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Pulmonary Hypertension in Sickle Cell Anemia


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361. Screening for Alpha Thalassemia in Healthy Volunteers

thalassemia trait is when someone has only two out of the normal four alpha globin genes. In some people, they lead to no symptoms. Others have changes that lead to disease, including mild anemia. Researchers want to learn more about alpha thalassemia and blood vessels. This may allow them to develop new treatments for blood diseases such as sickle cell disease. Objective: To better understand how alpha globin deletions in healthy people affect blood vessels. Eligibility: Healthy volunteers ages 18 50 who (...) ulceration,pulmonary hypertension, and cardiac hypertrophy are prevented, delayed or reduced by inheritance of one of more deletions of the alpha globin genes. Our long-term research goal is to understand how deletions of alpha globin protect against the vascular complications of sickle cell disease. Deletions of alpha globin are common and found in approximately 5% of the world s population.They are especially common among Africans and people of African ancestry, as well as in India, China

2016 Clinical Trials

362. HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

): Indira Sahdev, Northwell Health Study Details Study Description Go to Brief Summary: This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a routine stem cell transplant in order to assess whether or not it is as effective as myeloablative high dose chemotherapy and transplant. Condition or disease Intervention/treatment Phase (...) of TRM. In addition to providing a less toxic regimen, the reduced intensity chemotherapy preparative regimen also remains immunosuppressive enough to allow donor engraftment. Recent report of non-myeloablative regimens which resulted in engraftment of allogeneic stem cell in hematological malignancies raises the possibility that this conditioning regimen might be useful in achieving engraftment in non hematological disorder. In an effort to achieve stable engraftment with any suitable donor stem

2015 Clinical Trials

363. A Phase 2a, Single-Dose, Double-Blind, Placebo-Controlled, 2-Way Crossover Study Using Penile Plethysmography to Evaluate the Efficacy and Safety of SST-6006, a Topical Sildenafil Cream (5% w/w), Compared to Placebo in the Treatment of Erectile Dysfunctio

containing medication(s). 12. Subject has retinitis pigmentosa or sickle cell anemia or related anemias, even if the subject feels clinically well at the time of screening. Subjects with retinitis pigmentosa will be identified by specifically asking whether they have the condition, if there are visual signs and symptoms of the condition (including questioning subjects as to whether they have difficulty seeing at night or in low light, and if they have any visual field deficits that indicate a loss (...) , or life-threatening arrhythmia within 6 months prior to Visit 1; a history of coronary disease causing angina; or congestive heart failure requiring medical intervention. 7. Subject has uncontrolled hypertension or untreated hypertension. 8. Subject has a history of orthostatic hypotension or orthostatic hypotension. 9. Subject is unable to walk up and down two flights of stairs briskly without chest pain. 10. Subject is using alpha blockers. 11. Subject is using or has possession of nitrate

2015 Clinical Trials

364. Sildenafil for the Treatment of Lymphatic Malformations

diagnosis of the soft tissue tumor as LM not clinically certain. Participating in another clinical study which may interfere. Has a history of priapism or is diagnosed with sickle cell anemia or any other disorder which may predispose to priapism. Contacts and Locations Go to Information from the National Library of Medicine To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor. Please refer to this study by its (...) with sildenafil oral therapy for pulmonary arterial hypertension. We have subsequently evaluated additional subjects who improved with sildenafil. The goal of this clinical research trial is to document the benefit or absence of benefit of sildenafil therapy for LMs and identify which type of patient will benefit from sildenafil. This study is a double-blind placebo-controlled trial which involves precise documentation of volume changes associated with therapy or placebo by using MRI segmentation techniques

