Gaucher Disease
Conditions
Brief summary
The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with VPRIV (Velaglucerase alfa)at their home setting compared to receiving the infusions at the clinic or at doctor's practice.
Interventions
None listed
Sponsors
Shire
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female patients with a confirmed diagnosis of Gaucher disease type 1 * Age\> 2 years * patients who have at least 3 infusions (6 weeks) at least 5-year or 5-6 infusions (10-12 weeks) at 2 - to 4-year patients have received VPRIV ® and tolerate well * The patient is compliant, the previous VPRIV ® infusions were / performed approximately every 2 weeks in the center during office visits * The patient was already before inclusion in this study for a home infusion therapy and has consented to (or their legal representative) * The patient / be lawful. Representative has consented in writing to participate in this study.
Exclusion criteria
• The patient is participating in a clinical trial with a medicinal product
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Patient satisfaction estimated on a 10-ary Likert scale, quality of life estimated by SF-36 questionnaire | comparison of baseline to 12 months value |
Secondary
| Measure | Time frame |
|---|---|
| Number (per infusion) and severity of infusion-related side effects | baseline compared to 12 months |
Countries
Austria, Germany
Outcome results
None listed