Bone marrow transplantation for severe aplastic anemia: influence of conditioning and graft-versus-host disease prophylaxis regimens on outcome

Abstract

Data for 595 patients with severe aplastic anemia receiving HLA- identical sibling bone marrow transplants were analyzed to determine the effect of pretransplant conditioning and graft-versus-host disease (GVHD) prophylaxis on outcome. Transplants were performed between 1980 and 1987 and reported to the International Bone Marrow Transplant Registry. Three conditioning regimens (cyclophosphamide alone, cyclophosphamide plus limited field radiation, and cyclophosphamide plus total body radiation) were studied; none was associated with superior long-term survival. Three GVHD prophylaxis regimens (methotrexate, cyclosporine, and methotrexate plus cyclosporine) were studied. Recipients of cyclosporine with or without methotrexate had a significantly higher probability of 5-year survival (69%, 95% confidence interval 63% to 74%) than patients receiving methotrexate only (56%, 49% to 62%, P less than .003). Higher survival with cyclosporine resulted from decreased risks of interstitial pneumonia (P less than .0002) and chronic GVHD (P less than .005). Additional risk factors adversely associated with survival included infection pretransplant (P less than .004), use of parous or transfused female donors (P less than .005), older patient age (P less than .005), and 20 or more pretransplant transfusions (P less than .006). These data may prove useful in planning randomized clinical trials and in identifying patients at high-risk of treatment failure.

Unrelated donor bone marrow transplantation for children and adolescents with aplastic anaemia or myelodysplasia

“…Results of allogeneic matched sibling donor BMT for SAA are excellent, with 60-80% long-term survival reported in most series (Storb et al, 1986;McGlave et al, 1987;Gluckman et al, 1992b;Locasciulli et al, 1990). A significant proportion of patients without a suitable family member donor will respond to immunosuppression with combinations of ATG, cyclosporin and steroids (Frickhofen et al, 1991;Rosenfeld et al, 1995;Doney et al, 1992;Gluckman et al, 1992a).…”

“…Allogeneic transplantation from an HLA-matched family member has been shown to be effective in reconstituting normal haemopoiesis in patients with myelodysplastic syndrome (MDS) and severe aplastic anaemia (SAA) (Storb et al, 1986;Gluckman et al, 1992b;Locasciulli et al, 1990;Anderson et al, 1993;Guinan et al, 1989;O'Donnell et al, 1987O'Donnell et al, , 1995. Due to limitations of family size, the majority of patients with these disorders do not have a suitable family member donor, and for these patients the increasing availability of unrelated donors in bone marrow registries worldwide offers another therapeutic choice (Kernan et al, 1993).…”

Periurethral injection of autologous adipose‐derived regenerative cells for the treatment of male stress urinary incontinence: Report of three initial cases

“…[5][6][7][8] Thus far, MSC have mainly been harvested from bone marrow, a tissue source that has many limitations. These include donor-site morbidity in the bone marrow, which limits the amount of marrow that can be obtained , 9 MSC represent less than 0.01% of all nucleated bone marrow cells in healthy volunteers, 10,11 and an extended culture time is required to obtain therapeutic cell doses of MSC by using the ex vivo cell expansion method.…”

Increased urethral resistance by periurethral injection of low serum cultured adipose‐derived mesenchymal stromal cells in rats

“…These include donor-site morbidity in the bone marrow, which limits the amount of marrow that can be obtained. 8,9 In contrast, adipose tissue contains multipotent cells that are similar to MSC, 10,11 and the number of stem cells in adipose tissue is 100-fold higher than that in the bone marrow. This finding has generated significant interest, because unlike bone marrow cells, adipose tissue can be easily and safely harvested in large quantities with minimal morbidity.…”