What are the recommended treatments for candidemia? When should you use an azole vs. an echinocandin?

Comment by InpharmD Researcher

There is moderate and consistent evidence supporting echinocandins as preferred initial treatment for candidemia. Across guidelines, first-line agents include caspofungin, micafungin, and anidulafungin, which have demonstrated early treatment success and more reliable activity against C. glabrata, C. krusei, and other non-albicans species in comparative trials. Fluconazole is generally considered an acceptable initial therapy only for clinically stable, non-neutropenic patients without recent azole exposure and with low risk for resistant species, and it remains the standard step-down therapy once blood cultures clear and susceptibility is confirmed. Voriconazole may be used in place of fluconazole for step-down when C.krusei is identified or when broader azole coverage is needed. Liposomal amphotericin B is reserved for patients who cannot receive an echinocandin or azole, or when tissue penetration or resistance concerns necessitate broader antifungal activity. Overall, treatment selection is generally guided by clinical stability and susceptibility results, with guidelines recommending echinocandins for initial therapy and reserving azoles for step-down once blood cultures have cleared.

PubMed/Google Scholar (and/or Embase): (“candidemia” OR “Candida bloodstream infection” OR “invasive candidiasis”) AND (“echinocandin*” OR caspofungin OR micafungin OR anidulafungin OR “azole*” OR fluconazole OR voriconazole OR isavuconazole OR amphotericin B) AND (guideline* OR consensus OR “systematic review” OR “meta-analysis”); limit to humans/adults, English, and most recent 10 years, then screen for recommendations on initial therapy vs step-down and criteria for azole use.

Background

The European Society of Intensive Care Medicine (ESICM) and the Critically Ill Patients Study Group of the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) published 2019 guidelines for management of invasive candidiasis. For a non-neutropenic, critically ill patient with invasive candidiasis, echinocandins (caspofungin, micafungin, anidulafungin) are the preferred first-line empirical therapy, particularly for patients with septic shock or multiple organ failure. This recommendation is supported by their fungicidal activity, broader spectrum against non-albicans species like C. glabrata and C. krusei, and studies showing a potential mortality benefit in critically ill populations. However, fluconazole is considered a suitable first-line option for critically ill patients with low disease severity (without septic shock) in settings with low fluconazole resistance, provided the patient has had no recent azole exposure. Its value lies in its tolerability and lower cost. If fluconazole is used, a weight-based loading dose of 12 mg/kg, followed by 6 mg/kg maintenance dosing, is strongly recommended to overcome the pharmacokinetic variability common in ICU patients and achieve target drug levels. [1]

According to the 2016 IDSA guidelines for management of candidiasis, echinocandins (caspofungin, micafungin, anidulafungin) are favored as initial therapy for most adult patients with candidemia due to their fungicidal efficacy, favorable safety profile, and the rising concern of fluconazole resistance. Fluconazole is considered an acceptable first-line alternative only for patients who are not critically ill, have had no recent azole exposure, and are at low risk for infection with C. glabrata. For patients who respond well to initial echinocandin therapy, have documented clearance of Candida from the bloodstream, and are infected with a susceptible isolate, step-down therapy to oral fluconazole (or voriconazole for C. krusei) is recommended, typically after 5-7 days. This guideline also notes that while C. parapsilosis has higher echinocandin MICs in vitro, clinical data do not show superiority of fluconazole at the time, so echinocandins remain a recommended first-line option even for this species. [2]

Per the 2025 ECMM guidelines, echinocandins are also strongly recommended as first-line treatment for candidemia, favored for their broad spectrum (including against C. auris), safety, and limited drug interactions. Fluconazole is only marginally recommended for initial therapy due to high rates of treatment failure and increasing antifungal resistance in many regions. However, switching to oral fluconazole or voriconazole is a moderately recommended option for step-down therapy after at least 5 days of echinocandin treatment, provided the patient is hemodynamically stable, has documented bloodstream clearance, and the isolate is susceptible. [3]

Based on the 2017 European Conference on Infections in Leukemia (ECIL) guidelines, echinocandins (anidulafungin, caspofungin, micafungin) are strongly recommended as the first-line therapy for invasive candidiasis, including in hematologic and neutropenic patients, though the quality of evidence is lower for this specific group due to limited trial data. Fluconazole and voriconazole are considered potential alternatives for first-line treatment only in non-severe cases where there has been no prior azole exposure. After species identification, echinocandins remain the preferred drug class for most species. The guideline notes that, while fluconazole was traditionally considered more appropriate for C. parapsilosis, recent observational data showed no difference in outcomes with echinocandin-based regimens, so continuing an echinocandin is acceptable if the patient is responding. Step-down therapy to oral fluconazole (or voriconazole for C. krusei) is recommended after five days of intravenous therapy in stable patients with a susceptible isolate. A critical accompanying recommendation is the prompt removal of central venous catheters, which is independently associated with decreased mortality. [4]

