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Hematopoietic stem cell gene transfer for the treatment of hemoglobin disorders.

Hematopoietic stem cell (HSC)-targeted gene transfer is an attractive approach for the treatment of a number of hematopoietic disorders caused by single gene defects. Indeed, in a series of gene transfer trials for two different primary immunodeficiencies beginning early in this decade, outstanding success has been achieved. Despite generally low levels of engrafted, genetically modified HSCs, these trials were successful because of the marked selective advantage of gene-corrected lymphoid precursors that allowed reconstitution of the immune system. Unlike the immunodeficiencies, this robust l

Do Weekly Surveillance Cultures Contribute to Antibiotic Stewardship and Correlate with Outcome of HSCT in Children? A Multicenter Real-World Experience of 5 Years from the Indian Subcontinent.

The utility of weekly rectal swab surveillance cultures (RSSCs) as a resource to identify gut colonization with extended-spectrum beta-lacta … plantation outcomes. This retrospective analysis of 317 successive first HSCTs performed mainly for hemoglobinopathies was conducted in 3 pediatric bone marrow transplantation centers in the Indian subcontinent between April 2016 and April 2021. Transplantation, infection control, and febrile neutropenia management protocols were identical in the 3 centers. First-li …

Did Osteoblastic Cell Therapy Improve the Prognosis of Pre-fracture Osteonecrosis of the Femoral Head? A Randomized, Controlled Trial.

BACKGROUND: In patients with nontraumatic osteonecrosis of the femoral head (ONFH), implantation of bone marrow aspirate concentrate (BMAC) … -blind trial, patients needed to have an ONFH Stage I or II; we excluded those with traumatic ONFH, hemoglobinopathies and positive serology for hepatitis B, C or HIV. Of those treated surgically for this diagnosis during the study period, 24% (67) agreed to participate in this randomized trial. Hips with pre-fracture ONFH were randomly treated with a core decompres …

Incidence of haemoglobinopathies detected through neonatal screening in the United Arab Emirates.

In January 2002, a pilot programme of neonatal screening for sickle cell disease was launched in the United Arab Emirates (UAE) in 3 districts of Abu Dhabi emirate. This paper reports the incidence of sickle cell diseases, other haemoglobinopathies and haemoglobinopathy carriers over a 12-month period using high performance liquid chromatography as a primary screening method. The overall incidence of sickle cell disease among 22 200 screened neonates was 0.04% (0.07% for UAE citizens and 0.02% for non-UAE citizens). The incidence of sickle cell trait was 1.1% overall (1.5% for UAE citizens and

Oxidative Stress, Antioxidant Capacity, Dyslipidemia and Cardiovascular Risk in Sickle Cell Disease: A Systematic Review and Meta-Analysis.

BACKGROUND: Sickle cell disease is a human hemoglobinopathy associated with high hemolytic capacity. Hemoglobin S (HbS) polymerization is a primary pathophysiological event in sickle cell anemia. Despite numerous studies conducted to understand the pathophysiology of the disease, including oxidative imbalance and cardiovascular risk, questions still arise. This systematic review and meta-analysis aimed to evaluate studies linking oxidative stress, lipid profile, and dyslipidemia in sickle cell patients, predisposing them to atherogenic risk. METHODS: The systematic review of databases and sea

Maternal and Neonatal Outcomes of Iron Deficiency Anemia: A Retrospective Cohort Study.

INTRODUCTION: Understanding the outcomes of anemia in pregnancy is critical. Since no study has been conducted regarding the maternal and neonatal outcomes of iron-deficiency anemia in Hormozgan province of Iran, this study aims to assess the maternal and neonatal outcomes of iron-deficiency anemia in women who gave birth in Hormozgan province from January 2020 to January 2022. METHODS: We retrospectively assessed all singleton pregnant women who gave birth at a tertiary hospital in Bandar Abbas, Hormozgan province, Iran, for two years. We divided all women into iron-deficiency anemic and non

Humoral Immune Reconstitution Kinetics after Allogeneic Hematopoietic Stem Cell Transplantation in Children: A Maturation Block of IgM Memory B Cells May Lead to Impaired Antibody Immune Reconstitution.

Although T cell immune reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been well studied, long-term B cell immune reconstitution remains less characterized. We evaluated humoral immune reconstitution among 71 pediatric allo-HSCT recipients. Although tetanus toxoid antibody levels were normal at 1 year after allo-HSCT, antipolysaccharide carbohydrate antibodies remained persistently low for up to 5 years. While naive B cell counts normalized by 6 months, IgM memory B cell deficiency persisted for up to 2 years (P = .01); switched memory B cell deficiency

Stillbirth is associated with increased risk of long-term maternal renal disease: a nationwide cohort study.

