Antithymocyte Globulin and Cyclosporine for Severe Aplastic Anemia
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- 5 March 2003
- journal article
- clinical trial
- Published by American Medical Association (AMA) in JAMA
- Vol. 289 (9), 1130-1135
- https://doi.org/10.1001/jama.289.9.1130
Abstract
Research from JAMA — Antithymocyte Globulin and Cyclosporine for Severe Aplastic Anemia — Association Between Hematologic Response and Long-term Outcome — ContextIn most patients, aplastic anemia results from T-cell–mediated immune destruction of bone marrow. Aplastic anemia can be effectively treated by stem cell transplantation or immunosuppression.ObjectiveTo assess long-term outcomes after immunosuppressive therapy.Design, Setting, and PatientsCohort of 122 patients (31 were ≤18 years and 91 were >18 years) with severe aplastic anemia, as determined by bone marrow cellularity and blood cell count criteria, were enrolled in a single-arm interventional research protocol from 1991 to 1998 at a federal government research hospital.InterventionsA dose of 40 mg/kg per day of antithymocyte globulin administered for 4 days, 10 to 12 mg/kg per day of cyclosporine for 6 months (adjusted for blood levels), and a short course of corticosteroids (1 mg/d of methylprednisolone for about 2 weeks).Main Outcome MeasuresSurvival, improvement of pancytopenia and transfusion-independence, relapse, and evolution to other hematologic diseases.ResultsResponse rates were 60% at 3 months after initiation of treatment, 61% at 6 months, and 58% at 1 year. The blood cell counts of patients who responded no longer satisfied severity criteria and they were transfusion-independent. Overall actuarial survival at 7 years was 55%. Survival was associated with early satisfaction of response criteria (86% vs 40% at 5 years; P<.001) and by blood counts at 3 months (reticulocyte count or platelet count of >50 × 103/µL predicted survival at 5 years of 90% [64/71] vs 42% [12/34] for patients with less robust recovery [P<.001 by log-rank test]). There were no deaths among responders more than 3 years after treatment. Relapse was common, but severe pancytopenia usually did not recur. Relapse did not influence survival. Thirteen patients showed evolution to other hematologic diseases, including monosomy 7.ConclusionsApproximately half of patients with severe aplastic anemia treated with antithymocyte globulin and cyclosporine have durable recovery and excellent long-term survival. These outcomes were related to the quality of hematologic recovery.Keywords
This publication has 33 references indexed in Scilit:
- Treatment of acquired severe aplastic anemia: Bone marrow transplantation compared with immunosuppressive therapy-the European group for blood and marrow transplantation experienceSeminars in Hematology, 2000
- Alternative-donor hematopoietic stem-cell transplantation for severe aplastic anemiaSeminars in Hematology, 2000
- Current status of allogeneic bone marrow transplantation in acquired aplastic anemiaSeminars in Hematology, 2000
- Myelodysplastic syndrome and aplastic anemia: Distinct entities or diseases linked by a common pathophysiology?Seminars in Hematology, 2000
- Rabbit antithymocyte globulin (r‐ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapyBritish Journal of Haematology, 1999
- Marrow transplantation from unrelated donors for patients with severe aplastic anemia who have failed immunosuppressive therapyTransplantation and Cellular Therapy, 1999
- Unrelated donor bone marrow transplantation to treat severe aplastic anaemia in children and young adultsBritish Journal of Haematology, 1996
- Late Clonal Complications in Severe Aplastic AnemiaLeukemia & Lymphoma, 1994
- Relapse of aplastic anaemia after immunosuppressive treatment: a report from the European Bone Marrow Transplantation Group SAA Working PartyBritish Journal of Haematology, 1993
- TREATMENT OF APLASTIC ANÆMIA BY ANTILYMPHOCYTE GLOBULIN WITH AND WITHOUT ALLOGENEIC BONE-MARROW INFUSIONSThe Lancet, 1977