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Lessons Learned from Approval of Aducanumab for Alzheimer's Disease Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Judith L. Heidebrink, Henry L. Paulson
When the US Food and Drug Administration used the accelerated approval process to authorize the use of the antiamyloid drug aducanumab to treat Alzheimer's disease (AD), many people hoped this signaled a new era of disease-modifying treatment. But 2 years later, aducanumab's failure to launch provides a cautionary tale about the complexities of dementia and the need for a thorough and transparent review
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Complex Congenital Heart Disease in the Adult Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Sarah A. Goldstein, Richard A. Krasuski
Congenital heart disease (CHD), a heterogeneous group of structural abnormalities of the cardiovascular system, is the most frequent cause of severe birth defects. Related to improved pediatric outcomes, there are now more adults living with CHD, including complex lesions, than children. Adults with CHD are at high risk for complications related to their underlying anatomy and past surgical palliative
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Spectrum of Diabetic Neuropathy: New Insights in Diagnosis and Treatment Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Brendan R. Dillon, Lynn Ang, Rodica Pop-Busui
Diabetic neuropathy is a highly prevalent complication of diabetes. It consists of a broad range of neuropathic conditions, such as distal symmetric polyneuropathy and various forms of autonomic neuropathies involving the cardiovascular, gastrointestinal, and urogenital systems. Prevention or diagnosis in early stages of disease is crucial to prevent symptomatic onset and progression, particularly
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Prostate-Specific Membrane Antigen: Gateway to Management of Advanced Prostate Cancer Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Lena M. Unterrainer, Jeremie Calais, Neil H. Bander
Prostate-specific membrane antigen (PSMA) as a transmembrane protein is overexpressed by prostate cancer (PC) cells and is accessible for binding antibodies or low-molecular-weight radioligands due to its extracellular portion. Successful targeting of PSMA began with the development of humanized J591 antibody. Due to their faster clearance compared to antibodies, small-molecule radioligands for targeted
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Emerging Technologies in Cardiac Pacing Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Ramya Vajapey, Mina K. Chung
Cardiac pacing to treat bradyarrhythmias has evolved in recent decades. Recognition that a substantial proportion of pacemaker-dependent patients can develop heart failure due to electrical and mechanical dyssynchrony from traditional right ventricular apical pacing has led to development of more physiologic pacing methods that better mimic normal cardiac conduction and provide synchronized ventricular
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Novel Therapeutic and Program-Based Approaches to Opioid Use Disorders Annu. Rev. Med. (IF 10.5) Pub Date : 2024-01-29 Patricia Liu, P. Todd Korthuis, Bradley M. Buchheit
Opioid use disorder continues to drive overdose deaths in many countries, including the United States. Illicit fentanyl and its analogues have emerged as key contributors to the complications and mortality associated with opioid use disorder. Medications for opioid use disorder treatment, such as methadone and buprenorphine, are safe and substantially reduce opioid use, infectious complications, and
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Home-Based Dialysis: A Primer for the Internist Annu. Rev. Med. (IF 10.5) Pub Date : 2023-12-01 Robert Rope, Eric Ryan, Eric D. Weinhandl, Graham E. Abra
Home-based dialysis modalities offer both clinical and practical advantages to patients. The use of the home-based modalities, peritoneal dialysis and home hemodialysis, has been increasing over the past decade after a long period of decline. Given the increasing frequency of use of these types of dialysis, it is important for clinicians to be familiar with how these types of dialysis are performed
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More Tailored Approaches to Tuberculosis Treatment and Prevention Annu. Rev. Med. (IF 10.5) Pub Date : 2023-11-20 Charles M. Bark, W. Henry Boom, Jennifer J. Furin
Recent advances in the treatment of tuberculosis (TB) have led to improvements unprecedented in our lifetime. Decades of research in developing new drugs, especially for multidrug-resistant TB, have created not only multiple new antituberculous agents but also a new approach to development and treatment, with a focus on maximizing the benefit to the individual patient. Prevention of TB disease has
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Wearable Devices: Implications for Precision Medicine and the Future of Health Care Annu. Rev. Med. (IF 10.5) Pub Date : 2023-11-20 Mohan Babu, Ziv Lautman, Xiangping Lin, Milan H.B. Sobota, Michael P. Snyder
