More PAH Cases in US Southwest than Registry Reports, Study Says - Pulmonary Hypertension News

Posted: 11 Mar 2020 12:00 AM PDT

The number of both new and existing cases of pulmonary arterial hypertension (PAH) in the southwestern U.S. population was found in a recent study to be higher than that reported in the largest U.S. PH registry, called REVEAL, or the Registry to Evaluate Early and Long-term PAH.

The study also pointed out that Hispanic and Native American patients are underrepresented in the national registry, although neither minority was found to have greater PAH prevalence — the number of existing cases of a disease — compared with Caucasian patients.

Moreover, living at a higher altitude was found to be associated with PAH. But this observation warrants further study and input from other PAH centers, the researchers said.

Titled "High geographic prevalence of pulmonary artery hypertension: associations with ethnicity, drug use, and altitude," the study was published in the journal Pulmonary Circulation.

PAH, part of group 1 in the World Health Organization (WHO) classification of pulmonary hypertension, can have several causes, including congenital heart disease, exposure to certain drugs and toxins, and connective tissue diseases (CTDs). It also can be of unknown cause, and thus called idiopathic PAH.

Registries like REVEAL, for PAH, are important to better understand the demographics, causes, and distribution of the disease in different populations. They also are used by researchers in designing clinical trials for testing current and future treatments.

The REVEAL registry is the largest in the U.S., reporting an estimated incidence — the number of new cases reported within a specific time period — of PAH in adults of 2.3 cases per million. The prevalence of PAH is 12.4 cases per million, according to REVEAL.

Now, the researchers at a single PAH specialty clinic in New Mexico, in the southwestern U.S., sought to further describe the disease burden and characteristics of adults with PAH in their center. Of note, the population seen at this clinic also includes patients from two ZIP codes in Arizona and one in Texas.

"Clinical experience at our institution suggested disease prevalence greater than these previous population-level descriptions of PAH," the researchers said.

The team also aimed to provide specific, relevant data on minorities, namely Hispanics and Native Americans, both underrepresented in the REVEAL study. Of relevance, native Americans are absent from any prior PAH registry. Ethnicity is thought to play a role in both the symptoms and outcomes of PAH, and a recent study in North America found significant variability of PH subtypes and survival among children of different racial and ethnic groups.

The association of PAH with altitude and known toxins and drugs were other potential links examined by the researchers. Altitude has been considered a possible contributing factor for PAH, due to hypoxia, or low oxygen, and its effects on pulmonary artery pressures.

A total of 154 PAH patients, analyzed at the southwestern center in 2016, were included in the retrospective study. The patients' mean age was 58.3 years, with 60.4% age 64 or younger; 70.8% were female.

The team calculated the prevalence of PAH in this group at 93 cases per million, which is significantly greater than in the REVEAL registry. Likewise, incidence was established at 14 cases per million, also significantly greater than that observed in the REVEAL registry.

According to the team, at least part of this marked difference is attributed to the underestimation of PAH prevalence and incidence in the REVEAL registry.

"REVEAL study investigators are forthright about these and other limitations," the researchers said.

Racial and ethnic distribution revealed that less than half of the patients in the southwestern center's group were white (48.7%). Hispanics were the next most represented ethnicity/race at 35.7%, followed by Native Americans at 10.4%. This was significantly different from the REVEAL study.

"Our cohort contained significantly more Hispanic and fewer white and fewer black patients than the REVEAL cohort," the researchers said. However, the percentage of Hispanic patients in the center's PAH group was still significantly lower relative to the total at-risk population (48.4%) in the area analyzed.

Data on Native Americans was combined with that of the Asian population — since no specific data is available in REVEAL regarding Native Americans — and corresponded to a previously published racial/ethnic group named "other." The percentage of "other" was found to be higher than in the REVEAL cohort, but this difference was not significant.

"Neither group demonstrated prevalence greater than among whites, indicating that neither Hispanic nor Native American populations specifically contributed to the overall increased prevalence of PAH in our cohort," the team said.

The most common PAH-associated condition in the group analyzed was CTDs (34.4%), followed by idiopathic PAH, or IPAH (29.6%), and exposure to drugs and toxins (20.7%). In this group, most patients (26 patients, 16.8%) had been exposed to drugs and toxins categorized as definite causes of PAH, most commonly amphetamines and methamphetamines (22 patients).

In comparison with the REVEAL study, IPAH was significantly less common in this southwestern group, and drug/toxins exposure was significantly higher.

"Public health data from our state indicates exposure rates to methamphetamine above the U.S. national average, raising the possibility of its contribution to PAH in our patient cohort," the researchers said.

Concerning altitude, the team found 152 cases in the group from ZIP codes with mean altitudes greater than 4,000 ft. Four of the individuals resided at very high altitude, above 2,500 m (about 8,202 ft).

However, given the existence of several other variables, the researchers did not make any final determinations about this factor.

"Our data are insufficient to draw definitive conclusions about the effects of moderate altitude on PAH burden," they said.

