A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophenolate
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 123
- 试验地点
- 1
- 主要终点
- Time from randomisation to treatment failure.
研究概览
简要总结
This is a study of two treatment pathways [Standard steroid treatment versus combined steroid and Mycophenolate (MMF)] for subjects with newly diagnosed Immune Thrombocytopenia (ITP). ITP is an illness that causes bruising and bleeding due to a low platelet count (blood cells essential for normal clotting). Patients are first given high dose steroids but most suffer side effects (e.g. difficulty sleeping, weight gain, moods swings, high blood pressure and diabetes). In addition, the majority of patients become ill again when the steroids are stopped - only about 20% stay well long term. ITP is relatively rare, non-cancerous in nature and the rare impact on survival of ITP have prevented it from being a priority for research funding, with first line treatment being unsatisfactory and unchallenged for decades. This underestimates the profound adverse impact an ITP diagnosis and its treatment has on individual patients, many of whom are young.
MMF is often used as the next stage treatment for ITP and it works well. However, it can take up to 2 months to work during which patients continue to be at risk of bleeding, bruising, fatigue and usually need more steroids which they find intolerable. They are required to come to hospital for weekly blood tests and for many this impacts on work. We want to find out whether it would benefit more patients if everyone takes MMF at diagnosis instead of current practice (waiting for the illness to come back). We plan to test this by comparing the current way we treat patients to a new way with patients given MMF right at the start of their treatment. 120 patients from 20 different hospitals will be asked to take part and half will be randomly chosen for the new pathway.
详细描述
This is a multicentre, randomised clinical trial of MMF with steroid as a first line treatment for participants with ITP against the standard care pathway involving steroids alone as first line treatment. This is not a blinded study, therefore patients and research team will know which treatment arm the participant will be randomised to.
There are no additional appointments or separate trial visits for this trial. Participants will be seen at their usual hospital appointments, which may take slightly longer than they do usually to gather all the information needed to carefully record information for the trial and to see how the participants are.
Participants will be screened and given up to one week of steroid prior to randomisation to enable sufficient time to read information, discuss and ask questions with informed consent in an appropriate setting.
Participants will then be randomised to one of either two treatment pathways below and be asked to complete quality of life questionnaires:
- Steroid +MMF pathway: 1mg/kg once daily prednisolone 4 days (maximum of 100mg), 40mg once daily 2 weeks, 20mg once daily 2 weeks, 10mg once daily 2 weeks, 5mg once daily 2 weeks then 5mg alternate days 2 weeks then stop, (Dexamethasone 20mg or 40mg daily for 4 days is an alternative option to prednisolone if deemed clinically more appropriate for individual circumstances).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients (males and females) >16 years old with a diagnosis of ITP, a pl count <30x109/L AND a clinical need for first line treatment.
- •Patients have provided written informed consent
排除标准
- •The exclusion criteria include pregnancy and breastfeeding
- •Patients with HIV, Hepatitis B or C, or Common Variable immunodeficiency.
- •Women of child bearing potential require a pregnancy test result within 7 days prior to randomisation (as per 7.1 below) to rule out unintended pregnancy
- •Contraindications to MMF or steroid (see SPC, Appendix 2) including patients with hypersensitivity to mycophenolate mofetil, mycophenolic acid or to any of the excipients or active significant infection
- •Patients not capable of giving informed consent (e.g. due to incapacity)
- •Patients unwilling to follow contraceptive advice if allocated to MMF treatment arm.
研究组 & 干预措施
Steroid & Mycophenolate mofetil 1st line
Mycophenolate mofetil: 1 gm bd Non-IMP Steroid: 1mg/kg od 4 days (maximum of 100mg), 40mg od 2 weeks, 20mg od 2 weeks, 10mg od 2 weeks, 5mg od 2 weeks then 5mg alternate days 2 weeks.
干预措施: Mycophenolate Mofetil (Drug)
Prednisolone (Steroid) alone 1st line
Non-IMP steroid: 1mg/kg od 4 days (maximum of 100mg), 40mg od 2 weeks, 20mg od 2 weeks, 10mg od 2 weeks, 5mg od 2 weeks then 5mg alternate days 2 weeks.
干预措施: Prednisolone (Drug)
结局指标
主要结局
Time from randomisation to treatment failure.
时间窗: 1 year
To include patients who are refractory (platelet count \<30x109/L in spite of 2 weeks treatment in the steroid arm or platelet count \<30x109/L in spite of 2 months treatment in the steroid +MMF arm) or who initially respond but then relapse (defined clinically as platelet count \<30x109/L and need for further therapy).
次要结局
- Patient reported outcomes - Quality of Life(Up to 12 months post randomisation)
- Time to relapse and time to next therapy(Up to 12 months post randomisation)
- Cumulative cortiocosteroid dose(Up to 12 months post randomisation)
- Remission rates(Up to 12 months post randomisation)
- Medication side effects, toxicity and other adverse events (including infection episodes)(Up to 12 months post randomisation)
- Bleeding events(up to 12 months post randomisation)
- Need for rescue therapies(up to 12 months post randomisation)
- Need for splenectomy(Up to 12 months post randomisation)
- Socioeconomic costs(Up to 12 months post randomisation)
- Patient reported outcomes - Care Costs(Up to 12 months post randomisation)
- Patient reported outcomes - Fatigue(Up to 12 months post randomisation)
- Patient reported outcomes - Impact of bleeding(Up to 12 months post randomisation)