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 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.

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.

Study of the diagnostic criteria for hemophagocytic lymphohistiocytosis in neonatal and pediatric patients with severe sepsis or septic shock.

Septic shock is a major public health concern. However, the clinical and laboratory criteria for sepsis overlap with those for hemophagocytic lymphohistiocytosis (HLH), and their differentiation can be challenging. The aim of this study was to compare HLH criteria among patients diagnosed with neonatal sepsis and childhood sepsis and to study the outcomes in patients fulfilling the diagnostic criteria for HLH.

Intracranial hemorrhage in primary immune thrombocytopenia (ITP): 20 years' experience in pediatrics.

Incidence of intracranial hemorrhage (ICH) among children with primary immune thrombocytopenia (ITP) varies among different studies. We published data during the period of 1997-2007 of ICH in children with primary ITP, addressing risk factors and outcome. The aim of this study is to assess changes in incidence, risk factors, and outcome of ICH in children with ITP from last decade and to report the overall 20 years' experience.

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.

Thyroid hemodynamic alterations in Egyptian patients with sickle cell disease: relation to disease severity, total body iron and thyroid function.

: Intraparenchymal thyroid Doppler measurements might be considered a useful index of the thyroid status as well as micro-circulation elsewhere in the body among sickle cell disease (SCD) patients. The authors aim to evaluate the intra-thyroidal hemodynamic changes and thyroidal volume in SCD patients and its relation to the disease severity, and thyroid functions tests as well as iron overload state. : Sixty SCD patients, randomly recruited from the regular attendants of the Pediatric Hematology Clinic, Ain Shams University, Cairo, Egypt, were studied focusing on the disease duration, the transfusion history, the recorded Hydroxyurea, and chelation therapies and the vaso-occlusive crises history.

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.

Auditory brainstem response in full-term neonates born to mothers with iron deficiency anemia: relation to disease severity.

Iron is crucial for fetal brain development; however, there are insufficient data regarding the effects of maternal iron deficiency anemia (IDA) on auditory neural maturation. We evaluated the effect of maternal IDA on auditory brainstem response (ABR) in full-term neonates. Out of 223 pregnant women, 50 were diagnosed as having IDA and 50 healthy mothers were enrolled as controls.

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.

A pilot study on sperm DNA damage in β-thalassemia major: is there a role for antioxidants?

Excess iron deposition in patients with beta thalassemia major (BTM) causes excess free radical formation, damages the hypothalamic pituitary testicular axis and production of sperms with DNA defects. As antioxidants were reported to improve fertility in healthy males; their effectiveness to improve sperm DNA defects in adult males with BTM was studied. Twenty fully pubertal BTM patients were included consecutively, all had semen analysis; 10 were found to be azoospermic, so further analysis for sperms and DNA defects was conducted on the remaining 10 participants.

Left Ventricular Structural and Functional Changes in Children With β-Thalassemia and Sickle Cell Disease: Relationship to Sleep-disordered Breathing.

Cardiovascular complications are well recognized in β-thalassemia and sickle cell disease (SCD). The objective of this study was to evaluate left ventricular (LV) structural and functional changes and their relationship to sleep-disordered breathing (SDB) in children with β-thalassemia and SCD. One hundred patients recruited from the hematology clinic were subjected to Pittsburgh Sleep Quality Index score; 26 patients had positive score (Pittsburgh Sleep Quality Index ≥5) (15 β-thalassemia major and 11 SCD) and were compared with 25 age-matched and sex-matched controls.

Negative Impact of Prolonged Antibiotics or Persistent Diarrhea on Vitamin K1 Levels in 2-24 Weeks aged Egyptian Infants.

Background: To evaluate the hazard of prolonged antibiotic therapy and/or persistent diarrhea on vitamin K1 (VK1) level and bleeding profile in infants (2-24 weeks).

Methods: A one-year case-control study, conducted at Ain Shams University, Egypt. 338 infants (2-24 weeks) were recruited and divided into 3 groups (1:1:3 ratios); group A (n=67) patients who received antibiotics for ≥10 days, group B (n=67) who had persistent diarrhea ≥ 14 days and group C (n=204) age- and gender- matched infants who had not either received antibiotics nor had diarrhea.

Safety and efficacy of early start of iron chelation therapy with deferiprone in young children newly diagnosed with transfusion-dependent thalassemia: A randomized controlled trial.

Iron overload is inevitable in patients who are transfusion dependent. In young children with transfusion-dependent thalassemia (TDT), current practice is to delay the start of iron chelation therapy due to concerns over toxicities, which have been observed when deferoxamine was started too early. However, doing so may increase the risk of iron accumulation that will be manifested as toxicities later in life.

Klf10 Gene, a Secondary Modifier and a Pharmacogenomic Biomarker of Hydroxyurea Treatment Among Patients With Hemoglobinopathies.

Background: The klf10 gene could indirectly modify γ-globin chain production and hence the level of fetal hemoglobin (HbF) ameliorating the phenotype of β-hemoglobinopathies and the response to hydroxycarbamide (hydroxyurea [HU]) therapy. In this study, we aimed to evaluate the frequency of different genotypes for the klf10 gene in β-thalassemia major (B-TM), β-thalassemia intermedia (B-TI), and sickle cell disease (SCD) patients by polymerase chain reaction and to assess its relation to disease phenotypes and HU response.

Methods: This cross-sectional study included 75 patients: 50 B-TM, 12 SCD, and 13 B-TI patients (on stable HU dose).

Viruses, anti-viral therapy, and viral vaccines in children with immune thrombocytopenia.

Immune thrombocytopenia (ITP) might be preceded by silent or overt viral infections. Similarly, anti-viral drugs and viral vaccines could also trigger ITP and might play a central role in its pathogenesis. The seasonal nature of childhood ITP suggests that viral infections might initiate immune responses that increase the predisposition and occurrence of ITP.

Determination of Human Hemoglobin Derivatives.

The levels of the inactive hemoglobin (Hb) pigments [such as methemoglobin (metHb), carboxyhemoglobin (HbCO) and sulfohemoglobin (SHb)] and the active Hb [in the oxyhemoglobin (oxyHb) form] as well as the blood Hb concentration in healthy non pregnant female volunteers were determined using a newly developed multi-component spectrophotometric method. The results of this method revealed values of SHb% in the range (0.0727-0.

Role of vitamin C as an adjuvant therapy to different iron chelators in young β-thalassemia major patients: efficacy and safety in relation to tissue iron overload.

Background: Vitamin C, as antioxidant, increases the efficacy of deferoxamine (DFO).

Aim: To investigate the effects of vitamin C as an adjuvant therapy to the three used iron chelators in moderately iron-overloaded young vitamin C-deficient patients with β-thalassemia major (β-TM) in relation to tissue iron overload.

Methods: This randomized prospective trial that included 180 β-TM vitamin C-deficient patients were equally divided into three groups (n = 60) and received DFO, deferiprone (DFP), and deferasirox (DFX).