Abstract Number: PB0634
Meeting: ISTH 2021 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: Emicizumab is a humanized bispecific monoclonal antibody that bridges activated factors IX and X to restore the function of factor VIII. It is indicated for prophylaxis of bleeding episodes in patients with haemophilia A.
Aims: To describe the effectiveness and safety profile of emicizumab in patients with haemophilia A in clinical practice. We also evaluated the economic impact of emicizumab in comparison with previous treatments.
Methods: A retrospective and descriptive study was conducted involving patients treated with emicizumab between July 2020 and February 2021 in a tertiary hospital. Data were obtained from medical and pharmacy claim records. We collected demographic and clinical variables (sex, age, presence of FVIII inhibitor and haemophilic arthropathy) and related to previous and current therapy (dosification, number of bleeding events and adverse effects). We considered the wholesale acquisition cost of pharmacologic treatments within the Spanish public healthcare system.
Results: Five patients were included. All of them were men with a mean age of 32 (20-48) and haemophilic arthropathy. Only one patient (No.3) presented FVIII inhibitors at baseline. Mean number of days under emicizumab treatment was 125 (56-224). Main outcomes are depicted at Table 1.
| TABLE 1 | ||||||
| Patient | PREVIOUS TREATMENT | EMICIZUMAB TREATMENT | ||||
| Coagulation factor | Dosification | Bleeding episodes | Dosification | Days under treatment | Bleeding episodes | |
| 1 | Rurioctocog | 3000 UI/72h | 5 | 240 mg/2w | 224 | 1 |
| 2 | Efmoroctocog | 3000 UI/48h | 1 | 300 mg/2w | 161 | 0 |
| 3 | von Willebrand factor/FVIII | 3000 UI/48h |
0 | 195 mg/2w | 126 | 0 |
| 4 | Efmoroctocog Moroctocog |
3000 UI/48-72h 3000 UI/48h |
1 | 270 mg/2w | 56 | 0 |
| 5 | PEGylated- Damoctocog | 3000 UI/72h | 0 | 127 mg/w | 58 | 1 |
Concerning effectiveness, four patients achieved clinical response. However, in one patient (No.5) emicizumab treatment was stopped due to lack of effectiveness (haematuria) and a severe injection site reaction. The other four patients did not experience any remarkable side effects.
As for costs, emicizumab treatment was more cost-effective in comparison to previous treatments. Due to confidentiality reasons, we are not allowed to disclose the exact acquisition costs.
Conclusions: Our results suggest that emicizumab is a cost-effective treatment for haemophilia A. Despite the favourable safety profile observed in clinical trials, our results show that severe adverse effects can appear and contribute to treatment discontinuation. Further studies are warranted to establish the effectiveness and safety in clinical practice.
To cite this abstract in AMA style:
Riera A, Plaza A, Pagès N, Masip M, Riera P, Carrasco M, Mateo J, Vilalta N. Effectiveness, Safety and Cost Assessment of Emicizumab in Clinical Practice [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/effectiveness-safety-and-cost-assessment-of-emicizumab-in-clinical-practice/. Accessed November 29, 2023.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/effectiveness-safety-and-cost-assessment-of-emicizumab-in-clinical-practice/