Wearable devices are integrated analytical units equipped with sensitive physical, chemical, and biological sensors capable of noninvasive and continuous monitoring of vital physiological parameters. Recent advances in disciplines including electronics, computation, and material science have resulted in affordable and highly sensitive wearable devices that are routinely used for tracking and managing
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Precision Approaches to Chronic Obstructive Pulmonary Disease Management Annu. Rev. Med. (IF 10.5) Pub Date : 2023-10-12 Matthew Moll, Edwin K. Silverman
Chronic obstructive pulmonary disease (COPD) is a leading cause of morbidity and mortality worldwide. COPD heterogeneity has hampered progress in developing pharmacotherapies that affect disease progression. This issue can be addressed by precision medicine approaches, which focus on understanding an individual's disease risk, and tailoring management based on pathobiology, environmental exposures
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Interventional Pulmonology: Extending the Breadth of Thoracic Care Annu. Rev. Med. (IF 10.5) Pub Date : 2023-10-12 Yaron B. Gesthalter, Colleen L. Channick
Interventional pulmonary medicine has developed as a subspecialty focused on the management of patients with complex thoracic disease. Leveraging minimally invasive techniques, interventional pulmonologists diagnose and treat pathologies that previously required more invasive options such as surgery. By mitigating procedural risk, interventional pulmonologists have extended the reach of care to a wider
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The Hospitalist Movement 25 Years Later Annu. Rev. Med. (IF 10.5) Pub Date : 2023-10-06 Shradha A. Kulkarni, Robert M. Wachter
Hospitalists are generalists who specialize in the care of hospitalized patients. In the 25 years since the term hospitalist was coined, the field of hospital medicine has grown exponentially and established a substantial footprint in the medical community. There are now more hospitalists than practicing physicians in any other internal medicine subspecialty. Several key forces catalyzed the growth
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Genetics of Dilated Cardiomyopathy Annu. Rev. Med. (IF 10.5) Pub Date : 2023-10-03 Ramone Eldemire, Luisa Mestroni, Matthew R.G. Taylor
Dilated cardiomyopathy (DCM) is defined as dilation and/or reduced function of one or both ventricles and remains a common disease worldwide. An estimated 40% of cases of familial DCM have an identifiable genetic cause. Accordingly, there is a fast-growing interest in the field of molecular genetics as it pertains to DCM. Many gene mutations have been identified that contribute to phenotypically significant
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Mpox: The Reemergence of an Old Disease and Inequities Annu. Rev. Med. (IF 10.5) Pub Date : 2023-10-03 J.P. Thornhill, M. Gandhi, C. Orkin
Mpox, previously known as monkeypox, is caused by an Orthopoxvirus related to the variola virus that causes smallpox. Prior to 2022, mpox was considered a zoonotic disease endemic to central and west Africa. Since May 2022, more than 86,000 cases of mpox from 110 countries have been identified across the world, predominantly in men who have sex with men, most often acquired through close physical contact
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Leveraging TROP2 Antibody-Drug Conjugates in Solid Tumors Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-27 Blessie Elizabeth Nelson, Funda Meric-Bernstam
Antibody-drug conjugates (ADCs) have become the cornerstone of effective therapeutics in solid and hematological malignancies by harnessing potent cytotoxic payloads with targeted tumoricidal delivery. Since the monumental shift occurred with HER2-targeted ADCs, the discovery of the TROP2 antigen has revolutionized the landscape of ADC development. Moving beyond the traditional ADC design, multiple
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MELD 3.0 in Advanced Chronic Liver Disease Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-26 Nikhilesh R. Mazumder, Robert J. Fontana
The MELD (model for end-stage liver disease) 3.0 score was developed to replace the MELD-Na score that is currently used to prioritize liver allocation for cirrhotic patients awaiting liver transplantation in the United States. The MELD 3.0 calculator includes new inputs from patient sex and serum albumin levels and has new weights for serum sodium, bilirubin, international normalized ratio, and creatinine
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Health Effects of Wildfire Smoke Exposure Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-22 Carlos F. Gould, Sam Heft-Neal, Mary Johnson, Juan Aguilera, Marshall Burke, Kari Nadeau
We review current knowledge on the trends and drivers of global wildfire activity, advances in the measurement of wildfire smoke exposure, and evidence on the health effects of this exposure. We describe methodological issues in estimating the causal effects of wildfire smoke exposures on health and quantify their importance, emphasizing the role of nonlinear and lagged effects. We conduct a systematic
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Management of Resistant Hypertension Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-22 Lucas Lauder, Felix Mahfoud, Michael Böhm
