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Total 5940610 results found since Jan 2013.
A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease
This report describes results of voxelotor (500-1000 mg per day) in patients with sickle cell anemia. The study evaluated the safety, tolerability, pharmacokinetic, and pharmacodynamic properties of voxelotor and established proof of concept by improving clinical measures of anemia, hemolysis, and sickling. Thirty-eight patients with SCD received 28 days of voxelotor 500, 700, or 1000 mg per day or placebo; 16 patients received 90 days of voxelotor 700 or 900 mg per day or placebo. Four patients from the 90-day cohort were subsequently enrolled in an extension study and treated with voxelotor 900 mg per day for 6 months. A...
Source: Blood - April 24, 2019 Category: Hematology Authors: Howard, J., Hemmaway, C. J., Telfer, P., Layton, D. M., Porter, J., Awogbade, M., Mant, T., Gretler, D. D., Dufu, K., Hutchaleelaha, A., Patel, M., Siu, V., Dixon, S., Landsman, N., Tonda, M., Lehrer-Graiwer, J. Tags: Sickle Cell Disease, Red Cells, Iron, and Erythropoiesis, Clinical Trials and Observations Source Type: research
Alteration of grey matter volume is associated with pain and quality of life in children with sickle cell disease: Structural brain alteration and association with pain in sickle cell disease
Transl Res. 2021 Aug 18:S1931-5244(21)00194-8. doi: 10.1016/j.trsl.2021.08.004. Online ahead of print.ABSTRACTPain is the most common symptom experienced by patients with sickle cell disease (SCD) and is associated with poor quality of life. We investigated the association between grey matter volume (GMV) and the frequency of pain crises in the preceding 12 months and SCD-specific quality of life (QOL) assessed by the PedsQLTM SCD module in 38 pediatric patients with SCD. Using voxel-based morphometry methodology, high-resolution T1 structural scans were preprocessed using SPM and further analyzed in SPSS. The whole brain ...
Source: Cell Research - August 21, 2021 Category: Cytology Authors: Ying Wang Steven J Hardy Eric Ichesco Pengyue Zhang Richard E Harris Deepika S Darbari Source Type: research
Preferences for Using a Mobile App in Sickle Cell Disease Self-management: Descriptive Qualitative Study
CONCLUSIONS: This study expands our knowledge on the use of mHealth technology to reduce information access barriers pertaining to SCD. The findings can be used to develop a patient-centered, user-friendly mHealth app to facilitate disease self-management, thus increasing access to resources for families of patients with SCD residing in rural communities.PMID:34851295 | DOI:10.2196/28678
Source: Cell Research - December 1, 2021 Category: Cytology Authors: Tilicia L Mayo-Gamble Delores Quasie-Woode Jennifer Cunningham-Erves Margo Rollins David Schlundt Kemberlee Bonnet Velma McBride Murry Source Type: research
An impact evaluation of two modes of care for sickle cell disease crises
Conclusion: Expansion of adult sickle cell disease centers across the nation could lead to considerably better economic outcomes in the form of reduced costs and hospital length of stay in addition to improved clinical outcomes as reported in the existing literature.PMID:35187950 | DOI:10.2217/cer-2021-0257
Source: Cell Research - February 21, 2022 Category: Cytology Authors: Robert Skinner Andrew Breck Dominick Esposito Source Type: research
Application of validated mapping algorithms between generic PedsQL scores and utility values to individuals with sickle cell disease
CONCLUSIONS: The existing mapping algorithms show acceptable construct validity in children and adolescents with SCD. Additional algorithms are needed for adults and for specific SCD comorbidities.PMID:35715626 | DOI:10.1007/s11136-022-03167-2
Source: Cell Research - June 17, 2022 Category: Cytology Authors: Boshen Jiao Jane S Hankins Beth Devine Martha Barton M Bender Anirban Basu Source Type: research
Genetic modulation of anemia severity, hemolysis level, and hospitalization rate in Angolan children with Sickle Cell Anemia
