Use Of Allogeneic Marrow Transplantation Research Articles

Success of allogeneic marrow transplantation for children with severe aplastic anaemia

Allogeneic marrow transplantation offers curative therapy for children with severe aplastic anaemia (SAA). We report the outcomes of 148 children with SAA who received human leucocyte antigen (HLA)-matched related marrow grafts between 1971 and 2010. Patients were divided into three groups, reflecting changes in conditioning and graft-versus-host disease (GVHD) prophylaxis regimens that occurred over time. Patients in Group 1 were conditioned with cyclophosphamide (CY; 200 mg/kg) followed by 'long' (102 d) methotrexate (MTX). Patients in Groups 2 and 3 received CY alone (Group 2) or combined with anti-thymocyte globulin (Group 3) followed by 'short' (days 1, 3, 6, and 11) MTX and ciclosporin (until day 180). With a median follow-up of 25 years, the 5-year survivals were 66%, 95%, and 100% for Groups 1, 2, and 3, respectively (overall P < 0·0001). The 3-year estimates of graft rejection were 22%, 32%, and 7%, respectively. The probabilities of grades III-IV acute and 2-year chronic GVHD were 15%, 0%, and 3%, and 21%, 21%, and 10%, respectively. Advances in preparative and GVHD prophylaxis regimens, and supportive care during the past 40 years have led to improved outcomes for children with SAA. These results confirm the use of allogeneic marrow transplantation for children with SAA who have HLA-matched related donors.

Current issues and future directions in marrow transplantation.

The use of allogeneic marrow transplantation as treatment for patients with various hematological diseases has increased in recent years [1–6]. A survey by the International Bone Marrow Transplantation Registry [7] estimated the number of transplants carried out through the year 1987 to be in the order of 20000, more than 10000 of these during the years of 1985 through 1987. Marrow transplantation has been employed in most cases (≥ 80%) for therapy of malignant hematologic diseases. Roughly 10% of all transplants have been for the treatment of patients with acquired or inherited marrow dysfunction (aplastic anemia), and 5%–6% have been for treatment of congenital defects of the hematopoietic and immune systems (thalassemia major, severe combined immuno-deficiency disease, and other inborn errors).KeywordsBone Marrow TransplantationAplastic AnemiaAcute GVHDChronic GVHDSevere Combine Immunodeficiency DiseaseThese keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.