Resistant hypertension (RH) is a severe form of hypertension associated with increased cardiovascular risk. Although true RH affects less than 10% of the patients receiving antihypertensive therapy, the absolute number is high and continues to increase. The workup of these patients requires screening for secondary hypertension and pseudoresistance, including poor adherence to prescribed medicines and
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Emerging Therapies in Hypothyroidism Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-22 Antonio C. Bianco
Levothyroxine (LT4) is effective for most patients with hypothyroidism. However, a minority of the patients remain symptomatic despite the normalization of serum thyrotropin levels. Randomized clinical trials including all types of patients with hypothyroidism revealed that combination levothyroxine and liothyronine (LT4+LT3) therapy is safe and is the preferred choice of patients versus LT4 alone
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Ketamine: Mechanisms and Relevance to Treatment of Depression Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-20 Ji-Woon Kim, Kanzo Suzuki, Ege T. Kavalali, Lisa M. Monteggia
Major depressive disorder (MDD) is a leading cause of suicide in the world. Monoamine-based antidepressant drugs are a primary line of treatment for this mental disorder, although the delayed response and incomplete efficacy in some patients highlight the need for improved therapeutic approaches. Over the past two decades, ketamine has shown rapid onset with sustained (up to several days) antidepressant
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Multi-Cancer Early Detection: The New Frontier in Cancer Early Detection Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-20 Carmen E. Guerra, Prateek V. Sharma, Brenda S. Castillo
The new generation of cancer early detection tests holds remarkable promise for revolutionizing and changing the paradigm of cancer early detection. Dozens of cancer early detection tests are being developed and evaluated. Some are already commercialized and available for use, most as a complement to and not in place of existing recommended cancer screening tests. This review evaluates existing single-
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The Evolving Practice of Hospital at Home in the United States Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-20 Tuyet-Trinh Truong, Albert L. Siu
Hospital at Home (HaH) provides hospital-level services in the home to eligible patients who would otherwise require facility-based hospitalization. In the last two decades, studies have shown that HaH can improve patient outcomes and satisfaction and reduce hospital readmissions. Improved technology and greater experience with the model have led to expansion in the scope of patients served and services
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New Biological Therapies for Multiple Myeloma Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-20 Alfred L. Garfall
Multiple myeloma is a cancer of bone marrow plasma cells that represents approximately 10% of hematologic malignancies. Though it is typically incurable, a remarkable suite of new therapies developed over the last 25 years has enabled durable disease control in most patients. This article briefly introduces the clinical features of multiple myeloma and aspects of multiple myeloma biology that modern
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Polypharmacy and Deprescribing in Older Adults Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-20 Wade Thompson, Emily G. McDonald
Older adults commonly end up on many medications. Deprescribing is an important part of individualizing care for older adults. It is an opportunity to discuss treatment options and revisit medications that may not have been reassessed in many years. A large evidence base exists in the field, suggesting that deprescribing is feasible and safe, though questions remain about the potential clinical benefits
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High-Sensitivity Cardiac Troponin Assays: Ready for Prime Time! Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-19 Konstantin A. Krychtiuk, L. Kristin Newby
Rapid and accurate triage of patients presenting with chest pain to an emergency department (ED) is critical to prevent ED overcrowding and unnecessary resource use in individuals at low risk of acute myocardial infarction (AMI) and to efficiently and effectively guide patients at high risk to definite therapy. The use of biomarkers for rule-out or rule-in of suspected AMI has evolved substantially
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Treatments for COVID-19 Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-19 Hayden S. Andrews, Jonathan D. Herman, Rajesh T. Gandhi
The treatment for COVID-19 has evolved rapidly since the start of the pandemic and now consists mainly of antiviral and immunomodulatory agents. Antivirals, such as remdesivir and nirmatrelvir-ritonavir, have proved to be most useful earlier in illness (e.g., as outpatient therapy) and for less severe disease. Immunomodulatory therapies, such as dexamethasone and interleukin-6 or Janus kinase inhibitors
