This is a multicenter 48-52 month blinded-outcomes follow up study of subjects who received stannsoporfin or placebo in clinical trial 64,185-204. This clinical trial consists of clinic visits over 4 year period. The developmental tools used in this trial will identify children who are developmentally delayed or at risk for delay.
Extensive blood loss and multiple blood transfusions are a major source of patient complications during and after infant cardiac surgery. The activation of the fibrinolytic system (an enzyme sytem that breaks down blood clots) during surgery is thought to be a main cause of blood clot instability in these patients. Although medications that inhibit this system are routinely used, they also display very variable efficacy and can cause serious side effects. The primary hypothesis of this study is that direct measurement of the activation status of the fibrinolytic system directly inside local tissue (e.g. muscle or wound surfaces) will enable physicians to better evaluate the blood clotting ability of a pediatric heart surgery patient and will substantially improve diagnostic accuracy and drug dosing, thereby reducing the observed side effects such as kidney injury and increased risk of thrombosis.
This study would be the first to examine the application of pediatric constraint induced movement therapy (P-CIMT) in a developing country and could provide important pilot data to expand P-CIMT to larger trials and in other countries. The overarching aim of this proposal is to explore a culturally-relevant and feasible Ethiopian P-CIMT Treatment Model through collaboration with CURE Ethiopia Hospital in Addis Ababa, Ethiopia. If the proposed project demonstrates success at the CURE Ethiopia Hospital, there is immediate potential to educate providers from 28 connected orthopedic clinics within Ethiopia and a network of nine additional CURE Children’s Hospitals in surrounding countries.
Identification of swallowing disorders (dysphagia) in children has increased dramatically consequent to new medical technologies and life sustaining therapies in infants with serious medical conditions. Children with dysphagia risk aspiration-induced chronic lung disease, malnutrition, and chronic neurodevelopmental problems, negatively impacting health and the economic well-being of the family and health systems. Awareness of these risks brings an exponential increase in the number of videofluoroscopic swallowing studies (VFSSs) conducted in these fragile children. Despite 20 years of research, VFSS, a radiographic x-ray study is devoid of a standardized approach to characterize and quantify the type and severity of swallowing impairment necessary to effectively target treatments. There are no data demonstrating the clinical validity of the VFSS relative to health or developmental outcomes of children undergoing the procedure.
The goal of this project is to develop and test a standardized measurement tool and scoring schema for the quantification of swallowing impairment in bottle-fed children. To develop and test a validated and standardized tool for bottle-fed children, critical next steps are to determine: (1) intra- and inter-rater reliability of component scores in a larger cohort of SLPs, (2) internal consistency reliability and construct validity of the aggregate components, and (3) changes in physiologic swallowing impairment with a common intervention introduced during the VFSS. The specific aims will test the overall hypothesis that higher component scores of swallowing impairment will be associated with poorer indicators of growth, health, development, and quality of life. The long term goal of the project is to develop a clinically practical, reliable, and valid standardized tool for assessing, measuring, and reporting physiologic swallowing impairment in bottle-fed children.
Nearly 9 million U.S. children (1 in 8) meet criteria for at least one mental health disorder at any point in time. Effective treatments exist for these disorders, but children and families who seek services rarely receive them; mental health providers need more support in the delivery of these interventions to ensure that children and families are receiving the best quality care. This project aims to improve the delivery of best practices for families who seek mental health care by developing creative, technology-based resources for providers to enhance the care they already are providing.
