The purpose of this study is to collect a bank of information about people living with HHT to better understand HHT, the symptoms and complications it causes and how the disease impacts people's lives.
We are developing a computer-based tool for people who have cancers of the blood, bone marrow, and lymph nodes. This tool will help us understand what is most important to patients when it comes to the results of their treatment. We are looking for healthy volunteers (those without cancer) to help us test the usability of this tool.
The purpose of this study is to learn more about brain AVMs (BAVMs) and bleeding in HHT. We plan to study risk factors for rupture of BAVMs and also for other bleeding and features of HHT disease, including primarily genetics and imaging characteristics of the BAVMs. We hope that knowledge about risk factors will help improve the care and management of HHT patients.
To test the effect of automated RBC exchange transfusion and standard of care compared with standard of care on the number of episodes of clinical worsening of sickle cell disease (SCD)requiring acute health care encounters (non-elective infusion center/ER/Hospital visits) or resulting in death over 12 months in high risk adult SCD patients.
The purpose is to evaluate coagulation activation and the impact of iron status in patients with Hereditary Hemorrhagic Telangiectasia (HHT). Participants on this study will include patients with HHT, patients with iron deficiency +/- anemia but without HHT, and healthy volunteers The study team will procure single and/or serial blood samples from participants and assess various markers of hemostasis and activation of coagulation, and host iron status.
The primary objective of the study is to evaluate the safety and feasibility of treating patients with a history of iTTP with efgartigimod, an FcRn inhibitor that reduces IgG autoantibodies, including those responsible for ADAMTS13 depletion.
We want to test adults with sickle cell disease, using both the conventional in-person Montreal Cognitive Assessment (MOCA) and, within 2-4 months, the abbreviated 'MOCA-blind', which can be given remotely. In this way we can begin to evaluate whether these tests are sensitive to similar degrees of impairment when given remotely compared with in-person in people with sickle cell disease (SCD).
To evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in subjects with sickle cell disease (SCD) and nephropathy
Study the effectiveness and safety of Epeleuton
This study is being done to answer the following question: Do two different anti-cancer drugs, paclitaxel (PTX) and pegylated liposomal doxorubicin (PLD) have similar effects on treating KS in PLWH in sub-Saharan Africa? We are doing this study because we want to find out if one of these approaches, PTX or PLD, is better than the other for KS. Both drugs may be given as part of the usual approach for your KS. The usual approach is defined as care most people get for KS.