A 2021 Australasian consensus guideline for the diagnosis and management of invasive candidiasis recommends echinocandins as first-line therapy for most adults with candidemia, particularly those who are critically ill, neutropenic, or at higher risk of poor outcomes. This recommendation is based on randomized controlled trials and meta-analyses demonstrating higher overall treatment success with echinocandins compared with azoles, including in patients infected with azole-susceptible isolates. Azole therapy (most commonly fluconazole) is considered appropriate in selected patients who are clinically stable, non-neutropenic, and have no risk factors for azole resistance, and remains a key option for step-down therapy once blood cultures have cleared, the patient is hemodynamically stable, able to tolerate oral therapy, and the infecting Candida species is confirmed to be azole-susceptible. The guidelines also note that species-specific susceptibility is central to treatment selection, as reduced fluconazole susceptibility is more common in Candida glabrata complex and Candida tropicalis, while Pichia kudriavzevii is intrinsically resistant to fluconazole. Liposomal amphotericin B is presented as an alternative option with comparable efficacy to echinocandins but higher toxicity, reserved for situations where echinocandins cannot be used or resistance is suspected. For uncomplicated candidemia, a minimum treatment duration of 14 days after documented bloodstream clearance is recommended, with antifungal choice and de-escalation guided by clinical response and susceptibility results. [5]

According to a 2025 comprehensive review, echinocandins are strongly recommended as first-line treatment for severe candidemia in non-neutropenic patients, supported by a 2012 meta-analysis evidence showing they reduce mortality (odds ratio [OR] 0.50) and improve therapeutic success compared to other antifungals. This recommendation is based on their broad spectrum, favorable safety profile, and low resistance risk. However, the review highlights a critical challenge in intra-abdominal candidiasis, a common presentation in ICU patients. In this specific scenario, echinocandins may have suboptimal efficacy due to significantly lower drug concentrations in peritoneal fluid (up to 33% lower than in serum), which often fail to reach target levels needed to treat common species like C. albicans and N. glabratus. This pharmacokinetic limitation, compounded by physiological changes in critically ill patients, creates a niche for developing antifungal resistance. Consequently, the review suggests optimization strategies such as dose adjustment in obesity or considering amphotericin B formulations for their better peritoneal penetration and anti-biofilm activity in complex abdominal infections. [6], [7]

A 2015 review concluded from a literature review that while echinocandins and fluconazole each have distinct roles in managing candidemia, neither is ideal for every scenario. Based on the available evidence at the time, primarily from the single randomized trial comparing anidulafungin to fluconazole and a patient-level meta-analysis, the authors state that echinocandins had not been shown to be clearly superior to fluconazole for overall treatment, though anidulafungin was at least non-inferior and possibly more effective. The authors note significant nuance for specific situations: for C. glabrata, echinocandins were associated with better clinical success but not improved survival, and emerging echinocandin resistance linked to prior drug exposure was a concerning trend; for C. parapsilosis, despite higher echinocandin MICs, clinical outcomes appeared similar to fluconazole. In critically ill patients, they highlight conflicting data from subgroup analyses. Ultimately, their recommendations advocate for using institution-specific resistance patterns to guide empirical therapy, suggesting an echinocandin is reasonable for critically ill patients or where fluconazole-resistant C. glabrata is prevalent, while fluconazole remains appropriate for stable, non-immunosuppressed patients and for specific sites like endophthalmitis. They strongly endorse a strategy of starting with an echinocandin and stepping down to fluconazole for stable patients with susceptible isolates and emphasize the critical importance of proper, often weight-based, fluconazole dosing. The authors ultimately argue that optimizing the use of existing antifungal agents through better diagnostics and stewardship is paramount, as rapid treatment initiation may be more crucial than the specific agent choice. [8]

A 2012 individual patient-level quantitative review of seven RCTs evaluating antifungal therapy for candidemia and other forms of invasive candidiasis analyzed outcomes in 1,915 patients and assessed the impact of host, organism, and treatment-related factors on mortality and clinical success. In this pooled analysis, overall 30-day mortality was 31.4%, and multivariable logistic regression demonstrated that treatment with an echinocandin antifungal was independently associated with reduced mortality compared with polyenes or triazoles (OR 0.65; 95% confidence interval [CI] 0.45 to 0.94; p= 0.02), while triazole therapy was not associated with a survival benefit. Similar associations were observed for the composite endpoint of clinical and microbiologic success. The survival benefit of echinocandins was consistent across patients with candidemia due to Candida albicans and non-albicans Candida species and across a range of disease severities, as measured by APACHE II scores, although drug class did not significantly affect outcomes in patients with the highest severity of illness. In addition to antifungal choice, removal of a central venous catheter during treatment was independently associated with improved survival (OR 0.50; 95% CI 0.35 to 0.72; p= 0.0001). These findings support the use of an echinocandin as initial therapy for candidemia, particularly when compared with azole therapy, based on observed associations with improved mortality and treatment success in randomized trial data. [9]

