PB1243 - Comparison of Quality of Life and Bleeding Phenotypes in People with Congenital Hemophilia A: Longitudinal Study from the PicnicHealth Database of PROMIS®-29 v2.0 scores

Background: Congenital hemophilia A (HA) is characterized by a deficiency in factor (F)VIII. Severity is usually classified by FVIII level; however, a severe bleeding phenotype can also occur in people with non-severe HA. People with HA (PwHA) require frequent disease management to control bleeding, which can affect their quality of life (QoL). Real-world data on the association between bleeding frequency and QoL are limited.

Aims: To evaluate QoL in PwHA by bleeding phenotype.

Methods: The PicnicHealth platform is a real-world, patient-centric database, collating demographics and disease characteristics for PwHA. Enrolled participants report details of bleeds, treatment, pain, and QoL via an online platform. Informed consent and ethics approval were obtained. In the present analysis (funding: F. Hoffmann-La Roche), bleeding frequency was derived from the biweekly question 'How many bleeds did you have in the past 14 days?'. The Patient-Reported Outcomes Measurement Information System (PROMIS®-29 v2.0; National Institutes of Health) is a concise, disease-agnostic QoL measure that captures comprehensive data across multiple health domains. Raw PROMIS®-29 scores are rescaled into a standardized T-score metric, with a population mean of 50 and standard deviation of 10.

Results: Baseline demographics, stratified by bleeding frequency over 10-week periods, were obtained for 174 PwHA with QoL data available who had completed ≥2 surveys (Table 1). Mean PROMIS®-29 T-scores reflected poorer QoL for PwHA with higher bleeding frequency, although there was no adjustment for differences in baseline characteristics. ‘Physical Function’ and ‘Ability to Participate in Social Role and Activities’ scores were higher for PwHA with zero bleeds compared with ≥3 bleeds; scores for ‘Sleep Disturbance’, ‘Fatigue’, ‘Pain Interference’, ‘Pain Intensity’, ‘Anxiety’ and ‘Depression’ were higher for those with ≥3 bleeds compared with zero bleeds (Figure 1).

Conclusion(s): These data provide further evidence that bleeding frequency, independent of disease severity classified by FVIII level, can affect multiple aspects of QoL for PwHA.