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2529 Association between Aging and Health Status in Persons Living with Hemophilia and Controls without a Bleeding Disorder – Insights from the Probe Study

Program: Oral and Poster Abstracts
Session: 904. Outcomes Research—Non-Malignant Conditions: Poster II
Hematology Disease Topics & Pathways:
Hemophilia, Diseases, Bleeding and Clotting, Clinically relevant
Sunday, December 6, 2020, 7:00 AM-3:30 PM

Federico Germini, MD, MSc1,2*, Suzanne O'Callaghan, BA3*, Chatree Chai-Adisaksopha, MD, PhD4,5, Randall Curtis, MBA6*, Neil Frick7*, Michael B. Nichol, PhD8*, Declan Noone, MSc9,10*, Brian O'Mahony11,12*, David Page13*, Jeffrey Stonebraker14*, Mark W Skinner, JD1,15* and Alfonso Iorio, MD, PhD1,16

1Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada
2Department of Medicine, McMaster University, Hamilton, Canada
3Haemophilia Foundation Australia, Melbourne, Australia
4Medicine, McMaster University, Muang, Thailand
5Division of Hematology, Department of Internal Medicine, Chiang Mai University, Chiang Mai, Thailand
6Hematology Utilization Group Study (HUGS), Walnut Creek, CA
7National Hemophilia Foundation, Berkeley
8University of Southern California, Los Angeles, CA
9Health Decisions Consultants, Dublin, Ireland
10European Haemophilia Consortium, Brussels, Belgium
11Irish Haemophilia Society, Dublin, Ireland
12Trinity College Dublin, Dublin, Ireland
13Canadian Hemophilia Society, Montreal, Canada
14North Carolina State University, Poole College of Management, Raleigh
15Institute for Policy Advancement Ltd, Washington, DC
16McMaster-Bayer Endowed Research Chair in Clinical Epidemiology of Congenital bleeding Disorders, Department of Medicine, McMaster University, Hamilton, Canada

Background: People with hemophilia (PWH) have a life expectancy disadvantage as compared to the general population but little is known about the impact of aging on health status and health-related quality of life (HRQL).

Aims: Assessing the characteristics of the association between aging, health status and HRQL in PWH and people with no bleeding disorders (PWNoBD).

Methods: A cross-sectional, multinational study was conducted as part of the Patient Reported Outcomes, Burdens and Experiences (PROBE) project. PWH and PWNoBD were asked to complete the PROBE questionnaire. Measures of health status and HRQL were the PROBE score, the EQ-5D-5L utility index, and the EuroQol visual analog scale (EQ-VAS) of global health. The association between these measures and the age of participants was explored using a multivariable model adjusting for sex (all) and disease severity (PWH only) and stratifying by country.

Results: 1157 PWH and 690 PWNoBD completed the questionnaire in 33 countries from 2016 to 2019. Study population demographics are reported in Table 1. Eight percent of the PWH and 12% of the PWNoBD were aged ≥65 years. Multivariable analysis results are reported in Table 2. As expected, the EQ-5D utility index and EQ-VAS did not show a variation with aging in PWNoBD, while in PWH they were reduced respectively by 0.031 (95% confidence interval [CI] 0.021-0.041) and 0.030 (95% CI 0.014-0.047) every 10 years. Aging was associated with a mean reduction in the PROBE score of 0.010 (95% CI 0.004 to 0.017) every 10 years in PWNoBD. PWH showed an additional reduction in the PROBE score of 0.015 (95% CI 0.008 to 0.022) every 10 years.

Conclusions: Aging is associated with a steeper decrease in health status and HRQL in PWH than in PWNoBD. PROBE is more sensitive than EQ5D in measuring the association of aging for the specific domains measured in both PWH and PWNoBD.


Disclosures: Germini: NovoNordisk: Research Funding; Roche: Research Funding; Takeda: Research Funding; Bayer: Research Funding. Curtis: USC Hemophilia Utilization Group Study (HUGS): Consultancy; Patient Reported Outcomes, Burdens and Experiences: Consultancy; Bayer: Consultancy; Novo Nordisk: Consultancy. Nichol: Octapharma: Research Funding; Genentech Inc.: Research Funding; Global Blood Therapeutics: Research Funding; CSL Behring: Research Funding; Pfizer: Research Funding; Baxalta US Inc., Bannockburn, IL (a Takeda Company): Research Funding. Noone: European Haemophilia Consortium: Membership on an entity's Board of Directors or advisory committees; Healthcare Decision Consultants: Membership on an entity's Board of Directors or advisory committees; Research Investigator PROBE: Research Funding. O'Mahony: UniQure: Honoraria; Freeline: Honoraria; Biomarin: Honoraria, Membership on an entity's Board of Directors or advisory committees. Skinner: Takeda: Honoraria, Research Funding; Sobi: Research Funding; Sanofi: Honoraria, Research Funding, Speakers Bureau; Pfizer: Other, Speakers Bureau; Biomarin: Consultancy, Research Funding; Bayer: Consultancy, Research Funding; uniQure: Research Funding; Spark Therapeutics: Other, Speakers Bureau; Genentech: Consultancy, Honoraria; Roche: Honoraria, Research Funding; Novo Nordisk: Honoraria, Research Funding; Freeline Therapeutics: Research Funding; CSL Behring: Research Funding. Iorio: CSL: Research Funding; BioMarin: Research Funding; Freeline: Research Funding; Takeda: Research Funding; Spark: Research Funding; Bayer: Research Funding; Uniqure: Research Funding; Roche: Research Funding; Sanofi: Research Funding; Pfizer: Research Funding; Octapharma: Research Funding; NovoNordisk: Research Funding; Grifols: Research Funding.

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