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Use of amphotericin B colloidal dispersion in children
E S Sandler1, M M Mustafa, I Tkaczewski
1University of Texas Southwestern Medical Center at Dallas and Children's Medical Center of Dallas, USA.
Insights
Amphotericin B colloidal dispersion (ABCD) showed significantly less renal toxicity in children with febrile neutropenia compared to traditional amphotericin B. This new lipid-based antifungal agent was well-tolerated and effective in high-risk pediatric patients.
Area of Science:
- Pediatric Infectious Diseases
- Mycology
- Pharmacology
Background:
- Fungal infections pose a significant risk to children with febrile neutropenia.
- Traditional amphotericin B, while effective, is associated with substantial renal toxicity.
- Novel antifungal formulations are needed to improve treatment outcomes and reduce adverse effects in pediatric oncology patients.
Purpose of the Study:
- To evaluate the safety and efficacy of amphotericin B colloidal dispersion (ABCD), a new lipid-based formulation, in pediatric patients.
- To compare the renal toxicity of ABCD with conventional amphotericin B in children with febrile neutropenia.
- To assess the tolerability and effectiveness of ABCD in children with presumed or proven fungal infections across various clinical scenarios.
Main Methods:
- A prospective, randomized trial compared ABCD to amphotericin B in 49 children with febrile neutropenia.
- An additional 70 children received ABCD in open-label studies for reasons including treatment failure, renal impairment, or dose-escalation.
- Comprehensive data on treatment response and toxicity were collected for all participants.
Main Results:
- The randomized trial demonstrated significantly lower renal toxicity with ABCD (12.0%) compared to amphotericin B (52.4%, P = 0.003).
- In open-label studies, 80% of patients experienced adverse symptoms, primarily infusion-related, with only 12% reporting nephrotoxicity.
- ABCD was tolerated at doses up to five times higher than conventional amphotericin B.
Conclusions:
- Amphotericin B colloidal dispersion (ABCD) offers a favorable safety profile, particularly regarding renal toxicity, in pediatric patients.
- ABCD is well-tolerated and effective, even at higher doses, in managing fungal infections in high-risk children.
- Further randomized trials are warranted to fully establish the role of ABCD and other liposomal antifungal agents in pediatric care.
Purpose:
To describe the experience with a new lipid-based amphotericin product (amphotericin B colloidal dispersion or ABCD) in children with fever and neutropenia who are at high risk for fungal infection.
Patients And Methods:
Forty-nine children with febrile neutropenia were treated in a prospective, randomized trial comparing ABCD with amphotericin B. An additional 70 children with presumed or proven fungal infection were treated with 5 different open-label studies of ABCD. Patients were registered into these studies for reasons of: 1) failure to respond to amphotericin B; 2) development of nephrotoxicity or preexisting renal impairment; or 3) willingness to participate in a dose-escalation study. Extensive data detailing response and toxicity were collected from each patient.
Results:
In the randomized trial, there was significantly less renal toxicity in the children receiving ABCD than in those receiving amphotericin B (12.0% vs. 52.4% [P = 0.003]). Other adverse symptoms were not significantly different. In the additional open-label studies, although 80% of patients receiving ABCD reported some adverse symptom, the majority of these were infusion related, and nephrotoxicity was reported in only 12% of these patients.
Conclusions:
ABCD was well-tolerated at doses up to 5 times greater then those usually tolerated with amphotericin B. Renal toxicity was markedly less than expected, and there were no other unexpected severe toxicities. Further randomized studies are needed to further define the role of this and other liposomal products in children.