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New Therapeutic Approaches to Large-Vessel Vasculitis Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-08 Mahmut S. Kaymakci, Kenneth J. Warrington, Tanaz A. Kermani
Giant cell arteritis (GCA) and Takayasu arteritis (TAK) are large-vessel vasculitides affecting the aorta and its branches. Arterial damage from these diseases may result in ischemic complications, aneurysms, and dissections. Despite their similarities, the management of GCA and TAK differs. Glucocorticoids are used frequently but relapses are common, and glucocorticoid toxicity contributes to significant
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Expansion of Anticomplement Therapy Indications from Rare Genetic Disorders to Common Kidney Diseases Annu. Rev. Med. (IF 10.5) Pub Date : 2023-09-05 Takashi Miwa, Sayaka Sato, Madhu Golla, Wen-Chao Song
Complement constitutes a major part of the innate immune system. The study of complement in human health has historically focused on infection risks associated with complement protein deficiencies; however, recent interest in the field has focused on overactivation of complement as a cause of immune injury and the development of anticomplement therapies to treat human diseases. The kidneys are particularly
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Effects on Cancer Prevention from the COVID-19 Pandemic Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-25 Stephen W. Duffy, Lucie de Jonge, Thomas E. Duffy
The COVID-19 pandemic led to disruption of health services around the world, including cancer services. We carried out a narrative review of the effect of the pandemic on cancer prevention services, including screening. Services were severely affected in the early months of the pandemic, and in some areas are still recovering. Large numbers of additional cancers or additional late-stage cancers have
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Adverse Impact of Cannabis on Human Health Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-15 Mark Chandy, Masataka Nishiga, Tzu-Tang Wei, Naomi M. Hamburg, Kari Nadeau, Joseph C. Wu
Cannabis, the most commonly used recreational drug, is illicit in many areas of the world. With increasing decriminalization and legalization, cannabis use is increasing in the United States and other countries. The adverse effects of cannabis are unclear because its status as a Schedule 1 drug in the United States restricts research. Despite a paucity of data, cannabis is commonly perceived as a benign
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Carbon Monoxide Poisoning: From Microbes to Therapeutics Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-15 Matthew R. Dent, Jason J. Rose, Jesús Tejero, Mark T. Gladwin
Carbon monoxide (CO) poisoning leads to 50,000–100,000 emergency room visits and 1,500–2,000 deaths each year in the United States alone. Even with treatment, survivors often suffer from long-term cardiac and neurocognitive deficits, highlighting a clear unmet medical need for novel therapeutic strategies that reduce morbidity and mortality associated with CO poisoning. This review examines the prevalence
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Novel Approaches to Immunomodulation for Solid Organ Transplantation Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-10 Irma Husain, Xunrong Luo
Despite significant advances in the field of transplantation in the past two decades, current clinically available therapeutic options for immunomodulation remain fairly limited. The advent of calcineurin inhibitor–based immunosuppression has led to significant success in improving short-term graft survival; however, improvements in long-term graft survival have stalled. Solid organ transplantation
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Novel Antigens and Clinical Updates in Membranous Nephropathy Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-08 Rupali Avasare, Nicole Andeen, Laurence Beck
Membranous nephropathy (MN), an autoimmune kidney disease and leading cause of nephrotic syndrome, leads to kidney failure in up to one-third of affected individuals. Most MN cases are due to an autoimmune reaction against the phospholipase A2 receptor (PLA2R) located on kidney podocytes. Serum PLA2R antibody quantification is now part of routine clinical practice because antibody titers correlate
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Update on the Porphyrias Annu. Rev. Med. (IF 10.5) Pub Date : 2023-08-04 Amy K. Dickey, Rebecca Karp Leaf, Manisha Balwani
The porphyrias are a group of rare diseases, each resulting from a defect in a different enzymatic step of the heme biosynthetic pathway. They can be broadly divided into two categories, hepatic and erythropoietic porphyrias, depending on the primary site of accumulation of heme intermediates. These disorders are multisystemic with variable symptoms that can be encountered by physicians in any specialty
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Chronic Neuromuscular Respiratory Failure and Home Assisted Ventilation Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Hugo Carmona, Andrew D. Graustein, Joshua O. Benditt
