BACKGROUND: Both immunosuppressive therapy and bone marrow transplantation are accepted treatments for patients with aplastic anemia. Choosing one of these therapies for a given patient depends not only on donor availability but also on such factors as patient age. OBJECTIVE: To compare survival rates and long-term complications after bone marrow transplantation or immunosuppressive therapy in patients with acquired aplastic anemia and to identify prognostic factors associated with improved survival. DESIGN: Center-based, retrospective analysis. SETTING: Referral center for patients with aplastic anemia. PATIENTS: 395 patients with acquired aplastic anemia. INTERVENTION: Bone marrow transplant from an HLA-identical, related donor or immunosuppressive therapy. MEASUREMENTS: Kaplan-Meier survival curves, results of log rank tests, and cumulative incidence curves. RESULTS: Of 168 bone marrow transplant recipients, 89% had sustained engraftment. Forty-six patients developed grade II to IV acute graft-versus-host disease, and 68 developed chronic graft-versus-host disease that required therapy. Of 227 patients who received immunosuppressive therapy, 44% achieved a complete, partial, or minimal response. Fifty-four percent died or had no response to therapy. Actuarial survival at 15 years was 69% for bone marrow transplant recipients and 38% for patients receiving immunosuppressive therapy (P < 0.001). Improved survival was associated with having bone marrow transplantation as primary therapy, being younger, having no transfusion before transplantation, and having a higher absolute neutrophil count. Disease duration, year of therapy, sex, refractoriness to platelet transfusions, and previous treatment with androgens or corticosteroids did not significantly affect survival. CONCLUSIONS: Data from this center suggest that bone marrow transplantation may be preferred for younger patients with acquired aplastic anemia who have matched, related donors. Long-term survival is excellent for patients who respond to either form of therapy.
BACKGROUND: Both immunosuppressive therapy and bone marrow transplantation are accepted treatments for patients with aplastic anemia. Choosing one of these therapies for a given patient depends not only on donor availability but also on such factors as patient age. OBJECTIVE: To compare survival rates and long-term complications after bone marrow transplantation or immunosuppressive therapy in patients with acquired aplastic anemia and to identify prognostic factors associated with improved survival. DESIGN: Center-based, retrospective analysis. SETTING: Referral center for patients with aplastic anemia. PATIENTS: 395 patients with acquired aplastic anemia. INTERVENTION: Bone marrow transplant from an HLA-identical, related donor or immunosuppressive therapy. MEASUREMENTS: Kaplan-Meier survival curves, results of log rank tests, and cumulative incidence curves. RESULTS: Of 168 bone marrow transplant recipients, 89% had sustained engraftment. Forty-six patients developed grade II to IV acute graft-versus-host disease, and 68 developed chronic graft-versus-host disease that required therapy. Of 227 patients who received immunosuppressive therapy, 44% achieved a complete, partial, or minimal response. Fifty-four percent died or had no response to therapy. Actuarial survival at 15 years was 69% for bone marrow transplant recipients and 38% for patients receiving immunosuppressive therapy (P < 0.001). Improved survival was associated with having bone marrow transplantation as primary therapy, being younger, having no transfusion before transplantation, and having a higher absolute neutrophil count. Disease duration, year of therapy, sex, refractoriness to platelet transfusions, and previous treatment with androgens or corticosteroids did not significantly affect survival. CONCLUSIONS: Data from this center suggest that bone marrow transplantation may be preferred for younger patients with acquired aplastic anemia who have matched, related donors. Long-term survival is excellent for patients who respond to either form of therapy.
Authors: Lauri M Burroughs; Ann E Woolfrey; Barry E Storer; H Joachim Deeg; Mary E D Flowers; Paul J Martin; Paul A Carpenter; Kris Doney; Frederick R Appelbaum; Jean E Sanders; Rainer Storb Journal: Br J Haematol Date: 2012-04-26 Impact factor: 6.998
Authors: H Joachim Deeg; Margaret O'Donnell; Jakub Tolar; Rajni Agarwal; Richard E Harris; Stephen A Feig; Mary C Territo; Robert H Collins; Peter A McSweeney; Edward A Copelan; Shakila P Khan; Ann Woolfrey; Barry Storer Journal: Blood Date: 2006-05-09 Impact factor: 22.113
Authors: A Piccin; A O'Marcaigh; O Smith; J O'Riordan; M Crowley; E Vandenberg; N Gardiner; S McCann Journal: Ir J Med Sci Date: 2005 Jul-Sep Impact factor: 1.568
Authors: S H Shin; Y W Jeon; J H Yoon; S A Yahng; S E Lee; B S Cho; K S Eom; Y J Kim; S Lee; C K Min; H J Kim; S G Cho; D W Kim; W S Min; J W Lee Journal: Bone Marrow Transplant Date: 2016-06-27 Impact factor: 5.483
Authors: D Gómez-Almaguer; A Vázquez-Mellado; J R Navarro-Cabrera; V Abello-Polo; V Milovic; J García; A L Basquiera; S Saba; G Balladares; J Vela-Ojeda; S Gómez; A Karduss-Aurueta; A Bustinza-Álvarez; A Requejo; L Feldman; J C Jaime-Pérez; S Yantorno; G Kusminsky; C H Gutiérrez-Aguirre; J Arbelbide; J Martinez-Rolon; G Jarchum; G Jaimovich; L Riera; E Pedraza-Mesa; L Villamizar-Gómez; M Á Herrera-Rojas; M M Gamboa-Alonso; C Foncuberta; G Rodríguez-González; M A García Ruiz-Esparza; E Hernández-Maldonado; M Paz-Infanzón; E González-López; G J Ruiz-Argüelles Journal: Bone Marrow Transplant Date: 2016-08-22 Impact factor: 5.483