Types of Treatment

Until 1990, there were very few treatments available for PAH. Since that time, a number of approved therapies have become available, and even newer treatments are in clinical development.^2,3

Available treatments for PAH include oral medication, inhaled medication, infused medication and a combination of these therapies.

Before a medication or combination therapy is prescribed, your physician will determine the cause and severity of your PAH.⁶ If an associated disease is involved, treatment of this disease or condition should be part of your overall PAH care.

Any increase in the severity of your symptoms or unwanted side effects might indicate an adjustment in treatment is necessary. Keep track of these markers with the Living PAH Self-Assessment Survey.

Common PAH Pathways

Three pathways have been identified as causing the constriction and cellular changes that lead to PAH. Nitric oxide, prostacyclin, and endothelin are three substances that are present in the body. When these levels become unbalanced, it can cause the blood vessels—specifically those in the lungs—to constrict and narrow.⁹

Three major therapeutic classes of PAH treatment address the 3 key pathways:

• Prostacyclin Class (prostacyclins and prostacyclin
  analogues)^1,6,16-19

  Prostacyclin is a natural substance found in your body. It is a potent vasodilator, meaning it keeps blood vessels open. Prostacyclin helps to keep the arteries in your lungs open and working properly.

  If you have PAH, your body has lower levels of prostacyclin, which can lead to constriction and narrowing of the arteries in your lungs. Prostacyclin class therapy mimics the effect of prostacyclin, making it easier for your heart to pump blood through your lungs.

• ERAs (Endothelin Receptor Antagonists)^3,6

  Endothelin-1 is a substance that causes blood vessels to constrict, and stimulates the pulmonary artery smooth muscle cells to multiply abnormally. ERA treatment is aimed at blocking the chemical endothelin that causes these unwanted effects.

• PDE-5 Inhibitors (phosphodiesterase type 5 inhibitors)⁶

  The ability of nitric oxide (NO) to dilate pulmonary arteries is dependent on the production of a substance in the pulmonary vessels called cyclic GMP. PDE-5 is a substance in the blood that can break down cyclic GMP in the vessels PDE-5 Inhibitor treatment is aimed at enhancing or prolonging the vasodilatory effects of cyclic GMP.

Additional Treatments Include:^6,11

• The use of calcium channel blockers may help dilate the blood vessels and was one of the earliest treatments used for PAH.
• Anticoagulants help prevent the blood from clotting.
• Diuretics help the body eliminate excess fluid and reduce swelling.
• Antiproliferative agents are anti-cancer drugs that may reduce abnormal cell growth in the pulmonary arteries.
• Oxygen therapy increases the amount of oxygen available to the blood.

Treatment for the Most Severe Cases:

Balloon atrial septostomy
As right heart function worsens, and a patient's symptoms become progressively worse, an atrial septostomy may be considered. This procedure creates a right to left inter-atrial shunt, helping to ease the pressure in the right heart and improve function. With improved cardiac output, overall oxygenation in the pulmonary system is increased.⁶

Heart and lung transplantation
A lung or heart and lung transplant is used only in rare cases for select patients with the most severe cases of PAH. These procedures are limited by organ availability.⁶

This page is not intended to provide an exhaustive review of treatment options, nor replace a full discussion with your doctor.