This study enrolls donors and recipients involved in a bone marrow transplant. Donors are participants that are already planning on being a blood and marrow transplant (BMT) donor for a family member. This research study is asking for the donor's permission to take a small number of either the bone marrow cells or the peripheral blood (apheresis) cells for a research project, before they are transplanted into the family member. The extra bone marrow or peripheral blood stem cells will be analyzed in the laboratory. The researchers are trying to determine if there are certain cells, proteins, or genes (DNA) in the donor bone marrow or peripheral blood stem cell product that increase the risk of the recipient getting cGVHD. The recipients are participants that are about to undergo a blood and marrow transplantation (either bone marrow, peripheral blood stem cells, or umbilical cord blood – collectively referred to as “blood and marrow transplantation” or BMT). Chronic graft-versus-host disease (cGVHD) is one of the most important complications following BMT, occurring in around 10-20% of children who receive a transplant. This research study is trying to determine if it is possible to identify children earlier on who are at the highest risk for developing cGVHD. The goal would be to see if we can predict which children are at the highest risk of developing cGVHD after BMT, before cGVHD develops. If it is possible to separate children with lower or higher risks of cGVHD at an earlier time point after BMT (by day 100 post-BMT), it might mean that we could do something to try to prevent cGVHD from forming (e.g. give a medication to the higher risk group). The purpose of this study, however, is only to look at whether we can predict which patients are at a higher or lower risk of cGVHD – it is NOT a medication /drug trial.
This study is for patients that have acute leukemia or myelodysplastic syndrome and are going to have a bone marrow, blood stem cell, or cord blood transplant to treat blood cancer. Stem cells are cells found in the bone marrow and blood and are able to grow into all the types of blood cells that the body needs.
The purpose of this research study is to see if adding the investigational drug, treosulfan, to the transplant conditioning regimen of fludarabine and total body irradiation (TBI) is safe and effective in subjects undergoing bone marrow, blood stem cell, or cord blood transplantation. Participants will be in the study for 1 year. After participants have recovered from any immediate transplant related complications, we will continue to collect information on how their marrow is functioning at routine follow-up visits.
This research study is investigating a new antibiotic for children who have a skin infection requiring IV antibiotics in the hospital. This type of infection is called Acute Bacterial and Skin Structure Infection (ABSSSI).
Ceftaroline represents a new class of cephalosporin antibiotics that has activity against Methicillin Resistant Staphylococcus Aureus. Currently, there are very few antibiotics with activity against this bacteria. If ceftaroline is effective, it will constitute a major contribution to antibiotic therapy.
This research study is being done to learn more about the safety and how the study drug known as ceftaroline fosamil works in children. Ceftaroline fosamil is also called “ceftaroline” in this form and is also known as Teflaro ® in the U.S. Ceftaroline is an antibiotic. Antibiotics are drugs used to treat infections. The U.S. Food and Drug Administration (FDA) has approved ceftaroline fosamil to be used to treat adults with community-acquired bacterial pneumonia (CABP) and acute bacterial skin and skin structure infections (ABSSSI). In this study, the use of ceftaroline is considered investigational (experimental) because the FDA has not approved it for use in subjects less than 18 years of age.
The primary purposes of this study are to:
•Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study.
•Assess how well and how quickly blood counts return to normal after transplant in recipients on this study.
Genetic Testing of neonates undergoing surgery for single ventricle cardiac defects (SVCD) and other congenital cardiac defects. DNA testing with an aim to identifying genetic factors that aid survival and recovery in SCD patients.
Genetic contribution to patient outcomes: Over the past two decades, there has been dramatic improvement in the survival and functional outcome of patients with all forms of congenital cardiac defects. Yet, there exists significant variability in outcomes that becomes more pronounced as the level of surgical intervention increases and the exposure to adverse hemodynamic conditions becomes more prolonged and more profound. This is particularly noticeable in the SCD patient group where there are continued high levels of mortality and levels of disability that can be quite severe. While these poor outcomes can on occasion be attributed to technical difficulties, complex cardiac anatomy or patient co-morbidities, more commonly they occur in patients that do not superficially appear to be any different than those that will ultimately have excellent outcomes. What is becoming increasingly apparent is that every patient differs in their ability to tolerate the challenges presented by the peri-operative environment. Therefore, significant improvements in outcomes may depend on identification of the genetic factors that place some patients at greater risk and designing treatment protocols to minimize those risks.