Table 1 Clinical and laboratory data of parkinsonian patients with Gaucher disease (GD)
Examinations | Case 1 | Case 2 | Case 3 | Case 4 |
---|---|---|---|---|
ND = not determined; OMA = ocular movement analysis; VEP = visual evoked potentials; auditory brain responses; SPET = single-photon emission tomography; ERT = enzyme replacement therapy. | ||||
Sex | M | F | F | F |
Genotype | N370S/L444P | G377S/G377S | N370S/? | N370S/? |
Gaucher diagnosis, y | 22 | 23 | 53 | 64 |
Onset of parkinsonian symptoms, y | 44 | 43 | 59 | 55 |
Follow-up, y | 11 | 7 | 4 | 8 |
Clinical examination at onset of GD1 symptoms | Lymphadenomegaly, hepatosplenomegaly, anemia, thrombocytopenia | Hepatosplenomegaly, bleeding, anemia, thrombocytopenia | Hepatosplenomegaly, anemia, thrombocytopenia | Hepatosplenomegaly, anemia, thrombocytopenia |
Neurological examination at onset of PD symptoms | Asymmetric onset (right), rigidity, and resting tremor | Asymmetric onset (right), rigidity, and resting tremor | Left limbs resting tremor and rigidity | Asymmetric onset (right) of rigidity, resting tremor, akinesia |
Splenectomy | No | Yes | Yes | No |
Ferritin, ng/mL; normal: man, 28–397; woman, 10–159 | 1320 | 831.6 | 1277 | ND |
β-glucosidase, nmol/mg/h; normal: 11.37 ± 2.85 | 2.03 | 2.15 | 1.57 | 1.42 |
EEG | Slow left temporal activity | Diffuse nonspecific encephalic sufferance | Normal | ND |
OMA | Normal | Normal | Normal | ND |
VEP | Latency normal, reduced amplitude | Normal | Normal | ND |
ABR | Mild latency of I–III waves | Mild latency of I–III waves | Normal | ND |
Audiometry | Perceptive hearing loss | Perceptive hearing loss | Normal | ND |
Cerebral RMN | Mild cortical atrophy | Mild cortical atrophy | Normal | ND |
Cerebral SPET | ND | Left basal ganglia hypoperfusion | ND | ND |
ERT, alglucerase/imiglucerase, y | 51 | 44 | 58 | No |
l-Dopa efficacy | Good transient | Good transient | Good | Good |
Surgical therapy | Bilateral pallidotomy: transient efficacy | Deep brain stimulation: efficacy | No | No |
Effect of ERT on PD | None | None | None | ND |