Pulmonary Arterial Hypertension (PAH)
BASICS
- Definition: Disease of small pulmonary arteries (precapillary PH) causing increased pulmonary arterial pressure (PAP) and vascular resistance → right-sided heart failure.
- Diagnosis (Right Heart Catheterization):
- Mean PAP ≥20 mm Hg at rest
- Pulmonary vascular resistance ≥3 Wood units
- Pulmonary capillary wedge pressure ≤15 mm Hg
- Excludes left heart, lung, thromboembolic, or systemic/metabolic causes
- WHO Group 1 PH: PAH (idiopathic, heritable, drug/toxin-induced, connective tissue disease, HIV, portal HTN, congenital heart disease, schistosomiasis)
- Related but distinct: PVOD and PCH (pulmonary venous/capillary diseases)
EPIDEMIOLOGY
- Any age; mean age 37 yrs
- Sex: Female > Male (ratio 1.7–4.8:1 in idiopathic PAH)
- Incidence/Prevalence:
- PAH: 5–52/million; IPAH: 2–6/million
- PAH prevalence: 15–50/million
- Drug-induced: 1/25,000 for >3 months anorectic use
- High risk: scleroderma (6–60%), portal HTN, HIV
ETIOLOGY & PATHOPHYSIOLOGY
- Vascular: Inflammation, vasoconstriction, endothelial dysfunction, intimal proliferation, increased cell proliferation, reduced apoptosis → vascular remodeling & obstruction.
- Cardiac: Right ventricular hypertrophy/remodeling → RV failure, ischemia.
- Idiopathic (IPAH): Unknown cause, often sporadic, sometimes familial.
- Genetic: 75% heritable PAH & 25% IPAH have BMPR2 mutations (autosomal dominant).
RISK FACTORS
- Female sex
- Illicit anorectic drug use
- Recent pulmonary embolism
- Family history (first-degree relatives of familial PAH)
DIAGNOSIS
History
- Symptoms: Dyspnea, weakness, syncope, dizziness, chest pain, palpitations, LE edema
Physical Exam
- Loud P2, early pulmonary valve click, tricuspid regurgitation murmur, pulmonic insufficiency murmur (Graham Steell), JVD, ascites, hepatomegaly, peripheral edema
Differential Diagnosis
- Other causes of dyspnea (lung parenchymal disease, thromboembolism, cardiac disease)
Diagnostic Tests
- ECG: RVH, right axis deviation, RV strain, R/S >1 in V1
- PFT: ↓DLCO
- ABG: Hypoxemia, hypocapnia
- V/Q scan: Rule out CTEPH (group 4)
- 6MWD: Reduced max O2 consumption; correlates with severity
- BNP/NT-proBNP: Early marker, prognosis
- CXR: Prominent central PAs, peripheral pruning, RV enlargement (late)
- Echo Doppler: Screening; suggests RV/RA enlargement, tricuspid regurgitation
- Right heart catheterization: Gold standard
- Other: LFTs (portal HTN), HIV, ANA, TSH, sickle cell, genetic screening (BMPR2) if indicated
TREATMENT
Goals
- NYHA/WHO functional class I or II
- Normal/near-normal RV size/function, hemodynamics (RAP <8 mm Hg, CI >2.5–3 L/min/m²)
- 6MWD >380–440 m, normal BNP
General Measures
- Supervised exercise (avoid strenuous activity), psychosocial support, avoid pregnancy, influenza/pneumococcal vaccination, oxygenation (PaO₂ >60 mm Hg)
Medication
- Acute vasodilator test (during cath): Inhaled NO, IV epoprostenol, IV adenosine
- If positive: (fall in mean PAP ≥10 mm Hg and <40 mm Hg, CO unchanged/increased)
- CCBs (nifedipine LA, diltiazem, amlodipine)
- Avoid verapamil; avoid CCBs if low cardiac index or high RA pressure
- If negative/WHO-FC II–IV:
- Oral therapy: ambrisentan, bosentan, macitentan, riociguat, sildenafil, tadalafil
- Advanced (FC III–IV): IV epoprostenol (first-line FC IV), treprostinil (IV/SC/inhaled), inhaled iloprost, selexipag, combination regimens as needed
- Endothelin receptor antagonists (ERA): bosentan, ambrisentan, macitentan
- PDE5 inhibitors: sildenafil, tadalafil, vardenafil
- Guanylate cyclase stimulator: riociguat
- Prostacyclin analogs: epoprostenol, treprostinil, iloprost, beraprost
- Anticoagulation: Consider in IPAH/HPAH (warfarin INR 1.5–2.5) unless contraindicated
- Diuretics: For RV overload (edema, ascites)
- Digoxin: For RV failure/atrial arrhythmia
REFERRAL / SURGERY
- Specialist referral: Pulmonology/cardiology for PAH suspicion/management
- Atrial septostomy: For severe, refractory PAH as bridge/option before transplant
- Lung/heart-lung transplant: Advanced, refractory cases
ADMISSION, INPATIENT, NURSING
- Most therapy is palliative; focus on symptom management and optimizing quality of life
ONGOING CARE
- Exercise (low level, as tolerated), respiratory training
- Diet: Fluid/salt restriction (esp. RV failure)
PATIENT EDUCATION
- Discuss prognosis, need for lifestyle changes, potential for transplant
PROGNOSIS
- Median survival: 2–3 yrs from diagnosis (older data), now 5-yr survival ~70% with modern therapy
- Most common cause of death: Right heart failure
- Poor prognostic markers: Rapid progression, RV failure, NYHA/WHO FC III–IV, 6MWD <300 m, peak VO₂ <10.4 mL/kg/min, pericardial effusion, RV enlargement, RAP >20 mm Hg, CI <2 L/min/m², high BNP/NT-proBNP, scleroderma
COMPLICATIONS
- Thromboembolism, heart failure, pleural effusion, sudden death, high maternal/fetal risk in pregnancy
ICD-10 Codes
- I27.0 Primary pulmonary hypertension
- I27.2 Other secondary pulmonary hypertension
CLINICAL PEARLS
- PAH is a precapillary form of PH—diagnosis by right heart catheterization.
- Positive vasodilator test: CCBs are first-line in IPAH.
- Refer early to specialists; prognosis improved with modern therapies but disease remains serious.