BACKGROUND Pulmonary veno-occlusive disease (PVOD) is a rare condition with poor prognosis, and lung transplantation is recommended as the only curative therapy. The role of pulmonary arterial hypertension targeted therapy in PVOD remains controversial, and long-term effects of targeted therapy have been rarely reported. This study aims to retrospectively evaluate the role of targeted therapy in PVOD patients and the long-term outcome. METHODS PVOD patients with good responses to targeted therapies were analyzed, and data pre- and post- targeted therapies were compared. An overview of the effects of targeted therapies on PVOD patients was also conducted. RESULTS Five genetically or histologically confirmed PVOD patients received targeted therapies and showed good responses. Their mean pulmonary arterial pressure by right heart catheterization was 62.0 ± 11.7 mmHg. Two receiving monotherapy got stabilized, and three receiving sequential combination therapy got improved, cardiac function and exercise capacity significantly improved after treatments. No pulmonary edema occurred. The mean time from the first targeted therapy to the last follow up was 39.3 months, and the longest was 9 years. A systematic review regarding the effects of targeted therapies on PVOD patients indicated majorities of patients got hemodynamics or 6-min walk distance improved, and 26.7% patients developed pulmonary edema. The interval from targeted drugs use to death ranged from 71 min to over 4 years. CONCLUSIONS Cautious use of targeted therapy could safely and effectively improve or stabilize hemodynamics and exercise capacity of some patients without any complications. PVOD patients could live longer than expected.

中文翻译：

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肺静脉闭塞性疾病的靶向治疗：是时候重新考虑了吗？

背景技术肺静脉闭塞性疾病（PVOD）是一种罕见的预后较差的疾病，建议将肺移植作为唯一的治疗方法。肺动脉高压靶向治疗在PVOD中的作用仍存在争议，很少报道靶向治疗的长期效果。这项研究旨在回顾性评估靶向治疗在PVOD患者中的作用和长期疗效。方法分析对靶向治疗反应良好的PVOD患者，并比较靶向治疗前后的数据。还概述了靶向治疗对PVOD患者的作用。结果5例经遗传或组织学证实的PVOD患者接受了靶向治疗，并显示出良好的反应。右心导管检查的平均肺动脉压为62.0±11.7 mmHg。治疗后两种接受单药治疗稳定，三种接受序贯联合治疗得到改善，心功能和运动能力得到显着改善。没有发生肺水肿。从首次靶向治疗到最后一次随访的平均时间为39.3个月，最长为9年。关于靶向治疗对PVOD患者的影响的系统评价表明，大多数患者的血流动力学或步行6分钟的步行距离得到改善，并且26.7％的患者出现了肺水肿。从有针对性的药物使用到死亡的间隔时间为71分钟到4年以上。结论谨慎使用靶向疗法可以安全有效地改善或稳定某些患者的血液动力学和运动能力，而无任何并发症。PVOD患者的寿命可能比预期的长。