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Pulmonary Arterial Hypertension (PAH) — Targeted Therapy

Group 1 pulmonary hypertension (mPAP >20 mmHg, PAWP ≤15 mmHg, PVR >2 WU on right heart catheterization) characterized by progressive pulmonary vasculopathy, right heart failure, and reduced exercise capacity, treated with combination targeted therapy (endothelin receptor antagonists, PDE5 inhibitors, prostacyclin pathway agents) and risk-stratified treatment escalation per ESC/ERS guidelines.

Written by: Saygı Hospital Health Guide Editorial Board
Last updated:

This content has been compiled by the Saygı Hospital Health Guide Editorial Board and is periodically reviewed by a specialist physician.

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This content is for informational purposes only and does not constitute medical advice. You can book an appointment at our Göğüs Hastalıkları department. Book Appointment →

What is Pulmonary Arterial Hypertension (PAH) — Targeted Therapy?

Pulmonary arterial hypertension (PAH) is Group 1 pulmonary hypertension by WHO/Nice classification, defined by precapillary hemodynamics on right heart catheterization (RHC): mean pulmonary arterial pressure (mPAP) >20 mmHg at rest (revised threshold 2018), pulmonary artery wedge pressure (PAWP) ≤15 mmHg, and pulmonary vascular resistance (PVR) >2 Wood units. Histopathology shows pulmonary arteriolar remodeling with intimal hyperplasia, medial hypertrophy, adventitial thickening, plexiform lesions, and in situ thrombosis, leading to progressive elevation of pulmonary vascular resistance and right heart failure.

Subtypes include idiopathic PAH (IPAH, 30-40%), heritable PAH (HPAH, BMPR2 mutations 70%, ALK1, ENG, others 10%), drug/toxin-induced (anorexigens fenfluramine/aminorex, methamphetamine, dasatinib, interferons), associated conditions (APAH): connective tissue disease (scleroderma 15-30% develop PAH), HIV (0.5-1%), portal hypertension (portopulmonary), congenital heart disease (Eisenmenger), schistosomiasis (most common cause worldwide), pulmonary veno-occlusive disease (PVOD), and pulmonary capillary hemangiomatosis (PCH).

Targeted therapy involves 4 pathways: 1) Endothelin pathway: endothelin receptor antagonists (ERAs) bosentan, ambrisentan, macitentan; 2) Nitric oxide pathway: phosphodiesterase-5 inhibitors (PDE5i) sildenafil, tadalafil; 3) Soluble guanylate cyclase (sGC) stimulator: riociguat (CHEST, PATENT trials); 4) Prostacyclin pathway: parenteral epoprostenol (gold standard for severe disease), treprostinil (IV/SC/inhaled/oral), iloprost (inhaled), selexipag (oral non-prostanoid IP receptor agonist). Risk stratification via ESC/ERS 4-strata model uses WHO functional class, 6MWT, NT-proBNP, RA pressure, cardiac index, and SvO2 to guide initial dual therapy (low/intermediate risk) or triple therapy with parenteral prostacyclin (high risk).

Symptoms

Progressive exertional dyspnea (universal)
Fatigue and weakness
Lightheadedness or syncope (severe disease)
Chest pain or pressure on exertion
Peripheral edema (right heart failure)
Hemoptysis (rare, advanced disease)
Hoarseness (Ortner syndrome - LA compression)

Risk Factors

Female sex (3:1)
Connective tissue disease (scleroderma, lupus)
HIV infection
Portal hypertension and cirrhosis
Congenital heart disease (Eisenmenger)
Family history of PAH (BMPR2 mutation testing)
Drug exposure: anorexigens, methamphetamine, dasatinib

When to See a Doctor?

If you experience any of the following symptoms, seek medical attention promptly:

  • Unexplained progressive dyspnea on exertion
  • Right heart failure symptoms (edema, ascites)
  • Syncope on exertion (high-risk PAH)
  • New murmur or right ventricular hypertrophy on echo
  • Connective tissue disease screening for PAH (annual echo)
  • Worsening WHO functional class despite therapy
  • Need for treatment escalation (dual to triple therapy)

Treatment Methods

01
ERA: bosentan, ambrisentan, or macitentan (oral, daily)
02
PDE5i: sildenafil 20 mg TID or tadalafil 40 mg/day
03
Riociguat (sGC stimulator) — alternative to PDE5i (do not combine)
04
Prostacyclin: IV epoprostenol or SC/IV/inhaled treprostinil for severe disease
05
Selexipag (oral IP receptor agonist) for intermediate-risk disease
06
Initial dual oral combination (ERA + PDE5i) per ESC/ERS guidelines
07
Triple therapy with parenteral prostacyclin for high-risk patients

Which Department to Visit?

You can visit our Göğüs Hastalıkları department for these complaints. Our specialist physicians will create the most suitable treatment plan for you.

Learn About Göğüs Hastalıkları Department

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Health Disclaimer: The information on this page is prepared for general informational purposes only. It does not replace medical diagnosis and treatment. Please consult your physician for your complaints. Saygı Hospital does not accept responsibility for actions taken based on the information on this page.