Objective: To describe the clinical presentation of transthyretin amyloid cardiomyopathy (ATTR-CM) and discuss current treatments and investigational products and their effect on patient outcomes. Data Sources: A literature search was performed in PubMed (September 2018 to December 2020) using the following keywords: transthyretin amyloidosis, cardiomyopathy, polyneuropathy and transthyretin amyloid cardiomyopathy, monoclonal light-chain, tafamidis, cardiac amyloidosis, ATTR cardiomyopathy, green tea and inhibition of cardiac amyloidosis, AG10, tolcapone, tolcapone and leptomeningeal ATTR, PRX004, NI006, patisiran, inotersen, vutrisiran, AKCEA-TTR-LRx, and NTLA-2001. Study Selection and Data Extraction: Clinical trials were evaluated for evidence supporting pharmacology, safety, efficacy, and measured outcomes. Data Synthesis: Until 2019, there were no approved treatments for ATTR-CM. Treatment consisted of symptom management and organ transplant. Nonpharmacological and pharmacological treatments focused on the symptoms of heart failure (HF) associated with ATTR-CM. However, there are several emerging therapies recently approved or in development to address the underlying pathophysiology. Treatment classes for ATTR-CM include transthyretin stabilizers, human monoclonal antibodies, gene silencers, and CRISPR/Cas9 gene editing. Relevance to Patient Care and Clinical Practice: ATTR-CM is a complex disease in which amyloidosis causes cardiomyopathy. Underdiagnosis is attributed to the clinical presentation being heterogeneous, indistinguishable from HF caused by other etiologies, and the need for invasive testing modalities, including endomyocardial biopsy. Improved diagnostic approaches along with targeted therapies can slow disease progression and enhance patient quality of life. Conclusion: Diagnostic modalities along with biomarker and genetic testing could detect disease earlier and target therapy more accurately. Novel therapies demonstrate potential treatment benefits and can help shape the standard of care for these patients.
The interaction between vascular endothelial growth factor and its receptor is an important therapeutic target due to the importance of this pathway in carcinogenesis. In particular, this pathway promotes and regulates angiogenesis as well as increases endothelial cell proliferation, permeability, and survival. Ramucirumab is a fully human monoclonal antibody that specifically targets the vascular endothelial growth factor receptor-2, the key receptor implicated in angiogenesis. Currently, ramucirumab is approved for the second-line treatment of metastatic non-small-cell lung cancer (NSCLC) in combination with docetaxel. In a Phase III clinical trial, ramucirumab was shown to improve the overall survival in patients with disease progression, despite platinum-based chemotherapy for advanced NSCLC. This review describes the pharmacology, pharmacokinetics and dynamics, adverse event profile, and the clinical activity of ramucirumab observed in Phase II and III trials in NSCLC.
Substance abuse and addiction are responsible for an assortment of health and financial concerns in the United States. Tools to identify and assist at-risk persons before they develop a substance use disorder are necessary. Screening, brief intervention, and referral to treatment (SBIRT) can be utilized by health-care professionals to identify those at risk to minimize healthrelated complications and the potential of developing a substance use disorder. The primary objective of this study was to provide educational training sessions on SBIRT to health-care students utilizing interprofessional education activities and assess perceptions of the training sessions and activities with regard to confidence to utilize SBIRT in at-risk patients and overall student satisfaction with SBIRT instruction. The research protocol enrolled students of pharmacy, nursing, medicine, behavioral health, and physician assistant studies who received interprofessional SBIRT training. Students completed an anonymous posttraining online survey, measuring student perceptions of knowledge gained and confidence to utilize training. A total of 303 students completed the SBIRT training. Approximately 70% of students were satisfied with the training materials, instruction, quality, and experience. After training, 78% were confident that they could perform screening for substance abuse, conduct a brief intervention (80%), and when to refer to treatment (71%). A total 73% of students reported that the asynchronous online-based activity was extremely effective in increasing knowledge of the roles and responsibilities of other disciplines and providing opportunities to interact with students from other health professions. Interprofessional education-trained students from multiple health-care disciplines feel comfortable performing SBIRT to identify persons at risk for substance misuse in practice.
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