Chai-Adisaksopha C, Skinner MW, Page D, Stonebraker J, Noone D, Curtis R, Frick N, Nichol M, O’Mahony B, Iorio A, and Patient Reported Outcomes Burdens and Experiences Study Group. Health Status of People Living with Non- severe Hemophilia – Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study. Abstract OC 32.4. Res Pract Thromb Haemost. 2019;3(S1):1-228. https://doi.org/10.1002/rth2/12227
Background
People living with non-severe hemophilia (PwNSH), FVIII/IX 2-40 IU/dL, generally receive factor concentrate episodically. In recent studies, life expectancy of PwNSH is comparable to the non-hemophilia male population. However, their health status has not been systematically investigated.
Aims
To evaluate the health status of PwNSH
Methods
PROBE data for PwNSH and participants without a bleeding disorder (controls) were analyzed. A match-paired analysis, using 1:1 ratio of PwNSH and controls was performed. Health status of the two groups were compared using parametric or non-parametric methods.
Results
366 participants were included in this analysis. Mean age was 44.7 (SD 17.4) years in PwNSH and 45.8 (SD 14.4) in controls. Table 1 demonstrates health status of participants. Acute pain and chronic pain were more commonly reported among PwNSH (72.4% and 68.1%) compared to controls (37.6% and 30.0%), P< 0.001. Pain medications were more commonly used among PwNSH as compared to controls (86.2% vs 68.0%, P< 0.001). Working full-time was more frequent among controls. PwNSH more frequently reported being on long-term sick or disability compared to controls. Mean sick days per year were significantly higher in PwNSH (3.7 days vs 44.9 days, P< 0.001). Self-evaluated health status using the visual analog scale (VAS) shows that PwNSH had a significantly lower VAS utility score compared to controls (72.8 vs 83.7, P< 0.001). Overall health status, which was evaluated by mean PROBE score, indicated that PwNSH had worse health status compared with controls (0.71 vs 0.89, P< 0.001).
Conclusions
PwNSH had more health-related problems, worse work/school life, and lower health-related quality of life when compared to controls. PROBE findings demonstrate non-severe hemophilia is not a benign disease. Traditional clinical outcomes, e.g., annualized bleeding rate or mortality, are less sensitive to detect health status in PwNSH. We encourage investigators and clinicians to evaluate PwNSH using a patient-reported outcome assessment tool.
View Presentation: Health Status of People Living with Non- severe Hemophilia
