Long-term safety of dexamethasone sodium phosphate encapsulated in autologous erythrocytes in pediatric patients with ataxia telangiectasia.

ABSTRACT:

Background

Dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes (EryDex) was developed as an alternative to standard glucocorticoids in an effort to eliminate chronic steroid toxicity while preserving efficacy. The primary objective of this report is to describe the safety of long-term use of EryDex in treatment of pediatric patients with ataxia telangiectasia.

Methods

This is a post-hoc analysis of patients treated with EryDex for a minimum of 24 months in two prospective clinical trials. Outcomes include adverse events, growth, hemoglobin and serum iron, glucose levels, HbA1c, CD4+ lymphocytes, and bone mineral density.

Results

Sixty-eight patients completed a minimum of 2 years of treatment with EryDex (mean treatment length 39 ± 11 months).

SUBMITTER: Koenig MK 

PROVIDER: S-EPMC11800360 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature