Table 1 Patients’ demographic and clinical data

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

-