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Long-term (5 to 20 years) Evolution of nongrafted aplastic anemias. The
Cooperative Group for the Study of Aplastic and Refractory Anemias
Y Najean and O Haguenauer
Department of Nuclear Medicine and Hematology, Hopital Saint-Louis, Paris,
France.
In the presence of aplastic anemia (AA), therapeutic choices should be
determined while taking into account not only changes for immediate
improvement, but also both the risks for late-occurring complications and
the following quality of life. We report here data concerning a long-term
clinical survey (5 to 18 years with a median of 12 years) including 156
nongrafted patients receiving androgen therapy; all patients were alive
more than 5 years after diagnosis (40% of patients included at time of
diagnosis in our multicentric analysis). Between the 5th and the 13th year
follow-up, 21 patients died of various causes either related to AA or to
its treatment: 12 of infection or hemorrhage secondary to pancytopenia (6
relapses and 6 that had never been improved; 2 with paroxysmal nocturnal
hemoglobinuria [PNH]); 5 of leukemia; 1 of a non-Hodgkin's lymphoma; 2 of
late side effects following transfusion (1 acquired immunodeficiency
syndrome and 1 chronic B hepatitis); and a single case of myocardial
infarction (the latter could possibly result of androgen therapy). Thirteen
patients in total developed PNH (among which 10 had clinical symptoms
including 2 deaths, and 3 exhibited only biologic abnormalities). Few
long-term side effects of androgens could be noticed. Adult height was
normal in patients treated during childhood and so was young women's
fertility. No malignant hepatoma occurred. This survey allows the recording
of late spontaneous hematologic improvement (between 5 and 10 years of
evolution). This occurred in 50% of patients that had remained cytopenic 5
years after diagnosis. Although bone marrow stem cell concentration
remained abnormal after 10 years of evolution. 85% of patients had a normal
red blood cell count, 80% a normal polymorphonuclear count, and 66% a
normal platelet count. All patients who did not show late complications had
an excellent quality of life.
Volume 76,
Issue 11,
pp. 2222-2228,
12/01/1990
Copyright © 1990 by The American Society of Hematology
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