A 2015 review concluded that while echinocandins and fluconazole each have distinct roles in managing candidemia, neither is ideal for every scenario. Based on the available evidence at the time, primarily from the single randomized trial comparing anidulafungin to fluconazole and a patient-level meta-analysis, the authors state that echinocandins had not been shown to be clearly superior to fluconazole for overall treatment, though anidulafungin was at least non-inferior and possibly more effective. They note significant nuance for specific situations: for C. glabrata, echinocandins were associated with better clinical success but not improved survival, and emerging echinocandin resistance linked to prior drug exposure was a concerning trend; for C. parapsilosis, despite higher echinocandin MICs, clinical outcomes appeared similar to fluconazole. In critically ill patients, they highlight conflicting data from subgroup analyses. Their recommendations advocate for using institution-specific resistance patterns to guide empirical therapy, suggesting an echinocandin is reasonable for critically ill patients or where fluconazole-resistant C. glabrata is prevalent, while fluconazole remains appropriate for stable, non-immunosuppressed patients and for specific sites like endophthalmitis. They strongly endorse a strategy of starting with an echinocandin and stepping down to fluconazole for stable patients with susceptible isolates and emphasize the critical importance of proper, often weight-based, fluconazole dosing. [10]

References: [1] Martin-Loeches I, Antonelli M, Cuenca-Estrella M, et al. ESICM/ESCMID task force on practical management of invasive candidiasis in critically ill patients. Intensive Care Med. 2019;45(6):789-805. doi:10.1007/s00134-019-05599-w
[2] Pappas PG, Kauffman CA, Andes DR, et al. Clinical Practice Guideline for the Management of Candidiasis: 2016 Update by the Infectious Diseases Society of America. Clin Infect Dis. 2016;62(4):e1-e50. doi:10.1093/cid/civ933
[3] Cornely OA, Sprute R, Bassetti M, et al. Global guideline for the diagnosis and management of candidiasis: an initiative of the ECMM in cooperation with ISHAM and ASM. Lancet Infect Dis. 2025;25(5):e280-e293. doi:10.1016/S1473-3099(24)00749-7
[4] Tissot F, Agrawal S, Pagano L, et al. ECIL-6 guidelines for the treatment of invasive candidiasis, aspergillosis and mucormycosis in leukemia and hematopoietic stem cell transplant patients. Haematologica. 2017;102(3):433-444. doi:10.3324/haematol.2016.152900
[5] Keighley C, Cooley L, Morris AJ, et al. Consensus guidelines for the diagnosis and management of invasive candidiasis in haematology, oncology and intensive care settings, 2021. Intern Med J. 2021;51 Suppl 7:89-117. doi:10.1111/imj.15589
[6] Cabrera-Guerrero JP, García-Salazar E, Hernandez Silva G, et al. Candidemia: An Update on Epidemiology, Risk Factors, Diagnosis, Susceptibility, and Treatment. Pathogens. 2025;14(8):806. Published 2025 Aug 14. doi:10.3390/pathogens14080806
[7] Andes DR, Safdar N, Baddley JW, et al. Impact of treatment strategy on outcomes in patients with candidemia and other forms of invasive candidiasis: a patient-level quantitative review of randomized trials. Clin Infect Dis. 2012;54(8):1110-1122. doi:10.1093/cid/cis021
[8] Eschenauer GA, Nguyen MH, Clancy CJ. Is Fluconazole or an Echinocandin the Agent of Choice for Candidemia. Ann Pharmacother. 2015;49(9):1068-1074. doi:10.1177/1060028015590838
[9] Andes DR, Safdar N, Baddley JW, et al. Impact of treatment strategy on outcomes in patients with candidemia and other forms of invasive candidiasis: a patient-level quantitative review of randomized trials. Clin Infect Dis. 2012;54(8):1110-1122. doi:10.1093/cid/cis021
[10] Demir KK, Butler-Laporte G, Del Corpo O, et al. Comparative effectiveness of amphotericin B, azoles and echinocandins in the treatment of candidemia and invasive candidiasis: A systematic review and network meta-analysis. Mycoses. 2021;64(9):1098-1110. doi:10.1111/myc.13290

Literature Review

A search of the published medical literature revealed 4 studies investigating the researchable question:

What are the recommended treatments for candidemia? When should you use an azole vs. an echinocandin?

Level of evidence

B - One high-quality study or multiple studies with limitations  Read more→



Please see Tables 1-4 for your response.


Isavuconazole Versus Caspofungin in the Treatment of Candidemia and Other Invasive Candida Infections: The ACTIVE Trial

Design

Phase 3, randomized, double-blind, double-dummy, multicenter, noninferiority study

N= 450

Objective

To compare the efficacy and safety of intravenous (IV) isavuconazole followed by oral isavuconazole versus IV caspofungin followed by oral voriconazole in the primary treatment of candidemia and invasive candidiasis

Study Groups

Isavuconazole (n= 199)

Caspofungin (n= 201)

Inclusion Criteria

Male and female patients ≥18 years of age with candidemia or invasive candidiasis with a positive blood or tissue culture within 96 hours prior to randomization, accompanied by clinical signs and symptoms of infection

Exclusion Criteria

Hepatic dysfunction, Candida osteomyelitis, Candida endocarditis, Candida meningitis, severe immunodeficiency, or more than 48 hours of systemic antifungal therapy for the current episode

Methods

Patients were randomized 1:1 to receive either isavuconazole 200 mg IV three times daily on days 1 and 2, then 200 mg IV once daily or caspofungin 70 mg IV on day 1, then 50 mg IV daily, increased to 70 mg daily for patients weighing more than 80 kg. After day 10, nonneutropenic patients could switch to oral therapy: oral isavuconazole 200 mg once daily in the isavuconazole arm, or oral voriconazole 400 mg twice daily on day 1 of oral dosing followed by 200 mg twice daily in the comparator arm. Treatment continued for at least 14 days after the last positive blood culture and could extend to 56 days. Central venous catheter removal was recommended in candidemia. Patients were followed for 6 weeks after end of therapy, with scheduled clinical and laboratory assessments at baseline; days 7, 14, 28, 42, and 56; end of IV therapy; end of therapy; and 2 and 6 weeks post therapy. Blood cultures were obtained daily until two sequential negative cultures on separate days.

Duration

Maximum of 56 days, with follow-up for 6 weeks after the end of therapy

Outcome Measures

Primary: Successful overall response at the end of IV therapy

Secondary: Overall response at 2 weeks after end of treatment, all-cause mortality at days 14 and 56, safety

Baseline Characteristics  mITT Population Isavuconazole (n= 199)

Caspofungin (n= 201)

Candidemia only

170 (85.4%) 163 (81.1%)
Invasive candidiasis ± candidemia

29 (14.6%)

38 (18.9%)
C. albicans

84 (42.2%)

74 (36.8%)
C. tropicalis

41 (20.6%)

38 (18.9%)
C. parapsilosis

26 (13.1%)

27 (13.4%)
C. glabrata

22 (11.1%)

21 (10.4%)
C. krusei

4 (2.0%)

7 (3.5%)
C. guillermondii 0 (0.0%)

5 (2.5%)

Abbreviations: mITT, modified-intent-to-treat.

In the intention-to-treat (ITT) population (isavuconazole n= 221; caspofungin n= 219), baseline demographics and clinical characteristics were similar between treatment arms. Mean age was 58.0 ± 17.5 years with isavuconazole and 57.9 ± 16.9 years with caspofungin, and males comprised 64.7% and 57.5% of patients, respectively. Mean Acute Physiology and Chronic Health Evaluation II (APACHE II) scores were 13.9 ± 7.2 vs 14.1 ± 7.3, neutropenia was present in 11.3% vs 11.0%, and mean body mass index was 24.8 ± 6.1 vs 24.5 ± 6.9 kg/m2 in the isavuconazole and caspofungin arms, respectively.

Results  

Isavuconazole (n= 199)

Caspofungin (n= 201) Adjusted Difference (95% CI)

Overall response at EOIVT

120 (60.3%) 143 (71.1%) -10.8 (-19.9 to -1.8)

Clinical response

152 (76.4%) 169 (84.1%) -8.2 (-15.4 to -0.9)

Microbiological response

141 (70.9%) 172 (85.6%) -14.9 (-22.7 to -7.0)

Overall response at EOT

122 (61.3%) 145 (72.1%) -10.9 (-19.9 to -1.9)

Overall response at 2 weeks after EOT

109 (54.8%) 115 (57.2%) -2.7 (-12.2 to 6.8)

Overall response at 6 weeks after EOT

86 (43.2%) 97 (48.3%) -5.4 (-15.0 to 4.2)

All-cause mortality

Day 14

Day 56

 

29 (14.6%)

61 (30.7%)

 

25 (12.4%)

60 (29.9%)

 

2.5 (−3.8 to 8.9)

1.4 (−7.1 to 10.0)

Abbreviations: CI, confidence interval; EOIVT, end of intravenous treatment; EOT, end of treatment.

