by Phyllis Hanlon, Contributing Writer
Patients with WHO Group 1 PH (PAH, pulmonary arterial hypertension), a serious, progressive and often fatal lung disease, typically have a number of symptoms and limitations that affect the quality of everyday living. Depending on the severity of the disease, patients may experience negative physical, social, emotional and psychological impacts. Research has indicated that >macitentan (Opsumit®), an endothelin receptor antagonist (ERA), might help improve the health-related quality of life (HRQoL) and reduce the risk of deterioration over time.
In a collaborative effort, researchers from European and South American countries, Australia, Canada, India, Mexico and the United States examined the short and long-term effects of macitentan on the HRQoL in patients with PAH in the “Study with an Endothelin Receptor Antagonist in Pulmonary Arterial Hypertension to Improve Clinical Outcome” (SERAPHIN). The authors also sought to determine if there is an association between a patient’s baseline HRQoL and the risk of future clinical deterioration. (https://www.ncbi.nlm.nih.gov/pubmed/27671974)
The SERAPHIN trial was designed primarily to assess the long-term effects of a new PAH medication, macitentan, on a composite clinical outcome, which included risk of death and risk of worsening of PAH, according to Sanjay Mehta, MD, and colleagues. In this specific report, the focus was on assessing the potential benefits of macitentan on PAH patients’ HRQoL, as well as on the risk of future deterioration. The patients in this study were allowed to take other medications, including phosphodiesterase type 5 inhibitors, oral or inhaled prostanoids, calcium channel blockers or L-arginine, while also taking macitentan. All study subjects were World Health Organization (WHO) Functional Class II to IV and had a six-minute walk distance (6MWD) of 50 meters or more.
The authors reported that 710 of 742 patients in the original study were adults with measured HRQoL; most were female (76.9 percent) and white (55.2 percent), and the average age of subjects was 45.5. The subjects were divided into three groups: 239 were in a placebo (inactive substance) group; 237 received 3 mg macitentan; and 234 received 10 mg macitentan.
To measure the HRQoL outcomes, the authors administered the 36-Item Short Form Survey (SF-36), which reports scores in eight different areas: physical functioning; limitations as a result of health; limitations due to emotional issues; mental health; physical pain; how the patient views his general health; vitality; and social functioning.
HRQoL scores for patients in the placebo group worsened six months into the study, consistent with the known progressive nature of PAH, according to the authors. Patients who received 10 mg macitentan “significantly improved in seven of the eight SF-36 domains (all domains except general health perceptions) compared with the placebo.” Those in the macitentan 3 mg group showed similar improvements.
Additionally, the study showed that patients who had higher baseline scores on physical and mental functioning and overall quality of life had better long-term outcomes than those with lower baseline scores; macitentan-treated patients also demonstrated improvement on 6MWD and WHO functional class.
The study authors also reported that macitentan, unlike some other therapies, is administered orally, which might be part of the reason for some of the quality of life benefits.
While the positive effects on physical scores are critical, Dr. Mehta pointed out that the mental component is equally as vital. “The effect on mental domains is important, as several studies have shown that patients with PAH suffer a decline in psychological health, often experiencing feelings of anxiety, depression and stress,” he wrote.
Finally, the study noted that both doses of macitentan (3 mg and 10 mg) “significantly reduced the risk for HRQoL deterioration.”
In spite of the promising results, the authors indicated that this study had some potential limitations. They reported that the SF-36 survey is not designed to evaluate how specific symptoms affect PAH; however, the SF-36 is the most common measurement tool previously used to assess HRQoL in PAH patients in other studies.
Also, there was some missing data; however, this did not appear to affect the final outcome. But the authors pointed out that the results over the course of the whole study indicated that use of macitentan can lead to a better quality of life, both physically and mentally.