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"Sickle Cell Anemia"

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https://www.readbyqxmd.com/read/28821470/design-of-the-drepagreffe-trial-a-prospective-controlled-multicenter-study-evaluating-the-benefit-of-genoidentical-hematopoietic-stem-cell-transplantation-over-chronic-transfusion-in-sickle-cell-anemia-children-detected-to-be-at-risk-of-stroke-by-transcranial
#1
Sylvie Chevret, Suzanne Verlhac, Elisabeth Ducros-Miralles, Jean-Hugues Dalle, Regis Peffault de Latour, Mariane de Montalembert, Malika Benkerrou, Corinne Pondarré, Isabelle Thuret, Corinne Guitton, Emmanuelle Lesprit, Maryse Etienne-Julan, Gisèle Elana, Jean-Pierre Vannier, Patrick Lutz, Bénédicte Neven, Claire Galambrun, Catherine Paillard, Camille Runel, Charlotte Jubert, Cécile Arnaud, Annie Kamdem, Valentine Brousse, Florence Missud, Marie Petras, Lydia Doumdo-Divialle, Claire Berger, Françoise Fréard, Olivier Taieb, Elise Drain, Monique Elmaleh, Manuela Vasile, Yacine Khelif, Myriam Bernaudin, Philippe Chadebech, France Pirenne, Gérard Socié, Françoise Bernaudin
BACKGROUND: Children with sickle cell anemia (SCA) have an 11% risk of stroke by the age of 18. Chronic transfusion applied in patients detected to be at risk by transcranial Doppler allows a significant reduction of stroke risk. However, chronic transfusion exposes to several adverse events, including alloimmunization and iron overload, and is not curative. Hematopoietic stem cell transplantation allows termination of the transfusion program, but its benefit has not been demonstrated...
August 15, 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28814957/induced-pluripotent-stem-cell-for-the-study-and-treatment-of-sickle-cell-anemia
#2
Luiza Cunha Junqueira Reis, Virgínia Picanço-Castro, Bárbara Cristina Martins Fernandes Paes, Olívia Ambrozini Pereira, Isabela Gerdes Gyuricza, Fabiano Tófoli de Araújo, Mariana Morato-Marques, Lílian Figueiredo Moreira, Everton de Brito Oliveira Costa, Tálita Pollyanna Moreira Dos Santos, Dimas Tadeu Covas, Lygia da Veiga Pereira Carramaschi, Elisa Maria de Sousa Russo
Sickle cell anemia (SCA) is a monogenic disease of high mortality, affecting millions of people worldwide. There is no broad, effective, and safe definitive treatment for SCA, so the palliative treatments are the most used. The establishment of an in vitro model allows better understanding of how the disease occurs, besides allowing the development of more effective tests and treatments. In this context, iPSC technology is a powerful tool for basic research and disease modeling, and a promise for finding and screening more effective and safe drugs, besides the possibility of use in regenerative medicine...
2017: Stem Cells International
https://www.readbyqxmd.com/read/28807656/opportunities-for-model-based-precision-dosing-in-the-treatment-of-sickle-cell-anemia
#3
Min Dong, Tomoyuki Mizuno, Alexander A Vinks
Hydroxyurea is the primary pharmacotherapy to prevent complications of sickle cell anemia (SCA). Accumulated clinical experience across multiple age ranges has suggested that the use of an individualized maximum tolerated dose (MTD) will achieve optimal benefit of hydroxyurea treatment. However, the current empirical and trial-and-error approach for dose escalation often results in a lengthy titration process and is not strictly implemented in many clinics. Opportunities exist for pharmacokinetics model-based precision dosing of hydroxyurea to quickly achieve individual MTD...
August 9, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28800727/a-phased-snp-based-classification-of-sickle-cell-anemia-hbb-haplotypes
#4
Elmutaz M Shaikho, John J Farrell, Abdulrahman Alsultan, Hatem Qutub, Amein K Al-Ali, Maria Stella Figueiredo, David H K Chui, Lindsay A Farrer, George J Murphy, Gustavo Mostoslavsky, Paola Sebastiani, Martin H Steinberg
BACKGROUND: Sickle cell anemia causes severe complications and premature death. Five common β-globin gene cluster haplotypes are each associated with characteristic fetal hemoglobin (HbF) levels. As HbF is the major modulator of disease severity, classifying patients according to haplotype is useful. The first method of haplotype classification used restriction fragment length polymorphisms (RFLPs) to detect single nucleotide polymorphisms (SNPs) in the β-globin gene cluster. This is labor intensive, and error prone...
