The aim of this study was to review the characteristics and outcome of 21 p
atients with invasive mucormycosis treated with amphotericin B colloidal di
spersion (ABCD) in five phase I and phase II studies. Mucormycosis is an in
creasing concern in immunocompromised patients, in whom mortality exceeds 6
0%. The standard treatment has been amphotericin B combined with surgical d
ebridement. Twenty-one patients with invasive mucormycosis treated with ABC
D, a lipid complex of amphotericin B and cholesteryl sulfate, were identifi
ed. Patients were given ABCD on the basis of pre-existing renal insufficien
cy, development of nephrotoxicity during amphotericin B therapy, or fungal
infection that failed to respond to amphotericin B. Response could be evalu
ated in 20 patients, all of whom had bone marrow or organ transplantation,
haematologic malignancies, or diabetes. Infection was disseminated in six p
atients and localised to the sinuses, lower respiratory tract, or skin in t
he other patients. ABCD was given at a mean dose of 4.8 mg/kg per infusion
for a mean duration of 37 days. Twelve of 20 patients responded to ABCD the
rapy. Response rates were similar when patients were treated with ABCD alon
e (4/7) and ABCD combined with surgery (8/13), with more complete response
obtained in the latter group. No difference in response rate was observed i
n leukaemic patients (3/5) or transplant recipients (6/10) compared to diab
etics (3/5). No renal or hepatic toxicity was observed. These results compa
re favourably with the results of standard treatment and suggest that ABCD
combined with surgery may be a useful therapy in patients with mucormycosis
.