Bridging the Gap: Efficacy of Low-Dose Emicizumab in Haemophilia A With or Without Inhibitors.

INTRODUCTION

Emicizumab has proven to be a turning point in the management of patients with Haemophilia A (HA). In resource-limited settings, data on low-dose emicizumab prophylaxis (LDEP) in HA patients are emerging.

METHODS

Thirty-seven patients previously on factor FVIII prophylaxis or bypassing agents (BPAs) received LDEP at a dose of 3-mg/kg SC for 4 weeks, followed by 1.5-mg/kg SC every 2 weeks. The primary objective was to assess the efficacy of LDEP measured in the form of reduction in annualised bleed rates (ABR), treated joint bleed and conversion of target joint to non-target joint. The secondary objectives were to study the improvement in joint scores (HJHS and FISH score) and to assess quality of life.

RESULTS

The median duration of LDEP was 15 months. On LDEP, the mean ABR was 0.41 ± 0.7 (median = 0; IQR: 0-0.6). Sixty-seven percent of patients had zero bleeds, and 66% of target joints converted to non-target joints. There was a significant improvement in HJHS, FISH and QoL in patients.

CONCLUSION

LDEP, although not optimal, remains an effective strategy in the prevention of bleeds, improvement of QOL and reduction of joint-related disability in HA patients in resource-constrained settings.