Study: PNH symptoms remain with treatment, impairing life quality
Patients interviewed described how fatigue affected every aspect of their lives
Fatigue affects the ability of adults with paroxysmal nocturnal hemoglobinuria (PNH) to perform daily tasks, interact socially, and stay focused, even with treatment, showing how the disease limits physical, emotional, and social well-being, according to a real-world study.
The findings indicate how the impact on health-related quality of life and daily functioning must be considered when directing treatment, “even among patients who are not newly diagnosed or who have not recently switched treatments,” wrote the study’s researchers. “Despite receiving treatment for PNH, patients experienced deficits in [health-related quality of life] and functional impairment, suggesting that opportunities to improve patient-relevant outcomes through treatment should be identified.”
The study, “Health-related quality of life and symptom-specific functional impairment among patients treated with parenterally administered complement inhibitors for paroxysmal nocturnal hemoglobinuria,” was published in the Annals of Hematology.
PNH occurs when the complement system, a cascade of proteins that are part of the immune system, attacks red blood cells that lack certain protective proteins on their surface, which normally signal to the immune system that they are a healthy part of the body. This triggers hemolysis, or the breakdown of red blood cells, causing symptoms.
Complement inhibitors — therapies that block specific steps of the complement cascade — help ease some of these symptoms, but the degree to which unresolved symptoms interfere with daily life isn’t known.
Challenges, even with treatment
Here, researchers surveyed adults with PNH in the U.S. who’d been treated with parenterally administered complement inhibitors — those administered in ways other than orally — for at least six months. The study included 61 patients who answered questions about their symptoms and quality of life. Twenty-five of the patients also took part in interviews to describe their experiences in more detail. For each patient, the study included three adults from the general population, matched by age and sex.
Compared with the general population, patients had significantly worse health-related quality of life on the PROMIS 29 + 2 Profile, which assesses physical, emotional, and social well-being. They also had a higher degree of functional impairment.
This means that, even with treatment, patients face more challenges in their daily life, with “greater functional impairment due to fatigue, dyspnea [shortness of breath], and cognitive issues than age- and sex-matched adults from the general population,” the researchers wrote.
More severe fatigue was linked to poorer health-related quality of life and daily functioning across most domains. However, sleep didn’t appear to worsen with greater fatigue.
During interviews, patients explained how fatigue affected every aspect of their lives. Many struggled with physical tasks, socializing, and concentrating at work. These difficulties remained despite treatment with complement inhibitors.
“New treatments, including those with different half-lives or methods of administration, have the potential to improve patients’ [health-related quality of life] in meaningful ways,” the researchers wrote. “Future research should focus on exploring interventions that could improve patient outcomes, particularly to better understand how reductions in fatigue may contribute to better [health-related quality of life].”
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