Emicizumab: Reshaping Lives

Abstract

Objective: To determine the effectiveness of Emicizumab in patients with high annual bleeding rate (ABR) and in patients who needed rehabilitation (physical/psychological) along with any side effects.

Methodology: This prospective cohort study was conducted at Hemophilia Patients Welfare Society (HPWS) Lahore Chapter. A total of 37 patients were followed up for a period of 1 year after starting treatment with Emicizumab. Mean annual bleeding rate (ABR) per patient, attendance status at School/college/job, mental health issues, number of hospital visits and Hemophilia joint health society score (HJHS score) in last 2 years prior to the start of therapy were recorded and were then compared with the results 1 year after the start of treatment. Data was analyzed using SPSS version 23.00. Mean and standard deviation were calculated for continuous variables, median and IQR were calculated for discrete data. Frequencies and percentages were calculated for categorical variables. Shapiro-wilk test was used to test the normality of the data. Wilcoxon-signed rank test was used evaluate the difference in ABR and HJHS before and after treatment.  A p-value of less than 0.05 was considered as significant.

Results: After start of treatment patients showed 100% improvement in their attendance status. Only 6 patients (16.2%) had major breakthrough bleeds at different sites after start of treatment. The median Hemophilia Joint Health Society Score (HJHS) reduced from 8 (IQR: 8-15) to 7 (IQR: 5.5-14.5) p value = 0.001. The median number of hospital visits per year were 6 (IQR: 2-12) before the start of treatment and after intervention the median number of visits reduced to 0 (IQR: 0-0). The ABR median values before start of treatment were 3.5 (IQR: 0.5-8.25) and after start of treatment the median value reduced to 0 (IQR: 0-0) (p value <0.001).

Conclusion: Emicizumab has a promising role in enhancing the quality of life of hemophiliacs, thereby we strongly suggest the last scale local production and use of Emicizumab