Most of the patients suffering from non-malignant hematologic conditions are cured of the original disease with Hematopoitec Stem Cell Transplant (HSCT) but still their survival is less compared to age matched general population, and additionally they suffer from unique complications of HSCT culminating into a variety of late physical, psychologic, financial, and social complications (“late effects”). Considerable improvements ...more »

Can new advances in allogeneic blood or marrow transplantation (BMT) make the procedure a safe and universally available therapeutic strategy for treating non-malignant blood and immune disorders such as sickle cell anemia, thalassemia, aplastic anemia, and severe combined immune deficiency?

Allogeneic stem cell transplantation is curative in aplastic anemia with much less intrinsic toxicity than transplantation in hematologic malignancies. The recent BMT-CTN trial demonstrated 97% survival at one year with little subsequent decline. However patients without matched related or unrelated donors have graft-rejection rates of up to 50%. Preliminary data from the Netherlands suggests that anti-thymocyte globulin ...more »

In neonatal, pediatric, and adult patients with critical illness, what is the best means to identify: (1) the degree to which anemia contributes to insufficient oxygen (O2) delivery and (2) the likelihood that O2 delivery will be improved by red blood cell (RBC) transfusion? These questions are most relevant to critically ill populations that exhibit unique physiology, including those with low cardiac output (cardiac ...more »

Many forms of anemia are associated with lowering HbA1C% but iron deficiency anemia modestly raises HbA1C%. The exact mechanism of anemia and iron deficiency anemia's effect on HbA1c levels is unclear. This impacts our treatment of diabetes mellitus and diabetes mellitus co-morbidity with HIV, Hepatitis C and other diseases in the presence of anemia.

Experimental mouse models of ITP and AIHA are dependent on FcgR-signaling, which in turn depends on Syk kinase activity for activation of phagocytes. A significant and refractory complication of stem cell transplantation is immune hemolytic anemia. This can be either autoimmune or alloimmune. In either case it is likely that clearance of RBC and RBC progenitors is dependent on extravascular mechanisms involving activation ...more »

Why are some blood diseases called by genetic mutations in ubiquitous pathways. Diamond Blackfan anemia is due to a mutation in ribosomal proteins.

There is a need to investigate hemoglobin biosynthesis in order to develop novel approaches to treat sickle cell disease, thalassemia, and other anemias.

What is the optimal approach to prevent and treat immune mediated hematologic disorders (autoimmune hemolytic anemia, immune thrombocytopenic purpura, etc) and complications of hematologic disease (inhibitors in hemophilia, transfusion-related alloimmunization, etc)