Long-term efficacy and safety of luspatercept for the treatment of anaemia in patients with transfusion-dependent β-thalassaemia (BELIEVE): final results from a phase 3 randomised trial.

Background: Treatments to reduce red blood cell (RBC) transfusion burden among patients with transfusion-dependent β-thalassaemia remain limited. Here, we report long-term follow-up data from the phase 3 BELIEVE trial of luspatercept for transfusion-dependent β-thalassaemia.

Methods: BELIEVE was a phase 3, randomised, double-blind, placebo-controlled study performed at 65 sites in 15 countries.

Post-treatment Recovery in Orbital Arteriovenous Fistulas: A Systematic Review.

Orbital arteriovenous fistulas (AVFs) are rare vascular malformations that can cause severe ocular complications. This review evaluates the effectiveness of treatment strategies, focusing on post-treatment recovery and recurrence. A systematic review was conducted using PubMed and Scopus with no date restrictions.

Safety and efficacy of mitapivat in sickle cell disease (RISE UP): results from the phase 2 portion of a global, double-blind, randomised, placebo-controlled trial.

Background: Sickle cell disease, a debilitating, inherited haemolytic anaemia with premature morbidity and mortality, affects millions globally. Mitapivat, a first-in-class, oral, allosteric activator of pyruvate kinase, improves red blood cell survival by increasing ATP and diminishes sickling by decreasing 2,3-diphosphoglycerate. We aimed to evaluate the efficacy and safety of mitapivat in patients with sickle cell disease.

Luspatercept: a treatment for ineffective erythropoiesis in thalassemia.

Patients with β-thalassemia continue to have several unmet needs. In non-transfusion-dependent patients, untreated ineffective erythropoiesis and anemia have been associated with a variety of clinical sequelae, with no treatment currently available beyond supportive transfusions. In transfusion-dependent forms, lifelong transfusion and iron chelation therapy are associated with considerable clinical, psychological, and economic burden on the patient and health care system.

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.

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-reported outcome (NTDT-PRO) tiredness/weakness (T/W) and shortness of breath (SoB) scores. A secondary objective was to determine the symptom severity threshold for the NTDT-PRO T/W domain to identify patients with symptomatic T/W.

Design: Pooled blinded data from the phase 2, double-blind, placebo-controlled, randomised BEYOND trial in NTDT (NCT03342404) were used.

Posttraumatic Osteomyelitis in Mosul and Gaza: A Retrospective Cohort Study, 2018-2022.

Background: The history of conflicts in the Middle East has resulted in a high burden of complications from conflict-related wounds like posttraumatic osteomyelitis (PTO). This is particularly challenging to manage in settings like Mosul, Iraq and Gaza, Palestine, where healthcare systems are weakened. In nonconflict settings, PTO caused by (PAPTO) can lead to >20% of treatment failures.

Anemia and iron overload as prognostic markers of outcomes in β-thalassemia.

Introduction: Ineffective erythropoiesis and subsequent anemia as well as primary and secondary (transfusional) iron overload are key drivers for morbidity and mortality outcomes in patients with β-thalassemia.

Areas Covered: In this review, we highlight evidence from observational studies evaluating the association between measures of anemia and iron overload versus outcomes in both non-transfusion-dependent and transfusion-dependent forms of β-thalassemia.

Expert Opinion: Several prognostic thresholds have been identified with implications for patient management.

Recommendations for diagnosis, treatment, and prevention of iron deficiency and iron deficiency anemia.

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 amount that is replaced through dietary iron absorption.

Iron chelation therapy for children with transfusion-dependent β-thalassemia: How young is too young?

In this review, we provide a summary of evidence on iron overload in young children with transfusion-dependent β-thalassemia (TDT) and explore the ideal timing for intervention. Key data from clinical trials and observational studies of the three available iron chelators deferoxamine, deferiprone, and deferasirox are also evaluated for inclusion of subsets of young children, especially those less than 6 years of age. Evidence on the efficacy and safety of iron chelation therapy for children ≥2 years of age with transfusional iron overload is widely available.

Machine learning in action: Revolutionizing intracranial hematoma detection and patient transport decision-making.

