Table 1.
Baseline characteristics of the patient groups
| Characteristic | Regular-Dose (n=24) | Dose-Reduction-Switch (n=28) | Switch (n=37) | Total (N=89) |
|---|---|---|---|---|
| Patients | ||||
| Women | 3 (12.5)a,b | 13 (46.4) | 17 (46.0) | 33 (37.1) |
| Age, y | 43.5±11.3 | 47.4±12.7 | 46.1±13.9 | 45.5±12.5 |
| Weight, kg | 74.0±15.1 | 74.5±13.1 | 72.8±13.4 | 73.9±13.6 |
| Height, cm | 177.0±7.9 | 172.3±10.2 | 171.5±10.2 | 173.4±13.6 |
| Heart rate, bpm | 62.5±20.3 | 67.4±7.8 | 65.3±11.8 | 65.2±13.5 |
| Systolic BP, mmHg | 118.2±13.9 | 118.6±12.2 | 120.0±10.1 | 118.9±11.8 |
| Diastolic BP, mmHg | 78.9±10.4 | 79.0±11.6 | 80.5±8.4 | 79.5±10.1 |
| GLA activity, nmol/min per mg of protein | 0.04±0.05a,b | 0.09±0.12 | 0.10±0.10 | 0.08±0.10 |
| GLA activity in men, nmol/min per mg of protein | 0.02±0.01 | 0.03±0.01 | 0.02±0.01 | 0.02±0.01 |
| Diuretics | 6 (40.0) | 6 (30.0) | 9 (47.4) | 21 (38.9) |
| RAAS blockers | 9 (60.0) | 14 (73.7) | 12 (60.0) | 35 (64.8) |
| Analgesics | 4 (30.8) | 6 (31.6) | 6 (31.6) | 16 (31.4) |
| Premedication | 2 (11.8) | 2 (8.7) | 1 (2.9) | 5 (6.8) |
| Duration of ERT, mo | 45.5±22.9 | 44.0±27.9 | 42.6±27.9 | 44.2±26.3 |
| Fabry-typical manifestations and symptoms | ||||
| Angiokeratoma | 13 (65.0) | 11 (45.8) | 14 (43.8) | 38 (50.7) |
| Edema | 11 (55.5)b | 2 (8.3) | 8 (24.4) | 21 (27.6) |
| Gastrointestinal pain | 5 (25.0) | 3 (12.5) | 3 (9.1) | 11 (14.5) |
| Diarrhea | 5 (25.0) | 7 (30.4) | 4 (11.8) | 16 (20.8) |
| Hypohidrosis | 15 (75.0) | 16 (66.7) | 16 (51.6) | 47 (62.2) |
| Cornea verticillata | 12 (60.0) | 8 (33.3) | 15 (46.9) | 35 (45.3) |
| Tinnitus | 8 (40.0) | 6 (25.0) | 7 (21.2) | 21 (26.3) |
| Hypoacusis | 5 (25.0) | 7 (29.2) | 5 (15.2) | 17 (22.4) |
| Paresthesia | 15 (75.0) | 18 (75.0) | 21 (63.6) | 54 (69.7) |
| Pain attacks | 5 (25.0)a | 6 (25.0)c | 1 (3.0) | 12 (15.8) |
| Permanent pain | 8 (40.0) | 13 (54.2) | 6 (18.2) | 27 (35.5) |
| Pain crisis | 4 (20.0) | 3 (12.5) | 7 (21.2) | 14 (18.4) |
| TIA/stroke | 2 (8.3) | 0 (0.0) | 1 (3.0) | 3 (4.0) |
| Fatigue | 4 (20.0) | 6 (25.0) | 4 (12.1) | 14 (17.1) |
| Stroke/TIA | 2 (10.0) | 0 (0.0) | 1 (3.0) | 3 (3.9) |
| MSSI score | 26.5±12.1 | 18.5±9.5 | 17.6±8.3 | 20.3±10.4 |
Values are given as mean±SD or n (%). Differences between groups have been tested via one-way ANOVA for continuous variables or Fisher’s exact test for categorical variables. Because of the multicenter approach, some parameters were not available for the entire study cohort. RAAS, renin-angiotensin-aldosterone system; TIA, transitory ischemic attack.
a
P<0.05 for regular-dose versus switch group.
b
P<0.05 for regular-dose versus dose-reduction-switch group.
c
P<0.05 for dose-reduction-switch versus switch group.