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Effectiveness of Immunosuppressive Therapy in Older Patients with Aplastic Anemia

André Tichelli, MD; Gérard Socié, MD; Michel Henry-Amar, MD; Judith Marsh, MD; Jakob Passweg, MD; Hubert Schrezenmeier, MD; Shaun McCann, MD; Jill Hows, MD; Per Ljungman, MD; Pedro Marin, MD; Aruna Raghavachar, MD; Anna Locasciulli, MD; Alois Gratwohl, MD; Andrea Bacigalupo, MD, for the European Group for Blood and Marrow Transplantation Severe Aplastic Anaemia Working Party
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From Kantonsspital Basel, Basel, Switzerland; Hôpital St. Louis, Paris, France; Centre François Baclesse, Caen, France; St. George's Hospital, London, United Kingdom; Universität Ulm, Ulm, Germany; St. James Hospital Trinity College, Dublin, Ireland; Southmead Health Services, Bristol, United Kingdom; Huddinge Hospital, Huddinge, Sweden; Postgraduate School of Haematology, Farreras Valenti, Barcelona, Spain; Ospedale San Gerardo, Monza, Italy; and Ospedale San Martino, Genova, Italy.

Ann Intern Med. 1999;130(3):193-201. doi:10.7326/0003-4819-130-3-199902020-00004
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Background: Immunosuppressive therapy has been used for successful treatment of severe aplastic anemia, but little information is available on outcome in older patients.

Objective: To evaluate outcome in patients older than 50 years of age who received immunosuppressive therapy for aplastic anemia.

Design: Retrospective cohort study.

Setting: 56 centers of the European Group for Blood and Marrow Transplantation (EBMT).

Patients: 810 patients with aplastic anemia reported between 1974 and 1997. Patients were evaluated according to age group: 60 years of age or older (n = 127), 50 to 59 years of age (n = 115), and 20 to 49 years of age (n = 568; reference group).

Intervention: Antilymphocyte globulin, cyclosporine, or both.

Measurements: Survival, cause of death, response to treatment, relapse rate, and risk for late complications were analyzed in all patients and by age group.

Results: The 5-year survival rate was 57% (95% CI, 46% to 66%) in patients 50 to 59 years of age and 50% (CI, 39% to 60%) in patients 60 years of age or older compared with 72% (CI, 68% to 76%) in patients younger than 50 years of age (P < 0.001). Response to therapy, relapse rate, and risk for clonal complications were similar in all three age groups (P > 0.2). Age was significantly associated with an increased risk for death (relative risk compared with patients 20 to 49 years of age, 1.80 [CI, 1.29 to 2.52] for patients 50 to 59 years of age and 2.57 [CI, 1.87 to 3.53] for patients ≥ 60 years of age), mainly because of bleeding or infection (P = 0.02). Response to immunosuppressive therapy in all patients at 12 months was 62% (CI, 58% to 66%); no difference was seen among the age groups in multivariate analysis (P > 0.2). Sixty-six of the 379 responding patients (17%) subsequently had relapse. The risk for clonal disorders at 10 years was 20% (CI, 15% to 27%).

Conclusions: Response to immunosuppression in aplastic anemia is independent of age, but treatment is associated with increased mortality in older patients.


Grahic Jump Location
Five-year survival curves by age groups (115 patients 50 to 59 years of age [solid line], 127 patients ≥ 60 years of age [dotted line], and 568 reference patients 20 to 49 years of age [dashed line]), adjusted for neutrophil count and year of treatment.
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