Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study.

In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.

Fibrinolytic activity in infants undergoing cardiac surgery on cardiopulmonary bypass with routine tranexamic acid: A prospective cohort substudy within the FIBrinogen CONcentrate randomised control trial.

Background: Fibrinolytic activity contributes to bleeding after cardiopulmonary bypass (CPB).

Objective: Our objectives were, in a group of infants undergoing cardiac surgery with CPB: to document the extent of peri-operative fibrinolysis using rotational thromboelastometry (ROTEM) and standard biomarkers; to compare the agreement between these fibrinolytic measures; to assess whether fibrinolytic activity is associated with early postoperative mediastinal bleeding and assess whether supplementation with fibrinogen concentrate affected fibrinolysis.

Design: Prospective cohort, mechanistic substudy, nested within the FIBrinogen CONcentrate (FIBCON) randomised controlled trial.

EGLN1 mutations in Cis can induce congenital erythrocytosis with thromboses by increasing protein instability.

Hereditary congenital erythrocytosis results from constitutive activation of the hypoxia pathway. This pathway is controlled by regulation of the α isoforms of the hypoxia-inducible factor α/β heterodimer, notably via hydroxylation by prolyl hydroxylase domain 2 (PHD2). Mutations affecting PHD2 are involved in Type 3 erythrocytosis.

Monoclonal gammopathy of undetermined significance and the risk of thrombotic events: Results from iStopMM, a prospective population-based screening study.

Monoclonal gammopathy of undetermined significance (MGUS) is the asymptomatic precursor of multiple myeloma and related diseases but has also been associated with thrombosis. Prior studies have not been based on screened cohorts leading to bias. We assessed the risk of thrombosis in a cohort of 75 422 individuals over 40 years old who were screened for MGUS in Iceland.

Development and validation of a deep learning model for morphological assessment of myeloproliferative neoplasms using clinical data and digital pathology.

The subjectivity of morphological assessment and the overlapping pathological features of different subtypes of myeloproliferative neoplasms (MPNs) make accurate diagnosis challenging. To improve the pathological assessment of MPNs, we developed a diagnosis model (fusion model) based on the combination of bone marrow whole-slide images (deep learning [DL] model) and clinical parameters (clinical model). Thousand and fifty-one MPN and non-MPN patients were divided into the training, internal testing and one internal and two external validation cohorts (the combined validation cohort).

Association of human leucocyte antigen loci with vaccine-induced immune thrombotic thrombocytopenia: Potential role of the interaction between platelet factor 4-derived peptides and MHC-II.

No risk factors have been identified for vaccine-induced immune thrombotic thrombocytopenia (VITT) so far. The aim of this study was to identify human leucocyte antigen (HLA) alleles potentially associated with VITT susceptibility. Specific HLA class II alleles were detected with significantly higher frequency in VITT patients compared with Italian controls: DPB1*17:01, DQA1*05:01, and DRB1*11:04.

Standardized Data Elements for Patients With Acute Pulmonary Embolism: A Consensus Report From the Pulmonary Embolism Research Collaborative.

Recent advances in therapy and the promulgation of multidisciplinary pulmonary embolism teams show great promise to improve management and outcomes of acute pulmonary embolism (PE). However, the absence of randomized evidence and lack of consensus leads to tremendous variations in treatment and compromises the wide implementation of new innovations. Moreover, the changing landscape of health care, where quality, cost, and accountability are increasingly relevant, dictates that a broad spectrum of outcomes of care must be routinely monitored to fully capture the impact of modern PE treatment.

Exploring the role of Large Language Models in haematology: A focused review of applications, benefits and limitations.

Large language models (LLMs) have significantly impacted various fields with their ability to understand and generate human-like text. This study explores the potential benefits and limitations of integrating LLMs, such as ChatGPT, into haematology practices. Utilizing systematic review methodologies, we analysed studies published after 1 December 2022, from databases like PubMed, Web of Science and Scopus, and assessing each for bias with the QUADAS-2 tool.

Prevention and management of venous thrombosis in patients with cirrhosis.

Venous thromboembolism (VTE), particularly portal vein thrombosis, is common in patients with cirrhosis. Misconceptions around the increased bleeding risk in this patient group may lead to delayed and/or inadequate anticoagulation. This nutshell review focusses on the approach to management including the role of direct oral anticoagulants in the treatment of VTE in patients with cirrhosis.

A call for subspecialty training programmes in women's thrombosis and haemostasis.

Multidisciplinary collaboration to create formal education/training programmes in women's thrombosis and haemostasis will ideally lead to improved knowledge and health equity and potentially improve patient outcomes.

Vitamin B12 deficiency misdiagnosed as TTP: What can we learn from it?

The case report by Dwyre et al. shows that vitamin B12 deficiency may be misdiagnosed as acute thrombotic thrombocytopenic purpura. Together with similar observations, this underlines that acquired vitamin B12 deficiency-besides the inherited disorder of intracellular cobalamin metabolism, cbl C disease-should be listed as a separate entity of the thrombotic microangiopathies.

Revisiting the full blood count: Circulating blood cells and their role in coagulation.

There has been an expansion in our understanding of the multifaceted roles of circulating blood cells in regulating haemostasis and contributing to thrombosis. Notably, there is greater recognition of the interplay between coagulation with inflammation and innate immune activation and the contribution of leucocytes. The full blood count (FBC) is a time-honoured test in medicine; however, its components are often viewed in isolation and without consideration of their haemostatic and thrombotic potential.

Anti-platelet factor 4 immunothrombotic syndromes.

Anti-platelet factor 4 immunothrombotic syndromes comprise a group of disorders that include heparin-induced thrombocytopenia and vaccine-induced immune thrombocytopenia and thrombosis. These are highly prothrombotic, immunological disorders characterised by specific clinical and pathological criteria which include thrombocytopenia and thrombosis. While they are predominantly triggered by heparin and the adenoviral vector vaccines, respectively, other provoking factors have been described, as well as spontaneous forms.