Table 2.
Baseline data for all patients receiving agalsidase-beta, 1.0 mg/kg every other week
| Variable | Agalsidase-Beta Regular- Dose Group (ERT, 1.0 mg/kg) | Agalsidase-Beta Dose-Reduction Group (ERT, 0.5 mg/kg) | Agalsidase-Alfa Switch Group (ERT, 0.2 mg/kg) | Total |
|---|---|---|---|---|
| Patients (n) | 38 | 29 | 38 | 105 |
| Women | 12 (31.6) | 10 (34.5) | 21 (55.3)a,b | 43 (40.9) |
| Age (yr) | 46.1±12.4 | 45.1±13.7 | 44.7±12.9 | 45.3±12.8 |
| Weight (kg) | 71.9±14.2 | 73.9±13.3 | 75.2±14.7 | 73.7±14.0 |
| Height (cm) | 174.9±8.4 | 173.7±8.9 | 170.5±10.8 | 173.0±9.5 |
| Heart rate (beats/min) | 65.7±11.1 | 63.9±10.1 | 66.9±10.4 | 65.6±10.5 |
| Systolic BP (mmHg) | 119.1±11.5 | 120.7±13.5 | 120.3±12.2 | 119.9±12.3 |
| Diastolic BP (mmHg) | 79.6±7.5 | 81.1±10.6 | 80.9±8.3 | 80.5±8.8 |
| Nonclassic mutation | 1 (2.6) | 0 | 1 (2.6) | 2 (1.9) |
| α-Galactosidase A activity (nmol/mg per ml)c | 0.06±0.07 | 0.06±0.09b | 0.12±0.12a,b | 0.08±0.09 |
| α-Galactosidase A activity (nmol/mg per ml) in menc | 0.03±0.01 | 0.03±0.01 | 0.02±0.01 | 0.03±0.01 |
| Patients receiving RAAS blocker | 13 (34) | 17 (58) | 9 (24) | 39 (37) |
| Patients receiving analgesic drugs | 7 (18) | 8 (28) | 8 (21) | 23 (22) |
| Duration of ERT (mo) | 31±24 | 45±25d | 41±33 | 39±29 |
| Clinical presentation | ||||
| Angiokeratoma | 21 (55) | 15 (52) | 15 (41) | 51 (49) |
| Edema | 11 (29) | 4 (14) | 8 (21) | 23 (22) |
| Gastrointestinal pain | 8 (21) | 6 (21) | 5 (13) | 19 (18) |
| Diarrhea (d/mo) | 1.5±5.1 | 2.0±5.7 | 0.8±1.7 | 1.4±4.4 |
| Hypohidrosis | 25 (66) | 22 (76) | 18 (47)b | 65 (62) |
| Cornea verticillata | 11 (29) | 12 (43) | 19 (50)a | 54 (40) |
| Tinnitus | 13 (34) | 7 (24) | 10 (26) | 30 (29) |
| Hypacusis | 8 (21) | 11 (38) | 6 (16) | 25 (24) |
| Paresthesia | 23 (61) | 18 (64) | 22 (58) | 63 (60) |
| Pain attacks | 6 (16) | 6 (21) | 3 (8.1) | 15 (14) |
| Chronic pain | 15 (40) | 15 (52) | 9 (24)b | 39 (37) |
| Pain crises | 8 (21) | 5 (17) | 7 (18) | 20 (19) |
| TIA/stroke | 4 (11) | 1 (3.4) | 2 (5.3) | 7 (6.7) |
| Fatigue | 6 (16) | 8 (28) | 11 (29) | 25 (24) |
| Disease severity scores | ||||
| DS3 | 13.5±8.3 | 12.2±8.4 | 14.6±8.4 | 13.5±8.3 |
| MSSI | 20.5±11.6 | 21.8±10.5 | 18.1±9.8 | 19.9±10.6 |
| Cardiac measures | ||||
| Dyspnea | 1 (2.6) | 0 | 0 | 1 (1) |
| NYHA class | ||||
| I | 19 (50) | 16 (55) | 24 (63) | 59 (56) |
| II | 12 (32) | 7 (24) | 8 (23) | 27 (27) |
| III | 6 (16) | 6 (21) | 3 (9) | 15 (15) |
| IV | 1 (2.6) | 0 | 0 | 1 (1) |
| LV diastolic diameter (mm) | 48.1±7.9a | 49.5±5.1 | 50.2±5.5 | 47.8±6.5 |
| LV systolic diameter (mm) | 30.7±6.6 | 31.2±6.7 | 33.0±7.7 | 30.0±6.4 |
