Poor Right Heart Function Linked to Worse Life Quality for PAH Children

Quality of life found similar to that of their peers with severe heart disease

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Children with pulmonary arterial hypertension (PAH) experience lower quality of life because of their functional impairments, especially if they have poor right heart function, according to a study conducted in Hungary.

Despite PAH treatment, the health-related quality of life (HRQoL) in these children was similar to that of pediatric patients with severe heart disease due to other causes, and was correlated with reduced systolic function in the right ventricle of the heart, which is the chamber responsible for pumping oxygen-depleted blood to the lungs.

“These results point to the need for psychosocial rehabilitation […] to improve the [quality of life] in this severely ill population,” researchers wrote.

The study, “Assessment of Quality of Life in Children With Pulmonary Hypertension Using Parent and Self-report Questionnaires,” was published in the journal Transplantation Proceedings.

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PAH is characterized by a narrowing of the pulmonary arteries that supply blood to the lungs, leading to high blood pressure and symptoms such as shortness of breath and fatigue. As a result, the heart’s right ventricle needs to work harder to pump blood.

Recent therapeutic advances have greatly enhanced the survival of children with PAH. The improvement of functional status and quality of life has now become the main goal of PAH therapeutic strategies.

Prior studies have suggested that PAH children experience a lower quality of life than healthy children, but also when compared to children with cardiac disorders or cancer.

Health-related quality of life assessed based on reports from patients, parents

Now, researchers in Hungary conducted a survey study directed at children and their parents to assess HRQoL of pediatric PAH patients.

In total, they analyzed data of 25 children undergoing treatment (age 2 to 18, 17 boys). As for PAH classification, 15 patients had PAH associated with congenital heart disease, one had idiopathic (no known cause) PAH, two had PAH associated with lung disease, and the other seven had multifactorial PAH associated with heart disease.

For PAH medications, 18 patients were being treated with phosphodiesterase type 5 inhibitors or endothelin receptor antagonists, while six patients underwent a combo therapy with both. One patient was on a triple PAH-specific combination with the addition of prostacyclin.

Growth delay was seen in five of the 25 children (20%) when height or body mass index (a measure of body fat) were the criteria, and observed in six children when it was defined by weight.

Quality of life was assessed with two questionnaires, the Pediatric Quality of Life Inventory 4.0 generic core scales (PedsQL-GM) and the PedsQL 3.0-cardiac module (PedsQL-CM).

The PedsQL-GM evaluates four domains — physical, emotional, social, and schooling function — while the PedsQL-CM evaluates heart problems-symptoms, treatment, perceived physical appearance, treatment anxiety, cognitive problems, and communication.

Patients age 5 to 18 with appropriate cognitive capacity were asked to complete the two questionnaires. Each question was scored from zero to five, with a total score ranging from zero to 100. Higher scores indicate better quality of life.

The questionnaire’s outcomes were compared to data from the general pediatric population in Hungary and to data from children with heart disease who were patients at a cardiac unit.

In total, the researchers analyzed 25 parent reports and 15 patient self-reports.

These results point to the need for psychosocial rehabilitation […] to improve the [quality of life] in this severely ill population

PAH children score lower in four quality-of-life domains

The PedsQL-GM scores in the four quality-of-life domains were significantly lower for PAH children compared to the general pediatric population. PAH children scored lower in the PedsQL-CM total scores and in scores related to the physical, emotional, social, and psychosocial health domains when compared to children with other heart diseases.

“Using the detailed breakdown of patients with different disease severity, PedsQL-CM scores of patients with PAH were very similar to those with chronic heart disease with great complexity,” the researchers wrote.

At the time of the survey, 12 children (48%) were in group 1 of the World Health Organization functional classification (WHO FC), 11 patients were in group 2 (44%), and two children were in group 3 (8%).

Overall, PedsQL-CM scores in parent reports showed a significant negative correlation with children’s age — meaning higher scores were found for younger patients.

In addition, PedsQL-GM patient self-report scores showed a positive correlation — meaning the greater one, the greater the other — with the z-score of the tricuspid annular plane systolic excursion (TAPSE), a measure of right ventricle cardiac function. A z-score measures how far a certain data point is from a reference value. In this case, a TAPSE z-score below 2.0 was deemed a clinically significant PAH-specific parameter.

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No major differences seen between children given single or combo therapy

The researchers conducted further analysis looking at specific WHO groups. Children in WHO FC group 1 had higher (better) scores in the parent PedsQL-GM domain related to school functioning, and in parent PedsQL-CM scores in heart problems and symptoms.

Also, the data showed that children with higher TAPSE z-scores had significantly higher PedsQL-GM parent scores in the physical, emotional, social, and school function domains. Also, higher TAPSE z-scores correlated with fewer cognitive problems as reported by parents in the PedsQL-CM.

The researchers also assessed the influence of treatment regimens in PedsQL scores. No significant differences were observed between children given single or combo therapy.

Overall, these findings suggest that HRQoL of children with PAH “is impaired in almost every domain compared with healthy children and is similar to the group of patients with the most severe chronic heart disease with great complexity,” the researchers wrote.

“We were also able to establish an association between the reduced systolic function of the right ventricle, as assessed by TAPSE, and the significantly lower HRQOL in all core domains” they concluded.

According to the team, the small number of participants and the limited amount of data precluded a detailed subgroup analysis and more precise correlations with disease severity.

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