From the Guidelines
The treatment of pulmonary arterial hypertension (PAH) should prioritize the use of endothelin receptor antagonists, such as macitentan 10 mg once daily, as first-line therapy due to their efficacy in improving exercise capacity and delaying clinical worsening, as supported by the most recent guidelines 1. The management of PAH involves a multifaceted approach, including general measures and specific PAH therapies. General measures may include oxygen therapy, diuretics, and anticoagulation with warfarin in selected cases. For specific PAH therapy, the choice of medication depends on the disease severity and patient response.
- Key considerations in PAH management include:
- The use of prostacyclin derivatives, such as epoprostenol, iloprost, and treprostinil, which are effective in improving symptoms and exercise capacity 1.
- Endothelin receptor antagonists, including bosentan, ambrisentan, and macitentan, which have been shown to improve exercise capacity and delay clinical worsening 1.
- Phosphodiesterase type-5 inhibitors, such as sildenafil and tadalafil, which are effective in improving exercise capacity and reducing symptoms 1.
- Soluble cGMP stimulators, like riociguat, which have been shown to improve exercise capacity and reduce symptoms in patients with PAH 1.
- Prostacyclin receptor agonists, such as selexipag, which have been demonstrated to reduce the risk of clinical worsening and improve exercise capacity 1. Combination therapy with multiple PAH-specific medications is often necessary for patients with inadequate response to monotherapy, and should be guided by the patient's clinical response and tolerance to the medications 1. Regular follow-up with assessments such as echocardiography, 6-minute walk tests, and evaluation of functional class is crucial to monitor treatment response and disease progression, allowing for timely adjustments to the treatment plan as needed 1.
From the FDA Drug Label
Epoprostenol for injection is indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1) to improve exercise capacity. Selexipag tablets are indicated for the treatment of pulmonary arterial hypertension (PAH, WHO Group I) to delay disease progression and reduce the risk of hospitalization for PAH.
Treatment Options for Pulmonary Artery Hypertension:
- Epoprostenol (IV) is indicated for the treatment of PAH to improve exercise capacity, particularly in patients with NYHA Functional Class III-IV symptoms and etiologies of idiopathic or heritable PAH or PAH associated with connective tissue diseases 2.
- Selexipag (PO) is indicated for the treatment of PAH to delay disease progression and reduce the risk of hospitalization for PAH, particularly in patients with WHO Functional Class II-III symptoms 3.
From the Research
Treatment Options for Pulmonary Artery Hypertension
- The treatment of pulmonary artery hypertension (PAH) typically involves a combination of drugs that target multiple biological pathways, such as the nitric oxide-cyclic guanosine monophosphate and endothelin pathways 4.
- Currently approved therapies for PAH include drugs that enhance the nitric oxide-cyclic guanosine monophosphate biological pathway (e.g., sildenafil, tadalafil, or riociguat), prostacyclin pathway agonists (e.g., epoprostenol or treprostinil), and endothelin pathway antagonists (e.g., bosentan and ambrisentan) 4.
- Endothelin receptor antagonists (ERAs) have shown efficacy in the treatment of PAH, improving exercise capacity, World Health Organization (WHO) functional class, and cardiopulmonary hemodynamics, and may reduce mortality 5.
Combination Therapy
- Upfront combination therapy with an endothelin receptor antagonist and a phosphodiesterase 5 inhibitor is now widely accepted as the standard of care for PAH 6.
- Combination therapy has been shown to improve morbidity and mortality compared to single-pathway targeted monotherapy 4.
- The use of prostanoids, such as epoprostenol or treprostinil, may also be considered in combination with other therapies to delay time to clinical worsening 6.
Interventional Strategies
- Percutaneous balloon atrioseptostomy and pulmonary artery catheter-based denervation may be considered in combination with specific drugs at an earlier stage, rather than late in the progression of PAH 7.
- These interventional strategies may help to improve symptoms and delay disease progression, but more research is needed to fully understand their role in the treatment of PAH.
Monitoring and Adverse Effects
- Hepatic toxicity is a potential adverse effect of ERA treatment, and monitoring of liver function is recommended 5.
- Irreversible liver failure has been reported with the use of sitaxsentan, an ERA that has been withdrawn from the market 5.
- The importance of monitoring and managing adverse effects, as well as individualizing treatment approaches, is emphasized in the management of PAH 6.