RESULTS OF A PROSPECTIVE MULTICENTER OPEN-LABEL OBSERVATIONAL STUDY OF ENZYME REPLACEMENT THERAPY WITH AN IMIGLUCERASE BIOSIMILAR IN CHILDREN WITH TYPE 1 GAUCHER DISEASE

Abstract

Enzyme replacement therapy (ERT) is the mainstay for type 1 Gaucher disease (GD1) treatment. Imiglucerase biosimilar usage allows to control GD1 manifestations and improve the patients’ life quality according to interventional clinical studies. Objective: To evaluate the results of Glurazyme® use during long-term ERT in patients younger than 18 years with GD1 within a prospective multicenter observational study. Materials and methods: A total of 7 children aged 8–16 years with GD1 who receive ERT with Glurazim® were selected to participate in this prospective multicenter, non-comparative observational study. The study included a screening period (28 days) and a treatment period (52 weeks). Efficacy was assessed based on the hemoglobin dynamics over the observation period. Additionally, changes in platelet count, spleen and liver volumes, bone tissue condition, and the need for dose adjustments were studied. All reported adverse events (AE) were analyzed to assess treatment safety. Results: The average duration of the study for one patient was 363 [354.5; 376] days. The median values of a Glurazim® single dose were 1400 [900; 1900] U. During the study, two patients (28.6%) required a drug dose increase due to children growth. Hemoglobin and platelet concentrations remained stable, no significant changes were registered (p=0.32 and p=0.85, respectively). No increase in the spleen or liver volume was observed: p=0.78 and p=0.37, respectively. There were no new cases of aseptic bone necrosis during the study. All AEs were not related to the study drug. Conclusion: In this group of children with GD1, one year of Glurazyme® therapy was associated with stable hematological, visceral, and bone parameters, with no drug-related AEs observed. These findings support the evidence that long-term treatment with the imiglucerase biosimilar is effective in achieving therapeutic goals in pediatric patients with Gaucher disease.