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.
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).
We are interested in understanding the type of recommendations doctors give, to manage and prevent pregnant and laboring women with a problem with their platelets, who are getting spinal medicine to help reduce their pain during labor.
The primary aim of this study is to assess how the COVID-19 pandemic has affected resident-physicians' behavior regarding the 2020-2021 fellowship cycle. The secondary aim is to assess if the COVID-19 pandemic has disproportionately affected fellowship applicants' behavior according to age, gender, ethnic/racial minority group, and those currently training in areas where there has been a higher rate of infections.
A phase III randomized multi-center study designed to compare the efficacy of crenolanib with that of midostaurin when administered following induction chemotherapy, consolidation chemotherapy and bone marrow transplantation in newly diagnosed AML subjects with FLT3 mutation. About 510 subjects will be randomized in a 1:1 ratio to receive either crenolanib in addition to standard first line treatment of AML (chemotherapy and if eligible, transplantation) (arm A) or midostaurin and standard treatment (arm B). Potentially eligible subjects will be registered and tested for the presence of FLT3 mutation. Once the FLT3 mutation status is confirmed and additional eligibility is established, subject will be randomized and enter into the treatment phase.
To examine the impact of sustained increase in FVIII and FIX levels after liver transplantation on quality of life (QoL) as measured by pain, health outcomes, work, activity, and mental and physical health in adults with hemophilia A and B.
We are surveying individuals with sickle cell disease (SCD) and/or parents of a child with SCD to get a sense of the types of treatment they would choose based on the harms and benefits of those treatment options. The proposed study will be designed to meet the following objectives: 1) Elicit individual respondent's maximum acceptable risk (MAR) for treatment benefits 2) Elicit individual respondent's minimum 3) Characterize heterogeneity in MARs and MABs among respondents
The purpose of this study is to see how talquetamab in combination with daratumumab and pomalidomide (Tal-DP) or talquetamab and daratumumab (Tal-D) compares to daratumumab, pomalidomide, and dexamethasone (DPd) in treating patients with multiple myeloma, who have not responded to previous treatment. Talquetamab is an experimental medication being studied to see if it may be beneficial in the treatment of multiple myeloma.