The impact of oral health promotion on the quality of life of children with bleeding disorders: fighting misconceptions.

Background: Understanding the disease-specific risks and patient-related barriers of children with bleeding disorders is necessary for primary oral health promotion. Our goal was to assess the oral health status and the impact of oral health promotion among patients with bleeding disorders.

Research Design And Methods: At baseline, 70 patients with inherited and acquired bleeding disorders had a complete intraoral examination, completed the oral health-related quality of life (OHRQoL) questionnaires, and an oral health education was given.

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.

Psychosocial and mental profile of children with sickle cell disease and their caregivers.

Sickle cell disease (SCD), a chronic debilitating disorder that may negatively affect health-related quality-of-life (HRQoL). In this observational, case-control study, we aim to assess the prevalence of impaired psychosocial profile and poor HRQoL among SCD patients and their caregivers as well as to determine the association of such impairment with parameters of disease severity. Sixty-five children and adolescents with SCD and 65 age- and sex-matched healthy controls and their caregivers were recruited.

Efficacy and safety of early-start deferiprone in infants and young children with transfusion-dependent beta thalassemia: Evidence for iron shuttling to transferrin in a randomized, double-blind, placebo-controlled, clinical trial (START).

Children with transfusion-dependent thalassemia (TDT) require regular blood transfusions that, without iron-chelation therapy, lead to iron-overload toxicities. Current practice delays chelation therapy (late-start) until reaching iron overload (serum ferritin ≥1000 μg/L) to minimize risks of iron-depletion. Deferiprone's distinct pharmacological properties, including iron-shuttling to transferrin, may reduce risks of iron depletion during mild-to-moderate iron loads and iron overload/toxicity in children with TDT.

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.

Sleep disordered breathing and its relation to stroke and pulmonary hypertension in children with sickle cell disease: a single-center cross-sectional study.

Sleep disordered breathing (SDB) is a common underdiagnosed sequela of sickle cell disease (SCD) that has been linked to the frequency of vaso-occlusive crises. To determine the frequency of SDB in children with SCD and its association to SCD-related complications, thirty children and adolescents with SCD at their steady state underwent clinical, laboratory, and radiological assessment using transcranial duplex (TCD) and echo assessment of tricuspid regurge velocity (TRV). All participants had an overnight polysomnography after completing the modified STOP-Bang questionnaire.

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.

Deferiprone for transfusional iron overload in sickle cell disease and other anemias: open-label study of up to 3 years.

Long-term safety and efficacy data on the iron chelator deferiprone in sickle cell disease (SCD) and other anemias are limited. FIRST-EXT was a 2-year extension study of FIRST (Ferriprox in Patients With Iron Overload in Sickle Cell Disease Trial), a 1-year, randomized noninferiority study of deferiprone vs deferoxamine in these populations. Patients who entered FIRST-EXT continued to receive, or were switched to, deferiprone.

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.

Ledipasvir-sofosbuvir in Adolescents With Chronic Hepatitis C and Hematological Malignancies Undergoing Chemotherapy.

Objectives: In children with hematological malignancies, chronic hepatitis C virus (HCV) infection has been associated with more rapid liver disease progression and higher risk of malignancy relapse due to chemotherapy interruption. We evaluated the safety and efficacy of ledipasvir-sofosbuvir for 12weeks in these patients.

Methods: In a phase 2, open-label study, at one site in Egypt, patients ages 12-<18years with chronic HCV genotype 1 or 4 infection undergoing maintenance chemotherapy for hematological malignancies received ledipasvir-sofosbuvir (90 mg/400 mg) once daily for 12weeks.

Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study.

Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload that requires chelation therapy. The iron chelator deferiprone is frequently used in individuals with thalassemia syndromes, but data in patients with SCD are limited. This open-label study assessed the efficacy and safety of deferiprone in patients with SCD or other anemias receiving chronic transfusion therapy.

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.

Association between rs12979860, rs17047200 and rs4618569 Variant Polymorphisms with the Course and Outcome of SARS-CoV-2 Patients.

Background: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection provides a critical host-immunological challenge.

Aim: We explore the effect of host-genetic variation in interferon-lambda-3 rs12979860, Tolloid Like-1 () rs17047200 and Discoidin domain receptor 1() rs4618569 on host response to respiratory viral infections and disease severity that may probe the mechanistic approach of allelic variation in virus-induced inflammatory responses.

