Background Invasive fungal diseases (IFDs) increase the mortality rate
of patients with neutropenia who receive chemotherapy or have previously
undergone hematopoietic stem cell transplantation (HSCT). Micafungin is
a broad-spectrum echinocandin, with minimal toxicity and low drug
interactions. We therefore investigated the efficacy and safety of
prophylactic micafungin in pediatric and adolescent patients who
underwent autologous HSCT. Methods This was a phase II, prospective,
single-center, open-label, and single-arm study. From November 2011 to
February 2017, 125 patients were screened from Seoul National University
Children’s Hospital, Korea, and 112 were enrolled. Micafungin was
administered intravenously at a dose of 1 mg/kg/day (maximum 50 mg/day)
from day 8 of autologous HSCT until neutrophil engraftment. Treatment
success was defined as the absence of proven, probable, or possible IFD
up to 4 weeks after therapy. Results The study protocol was achieved
without premature interruption in 110 patients (98.2%). The reasons
interrupting micafungin treatment included early death (n=1) and patient
refusal (n=1). Treatment success was achieved in 109 patients (99.1%).
Only one patient was diagnosed with probable IFD. No patients were
diagnosed with possible or proven IFD. In the full analysis set, 21
patients (18.8%) experienced 22 adverse events (AEs); however, all AEs
were classified as “unlikely” related to micafungin. No patient
experienced grade IV AEs nor discontinued treatment and none of the
deaths were related to micafungin. Conclusions Our study demonstrated
that micafungin is a safe and effective option for antifungal
prophylaxis in pediatric patients who underwent autologous HSCT, with
promising efficacy without significant AEs.
Defibrotide for Prophylaxis of Hepatic Veno-Occlusive Disease in Pediatric Hematopoietic Stem Cell Transplantation: Subanalysis Data from an Open-Label, Phase III, Randomized Trial
