Tyler W Buckner1, Michelle Witkop2, Christine Guelcher3, Robert Sidonio4, Craig M Kessler5, David B Clark6, Wendy Owens7, Neil Frick8, Neeraj N Iyer9, David L Cooper9. 1. University of Colorado School of Medicine, Aurora, CO, USA. 2. Munson Medical Center, Traverse City, MI, USA. 3. Children's National Health System, Washington, DC, USA. 4. Emory University and Children's Healthcare of Atlanta, Atlanta, GA, USA. 5. Georgetown University Hospital, Washington, DC, USA. 6. The Coalition for Hemophilia B, New York, NY, USA. 7. Hemophilia Federation of America, Washington, DC, USA. 8. National Hemophilia Foundation, New York, NY, USA. 9. Novo Nordisk Inc., Plainsboro, NJ, USA.
Abstract
INTRODUCTION: Health-related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact in mild/moderate hemophilia B and affected women is not well characterized. OBJECTIVE: To evaluate factors that affect HRQoL in adults with hemophilia B and caregivers of affected children. METHODS: US adult patients and caregivers of affected children completed distinct ~1-hour online surveys including patient-reported outcome instruments. RESULTS: In total, 299 adult patients and 150 caregivers participated. Adults with moderate hemophilia reported poorer health status (median EQ-5D-5L index score, 0.63) than those with mild (0.73) or severe (0.74) hemophilia. Women reported greater pain severity than men on the Brief Pain Inventory v2 Short Form (median, 7.00 vs 5.00). Based on the Patient Health Questionnaire, mild or worse depression was observed in >50% of adult respondents, and depression was reported more often in those with moderate and severe hemophilia vs those with mild hemophilia. Most caregivers reported at least mild depression. CONCLUSION: Pain, functional impairment, and depression/anxiety are present at higher-than-expected levels in individuals with hemophilia B. The large proportion of individuals with mild/moderate hemophilia and women with reduced health status suggests significant unmet needs in this population.
INTRODUCTION: Health-related quality of life (HRQoL) is impaired in patients with hemophilia; however, the impact in mild/moderate hemophilia B and affected women is not well characterized. OBJECTIVE: To evaluate factors that affect HRQoL in adults with hemophilia B and caregivers of affected children. METHODS: US adult patients and caregivers of affected children completed distinct ~1-hour online surveys including patient-reported outcome instruments. RESULTS: In total, 299 adult patients and 150 caregivers participated. Adults with moderate hemophilia reported poorer health status (median EQ-5D-5L index score, 0.63) than those with mild (0.73) or severe (0.74) hemophilia. Women reported greater pain severity than men on the Brief Pain Inventory v2 Short Form (median, 7.00 vs 5.00). Based on the Patient Health Questionnaire, mild or worse depression was observed in >50% of adult respondents, and depression was reported more often in those with moderate and severe hemophilia vs those with mild hemophilia. Most caregivers reported at least mild depression. CONCLUSION:Pain, functional impairment, and depression/anxiety are present at higher-than-expected levels in individuals with hemophilia B. The large proportion of individuals with mild/moderate hemophilia and women with reduced health status suggests significant unmet needs in this population.
Authors: Tyler W Buckner; Robert Sidonio; Michelle Witkop; Christine Guelcher; Susan Cutter; Neeraj N Iyer; David L Cooper Journal: Patient Relat Outcome Meas Date: 2019-09-18
Authors: Isolde A R Kuijlaars; Janjaap van der Net; Tyler W Buckner; Christine L Kempton; Roger E G Schutgens; Kathelijn Fischer Journal: Haemophilia Date: 2021-09-08 Impact factor: 4.263
Authors: Erik Berntorp; Petra LeBeau; Margaret V Ragni; Munira Borhany; Yasmina L Abajas; Michael D Tarantino; Katharina Holstein; Stacy E Croteau; Raina Liesner; Cristina Tarango; Manuela Carvalho; Catherine McGuinn; Eva Funding; Christine L Kempton; Christoph Bidlingmaier; Alice Cohen; Johannes Oldenburg; Susan Kearney; Christine Knoll; Philip Kuriakose; Suchitra Acharya; Ulrike M Reiss; Roshni Kulkarni; Michelle Witkop; Stefan Lethagen; Rebecca Krouse; Amy D Shapiro; Jan Astermark Journal: Haemophilia Date: 2022-03-08 Impact factor: 4.263