HSC Transplants for Nonhematologic Diseases

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HSC Transplants for Nonhematologic Diseases

HSCs are being used to treat patients with nonmalignant, nonhematologic diseases. A list of some of these diseases is included in the table.
HSC Therapy for Nonmalignant Diseases
DiseaseBenefit
Multiple sclerosisSignificant improvement in neurologic function
Systemic sclerosisImproved joint flexibility, reversal of skin tightness, improved quality of life
Lupus erythematosisRemission and survival after 5 years
Crohn's disease Remission in some cases
Myocardial infarctionImproved recovery of left ventricular contractility
Peripheral vascular diseasePain relief, limb salvage, functional improvement
Type 1 diabetes mellitus Increased insulin production, decreased glycosylated hemoglobin
Rheumatoid arthritisReduction in the rate of joint damage
Vasculitis Complete remission in some cases

HSC infusion treatment for patients with severe autoimmune diseases began in the late 1990’s. Most of the transplants have been autologous with minimal myeloablative or nonmyeloablative conditioning. Allogeneic transplants although potentially a cure, are rarely performed due to increased complications such as GVHD and myelodysplasia. Since these are often chronic diseases, treatment mortality needs to be low. In one study, treatment mortality was less than 1% in patients who received nonmyeloablative therapy, 2% for patients treated with low-intensity myeloablative therapy, and 13% with high-intensity myeloablative therapy. The current nonmyeloablative conditioning therapy uses rituximab, cyclophosphamide, and recombinant antithymocyte globulin (rATG).