Table 3.
Description of the constraint period limited to duration of first stable modification*
| Patients who discontinued imiglucerase (N = 61) | Patients receiving a reduced imiglucerase dose (N = 32) | |||||
|---|---|---|---|---|---|---|
| Imiglucerase treatment | ||||||
| n | Median (range) | n | Median (range) | |||
| Time between treatment start and first modification (years) | 9.7 (1; 14) | 8.4 (1; 13) | ||||
| Duration of the limited period of constraint (months) | 5.1 (3; 15) | 6.0 (3; 15) | ||||
| Imiglucerase dose (U/kg/4-weeks): | ||||||
| At treatment initiation | 58 | 118.5 (51; 246) | 31 | 113.1 (58; 267) | ||
| At pre-constraint timepoint | 56 | 89.4 (10; 223) | 32 | 94.7 (46; 239) | ||
| At first modification | 61 | 0.0 (0.0; 0.0) | 32 | 52.4 (32; 181) | ||
| Dose decrease from pre-constraint dose (%): | n (%) patients | |||||
| <25% | 8 (25.0%) | |||||
| ≥25%; <50% | 13 (40.6%) | |||||
| ≥50%; <75% | 11 (34.4%) | |||||
| Evolution of biological parameters | ||||||
| n | Slope Estimate (SE) | Time-effect (p-value) | n | Slope Estimate (SE) | Time-effect (p-value) | |
| Hemoglobin (g/L) | 47 | −0.8 (0.2) | 0.003 | 26 | 0.6 (0.4) | 0.196 |
| Platelets (103/mm) | 47 | −5905 (1721) | 0.002 | 26 | 1762 (1720) | 0.322 |
| Serum ferritin (μg/L) | 22 | 12.4 (6.4) | 0.078 | 10 | −3.4 (13.3) | 0.812 |
| Chitotriosidase (nmol/mL/h) | 31 | 537 (208) | 0.021 | 15 | 179 (259) | 0.510 |
| ACE (IU/L) | 16 | 3.6 (1.1) | 0.012 | 11 | −2.8 (1.9) | 0.228 |
| Spontaneously reported GD-related events (by MedDRA Preferred Term) | ||||||
| Number (%) of patients | Number (%) of patients | |||||
| All events | 15 (24.6%) | 7 (21.9%) | ||||
| Bone events | ||||||
| Bone infarction | 1 (1.6%) | - | ||||
| Hand fracture | 1 (1.6%) | - | ||||
| Hematological events | ||||||
| Thrombocytopenia | 3 (4.9%) | 1 (3.1%) | ||||
| Pancytopenia | 1 (1.6%) | - | ||||
| Epistaxis | 1 (1.6%) | 1 (3.1%) | ||||
| Gingival bleeding | 1 (1.6%) | - | ||||
| Hematoma | 1 (1.6%) | - | ||||
| Visceral events | ||||||
| Hepatomegaly | - | 1 (3.1%) | ||||
| Other events | ||||||
| Asthenia/ Fatigue | 4 (6.6%) | 3 (9.4%) | ||||
| Bone pain | 2 (3.3%) | 1 (3.1%) | ||||
| Musculoskeletal pain | - | 1 (3.1%) | ||||
| Arthralgia | 1 (1.6%) | - | ||||
| Serum ferritin increased | - | 2 (6.3%) | ||||
| ACE increased | - | 1 (3.1%) | ||||
| Chitotriosidase increased | 1 (1.6%) | - | ||||
| Parkinson’s disease | 1 (1.6%) | - | ||||
| Stress | 1 (1.6%) | - | ||||
*”First stable modification” refers to discontinuation or reduced dose for at least 3 consecutive months.
Numerical values in bold print indicate statistical significance.