Table 4.
Hematological and visceral manifestations at baselinea for GD1 patients with and without Parkinsonism, after matching.
| Patients without Parkinsonism reported |
Patients with Parkinsonism reported |
p-value | |
|---|---|---|---|
| Patients enrolled | 649 | 68 | |
| Anemiab, n (%) | n = 519 | n = 44 | 0.0041 |
| Yes | 208 (40.1) | 8 (18.2) | |
| No | 311 (59.9) | 36 (81.8) | |
| Thrombocytopeniac (platelet count, × 103/mm3) [non-splenectomized patients only], n (%) | n = 293 | n = 33 | 0.0596 |
| Present (<150) | 263 (89.8) | 26 (78.8) | |
| Absent (≥150) | 30 (10.2) | 7 (21.2) | |
| Splenomegaly (spleen volume in multiples of normal), n (%) | n = 186 | n = 21 | 0.5997 |
| Mild or none (≤5) | 47 (25.3) | 6 (28.6) | |
| Moderate (>5 to ≤15) | 84 (45.2) | 11 (52.4) | |
| Severe (>15) | 55 (29.6) | 4 (19.0) | |
| Hepatomegaly (liver volume in multiples of normal), n (%) | n = 282 | n = 25 | 0.0897 |
| Mild or none (≤1.25) | 118 (41.8) | 13 (52.0) | |
| Moderate (>1.25 to ≤2.5) | 118 (41.8) | 12 (48.0) | |
| Severe (>2.5) | 46 (16.3) | 0 (0.0) |
For ‘never on imiglucerase’ patients, baseline is defined as the most recent date for which a data point for a given variable was available. Therefore different variables may have different assessment dates. For ‘ever on imiglucerase patients,’ baseline is defined as the data point closest to the first infusion date, no more than −3 months/+4 weeks (inclusive) from first infusion for hemoglobin/platelet, and −6 months/+6 weeks (inclusive) from first infusion for liver/spleen. Treated patients with no infusion date were excluded from the analysis for each hematological and visceral assessment.
Anemia is defined according to age and gender norms for hemoglobin concentrations as follows: <12 g/dL for males older than 12 years; <11 g/dL for females older than 12 years; <10.5 g/dL for children ages >2 to 12 years; <9.5 g/dL for children ages 6 months to 2 years; and <10.1 g/dL for children younger than 6 months of age.
For patients without Parkinsonism, among the 235 partial or total splenectomy patients, thrombocytopenia was classified as present in 65 (28%) and absent in 170 (72%). For patients with Parkinsonism, among the 11 partial or total splenectomy patients, thrombocytopenia was classified as present in 2 (18%) and absent in 9 (82%).