Department of Pediatrics, Division of Pediatric Cardiology, Stanford University School of Medicine, Palo Alto, California.
Department of Pediatrics, Division of Pediatric Cardiology, Medical College of Wisconsin, Milwaukee, Wisconsin.
Semin Thorac Cardiovasc Surg. 2023;35(4):733-743. doi: 10.1053/j.semtcvs.2022.07.011. Epub 2022 Aug 2.
Single ventricle (SV) patients with pulmonary vascular disease (SV-PVD) are considered poor surgical candidates for Glenn or Fontan palliation. Given limited options for Stage 1 (S1) and Stage 2 (S2) SV patients with SV-PVD, we report on the use of subcutaneous treprostinil (TRE) to treat SV-PVD in this population. This single-center, retrospective cohort study examined SV patients who were not candidates for subsequent surgical palliation due to SV-PVD and were treated with TRE. The primary outcome was ability to progress to the next surgical stage; secondary outcomes included changes in hemodynamics after TRE initiation. Between 3/2014 and 8/2021, 17 SV patients received TRE for SV-PVD: 11 after S1 and 6 after S2 (median PVR 4.1 [IQR 3.2-4.8] WUm and 5.0 [IQR 1.5-6.1] WUm, respectively). Nine of 11 (82%) S1 progressed to S2, and 2 (18%) underwent heart transplant (HTx). Three of 6 (50%) S2 progressed to Fontan, 1 underwent HTx and 2 are awaiting Fontan on TRE. TRE significantly decreased PVR in S1 patients with median post-treatment PVR of 2.0 (IQR 1.5-2.6) WU*m. TRE can allow for further surgical palliation in select pre-Fontan patients with SV-PVD, obviating the need for HTx. Improvement in PVR was significant in S1 patients and persisted beyond discontinuation of therapy for most patients.
单心室（SV）合并肺血管疾病（SV-PVD）的患者被认为不适合进行 Glenn 或 Fontan 姑息手术。鉴于 SV-PVD 合并 Stage 1（S1）和 Stage 2（S2）SV 患者的治疗选择有限，我们报告了使用皮下曲前列尼尔（TRE）治疗该人群的 SV-PVD。这项单中心回顾性队列研究检查了由于 SV-PVD 而不适合进一步手术姑息治疗的 SV 患者，并对 TRE 进行了治疗。主要结局是能否进展到下一个手术阶段；次要结局包括 TRE 开始后血液动力学的变化。在 2014 年 3 月至 2021 年 8 月期间，17 名 SV 患者因 SV-PVD 接受 TRE 治疗：S1 后 11 名，S2 后 6 名（中位 PVR 分别为 4.1[IQR 3.2-4.8]WUm 和 5.0[IQR 1.5-6.1]WUm）。11 名 S1 患者中有 9 名（82%）进展到 S2，2 名（18%）接受心脏移植（HTx）。6 名 S2 患者中有 3 名（50%）进展到 Fontan，1 名接受 HTx，2 名在 TRE 等待 Fontan。TRE 显著降低了 S1 患者的 PVR，中位治疗后 PVR 为 2.0（IQR 1.5-2.6）WU*m。TRE 可使选择的前 Fontan SV-PVD 患者进一步接受手术姑息治疗，避免 HTx 的需要。S1 患者的 PVR 改善显著，且大多数患者在停止治疗后仍持续。