In candidemia only, EOIVT success was 64.7% vs 72.4%; in invasive candidiasis with or without candidemia, 34.5% vs 65.8%.

Oral step down occurred in 34.7% of isavuconazole and 39.8% of caspofungin patients. Median total therapy duration was 15 vs 16 days, and median oral duration was 8 days in both groups. Among step down patients, EOIVT success was 84.1% vs 88.8%; at 2 weeks after EOT, 82.6% vs 77.5%. Breakthrough, emergent, or recurrent Candida infections were documented in 5 isavuconazole and 11 caspofungin patients.

Seventy five percent of mITT patients had an intravascular catheter at baseline; approximately 90% had removal during IV therapy in both arms. Median time to negative blood culture was 4 days with isavuconazole and 3 days with caspofungin (log rank p= 0.59). Persistent candidemia at EOIVT occurred in 18.3% vs 17.6% of those with baseline candidemia.

Adverse Events

Approximately 95% of patients in both arms experienced at least one treatment emergent adverse event (TEAE). Study drug related TEAEs occurred in 35.5% of isavuconazole patients and 32.3% of caspofungin patients, serious TEAEs in 50.9% vs 48.2%, and deaths in 30.0% vs 30.9%, respectively. TEAEs related to septic shock (9.1% vs 5.0%) and sepsis (8.2% vs 5.0%) were reported more frequently with isavuconazole. Pyrexia (2.3% vs 0.5%) and infusion site pain (2.3% vs 0%) were more common in the isavuconazole arm, whereas increased serum alkaline phosphatase occurred more often with caspofungin (2.7% vs 0.9%).

Study Author Conclusions

This study did not demonstrate non-inferiority of isavuconazole to caspofungin for primary treatment of invasive candidiasis. Secondary endpoints were similar between both groups.

Critique

The study was well-designed as a multicenter, double-blind trial, providing robust data on the efficacy and safety of isavuconazole versus caspofungin. However, the study did not meet its primary endpoint of non-inferiority, and the exclusion of pediatric patients and the small proportion of patients with neutropenia may limit the generalizability of the findings.

References:
[1] Kullberg BJ, Viscoli C, Pappas PG, et al. Isavuconazole Versus Caspofungin in the Treatment of Candidemia and Other Invasive Candida Infections: The ACTIVE Trial. Clin Infect Dis. 2019;68(12):1981-1989. doi:10.1093/cid/ciy827

Voriconazole versus a Regimen of Amphotericin B Followed by Fluconazole for Candidaemia in Non-Neutropenic Patients: A Randomised Non-Inferiority Trial

Design

Multicenter, randomized, non-inferiority study

N= 422

Objective

To assess the efficacy and safety of primary treatment with intravenous or oral voriconazole compared with amphotericin B followed by fluconazole in non-neutropenic patients with candidaemia

Study Groups

Voriconazole (n= 283)

Amphotericin B/fluconazole (n= 139)

Inclusion Criteria

Patients aged 12 years or older with one or more positive blood culture for Candida species obtained up to 96 h before study entry and clinical findings consistent with infection

Exclusion Criteria

Received more than 2 days of systemic antifungal therapy during the 96 h before the study, failed previous antifungal treatment, unlikely to survive longer than 24 h, receiving drugs interacting with azole antifungals, pregnant, allergy to azoles or amphotericin B, neutrophil count <0.5×10^9/L, AIDS, aplastic anaemia, chronic granulomatous disease, moderate or severe liver disease, severe renal impairment

Methods

Patients were randomly assigned to receive either voriconazole or amphotericin B followed by fluconazole. Voriconazole was given intravenously at 6 mg/kg every 12 h for 24 h, then 3 mg/kg every 12 h, with a switch to oral voriconazole 200 mg twice daily after 3 days. Amphotericin B was given at 0.7–1.0 mg/kg per day, with a switch to fluconazole 400 mg/day after 3-7 days. Treatment lasted at least 2 weeks after the last positive blood culture, up to 8 weeks. Follow-up was until 12 weeks after the end of treatment.

