L-AmB_ Retrospective MUlticenter Study on Mycosis PrOphylaxis

• Conditions: Invasive Fungal Infections; Prophylaxis; Invasive Fungal Disease
• Interventions: Drug: Liposomal Amphotericin B (LAmB)

• Registration Number: NCT06640296
• Lead Sponsor: Istituto Giannina Gaslini

Brief Summary

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)

Detailed Description

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).

Study Timeline

• Study Start: 2023-12-12
• Status Verified: 2024-10
• Study First Submitted: 2024-10-08
• Study First Submitted QC: 2024-10-10
• Last Update Submitted: 2024-10-10
• Study First Posted: 2024-10-14
• Last Update Posted: 2024-10-14
• Primary Completion: 2024-12-31
• Study Completion: 2025-05-31

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

• all patients at high risk of developing IFD receiving chemotherapy for (1st line or relapse treatment) or allo-HSCT for which primary fungal prophylaxis with extended dosing of liposomal amphotericin B has been administered

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Exclusion Criteria

• previous diagnosis of IFD (secondary prophylaxis),
• contraindications to liposomal amphotericin B prophylaxis

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients at high risk of developing IFD	Liposomal Amphotericin B (LAmB)	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

Primary Outcome Measures

Name	Time	Method
Tolerability of extended dosing of liposomal amphotericin B	In the period between the first and last administration of L-AmB	Number of participants with treatment-related adverse events (both clinical and laboratoristic adverse events) as assessed by CTCAE v4.0

Secondary Outcome Measures

Name	Time	Method
Effectiveness of extended dosing of liposomal amphotericin B	Up to 6 months after the last administration of L-AmB	Number of new diagnoses of IFD classified as proven, probable or possible according to EORTC/MSG criteria

Trial Locations

Locations (1)

Istituto Giannina Gaslini; 🇮🇹 Genoa, Italy