Steroid Treatment for Sickle Cell Pain Crisis
Sickle Cell DiseaseVaso-occlusive CrisisThe painful episode is the most common problem experienced by children with sickle cell disease. Although various treatments are available during painful episodes, the medication most commonly given for pain is a pain medication such as morphine. Fluids are also used. Even with these treatments, many children still have severe pain that is difficult to control. In addition to pain medications, there are other medications that may be useful. Methylprednisolone (solumedrol) and prednisone are a group of medications called steroids that may be helpful for painful episodes. These medications are known to lower the amount of inflammation (this means swelling, tenderness, and soreness) in the body. Because this medication may help with your pain, you are being asked to be a part of this study. These types of medications are used in other illnesses such as asthma, especially during times when the illness has gotten worse. The main purpose of this study is to see if the methylprednisolone and prednisone will lower the amount of pain and the length of hospital stay. In addition to the pain medication you will normally receive, you will be assigned to one of 2 groups: 1) the experimental group with the active form of the medicine, or 2) a comparison group without the active form of the medicine. In either group, you will still receive all of the treatments you would normally receive for a painful episode, including pain medicines and fluids. You and your doctors will not know what group you will be assigned. If you decide to be a part of the study the following will happen: For the first 5 days, you will be asked to: 1) describe your current pain (0=no pain to 10=a lot of pain), worst pain (0=no pain to 10=a lot of pain), least pain (0=no pain to 10=a lot of pain), and the amount of pain relief (0=no relief to 10=complete relief); 2) describe any signs or symptoms you feel, including filling out a pain scale form each day; 3) and take the medicines for 5 days, either at home or when in the hospital. Thirty days after the study, a study researcher will call and will ask questions about your pain, any painful episodes, and any medications you had. If you are discharged home sooner than 5 days after the start of the study, research staff will call you to ask you these questions, remind you to fill out your pain forms, and remind you to take your medicine. If you are discharged home, you will be given pain scales to fill out each day at home.
Predictors and Outcomes in Patients With Sickle Cell Disease
Sickle Cell DiseaseChildren with sickle cell disease (SCD) are living longer with the advent of medical advances such as prophylactic penicillin, chronic transfusion, and hydroxyurea. Despite greater longevity in SCD, the period following the transition from pediatric to adult care is critical; youth aged 18-30 years are at high risk for mortality and have high rates of healthcare utilization, leading to high healthcare costs. As such, health care transition (HCT) programs have been created to prepare patients for adult-centered care and subsequently, improve health outcomes. However, very few programs have been evaluated for effectiveness in achieving optimal health outcomes in SCD. This paucity of program evaluation is attributed to a lack of identifiable predictors and outcomes. Researchers at St. Jude Children's Research Hospital want to identify factors and patterns of successful HCT. This information will be used to develop approaches to best evaluate HCT interventions and identify areas of improvement of HCT programming. PRIMARY OBJECTIVE: Describe hospital utilization, treatment adherence, and health-related quality of life in a cohort of patients with sickle cell disease (SCD) who will transfer to adult care during the study period. SECONDARY OBJECTIVE: Examine the associations between various factors and health care transition (HCT) outcomes.
Kidney Function in Sickle Cell Anemia
Sickle Cell DiseaseKidney Failure1 moreThis is a prospective clinical cohort study that involves a baseline study visit followed by up to 3 annual follow-up study visits for a total follow-up of 36-48 months to evaluate the age- and sex-adjusted rate of change in kidney function, and to identify biomarkers of endothelial function, metabolomic profiles and clinical characteristics for the worsening of kidney function and for a rapid decline in kidney function. "Funding Source - FDA OOPD"
Omega-3 Fatty Acids in Sickle Cell Disease
Sickle Cell DiseaseThe purpose of this study is to determine the safety of a new formulation of the omega-3 fatty acids Docosahexaenoic Acid (DHA) and Eicosapentaenoic Acid (EPA) and to assess whether it decreases inflammation and inflammatory pain in children and young adults with Sickle Cell Disease.
A Study to Evaluate Safety, Pharmacokinetic, and Biological Activity of INCB059872 in Subjects With...
Sickle Cell DiseaseThe purpose of this study was to evaluate the safety and tolerability, and the pharmacokinetic and biologic activity of INCB059872 in participants with sickle cell disease.
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Sickle Cell DiseaseSickle Cell AnemiaSickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape. People who have SCD have a different type of hemoglobin (protein that carries oxygen). This different type of hemoglobin makes the red blood cells change into a crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood and can cause inflammation and injury to important areas of the body. All babies are born with hemoglobin called fetal hemoglobin (HbF). Soon after birth, HbF production slows down and another hemoglobin called adult hemoglobin (HbA) is made. Clinical studies have shown that increasing the amount of HbF in the blood may prevent sickling of the red blood cells. Vorinostat has been used in the treatment of cancers and in other research studies and information from those suggests that it may help treat SCD by increasing the amount of HbF in the blood. The purpose of this research study is to determine the effectiveness and safety of vorinostat when used to treat SCD.
Low-Dose Ketamine Infusion for Children With Sickle Cell Disease-Related Pain
Sickle Cell DiseaseAcute pain episodes associated with sickle cell disease (SCD) are very difficult to manage effectively. Opioid tolerance and side effects have been major roadblocks in our ability to provide these patients with adequate pain relief. This pilot study is designed to examine the safety and feasibility of using ketamine, an N-methyl-D-aspartate (NMDA) receptor antagonist, in the inpatient seeing with children and adolescents who have sickle cell vasoocclusive pain. Previous research suggests that in subanesthetic doses, ketamine may be able to prevent the development of opiate tolerance and facilitate better pain relief with lower opiate doses, allowing for less respiratory depression, less sedation, easier ambulation, less deconditioning, shorter hospital stays, and better quality of life. The goal of this pilot study is to evaluate the safety and feasibility of using a continuous infusion of ketamine, in conjunction with opiates, in the inpatient setting for sickle cell vasoocclusive pain. It is hypothesized that using a low dose ketamine infusion in conjunction with opiates will be a safe and feasible practice for the treatment of sickle cell pain.
TRF-1101 Assessment in Sickle Cell Disease
Sickle Cell DiseaseThis study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
A Safety Study of Eptifibatide in Patients With Sickle Cell Disease
Sickle Cell DiseaseThis study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises. The overall hypothesis that we seek to test is that increased platelet activation and the resultant inflammatory responses are important contributors to the problems of sickle cell disease. Sickle cell disease has been referred to both as a condition associated with increased risk of blood clots and increased inflammation. A painful crisis represents the most common cli nical problem in sickle cell disease, but the treatment of these crises remains inadequate.
Ibuprofen and Opioid (Morphine or Diamorphine) for Acute Pain in Sickle Cell Disease - Sickle With...
Sickle Cell DiseaseThe use of oral ibuprofen combined with Opioid (Morphine or Diamorphine) administered through patient controlled analgesia (PCA) will be clinically effective for acute pain crisis in adults with sickle cell disease (SCD).