Use of Darvadstrocel (Allogenic Stem Cell Therapy) for Crohn's Fistula in Real Clinical Practice: the National Project to Implement Mesenchymal Stem Cell for the Treatment of Perianal Crohn's Fistula (the PRIME Study)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 73
- 试验地点
- 1
- 主要终点
- Number of Participants with combined clinical-radiological response
研究概览
简要总结
Background Perianal fistula may affect 15-50% of patients with Crohn's disease (CD). Treatment is complex, requiring a multidisciplinary approach. Darvadstrocel (allogeneic mesenchymal cells obtained from lipoaspirates) was approved in 2018 by the European Medicines Agency and Spanish Agency of Medicines and Medical Products as a treatment for fistulas in CD. Recent European Crohn's and Colitis Organisation (ECCO) andSpanish Working Group on Crohn's Disease and Ulcerative Colitis (GETECCU) guidelines state that darvadstrocel is effective with a favourable safety profile,with a strong level of evidence (level 2).With this study we want to see the real efficacy of darvadstrocel in a Spanish population after 6 months of follow-up.
详细描述
Methods: The criteria for the Spanish National Health System to fund darvadstrocel treatment are: 1)complex fistula in a patient with CD; 2)failure of conventional and anti-tumour necrosis factor treatment; and 3)absence of collection s>2cm confirmed by pelvic MRI scan at the time of surgery. From November 2019 to April 2022, 73 patients were treated with darvadstrocel at 14Spanish centres and evaluated clinically and radiologically 6 months after treatment.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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
- •complex fistula in a patient with CD
- •failure of conventional and anti-tumour necrosis factor treatment
- •absence of collection s>2cm confirmed by pelvic MRI scan at the time of surgery
排除标准
- •Patients with a diagnosis of rectovaginal fistula, rectal stenosis or severe active proctitis were excluded
研究组 & 干预措施
Group of patients treated with darvadstrocel
干预措施: Darvadstrocel (Drug)
结局指标
主要结局
Number of Participants with combined clinical-radiological response
时间窗: 6-month
Combined clinical-radiological response, a new concept, was used to describe patients who achieved both clinical remission and complete radiological healing.
Number of Participants with clinical radiographic healing
时间窗: 6-month
Complete radiographic healing was defined as an MRI result with no fluid collection \>2cm in all dimensions, no oedema and no inflammation nor sign of active inflammatory response;a remnant fistula tract scar may remain
Number of Participants with clinical remission
时间窗: 6-month
Clinical remission was defined as closure of all previously draining external openings treated with darvadstrocel, despite gentle finger compression
Number of Participants with clinical response
时间窗: 6-month
Clinical response was defined as closure of ≥50% of previously draining external openings treated with darvadstrocel, despite gentle finger compression
次要结局
未报告次要终点
研究者
Dolores Herreros Marcos
Principal Investigator
Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz