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Real-Life Use of Posaconazole in Clinical Practice for Onco-Hematological Children: A National Survey by AIEOP
Lorenzo Chiusaroli1, Manuela Spadea2, Cecilia Liberati1
1Division of Pediatric Infectious Diseases, Department for Women's and Children's Health, University Hospital of Padua, 35128 Padova, Italy.
Insights
Posaconazole is widely used for antifungal prophylaxis and treatment in pediatric cancer patients across Italy. However, its application varies significantly between centers, highlighting the need for standardized protocols.
Area of Science:
- Pediatric Hematology-Oncology
- Infectious Diseases
- Pharmacology
Background:
- Posaconazole is an antifungal approved for invasive fungal infections (IFI) in pediatric onco-hematological patients.
- Recent pediatric approval means limited data and applicable limitations exist.
- Safety and efficacy appear favorable in children, prompting further investigation into its use.
Purpose of the Study:
- To describe the current utilization of posaconazole in pediatric cancer patients within the Associazione Italiana Ematologia e Oncologia Pediatrica (AIEOP) network.
- To identify patterns in posaconazole prophylaxis and treatment across Italian pediatric hematology-oncology centers.
- To assess the variability in posaconazole formulations, indications, and monitoring practices.
Main Methods:
- A national survey was conducted among physicians in the AIEOP network.
- A 25-item web questionnaire assessed posaconazole use in pediatric cancer patients, including those undergoing hematopoietic stem cell transplantation (HSCT).
- Descriptive statistics were used to analyze the collected data.
Main Results:
- Sixty-eight percent of AIEOP centers responded, indicating availability of oral suspension, delayed-release tablets, and intravenous posaconazole.
- Primary uses included prophylaxis for acute lymphoblastic leukemia (38%), acute myeloid leukemia (38%), and aplastic anemia (19%).
- Posaconazole was also used for secondary prophylaxis and salvage therapy, with common but varied drug plasma level monitoring and discontinuation due to drug-drug interactions.
Conclusions:
- Posaconazole is extensively utilized in AIEOP centers for pediatric onco-hematological patients.
- Significant variability exists in its application, including indications, dosing, and monitoring.
- Prospective studies are essential to establish standardized protocols for optimal pediatric posaconazole use.
Background:
Posaconazole is an antifungal medication used to treat invasive fungal infections (IFI) in pediatric onco-hematological patients. Its approval for pediatric use was recent, and limitations still apply. Despite limited data, the safety and efficacy profile appear generally favorable in children. This study describes how posaconazole is used across centers affiliated with the Associazione Italiana Ematologia e Oncologia Pediatrica (AIEOP).
Methods:
A national survey was conducted among physicians within the AIEOP network to evaluate current use of posaconazole in pediatric cancer patients, including those undergoing hematopoietic stem cell transplantation (HSCT). A 25-item web questionnaire was developed and distributed in June 2024. Data analysis involved descriptive statistics.
Results:
Twenty-one of thirty-one centers (68%) responded, reporting availability of various posaconazole formulations: oral suspension (76%), delayed-release tablets (95%), and intravenous solution (14%). Posaconazole was primarily used for prophylaxis in patients with acute lymphoblastic leukemia (ALL, 38%), acute myeloid leukemia (AML, 38%), and aplastic anemia (19%). It was also used as secondary prophylaxis against previous possible or probable IFI or as salvage therapy for probable or confirmed aspergillosis or mucormycosis, often combined with other treatments. Drug plasma level monitoring was common but varied in scheduling across centers. Most centers (74%) discontinued posaconazole if adverse events suspected drug-drug interactions, such as with vincristine.
Conclusions:
Posaconazole is widely used in AIEOP centers, though application varies significantly. This variability emphasizes the need for prospective studies to better define indications, dosing, and monitoring protocols for pediatric use of this antifungal.
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