| Septal diameter (mm) | 12.9±3.1 | 12.8±3.6 | 12.5±3.2 | 12.8±3.0 |
| Posterior wall diameter (mm) | 12.5±2.1 | 11.2±2.8d | 12.8±2.4 | 12.2±2.5 |
| Ejection fraction (%) | 63.1±8.3 | 61.3±7.1 | 61.5±7.4 | 62.0±7.6 |
| Myocardial mass (g/m2) | 95.7±35.8 | 91.5±34.7 | 82.8±26.9a,b | 90.3±32.6 |
| Diastolic function | ||||
| Normal | 18 (56) | 15 (60) | 15 (48) | 48 (46) |
| Relaxation abnormality | 9 (28) | 5 (20) | 9 (29) | 23 (22) |
| Pseudo-normal | 4 (13) | 4 (16) | 7 (23) | 15 (14) |
| Restriction | 1 (3.1) | 1 (3.4) | 0 | 2 (1.9) |
| ECG abnormalities | 10 (28) | 9 (31) | 7 (21) | 26 (26) |
| Pacemaker | 5 (13) | 4 (14) | 1 (2.8) | 10 (9.7) |
| Heart transplant | 0 | 0 | 0 | 0 |
| MRI late enhancement | 9 (60) | 11 (61) | 8 (62) | 28 (61) |
| Renal measures | ||||
| Albumin-to-creatinine-ratio (mg/g creatinine)c | 173 (0–375) | 273 (0–1010) | 114 (0–606) | 205 (0–1010) |
| Cystatin C GFR (ml/min per 1.73m2)c | 104 (18–158) | 109 (65–132) | 101 (52–151) | 103 (18–158) |
| eGFR (CKD-EPI) (ml/min per 1.73m2)c | 99 (63–119) | 98 (86–114) | 98 (66–112) | 99 (63–119) |
| Creatinine (mg/dl)c | 0.89 (0.50–3.40) | 0.80 (0.60–1.20) | 0.87 (0.60–2.30) | 0.90 (0.50–3.40) |
| Hemoglobin (mg/dl) | 13.8±1.2 | 13.6±1.2 | 13.4±1.3 | 13.6±1.2 |
| Dialysis | 6 (16) | 5 (17) | 5 (13) | 16 (15) |
| Kidney transplant | 1 (2.6) | 5 (17) | 3 (8) | 8 (7.6) |
| Neurologic measures | ||||
| CES-D score | 18.6±10.1 | 15.3±12.4 | 15.0±8.7 | 16.2±10.5 |
| GCPS 2 (maximum pain) | 4.3±3.7 | 4.1±2.6 | 4.0±3.3 | 4.1±3.1 |
| GCPS 5 (impairment) | 2.2±2.8 | 2.0±2.9 | 2.2±3.2 | 2.1±2.9 |
| NPSI sum score | 0.18±0.25 | 0.19±0.25 | 0.13±0.11 | 0.17±0.21 |
| Sural nerve | ||||
| SNAP (µV) | 16.2±8.5 | 18.4±11.0 | 17.4±12.9 | 17.3±10.6 |
| NCV (m/s) | 43.1±4.1 | 44.6±5.9 | 42.9±8.0 | 43.5±6.0 |
| CDT | −16.2±7.1 | −16.6±7.7 | −11.5±8.2 | −14.9±7.8 |
Categorical data are presented as number (percentage) of patients. Data expressed with a plus/minus sign are the mean±SD. RAAS, renin-angiotensin-aldosterone system; TIA, transient ischemic attack; DS3, Disease Severity; NYHA, New York Heart Association; LV, left ventricular; ECG, electrocardiographic; MRI, magnetic resonance imaging; CKD-EPI, CKD–Epidemiology Collaboration; CES-D, Center for Epidemiologic Studies-Depression scale; SNAP, sensory nerve action potential; NCV, nerve conduction velocity; CDT, cold detection threshold.
P<0.05 for agalsidase-beta, 1 mg/kg, versus agalsidase-alfa, 0.2 mg/kg, every other week.
P<0.05 for agalsidase-beta, 0.5 mg/kg, versus agalsidase-alfa, 0.2 mg/kg, every other week.
Expressed as median (range).
P<0.05 for agalsidase-beta, 1 mg/kg versus 0.5 mg/kg every other week.