The article discusses the results of a phase III clinical trial to compare the pharmacokinetics, efficacy and safety of the biosimilar medicinal product Tigerase® (dornase alpha) (Generium JSC, Russia) and the reference medicinal product Pulmozyme® (F.Hoffmann-La Roche Ltd, Switzerland) with the purpose of establishing their comparability for symptomatic treatment of patients with cystic fibrosis (CF).Methods. The study included 100 patients aged 18 years and older with a confirmed diagnosis of CF, who were divided into two groups by stratified randomization in a ratio of 1 : 1 based on the initial level of FEV1 (40–60% or > 60–100% from due value). Tigerase® or Pulmozyme® were used in a dose of 2.5 mg daily, once a day in the form of inhalations using a jet nebulizer compressor for 24 weeks.Results and discussion: The analysis of the data regarding the primary efficacy endpoint – changes in FEV1 – showed that in both groups (FAS population (Full analyses set) and PP population (Per protocol)), similar changes in FEV1 were observed. The average value of changes in FEV1 after 24 weeks of treatment compared with the initial level in the FAS population was –1.3% ± 9.8 % (95% CI (–4.1; 1.6)) in Group I (Tigerase®) and –1.9% ± 10.0% (95% CI (–4.7; 1.0)) in Group II (Pulmozyme®). The point estimate for the intergroup difference in changes in FEV1 (Group I – Group II) was 0.6%. The calculated 95% CI for the difference in changes in FEV1 in the FAS population was (–3.3; 4.6%]. In both populations studied, the intergroup difference in changes in FEV1 did not exceed 6%. During long-term treatment of patients with CF, no statistically significant differences were found in terms of efficacy (changes in FEV1 and FVC; number of exacerbations of chronic pulmonary disease and the number of days before its development; change in body weight; quality of life) between medicinal products in both studied populations (FAS and PP).Conciusion. A safety analysis demonstrated the comparability of medicinal products in terms of the incidence of adverse events. The frequency of detection of antibodies to dornase alpha during the study was similar in the treatment groups; the formation of antibodies did not lead to a decrease in the efficacy and safety of therapy.
Study of efficacy, safety, and patient satisfaction with inhaled tobramycin (Tobramycin-Gobbi) in children with cystic fibrosis and pseudomonas infectionAim of the study was to assess efficacy and safety of Tobramycin-Gobbi in CF, as well as the patients’ satisfaction with the treatment.Methods. 35 children from 6 to 18 years with CF were enrolled in this non-interventional prospective cohort multicenter study. All children had P. aeruginosa in the respiratory tract (newly diagnosed, recurrent, or chronic infection). The children received inhalation treatment with Tobramycin-Gobbi in the following cycles: 28-day treatment/28-day break, for 6 months. The studied parameters included respiratory function, bacterial cultures of the respiratory tract with a bacterial count, growth and body weight, antibiotic therapy for the respiratory episodes. The children and parents filled in a questionnaire “Treatment satisfaction assessment” and assessed their state of health on the visual-analog scale before and after each treatment cycle.Results. P. aeruginosa was eradicated in 17.7% of cases (6 patients, including 2 newly diagnosed, 3 recurrent infections, and 1 chronic infection), reduced bacterial count, decreased number of courses of antibiotic therapy, improvement of FEV1. Adverse reactions were reported by one patient.Conclusion. The efficacy, safety, and tolerability of Tobramicine Gobbi were confirmed in the patients with newly diagnosed, recurrent, and chronic infection caused by P. aeruginosa.
The aim of the program was to study the tolerability of Tigerase® in patients with cystic fibrosis (CF) of all ages in rutine clinical practice. Study design: retrospective open uncontrolled comparative multicenter solid. Materials and methods of research: retrospective data of clinical observations were collected from medical records of patients with CF on the use of Tigerase®. Results: therapy with Tigerase® was well tolerated by 668 (93,4%) of 715 patients included in the study. In 47 (6,6%) patients, 127 adverse reactions (ADRs) associated with the use of Tigerase® were recorded. ADRs from the respiratory system were the most common. Of these, 24 (3,4%) were coughing and 10 (1,4%) had increased viscosity of bronchial secretion. Among all patients included in the study, the proportion of patients in whom ADRs were registered based on clinical manifestations (3,9%) did not differ statistically significantly from the proportion of patients in whom ADRs were recorded based on complaints only (2,8%) (p=0,30). The distribution of ADRs by the source of registration and place of residence of patients did not depend on their gender and age. Registration of ADRs in different regions of the country differed statistically significantly both in frequency and in the source of detection (p<0,001). ADRs were not recorded in several regions, and the largest number of ADRs were registered in patients living in Moscow, and most of them were based only on patient complaints. 22 patients (47% of the number of patients with ADR) had medical commissions for ADR, and only in 8 (17% of the number of patients with ADR) of them had expertise of specialists with experience in the treatment of patients with CF. In 29 patients (62% of the number of patients with ADR), the development of ADR did not require cessation of the Tigerase® therapy. Conclusion: in the majority of CF patients (93,4%) tolerated the Tigerase® therapy well.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.