Review
Hematology
Tania Jain, Timothy S. Olson, Frederick L. Locke
Summary: The increasing use of CAR-T therapy has led to the discovery of diverse toxicities, such as cytopenias, which require specific recognition and management. These cytopenias usually occur early after CAR-T infusion (<30 days), can be prolonged (30-90 days), and sometimes persist or happen late (>90 days). The causes of these cytopenias are variable and some are not fully understood, creating clinical challenges and uncertainties in choosing the best management strategies. These cytopenias can result in additional complications, reduced quality of life, and increased resource utilization.
Article
Hematology
Lucy B. Cook, Adrienne A. Phillips
Summary: ATL is an aggressive T-cell malignancy with limited survival outcomes with chemotherapy, but novel therapies like mogamulizumab show promise for different ATL subtypes. The implementation of molecular methods may guide diagnosis and treatment, but universal availability remains a challenge worldwide.
Review
Hematology
Fadi Fakhouri, Nora Schwotzer, Veronique Fremeaux-Bacchi
Summary: In the past decade, there has been a significant improvement in our understanding and management of atypical hemolytic uremic syndrome (aHUS). C5 blockade has been established as an efficacious treatment for this devastating disease. However, the positive diagnosis of aHUS remains challenging and the spectrum of complement-mediated renal thrombotic microangiopathy is still debated. Individualized long-term management for aHUS is now prioritized, and the potential benefit of complement blockade in other forms of HUS is uncertain.
Review
Hematology
Jennifer E. Amengual, Barbara Pro
Summary: Posttransplant lymphoproliferative disorder (PTLD) is a significant and potentially life-threatening complication of solid organ transplant and hematopoietic stem cell transplant (HSCT). Treatment of PTLD is challenging and includes methods such as immunosuppression reduction, immunotherapy, and chemotherapy. Novel treatments are needed for high-risk patients or those who do not respond to frontline therapies.
Review
Hematology
Alessandro Casini, Philippe de Moerloose
Summary: Congenital dysfibrinogenemia is a disorder caused by structural changes in fibrinogen, leading to bleeding tendencies and thrombosis. Most patients are asymptomatic at diagnosis and genetic testing is needed for confirmation. Treatment involves fibrinogen supplementation and the use of antifibrinolytic agents in certain clinical settings.
Review
Hematology
Jeffrey E. Rubnitz, Gertjan J. L. Kaspers
Summary: Treatment outcomes for pediatric patients with acute myeloid leukemia (AML) are behind those with acute lymphoblastic leukemia (ALL) due to disease heterogeneity, lack of targeted therapies, and slow development of immunotherapy. Further intensification of conventional chemotherapy is unlikely to reduce relapse rates, but comprehensive genomic analyses and understanding of leukemic stem cells may lead to more effective tailored therapies in the future. New therapies like venetoclax and CAR T-cell therapy offer hope for improved outcomes in pediatric AML treatment.
Article
Hematology
Ross Baker, James S. O'Donnell
Summary: Recent studies show an increasing frequency in the diagnosis of BDUC, with a need for consensus on clinical and laboratory criteria. Objective assessment of bleeding phenotype using BAT is crucial for the diagnosis of BDUC. Despite the high prevalence of BDUC, diagnosing and managing these patients present significant clinical dilemmas.
Review
Oncology
J. Taieb, M. Karoui, D. Basile
Summary: The key in treatment decision-making for stage II colon cancer lies in classifying tumors based on molecular and clinico-pathological features, then choosing appropriate treatment methods including surveillance, adjuvant therapy, and drug treatment. New tools in the future may help to more accurately identify patients for a more personalized treatment approach.
Article
Hematology
Giovanni Palladini, Giampaolo Merlini
Summary: The treatment of AL amyloidosis is a challenge for hematologists, but early diagnosis and personalized therapy can improve patient outcomes.
