Clinical Trials

Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the nociceptive system, and as a receptor antagonist, ketamine mitigates this. Intravenous (IV) ketamine has demonstrated efficacy in reducing post-operative, chronic, and cancer-related pain in pediatrics, as well as in reducing time to pain control in the emergency department (ED) in adults. Limited studies suggest efficacy in adult opioid-refractory SCD patients.

Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.

The sickle cell disease registry of the Society for Paediatric Oncology/Haematology aims at describing the epidemiology of sickle cell disease in German-speaking central Europe. Patients with sickle cell disease will be characterized clinically and genetically and treatment will be documented with the aim to find predictors of the course of disease.

This study looks at why some children develop very severe anemia that leads to a blood transfusion. It also checks for diseases in the blood they receive. Researchers are using small samples of dried blood (called dried blood spots) from both the children and the donated blood to find out what might be causing the anemia.

The purpose of this study is to determine if warming the intravenous (IV) fluid given to patients with Sickle Cell Disease who are experiencing painful episodes known as Vaso-Occlusive Episodes; will decrease rates of hospital admission, decrease amounts of IV pain medications given, improve pain and improve global comfort.

Children with sickle cell disease may experience frequent painful episodes. This, together with the traumatic experiences during a hospitalization, can lead to the development of posttraumatic stress reactions. As the stress can trigger painful episodes (pain crisis) in children with sickle cell disease, the investigators think that treating these stress symptoms can reduce the pain-related problems in their lives. Eye Movement Desensitization and Reprocessing (EMDR) is proven to be an effective trauma treatment for posttraumatic stress disorder.

This study will prospectively investigate the feasibility, safety, and transfusion requirements of adding hydroxyurea to simple chronic transfusions for patients with sickle cell anemia already on chronic transfusions.

This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a genetically matched sibling.

This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.

It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age. Data describing the magnitude of the sickle cell problem are lacking in most African countries. The available data on prevalence were mainly from older studies and small numbers of hospitalized patients. In Uganda, approximately 25,000 children are born with SCD but 70-80% die before their 5th birthday.

Zinc is a nutritionally essential trace element found in previous studies to reduce growth retardation and improve immune function, which may also result in decreased incidence of infectious diseases including malaria, pneumonia and diarrhea. Sickle Cell Disease (SCD) patients are known to be susceptible to zinc deficiency and appear to benefit from zinc supplementation.