2015 Clinical Trials

365. Nitrite, Isoquercetin and Endothelial Dysfunction (NICE) Trial

infection (i.e. systemic or osteomyelitis) Class III or IV heart failure History of hemolytic anemia including sickle cell disease Hemoglobin <10 History of chronic obstructive pulmonary disease (COPD) Have a positive screen for glucose-6-phosphate dehydrogenase (G6PD) deficiency at screening Involvement in other clinical trials Current alcohol or other substance abuse Current smokers Unwillingness to stop flavonoid supplementation Unwillingness to stop nitrate and/or nitrite supplementation Contacts (...) /min/1.73 m2) Blood pressure [ Time Frame: 3 months ] Blood pressure (mm Hg) blood chemistry panel [ Time Frame: 3 months ] blood glucose, urea nitrogen, creatinine, calcium, phosphorus, bicarbonate Lipid panel [ Time Frame: 3 months ] cholesterol, triglycerides, LDL-cholesterol, HDL-cholesterol complete blood count [ Time Frame: 3 months ] complete blood count adverse events [ Time Frame: 3 months ] adverse events nitrate [ Time Frame: 3 months ] Plasma nitrate levels urinary albumin-to-creatinine

2015 Clinical Trials

366. Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements

to comply with the procedures in the study protocol. Exclusion Criteria: Acute episodes (infection, vaso-occlusive crises (VOC), acute chest syndrome (ACS), stroke, priapism, splenic sequestration) in the past one month before enrolment. Previous stroke, and other co-morbid diseases like Essential Hypertension, Cardiomyopathy and Heart failure, Diabetes, Chronic Renal Failure. Patients with a history of adverse reaction to omega-3 fatty acid supplementation. Blood transfusion in the previous 3 months (...) 17, 2015 Last Update Posted: August 17, 2015 Last Verified: August 2015 Keywords provided by Salam Alkindi, Sultan Qaboos University: Prevention of Vaso Occlusive Painful Crisis Additional relevant MeSH terms: Layout table for MeSH terms Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Hydroxyurea Antineoplastic Agents Antisickling Agents Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action

2015 Clinical Trials

367. A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects

Therapeutics Information provided by (Responsible Party): Global Blood Therapeutics Study Details Study Description Go to Brief Summary: The purpose of this study is to evaluate the relative bioavailability of a single 300 mg dose of GBT440 administered as a high strength (1 × 300 mg) capsule versus a low strength (3 × 100 mg) capsule formulation in healthy fasted subjects. Condition or disease Intervention/treatment Phase Sickle Cell Disease Drug: GBT440 Phase 1 Study Design Go to Layout table for study (...) , dermatological, hepatic, renal,hematological, pulmonary, cardiovascular, gastrointestinal, neurological, or psychiatric disorder History of hypersensitivity or allergy to drugs, foods, or other substances History or presence of abnormal electrocardiogram or hypertension History of alcohol abuse, illicit drug use, significant mental illness, physical dependence to any opioid, or any history of drug abuse or addiction within 1 year of screening Participated in another clinical trial of an investigational drug

2015 Clinical Trials

368. Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects

Director: Carla Washington, PhD Global Blood Therapeutics More Information Go to Layout table for additonal information Responsible Party: Global Blood Therapeutics Identifier: Other Study ID Numbers: GBT440-003 First Posted: October 5, 2015 Last Update Posted: April 12, 2017 Last Verified: April 2017 Keywords provided by Global Blood Therapeutics: anemia, sickle cell Additional relevant MeSH terms: Layout table for MeSH terms Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia (...) : April 12, 2017 Sponsor: Global Blood Therapeutics Information provided by (Responsible Party): Global Blood Therapeutics Study Details Study Description Go to Brief Summary: The purpose of this study to evaluate the effect of concomitant administration of GBT440 on caffeine (a CYP1A2 probe substrate), S warfarin (a CYP2C9 probe substrate), omeprazole (a CYP2C19 probe substrate), and midazolam (a CYP3A4 probe substrate) plasma concentrations. Condition or disease Intervention/treatment Phase Sickle

