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Table 2 Baseline a clinical characteristics for all imiglucerase-treated Latin American pediatric patients with type 1 Gaucher disease

From: Long-term effect of imiglucerase in Latin American children with Gaucher disease type 1: lessons from the International Collaborative Gaucher Group Gaucher Registry

Patients enrolled

443

 

Anemia b , n (%)

N = 353

 

Yes

189 (54)

 

No

164 (46)

 

Thrombocytopenia c (Platelet count, ×10 9 /L)

[Non-splenectomized patients only], n (%)

N = 339

 

Mild or none (≥120)

140 (41)

 

Moderate (60 to <120)

154 (45)

 

Severe (<60)

45 (13)

 

Splenomegaly (Spleen volume in multiples of normal [MN]), n (%)

n = 57

 

Mild or None (≤5)

2 (4)

 

Moderate (>5 to ≤15)

13 (23)

 

Severe (>15)

42 (74)

 

Qualitative splenomegaly d (Palpation), n (%)

n = 221

 

Yes

204 (96.7)

 

No

7 (3.3)

 

Hepatomegaly (Liver volume in MN), n (%)

n = 37

 

Mild or None (≤1.25)

5 (14)

 

Moderate (>1.25 to ≤2.5)

26 (70)

 

Severe (>2.5)

6 (16)

 

Qualitative hepatomegaly d (Palpation), n (%)

n = 230

 

Yes

204 (88.7)

 

No

26 (11.3)

 

Bone pain, n (%)

n = 248

 

Absent

160 (65)

 

Present

88 (35)

 

Prior bone crisis, n (%)

n = 242

 

Absent

212 (88)

 

Present

30 (12)

 

Radiologic bone disease, n (%) (Evidence of any bone disease)

n = 149

 

Absent

42 (28)

 

Present

107 (72)

 

Type of bone disease reported

Any data available, n

Abnormality present, n (%)

Avascular necrosis

103

20 (19)

Erlenmeyer flask deformity

110

65 (59)

Fractures

81

2 (2)

Infarction

97

22 (23)

Lytic lesions

99

20 (20)

Marrow infiltrations

75

56 (75)

Osteopenia

98

56 (57)

Decreased bone mineral density (Lumbar spine dual energy x-ray absorptiometry Z-score e ), n (%)

n = 19

 

Mild or None (≤ −1)

6 (32)

 

Moderate (> −2.5 to ≤ −1)

9 (47)

 

Severe (> −2.5)

4 (21)

 

Pediatric growth retardation, n (%)

n = 225

 

Observed

102 (45)

 

Expectedf

11 (5)

 
  1. a‘Baseline’ is defined as the data point closest to the first infusion date, at all time prior to first infusion through 2 weeks (inclusive) following first infusion. Patients with no infusion date were excluded from the analysis for each hematological, visceral and bone assessment.
  2. bAnemia 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; <10.1 g/dL for children younger than 6 months of age.
  3. cAmong the 12 partial or total splenectomy patients, thrombocytopenia was classified as mild or none in 11 (92%), moderate in 1 (8%), and severe in 0 (0%).
  4. dQualitative splenomegaly and hepatomegaly are determined by palpation on the basis of a yes/no response.
  5. eStandard deviations of age and sex-adjusted norms.
  6. fPediatric growth retardation is defined as the number of patients who are below the 5th percentile for height based on the age and gender of the normal healthy population and is calculated as 0.05 X the total number of patients.
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BMC Hematology

ISSN: 2052-1839

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