Important note: Information in this article was accurate in 1996. The state of the art may have changed since the publication date.
Complete remission in severe aplastic anemia after high-dose cyclophosphamide without bone marrow transplantation.
Blood. 1996 Jan 15;87(2):491-4. Unique Identifier : AIDSLINE MED/96141068 Brodsky RA; Sensenbrenner LL; Jones RJ; Johns Hopkins Oncology Center, Johns Hopkins Medical; Institutions, Baltimore, MD 21287-8967, USA.
Abstract:
Severe aplastic anemia (SAA) can be successfully treated with allogeneic bone marrow transplantation (BMT) or immunosuppressive therapy. However, the majority of patients with SAA are not eligible for BMT because they lack an HLA-identical sibling. Conventional immunosuppressive therapy also has major limitations; many of its remissions are incomplete and relapse or secondary clonal disease is common. Cyclophosphamide is a potent immunosuppressive agent that is used in all BMT conditioning regimens for patients with SAA. Preliminary evidence suggested that high-dose cyclophosphamide, even without BMT, may be beneficial to patients with SAA. Therefore, 10 patients with SAA and lacking an HLA-identical sibling were treated with high-dose cyclophosphamide (45 mg/kg/d) for 4 consecutive days with or without cyclosporine. A complete response (hemoglobin level, > 13 g/dL; absolute neutrophil count, > 1.5 x 10(9)/L, and platelet count > 125 x 10(9)/L) was achieved in 7 of the 10 patients. One of the complete responders died from the acquired immunodeficiency syndrome 44 months after treatment with high-dose cyclophosphamide. The 6 remaining patients are alive and in continuous complete remission, with a median follow-up of 10.8 years (range, 7.3 to 17.8 years). The median time to last platelet transfusion and time to 0.5 x 10(9) neutrophils/L were 85 and 95 days, respectively. None of the complete responders has relapsed or developed a clonal disease. These results suggest that high-dose cyclophosphamide, even without BMT, may be more effective than conventional immunosuppressive therapy in restoring normal hematopoiesis and preventing relapse or secondary clonal disorders. Hence, further studies confirming the efficacy of this approach in SAA are indicated.
Keywords: Acquired Immunodeficiency Syndrome/COMPLICATIONS Adolescence Adult Anemia, Aplastic/COMPLICATIONS/*DRUG THERAPY Bone Marrow/DRUG EFFECTS/PHYSIOLOGY Child Comparative Study Cyclophosphamide/ADMINISTRATION & DOSAGE/PHARMACOLOGY/ *THERAPEUTIC USE Cyclosporine/ADMINISTRATION & DOSAGE/THERAPEUTIC USE Female Follow-Up Studies Human Immunosuppressive Agents/ADMINISTRATION & DOSAGE/PHARMACOLOGY/ *THERAPEUTIC USE Male Regeneration Remission Induction Support, Non-U.S. Gov't Support, U.S. Gov't, P.H.S. Treatment Outcome CLINICAL TRIAL JOURNAL ARTICLE MULTICENTER STUDY RANDOMIZED CONTROLLED TRIAL 960530
M9651059
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Important note: Information in this article was accurate in 1996. The state of the art may have changed since the publication date.