Duration

September 1998 to January 2003

Outcome Measures

Primary: Clinical and mycological response 12 weeks after the end of treatment

Secondary: Time to first negative blood culture, time from randomisation to death

Baseline Characteristics  

Voriconazole (n= 248)

Amphotericin B/fluconazole (n= 122)
Mean age, years (range)

53.6 (13 to 90)

53.3 (13 to 87)
Female

103 (41.5%)

51 (41.8%)
Mean APACHE II score (range)

13.8 ± 6.5 (0 to 41)

14.7 ± 6.6 (2 to 36)

Predisposing factors

Abdominal surgery

Non-abdominal surgery

Non-surgical

 

95 (38%)

32 (13%)

121 (49%)

 

46 (38%)

15 (12%)

61 (50%)

In intensive care unit

119 (48%)

61 (50%)
Mechanically ventilated

89 (36%)

47 (39%)
Candidemia only

239 (96%)

117 (96%)
Candidemia and other sites of invasive candidiasis

9 (4%)

5 (4%)

Pathogen

C albicans

Non-albicans Candida species

C tropicalis

C parapsilosis

C glabrata

C krusei

Other Candida species

≥2 candida species

 

107 (43%)

150 (61%)

53 (21%)

45 (18%)

36 (15%)

4 (2%)

15 (6%)

12 (5%)

 

63 (51%)

61 (50%)

16 (13%)

19 (16%)

21 (17%)

1 (1%)

5 (4%)

3 (3%)

Results  

Voriconazole (n= 248)

Amphotericin B/fluconazole (n= 122) p-value
Primary success rate*

101 (41%)

50 (41%) 0.96

Success by pathogen

C albicans

C glabrata

C parapsilosis

C tropicalis

C krusei

 

46/107 (43%)

12/36 (33%)

24/45 (53%)

17/53 (32%)

1/4 (25%)

 

30/63 (48%)

7/21 (33%)

10/19 (53%)

1/16 (6%)

0/1

 

-

-

-

0.032

-

Secondary success rate†

162 (65%)

87 (71%) 0.25
Success rate at end of treatment‡

173 (70%)

90 (74%) 0.42
Success rate 2 weeks after end of treatment‡

130 (52%)

64 (53%) 0.99
Success rate 6 weeks after end of treatment‡

110 (44%)

56 (46%) 0.78
All-cause 14-week mortality

88 (36%)

51 (42%) 0.23
Favorable responses to treatment and all-cause mortality
 

*Sustained successes as assessed by data-review committee at 12-week follow-up visit only.

†Successes assessed by data-review committee at latest available study visit (including end of therapy, 2 weeks, or 6 weeks after end of treatment if 12-week assessment after end of treatment was not available).

‡Successes assessed by data-review committee.

Adverse Events

All-cause adverse events were higher in the amphotericin B/fluconazole group. Renal events were more frequent in the amphotericin B/fluconazole group (21% vs 8%). Visual events were more frequent in the voriconazole group (4% vs 1%).

Study Author Conclusions

In summary, the results of this large, multicentre study show that voriconazole is as effective as the commonly used strategy of amphotericin B followed by fluconazole in non-neutropenic patients for the treatment of candidaemia, including C albicans and non-albicans Candida species. The broader spectrum of voriconazole, its better safety than amphotericin B, and the availability of intravenous and oral formulations, provide an important new treatment option for candidemia.

Critique

This large, well-designed randomized trial reinforces echinocandins as preferred initial therapy for candidemia due to superior early efficacy. Azoles, including isavuconazole, appear best reserved for oral step-down therapy in stable patients with susceptible isolates rather than upfront treatment, especially in invasive disease.

References:
[1] Kullberg BJ, Sobel JD, Ruhnke M, et al. Voriconazole versus a regimen of amphotericin B followed by fluconazole for candidaemia in non-neutropenic patients: a randomised non-inferiority trial. Lancet. 2005;366(9495):1435-1442. doi:10.1016/S0140-6736(05)67490-9

Anidulafungin versus Fluconazole for Invasive Candidiasis

Design

Randomized, double-blind, non-inferiority trial

N= 261

Objective

To compare anidulafungin with fluconazole in the treatment of invasive candidiasis

Study Groups

Anidulafungin (n= 127)

Fluconazole (n= 118)

Inclusion Criteria

Patients 16 years or older with candidemia or other forms of invasive candidiasis within 96 hours before enrollment

Exclusion Criteria

More than 48 hours of systemic antifungal therapy for the current episode, prophylactic administration of any azole for more than 1 week within 30 days before enrollment, refractory candida infection, elevated levels of hepatic enzymes, Candida krusei infection, or osteomyelitis, endocarditis, or meningitis due to candida species

Methods

Patients received either intravenous anidulafungin 200 mg on day 1 followed by 100 mg daily, or intravenous fluconazole 800 mg on day 1 followed by 400 mg daily, with renal dose adjustment for fluconazole when indicated. Treatment duration was 14 to 42 days and continued for at least 14 days after a negative blood culture and clinical improvement. After at least 10 days of IV therapy, patients meeting prespecified clinical stability criteria could switch to oral fluconazole 400 mg daily at investigator discretion.

