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.
This study is being offerred to patients that have acute myelogenous leukemia (AML) which is a cancer of the blood and these patients are going to have a stem cell transplant. This study is looking to determine how accurate two different laboratory tests are at detecting residual, or small numbers of cancer cells in the body before and after stem cell transplant, as well as whether or not results of these two tests show how well a recipient might do after transplant.
This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered “investigational” products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.
We will conduct a screening and direct assessment study in the general population in an area already undergoing monitoring by ADDM. Specifically, all 8-year-olds in a three county region of South Carolina (n=8,000) will be screened for ASD, and those found to be at risk for ASD will be invited to participate in an in-depth diagnostic assessment. Evidenced-based strategies will be implemented to maximize participation in both the screening and diagnostic phases of the study to improve the accuracy of the findings. ASD prevalence estimates will be calculated using the number of children aged 8 years residing in the study area as the denominator, and the number of children identified as cases as the numerator, with adjustments made for missing data from nonparticipants. Prevalence estimates will be provided using both traditional ADDM and SUCCESS methods. Factors contributing to
differences in ASD prevalence estimates by methodology (e.g. sex, race/ethnicity, SES, previous diagnoses, behaviors, degree of impairment, co-morbidities) will be examined. Additionally we will compare prevalence using the DSM-Iv and DSM-5 criteria.
Power wheelchairs are defined as ‘Wheelchairs powered by electricity that provide mobility and body support for individuals with limited ability to walk’ (Shoemaker et al., 2010). For the purposes of this study, the term power mobility is any battery powered equipment used for mobility by children with disabilities. This can include powered ride-on-toys (e.g. Boss car, Cooper car), powered scooter-boards and powered standing devices (e.g. Gobot). This study seeks to expand on the new literature being published on the use of commercially available powered ride-on-toys to assist with the early mobility of children born with movement disorders. Children with neuromuscular impairments have significantly decreased early mobility which greatly affects their opportunities to explore their physical and social environment (Tefft, Guerette, & Furumasu, 1999; Uchiyama, Anderson, & Campos, 2008). The commercially available ride-on-toys could be used in the clinic, home, community, or school settings to improve independent mobility and are a low cost alternative to other mobility devices (Huang & Galloway, 2012). In addition, these devices provide a novel therapeutic tool to examine and/or treat body function level impairments such as cause-effect learning and head/upper extremity/trunk/lower extremity strength and control (Ragonesi & Galloway, 2012). The utilization of early power mobility allows for important early exploration and learning and may have tremendous effect on later perceptual, cognitive, social, and quality of life outcomes for children with movement disorders.