Abstract Number: PB1141
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Novel Biotherapeutics in Hemophilia
Background: Hemophilia A (HA) may be inherited or arise from spontaneous mutation. The development of inhibitory alloantibodies results in impairment of quality of life (QoL). The advent of emicizumab offers the potential for a paradigm shift in hemophilia treatment. There is a paucity of real-world data on emicizumab.
Aims: Therefore, we aimed to determine the demographic characteristics of our patients and to study their outcome.
Methods: This was a retrospective review of HA patients treated with emicizumab in East Malaysia between June 2018 and December 2019. The primary endpoint was reduction of bleeding events. The secondary endpoints included:
(a) improvement in QoL assessed using Haemo-QoL-A questionnaire; and
(b) adverse events.
Results: Three patients were started on emicizumab (Table 1). All were severe HA patients with inhibitors. The median duration of treatment was 56 weeks, ranging from 27 to 72 weeks. All patients were given 3mg/kg every 2 weeks. Two had achieved 100 percent reduction in bleeding events after receiving emicizumab. One had one episode of minor spontaneous bleed one day prior to the scheduled dose, which fully resolved after emicizumab administration without requiring any bypassing agents. All of those who required walking aids prior to emicizumab (two patients) were able to ambulate independently after receiving emicizumab. QoL assessment was shown in Table 2. None had any adverse events. No thromboembolic events were reported.
Conclusions: The results demonstrated that emicizumab has a favourable safety profile with encouraging efficacy in patients with HA. Study limitations include a small sample size and the results were generally descriptive. Additional studies are warranted to establish clinical data on its safety and to define suitable assays for hemostatic monitoring.
| Total | |
| Patients (n) | 3 |
| Gender, n (%) Male Female | 3 (100%) 0 (0%) |
| Age at diagnosis (months), median (range) | 12 (1.0 to 132.0) |
| Ethnicity, n (%) Chinese Malay Sarawak native | 1 (33.3%) 1 (33.3%) 1 (33.3%) |
| Inhibitor level (BU), median (range) | 3.0 (1.6-20.0) |
| Type of treatment Prior to Emicizumab, n (%) On demand Prophylaxis | 2 (66.7%) 1 (33.3%) |
[Table 1 Baseline characteristics of patients]
| Before emicizumab | After emicizumab | Difference | p-value | |
| Physical functioning | 11.87 (±8.44) | 77.77 (±3.87) | 65.90 (±11.23) | 0.010 |
| Role functioning | 29.03 (±13.74) | 81.73 (±11.06) | 52.70 (±15.86) | 0.029 |
| Worry | 46.67 (±39.46) | 84.00 (±10.58) | 37.33 (±34.49) | 0.202 |
| Consequences of bleeding | 17.13 (±15.04) | 75.20 (±14.67) | 58.07 (±26.52) | 0.063 |
| Emotional impact | 47.80 (±23.38) | 73.30 (±5.77) | 25.50 (±18.92) | 0.144 |
| Treatment concern | 37.80 (±42.26) | 64.40 (±36.75) | 26.60 (±23.04) | 0.184 |
| Overall transformed score | 31.72 (±12.95) | 76.10 (±2.27) | 44.38 (±10.68) | 0.019 |
[Table 2 Health-related quality of life scores by 6 domains (Haemo-QoL-A)]
To cite this abstract in AMA style:
Tang ASO, Yeo ST, Tan XY, Ko CT, Chew LP. Emicizumab in the Management of Hemophilia A: Real World Experience in East Malaysia [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/emicizumab-in-the-management-of-hemophilia-a-real-world-experience-in-east-malaysia/. Accessed September 21, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/emicizumab-in-the-management-of-hemophilia-a-real-world-experience-in-east-malaysia/