The Children's Hospital University of Illinois
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Northwestern University - Ann and Robert H. Lurie Children's Hospital of Chicago
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Medical College of Wisconsin - Children's Hospital of Wisconsin
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Rush University Children's Hospital
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University of Chicago - Comer Children's Hospital
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University of Iowa - Stead Family Children's Hospital
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University of Minnesota - Masonic Children's Hospital
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type.
September 1, 2020- More Dyskeratosis Congenita - University of Minnesota - Masonic Children's Hospital posts
University of Wisconsin - American Family Children's Hospital
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death. Bone marrow transplantation (BMT) can cure the blood system but can make the lung and liver disease and risk of cancer worse, because of DNA damaging agents such as alkylators and radiation that are typically used in the procedure. Based on the biology of DC, we hypothesize that it may be possible to avoid these DNA damaging agents in patients with DC, and still have a successful BMT. In this protocol we will test whether a regimen that avoids DNA alkylators and radiation can permit successful BMT without compromising survival in patients with DC.
September 1, 2020- More Dyskeratosis Congenita – University of Wisconsin - American Family Children's Hospital posts