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PubMed Journals Articles About "MAP To Provide Access To Crizanlizumab, For Sickle Cell Disease Patients" - Page: 3 RSS

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Showing "Provide Access Crizanlizumab Sickle Cell Disease Patients" PubMed Articles 51–75 of 77,000+

Dysfunction of the auditory system in sickle cell anemia: a systematic review with meta-analysis.

To describe the occurrence of both peripheral and central auditory system dysfunction in sickle cell anaemia (SCA) patients and discuss the different mechanisms hypothesized to be responsible for these alterations.


Reproductive intentions in mothers of young children with sickle cell disease.

Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy associated with morbidity and mortality. We sought to understand family planning intentions of parents of young children with SCD including the awareness of three reproductive options (adoption, in vitro fertilization with egg/sperm donation [IVFD], in vitro fertilization [IVF] with preimplantation genetic testing [IVF/PGT]) to decrease the risk of having a child with SCD.

Patients With Sickle Cell Disease Between Ages 0 and 20 Years Presenting With Acute Chest Syndrome in the United States.


Early Viral Reactivation Despite Excellent Immune Reconstitution Following Haploidentical Bone Marrow Transplant with Post-Transplant Cytoxan for Sickle Cell Disease.

Haploidentical bone marrow transplant (haplo-BMT) offers near universal donor availability as a curative modality for individuals with severe sickle cell disease (SCD). However, the required intense immunodepletion is associated with increased infectious complications. A paucity of data exists on immune reconstitution following haplo-BMT for SCD.

Vasopressin SNP pain factors and stress in sickle cell disease.

Frequencies of single nucleotide polymorphisms (SNPs) from pain related candidate genes are available for individuals with sickle cell disease (SCD). One of those genes, the arginine vasopressin receptor 1A gene (AVPR1A) and one of its SNPs, rs10877969, has been associated with pain and disability in other pain populations. In patients with SCD, clinical factors such as pain and stress have been associated with increased health care utilization, but it is not known if the presence of the AVPR1A SNP plays a ...

Red blood cells microparticles are associated with hemolysis markers and may contribute to clinical events among sickle cell disease patients.

Microparticles are sub-micron vesicles possessing protein and other materials derived from the plasma membrane of their parent cells, and literature suggests that they may have a role in the pathophysiology and downstream manifestations of sickle cell disease (SCD). The contributions of red blood cells microparticles (RMP) to the pathogenic mechanisms and clinical phenotypes of SCD are largely unknown. There is a controversy as to whether the proportions of intravascular hemolysis (approximately ≤ 30% o...

Dysregulated NO/PDE5 signaling in the sickle cell mouse lower urinary tract: Reversal by oral nitrate therapy.

Nitric oxide (NO) has a critical, but not well understood, influence in the physiology of the lower urinary tract. We evaluated the effect of NO/phosphodiesterase (PDE)5 signaling in voiding dysfunction in the sickle cell disease (SCD) mouse, characterized by low NO bioavailability.

Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

Haematopoietic stem cell transplantation (HSCT) is curative in sickle cell disease (SCD); however, the lack of available matched donors makes this therapy out of reach for the majority of patients with SCD. Alternative donor sources such as haploidentical HSCT expand the donor pool to nearly all patients with SCD, with recent data showing high overall survival, limited toxicities, and effective reduction in acute and chronic graft-versus-host disease (GVHD). Simultaneously, multiple gene therapy strategies ...

Hematologic Conditions: Common Hemoglobinopathies.

Hemoglobinopathies are genetic disorders that lead to abnormal structure of the hemoglobin molecule. Genetic mutation results in major changes in the hemoglobin structure, with dysfunctions related to changes in shape, oxygen-carrying capacity, or ability to clump together, causing obstruction of the vascular system. Sickle cell disease and beta thalassemia major are two common hemoglobinopathies worldwide. However, they occur infrequently in the United States, with approximately 101,000 individuals affecte...

Hemoglobin Alters Vitamin Carrier Uptake and Vitamin D Metabolism in Proximal Tubule Cells: Implications for Sickle Cell Disease.

Kidney disease, including proximal tubule (PT) dysfunction, and vitamin D deficiency are among the most prevalent complications in sickle cell disease (SCD) patients. Although these two comorbidities have never been linked in SCD, the PT is the primary site for activation of vitamin D. Precursor 25(OH)D bound to vitamin D binding protein (DBP) is taken up by PT cells via megalin/cubilin receptors, hydroxylated to the active 1,25(OH)D form, and released into the bloodstream. We tested the hypothesis that cel...

An experimental erythrocyte rigidity index (Ri) and its correlations with Transcranial Doppler velocities (TAMMV), Gosling Pulsatility Index PI, hematocrit, hemoglobin concentration and red cell distribution width (RDW).

Brain artery velocities (Time-Averaged Maximum Mean Velocity, TAMMV) by Transcranial Doppler (TCD), hematocrit, hemoglobin, Red blood cell (RBC) Distribution Width (RDW) and RBC rigidity index (Ri), when reported together with their correlations, provide a accurate and useful diagnostic picture than blood viscosity measurements alone. Additionally, our study included a sixth parameter provided by TCD, the Gosling Pulsatility Index PI, which is an indicator of CBF (Cerebral Blood Flow) resistance. All these ...

