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AIMS: Transthyretin amyloid cardiomyopathy (ATTR-CM) is characterized by the accumulation of transthyretin (TTR) protein in the myocardium. The aim of this scoping review is to provide a descriptive summary of the clinical trials and observational studies that evaluated the clinical efficacy and safety of various agents used in ATTR-CM, with a goal of identifying the contemporary gaps in literature and to reveal future research opportunities. METHODS AND RESULTS: The search was performed in line with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. A literature search using several databases for observational and clinical trials investigating the treatment modalities for ATTR-CM was undertaken. We extracted data including study characteristics, primary endpoints, and adverse events from each study. A total of 19 studies were included in our scoping review. 8 were clinical trials and 11 were observational analyses. The drugs evaluated included tafamadis, acoramidis, revusiran, TUDCA and doxycycline, diflusinil, inotersan, eplontersen, and patisiran. Tafamidis has shown to be efficacious in the management of ATTR-CM, particularly when initiated at earlier stages. RNA interference and antisense oligonucleotide drugs have shown promising impacts on quality of life. Additionally, this review identified gaps in the literature, particularly among long-term outcomes, comparative effectiveness, and the translation of research into economic contexts. CONCLUSIONS: Multiple pharmacological options are potential disease-modifying therapies for ATTR-CM. However, many gaps exist in the understanding of these various drug therapies, warranting further research. The future directions for management of ATTR-CM are promising in regard to improving prognostic implications.
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Background Gastroparesis symptoms seem to affect many diabetes mellitus patients. Pakistan has a high prevalence of diabetes, with an estimated 33 million people living with the condition. This study aimed to investigate the frequency of gastroparesis symptoms in patients with type-2 diabetes mellitus (T2DM). Methods This cross-sectional study was conducted from April to May 2022 in the outpatient Department of Medicine at the University College of Medicine and Dentistry, Lahore, Pakistan. Patients of both genders aged between 18 and 75 years and having T2DM were analysed. Data regarding demographic information, medical history, BMI assessment, and symptoms related to gastroparesis, as measured by the Gastroparesis Cardinal Symptoms Index (GCSI) were collected at the time of enrolment. For gastroparesis, a diagnostic cutoff of ≥1.90 was considered as per GCSI. Results Of a total of 148 T2DM patients, 85 (57.4%) were females. The mean age was calculated to be 54.0±11.3 years, ranging between 23 and 75 years. There were 134 (90.5%) patients who were using metformin. The most common symptom was fullness, reported by 66 (44.6%), while nausea, bloating, early satiety, retching, and vomiting were noted in 61 (41.2%), 59 (39.9%), 55 (37.2%), 39 (26.4%), and 22 (14.9%) patients, respectively. The frequency of gastroparesis was found in 17 (11.5%) T2DM patients. Stratification of gastroparesis revealed a significant association with female gender (82.4% vs. 54.2%, p=0.0272). Conclusion The study found a high frequency of gastroparesis symptoms in patients with T2DM. Nausea and bloating were the most commonly reported symptoms, while vomiting was the least common. The prevalence of gastroparesis was significantly higher in the female gender.