Review
Hematology
Brunangelo Falini, Lorenzo Brunetti, Maria Paola Martelli
Summary: Mutations of the nucleophosmin (NPM1) gene play a crucial role in adult acute myeloid leukemia (AML), with unique molecular, pathological, and clinical features. Accurate diagnosis and distinction of NPM1-mutated AML from other entities is important for guiding treatment decisions and assessing relapse risk. Monitoring measurable residual disease (MRD) using NPM1 mutations can provide valuable insights for therapeutic management after remission.
Review
Hematology
Brunangelo Falini, Luca De Carolis, Enrico Tiacci
Summary: Hairy cell leukemia responds well to frontline chemotherapy but becomes resistant to these drugs over time. In addition to traditional treatment options, targeting the mutant BRAF-V600E kinase and using combination therapy with other drugs is emerging as an attractive chemotherapy-free strategy. Other treatment options are also being explored in clinical trials.
Review
Hematology
Robert A. Brodsky
Summary: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare complement-mediated hemolytic anemia with diverse manifestations, requiring differentiated treatment approaches; terminal complement inhibition is effective for intravascular hemolysis treatment but not bone marrow failure; novel complement inhibitors under clinical development show promising prospects for future applications.
Article
Hematology
Efstathios Kastritis, Evangelos Terpos, Meletios A. Dimopoulos
Summary: Multiple myeloma is an incurable disease, and patients require long-term treatment. Despite the introduction of new therapies, drug resistance remains the most critical factor in choosing the appropriate treatment. Currently, three-drug regimens are the standard approach for managing relapsed disease.
Review
Hematology
Sigbjorn Berentsen
Summary: Significant progress has been made in the treatment of CAD in recent decades, with recommendations mainly based on nonrandomized trials and personal experience due to the lack of comparative trials. Individualized treatment options tailored to patients' symptoms and disease characteristics are crucial to avoid ineffective therapies.
Article
Medicine, General & Internal
Letizia Sabatini, Marcello Chinali, Alessio Franceschini, Margherita Di Mauro, Silvio Marchesani, Francesca Fini, Giorgia Arcuri, Mariachiara Lodi, Giuseppe Palumbo, Giulia Ceglie
Summary: Cardiovascular involvement is highly prevalent in pediatric sickle cell disease patients and is associated with specific laboratory findings and clinical phenotypes characterized by complications related to high hemodynamic load. This study found that left atrial dilatation and left ventricular hypertrophy were common cardiac abnormalities in sickle cell disease patients. Patients with left atrial dilatation had lower hemoglobin levels and higher reticulocyte counts, while left ventricular hypertrophy was negatively correlated with hemoglobin levels and positively correlated with reticulocyte counts. Additionally, patients with cardiac anomalies had higher transfusion needs and a lower frequency of pain crises.
JOURNAL OF CLINICAL MEDICINE
(2023)
Letter
Hematology
Nermi L. Parrow, Pierre-Christian Violet, Nisha Ajit George, Faris Ali, Shivam Bhanvadia, Ryan Wong, John F. Tisdale, Courtney Fitzhugh, Mark Levine, Swee Lay Thein, Robert E. Fleming
Article
Biochemistry & Molecular Biology
Sebastian Vogel, Sayuri Kamimura, Taruna Arora, Meghann L. Smith, Luis E. F. Almeida, Christian A. Combs, Swee Lay Thein, Zenaide M. N. Quezado
Summary: The NLRP3 inflammasome is upregulated in sickle cell disease and is dependent on BTK. Pharmacological inhibition of NLRP3 and BTK can decrease platelet aggregation and in vitro thrombus formation in SCD mice. Targeting the NLRP3 inflammasome may offer a novel approach for antiplatelet therapy in SCD.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS
(2021)
Editorial Material
Genetics & Heredity
Laxminath Tumburu, Swee Lay Thein
Summary: The study identified a new approach for therapeutically reactivating fetal hemoglobin by regulating the CHD4 protein subunit of the NuRD complex.
