Producing a Novel Symptom Burden Scale for People Living With Idiopathic Multicentric Castleman Disease (ISBUS)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 主要终点
- Completion of interviews (stage 2)
研究概览
简要总结
Development, validation of a novel symptom burden scale to assess and quantify the burden experienced by people living with Idiopathic Multicentric Castleman Disease (iMCD).
详细描述
Idiopathic Multicentric Castleman Disease (iMCD) is a rare lymphoproliferative disorder associated with systemic inflammation and organ dysfunction. The condition has an unknown aetiology and diagnostic criteria were established recently in 2017. It can be distinguished from other forms of Castleman Disease, including unicentric presentations and Multicentric Castleman Disease (MCD) that is associated with the Kaposi sarcoma herpesvirus. iMCD has an estimated prevalence of between 6.9 and 9.7 people per million. While clinical presentations vary, iMCD can be associated with a high degree of symptom burden, including constitutional, gastrointestinal, neuropsychiatric, dermatologic, respiratory, and hematologic or lymphoreticular problems. Both symptoms and the side-effects of treatment can impact the health-related quality of life (HRQoL) of people with iMCD
This international mixed-methods study will be conducted in four stages:
Stage 1: Item generation
- Drawing on the content, data, and analysis from a previous international patient online survey in iMCD with 65 patients, generate draft PROM content (including a longlist of candidate draft items under existing themes) to assess symptom burden in iMCD.
- Incorporate expert opinion and lived experience to achieve consensus on which items will be tested for inclusion in the symptom burden scale via an online multi-stakeholder workshop involving the funder, research team, patient and public involvement and engagement (PPIE) representative(s) (i.e., patients with iMCD and/or their carers), healthcare professionals, and any other relevant stakeholders.
Stage 2: Item testing and refinement
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
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
- •Diagnosis of idiopathic Multicentric Castleman Disease (iMCD)
- •Adults (aged 18+ years)
- •Fluent in English
排除标准
- •No diagnosis of iMCD
- •Children (aged < 18 years)
- •Not able to understand or communicate in English
- •People lacking the capacity to consent
结局指标
主要结局
Completion of interviews (stage 2)
时间窗: Within 6 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis)
completion of interviews by 10 patients from all recruiting sites
Long list of candidate items for the scale (stage 1)
时间窗: Sept 2023 - Nov 2023
completion of data analysis from a previous survey
Completion of questionnaire (stage 3)
时间窗: Within 9 months from start (With this being a rare condition, data from all patients will be collected at various levels of treatment and time from diagnosis). NB: Advisory comments noted.
completion of questionnaire which includes extra questions about treatments, time of diagnosis. Completion of EQ-5D for external validity of PROM. NB: EQ-5D is the full name of the instrument and not an acronym.
Completion of interviews (stage 4b)
时间窗: About 4 weeks after Stage 4a
completion of interviews by 10 patients from all recruiting sites
Completion of follow-up questionnaire (stage 4a)
时间窗: About 8 weeks after Stage 3 questionnaire. Note: Advisory comments noted.
completion of follow-up questionnaire from Stage 3 by same patients who participated in Stage 3. Note: The full name of the scale will be discussed and confirmed. At the moment, the working title is Idiopathic Multicentric Castleman Disease Symptom Burden Scale (ISBUS).
次要结局
未报告次要终点