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Invasive fungal disease (IFD) still represents an important cause of morbidity and mortality in immunocompromised patients, particularly in patients undergoing antineoplastic chemotherapy or allogeneic hemopoietic stem cell transplantation (allo-HSCT). International guidelines recommend primary antifungal prophylaxis to reduce mortality and morbidity in these patients. Liposomal amphotericin B (L-AmB) can represent a valid alternative for antifungal prophylaxis in pediatric age as its spectrum is extended to both molds and yeasts, has reduced pharmacological interactions with the antineoplastic drugs most frequently used in treatment protocols. All this despite the availability of an intravenous formulation which can ensure complete compliance with the treatment. L-AmB prophylaxis has been proposed with different dosages: 1 mg/kg every other day vs 2.5 mg/kg/dose twice-a-week vs 5 mg/kg/once-a-week)
Invasive fungal disease (IFD) still represents an important cause of morbidity and mortality in immunocompromised patients, particularly in patients undergoing antineoplastic chemotherapy or allogeneic hemopoietic stem cell transplantation (allo-HSCT). In this setting children presents some differences compared to adults in terms of risk factors for IFD: different treatment schemes, age-related comorbidities, diagnostic tools with different sensitivity. International guidelines strongly recommend primary prophylaxis in children at high risk of developing IFD to reduce disease-related morbidity and mortality (patients are considered to be at high risk in the presence of an IFD incidence ≥10%). Although the risk factors are well characterized in the literature and substantially unchanged over time, the local epidemiology must be considered in the evaluation of the best prophylaxis strategy. Drugs presently recommended for antifungal prophylaxis have been poorly studied in children, anyway triazoles (mainly posaconaozle) are indicated as possible prophylactic agents also in pediatrics. However, these drugs can present important interactions with drugs important for the treatment of pediatric leukemias (e.g. vinca alkaloids and posaconazole) and sometimes they lack of specific pediatric formulations, fact that can reduce compliance because of bad taste of the available formulations (oral solutions) or difficulties in swallowing and risk of poor absorption in presence of mucositis (bulky, non-divisible pills or capsules). Liposomal amphotericin B (L-AmB) can represent a valid alternative for antifungal prophylaxis in pediatric age as its spectrum is extended to both molds and yeasts, has reduced pharmacological interactions with the antineoplastic drugs most frequently used in treatment protocols. All this despite the availability of an intravenous formulation only which may require hospital access or activation of the home care team, but which can ensure complete compliance with the treatment. L-AmB prophylaxis has been proposed with different dosages: 1 mg/kg every other day vs 2.5 mg/kg/dose twice-a-week vs 5 mg/kg/once-a-week).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients at high risk of developing IFD | patients at high risk of developing IFD receiving chemotherapy for (1st line or relapse treatment) or allo-HSCT for which primary fungal prophylaxis with Liposomal amphotericin B has been administered between 01/01/2019 to 31/12/2023 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Liposomal Amphotericin B (LAmB) | Drug | collect data on tolerability and effectiveness of liposomal amphotericin B prophylaxis |
|
| Measure | Description | Time Frame |
|---|---|---|
| Tolerability of extended dosing of liposomal amphotericin B | Number of participants with treatment-related adverse events (both clinical and laboratoristic adverse events) as assessed by CTCAE v4.0 | In the period between the first and last administration of L-AmB |
| Measure | Description | Time Frame |
|---|---|---|
| Effectiveness of extended dosing of liposomal amphotericin B | Number of new diagnoses of IFD classified as proven, probable or possible according to EORTC/MSG criteria | Up to 6 months after the last administration of L-AmB |
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Inclusion Criteria:
Exclusion Criteria:
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All pediatric patients who received extended dosing of liposomal amphotericin B prophylaxis from 01/01/2019 to 31/12/2023
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Istituto Giannina Gaslini | Genoa | 16146 | Italy |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31802125 | Background | Donnelly JP, Chen SC, Kauffman CA, Steinbach WJ, Baddley JW, Verweij PE, Clancy CJ, Wingard JR, Lockhart SR, Groll AH, Sorrell TC, Bassetti M, Akan H, Alexander BD, Andes D, Azoulay E, Bialek R, Bradsher RW, Bretagne S, Calandra T, Caliendo AM, Castagnola E, Cruciani M, Cuenca-Estrella M, Decker CF, Desai SR, Fisher B, Harrison T, Heussel CP, Jensen HE, Kibbler CC, Kontoyiannis DP, Kullberg BJ, Lagrou K, Lamoth F, Lehrnbecher T, Loeffler J, Lortholary O, Maertens J, Marchetti O, Marr KA, Masur H, Meis JF, Morrisey CO, Nucci M, Ostrosky-Zeichner L, Pagano L, Patterson TF, Perfect JR, Racil Z, Roilides E, Ruhnke M, Prokop CS, Shoham S, Slavin MA, Stevens DA, Thompson GR, Vazquez JA, Viscoli C, Walsh TJ, Warris A, Wheat LJ, White PL, Zaoutis TE, Pappas PG. Revision and Update of the Consensus Definitions of Invasive Fungal Disease From the European Organization for Research and Treatment of Cancer and the Mycoses Study Group Education and Research Consortium. Clin Infect Dis. 2020 Sep 12;71(6):1367-1376. doi: 10.1093/cid/ciz1008. | |
| 21895857 |
| Label | URL |
|---|---|
| dictionary of the "Common Terminology Criteria for Adverse Events" of the National Cancer Institute | View source |
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| ID | Term |
|---|---|
| D000072742 | Invasive Fungal Infections |
| ID | Term |
|---|---|
| D009181 | Mycoses |
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
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| ID | Term |
|---|---|
| C068538 | liposomal amphotericin B |
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| Background |
| Bochennek K, Tramsen L, Schedler N, Becker M, Klingebiel T, Groll AH, Lehrnbecher T. Liposomal amphotericin B twice weekly as antifungal prophylaxis in paediatric haematological malignancy patients. Clin Microbiol Infect. 2011 Dec;17(12):1868-74. doi: 10.1111/j.1469-0691.2011.03483.x. Epub 2011 Sep 6. |
| 33811813 | Background | Groll AH, Pana D, Lanternier F, Mesini A, Ammann RA, Averbuch D, Castagnola E, Cesaro S, Engelhard D, Garcia-Vidal C, Kanerva J, Ritz N, Roilides E, Styczynski J, Warris A, Lehrnbecher T; 8th European Conference on Infections in Leukaemia. 8th European Conference on Infections in Leukaemia: 2020 guidelines for the diagnosis, prevention, and treatment of invasive fungal diseases in paediatric patients with cancer or post-haematopoietic cell transplantation. Lancet Oncol. 2021 Jun;22(6):e254-e269. doi: 10.1016/S1470-2045(20)30723-3. Epub 2021 Mar 31. |