The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from another therapeutic agent for Gaucher disease.
Study Type
OBSERVATIONAL
Enrollment
63
Konan
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAE) Following Initiation of Treatment With Velaglucerase Alfa
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.
Time frame: Baseline up to end of the study (8 years)
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Hemoglobin Concentration
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.
Time frame: Baseline, Every 12 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Platelet Counts
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.
Time frame: Baseline, Every 12 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Liver Volumes
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.
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Kōnan, Aichi-ken, Japan
Nagoya
Nagoya, Aichi-ken, Japan
Kitakyushu
Kitakyushu, Fukuoka, Japan
Kurume
Kurume, Fukuoka, Japan
Fukuyama
Fukuyama, Hiroshima, Japan
Higashihiroshima
Higashihiroshima, Hiroshima, Japan
Obihiro
Obihiro, Hokkaido, Japan
Sagamihara
Sagamihara, Kanagawa, Japan
Sendai
Sendai, Miyagi, Japan
Suita
Suita, Osaka, Japan
...and 25 more locations
Time frame: Baseline, Every 24 weeks up to 8 years
Number of Participants of Efficacy Categories in Assessment of Change From Baseline in Spleen Volumes
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.
Time frame: Baseline, Every 24 weeks up to 8 years
Lumbar Spine Bone Mineral Density (BMD) T-scores and Femur Neck BMD T-scores at Baseline and the Last Data Collection
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.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Lumbar Spine BMD Z-scores and Femur Neck BMD Z-scores 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.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Lumbar Spine BMD and Femur Neck BMD 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.
Time frame: Baseline, at the time of last data collection (up to 8 years)
Number of Participants With Positive Anti-velaglucerase Alfa Antibody Test Results
Number of participants with positive anti-velaglucerase alfa antibody test results was reported.
Time frame: Baseline up to end of the study (8 years)
Number of Participants With Adverse Drug Reaction and Serious Adverse Drug Reaction Following Initiation of Treatment With Velaglucerase Alfa
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.
Time frame: Baseline up to end of the study (8 years)
Number of Participants With Adverse Reactions Categorized as Infusion Reactions Following Initiation of Treatment With Velaglucerase Alfa
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.
Time frame: Baseline up to end of the study (8 years)