British Journal of Haematology最新文献

Medical scams or grifting has long been a societal issue, though in recent years, the problem has become increasingly mainstream, especially as it relates to transfusion medicine, apheresis and biotherapies. Unfortunately, unsubstantiated medical claims, or unfounded interventions such as therapeutic plasma exchange for longevity or dangerous stem cell injections, are leading to significant medical waste and patient harm. Herein, we review contemporary medical grifts, cognitive biases and present a framework for managing these issues to ensure legitimate care and safety for patients.

Commentary on: Schönung et al. Assessment of peripheral blood DNA methylation signatures as pharmacodynamic and predictive biomarkers during azacytidine therapy in juvenile myelomonocytic leukemia: Results of the EWOG-MESRAT study. Br J Haematol 2025; 207:1271–1278.

Most adults with sickle cell disease (SCD) attend the emergency department (ED), with significant interindividual and temporal variability. To identify the patterns of ED use, we analysed data from adults with SCD followed at a French reference centre who had ≥1 ED visit between 1 October 2013 and 31 December 2019. We used a survival model to estimate the cumulative risk of ED visits and its variability over time and applied clustering methods on these two dimensions to identify typical patterns of ED utilization. Among 656 adults (9080 ED visits), two clusters emerged: a low-use group with 529 individuals (81%) and 2924 ED visits (32%) and a high-use group with 127 individuals (19%) and 6156 ED visits (68%). All high-use group members experienced ED visits in bursts, defined as at least three visits within 3 months and at least three times more than during the previous 3 months. Bursts were uncommon in the low-use group (5% of individuals). Among individuals experiencing bursts, two ED visits less than a month apart increased the risk of another ED visit within the next month (adjusted HR 3.51 [95% CI 2.95-4.16]). Understanding factors triggering bursts of ED visits in adults with SCD may enable targeted interventions.

Despite therapeutic advances and improved survival, the long-term economic burden of chronic myeloid leukaemia (CML) remains under-recognised given evolving treatment practices and fluctuating drug costs. We aimed to estimate and project total prevalence costs for CML in Sweden from a healthcare sector perspective, representing the direct healthcare expenditures for all patients living with CML, based on real-world drug and procedure prices. We used data from the Swedish Cancer Register and the Swedish CML register to estimate and project prevalence and costs. The estimated numbers of prevalent cases were 1808 (95% confidence interval [CI], 1604–2011) in 2025 and 2120 (95% CI, 1916–2325) in 2030, driven by stable incidence and improved survival. Despite increasing prevalence, the annual total direct healthcare costs for all prevalent CML patients in Sweden decreased from USD 40.04 million (95% CI, 33.70–46.40) in 2015 to USD 26.04 million (95% CI, 23.13–28.97) in 2025, then projected to slightly increase to USD 30.67 million (95% CI, 27.70–33.64) in 2030. While CML prevalence proportions are expected to increase, declining treatment costs may mitigate the burden on the Swedish healthcare system. These population-based projections can inform long-term planning and pricing strategies for CML care.

Disulfidptosis is a newly recognized cell death induced by disulphide stress under glucose deprivation. However, its clinical implications in multiple myeloma (MM) remain largely unexplored. We identified disulfidptosis-related genes via co-expression analysis and developed a risk signature through univariate Cox, least absolute shrinkage and selection operator (LASSO) regression and multivariate Cox regression analyses. The model was internally and externally validated for overall survival prediction. Downstream analyses included functional enrichment, tumour mutation burden (TMB), tumour microenvironment (TME) characterization and immune-metabolic profiling. Preliminary in vitro validation was conducted with PLEC-knockout KMS-11 cells under glucose deprivation. A nine-gene signature was established and validated, showing robust prognostic performance across cohorts. High-risk patients displayed proliferative profiles, higher TMB, an immunosuppressive TME with neutrophils, eosinophils and exhausted T cells and activation of glycolysis and cystine/glutathione metabolism. This signature defines an aggressive MM subtype likely unresponsive to immune checkpoint therapy. Functional assays confirmed that PLEC mediates actin disulphide cross-linking during metabolic stress. We present a novel disulfidptosis-related gene signature with independent prognostic value in MM, offering insights into the molecular and immune landscape of MM and serving as a useful tool for risk stratification and therapeutic suggestion.