Impact of prophylactic echinocandin on the development of neurological complications in patients receiving busulfan-containing conditioning regimens for stem cell transplantation: A single-center retrospective study

Pediatr Transplant. 2024 May;28(3):e14728. doi: 10.1111/petr.14728.

Abstract

Background: Although neurotoxicity is a major adverse event associated with busulfan, little information is available regarding the association between drug interactions and neurological symptoms during busulfan-based regimens. This study evaluated the association between prophylactic echinocandins and neurological complications in patients receiving busulfan-containing conditioning regimens for stem cell transplantation.

Methods: We retrospectively included consecutive patients who administered intravenous busulfan as a conditioning regimen at our facility between 2007 and 2022. Prophylactic echinocandin use was defined as the use of an echinocandin antifungal drug to prevent invasive fungal disease in SCT recipients. The primary outcome was the incidence of neurological complications within 7 days of busulfan initiation and was compared between the echinocandin group (patients received prophylactic echinocandin) and nonechinocandin group (patients received prophylactic antifungal drugs other than echinocandin and those without antifungal prophylaxis).

Results: Among the 59 patients included in this study, the incidence of neurological complications in the echinocandin (n = 26) and nonechinocandin groups (n = 33) was 30.8% and 63.6%, respectively. We observed a negative association between prophylactic echinocandin use and the development of neurological complications after adjusting for the propensity score for receiving prophylactic echinocandins (adjusted odds ratio 0.294, 95% confidence interval 0.090 to 0.959). We observed a lower incidence of neurological complications in the echinocandin group than in the nonechinocandin group.

Conclusion: Our results suggested that the choice of antifungal prophylaxis is associated with busulfan neurotoxicity.

Keywords: busulfan; echinocandin; neurological complication; pediatrics; stem cell transplantation.

MeSH terms

  • Antifungal Agents / therapeutic use
  • Busulfan / adverse effects
  • Echinocandins / therapeutic use
  • Graft vs Host Disease* / etiology
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Nervous System Diseases* / etiology
  • Retrospective Studies
  • Stem Cell Transplantation
  • Transplantation Conditioning / adverse effects
  • Transplantation Conditioning / methods

Substances

  • Busulfan
  • Antifungal Agents
  • Echinocandins