Pulmonary Arterial Hypertension (PAH)
Pulmonary arterial hypertension (PAH) is a rare, progressive, life-threatening type of pulmonary hypertension without a cure. PAH is also called WHO PH Group 1. PAH causes narrowing, thickening and stiffness of the arteries that carry blood from the heart to the lungs, resulting in high blood pressure in the lungs and weakness in the right side of the heart.
Healthy artery
Artery of person with PAH
PAH Causes
PAH can be inherited or caused by another disease. Some people have PAH and it is not clear why.
PAH & Serotonin
Research has shown that serotonin can play a role in the development of PAH. Serotonin is a substance that occurs naturally in your body. It is mainly found in your brain and digestive tract and appears to affect and/or regulate a number of body functions. It is thought that too much serotonin may cause arteries in the lungs to narrow.
Doctors hope the ELEVATE Studies medicine, RVT-1201, can reduce the amount of serotonin affecting the lung arteries, which may open the arteries to allow blood to flow more easily.
PAH Symptoms
Early symptoms of PAH may include feeling tired and short of breath, especially when physically active. Unfortunately, since many other diseases share these symptoms, diagnosing PAH is often delayed. This means that people living with PAH often do not get treatment until the disease has worsened. As PAH progresses, the signs and symptoms may be experienced more often and may begin to impact everyday activities.
- Chest pain
- Trouble breathing
- High blood pressure in the lungs
- Blood clots
- Swelling in the legs, hands and belly
- Tiredness
- Dizziness
- Fainting
- Dry, sometimes bloody, cough
- Blue-colored lips or fingers
Diagnosis
PAH is typically diagnosed by a cardiologist (heart specialist) or pulmonologist (lung specialist). A cardiac catherization, also called a “right-heart cath” is used to confirm a diagnosis. The catheter measures the blood pressure in the right side of the heart and lungs.
Current Treatments
If left untreated, PAH can become life threatening. There is no cure, but there are more than a dozen treatments that can help people living with PAH improve their symptoms. If these medicines are not effective, some major surgical procedures (e.g. lung transplant or a heart procedure called atrial septostomy) can help, but also can have serious health consequences.
More options are needed to help improve PAH survival. The ELEVATE Studies will help researchers understand if RVT-1201 is safe and may be a treatment option for people living with PAH.
PAH Resources
- Empowered Patient Online Toolkit —customizable forms to organize medications, medical history, travel info, etc.
- PHA Classroom—online education platform for patients and families
- Podcast series featuring interviews with patients, caregivers, physicians, advocates
- Rare disease specialists can answer questions
- Rare disease information
- Report about PAH with detailed information
- Patient and Caregiver Resource Center for people living with rare diseases
References
- DP, McGoon. "REVEAL: A Contemporary US Pulmonary Arterial Hypertension Registry. - Pubmed - NCBI". Ncbi.Nlm.Nih.Gov, 2019, https://www.ncbi.nlm.nih.gov/pubmed/22379169. Accessed 7 Mar 2019.
- "Pulmonary Arterial Hypertension - NORD (National Organization For Rare Disorders)". NORD (National Organization For Rare Disorders), 2019, https://rarediseases.org/rare-diseases/pulmonary-arterial-hypertension/. Accessed 7 Mar 2019.
- "Genetic And Rare Diseases Information Center (GARD) – An NCATS Program | Providing Information About Rare Or Genetic Diseases.". Rarediseases.Info.Nih.Gov, 2019, https://rarediseases.info.nih.gov/. Accessed 7 Mar 2019.