Objectives: Traumatic intracranial hematomas represent a critical clinical situation where early detection and management are of utmost importance. Machine learning has been recently used in the detection of neuroradiological findings. Hence, it can be used in the detection of intracranial hematomas and furtherly initiate a management cascade of patient transfer, diagnostics, admission, and emergency intervention.

Quality of life, mood disorders, and cognitive impairment in adults with β-thalassemia.

Advances in understanding the disease process in β-thalassemia supported development of various treatment strategies that resulted in improved survival. Improved survival, however, allowed multiple morbidities to manifest and cemented the need for frequent, lifelong treatment. This has directly impacted patients' health-related quality of life and opened the door for various psychiatric and cognitive disorders to potentially develop.

von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease.

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, a prospective, noncontrolled, international phase 3 trial, investigated the efficacy and safety of Wilate prophylaxis in severe patients with VWD.

Αlpha-thalassemia: A practical overview.

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) survival, chronic hemolytic anemia, and subsequent comorbidities. Clinical presentation varies depending on the genotype, ranging from a silent or mild carrier state to severe, transfusion-dependent or lethal disease. Management of patients with α-thalassemia is primarily supportive, addressing either symptoms (eg, RBC transfusions for anemia), complications of the disease, or its transfusion-dependence (eg, chelation therapy for iron overload).

Unmet needs in β-thalassemia and the evolving treatment landscape.

β-thalassemias are genetic disorders causing an imbalance in hemoglobin production, leading to varying degrees of anemia, with two clinical phenotypes: transfusion-dependent thalassemia (TDT) and non-transfusion-dependent thalassemia (NTDT). Red blood cell transfusions and iron chelation therapy are the conventional treatment options for the management of β-thalassemia. Currently available conventional therapies in thalassemia have many challenges and limitations.

Cancer Research in Vulnerable Populations: A Call for Collaboration and Sustainability From MENAT Countries.

Purpose: Cancer is a major burden across Middle East, North Africa, Türkiye (MENAT). Many MENAT countries experience multiple conflicts that compound vulnerabilities, but little research investigates the linkages between vulnerability and cancer research. This study examines the current level and the potential for cancer research among vulnerable populations in the MENAT region, aiming to provide direction toward developing a research agenda on the region's vulnerable populations.

Eviscerated liver: an extremely rare complication of abdominal wound dehiscense through a midline incision.

Abdominal wound dehiscense, or burst abdomen, is a critical postoperative complication necessitating immediate intervention. We present an extremely rare case of left hepatic lobe evisceration through wound dehiscense in a 65-year-old female receiving palliative care for hypopharyngeal squamous cell carcinoma. The patient's midline incision that was performed for feeding jejunostomy tube displayed liver protrusion on Day 14 postoperatively.

Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East.

β-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators and more novel ineffective erythropoiesis-targeted therapy.

Compliance and clinical benefit of deferasirox granule and dispersible tablet formulation in pediatric patients with transfusional iron overload: in a randomized, open-label, multicenter, phase II study.

CALYPSO (clinicaltrials gov. Identifier: NCT02435212), a randomized, open-label, multicenter, phase II study evaluated the compliance, clinical benefits, and safety of deferasirox granules and dispersible tablets (DT) in pediatric patients with iron overload. Iron chelation therapy-naive and iron chelation therapy-pretreated patients aged 2 to <18 years with transfusion- dependent anemias were enrolled.

Enhancement of the Sensing Performance of Devices based on Multistimuli-Responsive Hybrid Materials.

Capturing environmental stimuli is an essential aspect of electronic skin applications in robotics and prosthetics. Sensors made of temperature- and humidity-responsive hydrogel and piezoelectric zinc oxide (ZnO) core-shell nanorods have shown the necessary sensitivity. This is achieved by using highly conformal and substrate independent deposition methods for the ZnO and the hydrogel, i.

Thalassemia and malignancies: Updates from the literature.

Thalassemia management has undergone significant development with the advancement in iron chelation therapy, which has led to a prolonged life expectancy. This has been accompanied by the emergence of several new morbidities and chronic diseases, including cancer. Over the years, multiple cases of solid and hematologic malignancies in thalassemia patients have been reported in the literature, with no clear mechanism for the development of cancer in these patients despite a number of potential mechanisms.