| Journal of Hematology, ISSN 1927-1212 print, 1927-1220 online, Open Access |
| Article copyright, the authors; Journal compilation copyright, J Hematol and Elmer Press Inc |
| Journal website https://jh.elmerpub.com |
Short Communication
Volume 14, Number 5, October 2025, pages 257-261
Emicizumab in Severe Hemophilia A: Clinical and Patient Determinants of Transition Within a Standardized Program
Tables
| Characteristic | All (n = 69) |
|---|---|
| PWHA: patients with severe hemophilia A; PROBE: Patient-Reported Outcomes, Burdens and Experiences; IQR: interquartile range; HIV: human immunodeficiency virus; HCV: hepatitis C virus. | |
| Age (years) at time of drug availability, median (IQR) | 31 (26, 40) |
| > 2 h by car from Vancouver, n (%) | 25 (36.2) |
| Hemophilic arthropathy (6 joints involved), n (%) | |
| 0 - 1 joints | 36 (52.2) |
| 2 - 4 joints | 29 (42.0) |
| 5 or more joints affected | 4 (5.8) |
| HIV, n (%) | 10 (14.5) |
| HCV (chronic diagnosis), n (%) | 23 (34.3) |
| If HCV - cleared, n (%) | 21 (95.5) |
| Filled out EQ5D/PROBE survey, n (%) | 52 (75.4) |
| Composite EQ5D, median (IQR) | 0.854 (0.714, 0.942) |
| Composite PROBE score, median (IQR) | 0.791 (0.650, 0.907) |
| Transition time (months) | N | Minimum | Maximum | Median | IQR 25% | IQR 75% |
|---|---|---|---|---|---|---|
| IQR: interquartile range. | ||||||
| Early | 28 | 0 | 9 | 7 | 4.5 | 8 |
| Late | 41 | 10 | 21 | 12 | 11 | 14 |
| All | 69 | 0 | 21 | 10 | 7 | 13 |
| Variable | Regression coefficient | 95% confidence limits | P value |
|---|---|---|---|
| CI: confidence interval. | |||
| Intercept | 10.59 | 8.87, 12.31 | < 0.0001 |
| Age (years) at time of emicizumab availability | -0.15 | -0.23, -0.06 | 0.0008 |
| > 2 h by car from Vancouver | 0.75 | -1.43, 2.92 | 0.50 |
| Hemophilic arthropathy (reference: 0 - 1 joints) | |||
| 2 - 4 joints affected | -0.26 | -2.45, 1.94 | 0.81 |
| 5 or more joints affected | 6.50 | 1.84, 11.16 | 0.007 |
| History of adverse events on a previous factor concentrate | -5.52 | -11.94, 0.90 | 0.09 |