2015 Clinical Trials

369. A RCT of CenteringPregnancy on Birth Outcomes

preclude prenatal care provision by nurse practitioners or participation in group care Pregestational diabetes, Severe chronic hypertension requiring medication, Morbid Obesity with BMI >49.99 Renal disease with baseline proteinuria >1g/24 hours Any disease requiring chronic immunosuppression (SLE, solid organ transplant) Active pulmonary tuberculosis Sickle cell anemia Human Immunodeficiency Virus Infection Other medical conditions that would exclude women from group care at the discretion of the PI (...) diagnosed with overt diabetes If PG at 1 h after load is ≥140 mg/dL (7.8 mmol/L), proceed to 100-g OGTT (Step 2), performed while patient is fasting GDM diagnosis made when two or more PG levels meet or exceed: Fasting: 95 mg/dL or 105 mg/dL (5.3/5.8) hr: 180 mg/dL or 190 mg/dL (10.0/10.6) hr: 155 mg/dL or 165 mg/dL (8.6/9.2) hr: 140 mg/dL or 145 mg/dL (7.8/8.0) Gestational hypertension [ Time Frame: Measured during pregnancy ] Hypertension (SBP/DBP: above 140/90 mm Hg) occurred during pregnancy

2015 Clinical Trials

370. Improving Quality by Maintaining Accurate Problems in the EHR

Coronary Artery Disease Congestive Heart Failure Hyperlipidemia Hypertension Myocardial Infarction Sickle Cell Disease Sleep Apnea Smoking Stroke Tuberculosis Other: Problem List Suggestion Not Applicable Detailed Description: The clinical problem list is a cornerstone of the problem-oriented medical record. Problem lists are used in a variety of ways throughout the process of clinical care. In addition to its use by clinicians, the problem list is also critical for decision support and quality (...) of our study is improving clinical outcomes, we have selected four outcome measures to evaluate from NCQA's Healthcare Effectiveness Data and Information Set (HEDIS) 2013 measure set: LDL control in patients with a history of myocardial infarction, LDL control in patients with coronary artery disease, blood pressure control in patients with coronary artery disease and blood pressure control in patients with hypertension. The details for the numerator and denominator for each measure are given

2015 Clinical Trials

371. Inspiratory Flow Rates Achieved by the COPD Patients Through Breezhaler®, Ellipta® and Handihaler® Inhaler Devices

major chronic illness including but not limited to a diagnosis of non-skin cancer, cystic fibrosis, bronchiectasis, α-1 anti-trypsin deficiency, myelomeningocele, sickle cell anemia, endocrine disease, congenital heart disease, unstable arrhythmia, congestive heart failure, stroke, severe hypertension, insulin-dependent diabetes mellitus, renal failure, liver disorders, immunodeficiency states, significant neurodevelopmental delay or behavioral disorders (excluding mild attention deficit (...) 4, 2015 Results First Posted : June 26, 2018 Last Update Posted : June 26, 2018 Sponsor: Novartis Pharmaceuticals Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals ) Study Details Study Description Go to Brief Summary: The purpose of this study was to compare dynamic inspiratory flow rates achieved by a population of Chronic Obstructive Pulmonary Disease (COPD) patients through the Breezhaler®, Ellipta® and Handihaler® dry powder inhaler (DPI) devices. No active

2015 Clinical Trials

372. COOL AMI EU Pilot Trial to Assess Cooling as an Adjunctive Therapy to Percutaneous Intervention in Patients With Acute Myocardial Infarction

or severe renal failure (clearance < 30ml/min/1.73m2). Fever (temperature > 37.5 °C) or infection with fever in the last 5 days. Previous CABG. Stroke within 90 days of admission. Cardio-pulmonary decompensation present or imminent Contraindications to hypothermia, such as patients with known hematologic dyscrasias which affect thrombosis (e.g., cryoglobulinemia, sickle cell disease, serum cold agglutinins) or vasospastic disorders (such as Raynaud's or thromboangitis obliterans) Hypersensitivity (...) or contraindication to aspirin, heparin, or sensitivity to contrast media, which cannot be adequately pre-medicated. History of bleeding diathesis, coagulopathy, cryoglobulinemia, sickle cell anemia, or will refuse blood transfusions. Height of <1.5 meters (4 feet 11 inches). Hypersensitivity to buspirone hydrochloride or Pethidine (Meperidine) and/or treatment with a monoamine oxidase inhibitor in the past 14 days. History of severe hepatic or renal impairment, untreated hypothyroidism, Addison's disease, benign

