View clinical trials related to Anemia, Aplastic.
Filter by:The study aims to evaluate the molecular mechanism underlying the erythroid response observed in some patients with myelodysplasia, myelofibrosis and aplastic anemia treated with Deferasirox or Deferoxamina.
To utilise extended platelet parameters in order to individuate Immune Thrombocytopenia (ITP) from hypo-proliferative causes of thrombocytopenia. To develop the clinical potential of the extended platelet parameters as they pertain to distinguishing different causes of thrombocytopenia from one another. To test the hypothesis that mean platelet component (MPC) and mean platelet mass (MPM) might distinguish between thrombocytopenia related to bone marrow dysfunction and immune mediated destruction of platelets.
Feasibility and toxicity of haploidentical transplantation of CD3/CD19 depleted stem cells in combination with a toxicity reduced conditioning regimen or with standard conditioning regimens according to underlying disease.
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem cell transplant for serious non-malignant diseases as well as to assess the immunological effects of abatacept. Participants will be followed for 2 years.
Background: - This research is being done to describe the types of bacteria found in the mouths of patients who have severe aplastic anemia (SAA) and are treated with drugs that suppress the immune system or with stem cell transplant. People with SAA who receive these treatments are more likely to get infections. Studies show that there might be a link between the bacteria in your mouth and those bacteria that can cause infections. The bacteria found in the mouths of patients with SAA will be described. Objectives: - To understand the changes in mouth bacteria that are related to treatment and to describe the oral bacterial environment. Eligibility: - Adults at least 18 years of age who are going to be treated for SAA. - Healthy volunteers at least 18 years of age. Design: - Participants will answer questions about their medical history and dental care. Their mouths will be examined. - Participants with SAA will be tested during treatment for their disease, over the course of 1 year. All participants with SAA will be tested at 3 scheduled appointments. Any participants who require a breathing tube will receive additional tests. - Healthy volunteers will be tested during 1 visit. - Participants will give two samples each time. A saliva sample will be taken with a disposable padded tool. Skin cells will be collected from the tongue with a small plastic brush.
Background: - Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients. However, researchers do not know how long the drug can and should be taken for this type of anemia. Objectives: - To look at whether 6 months of treatment with eltrombopag can improve patient s blood counts. Eligibility: - Individuals at least 2 years of age who are taking eltrombopag for severe aplastic anemia. Design: - Participants will take eltrombopag by mouth once a day for 6 months. - Blood samples will be collected every 2 weeks for the first 6 months. Bone marrow samples will be collected at 3 and 6 months. These samples will look at the effects of the study drug on the marrow. - Participants will continue to take the study drug for as long as it is effective and if the side effects are not severe.
Background: - Cord blood is blood that is taken from the umbilical cord and placenta of healthy newborns after childbirth. The cord blood collected from a baby is called a cord blood unit. Cord blood units are stored frozen in public cord blood banks. About 10,000 cord blood transplants have been performed in children and adults for blood cancers and other diseases in the world. These transplants have helped save lives and improve treatments. However, not all available units of cord blood have been collected, stored, and licensed according to specific government requirements. These unlicensed units can still be used in transplant, but they can only be given as part of specific research studies. This study will evaluate the safety of giving these unlicensed units by recording any problems that may occur during and after giving the cord blood. Objectives: - To test the safety and effectiveness of unlicensed cord blood units in people who need stem cell transplants. Eligibility: - Individuals who are scheduled to have a stem cell transplant. Design: - Participants will be screened with a medical history and physical exam. - Participants will receive the cord blood unit as part of their stem cell transplant procedure. The transplant will be performed according to the current standard of care for the procedure. - After the transplant, participants will be monitored for up to 1 year. Any problems or side effects from the transplant will be treated as necessary. All outcomes will be reported to the National Cord Blood Program and to the Center for International Blood and Marrow Transplant.
Septic shock remains a significant clinical problem associated with high rates of mortality among neutropenic patient despite antimicrobial therapy and supportive care. Recently, mesenchymal stromal cells (MSC) have demonstrated remarkable potential effect in sepsis. MSC treatment significantly reduced mortality in septic mice receiving appropriate antimicrobial therapy. MSCs reduced systemic inflammatory cytokine levels in mice, down-regulated of inflammation and inflammation-related genes (such as interleukin-10, interleukin-6). Bacterial clearance was greater in MSC-treated mice. Thus, MSCs have beneficial effects on experimental sepsis and suggest that MSСs-therapy may be an effective adjunctive treatment to reduce sepsis-related mortality. The safety of MSCs is proved by Graft-versus-host disease treatment MSCs in patients after bone marrow transplantation. This study hypothesis is that MSCs reduce organ dysfunction/injury, systemic inflammation and mortality in patients with septic shock and severe neutropenia. The main goal of the study is to evaluate the impact of MSCs therapy on organ dysfunction/injury, systemic inflammation and 28-day mortality in patients with septic shock and severe neutropenia. All patients will be randomized in two groups: control group (standard treatment of septic shock) and MSCs-group (standard treatment of septic shock + MSCs infusion of 1-2 millions/kg/ day).
The objective of this study is to evaluate the feasibility and effectiveness of immunosuppressive therapy (IST) using rabbit anti-thymocyte globulin (ATG) (Thymoglobuline, Genzyme) for patients with very severe aplastic anemia (VSAA) and severe aplastic anemia (SAA) as a primary therapy. The primary endpoint is the response rate (complete response (CR) + partial response (PR)) at day 180 after the start of IST. Secondary endpoints include evaluation of the presence and frequency of Epstein-Barr virus (EBV)-reactivation and EBV-associated lymphoproliferative disorder (EBV-LPD), Cytomegalovirus(CMV)-reactivation and CMV associated diseases, the response rate (CR+PR) on Day 360 after the start of IST, relapse rate and overall survival.
Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) without chelation therapy.