# Emicizumab prophylaxis in current haemophilia A care in the Czech Republic—data from the Czech National Haemophilia Programme Registry

## TL;DR

This study shows emicizumab significantly reduces bleeding in hemophilia A patients in the Czech Republic, including those with and without inhibitors.

## Contribution

Provides real-world evidence from a national registry on emicizumab's effectiveness in reducing bleeds in hemophilia A patients.

## Key findings

- Total annualized bleeding rate dropped from 3.44 to 0.46 after emicizumab initiation.
- 60.3% of patients had no reported bleeding while on emicizumab.
- Younger patients and those with inhibitors saw the most significant improvement.

## Abstract

Nonfactor therapy with emicizumab has become an important part of the haemophilia A treatment landscape recently.

We aimed to analyze data on the transition from previous treatment regimens to emicizumab in routine clinical practice in the Czech Republic.

Retrospective analysis of data from the Czech National Haemophilia Programme (CNHP) registry of all consecutive pediatric and adult persons treated with emicizumab prophylaxis. We evaluated bleeding control, injection frequency, and drug consumption in the period before and after emicizumab initiation.

Seventy-three persons with severe haemophilia A, median (IQR) age 4 (1-18) years, treated with emicizumab were reported in the CNHP registry up to the end of 2023. Two age categories were used for further stratification, 0-11 (n = 45) and 12+ (n = 28) years. For the whole group, we observed a significant reduction in total annualized bleeding rate (ABR) from a mean of 3.44 to 0.46 (P < .001), in joint ABR from 0.70 to 0.27 (P = .004), and in spontaneous ABR from 1.18 to 0.19 (P = .002) after starting emicizumab. The proportion of persons with no reported bleeding increased from 26.0% to 60.3% (P < .001). A sub-analysis, focused on inhibitors presence (present in 27 cases) and age, showed a more significant effect of emicizumab switch in observed parameters in persons with inhibitors and in the younger age group.

The retrospective analysis of nation-wide data from the CNHP registry supports the fact that emicizumab has the potential to reduce bleeding and to provide an opportunity for a significant proportion of patients, with and without factor VIII inhibitors, to achieve zero bleeds.

•Emicizumab has become an important part of the treatment landscape for haemophilia A.•Analysis of data from the Czech national registry of all consecutive persons on emicizumab.•We observed significant increase in the proportion of persons with zero bleeds on emicizumab.•Our data strongly support preference of emicizumab prophylaxis in various clinical scenarios.

Emicizumab has become an important part of the treatment landscape for haemophilia A.

Analysis of data from the Czech national registry of all consecutive persons on emicizumab.

We observed significant increase in the proportion of persons with zero bleeds on emicizumab.

Our data strongly support preference of emicizumab prophylaxis in various clinical scenarios.