Successful Salvage Therapy for Mucormycosis with Isavuconazole in Pediatric Patients: Cases Series

Background: Mucormycosis is a life-threatening fungal disease that occurs primarily in immunocompromised patients, but also in patients with diabetes mellitus or trauma. Isavuconazole is a new triazole that has shown efficacy in the adult population for primary and salvage treatment of mucormycosis. However, data regarding its efficacy and long-term safety in the pediatric population are scarce.

Methods: The demographic and clinical data of pediatric patients with mucormycosis who were treated with isavuconazole in 2017-2019 were collected. Children with histopathological evidence of the disease or growth on fungal cultures from a sterile body site were included.

Results: Four children of median age 10.5 years (range 7-14) met the study criteria. Two had rhino-orbital involvement and one each had cutaneous with deep tissue involvement or disseminated infection. Three had underlying hematological malignancies, and one had sustained major trauma. Isavuconazole was used as salvage therapy in all: in three patients for refractory disease, and in one after intolerance to another anti-fungal drug. Following initiation of isavuconazole, alone or combined with other anti-fungal agents, complete clinical, radiological, and mycological responses were documented in all patients. The median treatment duration was 2.5 months (range 2-4), during which no isavuconazole-related adverse events were noted.

Conclusions: Our limited experience showed that isavuconazole was well-tolerated and efficacious as salvage therapy in pediatric patients with mucormycosis. The overall complete response rate was 100%. Prospective clinical trials are needed to evaluate the safety and efficacy of isavuconazole in the pediatric population and its role as first-line treatment for mucormycosis.