Throughout 2020, Pulmonary Hypertension News brought daily coverage of groundbreaking research, treatment advancements, and clinical trial developments related to pulmonary hypertension (PH).
As we anticipate bringing you more PH news this year, we take a look back at our top 10 most-read stories of 2020, with a summary of their significance for the PH community.
A study found that in people with pulmonary arterial hypertension (PAH), treatment with Orenitram (oral treprostinil) is more cost-effective than Uptravi (selexipag). Both common treatments for PAH, the medications mimic the action of prostacyclin, a hormone that lowers blood pressure by opening blood vessels. Healthcare costs associated with Orenitram, marketed by United Therapeutics (which funded and participated in this study), were found to be 51.4% lower than those associated with Uptravi, and pharmacy costs 68.2% lower.
A presentation at the European Society of Cardiology Congress reported that the Phase 3b TRITON clinical trial found the triple combination of oral Uptravi, Opsumit (macitentan), and tadalafil not to be more effective than Opsumit plus tadalafil at reducing pulmonary vascular resistance or other clinical disease markers in newly diagnosed PAH patients. The triple combination therapy, however, was more effective at slowing disease progression.
A study in a rat disease model reported that a poorer gut microbiome diversity — a phenomenon called dysbiosis — may be involved in PAH onset and progression. Using a combination of antibiotics to alter gut bacteria composition, the researchers found that antibiotic-treated rats with PAH had lower strain and blood pressure in the right ventricle that pumps blood from the heart to the lungs. Antibiotic treatment seemed to prevent the enlargement of the heart’s right ventricle, which tends to happen over time as PAH progresses, and lessened blood vessel narrowing and obstruction in the animals’ lungs, another key hallmark of PAH.
A case study detailed a PAH patient who was successfully treated with PB1046, being developed by PhaseBio. The therapy is a combination of the company’s proprietary elastin-like polypeptide biopolymer and vasoactive intestinal peptide — a hormone secreted by nerve cells that increases blood flow by opening blood vessels. The patient experienced a reduction in mean blood pressure of the pulmonary artery, and in blood flow resistance in the lungs. The treatment also increased the patient’s stroke volume — the amount of blood the heart pumps out to the body in one heartbeat — and his cardiac output, or the amount of blood pumped by the heart in one minute, without raising his heart rate.
In a case report, a 34-year-old woman with PAH complicated by COVID-19 infection was successfully treated with an at-home inhaled nitric oxide tankless delivery system called Genosyl, developed by Vero Biotech, to lower blood pressure. The patient received inhaled nitric oxide for 12–14 hours each day for 17 days, before being weaned off treatment without incident.
In April, Pulmonary Hypertension News reported that the FDA had designated sotatercept, an investigational molecule developed by Acceleron Pharma to treat PAH by improving pulmonary artery health and function, a breakthrough therapy. The designation conveys regulatory and approval incentives to potential treatments for serious or life-threatening conditions.
The FDA months earlier ad designated a medical product developed by Aria CV to treat pulmonary hypertension a breakthrough device. The system is designed to restore damaged arteries in the lungs, reduce blood pressure, and increase blood flow. It consists of a balloon that is implanted in the pulmonary artery, which inflates and deflates in sync with the heart cycle. The company believes the device will improve quality of life in patients with PH.
A case report described a man in his 60s with PAH brought on by severe vitamin C deficiency. The patient, who was hospitalized with dangerously high blood pressure along with a rash, sore joints, and difficulty breathing, had low vitamin C levels likely caused by a poor diet. He was treated with vitamin C and D supplements, which alleviated his symptoms over the next five months. PAH resolved following repletion of vitamin C. The case highlights the importance of checking vitamin C levels in patients experiencing PAH, its researchers suggested, and supports “the potential therapeutic role of vitamin C supplementation in patients who are deficient.”
In March, Vero Biotech released early details from the case described in article No. 6 on this list. The patient, a 34-year-old female physician, was the first person with PAH complicated by COVID-19 to receive the at-home Genosyl inhaled nitric oxide treatment. She was treated under the terms of an emergency investigational new drug application approved by the FDA.
A study found that the composition of the gut microbiome is unique in patients with PAH, and could serve as a diagnostic marker for the disease. PAH patients had a distinct gut bacterial profile, with less diversity and a low number of beneficial bacterial species compared with healthy controls. This specific bacterial profile was able to predict the presence or absence of PAH with 83% accuracy. According to the research team, the identification of specific gut microorganisms may lead to earlier, less invasive diagnoses, as well as new avenues for treatment.
Pulmonary Hypertension News hopes our reporting in 2021 will continue to inform and improve the lives of everyone affected by PH.
Wishing all our readers a happy and bright 2021.
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