An observational study to investigate the relationship between plasma glucosylsphingosine (lyso-Gb1) concentration and treatment outcomes of patients with Gaucher disease in Japan

Table 2 Summary of achievement of therapeutic goals

Therapeutic goal	N = 20
Number of therapeutic goals achieved
≤ 1	0 (0)
2	1 (5.0)
3	0 (0)
4	3 (15.0)
5	2 (10.0)
6	14 (70.0)
Hepatomegaly score, achieved (score = 0)	17 (85.0)
0	17 (85.0)
1	3 (15.0)
2	0 (0)
3	0 (0)
Splenomegaly score, achieved (score = 0)	18 (90.0)
0	18 (90.0)
1	1 (15.0)
2	0 (0)
3	1 (5.0)
Anemia, hemoglobin count (g/dL), n	20
Achieved^a	17 (85.0)
Mean (SD)	12.7 (1.9)
Median (min–max)	12.5 (7.8–15.9)
Thrombocytopenia, platelet count (× 10³/μL), n	20
Achieved^b	18 (90.0)
Mean (SD)	248.2 (102.4)
Median (min–max)	233.0 (24.0–473.0)
Bone pain, achieved (absent or very mild, or mild)	18 (90.0)
Absent or very mild pain	17 (85.0)
Mild pain	1 (5.0)
Moderate pain	2 (10.0)
Severe pain	0 (0)
Intolerable pain	0 (0)
Bone crisis, achieved (absent)	20 (100)
Absent	20 (100)
Present	0 (0)

Data are n (%), unless otherwise stated
^aTreatment goal for anemia was achieved if hemoglobin level was ≥ 11.0 g/dL for children ≤ 12 years and female ≥ 13 years, and ≥ 12.0 g/dL for males ≥ 13 years
^bTreatment goal for thrombocytopenia was achieved if platelet count was > 120 × 10³/μL for those with platelet count ≥ 60 × 10³/μL at the first infusion of velaglucerase alfa or if no data at the first velaglucerase alfa infusion were available; or ≥ 2 times the platelet count at first velaglucerase alfa infusion for those with platelet count < 60 × 10³/μL at the first infusion of velaglucerase alfa
max, maximum; min, minimum; SD, standard deviation

ISSN: 1750-1172