A5003414034- Complement system in diseases
- Blood groups and transfusion
- Political and Economic history of UK and US
- Political Systems and Governance
- European and International Law Studies
- Irish and British Studies
- Hemoglobinopathies and Related Disorders
- Renal Diseases and Glomerulopathies
- Coagulation, Bradykinin, Polyphosphates, and Angioedema
- Acute Myeloid Leukemia Research
- Adenosine and Purinergic Signaling
- Cinema and Media Studies
- Histone Deacetylase Inhibitors Research
- Medieval Literature and History
- Chronic Lymphocytic Leukemia Research
- Chronic Myeloid Leukemia Treatments
- Liver Disease and Transplantation
- Legal Issues in South Africa
- Platelet Disorders and Treatments
- Corporate Governance and Law
- Linguistics and language evolution
- Music History and Culture
- Complex Systems and Decision Making
- Prostate Cancer Treatment and Research
- Scottish History and National Identity
St James's University Hospital
2010-2025
Cancer Australia
2025
Peter MacCallum Cancer Centre
2022-2025
The Alfred Hospital
2025
The University of Melbourne
2025
Leeds Teaching Hospitals NHS Trust
2011-2024
European Society for Blood and Marrow Transplantation
2024
Centre Hospitalier Universitaire de Toulouse
2024
German Red Cross
2024
Chinese Academy of Medical Sciences & Peking Union Medical College
2024
BackgroundPersistent hemolytic anemia and a lack of oral treatments are challenges for patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or not complement inhibitors. Iptacopan, first-in-class factor B inhibitor, has been shown to improve hemoglobin levels in these patients.MethodsIn two phase 3 trials, we assessed iptacopan monotherapy over 24-week period less than 10 g per deciliter. In the first, anti-C5–treated were randomly assigned switch continue...
Pegcetacoplan significantly improves outcomes for patients with paroxysmal nocturnal hemoglobinuria (PNH) experiencing extravascular hemolysis (EVH) on eculizumab, leading to approval in 2021/2022 (USA/Europe). We report the first collaborative real-world evidence pegcetacoplan use UK and France. A total of 48 were either currently receiving or previously received (2019-2023). 12 had participated PEGASUS clinical trial, continuing treatment after trial completion. Five combination C5...
Background Paroxysmal nocturnal hemoglobinuria is an acquired hemolytic anemia characterized by intravascular hemolysis which has been demonstrated to be effectively controlled with eculizumab. However, lactate dehydrogenase levels remain slightly elevated and haptoglobin low in some patients suggesting residual low-level hemolysis. This may due C3-mediated clearance of paroxysmal red blood cells through the reticuloendothelial system.Design Methods Thirty-nine samples from not treated...
Summary Pulmonary hypertension (PH) is a common complication of haemolytic anaemia. Intravascular haemolysis leads to nitric oxide (NO) depletion, endothelial and smooth muscle dysregulation, vasculopathy, characterized by progressive hypertension. PH has been reported in patients with paroxysmal nocturnal haemoglobinuria (PNH), life‐threatening disease. We explored the relationship between haemolysis, systemic NO, arginine catabolism measures 73 PNH enrolled placebo‐controlled TRIUMPH...
Population-based information on cancer incidence, prevalence and outcome are required to inform clinical practice research; but contemporary data lacking for many myeloid malignancy subtypes.Set within a socio-demographically representative UK population of ∼4 million, (N=5231 diagnoses) from an established patient cohort. Information survival (relative & overall), transformation/progression, is presented >20 subtypes.The median diagnostic age was 72.4years (InterQuartile Range 61.6-80.2),...
Abstract Background The GOSSAMER phase 2 study assessed the FMS ‐like tyrosine kinase 3 (FLT3) inhibitor gilteritinib as maintenance therapy in patients with FLT3 –internal tandem duplication ( ‐ITD) acute myeloid leukemia (AML) first complete remission without previous hematopoietic stem cell transplantation (HSCT). Methods Patients had to be within months of their last consolidation cycle and have completed recommended number cycles per local practice. inhibitors were allowed only during...