August 11, 2017: BMC Genomics
https://www.readbyqxmd.com/read/28783617/an-age-dependent-response-to-hydroxyurea-in-pediatric-sickle-cell-anemia-patients-with-alpha-thalassemia-trait
#5
Lisa Figueiredo, Kerry Morrone, Catherine Wei, Karen Ireland, Hillel W Cohen, Catherine Driscoll, Deepa Manwani
Hydroxyurea (HU) is a key drug therapy for individuals with sickle cell anemia (SCA), yet its clinical and hematologic responses can be variable. Various studies have reported the role of α-thalassemia as one of the most prevalent heritable traits that may modify HU response. We provide data from 62 pediatric and adolescent patients with SCA, 26 with co-inherited α-thalassemia trait. Our data suggest that altered hematologic and clinical responses to HU therapy are noted in adolescent SCA individuals with co-inherited α-thalassemia trait...
July 31, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28781827/deferasirox-associated-with-liver-failure-and-death-in-a-sickle-cell-anemia-patient-homozygous-for-the-1774delg-polymorphism-in-the-abcc2-gene
#6
Caroline C B Braga, Bruno Deltreggia Benites, Dulcineia M de Albuquerque, Marisa C Alvarez, Tiago Seva-Pereira, Bruno K L Duarte, Fernando F Costa, Simone C O Gilli, Sara T O Saad
This manuscript describes the case of a patient with sickle cell anemia who died of fulminant hepatitis after therapy with the iron chelator Deferasirox. The patient was homozygous for the -1774delG polymorphism in the Abcc2 gene, which raises the concern about the use of hepatotoxic drugs in this specific context.
August 2017: Clinical Case Reports
https://www.readbyqxmd.com/read/28766840/high-birth-prevalence-of-sickle-cell-disease-in-northwestern-tanzania
#7
Emmanuela E Ambrose, Julie Makani, Neema Chami, Tulla Masoza, Rogatus Kabyemera, Robert N Peck, Erasmus Kamugisha, Alphaxard Manjurano, Neema Kayange, Luke R Smart
BACKGROUND: Worldwide, hemoglobinopathies affect millions of children. Identification of hemoglobin disorders in most sub-Saharan African countries is delayed until clinical signs of the disease are present. Limited studies have been conducted to understand their prevalence and clinical presentation among newborns in resource-limited settings. METHODOLOGY: This was a prospective cohort study. Newborns (aged 0-7 days) at two hospitals in Northwestern Tanzania were enrolled and followed prospectively for 6 months...
August 2, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28766838/transfusion-transmitted-babesiosis-leading-to-severe-hemolysis-in-two-patients-with-sickle-cell-anemia
#8
Kristine Karkoska, James Louie, Abena O Appiah-Kubi, Lawrence Wolfe, Lorry Rubin, Sujatha Rajan, Banu Aygun
The intracellular parasites Babesia microti and Babesia duncani can be transmitted by blood transfusion and cause severe life-threatening hemolytic anemia in high-risk patients, including those with sickle cell disease. The rarity of the diagnosis, as well as its similar clinical presentation to delayed hemolytic transfusion reaction, may lead to a delay in diagnosis, as well as inappropriate treatment with steroids or other immunosuppressive agents. The morbidity caused by this disease in especially vulnerable populations justifies the need for a universal blood-screening program in endemic areas...
August 2, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28759962/effects-of-hydroxyurea-on-blood-rheology-in-sickle-cell-anemia-a-two-years-follow-up-study
#9
Nathalie Lemonne, Berenike Möckesch, Keyne Charlot, Yohann Garnier, Xavier Waltz, Yann Lamarre, Sophie Antoine-Jonville, Maryse Etienne-Julan, Marie-Dominique Hardy-Dessources, Marc Romana, Philippe Connes
The aim of the present study was to test the effects of hydroxyurea (HU) therapy on clinical, hematological and hemorheological parameters in adult patients with sickle cell anemia (SCA). Hematological and hemorheological parameters were measured in 28 SCA patients before HU therapy (i.e., baseline) and at 6, 12 and 24 months of treatment. RBC deformability was determined by ektacytometry at 30 Pa. RBC aggregation properties were investigated by light-backscatter method. Blood viscosity was measured at 225 s-1 by a cone-plate viscometer...
