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Table 1 Patients’ demographic and clinical data

From: Chronic pain in Gaucher disease: skeletal or neuropathic origin?

Characteristic N = 25
Female n° (%) 12 (48)
Male n° (%) 13 (52)
Age at start ERT (years)  
 Mean ± SD 27.2 ± 14.6
 Range 3–60
Duration of treatment (years)  
 Mean ± SD 15.0 ± 6.4
 Range 0–22
Patients ≤16y old at start of treatment, n° (%) 7 (31.8)
ERT variations and therapeutic results
  Baseline - T0 Follow-up evaluation - Tx  
  Mean ± SD (Range) Mean ± SD (Range) P
ERT dose (U/Kg/Month) 35.3 ± 14.2 (15–60) 66.5 ± 22.5 (35–115) -
Hemoglobin (g/dl) 11.9 ± 1.5 (9.4–14.8) 14.3 ± 1.6 (11.8–17.4) <0.01
Platelets (×10.000/mm3) 155.7 ± 101.3 (42–379) 214.3 ± 94.4 (107–526) <0.05
Gammaglobulin (mg/dL) 1632.5 ± 877.1 (587–4632) 1164.5 ± 339.6 (570–1832) <0.01
Liver volume (MN)* 1.99 ± 0.82 (0.8–3.6) 0.93 ± 0.20 (0.53–1.33) <0.01
Spleen volume (MN)* 11.48 ± 6.22 (1.3–20.3) 2.97 ± 1.59 (0.92–5.62) <0.01
BMD (Z-score) −1 ± 1.40 (−3.36;0.8) −0.26 ± 1.28 (−2.9;1.7) -
Pain 13   13   
Bone crisis 7   -   
Zimran severity score index**      
Mild (≥0 to 10) 16   21   <0.05
Moderate (≥11 to 25) 5   1   
Severe (>25) 1   -   
  1. *MN: multiple of normal value
  2. **Zimran Z-score was available for 22 patients
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