BACKGROUND: Stillbirth is a devastating adverse pregnancy outcome that may occur without any obvious reason or may occur in the context of fetal growth restriction, preeclampsia, or other obstetric complications. There is increasing evidence that women who experience stillbirths are at greater risk of long-term cardiovascular disease, but little is known about their risk of chronic kidney disease and end-stage renal disease. We conducted the largest study to date to investigate the subsequent risk of maternal chronic kidney disease and end-stage renal disease following stillbirth. OBJECTIVE:

Transfusion and the use of blood products.

I have reviewed areas of development in the use of blood and blood products, placing emphasis on the complications of transfusion, particularly transmission of infection. Alloimmunization in relation to transfusion of red cells and platelets has been covered and suggestions for reducing this problem assessed. The potential methods of avoiding the infective complications have been discussed including the screening of blood for infective agents, the virucidal treatment of blood products during the manufacturing process and white cell depletion. The use of recombinant DNA technology to produce co

Erythropoiesis: insights into pathophysiology and treatments in 2017.

Erythropoiesis is a tightly-regulated and complex process originating in the bone marrow from a multipotent stem cell and terminating in a mature, enucleated erythrocyte.Altered red cell production can result from the direct impairment of medullary erythropoiesis, as seen in the thalassemia syndromes, inherited bone marrow failure as well as in the anemia of chronic disease. Alternatively, in disorders such as sickle cell disease (SCD) as well as enzymopathies and membrane defects, medullary erythropoiesis is not, or only minimally, directly impaired. Despite these differences in pathophysiolo

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… These results hold promise for future disease modeling and development of novel therapeutic treatments for all hemoglobinopathies. … These results support the need for further evaluation of givinostat as a new candidate molecule for the treatment of hemoglobinopathies. …

Advancing Hematopoietic Stem Cell Transplantation (HSCT) in Africa: A Pathway to Curing Hematological Disorders.

BackgroundHematopoietic stem cell transplantation (HSCT) offers a curative option for numerous hematological disorders, notably sickle cell disease and thalassemia, which are highly prevalent in Africa. Despite its potential, access to HSCT remains limited due to infrastructural, economic, and socio-cultural challenges across the continent.MethodsThis review synthesizes current literature and reports on the status of HSCT in Africa, analyzing data from existing transplant centers, national registries, and peer-reviewed studies to assess progress, challenges, and prospects for expanding HSCT se

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… Introduction Among all the hematological disorders, hemoglobinopathies and hematological malignancies are a serious public health concern in Africa [ 1 , 2 ]. Africa is home to more than 70% of all hemoglobinopathies in the world. … Blood transfusions, infusion therapy, and drugs are among the available treatments for hemoglobinopathies [ 3 ]. …

Mandibular Radiographic Assessment in Sickle Cell Disease: A Systematic Review of Radiomorphometric Indices and Fractal Dimension.

AIM: This systematic review aimed to investigate the association between sickle cell disease (SCD) and reduced bone mineral density (BMD) using radiomorphometric indices (RMI) and fractal dimension analysis (FDA) on dental radiographic images. METHODS AND RESULTS: Observational studies evaluating BMD in individuals with SCD through RMI or FDA were included. A comprehensive search was conducted across six databases and grey literature in March 2025. Risk of bias was evaluated using the Joanna Briggs Institute Critical Appraisal Tool for cross-sectional studies, and certainty of the evidence wa

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… Reduced bone mineral density (BMD) is a well‐recognized complication in patients with hemoglobinopathies such as SCD, with prevalence rates estimated at 30%–50% in children and 60%–80% in adults [ 13 , 14 ]. … A., “Evaluation of the Effects of Hemoglobinopathies on the Mandible With Fractal Dimension Analysis,” Nigerian Journal of Clinical Practice 22 (2019): 1435–1440. 27. Temur K. …

Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment.

Background-This review provides a comprehensive overview of rare anemias, emphasizing their hereditary and acquired causes, diagnostic advancements, and evolving treatment strategies. It outlines the significance of rare anemias within public health, historical challenges in recognition and treatment, and the role of European initiatives like ENERCA and EuroBloodNet in advancing care. Content-This document discusses diagnostic technologies like next-generation sequencing and the impact of artificial intelligence, alongside the promising avenues of gene therapy, targeted drug treatments, and st

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… Hemoglobinopathies Hemoglobinopathies are genetic defects of the hemoglobin associated with chronic or acute anemia and other complications. They are classified into structural hemoglobinopathies and thalassemia’s. … In Europe, hemoglobinopathies are still rare diseases, but their prevalence has significantly increased in many countries due to mobility and migration flows. …