Methods: 141 COVID-19 positive patients and 100 healthy controls were tested for interferon-lambda-3 rs12979860, rs17047200 and DDR1 rs4618569 polymorphism by TaqMan probe-based genotyping.

COVID-19 in Children With Cancer: A Single Low-Middle Income Center Experience.

Background: Coronavirus disease-2019 (COVID-19) could be associated with morbidity and mortality in immunocompromised children.

Objective: The objective of this study was to measure the frequency of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection among hospitalized children with cancer and to detect the associated clinical manifestations and outcomes.

Methodology: A prospective noninterventional study including all hospitalized children with cancer conducted between mid-April and mid-June 2020 in Ain Shams University Hospital, Egypt.

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.

High Risk of HBV Infection Among Vaccinated Polytransfused Children With Malignancy.

Aim Of The Study: The national Egyptian hepatitis B virus (HBV) vaccination program coverage of all infants started in 1992. The study aimed to assess immunity against HBV and occurrence of HBV breakthrough infections in vaccinated polytransfused children with malignancies.

Patients And Methods: Eighty-nine polytransfused children with malignancies were recruited; 37 were on chemotherapy (male:female 20:17; mean age 7.

Micro-elimination of hepatitis C through testing of Egyptian pregnant women presenting at delivery: implications for screening policies.

Objectives: Despite the high burden of hepatitis C virus (HCV) infection in Egypt, screening of pregnant women is not yet universal, making national and global elimination unlikely. This study assessed the proportion of pregnant women who were screened for HCV infection at delivery, the prevalence and risk factors for HCV infection, the associated adverse neonatal outcomes, and the real-life linkage to care of infected women and follow-up of their infants' HCV status and timing of testing.

Methods: Data were collected from medical records of a retrospective cohort of all pregnant women who were admitted to a university hospital in Cairo for delivery between January and June 2018 (n = 6734).

The Adverse Impact of Thalassemia Major on Adolescents' Oral Health-related Quality of Life.

Background: Adolescents with beta-thalassemia major (βTM) had unfavorable quality of life (QOL).

Objectives: To assess oral health status of adolescents with βTM and its impact on their oral health-related QOL (OHRQoL).

Subjects And Methods: Forty adolescents with βTM were recruited and compared with 40 age-matched and sex-matched healthy adolescents.

Body Composition in Egyptian Children With Transfusion-dependent Thalassemia: The Impact of Nutrition and Metabolic Profile.

Background: Growth failure is a common complication in children with beta-thalassemia major (β-TM) that has persisted despite major treatment advances. It could stem from malnutrition, especially in those who live in poor countries and who have inadequate nutrient intake.

Aim: The aim of this study was to assess the influence of nutrition on growth, total body composition, and metabolic profile in Egyptian children with β-TM.

Reticulocyte Hemoglobin Content (Ret He): A Simple Tool for Evaluation of Iron Status in Childhood Cancer.

Background: Cancer-related anemia is a common complication of cancer and its treatment that may be mediated by nutritional deficiency or inflammatory cytokines inhibiting erythropoiesis.

Aim: We evaluated the value of reticulocyte hemoglobin content (Ret He) as a marker of iron availability for erythropoiesis in childhood cancer and the impact of oral iron supplementation on hematologic parameters in patients with low Ret He.

Materials And Methods: This prospective study included 100 pediatric patients with cancer on chemotherapy who were screened for the presence of anemia.

Angiopoietin-2 as a Marker of Retinopathy in Children and Adolescents With Sickle Cell Disease: Relation to Subclinical Atherosclerosis.

Objectives: Angiopoietin-2 (Ang-2) is a multifaceted cytokine that functions in both angiogenesis and inflammation. A proangiogenic state has been found in adults with sickle cell disease (SCD), mainly because of elevated Ang-2 levels. We determined Ang-2 level in 40 children and adolescents with SCD compared with 40 healthy controls and assessed its relation to retinopathy as well as carotid intimamedia thickness (CIMT).

Surfactant protein D as a marker for pulmonary complications in pediatric patients with sickle cell disease: Relation to lung function tests.

Background: Surfactant protein D (SP-D) is considered a candidate biomarker for lung integrity and for disease progression.

Aim: We determined the level of SP-D in children and adolescents with SCD and assessed its possible relation to pulmonary complications and lung function.

Methods: Serum SP-D levels were assessed in 50 SCD patients compared with 30 healthy controls.