A Case-Controlled Study with Dornase Alfa to Evaluate Impact on Disease Progression over a 4-Year Period

Abstract

Background: Chronic endobronchial sepsis and profuse airway secretions dominate pulmonary disease in cystic fibrosis. Recombinant human DNase I (dornase alfa) reduces the viscoelasticity of airway secretions and hence may improve clearance of airway secretions. Objectives: To evaluate the long-term influence of dornase alfa on disease progression by performing a case-controlled study with dornase alfa over a period of 4 years. Methods: A cohort of patients with cystic fibrosis who have been treated with dornase alfa were matched with a control group of patients with cystic fibrosis who had not received treatment with dornase alfa. The patients were matched by pulmonary function, age, and then sex. All available measurements of forced expiratory volume in one second (FEV₁), height, weight and sputum bacteriology were collected for periods when the patients were free from respiratory exacerbations. Results: Thirty-eight patients were matched. Slopes of median changes in FEV₁ were –2.19 (–3.32, –1.06) in the control group and –0.75 (–1.87, 0.36) in the dornase alfa-treated group (p = 0.076). There were more infective exacerbations per patient year in the control group [3.13 (1.25–4.25)] in comparison to the dornase alfa group [1.25 (0.63–3.0), p = 0.035] over the 4-year treatment period. Antibiotic requirements were also greater with a median 43.75 (17.5–60.0) days of intravenous antibiotic use per patient year in the control group and 16.25 (8.5–44.0) days in the dornase alfa group (p = 0.034). Conclusions: The trends suggest that dornase alfa may have some influence on disease progression but in view of the limitations of the current study the need for further long-term studies in larger cohorts of patients is emphasised.

Clinical Practice Guidelines From the Cystic Fibrosis Foundation for Preschoolers With Cystic Fibrosis

“…[75][76][77][78][79][80][81] Dornase alfa has been shown to have positive effects on CT changes and LCI [82][83][84] and improved health-related quality-of-life scores in children >6 years. 85 Safety and tolerability of dornase alfa has been demonstrated in children ages 3 months to 5 years.…”

The Register as a Means of Improving the Quality of Mucoviscidosis Patients’ Treatment

“…Положительный опыт применения препарата у детей с 2-месячного возраста в Российском центре МВ и данные литературы позволили расширить возрастные рамки и использовать его у больных, выявленных в ходе неонатального скрининга [13]. Длительное применение дорназы альфа способствует уменьшению числа обостре-ний и замедлению снижения легочной функции [14,15]. Недавние исследования подтверждают данные об эффек-тивности и безопасности использования дорназы альфа на любой стадии болезни.…”

Clinical Characteristics of Respiratory Involvement in Children With Cystic Fibrosis in the Chuvash Republic

“…Преимущество дорназы альфа среди средств, улучшающих реологи-ческие свойства мокроты, обусловлено не только уни-кальными муколитическими свойствами, но и проти-вовоспалительным и антибактериальным эффектом. Клиническая эффективность и безопасность препарата дорназы альфа была продемонстрирована в большом числе международных и отечественных иссследова-ний, проведенных за последние 20 лет [5][6][7][8][9][10][11][12]. Одним из значимых исследований стало PEIT (Pulmozyme Early Intervention Trial), началом которому послужи-ла точка зрения о возможном протективном влиянии Пульмозима при раннем его назначении на бронхоле-гочную систему у пациентов с незначительными нару-шениями функции легких (ФЖЕЛ у 70% от должно-го), но с доказанным воспалительным процессом [5].…”