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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,
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
SOURCE: National Library of Medicine. NOTICE: This material may be protected
by Copyright Law (Title 17, U.S.Code).
Copyright © 1996 – National
Library of Medicine. Reproduced under license with the National Library of
Medicine, Bethesda, MD.
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