Schranz Dietmar
Pediatric Heart Center, Johann-Wolfgang-Goethe University Clinic, Theodor-Storm-Kai 7, 60596, Frankfurt, Germany.
Pediatr Cardiol. 2025 Jan 5. doi: 10.1007/s00246-024-03759-4.
This proposal presents a proof of concept for the use of pulmonary flow restrictors (PFRs) based on MVP™-devices, drawing from clinical experience, and explores their potential role in the management of newborns with hypoplastic left heart syndrome (HLHS), other complex left heart lesions, and infants with end-stage dilated cardiomyopathy (DCM). At this early stage of age, manually adjusted PFRs can be tailored to patient's size and hemodynamic needs. Although currently used off-label, PFRs have substantial potential to improve outcomes in these vulnerable patient populations. When integrated into a holistic treatment strategy, they represent a promising advancement in care. There is significant potential to reduce mortality and improve the quality of life for neonates with HLHS and variants, as well as to support age-dependent functional regeneration in DCM, all while avoiding the need for advanced surgical procedures, including general anesthesia. The main barrier to broader adoption is the limited availability of appropriately sized MVP devices. However, with ongoing patient-centered knowledge sharing and refinement of transcatheter techniques, there is reason for optimism that PFRs, tailored for individual patients, could provide significant benefits to thousands of infants with congenital heart disease and DCM worldwide.
本提案基于MVP™设备，借鉴临床经验，提出了使用肺血流限制器（PFR）的概念验证，并探讨了其在治疗左心发育不全综合征（HLHS）、其他复杂左心病变的新生儿以及终末期扩张型心肌病（DCM）婴儿中的潜在作用。在这个早期阶段，手动调节的PFR可以根据患者的体型和血流动力学需求进行定制。尽管目前PFR的使用属于标签外使用，但它在改善这些脆弱患者群体的治疗结果方面具有巨大潜力。当融入整体治疗策略时，它们代表了护理方面的一项有前景的进展。对于患有HLHS及其变体的新生儿，有显著潜力降低死亡率并提高生活质量，同时支持DCM中与年龄相关的功能再生，且无需进行包括全身麻醉在内的先进外科手术。更广泛采用的主要障碍是尺寸合适的MVP设备供应有限。然而，随着以患者为中心的知识不断共享以及经导管技术的改进，我们有理由乐观地认为，为个体患者量身定制的PFR能够为全球数千名患有先天性心脏病和DCM的婴儿带来显著益处。
