View clinical trials related to Sickle Cell Disease.
Filter by:Children 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.
This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab).
Sickle cell disease (SCD) is a genetic disease characterized by abnormal hemoglobin, the main constituent of red blood cells. People with SCD have nutritional deficiencies, and vitamin D deficiency is one of the most common. Symptoms of vitamin D deficiency are similar to those of SCD and include chronic pain and bone complications. Correcting vitamin D nutrition of children with SCD represents a treatment that will improve their health. A single oral high-dose of vitamin D3 will be given to SCD children during one of their follow-up visits at the SCD clinic of CHU Sainte-Justine, Montreal, Canada. This mode of administration was chosen to ensure a better adherence to the treatment. The investigators will determine whether this dose is safe and its administration feasible in clinic. The impact of this dose on blood vitamin D and calcium, urinary calcium, growth, inflammation, bone health, pain and quality of life will also be assessed. This study intends to propose a new intervention to improve the nutrition of children with this disease.
Bearers of the sickle cell allele (S) are currently eligible for blood donations in Belgium. As blood donors are not tested for this allele, their heterozygous status is unknown. However, guidelines recommend to transfuse sickle cell patients with blood that is negative for the 'S' hemoglobin. To the investigator's knowledge, no study has been conducted to evaluate the impact of transfusion with blood originating from heterozygous donors on the transfusion performance and the improvement of clinical status of the sickle cell disease patients.
Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and the sickle cell patients of African origin. Genetic and environmental risk factors have also been described. The main risk factors that have been shown in retrospective or cross-sectional studies are some HLA alleles, the age of the patient, the number of leukocyte-depleted erythrocyte concentrates (CED) transfused, the number of transfusion episodes, the age of the CEDs, the existence of an inflammatory event at the time of transfusion and the presence of anti-erythrocyte autoantibodies.There is also evidence of an impaired TH response but the underlying immunological mechanism is not fully understood. The aim of this study is to study the prevalence and the risk factors for anti-erythrocyte alloimmunization and to try to understand the immunological mechanisms.
Sickle cell patients have a high prevalence of alloimmunization. This high rate of alloimmunization can be partially explained by the existence of an antigenic difference between the predominantly Caucasian donor population and the sickle cell patients of African origin. Genetic and environmental risk factors have also been described. The main risk factors that have been shown in retrospective or cross-sectional studies are some HLA alleles, the age of the patient, the number of leukocyte-depleted erythrocyte concentrates (CED) transfused, the number of transfusion episodes, the age of the CEDs, the existence of an inflammatory event at the time of transfusion and the presence of anti-erythrocyte autoantibodies.There is also evidence of an impaired TH response but the underlying immunological mechanism is not fully understood. The aim of this study is to study the prevalence and the risk factors for anti-erythrocyte alloimmunization in pediatric and adult patients with Sickle Cell Disease (with a SS genotype) who are being followed at Queen Fabiola University Children's Hospital (HUDERF) and at the CHU Brugmann Hospital. The identification of risk factors would allow the investigators to improve, or at least adapt, their transfusion policy to certain clinical or immuno-haematological situations.
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
Patients with sickle cell disease (SCD) and cancer often have complicated courses while hospitalized and often deal with pain, anxiety and depression. Advances in the field of technology provide potential avenues for innovative and improved care models for our patients. Virtual reality (VR) has been recently utilized to improve anxiety and pain in a variety of patient populations including children undergoing elective surgery and children experiencing intravenous cannulation in the Emergency Department. Patients with SCD and cancer, both adults and children, are a group of patients that can benefit from VR as part of their care. Over the past four years, our team has successfully implemented several self-developed mobile applications ("apps") for our patients, in addition to integrating objective data (heart rate, activity, stress) from wearable activity trackers. The investigators now propose implementing a feasibility study followed by a pilot study and randomized-controlled trial of the use of VR in patients with SCD and cancer. The investigators plan to assess pain and anxiety prior to the session as well as following the session in hospitalized patients and outpatients with SCD and cancer. The sessions will include a ten-minute relaxation response introductory narrative segment (deep breathing and mindfulness) followed by a ten-minute narrated and immersive VR. Heart rate will be tracked using an Apple iWatch for 30 minutes prior to the session, during the session, and following the session. We anticipate VR will not only be a feasible method to provide non-pharmacologic treatment, but will also significantly reduce pain and anxiety.
This study will test the hypothesis that Heart Rate Variability Biofeedback (HRV-B) restores autonomic balance and reduces pain and other symptoms among patients with sickle cell disease (SCD).The specific aims of this study are to: (1) conduct a randomized, wait list controlled, pilot intervention trial to determine whether HRV-B increases HRV coherence among SCD participants (minimum N of 30, up to 50 total); (2) determine whether HRV-B reduces pain, stress, fatigue, depression or insomnia among SCD participants; and (3) determine whether increases in HRV coherence are associated improvements in pain, stress, fatigue, depression, or sleep among study participants.
Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US. Silent and overt strokes contribute significantly to morbidity in adults with SCD, resulting in functional impairment, challenges with school and job performance, and premature death. Five NIH-funded randomized controlled trials have identified therapies to prevent silent and overt strokes in children with SCD, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). Despite the observation that at least 99% of children with SCD in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (~50% with silent strokes and ~10% with overt strokes), no stroke trials have established therapeutic approaches for adults with SCD. For adults with SCD, inadequate evidence-based guidelines exist for secondary stroke prevention strategies. Applying stroke prevention strategies in children may not be effective for stroke prevention in adults with SCD, particularly given the high rate of co-morbidities. Identifying subgroups of adults with SCD and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the requisite data required for the first-ever phase III clinical trials focused on secondary stroke prevention in adults.