Heart failure related to pulmonary arterial hypertension (PAH) is becoming more common, with patients living fewer years in full health, a trend that’s predicted to continue, with more cases and deaths expected by 2050, a study showed.
In the three decades from 1990 to 2021, women accounted for more cases of PAH than men, suggesting that future efforts should focus on better understanding sex-specific differences and tailoring interventions. However, age-standardized prevalence rates, which adjust the number of cases by accounting for differences in age, appear to be decreasing.
“The decrease in prevalence rates despite increasing numbers might be attributed to improved case detection, earlier diagnosis, and enhanced treatment strategies, leading to better disease management,” the researchers wrote.
The study, “Global Burden of Pulmonary Arterial Hypertension and Associated Heart Failure: Global Burden of Disease 2021 Analysis,” was published in JACC: Heart Failure based on data from the Global Burden of Disease, a resource to understand the health challenges faced by people across the world.
In PAH, the pulmonary arteries that take blood from the right side of the heart to the lungs narrow, increasing blood pressure. This makes it hard for the heart to pump enough blood through to the lungs, leading to symptoms such as shortness of breath. Left untreated, it can lead to heart failure.
Disease prevalence steady, higher rate of new cases
To understand how PAH and PAH-related heart failure have changed over time from 1990 to 2021, a team of researchers in China drew on data from the Global Burden of Disease 2021 database. The goal was to track trends at global, regional, and national levels and predict how the disease will evolve until 2050.
To do this, the researchers focused on how many people have the disease (prevalence), how many new cases occur (incidence), and how many people die from it. They also calculated disability-adjusted life years (DALYs), a measure of disease burden expressed as the number of years lost due to disability.
From 1990 to 2021, the global prevalence of PAH stayed stable (2.3-2.28 cases per 100,000 people), but incidence increased slightly (0.5-0.52 cases per 100,000 people). While death rates decreased significantly, population growth and aging contributed to an increase in the total number of deaths. The researchers noted, however, that all these trends showed variations over time.
While the number of cases of PAH increased for men and decreased for women, the disease was still more common in women. The number of DALYs and deaths decreased in both men and women, but more so in men, meaning that by 2021, women “surpassed men in DALYs and deaths rates,” the team wrote.
The greatest increase in PAH prevalence was observed in regions of low to middle sociodemographic index (SDI), a measure of development based on income, education, and birth rates. The deepest reductions in DALYs and death rates occurred in regions of high-middle SDI.
“At the regional level, Western Sub-Saharan Africa and Central Latin America reported rising prevalence rates … and Central Sub-Saharan Africa showed the largest decline,” the researchers wrote.
During the same time window, the total number of cases of PAH-related heart failure nearly doubled from 105,703 to 191,808 across the world, and years lived with disability increased from 9,789 to 17,765. Prevalence rates decreased slightly in women but increased in men.
In 2021, PAH-related heart failure was most common in regions with high and high-middle SDI. The number of years lived with disability was highest in regions with middle SDI. Western sub-Saharan Africa saw notable increases in both prevalence and years lived with disability.
“Our investigation into the global burden of PAH from 1990 to 2021 reveals that despite reduced or stable [age-standardized rates], the overall impact of PAH—measured by prevalence, incidence, mortality, and related [heart failure]—has escalated,” the team wrote.
The results also “underscore the global nature of the PAH-related [heart failure] burden, with significant and growing impacts in low-middle and middle SDI regions,” wrote the researchers, who noted that “access to advanced therapies, particularly pulmonary vasodilator treatments, remains largely concentrated in North America and Western Europe.”
The prevalence and incidence of PAH, as well as the number of deaths, are expected to increase by 2050 due to population growth and aging, so “prevention and control strategies should focus on managing elderly patients through multidisciplinary and individualized treatment approaches,” the researchers wrote. “Countries across the various SDIs should prioritize PAH prevention, prompt diagnosis, and integrated management to help control the disease’s escalating impact.”
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Pulmonary artery denervation (PADN), a procedure that targets overactive nerves involved in blood vessel constriction, significantly lessens high blood pressure in the lungs of people with pulmonary hypertension (PH), according to a meta-analysis of more than a dozen studies.
Pooled data also showed that the approach was associated with significant improvements in heart function and exercise capacity, further supporting its “promise as a therapeutic intervention for PH,” the researchers wrote.
“Pulmonary artery denervation has become a promising therapeutic intervention in the management of pulmonary hypertension,” James Jenkins, MD, study co-author and a cardiologist at Ochsner Health System, said in a Ochsner Health press release. “While our findings suggest a substantial role for PADN in treatment protocols, continuous research is crucial to fully understand its broader implications and long-term benefits.”
The study, “Pulmonary artery denervation in pulmonary hypertension: A comprehensive meta-analysis,” was published in the International Journal of Cardiology.
In PH, high blood pressure in the pulmonary arteries, the blood vessels that supply the lungs, makes it more difficult for the right side of the heart to pump blood to the lungs. This can lead to PH symptoms including shortness of breath during routine activities.
‘Potential game-changer’
“Conventional treatments primarily include vasodilators [blood vessel-widening medications], which provide symptomatic relief but do not effectively reverse the underlying [blood vessel abnormalities],” the researchers wrote.
PADN has in recent years “emerged as a potential game-changer in the treatment landscape of PH,” they wrote.
The procedure uses heat or radio waves to disable overactive nerves around the pulmonary arteries. This should help relax these blood vessels, lowering blood pressure and making it easier for the heart to pump blood to the lungs.
To better understand the benefits of PADN in PH patients, the researchers used a method called meta-analysis to combine data from studies published up to September 2023 and reporting on outcomes of PH patients treated with the procedure.
A total of 14 studies, covering 372 patients, were included in the final meta-analysis. The methods used, follow-up times, and outcomes evaluated differed from study to study.
The meta-analysis’ main goal was to watch for changes in a range of blood-related measures, including mean pulmonary artery pressure (mPAP); pulmonary vascular resistance (PVR), the resistance that must be overcome for blood to flow to the lungs; and mean right atrial pressure (mRAP), the pressure in the right upper chamber of the heart.
All 14 studies reported data on mPAP and PVR, and pooled data showed a significant decrease in both parameters after PADN. However, there was high variability across studies.
Eight studies, with a total of 276 patients, reported consistent results about mRAP, which also decreased significantly after PADN.
Data from 13 studies (360 patients) showed that PADN helped the heart pump significantly more blood. In nine studies (226 patients), the approach was also reported to significantly increase the distance patients could walk in six minutes, a common test for exercise capacity. The studies for each outcome showed some degree of variability among them.
“Our meta-analysis provides insight into the changing field of PADN as an upcoming treatment option for PH,” the researchers wrote. “However, the ongoing differences in patient responses and procedure modifications highlight the complex and subtle character of the results.”
That points to the need for clear guidelines on standard PADN procedures and larger, long-term, appropriately controlled clinical trials to confirm the results and identify patients who would benefit most from the procedure, the team said.
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Leading up to my heart and lung transplant in 2018, a friend asked if there were any specific activities I was looking forward to doing post-transplant. The hope was that my health would improve following the procedure. One of my very confident answers was that I was excited to begin engaging in marches and political activism.
As a woman who’s lived with rare and chronic illnesses, it’s probably not surprising that I’ve developed an interest in various aspects of political policy. That became more meaningful toward the end of my time with pulmonary hypertension (PH), when I was in college and starting to form my beliefs.
Unfortunately, my day-to-day life with PH made any physical activism difficult. My mobility was limited by poor heart and lung function, and I had little energy to spare. For this reason, I looked forward to a day when my body would give me the fortitude to speak out about policies and rights that I was passionate about. The idea of marching on a street with a sign in my hand was appealing and felt like a small way for me to take control of my circumstances.
Now, living as a transplant recipient with other complex issues, I have similar physical limitations as before. In addition, my immune system is incredibly vulnerable, and after several close calls, the risk of illness is no longer worth the exposure to large crowds.
