Figure 1:
Demographic characteristics and clinical outcomes of people with mild or moderate haemophilia A
| MILD HA (N=34) | MODERATE HA (N=79) | |
|---|---|---|
| Age | ||
| Mean (SD) | 36.6 (12.0) | 41.4 (14.1) |
| Median (IQR) | 37.0 (27.0, 42.0) | 41.0 (29.0, 54.0) |
| Age, n (%) | ||
| 18-35 | 15 (44) | 31 (39) |
| 36-59 | 16 (47) | 39 (49) |
| >60 | 3 (9) | 9 (11) |
| Body mass index, n (%) | ||
| Underweight | 0 (0) | 1 (1) |
| Normal weight | 18 (53) | 48 (61) |
| Overweight | 16 (47) | 28 (35) |
| Obese | 0 | 2 (3) |
| Current treatment regimen, n (%) | ||
| On-demand | 10 (29) | 24 (30) |
| No recent FVIII treatmenta | 24 (71) | 55 (70) |
| Prophylaxis | 0 | 0 |
| ABR (all cause) | ||
| Bleeding reported, n (%) | 24 (71) | 67 (85) |
| Mean (SD) | 1.1 (0.9) | 2.9 (2.7) |
| Median (Q1, Q3) | 1.0 (0.0, 2.0) | 2.0 (1.0, 4.0) |
| ABR (spontaneous)b | ||
| Spontaneous bleeding reported, n (%) | - | 57 (72) |
| Mean (SD) | - | 0.88 (0.93) |
| Median (Q1, Q3) | - | 0.6 (0, 1.2) |
| ABR, n (%) | ||
| 0 | 10 (29) | 12 (15) |
| 1 | 14 (41) | 14 (18) |
| >2 | 10 (29) | 53 (67) |
| Baseline factor level (overall) | ||
| Mean (SD) | 21.0 (10.3) | 3.5 (1.2) |
| Median (Q1, Q3, range) | 20 (11, 30, 5.01-40) | 4 (3, 5, 1-5) |
| Baseline factor level in those reporting a bleeding event | ||
| Mean (SD) | 21.3 (9.8) | 3.4 (1.3) |
| Median (Q1, Q3, range) | 20 (15, 27, 5.1-37) | 3.3 (2.3, 5, 1-5) |
| Target joints | ||
| Mean (SD) | 0.1 (0.3) | 0.3 (0.7) |
| Median (Q1, Q3) | 0 | 0 |
| Target joints, n (%) | ||
| 0 | 30 (88) | 62 (79) |
| 1 | 4 (12) | 11 (14) |
| >2 | 0 | 6 (8) |
| Problem joints | ||
| Mean (SD) | 0.1 (0.4) | 0.7 (0.8) |
| Median (Q1, Q3) | 0 | 1.0 (0.0, 1.0) |
| Problem joints, n (%) | ||
| 0 | 30 (88) | 39 (49) |
| 1 | 3 (9) | 26 (33) |
| >2 | 1 (3) | 14 (18) |
| Chronic pain level, n (%) | ||
| None | 22 (65) | 21 (27) |
| Mild pain | 11 (32) | 41 (52) |
| Moderate pain | 1 (3) | 17 (22) |
| Severe pain | 0 | 0 |
| Anxiety, n (%) | 4 (12) | 16 (20) |
| Depression, n (%) | 1 (3) | 12 (15) |
Clinical outcomes of participants who completed the patient self-complete questionnaire, by haemophilia A severity
| MILD HA (N=19) | MODERATE HA (N=25) | |
|---|---|---|
| Age | ||
| Mean (SD) | 37.7 (13.1) | 39.6 (14.6) |
| Median (Q1, Q3) | 37.0 (27.0, 46.0) | 31.0 (28.0, 47.0) |
| Age, n (%) | ||
| 18-35 | 8 (42) | 14 (56) |
| 36-59 | 9 (47) | 7 (28) |
| >60 | 2 (11) | 4 (16) |
| Body mass index, n (%) | ||
| Underweight | 0 (0) | 0 (0) |
| Normal weight | 11 (58) | 13 (52) |
| Overweight | 8 (42) | 11 (44) |
| Obese | 0 | 1 (4) |
| Current treatment regimen, n (%) | ||
| On-demand | 8 (42) | 9 (36) |
| No recent FVIII treatmenta | 11 (58) | 16 (64) |
| Prophylaxis | 0 | 0 |
| ABR (all cause) | ||
| Mean (SD) | 1.3 (0.9) | 3.0 (2.7) |
| Median (IQR) | 1.0 (1.0, 2.0) | 2.0 (1.0, 4.0) |
| Target joints | ||
| Mean (SD) | 0.1 (0.3) | 0.3 (0.6) |
| Median (IQR) | 0 | 0 |
| Target joints, n (%) | ||
| 0 | 17 (90) | 20 (80) |
| 1 | 2 (11) | 3 (12) |
| >2 | 0 | 2 (8) |
| Problem joints | ||
| Mean (SD) | 0.1 (0.5) | 0.6 (0.7) |
| Median (IQR) | 0 | 0 |
| Problem joints, n (%) | ||
| 0 | 18 (95) | 13 (52) |
| 1 | 0 | 9 (36) |
| >2 | 1 (5) | 3 (12) |
| Chronic pain level, n (%) | ||
| None | 11 (58) | 5 (20) |
| Mild pain | 8 (42) | 14 (56) |
| Moderate pain | 0 | 6 (24) |
| Severe pain | 0 | 0 |
| Anxiety, n (%) | 2 (11) | 3 (12) |
| Depression, n (%) | 0 | 2 (8) |