Survey reveals patients’ preferences for treating PAH

Patients prefer treatments with higher impact on physical limitations, survival

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by Andrea Lobo |

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This illustration shows a person making a choice of medicines.

When choosing a treatment regimen, people with pulmonary arterial hypertension (PAH) consider the impact on physical limitations and survival at three years as the most important parameters to consider, according to a study in Germany.

Meanwhile, unplanned hospitalizations within three years and short-term side effects were reported as less important when making treatment decisions.

“Patient preferences should be considered in treatment decision-making to better balance patient’s expectations regarding the known risk-benefit ratio of treatment,” the researchers wrote.

The study, “Patient preferences in Pulmonary Arterial Hypertension (PAH), a Latent Class Analysis to Identify Preference Heterogeneity,” was published in the journal Value in Health.

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PAH is caused by the narrowing of pulmonary arteries, which transport blood from the heart to the lungs. This restricts the blood flow across the lungs, causing high blood pressure, or hypertension, and making the heart work harder to pump blood.

PAH treatment requires a multidisciplinary strategy that encompasses medication, risk assessment, disease prognosis, and response to treatment. Several measures have contributed to better evaluating clinical outcomes, including lung and heart status, measure of blood flow dynamics, exercise capacity, and biomarkers.

“However, patient surveys indicate that more can be done to improve holistic care and patient engagement,” the researchers wrote. “Understanding of patient preferences can support patient-centered medicine development and medical care.”

Considering the limited data available about PAH patients’ preference in terms of treatment and disease management, researchers in Germany and at Janssen (now Johnson & Johnson Innovative Medicine) conducted the first patient preference study relating to drug treatment in PAH.

They conducted an online stated-preference survey on adult PAH patients with slight to marked limitations during physical activity, who were recruited through a patient organization in Germany.

How the patient survey was designed

In the survey, patients were asked to choose between three hypothetic treatment profiles defined by six treatment features (benefits or risks) at four clinically meaningful levels. Patients completed a total of 12 choice tasks, choosing the best and worst treatment regimen.

A total of 83 patients completed the survey; most were women (76%) and their mean age was 59. Nearly half (48%) self-reported their general health as fair, while 38% considered their health good and 12% poor.

More than half reported needing help in daily life (61%) and had marked limitations in physical activity (52%).

Survey results showed that a high survival rate (85% or higher) over three years and none-to-mild limitations during physical activity after three years, were the most important attributes when choosing a treatment regimen.

In terms of unplanned PAH-related hospitalizations at three years, responders clearly preferred regimens associated with a 90% chance of avoiding such hospitalizations. But this type of attribute, as well as short-term adverse events such as digestive symptoms, pain, and headache, were generally considered less important.

Four classes of patients

The researchers then identified four classes of patients based on variations in terms of preferences.

Those in class 1 (19%) preferred therapies that avoided side effects in the first six months of treatment. They also granted some importance to survival and physical limitation benefits, while reducing unplanned hospitalizations appeared to be the least important.

“Participants in other classes placed most importance on physical activity limitations after 3 years and/or survival at 3 years,” the researchers wrote.

Nearly two-thirds of respondents considered reductions in physical activity limitations alone (34%, class 2) or with high survival rates (30%, class 3), to be the most important treatment attributes. For patients in these classes, other attributes were considered relatively unimportant.

For patients in class 4 (17%), survival was the most important attribute in their decision-making process.

The variability in patient’s preferences did not seem to be affected by a patient’s characteristics and attitudes, but some differences were associated with self-reported limitations.

For instance, patients in class 4, who focused more on survival, were more likely to report marked physical activity limitations (79%) and needing daily help (100%) than those in the other classes.

“These results suggest that once patients adapt to living with marked limitations in their daily activities, they may focus treatment choices on survival,” the team wrote.

Positive effects on patient-caregiver relationship

“Understanding patient preferences in PAH can have a positive impact on the patient-caregiver relationship and facilitates shared decision-making,” the researchers wrote, considering that “patient preferences should be considered in personalized treatment planning decisions.”

Among the study’s limitations, the team noted the small sample size and the fact that all responders were German, which may limit the generalization to patients in other geographical areas with different healthcare systems.

“Future research should include a larger and more diverse sample, perhaps through collaboration with multiple patient organizations or health care providers,” the researchers concluded.

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