What is the most effective treatment of children with severe aplastic anemia who lack a matched sibling donor?
The most effective treatment currently available for children with severe aplastic anemia is bone marrow transplantation using a sibling donor who is completely matched with the patient at the major histocompatibility complex. Only a minority of patients, however, will have such a donor and other treatments which have been used include the use of single agent immunosuppression, multi-agent immunosuppression, multi-agent immunosuppression with hemopoietic growth factors, and the use of allogeneic stem cell transplantation using mismatched family members, unrelated individuals or placental blood. Of these approaches the most promising appears to be the use of combination immunosuppressive therapy plus hemopoietic growth factors. This paper reviews the current results obtained in using these various methods of treating children with severe aplastic anemia who lack a matched sibling donor.
Johnson
F L
FL
Division of Pediatric Hematology/Oncology, Oregon Health Sciences University, Portland 97201-3098, USA.
D000741:Anemia, Aplastic / Q000188:drug therapy / Q000628:therapy*; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002648:Child; D002986:Clinical Trials as Topic; D003131:Combined Modality Therapy; D016572:Cyclosporine / Q000627:therapeutic use; D005312:Fetal Blood / Q000166:cytology; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000517:prevention & control; D016178:Granulocyte-Macrophage Colony-Stimulating Factor / Q000627:therapeutic use; D016298:Hematopoietic Cell Growth Factors / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006648:Histocompatibility; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007377:Interleukin-3 / Q000627:therapeutic use; D008775:Methylprednisolone / Q000627:therapeutic use; D009679:Nuclear Family; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use; D013601:T-Lymphocytes; D014184:Transplantation, Homologous
D000961:Antilymphocyte Serum; D016298:Hematopoietic Cell Growth Factors; D007166:Immunosuppressive Agents; D007377:Interleukin-3; C078524:PIXY321 fusion protein, recombinant; D011993:Recombinant Fusion Proteins; D016178:Granulocyte-Macrophage Colony-Stimulating Factor; D016572:Cyclosporine; D008775:Methylprednisolone