Duration

March 2003 to October 2004

Outcome Measures

Primary: Global response (clinical and microbiologic) at the end of intravenous therapy

Secondary: Global response at the end of all study therapy and at follow-up visits at 2 weeks and 6 weeks, per-patient and per-pathogen microbiologic response, death from all causes

Baseline Characteristics

mITT Population

Anidulafungin Group (n= 127)

Fluconazole Group (n= 118)

Female

62 (49%)  58 (49%)

Age, years

57.0 ± 17.0  59.2 ± 16.5

Diabetes mellitus

44 (35%)  30 (25%)

Renal failure or insufficiency

47 (37%)  42 (36%)

Central venous catheter

99 (78%)  99 (78%) 

Broad-spectrum antibiotics

88 (69%)  82 (70%)

APACHE II score

≤20

>20

 

101 (80%)

26 (21%)

 

98 (83%)

20 (17%)

Absolute neutrophil count

>500/mm3

≤500/mm3

 

124 (98%)

3 (2%)

 

114 (97%)

4 (3%)

Abbreviations: APACHE II, Acute Physiology and Chronic Health Evaluation II; mITT, modified intention-to-treat.

The two study groups did not differ significantly in demographic characteristics, duration of treatment, frequency of switch to oral fluconazole, or exposure to oral fluconazole.

Results

 

Fluconazole Group (n= 118)

Anidulafungin Group (n= 127)

Absolute Percent Difference between Treatments (95% CI)

Global response at end of intravenous therapy

71 (60.2%) 96 (75.6%) 15.4 (3.9 to 27.0)

Global response at end of all therapy

67 (56.8%) 94 (74.0%) 17.2 (5.5 to 29.0)

Global response at 2-week follow-up

58 (49.2%) 82 (64.6%) 15.4 (3.1 to 27.7)

Global response at 6-week follow-up

52 (44.1%) 71 (55.9%) 11.8 (−0.6 to 24.3)

Abbreviations: CI, confidence interval.

Anidulafungin showed lower eradication rates for Candida parapsilosis than for other Candida species. No trend was observed between baseline fluconazole minimum inhibitory concentration (MIC) and eradication rates. Among fluconazole recipients, success was similar for isolates with MIC <16 μg/mL versus ≥16 μg/mL (75.7% vs 80.0%), though only five isolates had MIC ≥16 μg/mL. For C. glabrata, fluconazole eradication was 60.9% when MIC <16 μg/mL and 75.0% when MIC ≥16 μg/mL.

Anidulafungin had higher global success rates than fluconazole for all pathogens except C. parapsilosis. The largest difference was for C. albicans (81.1% vs 62.3%, p= 0.02). Global success for C. glabrata was similar (56.3% vs 50.0%). Central venous catheters were removed in 92.6% of patients overall (96.0% vs 89.0%). Among patients with catheters not removed before therapy, success occurred in 75.0% with anidulafungin and 27.3% with fluconazole. Persistent infection at end of intravenous (IV) therapy occurred in 6.3% vs 14.4% (p= 0.06). Early blood culture clearance at days 3 and 7 was numerically higher with anidulafungin but not statistically significant.

Adverse Events

Late complications were uncommon. Endophthalmitis occurred in one patient per group. Recurrent positive cultures occurred in two anidulafungin and one fluconazole patient. One fluconazole patient developed hepatic candidiasis. Treatment-related adverse events were similar (24.4% vs 26.4%). Drug-related hepatic enzyme elevations were more frequent with fluconazole (7.2% vs 1.5%, p= 0.03). Discontinuations due to adverse events occurred in 27 fluconazole and 15 anidulafungin patients (p= 0.02). All-cause mortality was 22.8% with anidulafungin and 31.4% with fluconazole (p= 0.13).

Study Author Conclusions

In conclusion, this study shows that anidulafungin is not inferior to and possibly is more efficacious than fluconazole for the primary treatment of the candidemic form of invasive candidiasis, with a safety profile similar to that of fluconazole.

InpharmD Researcher Critique

The study was well-designed as a randomized, double-blind, non-inferiority trial, providing robust data on the efficacy of anidulafungin compared to fluconazole. However, the small proportion of patients with neutropenia and noncandidemic invasive candidiasis, as well as the exclusion of pediatric patients, may limit the generalizability of the findings. Additionally, the potential center effect should be considered when interpreting the results.

