What if the Diagnosis Is Pulmonary Hypertension?

CTEPH Treatment, Management Varies Widely Across Globe

A new report shows wide variability in terms of which patients with chronic thromboembolic pulmonary hypertension (CTEPH) are deemed eligible for pulmonary endarterectomy and balloon pulmonary angioplasty.

Recent medical advances have improved the treatment of chronic thromboembolic pulmonary hypertension (CTEPH), but a new report shows wide variability in which patients receive which therapies.

The study was published in the journal Pulmonary Circulation.1

New and existing treatments can significantly improve outcomes for patients with CTEPH, noted the study authors. However, they also said treatment decisions can depend on a number of factors, and many patients are deemed ineligible for some of the most effective therapies.

For example, pulmonary endarterectomy (PEA) can lead to long-term improvement in most patients, but the investigators said some patients refuse the procedure, and others are ineligible due to distal disease or an unfavorable risk profile. Additionally, about a quarter of patients require additional intervention even after PEA.

Treatments for chronic thromboembolic pulmonary hypertension depend on several factors, which include patient refusal or ineligibility because of distal diseaseImage Credit: Dzmitry - stock.Adobe.Com

Patients who cannot undergo PEA or who have symptoms following the procedure can benefit from balloon pulmonary angioplasty (BPA), they added. Several drugs have also been shown to lead to improvement in some patients with CTEPH, including the oral guanylate cyclase stimulator riociguat (Adempas; Bayer), and some physicians prescribe off-label use of therapies designed to treat pulmonary arterial hypertension, they said.

Yet, the study investigators said there remains a lack of consensus about the optimal therapeutic targets for patients, the ideal sequencing of therapies, and the role of medical therapy in the preoperative and interventional settings. In the absence of that consensus, they sought to better understand what is happening in real-world practice.

The authors used the CTEPH Global Cross-Sectional Scientific Survey (or CLARITY) survey, which included 110 closed- and open-ended questions and was available in 12 languages. It was distributed via 21 international, regional, and global scientific societies between September 2021 and May 2022. Recipients were asked to complete the survey if they were involved in the treatment of acute pulmonary embolism or CTEPH.

In all, 416 people responded to the survey. The new data are based on the 212 responses from clinicians who said they were involved in the treatment of CTEPH. Most respondents (approximately 71%) were from the Asia-Pacific region or Europe. Half were pulmonologists and 37% were cardiologists. Other respondents indicated they specialized in cardiothoracic surgery, internal medicine, vascular medicine, hematology, and radiology. A plurality of respondents (46%) said they had been working in their specialization between 15 and 29 years. Most said they were at centers that performed up to 50 PEA and/or BPA procedures per year.

The investigators found wide variation when they asked about the proportion of patients with CTEPH who were deemed eligible for PEA. In Europe, North America, and South America, more than half of respondents said that at least half of their patients in the previous year were deemed eligible for PEA. However, only 9% of respondents from the Asia-Pacific region said a majority of their patients were eligible.

In terms of carrying out the procedure, European and North American respondents said more than half of their patients with CTEPH underwent PEA, while most respondents from South America and the Asia-Pacific region said they performed the procedure on less than half of their patients with CTEPH.

When asked why patients eligible for PEA refused the procedure, the most common reasons were fear of adverse events, a preference for BPA, a lack of awareness about the treatment and outcomes, and concerns about the costs of the procedure and/or the hospital stay.

In terms of BPA, respondents from the Asia-Pacific region were more likely to report higher proportions of patients as eligible for the procedure. In Europe, North America, and South America, most respondents said a minority of patients were eligible for BPA.

Asked about the treatment goals for BPA, most respondents said quality of life, improved New York Heart Association functional class, symptom relief, and improved right ventricle function.

The study authors found that the use of medications such as riociguat was common, but also varied.

The investigators also said their findings suggest that right heart catheterization is underutilized. They cited a 2022 study showing that among 103 patients in the US who underwent PEA, only 5% were subsequently given right heart catheterization.2 The authors of that study said the findings imply that patients are not being referred for full diagnostic workups despite post-PEA symptoms of pulmonary hypertension.

In all, the present investigators said, the survey shows best practices for CTEPH therapy remain unclear.

"The observed heterogeneity in treatment approaches highlights the need to address access barriers to PEA and BPA, taking into consideration country-specific health care system factors, including the financing and organization of care," the investigators concluded. "In addition, the optimal sequence of multimodal therapies, taking into account the disease distribution, patient status, and respective treatment goals, warrants further research.

References

1. Skoro-Sajer N, Sheares K, Forfia P, et al. Treatment and management of chronic thromboembolic pulmonary hypertension (CTEPH): a global cross-sectional scientific survey (CLARITY). Pulm Circ. 2024;14(2):e12406. Doi:10.1002/pul2.12406

2. Butler O, Ju S, Hoernig S, Vogtländer K, Bansilal S, Heresi GA. Assessment for residual disease after pulmonary endarterectomy in patients with chronic thromboembolic pulmonary hypertension. ERJ Open Res. 2022;8(2):00572-2021. Doi:10.1183/23120541.00572-2021

Economic Burden In PAH Mainly Due To Treatment, Hospital Costs

Healthcare costs were found to be more than eight times higher for people with pulmonary arterial hypertension (PAH) compared with patients without the disease in a real-world study — which contributed to the greater economic burden seen for those being treated for PAH.

