CHOICE Study illustrates that Person with Inhibitors (PWI) have a more bleeding and joint issues
Improvement is needed in treatment and disease management for this population
THSNA 2018 (San Diego, CA, March 7-10): The CHOICE Project was a partnership between the US Centers for Disease Control and Prevention (CDC) and Hemophilia Federation of America, a non-profit, community-based organization, to survey persons with bleeding disorders (PwBD) in the US, including those who do not receive care at federally-funded hemophilia treatment centers (non-HTC PwBD). This study analyzed and compared demographics, treatment, pain management, and quality of life issues among patients with hemophilia (PwH) A or B, aged 12 and above, including patients with inhibitors (PwI) in the CHOICE data set to better understand disease burden and gaps in healthcare. The goal was to identify how the burdens and gaps can be addressed to improve health outcomes.
This study analyzes a portion of the CHOICE data set for PwI compared to those without inhibitors (non-PwI) enrolled between April 2013 and July 2015; respondents were adults (age 18 or older) and caregivers of children (age <18). There were 576 PwBD (HTC and non-HTC), including 31 PwI and 191 non-PwI. For PwI and non-PwI, 92%/76% reported severe hemophilia A and 100%/55% severe hemophilia B; 10%/8% did not receive care at an HTC.
Almost all PwI/non-PWI (100%/99%) reported having had a bleeding the past; 81%/76% reporting a joint bleed in the past 12 months. They reported an average of 8.1/5.6 bleeds in the past 12 months. More than twice the percent of PwI had ER visits (55%) than non-PwI (23%) in the last 12 months; 45%/14% stayed overnight in a hospital for an average of 13.8/7.7 nights.
In terms of joint-related problems, 39%/33% always or frequently (22%/15%) experience joint problems; the average number of lost days due to joint problems in the past year was 40.1/22.6 days. To treat pain in the past 30 days, 30% of PwI and 6% of non-PwI use over-the-counter pain medication on most days and 17%/12% use prescription pain medication every day.
Due to the high disease burden among PwI in the US, improvement is needed in treatment and disease management for this population. In PwI, a higher bleed rate and more frequent joint issues confirm the profound effects of progressive hemarthropathy on patients. Longitudinal assessments of disease burden in PwI could serve as a means of measuring improvement in patient self-care and treatment effectiveness.