Pulmonary hypertension: what it is and how to recognize the disease that "takes away" your breath

A rare, progressive and disabling disease. This is how we can define, in scientific terms, pulmonary arterial hypertension. But behind these scientific terms there is the reality of a condition that suddenly leads to difficulty breathing, with a hunger for air that sometimes becomes almost constant and represents a traveling companion capable of heavily penalizing the quality of life of those who suffer from the condition. With damage that, in the end, spreads to the heart, with the onset of heart failure.

It is estimated that in Italy there are 3,000-3,500 people affected by PAH and in Europe about 30,000. The symptoms of PAH are subtle at the onset, but then progressively worsen. For this reason, it is essential to arrive at the diagnosis early, but this objective is not always achieved also because the symptoms are often nonspecific and therefore can lead astray.

An invisible disease

The pathology is characterized by an increase in blood pressure in the pulmonary arteries with consequent heart fatigue. Technically, IAP originates directly in the small pulmonary arteries and causes an increase in pressure in the small circulation with possible heart failure due to the involvement of the right side of the heart.

It generally tends to manifest itself mainly in women, but it can have a worse course in the male population. The difficulty in recognizing the picture is linked to the fact that the symptoms are really not very specific and perhaps make one think of other more common conditions: we are talking about shortness of breath, breathlessness, swelling of the legs.

For this reason, the diagnosis is difficult to obtain early and sometimes takes years. In short, we are faced with a disease that is defined as invisible because the air hunger does not cause pain and the symptoms are often neglected or confused with those of other cardio-respiratory diseases. It is therefore necessary to support those who suffer from it and those who assist the patients, also and above all with early recognition of the condition.

The role of therapies

In the past, the treatment of pulmonary arterial hypertension consisted of lung or heart-lung transplantation as the only therapeutic option; in the last 10 years the therapeutic path has changed, with the introduction of drugs that prolong survival and improve the quality of life of patients. In addition, patient and family support services have been developed, such as the Home Delivery of some therapies.

Meanwhile, research continues, as demonstrated by preliminary results from the Phase 3 HYPERION study evaluating a drug (sotatercept) versus placebo (both in combination with standard therapy) in adults with recently diagnosed PAH functional class II or III at intermediate or high risk of disease progression. HYPERION met its primary endpoint of time to clinical worsening. In the HYPERION study, sotatercept, administered in addition to standard therapy (72.2% of patients were on dual therapy) within 12 months of the initial diagnosis of PAH, demonstrated a statistically significant and clinically relevant reduction in the risk of clinical worsening events versus placebo.

“Pulmonary arterial hypertension (PAH) is a progressive disease with a poor prognosis and is a difficult disease to treat because the combination therapies needed to attack the disease from the early stages can be complex to manage,” said Stefano Ghio , President of the Italian Pulmonary Hypertension Network (IPHNET) and head of the Heart Failure, Cardiomyopathy and Pulmonary Hypertension Unit of the Cardiology Division, IRCCS Policlinico S. Matteo, Pavia. The HYPERION study demonstrated that sotatercept, in addition to basic therapy, achieved the primary endpoint of reducing the time to clinical worsening in patients with newly diagnosed PAH. These positive results from HYPERION add to the previous positive data from STELLAR and ZENITH obtained in prevalent patient populations, i.e. patients who had been on their treatment for years. Sotatercept is the first therapy approved for the treatment of PAH that acts correcting the imbalance between intracellular signals that promote and inhibit growth that characterizes PAH disease. The HYPERION results confirm the clinical robustness of sotatercept and potentially expand the patient population that can benefit from this new drug.”