This limitation is another restriction in my life that causes a bit of grief. Still, I’ve learned over time that there are other ways to take charge of advocacy, away from crowds and safe at home.
Ways to get involved
Contacting representatives: Getting in touch with local reps is what people often recommend, but not everyone takes advantage of this option. The point of a democracy is for the people to have a voice, so I try to remember to use mine.
By calling and emailing my representatives, I can put forth my specific views about policies and bills affecting me. People will often circulate specific instructions and scripts via social media for larger issues, making the process extremely straightforward. This guidance is a wonderful tool for people like me who can get anxious with conversational tasks.
Taking to social media: Speaking of social media, it’s a powerful tool for engaging in activism. Just the other day, I posted a video regarding the inevitable underrepresentation of the disabled community at protests and rallies because of our physical limitations. Creating content like that helps me find my voice and brings people into my comments who offer so much community and positive conversation regarding the issues I’m passionate about. Virtual community is a wonderful modern development that can go a long way in helping me remember that no one is alone.
Engaging in conversation with local community: Finally, speaking with those around me is the simplest way to be politically active. Holding conversations with my friends, loved ones, and local community allows me to share my perspective with others as a disabled woman while also receiving education from different perspectives. At the end of the day, we all belong to one country, and nothing can replace the impact of sharing our stories and needs with one another.
Note: Pulmonary Hypertension News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Hypertension News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary hypertension.
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Treatments approved for pulmonary arterial hypertension (PAH) may reduce by half the risk of serious complications or death in adults and adolescents who also have repaired congenital heart disease (CHD), pooled data analysis of three clinical trials shows.
This risk was similarly reduced among the whole PAH patient population who participated in the trials and given either Uptravi (selexipag), Opsumit (macitentan), or Tracleer (bosentan). All three therapies are marketed by Johnson & Johnson, which funded the analysis.
“This pooled analysis provides important evidence to guide medical management in this patient population,” the researchers wrote. The findings were described in the study “Outcomes in Patients Receiving Treatment for Pulmonary Arterial Hypertension Associated With Repaired Congenital Heart Disease,” which was published in JACC: Advances.
PAH occurs due to a narrowing of the pulmonary arteries, the blood vessels that supply the lungs. This causes blood to travel under higher pressure than normal, making it more difficult for the right side of the heart to pump blood to the lungs. This can lead to shortness of breath and other symptoms.
People with CHD, a problem with how the heart forms before birth, are at risk of PAH and those who develop it after a surgical repair may have a worse outlook. There’s little evidence about how well approved PAH treatments work for patients with repaired CHD. Here, researchers at Johnson & Johnson and several international institutes explored whether patients with repaired CHD-associated PAH (CHD-PAH) may benefit from such treatments.
Benefit of PAH treatments on repaired CHD
They pooled data from three placebo-controlled clinical trials: the Phase 3 GRIPHON (NCT01106014) that tested Uptravi, the Phase 3 SERAPHIN (NCT00660179) of Opsumit, and the Phase 4 COMPASS-2 (NCT00303459) that tested Tracleer.
A total of 1,982 people, 12 years and older, with PAH made up the whole patient population. Fewer than 1 in 10 (8.9%) had repaired CHD-PAH. The most common CHD was an atrial septal defect, wherein a hole allows blood to flow between the two top chambers of the heart.
More than three-quarters of the patients in the overall PAH and repaired CHD-PAH groups were female. A similar proportion of patients were given treatment compared with those on a placebo (49.2% vs. 50.8%). For those assigned one of the therapies, mean age at enrollment was lower for repaired CHD-PAH patients relative to the whole PAH population (41 vs. 48).
The main goal of the analysis was to measure the time until the first confirmed death or serious complications, including disease progression or PAH worsening leading to hospitalization, long-term oxygen therapy, a lung transplant, or atrial septostomy, which is a surgical procedure to reduce strain on the heart’s right side.
Death and serious complications were less frequent among patients given active treatment over those on a placebo (31% vs. 44%), with active treatment significantly reducing the risk of such events by 37% in the whole PAH patient population. The same was observed among patients with repaired CHD-PAH (17% vs. 31%), where active treatment was significantly associated with a 50% lower risk.