References:
[1] Reboli AC, Rotstein C, Pappas PG, et al. Anidulafungin versus fluconazole for invasive candidiasis. N Engl J Med. 2007;356(24):2472-2482. doi:10.1056/NEJMoa066906

A Randomized Trial Comparing Fluconazole With Amphotericin B For The Treatment Of Candidemia In Patients Without Neutropenia

Design

Multicenter, randomized, comparative trial

N= 237

Objective

To compare amphotericin B with fluconazole as treatment for candidemia in patients without neutropenia

Study Groups

Amphotericin B (n= 103)

Fluconazole (n= 103)

Inclusion Criteria

Age ≥13 years; ≥1 positive blood culture for Candida spp. within the previous 4 days; evidence of systemic illness or focal inflammation between first positive culture and enrollment (e.g., fever, hypotension, or signs of inflammation at infected site)

Exclusion Criteria

Neutrophils <500/mm³; hematologic cancer or major immunodeficiency states; azole/imidazole allergy/intolerance or moderate-to-severe liver disease (per protocol definitions); expected survival <24 hours; recent high-dose exposure to study drugs, prior failed systemic antifungal therapy for same infection, or other protocol-specified medication restrictions; pregnancy/lactation

Methods

Patients were assigned to receive either fluconazole or amphotericin B. Fluconazole was administered at 400 mg daily, initially intravenously with transition to oral therapy when feasible, with dose adjustments for renal function. Amphotericin B was given intravenously at 0.5 to 0.6 mg/kg/day, with allowance for reduced dosing frequency after the first week if the average daily dose was maintained.

Treatment was continued for at least 14 days after clinical resolution of candidemia or after the last positive sterile-site culture. Clinical assessments, blood cultures, and laboratory monitoring were performed throughout therapy, with follow-up visits after treatment completion. Treatment response was determined using predefined clinical and microbiologic criteria, and therapy could be modified or discontinued for toxicity or lack of response.

Duration

Mean days of therapy: amphotericin B 17 ± 1 days; fluconazole 18 ± 1 days

Follow-up after end of therapy: amphotericin B 61 ± 5 days; fluconazole 65 ± 5 days

Outcome Measures

Primary: Response rate (successful outcome vs failure/relapse) in patients meeting all entry criteria (primary-analysis group)

Secondary: Patients receiving ≥5 days of therapy

Baseline Characteristics

 

Amphotericin B (n= 103)

Fluconazole (n= 103)

 

Age, years

60 ± 2 58 ± 2  

Female

42 (40.8%) 59 (57.3%)  

APACHE II score (mean ± SE)

16 ± 1 16 ± 1  

Underlying disease, n

Cancer

Renal failure

Gastrointestinal disease

Pulmonary disease

Cardiovascular disease

Diabetus Mellitus

 

32

41

28

17

14

23

 

33

39

33

21

13

15

 

Risk factors for candidemia, n

Recent use of antibiotics

Recent use of central venous catheter

Recent hyperalimentation

Recent abdominal surgery

Recent non-abdominal surgery

Recent use of corticosteroids

 

100

84

63

35

35

24

 

98

79

57

28

28

22

 

Previous use of amphotericin B, n

23 ± 3

20 ± 3

 

Previous use of fluconazole, n

350 ± 80

400 ± 0

 

Results

Endpoint

Amphotericin B (n= 103)

Fluconazole (n= 103)

p-value

Successful outcome

81/103 (79%) 72/103 (70%) 0.22

Intention-to-treat successful outcome

89/111 (80%) 81/113 (72%) 0.17

Successful outcome (≥5 days therapy)

81/94 (86%) 70/93 (75%) 0.052

Bloodstream infection failed to clear

12 pts 15 pts NS

 

Abbreviations: NS, not stated

Adverse Events

Renal toxicity and electrolyte abnormalities occurred significantly more frequently in patients receiving amphotericin B compared with fluconazole. Elevations in blood urea nitrogen or serum creatinine were observed in 37% of patients treated with amphotericin B versus 2% of those receiving fluconazole (p< 0.001), and hypokalemia occurred in 10% versus 2%, respectively (p= 0.006). Elevations in liver enzyme levels were reported at similar rates between treatment groups (10% with amphotericin B vs 14% with fluconazole; p= 0.43). Treatment-limiting adverse events and serious adverse events were not reported as distinct categories, although protocol-defined toxicity could result in treatment modification or discontinuation.

Study Author Conclusions

In patients without neutropenia and without major immunodeficiency, fluconazole and amphotericin B are not significantly different in their effectiveness in treating candidemia.

InpharmD Researcher Critique

This randomized multicenter trial supports that, in non-neutropenic candidemia, fluconazole can achieve similar overall success to amphotericin B with substantially less nephrotoxicity, but the population was largely C. albicans and not profoundly immunosuppressed, so generalizability to today’s higher-C. glabrata/resistance settings are limited. Practically, it reinforces the modern concept of using an azole when the patient is stable, and the isolate is likely susceptible, while reserving broader agents when resistance/toxicity risks are higher.

References:
[1] Rex JH, Bennett JE, Sugar AM, et al. A randomized trial comparing fluconazole with amphotericin B for the treatment of candidemia in patients without neutropenia. Candidemia Study Group and the National Institute. N Engl J Med. 1994;331(20):1325-1330. doi:10.1056/NEJM199411173312001