2015 Clinical Trials

373. Pulmonary complications of hemoglobinopathies. (Abstract)

Pulmonary complications of hemoglobinopathies. Hemoglobinopathies are diseases caused by genetic mutations that result in abnormal, dysfunctional hemoglobin molecules or lower levels of normal hemoglobin molecules. The most common hemoglobinopathies are sickle cell disease (SCD) and the thalassemias. In SCD, an abnormal hemoglobin alters the erythrocyte, causing a chronic hemolytic anemia, which can lead to pulmonary parenchymal damage and impaired vascular function. Pulmonary complications (...) of SCD include the acute chest syndrome (ACS), reactive airways disease, pulmonary hypertension (PH), and pulmonary fibrosis. Episodes of ACS and the development of PH both increase the risk of death in patients with SCD. Both α and β thalassemia are characterized by impaired production of hemoglobin subunits, and severity of disease varies widely. Although screening studies suggest that PH is a common complication for patients with thalassemia, its impact on survival is unknown. Understanding

2010 Chest

374. Late Effects of Treatment for Childhood Cancer

. Tumor-induced organ dysfunction. Mechanical effects. Treatment-related factors Radiation therapy: Total dose, fraction size, organ or tissue volume, type of machine energy. Chemotherapy: Agent type, dose-intensity, cumulative dose, schedule. Surgery: Technique, site. Hematopoietic cell transplantation. Use of combined modality therapy. Blood product transfusion. Management of chronic graft-versus-host disease. Host-related factors Sex. Genetic predisposition. Premorbid health state. Developmental (...) and their potential health risks (e.g., chemotherapy type and cumulative dose, radiation treatment fields and dose, surgical procedures, blood product transfusions, and hematopoietic cell transplantation). Personalized health screening recommendations. Information about lifestyle factors that modify risks. A CCSS investigation that evaluated perceptions of future health and cancer risk highlighted the importance of continuing education of survivors during long-term follow-up evaluations. A substantial subgroup

2012 PDQ - NCI's Comprehensive Cancer Database

375. Wilms Tumor and Other Childhood Kidney Tumors

patients without hemihyperplasia.[ ] (Refer to the section of this summary for more information.) Other syndromic causes of Wilms tumor include the following: Perlman syndrome. Perlman syndrome—a rare, autosomal, recessively inherited, congenital overgrowth syndrome—is characterized by fetal gigantism, renal dysplasia and nephroblastomatosis, islet cell hypertrophy, multiple congenital anomalies, and mental retardation. Survivors have a high risk of developing Wilms tumor (75%).[ ] Germline (...) key roles in early renal development),[ , ] EP300 , CREBBP , and MYCN .[ ] Of the mutations in Wilms tumors, 30% to 50% appear to converge on the process of transcriptional elongation in renal development and include the genes MLLT1 , BCOR , MAP3K4 , BRD7 , and HDAC4 .[ ] Anaplastic Wilms tumor is characterized by the presence of TP53 mutations. Elevated rates of Wilms tumor are observed in a number of genetic disorders, including WAGR (Wilms tumor, aniridia, genitourinary anomalies, and mental

2012 PDQ - NCI's Comprehensive Cancer Database

376. Biopoin (epoetin theta)

treatment. In addition, rhEPO treatment did not enhance tumour growth in DS-sarcoma bearing anaemic and non-anaemic rats. Moreover, anaemia resulted in a worsening of tumour oxygenation in DS-sarcoma bearing rats which could partially be reversed by rhEPO administration or by transfusion with red blood cells in small tumours. In larger tumours, neither method of anaemia correction resulted in significant changes in tumour oxygenation • Safety pharmacology programme The effects of a single intravenous (...) theta exhibited changes in the hemodynamic profile including: transient reductions in blood pressure (systemic, left ventricular and pulmonary artery) and concurrent reductions and/or elevations in heart rate. These histamine-like reactions observed in all four animals were attributed to a detergent (Tween-20) in the vehicle formulation. An additional animal was instrumented separately and treated at a lower dose volume (0,3 ml/kg), proposed for use in a subsequent 6-week toxicity study