PAH treatment was superior to a placebo at improving patients’ functional capacity in the six-minute walk test, easing shortness of breath, and slowing the increase in NT-proBNP, a blood marker of heart train, an analysis of secondary measures showed
While the magnitude of treatment effect was generally similar between the whole PAH and repaired CD-PAH groups, differences relative to placebo reached statistical significance only in the whole PAH patient population.
“Treatment with approved PAH drugs provided a similar reduction in [mortality/morbidity] risk in patients with repaired CHD-PAH when compared with the overall PAH population,” wrote the researchers, who said more studies are needed to know if “improvements in clinical and functional parameters accompany the significant reduction in the risk of [mortality/morbidity] achieved with PAH treatment in patients with CHD-PAH.”
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Corvista Health’s point-of-care device, which can be used to predict pulmonary hypertension in a noninvasive way, also appears to work well at identifying heart failure, accurately detecting patients who have it while ruling it out in others, a study found.
Using machine learning to decode data from patients, the Corvista System accurately detected elevated pulmonary capillary wedge pressure, or PCWP — an indirect measure of pressure in the heart’s left top chamber, which receives oxygen-rich blood from the lungs.
In the study, researchers at Corvista calculated that the diagnostic odds ratio of the machine learning algorithm was 29, meaning a patient with heart failure was 29 times more likely to test positive than a patient without heart failure.
“The new data provide preliminary results for the feasibility of our noninvasive machine learning-based test to detect elevated PCWP with a high level of accuracy,” Charles R. Bridges, MD, Corvista’s executive vice president and chief scientific officer, said in a company press release.
Bridges presented these data in a poster at the American College of Cardiology’s Annual Scientific Session, held late last month in Chicago. The poster was titled “Point-of-Care Testing for Pulmonary Capillary Wedge Pressure Elevation Using Machine Learning on Non-invasive Signals.”
Symptoms of PH, heart failure overlap, complicating diagnosis
Pulmonary hypertension occurs when blood pressure in the vessels that carry blood from the heart to the lungs becomes abnormally high. This makes it harder for the heart to pump enough blood to the lungs, leading to symptoms such as shortness of breath. If left untreated, it can result in heart failure.
Diagnosing pulmonary hypertension and heart failure can be difficult because many of their symptoms overlap with those of other conditions. The process is often lengthy and involves several different tests.
With the Corvista System, signals naturally emitted by the heart are collected for 3.5 minutes while the person is at rest. A machine learning algorithm analyzes the data in the cloud — a remote data processing service — making the results quickly available to doctors through a dedicated web portal.
In a previous study, the company showed that its machine learning algorithm performed comparably to transthoracic echocardiography — a standard diagnostic test to measure blood flow through the heart — in predicting pulmonary hypertension in patients with new-onset symptoms of cardiovascular disease.
Bridges noted, however, that “although echocardiography is an excellent modality for estimating left ventricular ejection fraction (EF), its sensitivity for diagnosing diastolic dysfunction is rather abysmal.”
EF refers to how much blood the heart’s left bottom chamber can pump. Diastolic dysfunction, meanwhile, is the heart’s inability to relax and fill with blood between heartbeats, which, if impaired, can lead to heart failure with preserved EF, or HFpEF.
While it accounts for about half of all cases of heart failure, “the underdiagnosis of HFpEF is arguably the largest unmet need in cardiovascular medicine today,” according to Bridges, who noted that it represents “approximately $25 billion in direct costs to the U.S. healthcare system annually.”
Algorithm ‘promising’ for development of point-of-care test for PCWP
The new study involved 283 patients, according to the poster’s abstract.
According to the researchers, the machine learning algorithm “achieved a clinically relevant performance in the identification of [patients] with elevated PCWP.”
Its sensitivity was 90%, meaning it correctly identified 9 of every 10 patients with heart failure. Its specificity was 76%, meaning it correctly ruled out slightly more than three-quarters of patients without heart failure.
