A5069567664- Hemoglobinopathies and Related Disorders
- Iron Metabolism and Disorders
- Lysosomal Storage Disorders Research
- Blood groups and transfusion
- Hemophilia Treatment and Research
- Pharmacological Effects and Toxicity Studies
- Erythropoietin and Anemia Treatment
- Cellular transport and secretion
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Folate and B Vitamins Research
- Carbohydrate Chemistry and Synthesis
- Prenatal Screening and Diagnostics
- Platelet Disorders and Treatments
- Metabolism and Genetic Disorders
- Cancer-related gene regulation
- Erythrocyte Function and Pathophysiology
- Trypanosoma species research and implications
- Liver Disease Diagnosis and Treatment
- Epigenetics and DNA Methylation
- Chronic Myeloid Leukemia Treatments
- Blood Coagulation and Thrombosis Mechanisms
- Autism Spectrum Disorder Research
- Neonatal Health and Biochemistry
- Blood disorders and treatments
- Sepsis Diagnosis and Treatment
Cairo University
2015-2024
Cairo University hospitals
1999-2024
Faculty (United Kingdom)
2019
Pediatrics and Genetics
2011
Ain Shams University
2008
Texas Children's Hospital
2004
American University of Beirut Medical Center
2004
Boston Children's Hospital
2002
Deoxygenated sickle hemoglobin (HbS) polymerization drives the pathophysiology of cell disease. Therefore, direct inhibition HbS has potential to favorably modify disease outcomes. Voxelotor is an inhibitor.
The development of neutralizing anti–factor VIII alloantibodies (inhibitors) in patients with severe hemophilia A may depend on the concentrate used for replacement therapy.
Background Following a clinical evaluation of deferasirox (Exjade) it was concluded that, in addition to baseline body iron burden, ongoing transfusional intake should be considered when selecting doses. The 1-year EPIC study, the largest ever investigation conducted for an chelator, is first evaluate whether fixed starting doses deferasirox, based on intake, with dose titration guided by serum ferritin trends and safety markers, provides clinically acceptable chelation patients (aged >or=2...
I am honored to invite you enjoy reading this special issue of The Scientific World Journal. One review article is “Ineffective erythropoiesis in β-thalassemia.” authors concluded that ineffective could be the conjunction several mechanisms which final consequence arrest maturation and increased apoptosis erythroblasts during their terminal differentiation stage. Another discusses biologic complexity sickle cell disease. This likely one major barriers development successful new...
Many patients with transfusional iron overload are at risk for progressive organ dysfunction and early death poor compliance older chelation therapies is believed to be a major contributing factor. Phase II/III studies have shown that oral deferasirox 20-30 mg/kg/d reduces burden, depending on intake.The prospective, open-label, 1-yr ESCALATOR study in the Middle East was designed evaluate once-daily > or =2 yr beta-thalassaemia who were previously chelated deferoxamine and/or deferiprone....
Background Prospective data on cardiac iron removal are limited beyond one year and longer-term studies are, therefore, important.Design Methods Seventy-one patients in the EPIC substudy elected to continue into 3rd year, allowing be analyzed over three years.Results Mean deferasirox dose during 3 was 33.6±9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms ±39.1% at baseline 17.1 ±62.0% end of study (P
Purpose Magnetic resonance imaging (MRI)‐based techniques for assessing liver iron concentration (LIC) have been limited by single scanner calibration against biopsy. Here, the of spin‐density projection‐assisted (SDPA) R2‐MRI (FerriScan®) in iron‐overloaded β‐thalassemia patients treated with chelator, deferasirox, 12 months is validated. Methods SDPA measurements and percutaneous needle biopsy samples were obtained from a subgroup ( n = 233) ESCALATOR trial. Five different makes models...
The hemoglobin disorders are the most common clinically serious single gene in world. In Egypt, beta-thalassemia is type with a carrier rate varying from 5.3 to > or =9% and frequency of 0.03. So, it was estimated that 1,000/1.5 million per year live births will suffer thalassemia disease Egypt (total 1,936,205 2006). beta-Thalassemia creates social financial burden for patients' family Egyptian government. high carriers increasing newly born cases pressing reason importance develop...
The randomized comparison of deferasirox to deferoxamine for myocardial iron removal in patients with transfusion-dependent anemias (CORDELIA) gave the opportunity assess relative prevalence and body distribution overload screened patients.Patients aged ≥ 10 yr from 11 countries were screened. Data summarized descriptively, overall across regions.Among 925 (99.1% β-thalassemia major; 98.5% receiving prior chelation; mean age 19.2 yr), 36.7% had (myocardial T2* ≤ 20 ms), 12.1% low left...
Background The efficacy of cardiac iron chelation in transfusion-dependent patients has been demonstrated one-year prospective trials. Since normalization T2* takes several years, the and safety deferasirox was assessed for two years with β-thalassemia major sub-study EPIC trial.Design Methods Eligible myocardial greater than 5 to less 20 ms received deferasirox, primary endpoint being change from baseline years.Results Baseline severe (>5
This placebo-controlled phase II study evaluated the pharmacodynamics, efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), a fetal globin gene-inducing short-chain fatty acid derivative, administered orally at 15 mg/kg twice daily for 48 weeks in 76 subjects with sickle cell disease (SCD). The median age was 26 years (range: 12-55 years) 37 (49%) were treated previously hydroxycarbamide. Sixty (79%) had Hb SS 16 (21%) S/β(0) thalassemia. terminated after planned interim analysis showed...
In Gaucher disease (GD), deficiency of lysosomal acid β-glucosidase results in a broad phenotypic spectrum that is classified into three types based on the absence (type 1 [GD1]) or presence and severity primary central nervous system involvement 2 [GD2], fulminant neuronopathic form, type 3 [GD3], milder chronic form). Enzyme replacement therapy (ERT) with imiglucerase ameliorates prevents hematological visceral manifestations GD1, but data GD3 are limited to small, single-center series....
Abstract Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload that requires chelation therapy. The chelator deferiprone is frequently used in individuals thalassemia syndromes, but data patients SCD are limited. This open-label study assessed the efficacy and safety of receiving transfusion A total 228 (mean age: 16.9 [range, 3-59] years; 46.9% female) were randomized to receive either oral (n = 152) subcutaneous...