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Durable treatment-free remission after high-dose cyclophosphamide therapy for previously untreated severe aplastic anemia.

Abstract：

BACKGROUND:Severe aplastic anemia is a life-threatening bone marrow failure disorder. High-dose cyclophosphamide therapy followed by allogeneic bone marrow transplantation cures the disease. However, it requires a suitable donor and carries the risk for graft-versus-host disease. A small pilot study demonstrated that high-dose cyclophosphamide therapy without bone marrow transplantation leads to durable, treatment-free complete remission. OBJECTIVE:To confirm the safety and efficacy of high-dose cyclophosphamide therapy alone in patients with severe aplastic anemia. DESIGN:Uncontrolled clinical trial. SETTING:Three tertiary care hospitals. PATIENTS:19 patients with untreated severe aplastic anemia. INTERVENTION:Cyclophosphamide, 50 mg/kg of body weight per day for 4 consecutive days. MEASUREMENTS:Probability of response and overall survival were measured. Complete remission was defined as normal blood count for age and sex. Partial remission was defined as independence from transfusion and an absolute neutrophil count greater than 0.5 x 10(9) cells/L without growth factor support. Nonresponders were patients who remained transfusion dependent or died. Relapse was defined as no longer meeting criteria for partial or complete remission. RESULTS:The median time to an absolute neutrophil count of 0.5 x 10(9) cells/L was 49 days. The probability of survival was 84% (95% CI, 59% to 95%) at 24 months. The probability of achieving treatment-free remission was 73% (CI, 51% to 91%) at 24 months, and the probability of achieving complete remission was 65% (CI, 39% to 89%) at 50 months. No responding patients have had relapse or have developed secondary clonal disorders. CONCLUSIONS:High-dose cyclophosphamide therapy without bone marrow transplantation produces durable treatment-free remission in severe aplastic anemia. This approach deserves further study in patients with severe aplastic anemia who are not suitable candidates for allogeneic bone marrow transplantation.

journal_name

Ann Intern Med

journal_title

Annals of internal medicine

authors

Brodsky RA,Sensenbrenner LL,Smith BD,Dorr D,Seaman PJ,Lee SM,Karp JE,Brodsky I,Jones RJ

doi

10.7326/0003-4819-135-7-200110020-00006

subject

Has Abstract

pub_date

2001-10-02 00:00:00

pages

477-83

issue

eissn

0003-4819

issn

1539-3704

pii

200110020-00006

journal_volume

135

pub_type

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Durable treatment-free remission after high-dose cyclophosphamide therapy for previously untreated severe aplastic anemia.