Patients' Preferences for Primary Immune Thrombocytopenia Treatment
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Preference Weights for Treatment Attributes
研究概览
简要总结
This study aims to evaluate treatment preferences of patients with Primary Immune Thrombocytopenia (ITP) in the United States (US) by conducting a preference elicitation survey.
详细描述
This is a non-interventional study to assess treatment preferences from patients with ITP. Data will be collected from participants using a preference elicitation survey.
The study consists of two stages. Stage 1 includes the development of the survey and identification of key treatment attributes. Stage 2 involves administration of the finalized survey, followed by comprehensive analysis of the data to assess treatment preferences.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Currently living in the US.
- •Aged 18 years and older on the day of providing informed consent.
- •A diagnosis of primary ITP:
- •a. For participants receiving first-line (1L) treatment for ITP: i. Diagnosed with primary ITP and started 1L corticosteroids within 3 months of diagnosis ii. Responded to 1L corticosteroid treatment [with or without intravenous immunoglobulin (IVIg)] b. For participants receiving second-line (2L) treatment for ITP: i. Assessed as needing or are taking thrombopoietin receptor agonist (TPO-RA) treatment for 2L treatment ii. Has previously been treated with a 1L corticosteroid (with or without IVIg) with insufficient response or relapse
排除标准
- •For participants receiving 1L treatment for ITP only: Previous or current use of treatments different from corticosteroids and/or IVIg/anti-D immunoglobulin.
- •For participants receiving 2L ITP treatment: Previous or current use of treatments different from corticosteroids, IVIg/anti-D immunoglobulin or one TPO-RA (i.e. patients who have used any treatment in a third-line setting are ineligible).
研究组 & 干预措施
ITP Cohort
Adults with ITP in the US who are being treated in either 1L or 2L settings.
结局指标
主要结局
Preference Weights for Treatment Attributes
时间窗: 1 Day
MDT will be used to elicit trade-offs that participants with ITP are willing to make between multiple treatment attributes. Preference weights will be estimated for each treatment attribute included in the MDT. Higher preference weights indicate treatment attributes that have a stronger influence on preferences, while lower weights indicate treatment attributes that have a weaker influence.
Maximum Acceptable Risk (MAR) and Minimum Acceptable Benefit (MAB)
时间窗: 1 Day
To evaluate the benefit-risk trade-offs that participants are willing to make, marginal rates of substitutions (MRS) will be calculated based on the Dirichlet model. MRS will be represented as either MAR or MAB. * MAR measures the median maximum level of risk that participants are willing to tolerate in certain treatment attributes in return for a specified improvement in other treatment attributes. * MAB measures the median minimum level of benefit required in certain treatment attributes for participants to tolerate deteriorations in other treatment attributes.
Differences in Preference Weights Associated With Personal Characteristics
时间窗: 1 Day
Preference weights associated with personal characteristics will be assessed using covariate-adjusted Dirichlet regression. Personal characteristics include sociodemographic, clinical characteristics such as race/ethnicity, living location, employment status, level of education, clinical setting usually treated in, ITP symptoms, and treatment experience, as well as other characteristics such as participants' risk tolerance level, health literacy and numeracy level.
次要结局
未报告次要终点