The ConNeCT Study: Neurological Complications of Thrombotic Thrombocytopenic Purpura
试验速览
- 阶段
- 不适用
- 入组人数
- 250
- 试验地点
- 1
- 主要终点
- The percentage of patients with TTP with neurological complications at acute presentation
研究概览
简要总结
Thrombotic thrombocytopenic purpura (TTP) is a rare condition, which has a very high risk of death if not recognised and given immediate treatment. TTP is caused by a very low level of an enzyme in the body, called ADAMTS13. A lack of ADAMTS13 causes multiple small clots to form around the body which can disrupt the blood flow to important organs. Although survival has improved significantly, it is now being recognised that patients with TTP may suffer with longer term complications as a result of their condition; literature from the USA reports higher rates of major depression and also poor memory and reduced concentration in patients with TTP. The investigators aim to improve the understanding of the long-term complications and review, for the first time, forward-looking data at multiple time points in patients with TTP in the UK. Both patients with a new diagnosis and patients with a known diagnosis of TTP identified in NHS hospitals will be included, over a minimum duration of 2 years. This will be a questionnaire based study with both doctor led and participant led questionnaires at pre-determined points in time. By improving the understanding and comparing symptoms to that of the general population, the investigators hope to improve the support and tailor the treatments which can be offered to patients with TTP.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 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
- 是
入选标准
- •1.Acute episode TTP:
- •Adult male or female patient ≥18 years of age at the time of signing the consent form, with a confirmed diagnosis of TTP (initial or relapse) based on ADAMTS13 <10%
- •Known diagnosis of TTP:
- •Adult male or female patient ≥ 18 years of age at time of signing the consent form, with a historical confirmed diagnosis of TTP (based on ADAMTS13 at initial presentation <10%)
- •Healthy control:
- •Non-blood relative / friend / carer of patients under the care of Haematology clinics at the Royal Liverpool University hospital or other participating centres.
排除标准
- •Acute episode of TTP:
- •Participants less than 18 years old at the time of signing the consent form
- •Patient with ADAMTS13 greater than 10%
- •Patient with cancer or transplant associated MAHA will not be included
- •Patient (or NOK, where patient does not have capacity) not wishing to consent to trial
- •Known diagnosis of TTP:
- •Participants less than 18 years old at the time of signing the consent form
- •Patient with ADAMTS13 greater than 10%
- •Patient with cancer or transplant associated MAHA will not be included
- •Patient (or NOK, where patient does not have capacity) not wishing to consent to trial
- •For healthy control:
- •Participants less than 18 years old at the time of signing the consent form
- •Participant not wishing to consent to trial
- •Any personal or family history of thrombotic microangiopathy
结局指标
主要结局
The percentage of patients with TTP with neurological complications at acute presentation
时间窗: 1 week, 1 month, 3 months, 6 months, 12 months
The primary outcome is to estimate the proportion of both new acute and remission TTP patients developing neurological conditions. These will be reported as counts and percentages with 95% confidence intervals. If we recruit 100 patients in both groups, acute and remission, then we can estimate prevalence rates of 10% with an accuracy of +/- 6%, a 20% prevalence with an accuracy of +/-8% and a 40% prevalence with an accuracy of +/-10%.
The percentage of patients with TTP in remission with long-term neurological complications
时间窗: 6 months, 12 months, 18 months, 2 years
The primary outcome is to estimate the proportion of both new acute and remission TTP patients developing neurological conditions. These will be reported as counts and percentages with 95% confidence intervals. If we recruit 100 patients in both groups, acute and remission, then we can estimate prevalence rates of 10% with an accuracy of +/- 6%, a 20% prevalence with an accuracy of +/-8% and a 40% prevalence with an accuracy of +/-10%.
次要结局
- The percentage of 'follow-up' patients with TTP with a depressive disorder, based on PHQ-9 scoring system, compared to the general UK population.(6 month, 12 months, 18 months, 2 years)
- The percentage of 'follow-up' patients with TTP with neurocognitive deficit, based on TYM scoring system, compared to the general UK population.(6 month, 12 months, 18 months, 2 years)
- The percentage of 'follow-up' patients with TTP with reduced quality of life, based on SF-36 score, compared to the general UK population.(6 month, 12 months, 18 months, 2 years)