It is critically important to differentiate subtypes of pulmonary hypertension which have distinct often lifesaving guideline-directed treatments.
A total of 247 features collected from data were found to help differentiate patients with elevated PCWP (greater than 18 mmHg) from those with a normal measurement (equal to or less than 18 mm Hg). The data were measured using a procedure called invasive right heart catheterization.
The algorithm also showed a positive likelihood ratio of 3.8 and a negative likelihood ratio of 0.13. This means that if a patient tests positive, they are more likely to have heart failure; if a patient tests negative, they are unlikely to have heart failure.
It performed well across different groups, such as by sex and age, according to the researchers.
Bridges noted that pulmonary hypertension “is also vastly underdiagnosed,” saying “it is critically important to differentiate subtypes of pulmonary hypertension which have distinct often lifesaving guideline-directed treatments.” Based on guidelines, PCWP is required to differentiate subtypes of pulmonary hypertension.
The researchers suggest their algorithm “is promising for the future development of a noninvasive, point-of-care test for PCWP.”
Per the team, “estimating PCWP would facilitate earlier diagnosis and appropriate treatment of various conditions, including heart failure and [pulmonary hypertension].”
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The advocacy organization Phaware is launching a series of podcasts to explore new advances in the treatment of pulmonary hypertension associated with interstitial lung disease (PH-ILD).
The series will be launched as part of “I’m Aware That I’m Rare: the phaware podcast,” which can be found online and on Spotify, Apple Podcasts, and YouTube Music. The first episode of the PH-ILD series will premiere on April 7, with new ones dropping every Monday.
“These episodes [represent] a pivotal moment for the PH-ILD community,” Steve Van Wormer, cofounder of Phaware and executive producer of the “I’m Aware That I’m Rare” podcast, said in a press release from the nonprofit.
Interstitial lung diseases, known as ILDs, are a group of disorders marked by scarring in the lungs. Such scarring can set the stage for pulmonary hypertension, which is defined by abnormally high pressure in the vessels that carry blood through the lungs.
Phaware podcasts sponsored by 3 companies developing treatments
The upcoming podcast series will feature discussions of ongoing clinical trials in PH-ILD as well as treatments that are on the verge of potential approvals. The series will feature a range of experts in PH-ILD from U.S. institutions who will share their perspectives on these advances.
“We are thrilled to bring together such an distinguished panel of experts to discuss how research and innovation are driving meaningful change for patients worldwide,” Van Wormer said.
The PH-ILD podcast series is sponsored by Liquidia, Pulmovant, and Gossamer Bio. All three companies are working to develop new treatments for the condition.
Podcasts are an excellent channel for increasing disease awareness and fostering community among patients, families, and their providers.
Roger Jeffs, PhD, CEO of Liquidia, said the company is “grateful to join hands with Phaware on this important awareness campaign.”
The U.S. Food and Drug Administration is now reviewing Liquidia’s Yutrepia as a treatment for PH-ILD. A final decision by the regulatory agency is expected by May 23.
“Raising awareness of pulmonary hypertension associated with interstitial lung disease (or PH-ILD) alongside our advocacy partners is a critical component of our collective effort to ensure patients, caregivers and physicians feel informed and empowered,” Jeffs said. “We look forward to helping ensure this vastly underserved patient population can be properly diagnosed and treated effectively.”
Gossamer’s experimental therapy seralutinib is also being developed as a potential treatment for PH-ILD. Pulmovant’s mosliciguat, meanwhile, is being tested in a PH-ILD Phase 2 clinical trial (NCT06635850) at two U.S. locations.
Drew Fromkin, CEO of Pulmovant, noted that the podcast series will “[bring] forward stories and expert opinions to educate and empower patients about this life-threatening and often-misdiagnosed disease.”
Fromkin said Pulmovant is “proud to support the innovative work of Phaware global association” in this new venture.
“Podcasts are an excellent channel for increasing disease awareness and fostering community among patients, families, and their providers. Pulmovant is pleased to be at the forefront of this important initiative alongside some of our industry colleagues,” Fromkin said.
