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Mucolipidosis II (I-cell disease) Fucosidosis; Aspartylglucosaminuria; Alpha-mannosidosis; Other Wolman disease (acid lipase deficiency) Immunodeficiencies. T-cell deficiencies Ataxia-telangiectasia; DiGeorge syndrome; Combined T- and B-cell deficiencies Severe combined immunodeficiency (SCID), all types; Well-defined syndromes Wiskott ...
Stem-cell transplantation was pioneered using bone marrow-derived stem cells by a team at the Fred Hutchinson Cancer Research Center from the 1950s through the 1970s led by E. Donnall Thomas, whose work was later recognized with a Nobel Prize in Physiology or Medicine. Thomas' work showed that bone-marrow cells infused intravenously could ...
Drug therapy may include the medications lenalidomide, antithymocyte globulin, and azacitidine. [3] Some people can be cured by chemotherapy followed by a stem-cell transplant from a donor. [3] About seven per 100,000 people are affected by MDS; about four per 100,000 people newly acquire the condition each year. [4]
acute graft-versus-host disease, in combination with a calcineurin inhibitor and methotrexate, undergoing hematopoietic stem cell transplantation from a matched or 1 allele-mismatched unrelated donor Voxelotor: Global Blood Therapeutics Inc: sickle cell disease: Cabotegravir: ViiV Healthcare Co: pre-exposure prophylaxis for HIV
Hematopoietic stem cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood. [ 16 ] [ 17 ] [ 13 ] It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an ...
Stem-cell therapy uses stem cells to treat or prevent a disease or condition. [1] As of 2024, the only FDA-approved therapy using stem cells is hematopoietic stem cell transplantation. [2] [3] This usually takes the form of a bone marrow or peripheral blood stem cell transplantation, but the cells can also be derived from umbilical cord blood.
Instead, the drugs affect new blood cells that are being made by the bone marrow. [4] When myelosuppression is severe, it is called myeloablation. [5] Many other drugs including common antibiotics may cause bone marrow suppression. Unlike chemotherapy the effects may not be due to direct destruction of stem cells but the results may be equally ...
Younger individuals, if at high risk for dying from CLL, may consider allogeneic hematopoietic stem cell transplantation (HSCT). Myeloablative (bone marrow killing) forms of allogeneic stem cell transplantation, a high-risk treatment using blood cells from a healthy donor, may be curative, but treatment-related toxicity is significant. [80]
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