Treatment outcomes of clotting factor concentrates in patients with hemophilia

Abstract

background: Hemophilia is a genetic bleeding disorder that is characterized by easy bleeding and difficulty stopping bleeding due to a lack of clotting factors. Controlling bleeding symptoms has led to the development of treatments involving concentrated factors extracted from plasma to replace the missing factor VIII or IX. This includes treatments for episodic bleeding and prophylaxis, which yield different outcomes. Additionally, there are barriers affecting these treatments.

Objective: To evaluate treatment outcomes of clotting factor concentrate therapy and identify barriers to hemophilia management in children.

Method: A retrospective study was conducted in patients aged from birth to 21 years who were diagnosed between January 1, 2020, and December 31, 2022. Data record included sex, age, date of birth, date of diagnosis, type of hemophilia, severity level, factor inhibitor level, type of treatment, age at the start of prophylaxis treatment, frequency and dose of prophylaxis treatment, factor injector provider, annual bleeding rate during episodic clotting factor concentrate and prophylaxis treatment, number of hospital visits, annual bleeding rate after continuous prophylaxis treatment with a follow-up period of 1 year, target joint and obstacles affecting hemophilia treatment. The data were analyzed using descriptive statistics.

Results: Among 18 patients diagnosed with hemophilia at Khon Kaen Hospital, the median annual bleeding rate after continuous prophylactic treatment was 0 episode/year and was 2 episodes/year for episodic treatment. After 1 year of follow-up, patients who received continuous prophylactic treatment showed a statistically significant reduction in annual bleeding rates (–p value < 0.002). The main barrier to treatment in patients was the fear of needles by questionnaire.

Conclusion: The annual bleeding rate after continuous prophylaxis treatment decreased significantly compared to episodic treatment. Additionally, after 1 year follow up of continuous prophylaxis treatment, the annual bleeding rate decreased significantly. The most common barrier to treatment in patients was the fear of needles.