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Treatment with the approved therapy Winrevair (sotatercept-csrk) significantly reduced the risk of death, lung transplant, or hospitalization in people with pulmonary arterial hypertension (PAH), according to results from the Phase 3 ZENITH clinical trial.
Findings were published in The New England Journal of Medicine, in the study, “Sotatercept in Patients with Pulmonary Arterial Hypertension at High Risk for Death.” The results were simultaneously presented at the American College of Cardiology’s Annual Scientific Session and Expo. The work was funded by Merck, the company that sells Winrevair.
“These data support the practice-changing potential of Winrevair for a broad range of patients with PAH,” Marc Humbert, MD, PhD, co-author of the study at Université Paris-Saclay, said in a press release from Merck.
The results from the ZENITH study were so positive the placebo-controlled trial ended early so all patients could switch to receiving Winrevair at the recommendation of an independent data monitoring committee. Another trial testing Winrevair against a placebo in people with recently diagnosed PAH was also ended early so all patients could receive active treatment.
“The impressive results from ZENITH demonstrated that patients on Winrevair had a 76 percent risk reduction in the composite of all-cause death, lung transplantation and hospitalization for PAH compared to placebo, with improvement observed early in treatment and increasing benefit throughout the study,” said Eliav Barr, MD, chief medical officer, senior vice president and head of global clinical development at Merck Research Laboratories. “These results led to the ZENITH study being the first PAH clinical trial stopped early due to overwhelming efficacy, representing an important milestone in clinical research with promise for the PAH community.”
Winrevair reduces abnormal growth of blood vessel cells
Winrevair was approved last year to treat adults with PAH in the U.S. as well as the European Union. The therapy is given by subcutaneous (under-the-skin) injection every three weeks, and it is designed to reduce the abnormal growth of blood vessel cells that contributes to disease progression in PAH.
The ZENITH clinical trial (NCT04896008) enrolled 172 people with PAH who, based on standardized metrics, were judged to be at high risk of dying within one year despite available treatments. Most participants were white and female. The patients were randomly assigned to receive Winrevair or a placebo, given on top of standard therapies.
The study’s main goal was to assess whether Winrevair was better than the placebo at preventing patients from reaching a composite endpoint that included death, lung transplant, and/or being hospitalized due to worsening PAH for at least 24 hours.
Results showed the study met this goal: fewer than 1 in 5 (17.4%) patients on Winrevair experienced one of these adverse outcomes compared with more than half (54.7%) of patients on the placebo. Statistically, this works out to a 76% lower likelihood of adverse outcomes in patients given Winrevair.
“The ZENITH study represents the first PAH clinical trial with a primary endpoint comprised entirely of major outcome measures – all-cause death, lung transplantation and hospitalization for PAH,” Humbert said. “Winrevair had a significant and clinically meaningful impact on the composite of these outcomes.”
Looking at each outcome individually also indicated favorable effects of Winrevair. Patients given the therapy had lower rates of death (8.1% vs. 15.1%), transplant (1.2% vs. 7%), and PAH-related hospitalizations (9.3% vs. 50%). These differences weren’t all statistically significant, though the researchers stressed that since the trial was ended early there’s a lower likelihood of finding statistically meaningful results on these secondary endpoints because there wasn’t much data available to analyze.
Side effects include nosebleeds, damaged veins beneath the skin, low platelet count
The most common treatment-related safety issues seen in ZENITH included known side effects of Winrevair such as nosebleeds, bleeding from the gums, damaged veins visible beneath the skin (known as telangiectasias or spider veins), high counts of hemoglobin (the protein that carried oxygen in red blood cells), and low counts of platelets. Vomiting and back pain were also more common in patients given Winrevair than those given the placebo. Overall, “the safety profile was generally consistent with that seen in previous trials,” the researchers wrote.
After the ZENITH study ended, the participants had the option to continue into an open-label extension study called SOTERIA (NCT04796337) where all are being treated with Winrevair and monitored for long-term outcomes. Data from this study will help characterize the long-term safety profile of the therapy, researchers noted.
