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View article: Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study Open
Background Non-transfusion-dependent beta-thalassemia (β-NTDT) is characterized by ineffective erythropoiesis, increased intestinal iron absorption, and iron overload. The ferroportin inhibitor, vamifeport, has been shown to improve erythr…
View article: Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent β-thalassemia using real-world criteria
Revisiting erythroid response in the phase 3 BELIEVE trial of luspatercept in patients with transfusion-dependent β-thalassemia using real-world criteria Open
Introduction: In the phase 3 randomized, double blind, placebo-controlled BELIEVE trial (NCT02604433) in transfusion-dependent β-thalassemia, 21.4% of patients (pts) treated with luspatercept vs 4.5% with placebo achieved an erythroid resp…
Prevalence of Middle Ear and Sinus Barotrauma between Divers in Saudi Arabia Open
Introduction: Diving is considered one of the most common sports around the world. Due to the popularity of this sport, physicians must be aware of related diving injuries. Most diving injuries are related to fast changes in pressure, whic…
View article: Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study
Efficacy and safety of luspatercept in non–transfusion-dependent β-thalassemia: long-term results from the BEYOND study Open
Chronic anemia due to non–transfusion-dependent β-thalassemia (NTDT) can result in clinical morbidities, particularly with inadequate management. Luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the p…
The Bupa Arabia Value Pilot: A KSA-Based Payor-Provider Collaborative in Maternity Care Open
The Bupa Arabia and Bupa CareConnect Value Pilot explores the transformative journey of implementing Value-Based Healthcare (VBHC) in Maternity care across four hospitals in Saudi Arabia, using ICHOM’s Pregnancy and Childbirth Standard Set…
Impact of insulin resistance and hyperinsulinemia on pulmonary functions in asthmatic children: a cross-sectional study Open
A bstract Background Asthma and obesity prevalence has increased exponentially over the last few decades. Obesity is an inflammatory disease that exerts its negative effect on asthma through several mechanisms one of which is insulin resis…
590 Eviscerated Liver: An Extremely Rare Complication of Abdominal Wound Dehiscense Through a Midline Incision Open
Introduction Abdominal wound dehiscence, or burst abdomen, is a critical, challenging, and upsetting postoperative complication after. It is characterized by the partial or complete disruption without eviscerating the abdominal contents or…
A New Flexible Model for Three-Stage Phenomena: The Fragmented Kumar-Trapez Distribution Open
This article proposes a solution to the problem of limiting the representation of three-stage phenomena to linear forms and addresses the stability of the second stage by introducing a novel distribution, the Fragmented Kumar-Trapez (FKT) …
Reframing thalassaemia syndrome as a benign haematopoietic stem cell disorder Open
Summary Thalassaemia, caused by over 250 mutations in the beta globin gene, changes the haematopoietic stem cell (HSC) differentiation, leading to ineffective erythropoiesis. This Wider Perspective article overlooks its underlying nature a…
Luspatercept Improves Fatigue-Related Quality of Life through 5 Years of Treatment in Non-Transfusion Dependent Beta-Thalassemia Open
Introduction: Luspatercept has been shown to increase hemoglobin levels and improve health-related quality of life (HRQoL) in patients with non-transfusion-dependent beta-thalassemia (NTDT) participating in the phase 2, double-blind random…
View article: ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia
ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia Open
Introduction: Transfusions and iron chelators have improved survival and outcomes in transfusion-dependent (TD)-thalassemia. However, they are associated with adverse effects, such as end organ complications and high healthcare resource ut…
View article: Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial
Improvement of Iron Overload Parameters in Patients with Transfusion-Dependent β-Thalassemia Treated with Luspatercept: Data from the Phase 3b Long-Term Rollover Study Following the BELIEVE Trial Open
Introduction: Patients (pts) with transfusion-dependent (TD) β-thalassemia require lifelong red blood cell (RBC) transfusions, which can contribute to iron overload and increased morbidity and mortality. In the phase 3 BELIEVE trial (NCT02…
Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial Open
Objectives To estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13–24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-repor…
Squamous Cell Carcinoma: From Diagnosis to Effective Treatment-A Comprehensive Guide Open
Squamous cell carcinoma (SCC) is the second most common malignant skin cancer, and its occurrence is on the rise globally. The main risk factors are cumulative sun exposure, immunosuppression, and inflammation. Early diagnosis and treatmen…
Hypercoagulability in hemoglobinopathies: Decoding the thrombotic threat Open
Beta (β)‐thalassemia and sickle cell disease (SCD) are characterized by a hypercoagulable state, which can significantly influence organ complication and disease severity. While red blood cells (RBCs) and erythroblasts continue to play a c…
View article: ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA
ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA Open
Background: In thalassemia, ATP production in erythroid cells is too low to meet the demand of oxidative stress and ensuing cellular damage; this leads to ineffective erythropoiesis (IE) and chronic hemolytic anemia. Guidelines for non–tra…
IMPROVEMENTS IN FATIGUE AND 6-MINUTE WALK TEST IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA: THE PHASE 3 ENERGIZE TRIAL OF MITAPIVAT Open
Background: Thalassemia, a group of inherited disorders characterized by ineffective erythropoiesis and chronic hemolytic anemia, is associated with wide-ranging impacts on health-related quality of life (HRQoL), such as impaired physical …
View article: COVID-19 in patients affected by red blood cell disorders, results from the European registry ERN-EuroBloodNet
COVID-19 in patients affected by red blood cell disorders, results from the European registry ERN-EuroBloodNet Open
Background Despite several publications covering patients from multiple centers, no international registry covered all patients with red blood cell diseases (RBCD) affected by COVID-19. The ERN-EuroBloodNet's registry provided real-time re…
View article: Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia
Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia Open
Iron is an essential nutrient and a constituent of ferroproteins and enzymes crucial for human life. Generally, nonmenstruating individuals preserve iron very efficiently, losing less than 0.1% of their body iron content each day, an amoun…
View article: 131 Clinical outcomes comparison between radiofrequency ablation vs cryoballoon ablation for paroxysmal atrial fibrillation: an observational cohort study
131 Clinical outcomes comparison between radiofrequency ablation vs cryoballoon ablation for paroxysmal atrial fibrillation: an observational cohort study Open
Background Pulmonary vein isolation (PVI) is a standard ablation strategy that can be achieved with cryoablation (Cryo) with a single-shot cryoballoon catheter or point-by-point radiofrequency (RF) ablation to treat symptomatic paroxysmal …
View article: von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease
von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease Open
Long-term prophylaxis with a von Willebrand factor (VWF) concentrate is recommended in patients with von Willebrand disease (VWD) who have a history of severe and frequent bleeds. However, data from prospective studies are scarce. WIL-31, …
View article: Αlpha-thalassemia: A practical overview
Αlpha-thalassemia: A practical overview Open
α-Thalassemia is an inherited blood disorder characterized by decreased synthesis of α-globin chains that results in an imbalance of α and β globin and thus varying degrees of ineffective erythropoiesis, decreased red blood cell (RBC) surv…
Characteristics and Clinical Outcomes of Patients Referred to a Cardio-Oncology Clinic: The First Lebanese Experience Open
Introduction: Survival rates in cancer patients have improved dramatically in the past ten years, especially with the discovery of novel cancer therapy which was found to be associated with multiple cardiovascular complications. The develo…