Lysosomal Disease
Conditions
Keywords
Drug Therapy
Brief summary
During the COVID-19 pandemic, home treatment for conditions such as Fabry, Gaucher or Hunter became important because it is easier for people to stick to their treatment if medicines that need to be given as infusion (called intravenous or IV treatment) can be given at home or somewhere close to home. Additionally, many of the hospitals that provide infusions are centralized in Mexico. The main aim of the study is to find out whether people with Fabry, Gaucher or Hunter condition are more likely to continue and follow their treatment properly (called 'treatment adherence') when they receive it at home compared to when they receive it at a hospital. Other aims are to understand the factors that can influence treatment adherence, to learn about any regional differences in Mexico and to understand any medical problems with either treatment (home or hospital). No treatment will be given during this study. Only already existing data will be reviewed during this study.
Interventions
OTHERNo intervention
This is a non-interventional study.
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Participants who met the inclusion criteria of the "Modular infusion" program * Confirmed diagnosis of Fabry disease, Gaucher disease, or Hunter syndrome * Confirmatory diagnostic test * Referral to the PSP from the treating physician * Original PSP informed consent and * Privacy notice signed * Participants who met the inclusion criteria of the "Hospital infusion" program * Confirmed diagnosis of Fabry disease, Gaucher disease, or Hunter syndrome * Confirmatory diagnostic test * Referral to the PSP from the treating physician * Original PSP informed consent and * Privacy notice signed * Agreement that their anonymized information will be used for research purposes
Exclusion criteria
* Participants who do not agree or withdraw his/her explicit consent for research purposes may have the right to withdraw; however, once their data has been integrated into the database, it is no longer identifiable by any participant in the research team. * Discontinuation of Participants
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion) | Up to 6 months | Treatment adherence will be analyzed according to the definition of the ratio between prescribed vials vs currently administered vials. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion | Up to 6 months | AE is defined as any untoward medical occurrence in the participant notified to the pharmacovigilance unit, including Infusion-related reactions (observed within 24 hours of product administration). |
Countries
Mexico
Contacts
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed