A retrospective study was performed to collect information regarding effica
cy and toxicity of cidofovir (CDV) in allogeneic stem cell transplant patie
nts. Data were available on 82 patients. The indications for therapy were c
ytomegalovirus (CMV) disease in 20 patients, primary preemptive therapy in
24 patients, and secondary preemptive therapy in 38 patients. Of the patien
ts, 47 had received previous antiviral therapy with ganciclovir, foscarnet,
or both drugs. The dosage of CDV was 1 to 5 mg/kg per week followed by mai
ntenance every other week in some patients. The duration of therapy ranged
from 1 to 134 days (median, 22 days). Ail patients received probenecid and
prehydration. Ten of 20 (50%) patients who were treated for CMV disease (9
of 16 with pneumonia) responded to CDV therapy, as did 25 of 38 (66%) patie
nts who had failed or relapsed after previous preemptive therapy and 15 of
24 (62%) patients in whom CDV was used as the primary preemptive therapy. O
f the patients, 21 (25.6%) developed renal toxicity that remained after ces
sation of therapy in 12 patients. Fifteen patients developed other toxiciti
es that were potentially due to CDV or the concomitantly given probenecid.
No toxicity was seen in 45 (61.6%) patients. Cidofovir can be considered as
second-line therapy in patients with CMV disease failing previous antivira
l therapy. However, additional studies are needed before CDV can be recomme
nded for preemptive therapy. (C) 2001 by The American Society of Hematology
.