A5087536541- Hemoglobinopathies and Related Disorders
- Prenatal Screening and Diagnostics
- Hematopoietic Stem Cell Transplantation
- Acute Myeloid Leukemia Research
- Iron Metabolism and Disorders
- Blood groups and transfusion
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Immune Cell Function and Interaction
- Erythrocyte Function and Pathophysiology
- Transplantation: Methods and Outcomes
- Folate and B Vitamins Research
- Bone and Joint Diseases
- T-cell and B-cell Immunology
- Acute Lymphoblastic Leukemia research
- Epigenetics and DNA Methylation
- Chronic Myeloid Leukemia Treatments
- Hepatitis B Virus Studies
- Liver Disease Diagnosis and Treatment
- Parvovirus B19 Infection Studies
- MicroRNA in disease regulation
- Virus-based gene therapy research
- CRISPR and Genetic Engineering
- Sarcoma Diagnosis and Treatment
- Pregnancy and preeclampsia studies
- Pharmacological Effects and Toxicity Studies
National Institutes of Health
2016-2025
National Heart Lung and Blood Institute
2016-2025
Cellular Therapeutics (United Kingdom)
2025
Liverpool John Moores University
2024
Government of the United States of America
2019-2022
National Institute of Diabetes and Digestive and Kidney Diseases
2002-2021
Boston University
2019
Seattle University
2019
Vanderbilt University Medical Center
2016
National Cancer Institute
2009
Myeloablative allogeneic hematopoietic stem-cell transplantation is curative in children with sickle cell disease, but adults the procedure unduly toxic. Graft rejection and graft-versus-host disease (GVHD) are additional barriers to its success. We performed nonmyeloablative disease.Ten (age range, 16 45 years) severe underwent CD34+ peripheral-blood stem cells, mobilized by granulocyte colony-stimulating factor (G-CSF), which were obtained from HLA-matched siblings. The patients received...
<h3>Importance</h3> Myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) is curative for children with severe sickle disease, but toxicity may be prohibitive adults. Nonmyeloablative has been attempted degrees of preparative regimen intensity, graft rejection and graft-vs-host disease remain significant. <h3>Objective</h3> To determine the efficacy, safety, outcome on end-organ function this low-intensity phenotype or without thalassemia. <h3>Design, Setting,...
Sickle cell disease (SCD) is associated with early mortality. We sought to determine the incidence, cause, and risk factors for death in an adult population of patients SCD. All aged >/=18 years seen at Adult Cell Center Duke University Medical between January 2000 April 2005 were enrolled. Forty-three (21 males 22 females) died during study period. The median age survival was 39 females (95% CI: 34-56), 40 34-48), overall 35-48). Cardiac causes accounted 25.6% (11/43 patients); pulmonary,...
Peripheral blood stem cell transplantation (PBSCT) offers a curative option for sickle disease (SCD). Although HLA-matched sibling is promising, the vast majority of patients lack such donor. We sought to develop novel nonmyeloablative HLA-haploidentical PBSCT approach that could safely be used with severe organ damage. Based on findings in our preclinical model, we developed phase 1/2 trial using alemtuzumab, 400 cGy total body irradiation, and escalating doses posttransplant...
Abstract Background: Sickle cell disease (SCD) is a life-limiting inherited hemoglobinopathy that results in significant complications and affects quality of life. Hematopoietic stem transplantation (HSCT) currently the only curative intervention for SCD; however, guidelines are needed to inform how apply HSCT clinical practice. Objective: These evidence-based American Society Hematology (ASH) intended support patients, clinicians, health professionals their decisions about SCD. Methods: The...
To report the incidence and risk factors for secondary neoplasm after transplantation sickle cell disease.
Mixed donor chimerism after hematopoietic cell transplantation for sickle disease (SCD) can result in resolution of symptoms, but symptoms recur when is critically low. The relationship between chimerism, hemoglobin S (HbS) level, and symptomatic was correlated retrospectively 95 patients who had reports available at day 100 1 2 years transplantation. Recurrent defined as recurrence vaso-occlusive crises, acute chest syndrome, stroke, and/or HbS levels > 50%. Thirty-five maintained full...
Abstract Proteinuria in children with sickle cell anemia (SCA) is an early sign of nephropathy, and portends the development nephrotic syndrome chronic renal failure. Enalapril has been shown to reduce proteinuria adult patients SCA, but potential benefits hydroxyurea this clinical setting have not reported. A single institution retrospective analysis was performed. Children nephropathy were identified, laboratory effects enalapril therapy evaluated substantial proteinuria. Three developed...
Background Adults with sickle cell anemia (HbSS) are inconsistently treated hydroxyurea. Objectives We retrospectively evaluated the effects of elevating fetal hemoglobin hydroxyurea on organ damage and survival in patients enrolled our screening study between 2001 2010. Methods An electronic medical record facilitated development a database for comparison parameters based exposure dose. This is registered ClinicalTrials.gov, number NCT00011648. Results Three hundred eighty-three adults...
Abstract Sickle cell nephropathy results in chronic kidney disease (CKD), which is associated with significant morbidity and mortality sickle anemia (SCA). Albuminuria an early manifestation of nephropathy; however, little known about progression albuminuria or its correlation glomerular filtration rate (GFR) decline CKD. We studied 303 SCA participants a prospective, multicenter, longitudinal study. collected steady-state urine serum samples yearly assessed albumin/creatinine ratio (ACR),...
Decreased red cell deformability is characteristic of several disorders. In some cases, the extent defective can predict severity disease or occurrence serious complications. Ektacytometry uses laser diffraction viscometry to measure blood cells subject either increasing shear stress an osmotic gradient at a constant value applied stress. However, direct measurements are difficult interpret when measuring heterogenous that characterized by presence both rigid and deformable cells. This due...
Nephropathy is a common and progressive complication of sickle cell anemia (SCA). In SCA mice, we found that hyperangiotensinemia in the absence hypertension underlies nephropathy, its downregulation by losartan, an angiotensin-II-receptor-1 blocker, reduced albuminuria progression nephropathy. Therefore, performed phase-2 trial oral given for 6 months, to explore whether it children adults with SCA. Participants were allocated groups defined class baseline urinary albumin-to-creatinine...
Human cytomegalovirus (CMV) infection and disease remains a significant cause of morbidity mortality for hematopoietic cell transplantation (HCT) recipients. Disruption or weak reconstitution virus-specific cellular immune function, such as with certain HCT approaches, poses risk CMV-related complications. The incidence factors CMV the nature were evaluated retrospectively among 356 consecutive recipients transplanted at National Institutes Health using all graft sources, including bone...
Abstract Recent studies suggest that plerixafor mobilization and apheresis in patients with sickle cell disease (SCD) is safe can allow collection of sufficient CD34+ hematopoietic stem (HSC) for clinical gene therapy applications. However, the quantities plerixafor-mobilized cells vary between different SCD unknown reasons. Twenty-three participants underwent followed by apheresis, processing, HSC enrichment under a phase 1 safety efficacy study conducted at 2 institutions. Linear...