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Updated: Jun 9, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
Published on: May 27, 2011
Isavuconazole prophylaxis and treatment in pediatric HSCT recipients: Pharmacokinetics, safety, and efficacy
Hao Jiang1, Lixiao Cai2, Ningning Chen2
1Department of Stem cell Transplantation, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China.
Insights
Isavuconazole is safe and effective for preventing invasive fungal diseases in pediatric stem cell transplant patients. A trough concentration target of 1-3 mg/L is sufficient for prophylaxis, with TDM crucial for optimal dosing, especially in young children.
Area of Science:
- Pharmacology
- Hematology
- Infectious Diseases
Background:
- Pediatric hematopoietic stem cell transplantation (HSCT) recipients are at high risk for invasive fungal diseases (IFDs).
- Isavuconazole is a newer azole antifungal agent with a broad spectrum of activity.
- Optimal dosing and therapeutic drug monitoring (TDM) targets for isavuconazole in pediatric HSCT populations require further definition.
Purpose of the Study:
- To evaluate the pharmacokinetics, safety, and efficacy of isavuconazole for IFD prophylaxis in pediatric HSCT recipients.
- To define an optimal TDM trough concentration target for isavuconazole prophylaxis in this population.
Main Methods:
- Retrospective analysis of 30 pediatric patients undergoing HSCT, receiving isavuconazole for prophylaxis (n=23) or salvage (n=7).
- Evaluation of plasma trough concentrations (Cmin), concentration-to-dose ratios (C/D), breakthrough IFDs, and adverse events.
- Specific focus on a target Cmin of 1-3 mg/L for prophylaxis.
Main Results:
- A Cmin of 1-3 mg/L achieved 95.7% clinical success in the prophylaxis group.
- The defined target range demonstrated a favorable safety profile without dose-limiting toxicities.
- Children under 6 years showed significantly lower C/D ratios, indicating faster drug clearance and need for higher weight-based doses.
Conclusions:
- Isavuconazole is effective and safe for IFD prophylaxis in pediatric HSCT recipients.
- A Cmin target of 1-3 mg/L is adequate for prophylaxis, differing from treatment targets.
- Routine TDM is essential for optimizing isavuconazole dosing, particularly in children under 6 years.
Objectives:
To evaluate the pharmacokinetics, safety, and efficacy of isavuconazole in pediatric hematopoietic stem cell transplantation (HSCT) recipients, specifically defining an optimal therapeutic drug monitoring (TDM) trough concentration target for antifungal prophylaxis.
Methods:
This single-center, retrospective study included 30 pediatric patients receiving isavuconazole post-HSCT (April 2023-February 2026) for prophylaxis (n = 23) or salvage treatment (n = 7). For prophylaxis, a target trough concentration Cmin of 1-3 mg/L was evaluated. Plasma Cmin, steady-state concentration-to-dose (C/D) ratios, breakthrough invasive fungal diseases (IFDs), and adverse events were analyzed.
Results:
In the prophylaxis cohort, maintaining a Cmin between 1 and 3 mg/L achieved a 95.7% (22/23) clinical success rate. The single breakthrough IFD occurred in a high-risk re-transplantation patient. This lower target range demonstrated a favorable safety profile without dose-limiting toxicities. Pharmacokinetic analysis revealed that children <6 years exhibited significantly lower steady-state C/D ratios (median 0.74) compared to older patients (P = 0.0019), indicating accelerated drug clearance and a requirement for higher weight-based dosing in this younger subset.
Conclusions:
Isavuconazole provides effective and safe IFD prophylaxis in pediatric HSCT recipients. A Cmin target of 1-3 mg/L is sufficient for prophylaxis, distinct from the higher therapeutic targets typically recommended for treatment. Routine TDM remains critical to optimize dosing and ensure target attainment, particularly in children <6 years.
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