Current medical research and opinion
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Novel lipid-lowering therapies are being introduced. Few studies exist of the real-world effectiveness of adenosine-tri-phosphate citrate lyase inhibition with bempedoic acid. ⋯ This audit suggests that bempedoic acid therapy is effective but that adverse effects and discontinuation are common. This suggests nocebo effects might be generalizable to all lipid-lowering drug therapies in susceptible individuals.
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To develop and validate the Asthma Severity-Health Search (AS-HScore), predicting severe asthma risk in Italian primary care. According to the current asthma treatment guidelines, the AS-HScore intended to serve as a clinical decision support system (CDSS) for General Practitioners (GPs). ⋯ The AS-HScore emerges as an accurate tool predicting severe asthma risk in the Italian primary care. It therefore shows promising application to enhance asthma care by early identification of severe cases. Implementing a score-based CDSS for Italian GPs holds potential for significantly improving asthma management and patients' outcomes.
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Acromegaly is a rare hormonal disorder that results in enlargement of the face, hands and feet. It is associated with comorbidities, increased mortality, reduced quality of life and economic burden. Adequate treatment is critical to alleviate symptoms; however, the treatment burden is substantial. To understand how different treatment aspects might ease the burden, this study investigated preferences for treatment options among people with acromegaly in the US, using a choice experiment (CE). ⋯ The results indicate that new treatment options administered as subcutaneous injections right under the skin once every fourth week at home have the potential to lower the treatment burden among people with acromegaly, emphasizing the importance of taking each person's preference into consideration when choosing treatment.
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Limited real-world evidence exists to better understand the patient experience of living with symptoms and impacts of non-alcoholic steatohepatitis (NASH). This study aimed to (1) describe patient-reported perspectives of NASH symptoms and impacts on patients' daily lives and (2) develop a patient-centered conceptual NASH model. ⋯ Our study provides insights into patients' perspectives of NASH symptoms and their impact on their daily lives. These findings may guide patient-physician conversations, supporting patient-centered treatment decisions and disease management.
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To systematically review the literature and summarize the health-related quality-of-life (HRQoL) of patients undergoing treatment for mucopolysaccharidoses (MPS), a rare, hereditary lysosomal storage disorder. ⋯ This systematic literature review revealed the substantial humanistic burden of individuals affected by MPS as well as caregivers. Significant variation in HRQoL scores was observed, however studies indicate that the quality-of-life of MPS patients is influenced primarily by severity of disease (MPS type and phenotype), and then by time of diagnosis, pain, cognitive involvement, mobility, dependence, and time of treatment initiation. Further studies are needed to assess the global humanistic burden of MPS, particularly in MPS III, VI, VII, and IX subtypes, in adults, and for a longer follow-up period. Considering the vast array of HRQoL assessment tools available and used in this study, researchers should also consider using scales with condition-specific measures to ensure appropriate estimates of effectiveness.