Justin J Kochanski, Jeffrey A Feinstein, Michelle Ogawa, Victor Ritter, Rachel K Hopper, Gregory T Adamson
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引用次数: 0
摘要
严重的 1 类肺动脉高压(PAH)患儿对皮下注射曲普瑞替尼疗法(TRE)的反应难以预测,这可能受到年龄、疾病严重程度或开始治疗时其他未知变量的影响。在这项回顾性单中心队列研究中,我们假设开始 TRE 治疗时的年龄较小、早期血流动力学反应(随访导管检查时肺血管阻力下降≥30%)和较轻的基线血流动力学(Rp:Rs < 1.1)均与较好的临床预后相关。在接受皮下 TRE 治疗的 40 名年龄在 17 天-18 岁的 I 类 PAH 儿童患者中,开始接受 TRE 治疗时年龄较小(截止年龄为 6 岁,AUC 0.824)与较好的 5 年无不良事件发生率(94% 对 39%,P = 0.002)、较好的 WHO 功能分级(I 级或 II 级:88% 对 39% P = 0.003)以及最近随访时较好的右心室功能超声心动图指标有关。早期血流动力学反应和较轻的基线血流动力学反应都与更好的预后无关。在首次随访导管检查时对 TRE 没有明显早期血流动力学反应的患者,其 PVRi 随后不太可能出现改善(1/8,13%)。这些发现可帮助临床医生为患者家属提供咨询,并指导临床医生就晚期疗法的时机做出决策。
Younger age at initiation of subcutaneous treprostinil is associated with better response in pediatric Group 1 pulmonary arterial hypertension.
Children with severe Group 1 pulmonary arterial hypertension (PAH) have an unpredictable response to subcutaneous treprostinil (TRE) therapy, which may be influenced by age, disease severity, or other unknown variables at time of initiation. In this retrospective single-center cohort study, we hypothesized that younger age at TRE initiation, early hemodynamic response (a decrease in pulmonary vascular resistance by ≥30% at follow-up catheterization), and less severe baseline hemodynamics (Rp:Rs < 1.1) would each be associated with better clinical outcomes. In 40 pediatric patients with Group I PAH aged 17 days-18 years treated with subcutaneous TRE, younger age (cut-off of 6-years of age, AUC 0.824) at TRE initiation was associated with superior 5-year freedom from adverse events (94% vs. 39%, p = 0.002), better WHO functional class (I or II: 88% vs. 39% p = 0.003), and better echocardiographic indices of right ventricular function at most recent follow-up. Neither early hemodynamic response nor less severe baseline hemodynamics were associated with better outcomes. Patients who did not have a significant early hemodynamic response to TRE by first follow-up catheterization were unlikely to show subsequent improvement in PVRi (1/8, 13%). These findings may help clinicians counsel families and guide clinical decision making regarding the timing of advanced therapies.
期刊介绍:
Pulmonary Circulation''s main goal is to encourage basic, translational, and clinical research by investigators, physician-scientists, and clinicans, in the hope of increasing survival rates for pulmonary hypertension and other pulmonary vascular diseases worldwide, and developing new therapeutic approaches for the diseases. Freely available online, Pulmonary Circulation allows diverse knowledge of research, techniques, and case studies to reach a wide readership of specialists in order to improve patient care and treatment outcomes.