For a long time, deformities of the chest wall, such as pectus excavatum, a condition where the chest appears to have sunken, remained untreated or were treated using crude and invasive techniques. However, thanks to innovations led by surgeons such as Prof. Donald Nuss of Eastern Virginia Medical School, these procedures have undergone a remarkable transformation. Such work has shifted the paradigm from radical surgery to minimally invasive solutions, changing lives and restoring confidence for countless patients. Now, a Review Article published in the African Journal of Thoracic and Critical Care Medicine, and co-authored by Prof. Donald Nuss and Dr. Ivan Schewitz of the Department of Cardiothoracic Surgery at the University of Pretoria, South Africa, charts the remarkable progress in treating pectus excavatum.

Imagine living with an illness that can sap your energy levels so completely that even day-to-day tasks, such as doing laundry, walking the dog, or even getting out of bed can be insurmountable challenges. To make matters worse, this illness is not well understood either by the public or by medical staff, and is often dismissed and stigmatised, making it difficult to find understanding or treatment. This is the unfortunate lived experience of many people with myalgic encephalomyelitis/chronic fatigue syndrome (or ME/CFS for short). In a recent Communication article, researcher Caroline Kingdon of the London School of Hygiene and Tropical Medicine, and colleagues, discuss this misunderstood condition through the prism of the 2021 guideline for the treatment of those with ME/CFS, which have been published by the UK’s National Institute for Health and Care Excellence (or NICE for short). Their article aims to inform primary caregivers about the NICE guideline, and, happily, reveals that the new guideline prioritises an overdue shift toward compassionate and patient-focused care for ME/CFS.

Cholangiocarcinoma is an aggressive cancer that begins in the bile ducts. While the cancer is relatively rare, affecting approximately 8,000 people in the United States each year, unfortunately, it is often undiagnosed until an advanced stage. This late diagnosis makes cholangiocarcinoma very challenging to treat, and less than 10% of patients survive for five years after diagnosis. Traditional anti-cancer treatments, such as chemotherapy, have only limited effectiveness in cholangiocarcinoma, and can cause serious side effects. Recently developed immunotherapy and targeted therapies have provided promising options for this difficult-to-treat disease. Dr. Kim Saverno of the Incyte Corporation, a biopharmaceutical company headquartered in the U.S., and the study’s co-authors, have been studying the real-world use of an FDA-approved targeted drug for cholangiocarcinoma called pemigatinib. Pemigatinib was approved by the FDA in 2020. It can be specifically used for cholangiocarcinoma patients who have genetic changes in a protein known as fibroblast growth factor receptor 2, or FGFR2 for short. Their study, to the best of our knowledge, is the first to examine pemigatinib’s use in real-world settings, and reveals information about treatment patterns, FGFR2 testing patterns, and effectiveness of pemigatinib for cholangiocarcinoma when used in everyday practice.

Hypertrophic cardiomyopathy (or HCM for short) is a serious heart condition that involves thickening of the heart muscle wall and disruption of the normal tissue architecture, called ‘disarray’. This can result in sudden cardiac death caused by abnormal heart rhythms, known as arrhythmias. Identifying those HCM patients who are most at risk could permit preventative measures, such as implanting a cardioverter-defibrillator, which could potentially be lifesaving. However, current techniques to predict the risk of sudden death in HCM are limited, leaving patients underserved. In a recent study, Dr Richard Saumarez, an academic cardiologist formerly of the University of Cambridge, and colleagues, questioned whether conventional methods, which consider risk factors such as family history of sudden death or the degree of heart muscle thickening, are effective in predicting sudden death in HCM patients. Their research suggests that risk factor assessments might miss crucial information about the heart’s electrical behaviour, which could provide more accurate clues about the risk of sudden death. As an alternative, the researchers propose direct heart-investigation methods, called electrophysiological techniques, as a more reliable assessment. Although the study was concerned with HCM, the arguments put forward are more general and applicable to other diseases, particularly to survivors of myocardial infarcts who are also at risk of sudden death.

In their ongoing quest to improve diabetes management, researchers are searching for new insights into the mechanisms through which the body manages blood sugar levels. Prof. Eugenio Cersosimo and colleagues at the University of Texas Health Science Center recently reported a breakthrough that could change how we understand glucose control and increase our ability to manage type 2 diabetes. Their study examines two medications, dapagliflozin, an SGLT-2 inhibitor, and exenatide, a GLP-1 receptor agonist, and how they can work together to control blood sugar levels by exploiting a previously unknown kidney-to-liver signalling pathway. Their findings have unravelled some important underlying mechanisms that provide strong support for the cardio-renal protective effects reported in many large clinical trials with the use of SGLT-2 inhibitors in patients with type 2 diabetes. The demonstration that the kidney plays a central role in glucose regulation during exposure to SGLT-2 inhibitors represents a major advance in our understanding of diabetes treatment and the prevention of severe cardiovascular and renal complications.