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The U.S. Food and Drug Administration (FDA) is poised to soon issue a final determination on Yutrepia, an inhaled dry powder formulation of treprostinil, up for approval to treat both pulmonary hypertension associated with interstitial lung disease (PH-ILD) and pulmonary arterial hypertension (PAH).
The FDA granted Yutrepia tentative approval for PH-ILD last year and for PAH in 2021. These tentative approvals meant that the FDA judged that Yutrepia met all criteria for quality, safety, and efficacy, but the therapy was not able to be brought to market.
At issue was the fact that United Therapeutics had exclusive marketing rights for Tyvaso DPI, United’s inhaled dry powder formulation of treprostinil, which is currently the only version of the therapy approved in the U.S. United’s exclusivity period is set to expire on May 23, 2025. According to Liquidia, the developer of Yutrepia, the FDA has accepted a resubmission of an application seeking approval of the therapy, and announced an intent to make its final decision on Yutrepia public on May 24.
Treprostinil mimics the activity of hormone that widens blood vessels
“We are pleased that the FDA has responded promptly to the resubmission … We look forward to working with the FDA over the coming months as we seek final approval for Yutrepia and, in the meantime, will continue preparations to support a launch of Yutrepia as soon as possible,” Roger Jeffs, PhD, CEO of Liquidia, said in a company press release.
The active ingredient in Yutrepia, treprostinil, works by mimicking the activity of prostacyclin, which is a hormone that triggers blood vessels to relax and widen. The therapy aims to reduce blood pressure in the vessels of the lungs; both PAH and PH-ILD are marked by abnormally high pressure in lung blood vessels.
The FDA’s tentative approvals of Yutrepia were based on data from the Phase 3 clinical trial called INSPIRE (NCT03399604). The study tested Yutrepia in 121 people with PAH, including 66 who were not being treated with any form of inhaled treprostinil and 55 who had switched to Yutrepia from Tyvaso, which is nebulized treprostinil. Results showed that Yutrepia was generally safe and well tolerated, and the therapy led to improvements in measures of exercise capacity and quality of life.
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Pulmonary hypertension (PH) is associated with an increased risk for sleep disorders, including obstructive sleep apnea, and structural brain changes at the genetic level, according to a recent study.
These findings may help “develop new guidelines for the clinical management, personalized diagnosis, and treatment of PH, sleep disorder diseases, and their associated neurological comorbidities [coexisting disorders],” researchers wrote.
The study, “Triangular Causality Among Pulmonary Hypertension, Sleep Disorders, and Brain Structure at the Genetic Level: A Mendelian Randomization Study Focused on the Lung-Brain Axis,” was published in Nature and Science of Sleep.
PH is a progressive disease characterized by unusually high blood pressure in the pulmonary arteries, the blood vessels that supply the lungs. This restricts blood flow to the lungs and causes more strain on the heart which needs to work harder to pump blood.
Sleep disturbances common in PH
People living with PH may experience systemic (body-wide) complications, including sleep disorders and chronic kidney disease. The most common sleep disturbance is obstructive sleep apnea (OSA), which is when breathing is interrupted due to airway blockage, with impacts on sleep quality and daytime function.
However, existing research shows the prevalence of PH in patients with OSA varies significantly, with few studies investigating the prevalence of OSA in PH patients, the researchers noted.
To learn more about the relationship between PH and sleep disorders, researchers in China conducted a genetic-level study, based on genome-wide association study data. This type of approach looks at genetic variations to find genes associated with specific diseases.
For that, they analyzed data from four international studies, which included several sites across the U.S., France, Germany, the Netherlands, Italy, and the U.K. Then, identified gene variants were analyzed for their potential relationship with PH and sleep disorders, using a method called Mendelian randomization, which assesses the association between factors such as lifestyle habits or biomarkers and specific health outcomes.