In Paroxysmal nocturnal haemoglobinuria (PNH), pregnancy is associated with increased maternal and foetal complications to such an extent that the condition has been considered relatively contra-indicated in PNH. Eculizumab revolutionized treatment of We evaluate its use date. report on seven patients exposed eculizumab at different stages including first two receive drug from conception delivery. There was no evidence complement blockade cord blood samples taken appears safe this setting...
Abstract Purpose: Azacitidine (AZA) is a novel therapeutic option in older patients with acute myeloid leukemia (AML), but its rational utilization compromised by the fact that neither determinants of clinical response nor mechanism action are defined. Co-administration histone deacetylase inhibitors, such as vorinostat (VOR), reported to improve activity AZA, this has not been prospectively studied AML. Experimental Design: We compared outcomes 259 adults AML (n = 217) and MDS 42)...
Abstract Objectives A retrospective population‐based study to determine the incidence and prevalence of patients with rare blood disease paroxysmal nocturnal haemoglobinuria (PNH). Methods All were identified by flow cytometric detection cells deficient in glycosylphosphatidylinositol (GPI) linked proteins at a single diagnostic reference laboratory that serves Yorkshire based, Haematological Malignancy Research Network (HMRN) population 3.8 million. Results One hundred ninety‐seven...
Pulmonary hypertension is becoming a recognized complication of the hereditary and acquired haemolytic anaemias, associated with poor prognosis. Recently we reported that patients paroxysmal nocturnal haemoglobinuria (PNH) have high levels N-terminal pro-brain natriuretic peptide (NT-proBNP), biomarker both right left ventricular dysfunction cardiac dysfunction. In current study evaluated cohort (N = 29) PNH for elevated pulmonary artery systolic pressure function by...
Patients with paroxysmal nocturnal hemoglobinuria (PNH) experience complement-mediated intravascular hemolysis leading to anemia, fatigue, and potentially life-threatening thrombotic complications. Pegcetacoplan, a C3 inhibitor, demonstrated sustained improvements in hematologic clinical parameters the phase 3 PEGASUS trial patients PNH who remained anemic despite C5 inhibitor therapy. The present post hoc analysis describes 26 adverse events (AEs) experienced 19 during pegcetacoplan therapy...
To evaluate the survival benefit of chemotherapy intensification in older patients with AML who have not achieved a measurable residual disease (MRD)-negative remission.
Abstract Advanced melanoma was historically considered incurable, however a 52% 10-year melanoma-specific survival rate from seminal immunotherapy trials challenges that conclusion 1. There is no literature exploring clinicians’ discussion of treatment-intent with patients, or whether this represents cure. We performed multi-center retrospective cohort analysis to examine treatment-intent, using electronic medical records identify 278 patients unresectable stage IV consented for 2019-2023....
Abstract Relapsed/refractory (R/R) Acute Myeloid Leukemia (AML) is a genetically complex and heterogeneous disease with poor prognosis limited treatment options. Thus, there an urgent need to develop therapeutic combinations overcome drug resistance in AML. This open‐label, multicenter, international, phase 1b study evaluated the safety, efficacy, pharmacokinetics of venetoclax combination alvocidib patients R/R Patients were treated escalating doses (400, 600, 800 mg QD, orally, days 1–28)...
Improved survival in patients with hematological malignancy (HM) admitted to the intensive care unit (ICU) has largely been reported uncontrolled cohorts from single academic institutions. We compared hospital mortality between 147 HM and general medical admissions five non-specialist ICUs. The proportion of surviving discharge was significantly worse (27% vs. 56%; p < 0.001). Six-month 1-year 21% 18%, respectively. HM, greater age, mechanical ventilation (MV) acute physiology chronic health...
A Feeling for light and lighting starts with visual imagination, just as a painter's talent does. Think of the creation watercolor rendering—First, major highlights are imagined—then, graded washes different luminosity added and—then, detail minor lightplay makes idea clear entertains eye.