Chest
Clinical InvestigationsCystic FibrosisLong-term Benefits of Inhaled Tobramycin in Adolescent Patients With Cystic Fibrosis
Section snippets
Materials and Methods
The data presented here were collected during a 96-week series of clinical trials that consisted of a 24-week, double-blind, randomized, pivotal phase and 72-week, open-label phase. Figure 1 , upper panel, illustrates the two phases. In the pivotal phase, patients were randomized to treatment with either 300 mg of TSI bid or taste-masked placebo bid. In the open-label phase, all patients received TSI. The overall design of the open-label trials is identical to that of the controlled trials as
Patient Population
Patients were assigned to age subgroups (children, 6 to 12 years; adolescent, 13 to 17 years; adult, ≥ 18 years) according to their age at the first visit of the controlled trials (visit 3, week 0). One hundred twenty-eight adolescent patients were enrolled in the controlled trials. Of these 128 patients, 120 patients completed 24 weeks of randomized treatment. Ninety-three adolescent patients entered the open-label phase of the study and 65 adolescent patients completed the 96-week series.
Of
Discussion
Pulmonary function is the single best predictor of morbidity and mortality in patients with CF.5 Adolescent CF patients are at a particularly vulnerable stage in their life cycle when they are susceptible to rapid decline in pulmonary function and high rates of death through early adulthood. The 2-year TSI results show long-term improvement in pulmonary function in adolescent CF patients. The substantial improvements in FEV1 percent predicted and the maintenance of lung function above baseline
References (18)
- et al.
Longitudinal analysis of pulmonary function decline in patients with cystic fibrosis
J Pediatr
(1997) - et al.
A comparison of survival, growth, and pulmonary function in patients with cystic fibrosis in Boston and Toronto
J Clin Epidemiol
(1988) - et al.
Nutrition in adults with cystic fibrosis
Clin Nutr
(1998) Cystic fibrosis: molecular biology and therapeutic implications
Science
(1992)- et al.
Cystic fibrosis
Am J Respir Crit Care Med
(1996) Cystic fibrosis: pathogenesis, pulmonary infection, and treatment
Clin Infect Dis
(1995)Patient registry 1998 annual data report
(1999)- et al.
Prediction of mortality in patients with cystic fibrosis
N Engl J Med
(1992) - et al.
Efficacy of aerosolized tobramycin in patients with cystic fibrosis
N Engl J Med
(1993)
Cited by (149)
Nanoparticle-based platforms for targeted drug delivery to the pulmonary system as therapeutics to curb cystic fibrosis: A review
2024, Journal of Microbiological MethodsApplication of iPS cell-derived airway epithelial cells for cell-based therapy and disease models
2021, Recent Advances in iPSC-Derived Cell Types: Volume 4 in Advances in Stem Cell BiologyUnusual Cystic Fibrosis Transmembrane Conductance Regulator Mutations and Liver Disease: A Case Series and Review of the Literature
2019, Transplantation ProceedingsCitation Excerpt :There are 6 classes of CF mutations [6]. In class 1 there is premature mRNA termination secondary to nonsense or frameshift mutation, resulting in the CFTR protein being completely absent [9–11]. The class 2 mutation has a defect in protein processing due to abnormal posttranslational modification [9].
Aerosolized tobramycin for Pseudomonas aeruginosa ventilator-associated pneumonia in patients with acute respiratory distress syndrome
2017, Pulmonary Pharmacology and TherapeuticsCitation Excerpt :The efficacy and safety of TIS therapy has been well studied, especially in patients with cystic fibrosis. Many clinical studies have demonstrated that TIS therapy for cystic fibrosis reduced sputum production, P. aeruginosa density in sputum and antibiotic consumption; it also improved lung function, quality of life, and survival [34–37]. Few researches on VAP suggested that TIS therapy was associated with better outcomes with respect to systemic inflammation, weaning success, or survival in mechanically ventilated patients [38–40].
The treatment of the pulmonary and extrapulmonary manifestations of cystic fibrosis
2017, Presse Medicale
The author has received speaking and consulting fees from Chiron Corporation.
Presented in part at the 1999 North American Cystic Fibrosis Conference, Seattle, WA, October 7–10, 1999.