Targeted therapy in pulmonary veno-occlusive disease: time for a rethink?

Luo, Qin; Jin, Qi; Zhao, Zhihui; Zhao, Qing; Yu, Xue; Liu, Yan; Zhang, Yi; Xiong, Changming; Liu, Zhihong

doi:10.1186/s12890-019-1031-3

Cited by 5 publications

(10 citation statements)

References 32 publications

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“…15 However, the survival has improved according to more recent studies. 7 , 9 , 10 , 14 For example, the 1‐year survival rate was approximately 70% in 94 PVOD/PCH cases. 4 In our study, the 1‐year survival rate was 93%, and mean survival time was 3.9 years; these findings are better than those in early reports and at least comparable to those in recent studies.…”

Section: Discussionmentioning

confidence: 99%

“… 3 , 4 , 7 , 8 However, oral drugs for pulmonary arterial hypertension have made significant progress in recent years, and there have been some reports of their efficacy in PVOD/PCH. 9 , 10 In contrast to studies of early‐phase drugs such as intravenous prostaglandin I 2 and calcium channel blockers, these reports have been limited to a small number of cases, and detailed data on their efficacy and safety are lacking.…”

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confidence: 99%

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Efficacy and safety of oral pulmonary vasodilators in pulmonary veno‐occlusive disease

et al. 2022

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Pulmonary veno‐occlusive disease (PVOD) or pulmonary capillary hemangiomatosis (PCH) is a rare subtype of pulmonary hypertension with dismal prognosis. Limited data are available on the efficacy and safety of orally administered pulmonary vasodilators for PVOD/PCH. Whether and how systemic sclerosis (SSc) affects the clinical outcomes of PVOD/PCH is also unknown. This study aimed to determine the clinical and hemodynamic efficacy and safety of oral pulmonary vasodilators for PVOD/PCH and clarify the possible effects of SSc on the clinical presentation of PVOD/PCH. We retrospectively analyzed the clinical data of 15 patients with PVOD/PCH treated with oral pulmonary vasodilators in our department since 2001. Six of them had SSc. Oral pulmonary vasodilators were administered either as single agents ( n = 10) or in combination ( n = 5). Treatment improved the functional class of five patients, and pulmonary arterial pressure and pulmonary vascular resistance decreased by 10 ± 12 mmHg and 36 ± 19%, respectively ( p < 0.05 for both, n = 13), whereas pulmonary edema developed in three patients. The mean survival was 3.9 years, and the 1‐ and 3‐year survival rates were 93% and 65%, respectively. The clinical presentation, including survival, was similar between patients with and without SSc. In our PVOD/PCH cohort, oral pulmonary vasodilators caused pulmonary edema in 20% of patients, but more than 80% of patients experienced significant pulmonary vasodilatory effects, and the overall prognosis was better than that previously reported. SSc does not adversely affect the clinical outcomes of PVOD/PCH.

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Efficacy and safety of oral pulmonary vasodilators in pulmonary veno‐occlusive disease

et al. 2022

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“…It is speculated that the degree and severity of pulmonary vein involvement in these patients may be the basis of different responses to prostaglandins. [ 26 ] Nossent et al reported that in addition to typical veno-occlusive lesions, substantial pulmonary arterial lesions, and important microvascular remodeling were also found in 24 cases of PVOD. [ 27 ] These PVOD patients had pathological characteristics similar to PAH.…”

Section: Discussionmentioning

confidence: 99%

Good response to pulmonary arterial hypertension-targeted therapy in 2 pulmonary veno-occlusive disease patients

2021

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Rationale: Pulmonary veno-occlusive disease (PVOD) is a kind of rare and fatal pulmonary arterial hypertension (PAH). Different from other subtypes of PAH, PVOD patients have a very poor prognosis because of the progressive nature of pulmonary vascular involvement and fatal pulmonary edema induced by PAH-targeted drugs. Lung transplantation is the only choice for these patients.Patient concerns: We reported 2 cases of PVOD which was misdiagnosed as idiopathic pulmonary arterial hypertension initially due to the lack of typical findings of PVOD. Right heart catheterization was done. The results showed severe PAH with mean pulmonary artery pressure at 76 mmHg and 68 mmHg.Diagnosis: The diagnosis of idiopathic pulmonary arterial hypertension was corrected by eukaryotic translation initiation factor 2 alpha kinase 4 (EIF2AK4) mutation screening. Biallelic mutations (c.1387delT (p. Arg463fs); c.989-990 delAA (p. Lys330fs)) were detected by next-generation sequencing for whole exome from blood sample. The presence of biallelic EIF2AK4 mutation was sufficient to confirm the diagnosis of PVOD. Interventions:The 2 patients had good response to PAH-targeted therapy (Ambrisentan 10 mg once a day and tadalafil 20mg once a day) in the following 1 year.Outcomes: Because the patients had a good response to targeted drugs, the treatment of the 2 cases was unchanged. Over 1-year period, they still have a good response to PAH-targeted drugs. There was no sign of pulmonary edema.Lessons: All these results may indicate that PVOD is not so rare and typical findings of PVOD are lacking in some patients. EIF2AK4 mutation screening by next-generation sequencing maybe useful to differentiate PVOD from other PAH subtypes. PVOD is a heterogeneity population and different patients have different characteristics including response to PAH-targeted therapy. How to pick off this portion of patients timely is the core issue. Further study is necessary to answer this question.

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“…1 Experts in the field do favor a cautious trial of vasodilator therapy as a bridge to lung transplantation. 1,2 Up to 25% to 45% of patients will experience the development of pulmonary edema at any point during up titration or sequential addition of vasodilators; this is one of the most profound clinical clues to the diagnosis. It is recommended therefore to initiate monotherapy with slow titration and an aggressive diuretic regimen.…”

Section: Clinical Discussionmentioning

confidence: 99%

“…It is recommended therefore to initiate monotherapy with slow titration and an aggressive diuretic regimen. [1][2][3] Of note, pulmonary capillary hemangiomatosis is considered to be on the spectrum of the same disease as PVOD. Sporadic and familial cases of PVOD/pulmonary capillary hemangiomatosis have been associated with biallelic mutations in the eukaryotic translation initiation factor 2 alpha kinase 4 gene.…”

Section: Clinical Discussionmentioning

confidence: 99%