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treprostinil

✓ Approved

United Therapeutics · PTGIR · 小分子

什么是 treprostinil?

treprostinil 是一种小分子,由United Therapeutics研发。该药已获批,用于治疗相关适应症,给药途径:Injectable (Others)、Subcutaneous Injection。

药物档案

公司United Therapeutics
药物类别小分子
分子靶点PTGIR
给药途径Injectable (Others), Subcutaneous Injection
状态Approved

作用机制

分子靶点

treprostinil 作用于 1 个分子靶点:

PTGIRprostaglandin I2 receptor (IP, PRIPR)
需要更深入的分析?Noah AI 可解释复杂机制并与同类药物比较。

治疗适应症

treprostinil 针对 1 个适应症,涉及 1 个治疗领域。

治疗领域疾病/病症分期
Respiratory, thoracic and mediastinal disordersPulmonary hypertension✓ Approved

相关研究文献

PubMedMinerva medica2026-09-10

Pulmonary hypertension in interstitial lung disease: today's realities, tomorrow's possibilities.

Bandyopadhyay Debabrata D, Smith Jorden E JE, Asif Huda H, Uysal Askin A et al.

Interstitial lung disease-associated pulmonary hypertension (ILD-PH) is a frequent and life-limiting complication of fibrotic lung disease, associated with substantial morbidity, impaired functional capacity, and significantly reduced survival. Under-recognition remains common due to overlapping symptoms with ILD, evolving hemodynamic definitions, and the limited accuracy of current noninvasive screening tools. ILD-PH prevalence varies widely across ILD subtypes, ranging from 3-15% in early disease to more than 60% in lung transplant candidates. Even modest elevations in mean pulmonary artery pressure are prognostically meaningful, while right ventricular dysfunction represents a key determinant of mortality. Pathophysiology reflects a complex interaction between parenchymal fibrosis, chronic hypoxemia, endothelial injury, and progressive pulmonary vascular remodeling. Diagnostic evaluation requires a multimodal approach, with right heart catheterization remaining essential for definitive confirmation. Historically, vasodilator therapies have shown limited benefit; however, inhaled treprostinil has demonstrated improvements in exercise capacity, biomarkers, and clinical worsening, establishing the first evidence-based treatment for ILD-PH. Additional inhaled and targeted agents are in development, and the role of combination therapy is an emerging area of investigation. Optimal management also includes supplemental oxygen, pulmonary rehabilitation, and timely evaluation for lung transplantation. ILD-PH represents a distinct vascular phenotype requiring early detection and tailored treatment strategies. Continued advances in screening algorithms, precision therapeutics, and mechanistic research are critical to transforming ILD-PH from a terminal complication into a modifiable component of the ILD spectrum.

PMID 42720540
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PubMedCHEST pulmonary2026-09-03

Real-World Experience of Midodrine in Hospital Setting in Pulmonary Arterial Hypertension.

Giese Noah N, Safdar Zeenat Z, Mulumudi Anisha A, Momin Sarah S et al.

Pulmonary arterial hypertension (PAH), a progressive disease, is characterized by increased pulmonary vascular resistance (PVR) and leads to right ventricular failure and premature death. PAH therapies aim to reduce PVR; however, these treatments as vasodilators may also result in reduced systemic vascular resistance and mean arterial pressure (MAP), leading to clinical or symptomatic hypotension. Low MAP can limit the administration of optimal dosage of PAH drugs. Midodrine, an oral alpha-1 adrenergic agonist, as a promising intervention, can potentially increase mean MAP and improve tolerance to PAH therapies. Does the use of midodrine improve MAP and allow for simultaneous uptitration of PAH therapy while hospitalized? A retrospective analysis of 433 patients treated at Houston Methodist Lung Center was undertaken between January 2005 and September 2022. Of these, 57 patients were prescribed midodrine during their hospital stay. We matched 57 patients 1:1 with propensity score matching between patients with PAH not given midodrine (control patients) based on age, sex, World Health Organization functional class, B-type natriuretic peptide, and 6-minute walk distance. Among hospitalized patients with PAH, those receiving midodrine were more likely to undergo uptitration of their PAH medications compared with those not receiving midodrine (n = 30 vs n = 17, respectively; P < .05). Patients on midodrine during hospitalization received higher doses of epoprostenol (P < .001), treprostinil (P < .05), and selexipag (P < .05). Additionally, no adverse effects attributable to midodrine were reported. This study, to our knowledge the first large-scale analysis of PAH data, investigated the use of midodrine in hospitalized patients with PAH. In this single-center study, we share real-world experience of using midodrine to mitigate systemic hypotension, thereby facilitating the uptitration of PAH-targeted therapies.

PMID 42688414
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PubMedJournal of controlled release : official journal of the Controlled Release Society2026-08-14

Formation of a pulmonary drug-depot by a double-ester treprostinil prodrug enables sustained lung-selective delivery.

Leone Giuditta G, Akoumia Kouamé Kan Firmin KKF, Ucakar Bernard B, Esfahani Hrag H et al.

