You are being asked to take part in this research study because you have been diagnosed with pulmonary sarcoidosis. Sarcoidosis is a disease that can affect the lungs, skin and other organs of the body. Sarcoidosis also involves immune cells which fight bacteria. The purpose of this study is to see if using specific antibiotics will help these immune fighting cells get rid of bacterial proteins and how the antibiotics affect respiratory (breathing) function. The antibiotics used in this study are Levaquin, Ethambutol, Azithromycin, and either Rifampin or Rifabutin. You will by chance be assigned either these medicines or a placebo (an inactive substance).

This study is being conducted to assess safety and efficacy of bone marrow transplant in severe and less severely affected children. In this study, we will transplant bone marrow or cord blood from a HLA-matched brother or sister instead of the currently more common method with a HLA-identical sibling donor. This is a multicenter, single arm trial for patients between 2 and 10 years of age who have less severe HbSS or sickle beta zero thalassemia. Patient must have an HLA identical sibling donor who is less than 10 years old and who does not have HbSS, SC or sickle beta zero thalassemia.

Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM).

If you have been diagnosed with NTM, then your doctor may decide that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to you in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because you have a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because you have had side effects that prevent the use of other drugs.

This is a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.

A device called the "Liposorber LA-15 System" has been approved by the United States Food and Drug Administration for treating kids with focal segmental
glomerulosclerosis (FSGS). The "Liposorber LA-15 System" can only be used if other treatment options, like drugs, don't work or can't be used, but the kidneys are still working okay. It can also be used if the subject has had a kidney transplant and the FSGS comes back after the transplant. Although the Liposorber System can be used for FSGS, we are not sure how well the Liposorber System works. So, we are doing this study to find out how well the treatment works.

In this research study, there will be up to 5 children who have FSGS enrolled at MUSC. Subjects will come back for up to 12 treatments over 9 weeks and then 5 visits to their study doctor over the next 2 years.

INVESTED is a multi-site trial comparing high-dose (60 μg per vaccine viral strain) trivalent influenza vaccine to standard-dose (15 μg per viral strain) quadrivalent influenza vaccination for up to three influenza seasons in high-risk cardiovascular disease patients with a history of myocardial infarction in the previous 12 months OR history of heart failure hospitalization in the previous 24 months. Subjects will be randomly assigned to receive either the high-dose or standard-dose vaccine.

Current care patterns for patients with acute chest pain fail to provide optimal quality and value. To avoid missing acute
coronary syndrome (ACS), emergency physicians hospitalize >50% of patients who present to the Emergency Department with
chest pain. However, <10% are ultimately diagnosed with ACS, and this pervasive overtriage
costs $1013
billion annually.
The HEART Pathway, which was developed at Wake Forest Baptist Health (WFBH), is designed to improve care for patients with
chest pain. It uses a validated clinical decision aid and serial troponin measures to provide realtime
decision support to providers.
In our prior studies, the HEART Pathway decreased hospitalizations, stress testing, and hospital length of stay, without
increasing adverse events. These studies led to a learning health system project in collaboration with insurers, in which the
HEART Pathway was fully integrated into the WFBH EHR. Preliminary results demonstrate further reductions in hospitalizations
and stress testing. Given WFBH's success with the HEART Pathway, the next logical step is regional dissemination.
This project will leverage Carolinas Collaborative infrastructure to collect data specific to the HEART Pathway from all 4 health
systems, establishing rates of healthcare utilization and ACS outcomes for Emergency Department patients with chest pain in
the Carolinas. In addition, we will engage key stakeholders at each health system to develop an implementation strategy. This
proposal builds on our prior work and will provide pilot data essential for a larger grant application that will support rigorous
testing and implementation of the HEART Pathway across Carolinas Collaborative health systems.

Lymphangioleiomyomatosis (LAM) is a rare lung disease that is caused by genetic mutations. It results in the uncontrolled growth and proliferation of an atypical smooth muscle cells in the lung. These cells invade airways, blood vessels, and lymph vessels, and limit the flow of air, blood, and lymph, respectively. The source of the cells is unknown, but available evidence indicates they arise from an extrapulmonary source. Their aberrant behavior is due to mutations in tuberous sclerosis genes that results in mTOR activation. Respiratory failure, lung collapse (pneumothorax), and pleural effusions (chylothorax) are hallmarks of the disease. This study will evaluate the safety and durability of the mTOR inhibitors sirolimus and everolimus, which are FDA approved medications for prevention of rejection of transplanted organs, in stabilizing or improving lung function in people in LAM.

Transforming health care and outcomes for children with rare diseases is difficult within the current health care system. There is great variation in care delivery, inadequate and slow application of existing evidence, and ineffective use of available data to generate new knowledge. Individual care centers have inadequate numbers of patients for robust learning and improvement. In order to redesign the system, changes must take place at multiple levels, including the patient and family, clinician, practice and the network. The purpose of this project is to design, develop, and test further refinements to an improvement and research network focused on HLHS, the most severe congenital heart defect, and to use a registry to simultaneously improve clinical care, redesign care delivery systems and to conduct quality improvement, health services, outcomes, and comparative effectiveness research. The purpose of this initiative, specifically, is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes. We utilize a quality improvement methodology, known as the adapted learning collaborative model, which expedites the implementation of tools and strategies that facilitate changes such as systematic care coordination, cardiovascular monitoring, and nutritional monitoring into every day practice. The NPC-QIC registry is used to document the impact of these changes on various care processes and outcomes (e.g., mortality rate, readmissions, and weight gain).

PEPPER is a randomized study comparing the three most commonly used blood thinners in North America in patients who have elected to undergo primary or revision hip or knee joint replacement surgery. The blood thinners being compared are enteric coated aspirin, low intensity warfarin, and rivaroxaban.