July 25, 2017: Clinical Hemorheology and Microcirculation
https://www.readbyqxmd.com/read/28748763/induced-pluripotent-stem-cell-technology-a-paradigm-shift-in-medical-science-for-drug-screening-and-disease-modeling
#10
Meera Nair, Sardul Singh Sandhu, Anil Kumar Sharma
BACKGROUND: Induced Pluripotent Stem Cell (IPSC) Technology is the most advanced research as it offers an attractive alternative for establishing patient-specific IPSCs to recapitulate phenotypes of not only monogenic diseases (viz. Thalassaemia, Sickle cell anemia, Haemophilia, Tay-Sachs disease), but also late-onset polygenic diseases (viz. Parkinson's disease, Alzheimer's disease, schizophrenia). Over the hindsight, numerous studies of the past and current scientists have led to the production, maturation and understanding of induced pluripotent stem cell technology and its use in basic and clinical research...
July 27, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28746470/relationship-between-ventilation-heterogeneity-and-exercise-intolerance-in-adults-with-sickle-cell-anemia
#11
A J Lopes, C L Marinho, U D Alves, C E A Gonçalves, P O Silva, E C Botelho, R Bedirian, A R Soares, M C P Maioli
Sickle cell anemia (SCA) causes dysfunction of multiple organs, with pulmonary involvement as a major cause of mortality. Recently, there has been growing interest in the nitrogen single-breath washout (N2SBW) test, which is able to detect ventilation heterogeneity and small airway disease when the results of other pulmonary function tests (PFTs) are still normal. Thus, the objectives of the present study were to assess the heterogeneity in the ventilation distribution in adults with SCA and to determine the association between the ventilation distribution and the clinical, cardiovascular, and radiological findings...
July 20, 2017: Brazilian Journal of Medical and Biological Research, Revista Brasileira de Pesquisas Médicas e Biológicas
https://www.readbyqxmd.com/read/28744165/asymptomatic-multinodular-splenoma-splenic-hamartoma-in-a-child-with-sickle-cell-anemia
#12
Narcisse Elenga, Sylvain Labbé, Nicolas Leduc, Anicet Sika, Emma Cuadro, Laurence Long, Falucar Njuieyon, Rémi Kom-Tchameni, Thierry Basset
Splenoma is a rare and benign malformation usually fortuitously diagnosed during imaging, surgery or, unfortunately, at autopsy. Although splenoma was first described in 1861, its association with hematological pathology is a very unusual condition in children. We report the case of an asymptomatic splenoma in an 8-year-old boy with sickle cell anemia, whose diagnosis was confirmed after conventional splenectomy.
2017: International Medical Case Reports Journal
https://www.readbyqxmd.com/read/28743191/successful-utilization-of-an-electronic-pain-diary-in-a-multinational-phase-3-interventional-study-of-pediatric-sickle-cell-anemia
#13
Lori E Heath, Matthew M Heeney, Carolyn C Hoppe, Samuel Adjei, Tsiri Agbenyega, Mohamed Badr, Nicoletta Masera, Chunmei Zhou, Patricia B Brown, Joseph A Jakubowski, Carlton Dampier
BACKGROUND/AIMS: Patients with sickle cell anemia can experience recurrent pain episodes, which affect quality of life. The reported prevalence of pain is higher in studies using patient diaries than in healthcare facility utilization data. Determining Effects of Platelet Inhibition on Vaso-Occlusive Events was a multinational study that assessed the efficacy and safety of prasugrel in reducing the rate of vaso-occlusive events in children with sickle cell anemia (NCT01794000) and included an electronic patient-reported outcome diary to record pain occurrence...
July 1, 2017: Clinical Trials: Journal of the Society for Clinical Trials
https://www.readbyqxmd.com/read/28736939/fetal-hemoglobin-in-sickle-cell-anemia-the-arab-indian-haplotype-and-new-therapeutic-agents
#14
REVIEW
Alawi H Habara, Elmutaz M Shaikho, Martin H Steinberg
Fetal hemoglobin (HbF) has well-known tempering effects on the symptoms of sickle cell disease and its levels vary among patients with different haplotypes of the sickle hemoglobin gene. Compared with sickle cell anemia haplotypes found in patients of African descent, HbF levels in Saudi and Indian patients with the Arab-Indian (AI) haplotype exceed that in any other haplotype by nearly twofold. Genetic association studies have identified some loci associated with high HbF in the AI haplotype but these observations require functional confirmation...
