Haowei Linda Sun1,2, Kam A McIntosh1, Sandra J Squire1, Ming Yang1, Claude Bartholomew1, Deb S Gue1, Pat G Camp3,4, Shannon C Jackson1,2. 1. British Columbia Provincial Bleeding Disorders Program - Adult Division, St. Paul's Hospital, Vancouver, BC, Canada. 2. Division of Hematology, Department of Medicine, University of British Columbia (UBC) and St. Paul's Hospital, Vancouver, BC, Canada. 3. Centre for Heart Lung Innovation, Vancouver, BC, Canada. 4. UBC Department of Physical Therapy, Vancouver, BC, Canada.
Abstract
INTRODUCTION: Adults with severe haemophilia A (SHA) may experience breakthrough bleeds despite standard weight-based FVIII prophylaxis three times weekly. Individualized prophylaxis has evolved to optimize patient outcomes. AIMS: This study aimed to evaluate the impact of a standardized approach to individualized prophylaxis on annualized bleeding rates (ABR), factor utilization, physical activity and quality of life in adults with SHA. METHODS: In this prospective cohort study, patients with baseline FVIII:C <2% and ABR >3 on weight-based prophylaxis received a standardized approach to individualized prophylaxis. Changes in ABR, annualized FVIII consumption and adherence from the 12-month prestudy and 12-month intervention period were compared. Changes in Haemo-QoL-A total score, Physical Functioning (PF) subscale and physical activity level measured by accelerometry were also examined. RESULTS: Eighteen patients participated (median age 26 years). Individualized prophylaxis decreased total bleeds in the population by 69% and traumatic bleeds by 73%. The median ABR decreased from 7.5 to 2 (P<.001). Annualized factor consumption increased by 7.3%, as a result of 66% reduction in factor utilization for treatment of bleeds and 25% increase in factor utilization for prophylaxis. Adherence scores for frequency and dosing did not change. There was a significant increase in the Haemo-QoL-A total score (P=.02) and PF score (P=.01) from baseline to 4 months but no change in physical activity. CONCLUSION: Patients with SHA who switched from standard to individualized prophylaxis show reduced ABR and increased FVIII consumption, and also improved their health-related quality of life. The mechanism is independent of adherence to prescribed prophylactic regimen.
INTRODUCTION: Adults with severe haemophilia A (SHA) may experience breakthrough bleeds despite standard weight-based FVIII prophylaxis three times weekly. Individualized prophylaxis has evolved to optimize patient outcomes. AIMS: This study aimed to evaluate the impact of a standardized approach to individualized prophylaxis on annualized bleeding rates (ABR), factor utilization, physical activity and quality of life in adults with SHA. METHODS: In this prospective cohort study, patients with baseline FVIII:C <2% and ABR >3 on weight-based prophylaxis received a standardized approach to individualized prophylaxis. Changes in ABR, annualized FVIII consumption and adherence from the 12-month prestudy and 12-month intervention period were compared. Changes in Haemo-QoL-A total score, Physical Functioning (PF) subscale and physical activity level measured by accelerometry were also examined. RESULTS: Eighteen patients participated (median age 26 years). Individualized prophylaxis decreased total bleeds in the population by 69% and traumatic bleeds by 73%. The median ABR decreased from 7.5 to 2 (P<.001). Annualized factor consumption increased by 7.3%, as a result of 66% reduction in factor utilization for treatment of bleeds and 25% increase in factor utilization for prophylaxis. Adherence scores for frequency and dosing did not change. There was a significant increase in the Haemo-QoL-A total score (P=.02) and PF score (P=.01) from baseline to 4 months but no change in physical activity. CONCLUSION:Patients with SHA who switched from standard to individualized prophylaxis show reduced ABR and increased FVIII consumption, and also improved their health-related quality of life. The mechanism is independent of adherence to prescribed prophylactic regimen.
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
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Authors: Shadan Lalezari; Mark T Reding; Ingrid Pabinger; Pal Andre Holme; Claude Negrier; Pavani Chalasani; Ho-Jin Shin; Maria Wang; Despina Tseneklidou-Stoeter; Monika Maas Enriquez Journal: Haemophilia Date: 2019-10-17 Impact factor: 4.287
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