TY - JOUR T1 -前期三联治疗重度小儿肺动脉高压JF -欧洲呼吸杂志JO - Eur Respir J DO - 10.1183/13993003.01120-2020 VL - 57 IS - 1 SP - 2001120 AU - Haarman, Meindina G. AU - Lévy, Marilyne AU - Roofthooft, Marcus T.R. AU - Douwes, Johannes M. AU - visia - kazemier, Theresia R. AU - Szezepanski, Isabelle AU - Berger, Rolf M.F. AU - Bonnet，Damien Y1 - 201/01/01 UR - //www.qdcxjkg.com/content/57/1/2001120.abstract N2 -在过去的几年里，儿科肺动脉高压(PAH)的治疗策略已经发展，但生存率仍然很低。最近，在患有严重多环芳烃的成年人中，从诊断开始，预先三联治疗(uTCT)显示出显著的临床改善和良好的长期结果。这项回顾性观察性研究旨在评估uTCT在儿科PAH中的疗效。包括2010年至2019年期间诊断为PAH并开始接受uTCT的儿童。在基线时、3个月和6个月后以及最后的随访时评估世界卫生组织功能等级(WHO-FC)、血流动力学、超声心动图、6分钟步行距离和血清n端脑钠肽前体水平。事件定义为死亡、肺移植或波茨分流。包括21名儿童(中位年龄4.8岁(2.5-12.8)，57%为女性)。除1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，一个孩子死亡，一个孩子接受了波茨分流。其余19例患儿临床和超声心动图均有改善，持续6个月。 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.Upfront triple combination therapy in severe paediatric PAH resulted in significant clinical, haemodynamic and echocardiographic improvement and favourable 1-, 2- and 3-year survival rates, albeit with 47% receiving a Potts shunt during follow-up https://bit.ly/3iG92PA ER -