Overall, a total of 11,744 individuals of European ancestry were included, and 2,085 of them had a PH diagnosis. The researchers identified 24 genetic variants, in 18 genes, strongly associated with PH. Further analysis demonstrated these genes were enriched in biological pathways related to calcium-mediated signaling and ion protein channels, “both of which are strongly linked to the physiological mechanisms of PH.”
PH increases risk for sleep disorders by 1.8%
Results showed PH significantly increased the risk for sleep disorders by 1.8%, including OSA by 2.2%. On the contrary, no evidence was found to suggest having sleep disorders may increase the risk for PH.
A total of 153 genetic variants, linked to 15 brain structures, were identified. Results indicated PH could be a risk factor for changes in total brain structure and white matter, which is composed of nerve cell extensions. However, no specific changes in any brain region increased the risk for PH.
Although initial findings indicated changes in brain surface area could be a risk factor for developing OSA, this effect was not confirmed in a further analysis.
Still, changes in the amygdala, which is a brain region involved in processing memory and emotional responses, were associated with a lower risk of developing sleep disorders by 0.4%, and OSA by 0.3%.
“This study identifies significant causal links between PH, sleep disorders, and structural brain changes, establishing a triangular cyclic relationship that supports the lung-brain axis concept,” the researchers wrote. The lung-brain axis refers to the connections between neurological and respiratory functions.
The scientists also noted the potential relationships were obtained using a computational model, so applying “these results to clinical practice, extensive trials and observational clinical studies [is] necessary to ensure a robust and conclusive framework.”
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Following my heart and lung transplant in 2018, I really wanted a smartwatch. Admittedly, the dream was that I’d be using this piece of tech to track ambitious hikes and long walks. Maybe even runs. I had a very athletic vision of myself following recovery, which, of course, ended up not being my reality.
Still, I got a smartwatch, and it’s proven to be a helpful accessory in my post-transplant life. It’s just one of the many ways I rely on modern technology to keep track of my health and simplify my day-to-day activities.
My phone and smartwatch
While it’s not the same as what I envisioned, I do use my watch to track my workouts. Tracking my fitness has proven helpful in monitoring my long-term health. When recovering from a period of difficulty, I’m able to go back through this data and see when I was last regularly active. In the past, this has helped me understand my health trends over months and even years.
I also use my watch to monitor my heart rate, which sometimes runs high. When I don’t have a pulse oximeter nearby, my watch is helpful for quick check-ins, and I find the data to be fairly reliable. Once again, I can compare these trends over time, which is incredibly informative.
Finally, I rely on my phone and watch for medication reminders. The timing for transplant medications is critical. Every day, I take my meds at 10:30 a.m. and 10:30 p.m., and I set alarms on my phone for each of these. If I also wear my watch, the alarm will sound as well. This system is very effective and keeps me from missing doses.
Apps and patient portals
Using health-based applications also helps me stay on top of how I’m feeling. Countless apps allow for healthcare input, such as changes to medications and symptoms. An app as simple as a period tracker can bring clarity to the impact that menstruation has on my other symptoms, such as migraines.
Another modern improvement is the use of patient portals, which have become much more common since I was diagnosed with pulmonary hypertension in 1999. Patient information used to be so difficult to access. My mom’s initial record-keeping solution for my health information involved handwritten notes in a black notebook.
Now, as an avid portal user, with the click of a button, I can access the results of my lab work, information about my medications and diagnoses, and a calendar of all my upcoming appointments. Knowing that this information exists in one easily accessible place is a relief.
Modern conveniences
Finally, generic tech improvements in society have undeniably benefited the chronic illness community. Some examples include Uber, DoorDash, and Instacart. These app-based interfaces are all geared toward services of convenience. I’m a frequent user of these types of services, especially in times of increased illness when having food or groceries delivered makes the day much easier.
I’m not always excited about the task of managing my physical health, along with the massive amounts of record-keeping it requires. But I am thankful to live in a time of technological advancement that makes it all a bit easier. It’s always thrilling to learn that there is a new aspect of modern tech that I can take advantage of to better my knowledge of my health or to improve my quality of life.
Note: Pulmonary Hypertension News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Hypertension News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to pulmonary hypertension.
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