Outcomes of prophylactic treatment with factor VIII concentrate in hemophilia A patients in Lampang Hospital

Abstract

Background: Hemophilia is a congenital bleeding disorder. Most patients are diagnosed with hemophilia A resulting from a deficiency of clotting factor VIII. Bleeding has an effect on physical health and quality of life. The principle of cares includes bleeding prevention and treatment with factor replacement therapy. There are 2 factor replacement therapy protocols. They are episodic (on demand) treatment and continuous prophylactic treatment. Since 2013, Lampang Hospital has provided factor VIII concentrate as continuous prophylactic treatment for moderate and severe hemophilia patients.

Objectives: The primary objective was to study the clinical outcomes before and after prophylactic treatment with factor VIII concentrate in hemophilia A patients. The secondary objective was to study the quality of life after prophylactic treatment.

Methods: This research was a retrospective historical cohort study and a cross-sectional study. Data were gathered from Lampang Hospital medical records for all of hemophilia A patients who received factor VIII concentrate as continuous prophylactic treatment between 1 January 2014 and 31 December 2019. Medical and humanistic outcomes were analyzed and compared between before and after prophylactic treatment.

Results: There were 18 hemophilia A patients enrolled, all were male (100%), 13 (72%) patients were severe hemophilia A, and 5 (28%) patients were moderate hemophilia A. The median ages at diagnosis and at data collection were 20 months and 17 years respectively. Significantly higher amount of factor VIII concentrate used (p-value <0.001) and shorter length of hospital stay (p-value 0.007) were found after prophylactic treatment while hospitalization rate per year and bleeding episodes per year were not different. Among these 18 cases, 5.6% had HIV infection, 11.1% had hepatitis B virus infection, and 16.7% had hepatitis C virus infection. Three cases (17.6%) discovered factor VIII inhibitor  (33.3% with low titer and 66.7% with high titer). In joint arthropathy evaluation, from radiologic imaging by Arnold-Hilgartner classification, the most common staging of knees and ankles was 0 (normal joint), from hemophilia joint health score version 2.1, the median score was 15/124. For quality of life score, from Thai EQ-5D-5L, the mean utility score was 0.8 (SD 0.2) and the mean visual analog scale score was 79 (SD 18.6). From Thai SF-36, the highest quality of life score was mental health (mean + SD = 78.9 + 15.5), and the lowest quality of life score was physical function (mean + SD = 65.0 + 24.5).                        

Conclusion: After prophylactic treatment, analysis in medical outcomes showed the length of hospital stay was significantly shorter. Quality of life scores were close to the best score. From Thai SF-36, the highest score was mental health and the lowest score was physical function.