Presentation Details
| Clinical outcomes in mild-moderate and severe patients with hemophilia B: results from a real-world, multi-national survey Sheena Thakkar1, Lisa Wilcox1, Valeria Merla1, Anna Kane1, Jose Alvir1, Surya Pemmaraju1, Jennifer Mellor2, Ella Morton2, Jade Garratt Wheeldon2, James Pike2, Nathan Ball2, Stevie Olsen2. 1Pfizer Inc, New York, NY, USA.2Adelphi Real World, Bollington, United Kingdom |
Abstract
Background: Hemophilia B is a heritable X-linked disorder characterized by recurrent and extended bleeding episodes resulting from a deficiency of factor IX. Hemophilia severity is determined by the extent of factor IX deficiency. This study aims to compare occurrence of bleeding episodes and resultant joint damage in those with mild-moderate and severe presentations of hemophilia B. Objectives: To compare bleeding outcomes, joint health and hospitalizations between mild-moderate and severe hemophilia B patients in a real-world setting. Methods: Data were drawn from the Adelphi Real World Hemophilia Disease Specific Programme™, a cross-sectional survey of hemophilia-treating physicians in France, Germany, Italy, Spain, the United Kingdom, and the United States of America between February 2020 – May 2021. Hematologists and hematologist-oncologists reported data on consulting male patients with hemophilia B (PwHB), including demographics, bleeds, joint health and hospitalizations. Patients were grouped by physician-reported baseline factor activity level: <1% factor were defined as severe, ≥1% were mild-moderate. Bleed severity was determined by physician perception and classed as mild, moderate, or severe. Differences between groups were analyzed using Fisher’s Exact test or t-test, for categorical and continuous variables, respectively. Results: Overall, 142 physicians provided data for 312 PwHB. Mean (standard deviation; SD) age was 31.0 (17.8) years, 29% of patients had severe hemophilia, and 68% of patients were receiving treatment for hemophilia at data collection (mild-moderate: 57%; severe: 95%). Among those receiving treatment, 60% were treated prophylactically (mild-moderate: 39%; severe: 91%). Among severe PwHB, 57% had experienced ≥1 bleed in the 12 months prior to data collection compared to 45% of mild-moderate patients (p=0.08). Of those who experienced ≥1 bleed, mean (SD) number of total bleeds experienced was 2.1 (2.6) [mild-moderate: 1.9 (2.0); severe: 2.5 (4.0); p=0.03] and the mean (SD) number of joint bleeds was 0.8 (2.1) [mild-moderate: 0.7 (0.8); severe: 1.2 (3.5); p=0.15]. The most recent bleed for mild-moderate patients was determined by physician to be severe in 2% of cases, compared to 23% of cases for severe patients (p<0.01). Pain level associated with the bleed was determined to be severe in 1% of cases for mild-moderate patients compared to 14% for severe patients (p<0.01). Physicians reported 32% of PwHB had experienced joint problems due to hemophilia (mild-moderate: 26%; severe: 45%; p<0.01). Of PwHB, 18% had experienced synovitis in at least one joint (mild-moderate: 14%; severe: 28%; p<0.01) and 12% had been diagnosed with hemophilic arthropathy (mild-moderate: 6%; severe: 29%; p<0.01). In the 12 months prior to data collection, 15% of PwHB experienced at least one hospitalization due to hemophilia [mild-moderate: 11%; severe: 24%; p<0.01]. Conclusions: Despite high rates of prophylaxis prescription, patients with severe hemophilia B had problems with their joints and experienced multiple bleeds. Those bleeds were more commonly reported as severe and associated with severe pain in this dataset. In addition, a greater number of severe patients had a recent hospitalization compared to mild-moderate patients. These data suggest patients with severe hemophilia require improved treatment to control clinical outcomes and the pain associated with these.
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