Table 5.

Bone manifestations at baseline^a for GD1 patients with and without Parkinsonism, after matching.

	Patients without Parkinsonism reported	Patients with Parkinsonism reported	p-value
Patients enrolled	649	68
Bone pain, n (%)	n = 320	n = 42	0.6500
Absent	118 (36.9)	17 (40.5)
Present	202 (63.1)	25 (59.5)
Bone crisis, n (%)	n = 273	n = 31	0.5496
Absent	207 (75.8)	25 (80.6)
Present	66 (24.2)	6 (19.4)
Radiological bone disease, n (%)	n = 303	n = 37	0.7065
Evidence of any bone disease
Absent	12 (4.0)	1 (2.7)
Present	291 (96.0)	36 (97.3)

Type of bone disease reported	Any data available, n	Abnormality present, n (%)	Any data available, n	Abnormality present, n (%)
Avascular necrosis	155	77 (49.7)	19	5 (26.3)	0.0542
Erlenmeyer flask deformity	150	120 (80.0)	24	18 (75.0)	0.5745
Fractures	95	18 (18.9)	19	4 (21.1)	0.8319
Infarction	147	98 (66.7)	20	8 (40.0)	0.0201
Lytic lesions	78	31 (39.7)	13	6 (46.2)	0.6631
Marrow infiltration	163	153 (93.9)	18	18 (100.0)	0.2796
Osteopenia	149	127 (85.2)	20	17 (85.0)	0.9778
Decreased bone mineral density (lumbar spine DXA t-scoreb), n (%)	n = 49		n = 14		0.2309
Mild or none (> −1)	22 (44.9)		5 (35.7)
Moderate (>−2.5 to ≤−1)	10 (20.4)		6 (42.9)
Severe (≤ −2.5)	17 (34.7)		3 (21.4)

^aFor ‘never on imiglucerase’ patients, baseline is defined as the most recent date for which a data point for a given variable was available. Therefore different variables may have different assessment dates. For ‘ever on imiglucerase’ patients, baseline is defined as the data point closest to the first infusion date, no more than −2 years/+6 weeks (inclusive) from first infusion. Treated patients with no infusion date were excluded from the analysis for each bone assessment.

^bStandard deviations of age and gender-adjusted norms.