Chronic respiratory failure is a common, important complication of many types of neuromuscular and chest wall disorders. While the pathophysiology of each disease may be different, these disorders can variably affect all muscles involved in breathing, including inspiratory, expiratory, and bulbar muscles, ultimately leading to chronic respiratory failure and hypoventilation. The use of home assisted
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Exome/Genome Sequencing in Undiagnosed Syndromes Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Jennifer A. Sullivan, Kelly Schoch, Rebecca C. Spillmann, Vandana Shashi
Exome sequencing (ES) and genome sequencing (GS) have radically transformed the diagnostic approach to undiagnosed rare/ultrarare Mendelian diseases. Next-generation sequencing (NGS), the technology integral for ES, GS, and most large (100+) gene panels, has enabled previously unimaginable diagnoses, changes in medical management, new treatments, and accurate reproductive risk assessments for patients
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Use of Race in Kidney Function Estimation: Lessons Learned and the Path Toward Health Justice Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Dinushika Mohottige, Opeyemi Olabisi, L. Ebony Boulware
In 2020, the nephrology community formally interrogated long-standing race-based clinical algorithms used in the field, including the kidney function estimation equations. A comprehensive understanding of the history of kidney function estimation and racial essentialism is necessary to understand underpinnings of the incorporation of a Black race coefficient into prior equations. We provide a review
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Maternal Mortality in the United States: Trends and Opportunities for Prevention Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Siwen Wang, Kathryn M. Rexrode, Andrea A. Florio, Janet W. Rich-Edwards, Jorge E. Chavarro
Maternal mortality is unusually high in the United States compared to other wealthy nations and is characterized by major disparities in race/ethnicity, geography, and socioeconomic factors. Similar to other developed nations, the United States has seen a shift in the underlying causes of pregnancy-related death, with a relative increase in mortality resulting from diseases of the cardiovascular system
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New Frontiers in Obesity Treatment: GLP-1 and Nascent Nutrient-Stimulated Hormone-Based Therapeutics Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Ania M. Jastreboff, Robert F. Kushner
Nearly half of Americans are projected to have obesity by 2030, underscoring the pressing need for effective treatments. Glucagon-like peptide 1 receptor agonists (GLP-1 RAs) represent the first agents in a rapidly evolving, highly promising landscape of nascent hormone-based obesity therapeutics. With the understanding of the neurobiology of obesity rapidly expanding, these emerging entero-endocrine
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SGLT2 Inhibitors: The Sweet Success for Kidneys Annu. Rev. Med. (IF 10.5) Pub Date : 2023-01-27 Atit Dharia, Abid Khan, Vikas S. Sridhar, David Z.I. Cherney
Sodium-glucose cotransporter-2 inhibitors (SGLT2 inhibitors) were originally developed as antidiabetic agents, with cardiovascular (CV) outcome trials demonstrating improved CV outcomes in patients with type 2 diabetes mellitus (T2D). Secondary analyses of CV outcome trials and later dedicated kidney outcome trials consistently reported improved kidney-related outcomes independent of T2D status and
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Biological Phenotyping in Sepsis and Acute Respiratory Distress Syndrome Annu. Rev. Med. (IF 10.5) Pub Date : 2022-12-05 Pratik Sinha, Nuala J. Meyer, Carolyn S. Calfee
Heterogeneity in sepsis and acute respiratory distress syndrome (ARDS) is increasingly being recognized as one of the principal barriers to finding efficacious targeted therapies. The advent of multiple high-throughput biological data (“omics”), coupled with the widespread access to increased computational power, has led to the emergence of phenotyping in critical care. Phenotyping aims to use a multitude
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Club Cell Secretory Protein in Lung Disease: Emerging Concepts and Potential Therapeutics Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-30 Tereza Martinu, Jamie L. Todd, Andrew E. Gelman, Stefano Guerra, Scott M. Palmer
Club cell secretory protein (CCSP), also known as secretoglobin 1A1 (gene name SCGB1A1), is one of the most abundant proteins in the lung, primarily produced by club cells of the distal airway epithelium. At baseline, CCSP is found in large concentrations in lung fluid specimens and can also be detected in the blood and urine. Obstructive lung diseases are generally associated with reduced CCSP levels
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Clonal Hematopoiesis and Its Impact on Human Health Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-30 Herra Ahmad, Nikolaus Jahn, Siddhartha Jaiswal
