A Study of Home vs Hospital Treatment in People With Fabry, Gaucher or Hunter Conditions in Mexico

N/AActive Not RecruitingNCT07494058

Takeda222 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

222

Conditions

Lysosomal Disease

Interventions

No interventionOTHER

This is a non-interventional study.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion)

Treatment adherence will be analyzed according to the definition of the ratio between prescribed vials vs currently administered vials.

Time frame: Up to 6 months

Secondary Outcomes

Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion

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).