Clinical Characteristics and Mortality in Patients With Fibrodysplasia Ossificans Progressiva (FOP): A Non-Interventional US National Retrospective Cohort
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 131
- 试验地点
- 1
- 主要终点
- Prevalence of skeletal disorders
研究概览
简要总结
Primary Objective(s):
- To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.)
- To describe the prevalence of clinical characteristics of interest and the use of key medications in people living with FOP and a matched cohort of non-FOP people living in the U.S.
- To compare the prevalence of key clinical characteristics and medication use in people living with FOP to a matched cohort of non-FOP people living in the U.S.
- To estimate the crude mortality rate among people living with FOP
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
- •FOP Cohort(s):
- •Living with FOP in the United States who have been identified from the IFOPA membership database
- •Confirmed FOP diagnosis through IFOPA membership database will be tokenized and linked with HealthVerity's medical claims data, pharmacy claims data, laboratory data and obituary data, as defined in the protocol
- •Comparator Cohort:
- •People without FOP living in the U.S. randomly sampled from HealthVerity's closed medical claims data and matched to the FOP population
- •Index date are defined as each person's cohort entry date. People may exit and re-enter the cohort, with the period during which they were not in the cohort excluded from the analysis
- •The comparator cohort will be matched to the cohort of people with FOP on the following variables: age, sex, insurance type and first index date
排除标准
- •Failure to meet inclusion criteria
结局指标
主要结局
Prevalence of skeletal disorders
时间窗: Up to 5 years
Average number of dispensations per FOP patient per year
时间窗: Up to 5 years
Demographic characteristics
时间窗: Up to 5 years
Prevalence of cardiovascular disease
时间窗: Up to 5 years
Prevalence of metabolic disease
时间窗: Up to 5 years
Prevalence of infection
时间窗: Up to 5 years
Prevalence of respiratory disease
时间窗: Up to 5 years
Prevalence of reproductive disease
时间窗: Up to 5 years
Cumulative number of dispensations per FOP patient
时间窗: Up to 5 years
Incidence of all-cause death
时间窗: Up to 5 years
FOP Sub-Cohorts 1 and 2 Only
Prevalence of hematology disease
时间窗: Up to 5 years
Prevalence of neurologic disorders
时间窗: Up to 5 years
Prevalence of hearing loss
时间窗: Up to 5 years
Prevalence of dermatologic disorders
时间窗: Up to 5 years
Prevalence of neoplasms
时间窗: Up to 5 years
Prevalence of gastrointestinal disease
时间窗: Up to 5 years
次要结局
未报告次要终点