Cases of malaria in travellers with sickle cell disease - Chemoprophylaxis is important for this risk group.

Single cell analysis of bone marrow derived CD34+ cells from children with sickle cell disease and thalassemia.

Daily Associations between Child and Parent Psychological Factors and Home Opioid Use in Youth with Sickle Cell Disease.

Opioid analgesics are frequently used in the home setting to manage episodic pain in youth with sickle cell disease (SCD). Given the risk of adverse side effects, including constipation and sedation, understanding factors associated with at-home opioid use is important for maximizing pain relief while minimizing negative side effects.

Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: Rationale and design of a randomized, double-blind, parallel-group, multicenter phase 3 study (HESTIA3).

An unmet need for therapies exists to reduce sickle cell disease (SCD) complications in pediatric patients. Activated platelets contribute to the formation of cellular aggregates during sickling and vaso-occlusive crises (VOCs). Ticagrelor is an oral, direct-acting, and reversible adenosine diphosphate P2Y receptor antagonist that inhibits platelet activation and aggregation. Although ticagrelor was well tolerated in two phase 2 studies in children and young adults with SCD, larger and longer-term treatment...

Sensitivities to Thermal and Mechanical Stimuli: Adults with Sickle Cell Disease Compared to Healthy, Pain-free African American Controls.

Evidence supports but is inconclusive that sensitization contributes to chronic pain in some adults with sickle cell disease (SCD). We determined the prevalence of pain sensitization among adults with SCD pain compared with pain-free healthy adults. In a cross sectional, single session study of 186 African American outpatients with SCD pain (age 18-74 years, 59% female) and 124 healthy age, gender, and race matched control subjects (age 18-69 years, 49% female), we compared responses to standard thermal (Me...

New Option for Sickle Cell Disease.

Renal abnormalities among children with sickle cell conditions in highly resource-limited setting in Ghana.

Sickle cell disease (SCD) is associated with progressive multi-organ failure especially, the brain and kidney and leads to high morbidity and mortality rate. The aim of this study was to determine the prevalence of renal abnormalities among children with SCD. This cross-sectional study recruited 212 sickling positive patients comprising of 96 Hb AS, 48 Hb SC, and 68 Hb SS phenotypes from the Pediatric Unit of Wassa Akropong Government Hospital, Wassa Akropong, Ghana. Early morning urine and venous blood sam...

Familial Haploidentical Stem Cell Transplant in Children and Adolescents With High-Risk Sickle Cell Disease: A Phase 2 Clinical Trial.

Goal-oriented monitoring of cyclosporine is effective for graft-versus-host disease prevention after Hematopoietic Stem Cell Transplantation in Sickle Cell Disease and Thalassemia major.

Graft-versus-host disease (GvHD) is an important challenge and one of the major causes of morbidity and mortality in children after hematopoietic stem cell transplantation (HSCT). Herein, we report our institution's experience of a goal-oriented Bayesian monitoring for cyclosporine (CsA) used alone as GvHD prophylaxis during the post-transplant period in pediatric patients with Thalassemia Major (TM) or Sickle Cell Anemia (SCA) undergoing HLA-matched HSCT. We also studied evolution of chimerism. Twenty-six ...

Portal hypertension evolving from sickled hepatopathy: Could hepatic venous Doppler ultrasound be beneficial in its evaluation?

Sickle cell intrahepatic cholestasis involves sickling within hepatic sinusoids leading to vascular stasis and localized hypoxia resulting in ballooning of the hepatocytes causing a direct back pressure effect with resultant intracanalicular cholestasis. Vascular stasis may ultimately lead to portal hypertension. We proposed to document findings suggestive of portal hypertension evolving from hepatopathy in steady-state sickle cell disease (SCD) patients using hepatic venous Doppler ultrasound.

Translating exercise benefits in sickle cell disease.

Elevated levels of circulating invariant natural killer cell subsets are skewed toward Th2-like phenotype in children with sickle cell disease.

Invariant natural killer T (iNKT) cells are being considered as potential targets for immunotherapeutic strategies in a variety of conditions including sickle cell disease (SCD). However, relatively little is known about the fate of iNKT cell subsets in children with SCD. Herein, quantitative and qualitative analyses of circulating iNKT cell subsets were carried out in 120 children in steady state and 30 healthy controls. Children with SCD displayed significantly elevated levels of circulating iNKT cell sub...

Newborn screening of sickle cell disease and management of care.

Newborn screening of sickle cell disease -NBS- is systematic in the French West Indies and targeted in Metropolitan France to babies from "at risk" countries. The percentages of targeted babies range from 9.1% in Brittany to 73.6% in the Parisian area. NBS is performed on a drop of blood collected by heel stick and put on a blotting paper. Electrophoretic and chromatographic techniques are used to separate and quantify hemoglobin fractions. When a test finds abnormal results, confirmatory tests using a diff...

Study Evaluates Rapid Sickle Cell Disease Test.


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