Survey Study for Velaglucerase Alfa (VPRIV) in Japan

An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event.

Number of participants of efficacy categories in assessment of change from baseline in hemoglobin concentration during the study was reported. The efficacy categories were classified as Good: increased by at least 1.5g/dL, Moderate response: increase of at least 0.5 g/dL and less than 1.5 g/dL, and Ineffective: increase less than 0.5 g/dL.

Number of participants of efficacy categories in assessment of change from baseline in platelet counts during the study was reported. The efficacy categories were classified as Good: increase of at least 30 × 10^9/L, Moderate response: increase of at least 15 × 10^9/L and less than 30 × 10^9/L, and Ineffective: less than 15 × 10^9/L.

Number of participants of efficacy categories in assessment of change from baseline in liver volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.

Number of participants of efficacy categories in assessment of change from baseline in spleen volumes during the study was reported. The efficacy categories were classified as Good: reduction of at least 30%, Moderate response: reduction of at least 10% and less than 30%, and Ineffective: less than 10% reduction.

T-scores were the number of standard deviations (SDs) above or below the average for a young adult at peak BMD. A T-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD T-scores and femur neck BMD T-scores related to bone density at baseline and the last data collection.

Z-scores express the BMD as the number of SDs above or below the average BMD of a healthy participant of the same age and gender. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. The reported data in this outcome measure was lumbar spine BMD Z-scores and femur neck BMD Z-scores related to bone density at baseline and the last data collection.

The reported data in this outcome measure was lumbar spine BMD and femur neck BMD related to bone density at baseline and the last data collection.

Number of participants with positive anti-velaglucerase alfa antibody test results was reported.

An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is any event that results in: death; life-threatening; requires inpatient hospitalisation or results in prolongation of existing hospitalisation; persistent or significant disability/incapacity; a congenital anomaly/birth defect or a medically important event. Adverse drug reaction refers to AE related to administered drug.

An AE is any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Adverse drug reaction refers to AE related to administered drug. Infusion reactions were defined as reactions occurring up to 24 hours after the start of the infusion.