A medication that works for some patients with a rare disease may not be effective for others, even with the same disease. For rare diseases, this is important because sample sizes for drug trials may be small, thus its important that they’re representative of the multidimensional interaction of disease and personal body chemistry and display. As originally reported in Pulmonology Advisor; in a new analysis in Pulmonology Circulation, they have found that while systematic anticoagulation, a blood thinner with vitamin K antagonists (VKAs), effectively improves the quality of life for patients with idiopathic pulmonary arterial hypertension (PAH); for patients with the variation of PAH that is associated with connective tissue disease (CTD-PAH), these blood thinners may in fact, degrade the quality of life.

Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension is a rare disease that causes high blood pressure in the lungs. Patients with the disease typically cannot efficiently pump blood through the arteries which are blocked or narrow, causing the arteries in the lungs to thicken and harden, which puts pressure on the heart to pump harder to balance this out. As a result, oxygen is not distributed as efficiently across the body, and the heart gets worn out. The progressive disease is usually only noticed once it has advanced to later stages, with symptoms such as difficulty breathing, tiredness, fainting or dizziness, chest pain, and swelling. The disease can be the result of a genetic mutation or by other diseases such as HIV or other heart of lung diseases, as well as self-inflicted such as by cocaine. The majority of patients are women between age 30 and 60.

The New Findings

The new research has used the Quality Adjusted Life Year (QALY) to measure how the blood thinner effected patients with the distinct variations of PAH. They accounted for the many intersectional populations of the disease: including sex, use of birth control, and variation of PAH. They found that while use of the anticoagulation VKA´s ubiquitiously improved the quality of life index for patients with the idiopathic PAH, while 95% of patients with CTD-PAH would not choose to use the bloodthinner. However, the trials only measured anticoagulation with the vitamin K antagonists, but there are a whole host of other anticoagulation drugs that don´t have vitamin K and would presumably have different results.

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