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Treatment with caspofungin in immunocompromised paediatric patients: a multicentre survey
Andreas H Groll1, Andishe Attarbaschi, Friedhelm R Schuster
1Infectious Disease Research Program, Center for Bone Marrow Transplantation and Department of Paediatric Haematology/Oncology, Children's University Hospital, Muenster, Germany. grollan@ukmuenster.de
Insights
Caspofungin demonstrated good safety and tolerance in immunocompromised children with invasive fungal infections. This antifungal medication showed potential efficacy, with mild side effects and positive survival rates in a retrospective survey.
Area of Science:
- Paediatric Infectious Diseases
- Mycology
- Clinical Pharmacology
Background:
- Caspofungin is used off-label in paediatric patients despite no established dosage.
- Immunocompromised children are at high risk for invasive fungal infections.
Purpose of the Study:
- To gather data on the use of caspofungin in immunocompromised paediatric patients.
- To evaluate the safety, tolerance, and efficacy of caspofungin in this population.
Main Methods:
- A multicentre retrospective survey of 64 immunocompromised paediatric patients.
- Patients received caspofungin for proven, probable, possible, or empirical invasive fungal infections.
- Dosages were physician-determined based on individual patient needs and treatment goals.
Main Results:
- Caspofungin was administered for a median of 37 days, with dosages varying by weight and body surface area.
- No patients discontinued therapy due to adverse events; clinical adverse events were mild to moderate.
- Positive clinical responses and stabilization were observed in patients with proven, probable, and possible infections, with high survival rates.
Conclusions:
- Caspofungin exhibits favourable safety and tolerance in severely immunocompromised paediatric patients.
- The drug demonstrated potential antifungal efficacy in this challenging patient group.
- Further research into established paediatric dosing regimens is warranted.
Objectives:
Although a paediatric dosage has not been established, caspofungin is occasionally used in paediatric patients. We conducted a multicentre retrospective survey to obtain data on immunocompromised paediatric patients considered to require caspofungin therapy.
Methods:
The survey identified 64 patients (median age: 11.5 years; 25 females, 39 males) with haematological malignancies (48), marrow failure (9), solid tumours (3), haematological disorders (2) and congenital immunodeficiency (2) who received caspofungin for proven (17), probable (14) and possible (17) invasive fungal infections or empirically (16). Caspofungin was administered until intolerance or maximum efficacy at dosages individually determined by the responsible physician for refractory infection (38), intolerance of other agents (10) or as best therapeutic option (16).
Results:
The 64 patients received caspofungin for a median of 37 days (range 3-218) as single agent (20) or in combination (44). The median daily maintenance dosage was 1.07 mg/kg (95% CI 1.09-1.35; range 0.40-2.92) or 34.3 mg/m2 (95% CI 32.3-37.3; range 16.3-57.5). In none of the patients was therapy discontinued due to adverse events (AEs). Clinical AEs were mild to moderate and observed in 34 patients (53.1%). While mean glutamate pyruvate transaminase and glutamate oxalate transaminase values were slightly (P < 0.005) higher at the end of treatment (EOT), serum bilirubin, alkaline phosphatase and creatinine values were not different from baseline. Complete responses, partial responses or stabilization were observed in 5/7/3 of 17 patients with proven, in 3/4/3 of 14 patients with probable and in 7/6/1 of 15 evaluable patients with possible invasive infections. Thirteen of 16 patients on empirical therapy completed without breakthrough infection. Overall survival was 75% at the EOT and 70% at 3 months post-EOT, respectively.
Conclusions:
Caspofungin displayed favourable safety and tolerance and may have useful antifungal efficacy in severely immunocompromised paediatric patients.
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