Department of Medicine, Boston University School of Medicine, Boston, MA, USA.
Laboratory of Malaria and Vector Research, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Eur J Haematol. 2021 Jul;107(1):54-62. doi: 10.1111/ejh.13612. Epub 2021 Apr 15.
Sickle cell disease-related pulmonary hypertension (SCD-PH) is a complex disorder with multifactorial contributory mechanisms. Previous trials have evaluated the efficacy of pulmonary arterial hypertension (PAH) therapies in SCD-PH with mixed results. We hypothesized that a subset of patients with right heart catheterization (RHC) confirmed disease may benefit from PAH therapy.
We performed a retrospective chart review of patients with SCD-PH diagnosed by RHC who were treated with phosphodiesterase 5 inhibitor (PDE5-I) therapy for ≥4 months between 2008 and 2019 at two institutions.
Thirty-six patients were included in the analysis. The median age (IQR) upon PDE5-I initiation was 47.5 years (35-51.5 years); 58% were female and twenty-nine (81%) had HbSS disease. Of these, 53% of patients had a history of acute chest syndrome, 42% had a history of venous thromboembolism, and 38% had imaging consistent with chronic thromboembolic PH. Patients were treated for a median duration of 25 months (IQR 13-60 months). Use of PDE5-I was associated with a significant improvement in symptoms as assessed by NYHA Class (P = .002).
In SCD patients with PH defined by RHC, PDE5-I therapy was tolerated long-term and may improve physical activity.
镰状细胞病相关肺动脉高压（SCD-PH）是一种具有多因素致病机制的复杂疾病。先前的试验已经评估了肺动脉高压（PAH）治疗方法在 SCD-PH 中的疗效，但结果喜忧参半。我们假设，在经过右心导管检查（RHC）确诊的患者中，有一部分可能会从 PAH 治疗中获益。
我们对 2008 年至 2019 年期间在两家机构接受磷酸二酯酶 5 抑制剂（PDE5-I）治疗≥4 个月的 SCD-PH 患者进行了回顾性图表审查，这些患者的 RHC 诊断为 SCD-PH。
共纳入 36 例患者进行分析。PDE5-I 起始时的中位年龄（IQR）为 47.5 岁（35-51.5 岁）；58%为女性，29 例（81%）患有 HbSS 疾病。其中，53%的患者有急性胸痛综合征病史，42%有静脉血栓栓塞病史，38%有符合慢性血栓栓塞性 PH 的影像学表现。患者接受治疗的中位时间为 25 个月（IQR 13-60 个月）。NYHA 分级评估表明，PDE5-I 的使用与症状的显著改善相关（P=0.002）。
在通过 RHC 定义的 SCD 患者的 PH 中，PDE5-I 治疗是可以长期耐受的，并且可能改善体力活动能力。
