View clinical trials related to Anemia, Aplastic.
Filter by:This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.
The aim of this study was to evaluate the safety and efficacy of a modified allogeneic hematopoietic stem cell transplantation regimen for aplastic anemia.
Patients eligible undergoing total body irradiation as candidates for bone marrow or peripheral stem cell transplant.
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Abatacept and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.
Title: Efficacy and safety of Avatrombopag VS Avatrombopag combined with rhTPO in patients with severe aplastic anemia: a single-center, controlled study Observation group: Patients with severe aplastic anemia who did not respond to initial treatment (unconditional HSCT or ATG) or other treatments (except HSCT) Objective: To evaluate the efficacy and safety of Avatrombopag and rhTPO in the treatment of patients with severe aplastic anemia, to provide more treatment options for patients with severe aplastic anemia who are unable to undergo transplantation /ATG or have failed previous treatment, and to provide evidence-based evidence for the use of Avatrombopag or combined with rhTPO to promote hematopoietic recovery Experimental design: Single center, controlled study Total number of cases: 30 cases/group, 2 groups Case selection criteria: Inclusion criteria: 1. Age: > 18 years old, gender is not limited; 2. Patients clinically diagnosed with severe aplastic anemia (diagnostic criteria: ① myelocyte hyperplasia < 25% of normal; If ≥ 25% of normal but < 50%, the remaining hematopoietic cells should be < 30%. ② Blood routine must have two of the following three items: ANC < 0.5×109 /L; The absolute value of reticulocyte was < 20×109 /L; PLT < 20×109 /L). ③ If ANC < 0.2×109 /L, the diagnosis is very severe aplastic anemia), including patients who are newly diagnosed or have failed other treatments; 3. Patients currently undergoing hematopoietic stem cell transplantation or ATG without conditions; 4. Eastern Cancer Collaboration Group (ECOG) score 0-2; 5. Informed consent must be signed before participating in the study. Exclusion criteria: One of the following circumstances is not eligible for inclusion: 1. Patients with severe bleeding and/or infection that cannot be controlled after standard treatment; 2. Diagnosis of congenital hematopoietic failure (such as Fanconi anemia, congenital dyskeratosis, etc.); 3. Other causes of pancytopenia and bone marrow hypoproliferative diseases (such as hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated pancytopenia, etc.); 4. All laboratory or clinically confirmed HIV infection, hepatitis C infection, chronic hepatitis B infection, or evidence of active hepatitis during screening; 5. Cytogenetic evidence of bone marrow abnormalities in clonal blood diseases; 6. History of thromboembolism or current use of anticoagulants within the past 6 months; 7. Accompanied by any one or more malignant diseases; 8. Treatment with another investigational agent within 30 days prior to the first dose of Avatrombopag; 9. Patients who cannot understand or are unwilling to sign an informed consent form (ICF); 10. Pregnant or lactating women; 11. The female patient or the female spouse of the male patient is unable to take effective contraceptive measures; 12. The Investigator considers that there are any other circumstances that may cause the subject to be unable to complete the study or that pose a significant risk to the subject. Exit criteria: 1. The subject or his legal guardian voluntarily requests withdrawal; 2. Violation of inclusion/exclusion criteria; 3. Poor medication compliance; 4. The subject's condition requires treatment with drugs prohibited by the study; 5. Adverse events occur that cause subjects to be unable to continue the study; 6. Other unexplained severe comorbidities; 7. Pregnancy occurs during treatment; 8. Subjects deemed unsuitable for further study by the investigator. Test termination: 1. For safety reasons, the study sponsor proposes to stop the study; 2. The Ethics committee decides to stop the study; 3. The lead researcher decides to stop the study. Investigational drug: Avatrombopag: tablet, specification: 20 mg/ tablet. rhTPO: 15000 units /1 ml. Treatment plan: This clinical trial is planned to carry out a 3-month drug study. A: The Avatrombopag group was given Avatrombopag: 40 mg/ time, once a day, orally, for 3 months. B: Avatrombopag +rhTPO group, Avatrombopag: 40 mg/ time, once a day, orally; rhTPO: 15000U/ time, once a day, subcutaneous injection; Both were 3 months. Efficacy index: Main therapeutic indicators: Overall response rate at 3 months (OR); Secondary efficacy measures: Complete response rate at 3 months (CR); The time of the first occurrence of PR and CR within 3 months of medication; The proportion of subjects who were off platelet transfusion at 3 months; Hemorrhage score records of patients within 3 months of medication; Health-related quality of life score (SF-36 scale). Efficacy criteria: Complete response (CR) : HGB > 100 g/L; ANC > 1.5 × 109 /L; PLT > 100×109/L; Partial response (PR) : disengagement from component blood transfusion and no longer meeting the diagnostic criteria for SAA; Invalid (NR) : SAA diagnostic criteria are still met.
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation. Different medical entities classified as RADs by ORPHA classification are most of them chronic life threating disorders with many unmet needs for their proper clinical management creating an impact on European health systems. RADs present diagnostic challenges and their appropriate management requires from specialised multidisciplinary teams in Centers of expertise. Although there are some examples of well-established national registries on RADs in EU, the lack of recommendations for Rare disease registries implementation and the lack of standards for interoperability has led to the fragmentation or unavailability of data on prevalence, survival, main clinical manifestations or treatments in most of the European countries.
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).