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Found 20 clinical trials
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Background Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work. Other patients must get blood

cell counts
  • 1 views
  • 15 May, 2022
  • 1 location
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT). The procedure, however, is limited to children younger than the age of 16 because the risks are lower for children than for adults. The purpose of this study is to …

beta-hcg
blood transfusion
anemia
stroke
conjugated bilirubin
  • 95 views
  • 15 May, 2022
  • 1 location
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will …

fludarabine
blood transfusion
anemia
shortening fraction
stroke
  • 46 views
  • 15 May, 2022
  • 1 location
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)

This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of …

neutropenia
antithymocyte globulin
fludarabine
anemia
shwachman-diamond syndrome
  • 0 views
  • 11 May, 2022
  • 10 locations
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.

antithymocyte globulin
fludarabine
blood disorder
anemia
sickle cell anemia
  • 231 views
  • 22 Apr, 2022
  • 1 location
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer. Drugs used in chemotherapy, such as fludarabine phosphate and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the …

neutropenia
lymphoma
bone marrow procedure
imatinib
splenectomy
  • 134 views
  • 30 Apr, 2022
  • 1 location
Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine A and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also …

  • 0 views
  • 17 Jul, 2021
  • 1 location
Diamond Blackfan Anemia Registry (DBAR) (DBAR)

The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA).

white blood cell count
anemia
platelet count
malnutrition
  • 10 views
  • 27 Apr, 2022
  • 1 location
Cancer in Inherited Bone Marrow Failure Syndromes

-Blackfan anemia: pure red cell aplasia; elevated red cell adenosine deaminase. Dyskeratosis congenita: dysplastic nails, lacey pigmentation, leukoplakia; marrow failure. Shwachman-Diamond

neutropenia
anemia
fanca
shwachman-diamond syndrome
cancer
  • 43 views
  • 26 May, 2022
  • 2 locations
Investigation of the Genetics of Hematologic Diseases

The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood and bone marrow as well as health information …

hydroxyurea
blood disorder
hematologic disorder
sickle cell disease
sickle cell anemia
  • 112 views
  • 23 Feb, 2022
  • 1 location