Correction
Urology & Nephrology
Paul D. Jewell, Kate Bramham, James Galloway, Frank Post, Sam Norton, James Teo, Richard Fisher, Rohit Saha, Sam Hutchings, Phil Hopkins, Priscilla Smith, Jennifer Joslin, Satish Jayawardene, Sarah Mackie, Ali Mudhaffer, Amelia Holloway, Henry Kibble, Mosammat Akter, Benjamin Zuckerman, Kieran Palmer, Ciara Murphy, Domniki Iatropoulou, Claire C. Sharpe, Eirini Lioudaki
Article
Urology & Nephrology
Rachel K. Y. Hung, Elizabeth Binns-Roemer, John W. Booth, Rachel Hilton, Julie Fox, Fiona Burns, Mark Harber, Andrew Ustianowski, Lisa Hamzah, James E. Burns, Amanda Clarke, David A. Price, Stephen Kegg, Denis Onyango, Beatriz Santana-Suarez, Lucy Campbell, Kate Bramham, Claire C. Sharpe, Caroline A. Sabin, Cheryl A. Winkler, Frank A. Post
Summary: This study investigated the association between sickle cell trait (SCT) and kidney disease in people of African ancestry with HIV in the UK. The results showed that individuals with SCT were more likely to have lower glomerular filtration rate, proteinuria, and albuminuria. These associations were particularly significant in participants with low-risk APOL1 genotypes.
KIDNEY INTERNATIONAL REPORTS
(2022)
Article
Hematology
Julia Zhe Xu, Swee Lay Thein
Summary: Sickle cell disease (SCD) is characterized by chronic hemolytic anemia and intermittent acute pain episodes. Anemia in SCD not only reduces red cell mass and oxygen delivery, but also leads to red cell breakdown and release of cell-free hemoglobin, contributing to multiorgan damage. Balancing the benefits of raising hemoglobin levels and the potential risks of increasing blood viscosity is crucial in the treatment of anemia in SCD.
Article
Urology & Nephrology
Damien R. Ashby, Ben Caplin, Richard W. Corbett, Elham Asgari, Nicola Kumar, Alexander Sarnowski, Richard Hull, David Makanjuola, Nicholas Cole, Jian Chen, Sofia Nyberg, Kieran McCafferty, Faryal Zaman, Hugh Cairns, Claire Sharpe, Kate Bramham, Reza Motallebzadeh, Kashif Jamil Anwari, Alan D. Salama, Debasish Banerjee
Summary: This study found a significantly lower risk of severe COVID-19 in patients on dialysis who became infected with SARS-CoV-2 after receiving the COVID-19 vaccine.
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
(2022)
Article
Transplantation
Damien R. Ashby, Ben Caplin, Richard W. Corbett, Elham Asgari, Nicola Kumar, Alexander Sarnowski, Richard Hull, David Makanjuola, Nicholas Cole, Jian Chen, Sofia Nyberg, Suzanne Forbes, Kieran McCafferty, Faryal Zaman, Hugh Cairns, Claire Sharpe, Kate Bramham, Reza Motallebzadeh, Kashif Anwari, Tayeba Roper, Alan D. Salama, Debasish Banerjee
Summary: This multi-center study found that hemodialysis patients had a lower incidence of SARS-CoV-2 infection during the Omicron dominant period after vaccination. Vaccination reduced the risk of hospitalization and severe illness, particularly after the third dose.
NEPHROLOGY DIALYSIS TRANSPLANTATION
(2022)
Article
Urology & Nephrology
Sudha Priya Soundara Pandi, Michael J. Shattock, Bruce M. Hendry, Claire C. Sharpe
Summary: The role of different isoforms of TTCC in mouse mesangial cells was investigated in this study to determine the best therapeutic target for treating mesangioproliferative kidney diseases. Single and double knockout clones of the TTCC isoforms Ca(V)3.1 and Ca(V)3.2 were generated using CRISPR-cas9 gene editing. It was found that the Ca(V)3.1 isoform is required for stimulated phosphorylation of ERK1/2, while the Ca(V)3.2 isoform is not. Neither isoform was necessary for cell proliferation, and the anti-proliferative effects of mibefradil and TH1177 were not isoform-specific.
Editorial Material
Hematology
Swee Lay Thein, Vandana Sachdev
Summary: The study suggests that early initiation of disease-modifying therapy can mitigate sickle myocardial fibrosis by using extracellular volume fraction as a potential imaging biomarker.
