors: Denise E Sabatino; Steven W Pipe; Diane J Nugent; J Michael Soucie; W Craig Hooper; W Keith Hoots; Donna M DiMichele Journal: Haemophilia Date: 2019-07 Impact factor: 4.287
Authors: Steven W Pipe; Rebecca Kruse-Jarres; Johnny N Mahlangu; Glenn F Pierce; Flora Peyvandi; Peter Kuebler; Christian De Ford; Fabián Sanabria; Richard H Ko; Tiffany Chang; Charles R M Hay Journal: J Thromb Haemost Date: 2021-01 Impact factor: 5.824
Authors: Flora Peyvandi; Johnny N Mahlangu; Steven W Pipe; Charles R M Hay; Glenn F Pierce; Peter Kuebler; Rebecca Kruse-Jarres; Midori Shima Journal: J Thromb Haemost Date: 2021-01 Impact factor: 5.824