Table 3.
Distribution of disease complications and comorbid conditions for the Gaucher disease population, overall and by ERT use, 2014
| Total, n (%) n=500 |
ERT+, n (%) n=206 |
ERT–, n (%) n=294 |
P values* | |
| Complications (at any time) | ||||
| Anaemia | 348 (69.6) | 158 (76.7) | 190 (64.6) | 0.004 |
| Thrombocytopaenia | 310 (62.0) | 152 (73.8) | 158 (53.7) | <0.001 |
| Hepatomegaly | 37 (7.4) | 22 (10.7) | 15 (5.1) | 0.019 |
| Splenomegaly | 78 (15.6) | 35 (17.0) | 43 (14.6) | 0.474 |
| Bone event | 202 (40.4) | 89 (43.2) | 113 (38.4) | 0.285 |
| Calcification of cardiac valves | 19 (3.8) | 8 (3.9) | 11 (3.7) | 0.935 |
| Pulmonary hypertension | 20 (4.0) | 12 (5.8) | 8 (2.7) | 0.082 |
| Comorbidities (at any time) | ||||
| Cancer | 111 (22.2) | 42 (20.4) | 69 (23.5) | 0.415 |
| Multiple myeloma | 5 (1.0) | 4 (1.9) | 1 (0.3) | † |
| Diabetes | 64 (12.8) | 26 (12.6) | 38 (12.9) | 0.920 |
| Gallstones | 54 (10.8) | 21 (10.2) | 33 (11.2) | 0.715 |
| Parkinsonism | 15 (3.0) | 7 (3.4) | 8 (2.7) | 0.663 |
| Peripheral neuropathy | 2 (0.4) | 1 (0.5) | 1 (0.3) | † |
ERT+, users of ERT; ERT–, non-users of ERT.
*Adjustment for multiple hypothesis testing reduces the p values threshold by the number of tests (n=11; to p=0.005).
†Comparison tests were not calculated for prevalence rates with low numbers and high variance.
ERT, enzyme replacement therapy.