Review
Immunology
Claire C. Sharpe, Abid Suddle, Sara Stuart-Smith
Summary: Sickle cell disease is a common genetic disorder with a global prevalence of over 300,000 people. While standard care can help patients live into adulthood, complications can still arise, leading to end-stage organ disease. Solid organ transplantation is an established treatment option for sickle cell patients, but careful patient selection and management are crucial for success. Hematopoietic stem cell transplantation and gene therapy may offer future treatments for adult patients with chronic organ damage, but they are not yet widely available. Currently, early intervention and comprehensive care can minimize the need for solid organ transplantation later in life.
Letter
Education, Scientific Disciplines
Mohsen Tavakol, Claire C. Sharpe, Claire Stewart, David G. O'Brien
Article
Transplantation
James E. Neffendorf, Tracey Mare, Andrew R. H. Simpson, Cristina Soare, Varo Kirthi, Claire C. Sharpe, Timothy L. Jackson
NEPHROLOGY DIALYSIS TRANSPLANTATION
(2023)
Article
Critical Care Medicine
Callum Jackson, Iain Stewart, Tatiana Plekhanova, Peter S. Cunningham, Andrew L. Hazel, Bashar Al-Sheklly, Raminder Aul, Charlotte E. Bolton, Trudie Chalder, James D. Chalmers, Nazia Chaudhuri, Annemarie B. Docherty, Gavin Donaldson, Charlotte L. Edwardson, Omer Elneima, Neil J. Greening, Neil A. Hanley, Victoria C. Harris, Ewen M. Harrison, Ling-Pei Ho, Linzy Houchen-Wolloff, Luke S. Howard, Caroline J. Jolley, Mark G. Jones, Olivia C. Leavy, Keir E. Lewis, Nazir Lone, Michael Marks, Hamish J. C. McAuley, Melitta A. McNarry, Brijesh Patel, Karen Piper-Hanley, Krisnah Poinasamy, Betty Raman, Matthew Richardson, Pilar Rivera-Ortega, Sarah L. Rowland-Jones, Alex Rowlands, Ruth M. Saunders, Janet T. Scott, Marco Sereno, Ajay M. Shah, Aarti Shikotra, Amisha Singapuri, Stefan C. Stanel, Mathew Thorpe, Daniel G. Wootton, Thomas Yates, R. Gisli Jenkins, Sally J. Singh, William D-C Man, Christopher E. Brightling, Louise Wain, Joanna C. Porter, A. A. Roger Thompson, Alex Horsley, Philip L. Molyneaux, Rachael A. Evans, Samuel E. Jones, Martin K. Rutter, John F. Blaikley, PHOSP-COVID Study Collaborative Grp
Summary: This study investigated the prevalence and nature of sleep disturbance after discharge following hospital admission for COVID-19 and found that sleep disturbance is associated with dyspnoea, anxiety, and muscle weakness. Targeting sleep disturbance might be beneficial in treating the post-COVID-19 condition.
LANCET RESPIRATORY MEDICINE
(2023)
Article
Education, Scientific Disciplines
Mohsen Tavakol, David G. O'Brien, Claire C. Sharpe, Claire Stewart
Summary: Post-assessments psychometric reports are crucial in ensuring the reliability, validity, and fairness of assessments. Summarizing students' scores through frequency distributions, central tendency measures, and dispersion measures helps evaluate the quality of items and differentiate between students who have achieved or not achieved the learning outcomes. Estimating individual item reliability and validity indices enhances test-score reliability and validity. Assessing test reliability, consistency, and validity, along with measuring variation using the standard error of measurement, provides insights into test accuracy. The use of reality checks, such as the Hofstee method, P values, and correlation analysis, can improve the validity of standard setting. The Rasch model aids in modifying assessment questions and identifying areas requiring additional instruction. To support test developers, we propose 12 tips for interpreting structured psychometric reports, including analyzing and refining flawed items and ensuring fair assessments with accurate and defensible marks.
