Real-world Evidence of Modular Infusion Model in Lysosomal Disease: A Retrospective Observational Comparative Cohort Study From PSP (Patient Support Program) Data in Mexico
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Takeda
- 入组人数
- 232
- 试验地点
- 1
- 主要终点
- Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion)
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Participants with Lysosomal Diseases
All pediatric and adult participants diagnosed with lysosomal diseases (Fabry disease, Gaucher disease, or Hunter syndrome) who were enrolled in the Patient Support Program (PSP) and provided consent for the use of their anonymized data for research purposes will be included in the study. Retrospective data will be obtained from anonymized secondary sources, including the PSP database and the Global Safety Database (GSDB). Participants who received treatment through either modular (home-based) infusion or hospital-based infusion between August 2023 and July 2024 will be analyzed.
干预措施: No intervention (Other)
结局指标
主要结局
Ratio of Treatment Adherence by Infusion Setting (Modular Infusion versus Hospital Infusion)
时间窗: Up to 6 months
Treatment adherence will be analyzed according to the definition of the ratio between prescribed vials vs currently administered vials.
次要结局
- Percentage of Participants With Adverse Events (AE) Receiving Modular versus Hospital Infusion(Up to 6 months)