Safety and efficacy of L-Glutamine in reducing the frequency of acute complications among patients with sickle cell disease: A randomized controlled study.

To evaluate the safety and efficacy of L-glutamine in reducing vaso-occlusive crisis (VOC) and improving cerebral arterial blood flow in children with sickle cell disease (SCD). This is an interventional randomized controlled trial that recruited sixty SCD patients, aged 9.2 ± 3.

The implementation of automated red blood cell exchange (erythrocytapheresis) as a treatment modality in sickle cell disease patients: Single center experience.

Background: Improvements of health infrastructure, preventive care and clinical management is important to reduce the morbidity and mortality of sickle cell disease (SCD).

Objective: This prospective, investigator-initiated non-randomized open-label intervention, single centre study describes the implementation of the automated erythrocytapheresis in low-middle income country as a treatment modality for SCD patients to improve the standard of care and highlights its benefits and challenges.

Methodology: Eligible patients with SCD who had overt stroke, abnormal/conditional transcranial doppler (TCD), or other indications were subjected to regular automated erythrocytapheresis program.

Endothelin-1 gene polymorphism (G8002A) and endothelial monocyte-activating polypeptide II: Role in vascular dysfunction in pediatric patients with β-thalassemia major.

Background: Endothelin-1 (ET-1), a potent endogenous vasoconstrictor, stimulates production of reactive oxygen species. Endothelial monocyte-activating polypeptide-II (EMAP-II) is a multifunctional polypeptide.

Aim: To assess ET-1 gene polymorphism (G8002A) in pediatric patients with β-thalassemia major (β-TM) as a potential genetic marker for vascular dysfunction and its possible relation to EMAP II, oxidative stress and vascular complications.

The role of MRI-R2* in the detection of subclinical pancreatic iron loading among transfusion-dependent sickle cell disease patients and correlation with hepatic and cardiac iron loading.

Objectives: Pancreatic reserve could be preserved by early assessment of pancreatic iron overload among transfusion-dependent sickle cell disease (SCD) patients. This study aimed to measure pancreatic iron load and correlate its value with patients' laboratory and radiological markers of iron overload.

Materials And Methods: Sixty-six SCD children and young adults underwent MRI T2* relaxometry using a simple mathematical spreadsheet and laboratory assessment.

Epistaxis in a Pediatric Outpatient Clinic: Could It be an Alarming Sign?

 Epistaxis is a common presentation among children.  To investigate the suitability of a simple tool of assessment for patients with epistaxis that could guide in subgrouping those with possible bleeding tendencies who may need further assessment.  Children who presented to a tertiary outpatient clinic with epistaxis of an unknown cause were recruited.

Hidden brain iron content in sickle cell disease: impact on neurocognitive functions.

Children with sickle cell disease (SCD) are at a high risk for neurocognitive impairment. We aim to quantitatively measure cerebral tissue R2* to investigate the brain iron deposition in children and young adults with SCD in comparison to beta thalassemia major (BTM) and healthy controls and evaluate its impact on neurocognitive functions in patients with SCD. Thirty-two SCD, fifteen BTM, and eleven controls were recruited.

COVID-19 effect on clinical research: Single-site risk management experience.

Background: The ongoing coronavirus disease 2019 (COVID-19) pandemic is extensively impacting new and ongoing clinical trials of various medical products irrespective of indication. It has the potential to adverse effect not only in terms of recruitment and immediate patient care but is also likely to affect the data collection and analysis in the months to come.

Aim: The aim was to illustrate the effect of COVID-19 on the clinical research in one of the research centers in low limited-resource country as Egypt and the management plan performed to decrease this adverse impact.

Angiotensinogen M235T Gene Polymorphism is a Genetic Determinant of Cerebrovascular and Cardiopulmonary Morbidity in Adolescents with Sickle Cell Disease.

Background: Cerebrovascular stroke is a common critical complication of sickle cell disease (SCD). Angiotensinogen (AGT) M235T gene polymorphism is associated with risk of ischemic stroke and cardiovascular disease.

Aim: We investigated the potential association between angiotensinogen M235T gene polymorphism and susceptibility to cerebrovascular and cardiopulmonary complications in adolescents with SCD.

Immunological role of CD4CD28 T lymphocytes, natural killer cells, and interferon-gamma in pediatric patients with sickle cell disease: relation to disease severity and response to therapy.

Sickle cell disease (SCD) is associated with alterations in immune phenotypes. CD4CD28 T lymphocytes have pro-inflammatory functions and are linked to vascular diseases. To assess the percentage of CD4CD28 T lymphocytes, natural killer cells (NK), and IFN-gamma levels, we compared 40 children and adolescents with SCD with 40 healthy controls and evaluated their relation to disease severity and response to therapy.

Renal iron deposition by magnetic resonance imaging in pediatric β-thalassemia major patients: Relation to renal biomarkers, total body iron and chelation therapy.

Background: The reciprocal of multiecho gradient-echo (ME-GRE) T2* magnetic resonance imaging (MRI) R2*, rises linearly with tissue iron concentration in both heart and liver. Little is known about renal iron deposition in β-thalassemia major (β-TM).

Aim: To assess renal iron overload by MRI and its relation to total body iron and renal function among 50 pediatric patients with β-TM.