The team emphasized that more studies are needed in centers at different altitudes.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

IPF Patient Leads Lung Transplant Foundation to Help Others - Pulmonary Hypertension News

Posted: 27 Mar 2020 09:30 AM PDT

At 3 a.m. on July 6, 2003, Miami resident Jeffrey Goldstein got a call that would change his life. Doctors at the Duke University Lung Transplant Clinic had found a lung donor match for Goldstein, who had been diagnosed with pulmonary fibrosis two years earlier.

Nearly 17 years later, the 62-year-old Floridian leads the nonprofit Lung Transplant Foundation as it funds research to fight lung rejection, provides information and guidance for new transplant recipients, and manages a mentorship network.

Jeffrey Goldstein and his wife Martha Austrich. (Photos courtesy of Lung Transplant Foundation)

"We advocate and agitate on behalf of lung transplant patients," Goldstein said.

Thousands of patients receive lung transplants every year. However, only 56% of those patients are still alive five years later, he said. Transplant failure because of chronic lung allograft dysfunction accounts for 40% of deaths after one year.

Research into bronchiolitis obliterans syndrome (BOS), the most common lung rejection condition, is a funding focus for Goldstein and his foundation. Around half of patients develop BOS within five years of their transplant, according to the American Thoracic Society.

"Every day I wake up, I'm grateful, and then I'm fearful," Goldstein said in a phone interview with BioNews Services, publisher of this website. "Today could be the day that things change for me."

Diagnosis: IPF

Goldstein's journey from businessman to president of a nonprofit began in 2001, when he was CEO of a small company, Contract Resources, with five employees and annual sales of about $15 million.

While taking out the trash one night, he collapsed in his driveway, gasping for breath. He thought he was having a heart attack.

"I just laid there hoping I would survive," Goldstein said.

Twenty minutes later, he regained the strength to walk back to the house. The next day, he made an appointment with the only doctor available — an allergist. She listened to his lungs, and Goldstein immediately knew it was bad news.

"The facial expression was all I needed to see," Goldstein said. An X-ray at a nearby hospital confirmed he had idiopathic pulmonary fibrosis. No cure existed, and it was fatal; only a lung transplant could save him.

After the Duke University clinic accepted him, Goldstein and his wife moved from Miami to Durham, North Carolina, since transplant centers require patients to live within a three-hour drive in case a donor suddenly becomes available. The couple stayed for two years before heading back south because of his wife's relocation contract with Novartis.

Venture philanthropy

After the transplant, Goldstein said, he had nothing to do. He lost his business, and his friends were still working full-time.

In 2005, he connected with the now-defunct Transplant Foundation in Miami and helped with fundraising. Goldstein managed a marketing campaign, which included then-NBA player Alonzo Mourning, for Florida state license plates. A portion of every purchase went to the foundation. Four years later — shortly after Goldstein was elected president of the organization — he was diagnosed with chronic myeloid leukemia.

Jeffrey Goldstein and Stephanie Krenrich, daughter of former LTF board member, at an FDA hearing.

However, that diagnosis didn't stop Goldstein either. While undergoing chemotherapy, he built his foundation from the ground up.

Rather than fund transplants, Goldstein wanted to push research that would reduce post-transplant death and address the root cause. That decision led him to establish the Lung Transplant Foundation in April 2008.

With his business background, Goldstein added venture philanthropy to the foundation's fundraising playbook in 2015. By providing seed funding for a company in its early stages, the foundation receives a return on investment.

So far, the foundation's first and only venture philanthropy-funded project is with Paragonix Technologies, a company that builds transportation devices for solid organ transplants. The company's LUNGguard preserves donor lungs for up to eight hours.

The foundation is also looking at two potential projects that deal with diagnostic devices for transplant patients. In addition, Goldstein is involved with a company that aims to develop an anti-microbial product to prevent infections that cause lung rejection.

Improving the survival odds

While Goldstein and his foundation are early in their venture philanthropy, he hopes this kind of fundraising can provide a sustained source of income in the future. More recently, the foundation has been considering research projects such as a 3D printing model that prevents acute rejection after transplant and also treats BOS.

The odds of living five years after a lung transplant remain just over 50%, a statistic Goldstein believes he can improve by better preparing patients emotionally for a transplant. That's why he started a mentorship network in 2018.

"Being prepared mentally for every part of it is really important to the success of your transplant, both physically and emotionally," Goldstein said.

The network is named after Joseph J. Carter, who died 11 years ago at the age of 38 from congenital lung failure and is survived by his wife, Shari Carter, and two young boys. Shari started a foundation in honor of her husband, then disbanded it and donated its funds to the Lung Transplant Foundation to help build its mentorship program.

While Goldstein has helped many lung transplant recipients, he connected with his own donor family only recently, 15 years after his transplant.

"It's an overwhelming feeling of gratefulness," Goldstein said of their first encounter. "You are meeting loved ones who had the generosity and the charity in them to donate their loved one's organs."

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