Aging is associated with increased mutational burden in every tissue studied. Occasionally, fitness-increasing mutations will arise, leading to stem cell clonal expansion. This process occurs in several tissues but has been best studied in blood. Clonal hematopoiesis is associated with an increased risk of blood cancers, such as acute myeloid leukemia, which result if additional cooperating mutations
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Valvular Heart Disease: New Concepts in Pathophysiology and Therapeutic Approaches Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-19 Mackram F. Eleid, Vuyisile T. Nkomo, Sorin V. Pislaru, Bernard J. Gersh
This review discusses recent advancements in the field of valvular heart disease. Topics covered include recognition of the impact of atrial fibrillation on development and assessment of valvular disease, strategies for global prevention of rheumatic heart disease, understanding and management of secondary mitral regurgitation, the updated classification of bicuspid aortic valve disease, recognition
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All the Tau We Cannot See Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-15 Bradley Hyman
Alzheimer's disease (AD) was described in 1906 as a dementing disease marked by the presence of two types of fibrillar aggregates in the brain: neurofibrillary tangles and senile plaques. The process of aggregation and formation of the aggregates has been a major focus of investigation ever since the discoveries that the tau protein is the predominant protein in tangles and amyloid β is the predominant
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Primary Aldosteronism and the Role of Mineralocorticoid Receptor Antagonists for the Heart and Kidneys Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-14 Jordana B. Cohen, Irina Bancos, Jenifer M. Brown, Harini Sarathy, Adina F. Turcu, Debbie L. Cohen
Primary aldosteronism (PA) is the most common cause of secondary hypertension but is frequently underrecognized and undertreated. Patients with PA are at a markedly increased risk for target organ damage to the heart and kidneys. While patients with unilateral PA can be treated surgically, many patients with PA are not eligible or willing to undergo surgery. Steroidal mineralocorticoid receptor antagonists
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Genetics of Kidney Disease: The Unexpected Role of Rare Disorders Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-14 Mark D. Elliott, Hila Milo Rasouly, Ali G. Gharavi
Hundreds of different genetic causes of chronic kidney disease are now recognized, and while individually rare, taken together they are significant contributors to both adult and pediatric diseases. Traditional genetics approaches relied heavily on the identification of large families with multiple affected members and have been fundamental to the identification of genetic kidney diseases. With the
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Cytomegalovirus Therapy: Role of Letermovir in Prophylaxis and Treatment in Transplant Recipients Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-04 Jennifer L. Saullo, Rachel A. Miller
Cytomegalovirus (CMV) is a common viral pathogen in the transplant population and is associated with significant morbidity and mortality. CMV prevention is paramount; however, selecting the best preventive strategy depends on many factors including donor–recipient CMV serostatus, transplant-specific risks, antiviral toxicities and cost. Novel CMV therapeutics such as letermovir (LTV) are desperately
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Multispecific CAR T Cells Deprive Lymphomas of Escape via Antigen Loss Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-04 Fateeha Furqan, Nirav N. Shah
Chimeric antigen receptor (CAR) modified T cell therapy has transformed the management of relapsed/refractory B cell malignancies. Despite high overall response rates, relapse post CAR T treatment remains a clinical challenge. Loss of target antigen, specifically CD19, is one well-defined mechanism of disease relapse. The mechanism of CD19 loss and which patients are at higher risk of CD19 loss remain
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Update in Adult Transgender Medicine Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-02 Alyxandra Ramsay, Joshua D. Safer
Transgender people often face barriers in health care due to lack of access to care, lack of knowledgeable healthcare professionals, discrimination, and gaps in medical and mental health research. Existing research on transgender health has focused heavily on mental health, HIV/AIDS, sexually transmitted diseases/infections, and substance abuse. Gender-affirming hormone therapy and/or surgery allows
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Lessons Learned from the ISCHEMIA Trial for the Management of Patients with Stable Ischemic Heart Disease Annu. Rev. Med. (IF 10.5) Pub Date : 2022-11-01 William E. Boden, Peter H. Stone
The recent landmark International Study of Comparative Health Effectiveness With Medical and Invasive Approaches (ISCHEMIA) trial was undertaken to assess whether stable angina patients with moderate to severe baseline ischemia would benefit from an invasive approach with revascularization versus a conservative approach of intensive lifestyle intervention and pharmacologic secondary prevention. This
