Upfront triple combination therapy in severe paediatric pulmonary arterial hypertension
Upfront triple combination therapy in severe paediatric pulmonary arterial hypertension
复制标题
DOI：
10.1183/13993003.01120-2020
复制
发表时间：
2021-01-01
影响因子：
24.3
通讯作者：
Bonnet, Damien
中科院分区：
文献类型：
--
作者：
Haarman, Meindina G.;Levy, Marilyne;Bonnet, Damien
在过去的几年里，儿科肺动脉高压（PAH）的治疗策略不断发展，但生存率仍然很低。最近，在重度PAH成人患者中，据报告，从诊断开始的前期三联治疗（uTCT）显示出显著的临床改善和良好的长期结局。这项回顾性、观察性研究旨在评估uTCT在儿科PAH中的疗效，纳入了2010年至2019年期间诊断为PAH并开始接受uTCT治疗的儿童。在基线、3个月和6个月后以及末次随访时评估世界卫生组织功能分级（WHO-FC）、血流动力学、超声心动图、6分钟步行距离和血清N末端脑钠肽前体水平。事件定义为死亡、肺移植或Potts分流。纳入21例儿童（中位年龄4.8岁（2.5-12.8），57%为女性）。除一名儿童外，所有儿童均为WHO-FC III或IV（分别为28%和67%）。3个月后，一名儿童死亡，一名儿童接受了波茨分流术。其余19名儿童表现出临床和超声心动图改善，并持续到6个月。患有特发性和遗传性PAH的儿童的1年、2年和3年无移植生存率估计为100%、94%和87%，尽管其中47%在随访期间接受了Potts分流术。患有重度PAH但非肺静脉闭塞性疾病的儿童，通过uTCT得到显著改善，并显示出有益的3年生存率，尽管其中47%在随访期间接受了Potts分流术。Potts分流联合uTCT在儿科PAH中的作用需要进一步确定。
Treatment strategies in paediatric pulmonary arterial hypertension (PAH) have evolved over the last years, but survival is still poor. Recently, in adults with severe PAH, upfront triple combination therapy (uTCT) from diagnosis has been reported to show significant clinical improvement and excellent long-term outcome. This retrospective, observational study aimed to assess the efficacy of uTCT in paediatric PAH.Children diagnosed with PAH between 2010 and 2019 and started with uTCT were included. World Health Organization Functional Class (WHO-FC), haemodynamics, echocardiography, 6-min walking distance and serum level of N-terminal pro-brain-natriuretic-peptide were assessed at baseline, after 3 and 6 months and at last available follow-up. Events were defined as death, lung transplantation or Potts shunt.21 children (median age 4.8 years (2.5-12.8), 57% females) were included. All children except one were in WHO-FC III or IV (28% and 67%, respectively). After 3 months, one child had died and one child had received a Potts shunt. The remaining 19 children showed clinical and echocardiographic improvement, which persisted at 6 months. Children with idiopathic and heritable PAH showed one-, two- and three-year transplant-free survival estimates of 100%, 94% and 87%, albeit 47% of them receiving a Potts shunt during follow-up.Children with severe PAH, but not pulmonary veno-occlusive disease, improved significantly with uTCT and showed beneficial up to 3-year survival rates, albeit 47% of them receiving a Potts shunt during follow-up. The role of a Potts shunt in conjunction to uTCT in paediatric PAH needs to be further established.