2009 European Medicines Agency - EPARs

377. Kombiglyze XR (saxagliptin/metformin extended release) fixed dose combination tablets

hormones are released from the gastrointestinal tract during meals and stimulate insulin release from the pancreatic beta-cell in a glucose-dependent manner. DPP4 inhibitors increase the levels of intact glucagon-like peptide-1 (GLP-1). The intact GLP-1 regulates blood glucose via stimulation of glucose-dependent insulin secretion, delaying of gastric emptying, inhibiting of glucagon secretion, and as a result of all these actions, improves the pre-prandial and postprandial glycemic profile. Metformin (...) is a biguanide, that decreases elevated blood glucose with predominant effect on fasting hyperglycemia, reduces hepatic glucose output, improves peripheral glucose uptake and utilization, leads to improved insulin sensitivity. The combination therapy with DPP4 inhibitor and metformin lowers the glucose via different mechanisms of action. (b) (4)Clinical Review Arlet V. Nedeltcheva, M.D. NDA 200678 saxagliptin/metformin XR FDC 8 Metformin XR is proposed to be used as the metformin component in the fixed-dose

2009 FDA - Drug Approval Package

378. ACR-ASNR-SNIS-SPR Practice Guideline for the Performance of Cervicocerebral Magnetic Resonance Angiography (MRA)

following extracorporeal membrane oxygenation. 5. Intracranial aneurysm. 6. Vascular abnormalities, such as sickle cell anemia, large vessel vasculitis, and moyamoya disease. 7. Blood supply to vascular neoplasms for operative planning. 8. Etiology of intracranial hemorrhage and spinal hemorrhage. 9. Presence, nature, and extent of traumatic injury to cervicocerebral vessels, including dissection. 10. Nature and extent of other congenital or acquired vascular abnormality. B. Indications (...) stroke: assessment using parenchymal and angiographic magnetic resonance imaging. Ann Neurol 1991; 29:585-589. 5. Wiznitzer M, Ruggieri PM, Masaryk TJ, Ross JS, Modic MT, Berman B. Diagnosis of cerebrovascular disease in sickle cell anemia by magnetic resonance angiography. J Pediatr 1990; 117:551-555. 6. Debrey SM, Yu H, Lynch JK, et al. Diagnostic accuracy of magnetic resonance angiography for internal carotid artery disease: a systematic review and meta-analysis. Stroke 2008; 39:2237-2248. 7. JM

2010 American Society of Neuroradiology

379. Management of Priapism

(CBC) with special attention to the white blood count (WBC), white blood cell differential and platelet count. Acute infections or hematologic abnormalities that can cause priapism, such as sickled red blood cells, leukemia and platelet abnormalities, may be suggested or identified by the CBC. The reticulocyte count is often elevated in men with sickle cell anemia. Hemoglobin electrophoresis identifies the presence of sickle cell disease or trait as well as other hemoglobinopathies. Because (...) evacuation of old blood, and followed, when necessary, by a surgical shunting procedure. Close Ischemic Priapism Guideline Statement 2 In patients with an underlying disorder, such as sickle cell disease or hematologic malignancy, systemic treatment of the underlying disorder should not be undertaken as the only treatment for ischemic priapism. The ischemic priapism requires intracavernous treatment, and this should be administered concurrently. × Discussion Ischemic priapism is a compartment syndrome

2010 American Urological Association

380. Practice Parameters for the Respiratory Indications for Polysomnography in Children

children with suspected SRBDs. Findings indicate that PSG may be particularly useful in the evaluation of children with chronic health conditions such as obe- sity, metabolic syndrome, overt or evolving hypertension, and other conditions associated with cardiovascular risk. There is a need for further investigation of SRBD in children with neuro- developmental and neuromuscular disorders, sickle cell anemia, craniofacial disorders, and certain infants who experience ALTEs. The clinical utility and cost (...) treatment of children for obstructive sleep apnea syndrome with rapid maxillary expansion (2) Clinical parameters may be unreliable for predicting OSAS. (3) Children with certain medical disorders are at higher sur- gical risk (e.g., sickle cell anemia, HIV , coagulopathies, con- genital heart disease). (4) Children with severe OSAS have a higher risk for certain postoperative complications including respiratory compromise. The task force identified 30 papers pertaining to the clinical utility of PSG

2010 American Academy of Sleep Medicine

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