Results of a prospective observational study of imiglucerase biosimilar in adults with type I Gaucher disease
Year: 2025
Type: article
Study design: Observational Study
Abstract: AIM: To collect and analyze real-world data on long-term enzyme replacement therapy with Glurazyme® in patients with type I Gaucher disease (GD). MATERIALS AND METHODS: The study included 31 patients with type I GD (21 patients with intact spleen and 10 patients with a history of splenectomy) over 18 years. Efficacy was assessed by the change in hemoglobin level (primary endpoint), platelet count, spleen and liver volumes, prevalence of bone marrow infiltration, and the number of patients with quiet hip disease according to magnetic resonance imaging (secondary endpoints). Safety was assessed by the incidence of treatment-related adverse events and serious adverse events, as well as by the incidence of production of imiglucerase binding and neutralizing antibodies (IgG, IgE). The mean follow-up duration was 54 weeks. RESULTS: =0.08). The prevalence of specific bone marrow infiltration and the number of patients with quiet hip disease remained unchanged. During the safety analysis, one adverse reaction, a mild increase in alanine aminotransferase, was reported, which resolved spontaneously by the end of the study. The immunogenicity analysis showed the initial presence of anti-drug antibodies (ADAs) in 5 (16.7%) of the 30 examined patients. At the end of the study, ADAs were detected in only 3 (10%) patients. The detected ADAs had a low titer and no neutralizing activity, and they did not affect the treatment effectiveness. CONCLUSION: Long-term therapy with the imiglucerase biosimilar was associated with a stable course of GD without progression. It was characterized by a good safety profile and low immunogenicity.
Source: Terapevticheskii arkhiv
Authors: Elena A. Lukina, Rodion V. Ponomarev, Galina N. Salogub, Vadim A. Bezrukikh, Elena V. Saifullina, Igor L. Davydkin, Svetlana A. Volkova, Tatiana V. Shelekhova, Marina V. Kosinova, Evgeniy V. Vasiliev, Elena G. Kirillova, Olesya E. Danilova, Rais K. Khairetdinov, Oksana A. Markova, Evgene V. Zuev, Anton I. Borozinets
Institutions: National Medical Research Center for Hematology, Federal Almazov North-West Medical Research Centre, Bashkir State Medical University, Samara State Medical University, Privolzhsky Research Medical University, Saratov State Medical University, Krasnoyarsk Regional Clinical Hospital, Generium Pharmaceuticals (Russia)
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: diamond
OpenAlex ID: W4408838760