CONCLUSION: This study highlights, for the first time in the Angolan population, the importance of the genetic modifiers of vascular cell adhesion and nitric oxide metabolism in SCA pediatric phenotypic variability.PMID:36097125 | DOI:10.1007/s11033-022-07831-1
Source: Mol Biol Cell - September 12, 2022 Category: Molecular Biology Authors: Isabel Germano Br ígida Santos Mariana Delgadinho Catarina Ginete Pedro Lopes Ana Paula Arez Miguel Brito Paula Faustino Source Type: research
Individuals with Sickle Cell Disease Using SBAR as a Communication Tool: A Pilot Study
CONCLUSIONS: This pilot study supported the usefulness and acceptability of a web-based intervention in training adults with SCD to use SBAR to enhance patient-HCP communication. Enhancing communication may mitigate the barriers that individuals with SCD encounter during care seeking and improve the outcomes. Additional studies with larger samples need to be conducted.PMID:36360702 | DOI:10.3390/ijerph192113817
Source: Cell Research - November 11, 2022 Category: Cytology Authors: Deborah M Jean-Baptiste Maureen Wassef Susan Sullivan Bolyai Coretta Jenerette Source Type: research
A systematic review on hydroxyurea therapy for sickle cell disease in India
CONCLUSIONS: The findings of the present review suggest that there is still insufficient information presently to determine the long-term or major adverse effects on organ damage, fertility as well as pregnancy on the use of HU therapy for SCD. Long-term multi-centric studies are thus required to address these problems.PMID:36629190 | DOI:10.4103/ijmr.ijmr_3447_21
Source: Cell Research - January 11, 2023 Category: Cytology Authors: Apoorva Pandey Harpreet Kaur Sapan Borah Naveen Khargekar Vijay Kumar Karra Tulsi Adhikari Dipty Jain Manisha Madkaikar Source Type: research
Burden of sickle cell disease in tribal students in < em > Maa-Baadi < /em > institutions in southern Rajasthan - A pilot study
CONCLUSIONS: SCD is a significant problem among the tribes of southern Rajasthan, with the highest prevalence among the Garasia tribe. The present study recommends that a structured screening programme targeting the entire tribal population with appropriate counselling as well as providing treatment through the existing health system is the need of the hour.PMID:36629186 | DOI:10.4103/ijmr.ijmr_3195_21
Source: Cell Research - January 11, 2023 Category: Cytology Authors: S S Mohanty Anil Purohit P K Anand Ramesh Kumar Huda Source Type: research
Growing beyond sickle cell disease: A metasynthesis of children, adolescents, and young adult experiences living with sickle cell disease
Res Nurs Health. 2023 Apr 10. doi: 10.1002/nur.22310. Online ahead of print.ABSTRACTChildren, adolescents, and young adults living with sickle cell disease (SCD) often experience an unpredictable and complex disease course. Although there is a growing literature on the lived experience of patients with SCD, qualitative syntheses are lacking. Therefore, a qualitative metasynthesis was conducted to inform care and potential interventions. Noblit and Hare's phases of metaethnographic research were used to guide the synthesis of qualitative data. Data extracted from the identified studies were directly compared through recipro...
Source: Cell Research - April 10, 2023 Category: Cytology Authors: Megan Coco Wendy A Henderson Crystal L Park Angela R Starkweather Source Type: research
Markers of endothelial dysfunction differ between subphenotypes in children with sickle cell disease
Abstract: In adult patients with sickle cell disease two distinct subphenotypes have previously been defined: patients with the viscosity-vaso-occlusion subphenotype (VVO) suffer mainly from vaso-occlusive pain crises and have a relatively high hemoglobin concentration. Patients classified as the hemolysis-endothelial dysfunction subphenotype (HED) suffer from stroke and pulmonary hypertension and have an elevated concentration of lactate dehydrogenase. However, this classification is not possible in children due to low rates of complications. We used laboratory markers to classify children into the two subphenotypes, and ...