Prostacyclin analogues are effective treatments in pulmonary arterial hypertension (PAH), especially in advanced stages. Treprostinil, a stable prostacyclin analogue, can be administered as subcutaneous and intravenous infusions, oral extended-release tablets and inhalation. Inhalation offers several advantages over other routes of administration, including direct access to the lungs for localized therapy, reduced infection risk, and a painless, convenient mode of delivery. However, small lipophilic molecules like treprostinil are absorbed into the bloodstream within minutes after inhalation, resulting in a short duration of action in the lungs and systemic side effects. To address these limitations, we developed a novel strategy involving a double treprostinil prodrug tailored for pulmonary delivery. The prodrug consists of treprostinil di-esterified at its carboxylic acid with a polyethylene glycol (PEG) chain, and at its C11 hydroxyl group with butyric acid. The prodrug exhibited sustained treprostinil release in bronchoalveolar lavage fluid and serum, supporting its suitability for pulmonary delivery. It was cleaved by initial hydrolysis of the PEG chain, followed by subsequent cleavage of the short-chain fatty acid. Ex vivo studies in isolated pulmonary artery rings showed a delayed and prolonged vasorelaxation effect of the conjugate compared to the free drug. In vivo studies demonstrated significant lung retention, with detectable quantities of the compound remaining in the lungs 24 h after administration, and a markedly reduced peak serum concentration following inhalation. This double-prodrug approach represents a promising strategy for improving PAH treatment by optimizing local, sustained treprostinil delivery while minimizing systemic exposure.

PMID 42600698
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PubMedPulmonary circulation2026-08-14

Transitions From Parenteral Prostacyclin Analogues to Selexipag in Patients With Pulmonary Arterial Hypertension.

Kuebel Dalton J DJ, Collins Adele R AR, Jose Arun A, Guido Maria R MR et al.

Pulmonary arterial hypertension (PAH) is a progressive illness that may require therapy with parenteral prostacyclin pathway agents (PPA) (epoprostenol and treprostinil). These parenteral PPA's are continuous ambulatory infusions that require a high level of skill and knowledge to maintain safety and effectiveness. Depending on the clinical context, transition to oral prostacyclin receptor agonist therapy (selexipag) is sometimes pursued. To prevent abrupt withdrawal from parenteral PPA's, overlap transitions are typically used. This report describes 35 inpatient, rapid transitions from parenteral PPA's to oral selexipag. Planned hospital admissions for transition were common (n = 27, 77%), and all patients successfully transitioned without a return to parenteral PPA's in the immediate post-transition period. Most patients transitioned due to difficulties with current parenteral PPA therapy (n = 20, 57%), followed by an improved clinical status on parenteral PPA therapy (n = 8, 23%). The median length of transition was 36 h (IQR 24-48 h) and hospital stay was 3 days (IQR 2-6 days). Following transition, the cohort demonstrated stability in PAH-specific risk scores and functional status. All patients survived to outpatient pulmonary hypertension follow up (median 5.5 weeks, IQR 1.4-9.6 weeks), and the majority were alive at 1-year post-transition (n = 32, 91%). Based on these results, we conclude that transition from parenteral PPA's to selexipag can be done in the inpatient setting safely, quickly, and effectively.

PMID 42598408
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PubMedFrontiers in pharmacology2026-08-12

Exploring the therapeutic landscape of pulmonary hypertension associated with interstitial lung disease, with a focus on idiopathic pulmonary fibrosis: a narrative review.

Reccardini Nicolò N, Da Re Beatrice B, Mondini Lucrezia L, Salton Francesco F et al.

Pulmonary hypertension associated with idiopathic pulmonary fibrosis (PH-IPF) is a frequent and clinically relevant complication that worsens exercise capacity, quality of life, and survival. This narrative review summarizes the epidemiology, pathophysiology, diagnostic approach, and therapeutic landscape of PH-IPF. The development of PH in IPF reflects the combined effects of fibrotic parenchymal destruction, pulmonary vascular remodeling, hypoxic vasoconstriction, endothelial dysfunction, and altered vascular signaling. Diagnosis remains challenging because symptoms often overlap with those of advanced fibrotic lung disease and non-invasive tools have limited sensitivity; right heart catheterization remains the diagnostic gold standard. Antifibrotic agents are central to IPF management but have no established role as PH-targeted therapies. Most pulmonary arterial hypertension therapies have failed to show benefit in PH-IPF or have raised safety concerns, with ambrisentan and riociguat associated with harm. Inhaled treprostinil is currently the only approved therapy with randomized evidence of efficacy in PH associated with interstitial lung disease, including IPF. Supportive care, optimization of comorbidities, referral to expert centers, and timely lung transplantation evaluation remain essential components of management.

PMID 42582535
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PubMedJACC. Case reports2026-07-30

Pseudo-Infarction Reversal in Right Precordial Leads: ECG Evolution in Severe Pulmonary Hypertension.

Wang Lina L, Cui Yuxia Y, Song Jing J, Zhao Qinghao Q et al.

An acute pulmonary hypertension (PH) crisis causes dynamic electrocardiographic (ECG) changes, but complete post-therapy normalization-especially in post-transplant thrombotic microangiopathy (TMA) patients-is rarely documented. A 53-year-old man with acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation presented with dyspnea. Right heart catheterization confirmed an acute pulmonary hypertension crisis (mean pulmonary arterial pressure: 52 mm Hg) secondary to thrombotic microangiopathy. Treatment with inhaled nitric oxide and treprostinil led to improvement. Follow-up echocardiography showed normalized pulmonary arterial pressure (35 mm Hg), resolution of ECG abnormalities (including a myocardial infarction pattern in the right precordial leads), and no PH recurrence. This case highlights TMA as a rare cause of reversible, vasoreactive PH after hematopoietic stem cell transplantation and documents the full ECG evolution during PH crisis and recovery. ECG monitoring is essential for diagnosing and tracking acute PH crises in transplant patients. Post-transplant TMA can cause reversible PH, requiring prompt hemodynamic assessment and targeted therapy.

PMID 42530193
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