Ertical Expandable Prosthetic Titanium Rib (VEPTR) for Thoracic Insufficiency Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 7
- 试验地点
- 1
- 主要终点
- ffer expansion thoracoplasty therapy to TIS patients meeting certain criteria (provided in description below)
研究概览
简要总结
The hypothesis of the study is that individuals with poor pulmonary function secondary to TIS will benefit from expansion thoracoplasty. The investigators specific aims are to:
-
Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation
-
Offer expansion thoracoplasty therapy to TIS patients with
-
poor pulmonary function at enrollment
-
progressive decline in pulmonary function
-
progressive chest wall and/or spinal deformity
-
lack of appropriate increase in thoracic volume during growth
-
Document efficacy of expansion thoracoplasty for TIS patients
详细描述
Study procedures
- Patient Identification & Enrollment Patients meeting the inclusion criteria will be invited to enroll in the study as described above.
- Serial clinical observation
Patients will be followed over time for progression of thoracic insufficiency indicators:
- Worsening respiratory status oFrequent respiratory infections oElevated resting respiratory rate oEase of fatigability oHypoxia and hypercapnia oMarionette sign (trunk and shoulder elevation and depression with respiration)
- Primary diaphragmatic breathing (loss of chest wall mobility)
- Progressive scoliosis
- Failure of thoracic dimensions to increase with growth
Patients with progressive thoracic insufficiency will be offered surgical intervention with expansion thoracoplasty utilizing the VEPTR device.
Studies which are standard of care for TIS will be utilized to assess patients. Routine standard of care studies will be obtained:
- MRI of the spine will be obtained at presentation.
- Chest CT scan (every 2 years, or less frequently if clinically indicated)
- AP/Lat spine xrays (to include the chest wall)
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
- 6 Months 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Pediatric patients, age range from 6 months - 17 years with thoracic insufficiency syndrome (TIS) will be considered for inclusion. Categories of TIS include:
- •Flail chest syndrome
- •Constrictive chest wall syndrome (rib fusions and scoliosis)
- •Hypoplastic thorax syndrome (Jeune's syndrome, achondroplasia, Jarcho-levin syndrome, Ellis van Creveld syndrome)
- •Progressive scoliosis/kyphosis of congenital, neurogenic, or idiopathic origin without rib anomaly
- •Early onset scoliosis without rib anomaly at high risk for progression
排除标准
- •Failure to meet inclusion criteria.
结局指标
主要结局
ffer expansion thoracoplasty therapy to TIS patients meeting certain criteria (provided in description below)
时间窗: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Offer expansion thoracoplasty therapy to TIS patients with * poor pulmonary function at enrollment * progressive decline in pulmonary function * progressive chest wall and/or spinal deformity * lack of appropriate increase in thoracic volume during growth
Document efficacy of expansion thoracoplasty for TIS patients
时间窗: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
Pulmonary function and thoracic volume of TIS patients
时间窗: Enrollment (Baseline), Serial Observation Postop (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
1)Record pulmonary function and thoracic volume of TIS patients at enrollment and with serial observation postoperatively (Change from baseline at: 6 week, 12 week, 6 month, and every 6 months after that.)
次要结局
未报告次要终点
研究者
Jeffrey Martus
Assistant Professor of Orthopaedics
Vanderbilt University Medical Center