-
Hepatology (Baltimore, Md.) Oct 2023Acute liver failure (ALF) describes a clinical syndrome of rapid hepatocyte injury leading to liver failure manifested by coagulopathy and encephalopathy in the absence... (Review)
Review
Acute liver failure (ALF) describes a clinical syndrome of rapid hepatocyte injury leading to liver failure manifested by coagulopathy and encephalopathy in the absence of pre-existing cirrhosis. The hallmark diagnostic features are a prolonged prothrombin time (ie, an international normalized ratio of prothrombin time of ≥1.5) and any degree of mental status alteration (HE). As a rare, orphan disease, it seemed an obvious target for a multicenter network. The Acute Liver Failure Study Group (ALFSG) began in 1997 to more thoroughly study and understand the causes, natural history, and management of ALF. Over the course of 22 years, 3364 adult patients were enrolled in the study registry (2614 ALF and 857 acute liver injury-international normalized ratio 2.0 but no encephalopathy-ALI) and >150,000 biosamples collected, including serum, plasma, urine, DNA, and liver tissue. Within the Registry study sites, 4 prospective substudies were conducted and published, 2 interventional ( N -acetylcysteine and ornithine phenylacetate), 1 prognostic [ 13 C-methacetin breath test (MBT)], and 1 mechanistic (rotational thromboelastometry). To review ALFSG's accomplishments and consider next steps, a 2-day in-person conference was held at UT Southwestern Medical Center, Dallas, TX, entitled "Acute Liver Failure: Science and Practice," in May 2022. To summarize the important findings in the field, this review highlights the current state of understanding of ALF and, more importantly, asks what further studies are needed to improve our understanding of the pathogenesis, natural history, and management of this unique and dramatic condition.
Topics: Adult; Humans; Prospective Studies; Liver Failure, Acute; Prognosis; Liver Transplantation; Multicenter Studies as Topic
PubMed: 37183883
DOI: 10.1097/HEP.0000000000000458 -
Current Opinion in Neurology Oct 2023The purpose of this review is to summarise the recent developments in trial readiness, natural history studies, and interventional clinical trials for Becker muscular... (Review)
Review
PURPOSE OF REVIEW
The purpose of this review is to summarise the recent developments in trial readiness, natural history studies, and interventional clinical trials for Becker muscular dystrophy (BMD).
RECENT FINDINGS
As several treatment concepts have claimed to convert patients with Duchenne muscular dystrophy (DMD) into a BMD phenotype, BMD itself has moved into the focus of clinical research. Natural history studies have helped to better characterize patients with BMD and the disease is now a target for interventional trials. In parallel, there have been advances in diagnostics and in the development of preclinical models.
SUMMARY
Despite increased collaborative efforts to improve trial readiness amongst patients with BMD, there is still a lack of long-term natural history data, and the broad spectrum of disease severity remains a challenge for well designed clinical trials.
Topics: Humans; Muscular Dystrophy, Duchenne; Phenotype; Research Design
PubMed: 37591308
DOI: 10.1097/WCO.0000000000001191 -
Clinical Medicine (London, England) Sep 2023Atrial fibrillation (AF) is the most common cardiac arrhythmia and imposes a significant healthcare burden. The landscape of AF has changed considerably over the past... (Review)
Review
Atrial fibrillation (AF) is the most common cardiac arrhythmia and imposes a significant healthcare burden. The landscape of AF has changed considerably over the past few years, with the advent of novel diagnostic approaches, advances in therapies and changing recommendations on best practice from the latest major trials. In this article, we review our evolving understanding of the natural history of AF and explore the contemporary landscape of its diagnosis and management.
Topics: Humans; Atrial Fibrillation; Catheter Ablation
PubMed: 37775166
DOI: 10.7861/clinmed.2023-23.5.Cardio2