Aplastic Anemia
Pathophysiology
Progressive telomere shortening in aplastic anemia.
Ball SE, Gibson FM, Rizzo S, et al.
Blood. 1998;91:3582-3592.
Description: Correlation between telomere loss and disease duration in patients with acquired aplastic anemia.
PubMed citation number: 9572992
Antithymocyte globulin with or without cyclosporine A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia.
Frickhofen N, Heimpel H, Kaltwasser JP, et al.
Blood. 2003;101:1236-1242.
Description: Eleven-year follow-up on patients in 1991 study by the German Aplastic Anemia Study Group; further information on relapse and secondary malignancies.
PubMed citation number: 12393680
Haemopoietic growth factor production by normal and aplastic anaemia stroma in long-term bone marrow culture.
Gibson FM, Scopes J, Daly S, et al.
Br J Haematol. 1995;91:551-561.
Description: Differences in microenvironment of normal bone marrow stroma and stroma of patients with aplastic anemia.
PubMed citation number: 8555053
Increased frequencies of glutathione S-transferase (GSTMI and GSTTI) gene deletions in Korean patients with acquired aplastic anemia.
Lee KA, Kim SH, Woo HY, et al.
Blood. 2001;98:3483-3485.
Description: Patients with aplastic anemia have an increased incidence of homozygous deletions of glutathione S-transferase.
PubMed citation number: 11719393
Intracellular interferon-gamma in circulating and marrow T cells detected by flow cytometry and the response to immunosuppressive therapy in patients with aplastic anemia.
Sloand E, Kim S, Maciejewski JP, et al.
Blood. 2002;100:1185-1191.
Description: Presence of intracellular intereferon-gamma in T cells of aplastic anemia patients may predict response to immunosuppression and onset of relapse.
PubMed citation number: 12149196
Late complications following treatment for severe aplastic anemia (SAA) with high-dose cyclophosphamide (CY): follow-up of a randomized trial.
Tisdale JT, Maciejewski JP, Nunez O, et al.
Blood. 2002;100:4668-4670.
Description: Incidence of relapse and secondary malignancies may not be reduced by treatment with high-dose cyclophosphamide.
PubMed citation number: 12393567
Mutations in TERT, the gene for telomerase reverse transcriptase, in aplastic anemia
Yamaguchi H, Calado RT, Ly H, et al.
N Engl J Med. 2005;352:1413-1424.
Description: Mutations (which cause an amino acid change in the corresponding protein) were identified in TERT, the gene for the telomerase reverse transcriptase catalytic enzyme, among 7 unrelated patients with aplastic anemia; these mutations are thought to be risk factors for marrow failure.
PubMed citation number: 15814878
Diagnosis
Late presentation of dyskeratosis congenita as apparently acquired aplastic anaemia due to mutations in telomerase RNA.
Fogarty PF, Yamaguchi H, Wiestner A, et al.
Lancet. 2003;362:1628-1630.
Description: Bone marrow failure of variable severity due to dyskeratosis congenita, historically characterized by associated physical anomalies and early pancytopenia, may be present in otherwise phenotypically normal adults, and can masquerade as acquired aplastic anemia.
PubMed citation number: 14630445
Treatment
Treatment of acquired severe aplastic anemia: bone marrow transplantation compared with immunosuppressive therapy—The European Group for Blood and Marrow Transplantation experience.
Bacigalupo A, Brand R, Oneto R, et al.
Semin Hematol. 2000;37:69-80.
Description: Compares outcomes of 3,669 patients with aplastic anemia treated in Europe with immunosuppressive therapy or bone marrow transplantation between 1976 and 1998.
PubMed citation number: 10676912
Antilymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony – stimulating factor for severe aplastic anemia: an update of the GITMO/EVMT study on 100 patients.
Bacigalupo A, Bruno B, Saracco P, et al.
Blood. 2000;95:1931-1934.
Description: Evaluates 100 patients (median age, 16 years) with severe aplastic anemia. Treatment with antlymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony-stimulating factor results in high response raters and overall survival.
PubMed citation number: 10706857
Complete remission in severe aplastic anemia after high-dose cyclophosphamide without bone marrow transplantation.
Brodsky RA, Sensenbrenner LL, Jones RJ.
Blood. 1996;87:491-494.
Description: Treatment with high-dose cyclophosphamide may offer long-term hematologic remission, prevention of relapse, and relatively minimal secondary malignancies.
PubMed citation number: 8555470
Primary treatment of acquired aplastic anemia: outcomes with bone marrow transplantation and immunosuppressive therapy.
Doney K, Leisenring W, Storb R, et al.
Ann Intern Med. 1997;126:107-115.
Description: A comparison of 168 patients who received bone marrow transplantation with 227 patients who received immunosuppressive therapy. Actuarial survival and predictors of survival are emphasized in this study from Seattle.
PubMed citation number: 9005744
Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine: the German Aplastic Anemia Study Group.
Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al.
N Engl J Med. 1991;324:1297-1304.
Description: Randomized, multicenter trial of 84 patients showing benefit to addition of cyclosporine to antilymphocyte globuline and methylprednisolone.
PubMed citation number: 2017225
Long term outcome of acquired aplastic anaemia in children: comparison between immunosuppressive therapy and bone marrow transplantation.
Kojima S, Horibe K, Inaba J, et al.
Br J Haematol. 2000;111:321-328.
Description: One hundred children are included in this trial. Up-front bone marrow transplantation results in a significantly better 10-year failure free survival and overall survival than up-front treatment with immunosuppressive therapy.
PubMed citation number: 11091219
Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: a report from the EBMT SAA Working Party.
Locasciulli A, van’t Veer L, Bacigalupo A, et al.
Bone Marrow Transplant. 1990;6:211-217.
Description: A comparative analysis of 304 children under the age of 15 years who received bone marrow transplants or immunosuppression.
PubMed citation number: 2252962
Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporine for treatment of patients with nonsevere aplastic anemia: a report from the European Blood an Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party.
Marsh J, Schrezenmeier H, Marin P, et al.
Blood. 1999;93:2191-2195.
Description: Randomized, multicenter trial of 115 patients showing that combination of antithymocyte globulin and cyclosporine is superior to treatment with cyclosporine alone.
PubMed citation number: 10090926
Bone marrow transplantation for severe aplastic anemia: has outcome improved?
Passweg JR, Socie G, Henterberger W, et al.
Blood. 1997;90:858-864.
Description: Comparative survival data on patients with aplastic anemia who received bone marrow transplantation in time cohorts of 1976-1980, 1981-1987 and 1988-1992.
PubMed citation number: 9226187
Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia.
Rosenfeld SJ, Kimball J, Vining D, et al.
Blood. 1995;85:3058-3065.
Description: Triple therapy with anithymocyte globulin, methylprednisolone, and cyclosporine results in a high response rate when used as first-line therapy in patients with severe acquired aplastic anemia.
PubMed citation number: 7756640
Review
The pathophysiology of acquired aplastic anemia.
Young NS, Maciejewski J.
N Engl J Med. 1997;336:1365-1372.
Description: Review article.
PubMed citation number: 9134878
Aplastic anaemia.
Brodsky RA, Jones RJ.
Lancet. 2005;365:1647-1656.
Description: Review article.
PubMed citation number: 15885298