Background: In a double-blind randomized trial of empirical antifungal Rx for persistently febrile neutropenic pts, CAS was as effective as and better tolerated than L-AmB. We now examine the results of this study in the subgroup of pts with AML.

Methods: Randomization to CAS (70 mg x 1, then 50 mg/d) or L-AmB (3 mg/kg/d) was stratified by risk category [high risk = allogeneic hematopoietic stem cell transplant or relapsed acute leukemia] and use of antifungal prophylaxis. The primary efficacy endpoint was % of treated pts with documented fever and neutropenia who had a successful outcome defined by all the following: successful Rx of baseline (BL) invasive fungal infection (IFI) (if any), no breakthrough (BT) IFI to 7 d post-Rx, survival @7d post-Rx, no premature discontinuation (DC) due to lack of efficacy or study drug toxicity, and fever resolution x 48 hr during neutropenia.

Results: 703/1095 (64%) of the treated pts had AML, including 364/556 (65%) and 339/539 (63%) in the CAS and L-AmB groups, respectively. Demographic characteristics were similar in AML pts in both Rx groups. 27% of CAS and 22% of L-AmB pts with AML were high risk. Median days of Rx were: CAS, 12; L-AmB, 11. The table shows % AML pts with a successful outcome by Rx group.

% AML pts with a successful outcome by Rx group

CAS (N=364)L-AmB (N=339)Difference (CAS - L-AmB)95% CI for difference between treatment groups
† 7/14 CAS and 5/16 L-AmB pts had successful Rx of BL IFI. 
Success Rx of BL IFI † 50 31 19 (−16, 53) 
No BT IFI 93 95 −2 (−5, 2) 
Suvival @ 7d post-Rx 92 88 4 (0, 8) 
No premature DC 87 86 2 (−3, 7) 
Fever resolution 48 46 2 (−5, 9) 
All of the above 40 38 2 (−5, 10) 
CAS (N=364)L-AmB (N=339)Difference (CAS - L-AmB)95% CI for difference between treatment groups
† 7/14 CAS and 5/16 L-AmB pts had successful Rx of BL IFI. 
Success Rx of BL IFI † 50 31 19 (−16, 53) 
No BT IFI 93 95 −2 (−5, 2) 
Suvival @ 7d post-Rx 92 88 4 (0, 8) 
No premature DC 87 86 2 (−3, 7) 
Fever resolution 48 46 2 (−5, 9) 
All of the above 40 38 2 (−5, 10) 
View Large

The composite success rates for the high risk AML pts were 46/99 (46%) for CAS and 31/76 (41%) for L-AmB.

Conclusions: In this post hoc subgroup analysis, CAS provided an effective and generally well-tolerated option for the empirical Rx of persistently febrile neutropenic pts with AML.

Topics:
amphotericin b, amphotericin b liposomal, antifungal agents, brachial plexus neuritis, caspofungin, fever, leukemia, myelocytic, acute, neutropenia, prescriptions, drug, allogeneic hematopoietic stem cell transplant

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2005, The American Society of Hematology
2004
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