Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 388
- 试验地点
- 32
- 主要终点
- Adverse events
研究概览
简要总结
The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood.
Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables that have been proposed as treatment goals in PePH and the reasons for changes in treatment strategy.
The TOPP-2 registry uses the new clinical classification of PH as outlined at the 5th World Symposium for Pulmonary Hypertension (WSPH) in Nice 2013 and includes new characterizations for children with PH.
The registry is planned and implemented under the scientific leadership of the Association for Pediatric Pulmonary Hypertension (PePH), independently from the financial sponsors.
All enrolled patients will have a follow-up period of 18 months.
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
- 3 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient must be an incident patient, i.e.newly diagnosed with PH
- •Age at time of diagnosis is at least 3 months and less than 18 years
- •Patients must present with PH belonging to one of the following categories
- •Group 1 according to updated Nice clinical classification
- •Group 3 according to updated Nice clinical classification
- •Group 4 according to updated Nice clinical classification
- •Group 5 according to updated Nice clinical classification
- •PH confirmed by heart catheterisation (HC)
- •At HC, the patient must present with mean pulmonary arterial pressure (PAP) of at least 25 mmHg at rest, a pulmonary vascular resistance index (PVRi) equal to or below 3 Wood units*m^2 and mean pulmonary arterial wedge pressure (PAWP) below or equal to 15 mmHg
- •In case of congenital heart disease (CHD) patients who had undergone palliative procedure or repair to close systemic to pulmonary shunt, the diagnosis of PH must have been confirmed by HC at least 6 months after surgery/palliative procedure took place
- •For patients with PAH-CHD open shunt, only those considered not operable due to advanced pulmonary vascular disease are eligible
- •Patients to be included into the registry, and/or their legal guardians, must give informed consent. Where applicable patients will be asked for their written assent
- •Participating sites must agree to adhere to the recommendations of the WSPH 2015 Nice, Pediatric Taskforce.
排除标准
- •Patients belonging to Group 2 according to updated Nice clinical classification
结局指标
主要结局
Adverse events
时间窗: Over registry run-time (5.5 years)
Death
时间窗: Over registry run-time (5.5 years)
Transplantation
时间窗: Over registry run-time (5.5 years)
次要结局
- Atrial septostomy(Over registry run-time (5.5 years))
- Reasons for treatment change(Over registry run-time (5.5 years))
- Decline in WHO functional class(Over registry run-time (5.5 years))
- Use/initiation of i.v./s.c. prostanoids(Over registry run-time (5.5 years))
- Time to clinical worsening(Over registry run-time (5.5 years))
- Decline in 6-minute walk test (6MWT)(Over registry run-time (5.5 years))
- Type of treatment(Over registry run-time (5.5 years))
- Worsening of echocardiographic parameters (ECHO)(Over registry run-time (5.5 years))
- Increase in Brain Natriuretic Peptide (BNP)(Over registry run-time (5.5 years))
- Escalation of treatment(Over registry run-time (5.5 years))
- Hospitalisation related to pulmonary arterial hypertension (PAH)(Over registry run-time (5.5 years))
- Potts shunt(Over registry run-time (5.5 years))
- Switch in treatment(Over registry run-time (5.5 years))
- Increase in N-terminal-proBNP (NT-proBNP)(Over registry run-time (5.5 years))
- Decline in Panama functional class(Over registry run-time (5.5 years))