Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 244
- 试验地点
- 1
- 主要终点
- Estimated prevalence of haemophilia
研究概览
简要总结
This study will help to better understand the course of disease in people with haemophilia (A or B [with or without inhibitors]) and the haemophilia care scenario in the selected countries and the experience so far in dealing with the condition. This is a survey-based study; hence no medications or other treatments will be provided to participants as a part of this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •For Experienced/Senior Treating Physicians
- •Experienced/senior haematologist treating haemophilia (adult or paediatric) or physician treating patients with haemophilia. (PWH), with greater than 5 years of experience.
- •Able to communicate in English or country specific language.
- •Agree/sign informed consent before data collection. For Patients Organisations
- •Patient organisation supporting haemophilia.
- •Expert having greater than 3 years of work experience with the patient organisation.
- •Expert level understanding of haemophilia landscape and care pathway in the relevant country.
- •Able to communicate in English or country specific language.
- •Agree/sign informed consent before data collection. For Patients/Caregivers
- •Patients of all age groups, diagnosed with haemophilia A or haemophilia B (with or without inhibitors) for greater than 2 years.
- •For patients less than 18 years of age (*less than 19 years for South Korea), their caregivers aged greater than or equal to 18 years (*greater than or equal to19 years for South Korea) should sign the informed consent.
- •*For South Korea, the adult age is 19 years.
- •Able to communicate in English or country specific language.
- •Agree/sign informed consent before data collection.
排除标准
- •There is no explicit exclusion criterion in this study, thus any participants group who answers "no" to any of the inclusion criteria will be excluded from the study.
结局指标
主要结局
Estimated prevalence of haemophilia
时间窗: From start of data collection to end of data collection (approximately 5 months)
Proportion \[%\] of individuals in a population
Estimated incidence of haemophilia
时间窗: From start of data collection to end of data collection (approximately 5 months)
Number of new cases of haemophilia
Estimated proportion of patients with haemophilia (PWH) who develop inhibitors (%)
时间窗: From start of data collection to end of data collection (approximately 5 months)
proportion \[%\] of individuals in a population
次要结局
未报告次要终点