No therapeutic agent has yet been established as the definitive
therapy for
adenovirus infections. We describe the clinical experience of 13 immunocompromised patients who received
CMX001 (
hexadecyloxypropyl cidofovir), an orally bioavailable
lipid conjugate of
cidofovir, for adenovirus disease. We retrospectively analyzed 13 patients with adenovirus disease and
viremia treated with
CMX001; data were available for ≥ 4 weeks after initiation of
CMX001 therapy. Virologic response (VR) was defined as a 99% drop from baseline or undetectable adenovirus
DNA in serum. The median age of the group was 6 years (range, 0.92-66 years). One patient had
severe combined immunodeficiency, 1 patient was a small bowel transplant recipient, and 11 were allogeneic stem cell transplant recipients. Adenovirus disease was diagnosed at a median of 75 days (range, 15-720 days) after
transplantation. All patients received i.v.
cidofovir for a median of 21 days (range, 5-90 days) before
CMX001 therapy. The median absolute lymphocyte count at
CMX001 initiation was 300 cells/μL (range, 7-1500 cells/μL). Eight patients (61.5%) had a ≥ 1 log10 drop in viral load after the first week of
therapy. By week 8, 9 patients (69.2%) demonstrated a VR, with a median time to achieve VR of 7 days (range, 3-35 days). The change in absolute lymphocyte count was inversely correlated with the change in log10 viral load only at week 6 (r = -0.74; P = .03). Patients with VR had longer survival than those without VR (median 196 days versus 54.5 days; P = .04). No serious adverse events were attributed to
CMX001 during
therapy.
CMX001 may be a promising therapeutic option for the treatment of severe adenovirus disease in immunocompromised patients.