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Advances and Applications of Polygenic Scores for Coronary Artery Disease Annu. Rev. Med. (IF 10.5) Pub Date : 2022-10-31 Aniruddh P. Patel, Amit V. Khera
Polygenic scores quantify inherited risk by integrating information from many common sites of DNA variation into a single number. Rapid increases in the scale of genetic association studies and new statistical algorithms have enabled development of polygenic scores that meaningfully measure—as early as birth—risk of coronary artery disease. These newer-generation polygenic scores identify up to 8%
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Gender-Affirming Care of Transgender and Gender-Diverse Youth: Current Concepts Annu. Rev. Med. (IF 10.5) Pub Date : 2022-10-19 Janet Y. Lee, Stephen M. Rosenthal
Increasing numbers of transgender and gender-diverse (TGD) youth, from early puberty through late adolescence, are seeking medical services to bring their physical sex characteristics into alignment with their gender identity—their inner sense of self as male or female or elsewhere on the gender spectrum. Numerous studies, primarily of short- and medium-term duration (up to 6 years), demonstrate the
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Cytokine Storm Syndrome Annu. Rev. Med. (IF 10.5) Pub Date : 2022-10-13 Randy Q. Cron, Gaurav Goyal, W. Winn Chatham
Cytokine storm syndrome (CSS), which is frequently fatal, has garnered increased attention with the ongoing coronavirus pandemic. A variety of hyperinflammatory conditions associated with multiorgan system failure can be lumped under the CSS umbrella, including familial hemophagocytic lymphohistiocytosis (HLH) and secondary HLH associated with infections, hematologic malignancies, and autoimmune and
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Myocardial Infarction with Nonobstructive Coronary Arteries Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-30 H.R. Reynolds, N.R. Smilowitz
Myocardial infarction with nonobstructive coronary arteries (MINOCA) is an important subtype of myocardial infarction (MI) that occurs in approximately 6–8% of patients with spontaneous MI who are referred for coronary angiography. MINOCA disproportionately affects women, but men are also affected. Pathogenesis is more variable than in MI with obstructive coronary artery disease (MI-CAD). Dominant
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FGFR2 Inhibition in Cholangiocarcinoma Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-28 Arndt Vogel, Oreste Segatto, Albrecht Stenzinger, Anna Saborowski
Biliary tract cancer (BTC) is the second most common primary liver cancer after hepatocellular carcinoma and accounts for 2% of cancer-related deaths. BTCs are classified according to their anatomical origin into intrahepatic (iCCA), perihilar, or distal cholangiocarcinoma, as well as gall bladder carcinoma. While the mutational profiles in these anatomical BTC subtypes overlap to a large extent, iCCA
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Endocrine Disorders and COVID-19 Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-24 Seda Hanife Oguz, Bulent Okan Yildiz
The multifaceted interaction between coronavirus disease 2019 (COVID-19) and the endocrine system has been a major area of scientific research over the past two years. While common endocrine/metabolic disorders such as obesity and diabetes have been recognized among significant risk factors for COVID-19 severity, several endocrine organs were identified to be targeted by severe acute respiratory syndrome
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COVID-19 Thrombotic Complications and Therapeutic Strategies Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-21 Alexander C. Fanaroff, Renato D. Lopes
Shortly after the emergence of coronavirus disease 2019 (COVID-19) in late 2019, clinicians rapidly recognized an apparent association between the disease and both arterial and venous thrombotic complications, which was confirmed in epidemiologic studies. Based on these data, hospitals empirically developed and implemented protocols with different strategies for anticoagulation of hospitalized COVID-19
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COVID-19 and Kidney Disease Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-15 Maureen Brogan, Michael J. Ross
COVID-19 can cause acute kidney injury and may cause or exacerbate chronic kidney diseases, including glomerular diseases. SARS-CoV-2 infection of kidney cells has been reported, but it remains unclear if viral infection of kidney cells causes disease. The most important causes of kidney injury in patients with COVID-19 include impaired renal perfusion and immune dysregulation. Chronic kidney disease
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Adeno-Associated Virus Gene Therapy for Hemophilia Annu. Rev. Med. (IF 10.5) Pub Date : 2022-09-14 Benjamin J. Samelson-Jones, Lindsey A. George
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy. This effort is soon to bear fruit with completed pivotal adeno-associated viral (AAV) vector gene addition trials reporting encouraging results and regulatory approval widely