Version July 2025
For other causes of pediatric PH, established treatment guidelines are lacking and treatment decisions are made on a case-by-case basis. Children with PAH should be managed in centers with the experience, special expertise, and multidisciplinary teams necessary to provide care for these patients. In addition to pulmonary vasodilator therapy, other medical therapy for PAH may include oxygen therapy, diuretic therapy, and/or digoxin (eg, if the patient has evidence of right heart failure). Before starting targeted PAH therapy, all patients should undergo a baseline assessment that includes history and physical examination, electrocardiogram, BNP or NT-proBNP level, chest radiograph, and echocardiogram. In most cases, cardiac catheterization with AVT is also performed before starting therapy. This algorithm is intended for use in conjunction with additional UpToDate content on PAH in children. Refer to UpToDate topics on the evaluation and management of PAH in children for additional details of our approach to treatment and the efficacy of these treatments.
6MWD: six-minute walk distance; ASD: atrial septal defect; AVT: acute vasoreactivity testing; BNP: brain natriuretic peptide; BPD: bronchopulmonary dysplasia; CCB: calcium channel blocker; CDH: congenital diaphragmatic hernia; CHD: congenital heart disease; CI: cardiac index; D-TGA: D-transposition of the great arteries; ERA: endothelin receptor antagonist; FAC: fractional area change; IV: intravenous; mPAP: mean pulmonary artery pressure; mRAP: mean right atrial pressure; MRI: magnetic resonance imaging; mSAP: mean systemic arterial pressure; NT-proBNP: N-terminal pro-BNP; PACI: pulmonary arterial compliance index; PAH: pulmonary arterial hypertension; PAP: pulmonary artery pressure; PH: pulmonary hypertension; PDE-5i: phosphodiesterase type 5 inhibitor; PVRI: pulmonary vascular resistance indexed to body surface area; RA: right atrium; LV: left ventricle; RV: right ventricle; RVEF: right ventricular ejection fraction; SUBQ: subcutaneous; TAPSE: tricuspid annular plane systolic excursion; WHO: World Health Organization; WU: Wood unit.
* AVT is performed via cardiac catheterization and involves administrating a short-acting pulmonary vasodilator (eg, inhaled nitric oxide, epoprostenol, iloprost) and measuring the hemodynamic response. "Reactive" is generally defined as a decrease in mean PAP by at least 10 mmHg to a value of <40 mmHg with no decrease in cardiac output. For additional details, refer to UpToDate content on PH in children.
¶ For children in these categories, the CHD defect is unlikely to be the sole explanation for PAH. This is because isolated ASDs, small coincidental defects, corrected CHD, and lesions without prolonged initial shunt generally do not cause elevated PVRI in childhood. For example, PAH secondary to an unrepaired isolated ASD is a complication that would not be seen until the fourth or fifth decade of life. Thus, patients in these categories who present with PAH in childhood are likely to have an additional cause of PAH (eg, a genetic variant), and management is similar to management of IPAH/HPAH.
Δ CCBs that are used to treat PAH include nifedipine, amlodipine, diltiazem; but not verapamil. CCBs should not be used in patients with depressed RV function. Patients with depressed RV function are often classified as high-risk and are managed accordingly. Refer to UpToDate topic on management of PH in children for additional details.
◊ Oral ERAs include bosentan, ambrisentan, and macitentan; PDE5 inhibitors that are used for treatment of PH include sildenafil and tadalafil. The choice of agent is based largely on patient and provider preference. For additional details, refer to UpToDate content on PH in children.
§ The severity of PH is assessed using the clinical criteria outlined in the table. Categorization as low- or high-risk is not precise and is based in part on the clinical judgment of the treating clinician. For the low-risk category, all standard criteria should generally be met; for the high-risk category, patients typically meet multiple criteria, though all criteria need not be met. As a general principle, echocardiography and cardiac catheterization should be performed prior to starting PH therapy. Some patients have intermediate findings and do not fall clearly into a low- or high-risk category; consensus regarding management of such patients is lacking and practice varies considerably.
¥ In pediatric patients, epoprostenol is primarily used acutely in critically ill or unstable patients due the short half-life of the drug and ease of titration. By contrast, treprostinil is used more commonly for long-term management of more stable patients.