These costs were mainly driven by expenses in PAH medications and hospitalizations, which increased significantly in the last months of patients' lives, according to the study, led by scientists from Merck & Co. And IQVIA, a clinical research services company.

Overall, higher costs were primarily related to hospitalizations, indicating a higher care burden.

"The findings from this study suggest that PAH imposes substantial economic burden on the US healthcare system," the researchers wrote.

"Whether better management of the underlying disease, through improvements in time to diagnosis, adherence to treatment guidelines, or use of next‐generation medications in patients might help abrogate these costs warrants further consideration," the team added.

The study, "Excess healthcare resource utilization and costs for commercially insured patients with pulmonary arterial hypertension: A real-world data analysis," was published in Pulmonary Circulation. It was funded by a Merck & Co. Subsidiary.

Investigating the economic burden of PAH in the US

PAH is a type of pulmonary hypertension (PH) characterized by the narrowing of the pulmonary arteries. These blood vessels transport blood through the lungs, and such narrowing restricts blood flow and causes high blood pressure, known as hypertension.

In the U.S., the disease has been estimated to affect 109 patients per million adults ages 19-64, and 451 patients per million adults 65 and older. PAH has been associated with a substantial economic burden, at least in part due to delays in diagnosis and treatment initiation.

To estimate the real-world burden of the disease, scientists from the two companies, along with a researcher from Inova Fairfax Hospital, in Virginia, compared healthcare resource utilization and associated costs in commercially insured PAH patients to matched patients without PH. The team also explored the disease burden in PAH patients near the end of life. For that, they analyzed data from a health plan database in the U.S.

A total of 386 adults with PAH and 3,669,925 without any form of PH were identified as being treated between October 2014 and May 2020, or through March 2022 for end-of-life patients.

Overall, those with PAH were older than the group without PH (mean age 50.9 vs. 42.5 years), and had a higher mean Charlson Comorbidity Index (CCI) score (1.9 vs. 0.3). That score is an indication of mortality risk and the severity of comorbidities, or coexisting conditions. Also, the PAH patients had a higher frequency of lung infections (25.9% vs. 9.1 %).

The average healthcare costs were found to be significantly higher for those with PAH, with a mean healthcare cost of $2,914 per patient per month versus $297.

Next, the total group of PAH patients was matched to the same number of patients without PH, based on age, sex, payer type, plan type, CCI score, cardiovascular disorders, and other demographic and clinical characteristics.

As expected, shortness of breath, a PAH hallmark, was more common among PAH patients than those without PH, with rates of 67.6% versus 22.5%. Other issues also occurred more frequently among the PAH patients relative to those without the condition, including lung swelling, found in 4.9% versus 2.1%, coronary heart disease — when the heart arteries cannot deliver enough oxygen to the heart — as seen in 21.2% versus 16.1%, and alcohol-related disorders, seen for 6% versus 2.1%.

In contrast, hypertension (59.1% vs. 65.5%) and obesity (71.2% vs. 80.4%) were less frequent in the PAH group. Mean healthcare costs also remained higher in the PAH group ($3,069 vs. $1,571 per patient per month).

Total healthcare costs average $180K vs. $20K, per study

Patients were followed up to a median of 22.9 months or almost two years in the PAH group, and for slightly less time, 21.5 months, in the non-PH group. During follow-up, PAH patients had a higher rate of medical service use, including hospitalization — about 18% were hospitalized at least once in a year — and visits to the emergency department or as outpatients.

The risk of hospitalization due to any cause was almost seven times higher among PAH patients. This group's median length of hospital stay (6.7 vs. 2.4 days) also was longer than that of non-PAH patients.

Total healthcare costs were more than eight times higher in PAH patients ($183,616 vs. $20,212). These were mainly due to pharmacy costs ($115,926 vs. $7,862), which were 14 times higher in these patients and accounted for 63.1% of all costs. Hospitalizations were associated with 20% of all costs.

The study findings indicate substantial [healthcare resource utilization] and costs for PAH. While pharmacy costs were one of the major sources, hospitalization was the primary driver for [end of life]-related costs.

Among 28 patients who had died, 60.7% had at least one hospitalization within 30 days of death. That percentage was higher (82.1%) when considering six months before death.

Their mean healthcare costs per patient were $48,846 in the month before death, and $167,524 when looking at the six months before these individuals died. Hospitalizations were the main driver of these costs.

"The study findings indicate substantial [healthcare resource utilization] and costs for PAH. While pharmacy costs were one of the major sources, hospitalization was the primary driver for [end of life]-related costs," the researchers wrote.

According to the team, this "retrospective database study provides a contemporary estimate of the economic burden of patients with PAH compared to patients without PH."

"I Had To Face My Mortality At 28"

As a social media influencer, Gerry has found renewed purpose. She has begun sharing her story, building a following and connecting with important organizations. She has begun working on a collab with the American Lung Association. She has also traveled across the country speaking at events and meeting other patients with similar diagnoses.

"Having PAH has taught me that the cycle of grief is a cycle that you go through all the time and not just once. Being diagnosed with a chronic, rare, and progressive lung disease is very sobering and can emotionally take a toll on both you and your family," Gerry shared. "I would tell anyone dealing with a chronic diagnosis to show themselves grace. Show your body some grace as it tries to fight and heal. Don't be too hard on yourself because things look different than you imagined."

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