Targeted Therapies for Vascular Anomalies: A MultiCenter Real World Registry A Review of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 200
- 主要终点
- Baseline Demographics
研究概览
简要总结
This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies. The study will create an active registry for participating centers to enter data on patients with complex vascular anomalies being treated with sirolimus/everolimus (mTOR inhibitors), with/without trametinib (MEK inhibitor), or alpelisib (PIK3CA inhibitor). Tertiary care centers in the United States (US) that receive referrals for complex vascular anomaly cases and use Electronic Health Records (EHRs) will contribute patient medical chart reviews to this registry.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 0 Years 至 90 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with a spectrum of vascular anomalies including but not limited to congenital vascular and lymphatic anomalies, vascular tumors and lymphatic malformations, and acquired vascular malformations.
- •Treated with ≥1 of mammalian target of rapamycin (mTOR) inhibitors, mitogen-activated protein kinase/ERK kinase (MEK) inhibitors and phosphoinositide 3-kinase (PI3K) inhibitors continuously for 3 months.
排除标准
- •Patients diagnosed with a vascular anomaly who have not been treated with mTOR inhibitors, MEK inhibitors and PI3K inhibitors for at least 3 months.
- •Patients with other complex medical conditions; i.e. rare genetic syndromes.
- •Patients receiving many other systemic therapies making data collection not feasible.
- •Recurrent use of immunosuppressive agents, i.e. systemic steroids or targeted medical therapies for oncologic disorders, etc.
- •Patients with significant gaps in data collection.
- •Patients with concurrent enrollment in interventional trials.
- •Other protocol-defined inclusion/exclusion criteria may apply.
研究组 & 干预措施
Complex Vascular Anomaly Group
Patients with vascular anomalies that were treated with mTOR inhibitors (sirolimus/everolimus), a MEK inhibitor (trametinib), and a PIK3CA inhibitor (alpelisib) for a minimum of 3 months.
结局指标
主要结局
Baseline Demographics
时间窗: Baseline
Number of Patients by Clinical Characteristics
时间窗: Baseline
Characteristics include vascular anomaly diagnosis, family history of vascular anomalies, cancer diagnosis, disease severity and anatomic locations involved, associated complications, other medical and surgical interventions, and other medications used.
Number of Patients by Treatment Received in Each Line of Therapy
时间窗: Up to 10 years
次要结局
- Percentage of Patients Who Experience a Clinical Response(Up to 10 years)
- Number of Adverse Events per Person per Year (PPPY)(Up to 10 years)
- Total Number of Adverse Events(Up to 10 years)
- Percentage of Patients With Adverse Events(Up to 10 years)
- Number of Clinical Response Events PPPY(Up to 10 years)
- Total Number of Clinical Response Events(Up to 10 years)
- Number of Patients by Reason for Treatment Discontinuation(Up to 10 years)
- Frequency of Labs and Imaging for Disease Monitoring(Up to 10 years)
- Frequency of Adverse Events in Organ Systems(Up to 10 years)
- Percentage of Patients With Disease/Quality of Life (QoL) Impact(Up to 10 years)
- Percentage of Patients With Clinical Parameters Relevant to Routine Care(Up to 10 years)
- Treatment Duration(Up to 10 years)