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 Tool. Abstract 060. Haemophilia. 2021;27(S2):18-181. https://doi.org/10.1111/hae.14236
Introduction
In resource constrained countries such as the Kyrgyz Republic, there is very limited availability of clotting factor concentrates which has a determinantal effect on the long-term health-related quality of life (HRQoL) of people with haemophilia (PWH).
Methods
HRQoL data was collected from PWH > age 10 using the PROBE questionnaire which also collects haemophilia related sequalae, socioeconomic variables, and EQ-5D-5L. Descriptive methods were used to analyse the data set.
Results
37 PWH with a mean age (SD) of 22.76 (8.25) years participated. 29.7% (n=11), 50.5% (n=15), 2.7% (n=1) and 27.0% (n=10) were severe, moderate, mild or of those whom reported they do not know their severity level. 18.9% (n=7) reported access to some prophylaxis; 59.5% (n=22) and 16.2% (n=6) reported episodic (on-demand) or no treatment availability respectively. 35.1% (n=13) of adults (≥age 18) who did not otherwise report that they were a student reported they were either unemployed or on long-term disability. Overall, 70.3% (n=26) reported an impact on activities of daily living. PWH reporting acute or chronic pain in the past 12 months were 56.8% (n=21) and 75.7% (n=28) respectively. 67.6% (n=25) reported having a “target joint”. 41.5% (n=17) reported chronic pain in their target joint(s). 83.8% (n=31) reported having a joint(s) with reduced range of motion. 24.3% (n=9) reported having had life- or limb-threatening bleeds in the past 12 months. Overall HRQoL utility scores (SD) measured by EQ-5D and PROBE were 0.734 (0.23) and 0.715 (0.13) respectively reflecting the significant impact on quality of life for people living with haemophilia.
Discussion
This study was carried out in a cohort of PWH who did not have consistent access to factor concentrates. Among this cohort, the data demonstrate a significant impact on quality of life, joint health, employment, and chronic / acute pain. Early in life, the lack of accessible treatment has led to early disability, impacts on independence, and work-life.
Conclusion
There is evidence of significant joint damage and reduced quality of life without consistent availability of treatment regimens such as low-dose prophylaxis early in life. The introduction of prophylactic regimen has a demonstrated benefit for PWH. Implementation in the Kyrgyz Republic could significantly improve the health of future generations of PWH.
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