Prospective Study to Investigate the Relevance of Monitoring Area Under the Curve of Mycophenolic Acid in Patients Receiving Mycophenolate Mofetil to Treat a Diffuse Cutaneous or a Pulmonary Involvement of Systemic Sclerosis
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Skin efficacy
研究概览
简要总结
To define a target value of AUC MPA to improve the modified Rodnan score and / or respiratory impairment (DLCO or FVC) at one year in patients receiving MMF for the treatment of diffuse cutaneous or interstitial lung damage of systemic sclerosis.
详细描述
In the treatment of autoimmune diseases, MMF is almost always prescribed at a fixed dose, regardless of AUC, or based on the target of AUC determined for organ transplantation. One study looked at determining an "effective" AUC threshold in systemic lupus erythematosus, which appears to be 35mg / h / l. This was also done for ANCA vasculitis.
We therefore conducted this study to determine a correlation between AUC MPA and the effectiveness of MMF in systemic sclerosis.
Prospective, observational, open study.
Main objective: define a target value of AUC MPA to improve the modified Rodnan score and / or respiratory impairment (DLCO or FVC) at one year in patients receiving MMF for the treatment of diffuse skin involvement or pulmonary function in systemic sclerosis.
The main endpoint will be evaluated on the evolution of the modified Rodnan score at 1 year after the initiation of MMF and / or the evolution of FVC and DLCO at 1 year after the initiation of MMF.
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
- 否
入选标准
- •Systemic sclerosis meeting the ACR / EULAR criteria of 2013
- •Equal or more than 18 years old, able to freely consent to study
- •In patients treated for skin damage:
- •Diffuse skin sclerosis (rising above the elbows and / or knees)
- •First clinical sign of systemic sclerosis outside of Raynaud's phenomenon going back less than three years
- •Failure to take other concomitant immunosuppressive treatments or in the last 3 months except corticosteroids.
- •In patients treated for lung damage:
- •Interstitial lung damage identified on chest CT, chest x-ray
- •Any duration of progression of systemic scleroderma
- •Prescription of MMF in first line or in relay of a treatment with Cyclophosphamide.
- •Absence of biotherapy in the last 6 months.
排除标准
- 未提供
结局指标
主要结局
Skin efficacy
时间窗: 1 year
Modified Rodnan skin score (mRSS) : min 0 max 51. A diminution in the mRSS of more than 25% from the initial value was considered as improved. On the contrary, an increase in the mRSS over 25% was considered as deteriorated. All other variations of mRSS were classified as stable. Minimal clinically important difference was also tested (worsening of mRSS ≥ 4.7).
次要结局
- Pulmonary efficacy.2(6 months and 1 year)
- Pulmonary efficacy.1(1 year)