Source: Thrombosis Research - October 31, 2013 Category: Hematology Authors: Veronica van der Land, Marjolein Peters, Bart J. Biemond, Harriët Heijboer, Cornelis L. Harteveld, Karin Fijnvandraat Tags: Platelets and Cell Biology Source Type: research
How we treat sickle hepatopathy and liver transplantation in adults
Sickle cell disease (SCD) has evolved into a debilitating disorder with emerging end-organ damage. One of the organs affected is the liver, causing "sickle hepatopathy," an umbrella term for a variety of acute and chronic pathologies. Prevalence of liver dysfunction in SCD is unknown, with estimates of 10%. Dominant etiologies include gallstones, hepatic sequestration, viral hepatitis, and sickle cell intrahepatic cholestasis (SCIC). In addition, causes of liver disease outside SCD must be identified and managed. SCIC is an uncommon, severe subtype, with outcome of its acute form having vastly improved with exchange blood ...
Source: Blood - April 10, 2014 Category: Hematology Authors: Gardner, K., Suddle, A., Kane, P., O'Grady, J., Heaton, N., Bomford, A., Thein, S. L. Tags: Sickle Cell Disease, How I Treat, Free Research Articles, Red Cells, Iron, and Erythropoiesis Source Type: research
HC Activates Endothelial PDE4A Inhibiting Sickle RBC Adhesion Signal Transduction
Vaso-occlusive crises are the main acute complication in sickle cell disease. They are initiated by abnormal adhesion of circulating blood cells to vascular endothelium of the microcirculation. Several interactions involving an intricate network of adhesion molecules have been described between sickle red blood cells and the endothelial vascular wall. We have shown previously that young sickle reticulocytes adhere to resting endothelial cells through the interaction of α4β1 integrin with endothelial Lutheran/basal cell adhesion molecule (Lu/BCAM). In the present work, we investigated the functional impact of endothelial ...
Source: Journal of Biological Chemistry - March 10, 2014 Category: Chemistry Authors: Chaar, V., Laurance, S., Lapoumeroulie, C., Cochet, S., De Grandis, M., Colin, Y., Elion, J., Le Van Kim, C., El Nemer, W. Tags: Cell Biology Source Type: research
Acid sphingomyelinase is activated in sickle cell erythrocytes and contributes to inflammatory microparticle generation in SCD
In this study, we show that RBC membrane alterations in sickle cell disease enhance the activation acid sphingomyelinase by 13%, resulting in increased production and storage of sphingosine (2.6-fold) and S1P (3.5-fold). We also show that acid sphingomyelinase enhances RBC-derived microparticle (MP) generation. These MPs are internalized by myeloid cells and promote proinflammatory cytokine secretion and endothelial cell adhesion, suggesting that potential crosstalk between circulating inflammatory cells and MPs may contribute to the inflammation-rooted pathogenesis of the disease. Treatment with amitriptyline reduces MP g...
Source: Blood - September 18, 2014 Category: Hematology Authors: Awojoodu, A. O., Keegan, P. M., Lane, A. R., Zhang, Y., Lynch, K. R., Platt, M. O., Botchwey, E. A. Tags: Sickle Cell Disease, Red Cells, Iron, and Erythropoiesis Source Type: research
Sickle cell disease increases high mobility group box 1: a novel mechanism of inflammation
High mobility group box 1 (HMGB1) is a chromatin-binding protein that maintains DNA structure. On cellular activation or injury, HMGB1 is released from activated immune cells or necrotic tissues and acts as a damage-associated molecular pattern to activate Toll-like receptor 4 (TLR4). Little is known concerning HMGB1 release and TLR4 activity and their role in the pathology of inflammation of sickle cell disease (SCD). Circulating HMGB1 levels were increased in both humans and mice with SCD compared with controls. Furthermore, sickle plasma increased HMGB1-dependent TLR4 activity compared with control plasma. HMGB1 levels ...
Source: Blood - December 18, 2014 Category: Hematology Authors: Xu, H., Wandersee, N. J., Guo, Y., Jones, D. W., Holzhauer, S. L., Hanson, M. S., Machogu, E., Brousseau, D. C., Hogg, N., Densmore, J. C., Kaul, S., Hillery, C. A., Pritchard, K. A. Tags: Sickle Cell Disease, Free Research Articles, Red Cells, Iron, and Erythropoiesis, Vascular Biology, Brief Reports Source Type: research
