U.S. study highlights unmet needs in people living with CTEPH
Ongoing symptoms and work impacts reported despite treatment
People with chronic thromboembolic pulmonary hypertension (CTEPH) continue to experience significant symptoms that interfere with daily life, even when receiving treatment, according to a real-world U.S. study.
The study also found that people treated at Pulmonary Hypertension Association (PHA)-accredited centers tended to have more severe disease and were more likely to receive CTEPH-specific therapies.
The study, “Real-World Treatment Patterns and Burden of Chronic Thromboembolic Pulmonary Hypertension Patients across Consultation Settings in the USA,” was published in Pulmonary Therapy.
Understanding CTEPH and its treatment options
CTEPH is a rare form of pulmonary hypertension in which blood clots block the pulmonary arteries, leading to high pressure in the blood vessels that carry blood to the lungs.
Current treatment options for CTEPH include anticoagulants to prevent new clots, riociguat (marketed as Adempas, a generic formulation is available), and surgical procedures, such as pulmonary thromboendarterectomy (PTE) or balloon pulmonary angioplasty (BPA).
PTE is a surgery that removes blood clots and scar tissue from the lungs to restore blood flow and may be curative in some cases. BPA, a non-surgical procedure, involves widening blocked pulmonary arteries using small balloons and may be recommended for patients deemed unsuitable for surgery.
While PTE can significantly enhance survival and quality of life, many patients are considered inoperable due to the location of their blood clots or other health conditions. These patients, or those who continue to have pulmonary hypertension after surgery, may then be offered BPA.
Because treatment approaches for CTEPH can vary widely, “it is essential to understand how they are deployed across real-world settings,” the researchers wrote.
Looking at real-world care and daily life impact
With this in mind, a team of researchers, including scientists at Actelion Pharmaceuticals (a Johnson and Johnson company), set out to examine the clinical features, treatment patterns, and quality of life of people with CTEPH in the U.S.
The researchers analyzed data from the Adelphi Real World CTEPH Disease Specific Programme, a survey of U.S. physicians and patients conducted between September 2023 and May 2024.
In total, 98 physicians — 51% pulmonologists and 49% cardiologists — provided data on 153 people with CTEPH. Participants had a mean age of 59.2 years, and 59.5% were women. Most participants were white (71.9%), and 40.5% were former smokers.
Just under half of patients (44.4%) were treated at PHA-accredited centers. According to the physicians, the most commonly reported symptom was shortness of breath with exertion (73.2%), while hypertension was the most frequent co-occurring condition (45.1%).
Shortness of breath with exertion was also the most common symptom reported by patients themselves (52.1%), followed by chest pressure, tightness or pain (41.7%), and fatigue (39.6%).
This likely reflects previous findings that specialists in the USA are more willing to prescribe medication off-label. Accredited centers may also likely have more experience prescribing medication approved for use in [pulmonary hypertension] indications.
Physicians classified about half of the patients (50.3%) as having mild CTEPH, while 38.6% had moderate disease and 11.1% had severe or very severe disease. Most patients (54.1%) were classified as New York Heart Association functional class II, indicating slight limitations during physical activity.
About three-quarters of people were considered operable, according to their physicians. Among patients who underwent a procedure, 82.4% received PTE, while 19.1% underwent BPA. A combination of both procedures was performed in 7.3% of patients.
At the time of the survey, 82.4% of patients were prescribed at least one pulmonary hypertension medication. The vast majority (81%) were prescribed anticoagulants, and nearly half (44.4%) received riociguat. Other therapies — including phosphodiesterase-5 inhibitors and endothelin receptor antagonists — were also commonly prescribed. This pattern reflects “some off-label prescription despite the lack of proven efficacy in patients with CTEPH in the USA, particularly in accredited centers,” the investigators wrote.
“This likely reflects previous findings that specialists in the USA are more willing to prescribe medication off-label,” they added. “Accredited centers may also likely have more experience prescribing medication approved for use in [pulmonary hypertension] indications.”
How patients are being treated in everyday practice
Most patients (76.2%) were receiving their first CTEPH medication, while 23.8% were on a second treatment or later line of therapy.
Many patients (76.2%) were considered fully compliant, meaning they took more than 80% of their prescribed doses. Overall, 87.6% of physicians reported being satisfied with their patients’ level of disease control. A similarly high proportion of patients (83.4%) also reported being satisfied with their disease control.
Among patients who completed quality of life assessments, nearly a quarter reported moderate or severe problems with mobility (24.6%), daily activities (22.6%), and anxiety and depression (20.8%). Moderate to severe pain or discomfort was reported by 15.1% of patients.
The results also showed that 26.4% of patients changed how they work because of CTEPH, and those who were employed reported notable work-related impairment.
The researchers also found that patients treated at PHA-accredited centers tended to have more severe disease. Although treatment patterns generally followed guidelines, BPA appeared to be underutilized overall, particularly at non-accredited centers. Accredited centers were also more likely to prescribe riociguat.
Overall, the findings highlight the need for “better education surrounding novel treatment options and their benefits, improved patient/physician communication regarding unmet needs, and a demand for novel treatments that target ongoing symptoms even after surgical intervention,” the researchers concluded.
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