Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Number of Participants With Adverse Events (AEs) |
An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A serious adverse event (SAE) is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious adverse events (AEs) will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on charitable access program (CAP). |
Baseline up to approximately 12 months |
|
| Primary |
Number of Participants With Adverse Drug Reactions (ADRs) |
An ADR is an AE for which there is at least a reasonable suspicion of a causal relationship between an AE and a suspected medicinal product. Number of participants with ADRs will be reported using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Hemoglobin (Hb) Concentration Using Both Retrospective and Prospective Data |
Hemoglobin concentration will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Platelet Count Using Both Retrospective and Prospective Data |
Platelet count will be assessed in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Spleen Size Using Both Retrospective and Prospective Data |
Spleen size will be assessed using ultrasound or Magnetic Resonance Image (MRI) in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is defined as measurement from first time participants was dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Liver Size Using Both Retrospective and Prospective Data |
Liver size will be assessed using ultrasound or MRI in participants with Gaucher disease receiving VPRIV using both retrospective and prospective data when used as standard clinical practice. Baseline is first time participants were dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Treatment History Based on Previous VPRIV Treatment |
Presence of treatment history will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
At Baseline |
|
| Secondary |
Change From Baseline in Hemoglobin (Hb) Concentration Based on Previous VPRIV Treatment |
Hemoglobin concentration will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Platelet Count Based on Previous VPRIV Treatment |
Platelet count data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Spleen Size Based on Previous VPRIV Treatment |
Spleen size date will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Change From Baseline in Liver Size Based on Previous VPRIV Treatment |
Liver size data will be assessed using retrospective data of the previous VPRIV treatment. Baseline is first time participants were dosed on CAP. |
Baseline up to approximately 12 months |
|
| Secondary |
Number of Participants With AEs and SAEs Based on Previous VPRIV Treatment |
An AE is defined as any untoward medical occurrence (including a symptom or disease or an abnormal laboratory finding) in a participant or clinical investigation participants administered a medicinal product and which does not necessarily have a causal relationship with the treatment. A SAE is any event that results in: death; life-threatening event; requires inpatient hospitalization or results in prolongation of existing hospitalization; persistent or significant disability/incapacity; results in a congenital anomaly/birth defect or a medically important event. AEs include serious adverse events, unexpected AEs, non-serious AEs will be collected using retrospective data of the previous VPRIV treatment. Baseline is defined as measurement from first time participants was dosed on CAP. Data will be assessed retrospectively based on participant records. |
Baseline up to approximately 12 months |
|
