Vizcaya D, Torres L, Peñuela O, Forero MDR, Rivera M, Franco J-S. Characteristics and Self‐Reported Outcomes of Children and Adults with Severe Hemophilia A from Colombia. Abstract 060. Haemophilia. 2021;27(S2):18-181. https://doi.org/10.1111/hae.14236
Introduction
Patient-reported outcomes are useful to optimize care of persons with hemophilia, but little is known of the differences between adult and children reported outcomes.
Methods
Cross-sectional study conducted in the context of a hemophilia educational bootcamp held November 29 to December 1, 2019 in Medellin, Colombia. A hemophilia patient organization (Liga Antioqueña de Hemofilia), organized the bootcamp, contacted and invited people with Hemophilia A (PwHA), independently of their treatment prescribed. The objective was to provide a patient-centered framework to collect information on patients’ beliefs and perceptions of their disease. During the 3 days, focus groups, individual interviews and 2 questionnaires were administered: the EuroQoL five-dimension (EQ-5D-5L) and the Patient Reported Outcomes Burdens and Experiences (PROBE).
Results
Overall, 24 of 25 were severe PwHA and 15 (60%) came from urban areas. Thirteen patients (52%) were aged ≥ 18 years (mean age 32.4 yrs), 12 (48%) were ≤ 17 years old (mean age 13.4 yrs). When comparing PROBE results between adults and children, adults reported worse outcomes including acute pain in the last 12 months (100% vs 58%, respectively) and chronic pain in the last 12 months (61% vs 17%, respectively). With regards to bleeds in the past year, 42% of children reported no bleeds and the remainder reported less than three bleeds, whereas only 15% (2/3) of adults reported no bleeds and more than half reported four bleeds or more. All children reported being on a prophylactic treatment 2-3 times weekly, compared to 77% (10/13) of adults in this same treatment regimen. Self-reported overall health-related QoL, measured by EQ-5D VAS, was lower in adults than children (mean (SD): 75.5 (15.5) vs 90.4 (8.6), respectively). Adults also reported a worse health state than children in the EQ-5D dimensions of pain, mobility and usual activities. However, children reported greater anxiety/depression than adults: 100% moderate and 77% slight or no anxiety/depression, respectively in children and adults.
Discussion/Conclusion
Adults with severe Hemophilia A in Colombia report worse outcomes and a greater impact on their QoL compared to children except for the emotional disorders’ domain. Additional strategies must be implemented to ensure an early intervention with appropriate prophylaxis treatment regimen across all ages.
Disclosure of Interest
D. Vizcaya Employee of: Bayer Hispania, L. Torres Employee of: Bayer S.A. Colombia, O. Peñuela Employee of: Bayer S.A. Colombia, M. D. R. Forero Employee of: Bayer S.A. Colombia, M. Rivera Employee of: Bayer Hispania, J.-S. Franco Employee of: Bayer S.A. Colombia.
View Presentation: EAHAD21-ABS-1390
