Non-myeloablative haematopoietic stem cell transplantation for severe aplastic anaemia with various complications.
We report a 20-year-old-male with severe aplastic anaemia who was treated with nonmyeloablative haematopoietic stem cell transplantation (NSCT) from a sibling donor. As the patient presented with complications consisting of mental retardation, severe obesity, a bone fracture, and recurrent infections, we selected NSCT instead of a myeloablative regimen, to reduce regimen-related toxicity (RRT). Conditioning therapy consisting of busulfan, fludarabine, antithymocyte globulin and FK506 was used to obtain immune suppression. RRT was limited and he is now in complete remission 19 months after NSCT. On day 91, he developed chronic graft-vs.-host disease; it was resolved by the combination of FK506, corticosteroids, and mycophenolate mofetil. Our experience contributes to the growing interest in NSCT as a modality for treating not only malignant haematological disorders associated with complications, but also nonmalignant haematological diseases.
D000328:Adult; D000741:Anemia, Aplastic / Q000150:complications / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D002066:Busulfan; D005260:Female; D050723:Fractures, Bone / Q000150:complications; D006084:Graft Rejection / Q000517:prevention & control; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000209:etiology; D016179:Granulocyte Colony-Stimulating Factor; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008607:Intellectual Disability / Q000150:complications; D008297:Male; D008775:Methylprednisolone; D009173:Mycophenolic Acid / Q000031:analogs & derivatives / Q000627:therapeutic use; D009765:Obesity / Q000150:complications; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D011239:Prednisolone; D012074:Remission Induction; D035781:Siblings; D013601:T-Lymphocytes; D016559:Tacrolimus / Q000627:therapeutic use; D014019:Tissue Donors; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods*; D014740:Vidarabine / Q000031:analogs & derivatives*
D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor; D011239:Prednisolone; D014740:Vidarabine; D002066:Busulfan; D009173:Mycophenolic Acid; C024352:fludarabine; D016559:Tacrolimus; D008775:Methylprednisolone