July 24, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28730452/transcranial-color-doppler-in-stroke-free-adult-patients-with-sickle-cell-disease
#15
COMPARATIVE STUDY
G Graziadei, F M Casoni, F Annoni, I Cortinovis, P Ridolfi, I Gandolfi, A Marcon, E Di Pierro, M D Cappellini
The threshold velocity ≥200 cm/s at transcranial Doppler (TCD) evaluation is a useful cut-off for preventing the stroke (STOP trial) in pediatric patients with sickle cell disease (SCD), term including different types of sickle genotypes. Scanty data are available for adult SCD patients. We compared intracranial blood flow velocities between adult SCD patients and controls using transcranial color Doppler (TCCD), measuring the peak of systolic velocity (PSV) with the insonation angle correction and the pulsatility index (PI), an indicator of endothelial elasticity...
September 2017: Annals of Hematology
https://www.readbyqxmd.com/read/28705443/-impact-of-sickle-cell-trait-on-arterial-stiffness-in-african-subjects
#16
V Ouédraogo, R Soleti, I Signolet, M Diaw, M Hallab, A Samb, R Andriantsitohaina, A Ba, G Lefthériotis
Sickle cell trait (SCT) is the benign condition of sickle cell disease. Often asymptomatic, the carriers of the sickle cell trait have hemorheological disturbances with increased oxidative stress compared to healthy subjects. These disturbances can lead to structural and functional changes in large vessels. The aim of the study was to measure arterial stiffness, an independent marker of subclinical atherosclerosis, SCT carriers compared to sickle cell anemia (SCA) subjects. Nine SCT carriers aged 32±9 years (7 men) were compared to 14 SCA subjects aged 29±9 years (2 men) and 22 control subjects aged 34±9 years (11 men) recruited by the National blood transfusion center (CNTS) in Dakar (Senegal)...
February 2017: J Med Vasc
https://www.readbyqxmd.com/read/28700924/mesoscopic-adaptive-resolution-scheme-toward-understanding-of-interactions-between-sickle-cell-fibers
#17
Lu Lu, He Li, Xin Bian, Xuejin Li, George Em Karniadakis
Understanding of intracellular polymerization of sickle hemoglobin (HbS) and subsequent interaction with the membrane of a red blood cell (RBC) is important to predict the altered morphologies and mechanical properties of sickle RBCs in sickle cell anemia. However, modeling the integrated processes of HbS nucleation, polymerization, HbS fiber interaction, and subsequent distortion of RBCs is challenging as they occur at multispatial scales, ranging from nanometers to micrometers. To make progress toward simulating the integrated processes, we propose a hybrid HbS fiber model, which couples fine-grained and coarse-grained HbS fiber models through a mesoscopic adaptive resolution scheme (MARS)...
July 11, 2017: Biophysical Journal
https://www.readbyqxmd.com/read/28699284/a-monocyte-tnf-endothelial-activation-axis-in-sickle-transgenic-mice-therapeutic-benefit-from-tnf-blockade
#18
Anna Solovey, Arif Somani, John D Belcher, Liming Milbauer, Lucile Vincent, Rafal Pawlinski, Karl A Nath, Robert J Kelm, Nigel Mackman, M Gerard O'Sullivan, Kalpna Gupta, Gregory M Vercellotti, Robert P Hebbel
Elaboration of tumor necrosis factor (TNF) is a very early event in development of ischemia/reperfusion injury pathophysiology. Therefore, TNF may be a prominent mediator of endothelial cell and vascular wall dysfunction in sickle cell anemia, a hypothesis we addressed using NY1DD, S+S(Antilles) , and SS-BERK sickle transgenic mice. Transfusion experiments revealed participation of abnormally activated blood monocytes exerting an endothelial activating effect, dependent upon Egr-1 in both vessel wall and blood cells, and upon NFκB(p50) in a blood cell only...
July 11, 2017: American Journal of Hematology
https://www.readbyqxmd.com/read/28687959/stem-cell-therapy-for-fanconi-anemia
#19
Qing-Shuo Zhang
Stem cell therapy is the administration of stem cells to a patient to treat or prevent a disease. Since stem cells possess the long-term self-renewal capacity and provide daughter cells that differentiate into the specialized cells of each tissue, stem cell therapy will theoretically improve the disease condition for the lifetime of the patient. As the most widely used stem cell therapy, bone marrow transplantation is the treatment of choice for many kinds of blood disorders, including anemias, leukemias, lymphomas, and rare immunodeficiency diseases...
July 8, 2017: Advances in Experimental Medicine and Biology
https://www.readbyqxmd.com/read/28685705/chronic-active-parietal-osteomyelitis-due-to-salmonella-typhi-in-a-patient-with-sickle-cell-anemia
#20
Ahmad Antar, George Karam, Maurice Kfoury, Nadim El-Majzoub
No abstract text is available yet for this article.
July 4, 2017: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
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