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.