Browsing by Author "Heltshe, Sonya"

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    Multiple-Breath Washout as a Lung Function Test in Cystic Fibrosis: A Cystic Fibrosis Foundation Workshop Report
    (2015-06) Subbarao, Padmaja; Milla, Carlos; Aurora, Paul; Davies, Jane C.; Davis, Stephanie D.; Hall, Graham L.; Heltshe, Sonya; Latzin, Philipp; Lindblad, Anders; Pittman, Jessica E.; Robinson, Paul D.; Rosenfeld, Margaret; Singer, Florian; Starner, Tim D.; Ratjen, Felix; Morgan, Wayne; Department of Pediatrics, IU School of Medicine
    The lung clearance index (LCI) is a lung function parameter derived from the multiple-breath washout (MBW) test. Although first developed 60 years ago, the technique was not widely used for many years. Recent technological advances in equipment design have produced gains in popularity for this test among cystic fibrosis (CF) researchers and clinicians, particularly for testing preschool-aged children. LCI has been shown to be feasible and sensitive to early CF lung disease in patients of all ages from infancy to adulthood. A workshop was convened in January 2014 by the North American Cystic Fibrosis Foundation to determine the readiness of the LCI for use in multicenter clinical trials as well as clinical care. The workshop concluded that the MBW text is a valuable potential outcome measure for CF clinical trials in preschool-aged patients and in older patients with FEV1 in the normal range. However, gaps in knowledge about the choice of device, gas, and standardization across systems are key issues precluding its use as a clinical trial end point in infants. Based on the current evidence, there are insufficient data to support the use of LCI or MBW parameters in the routine clinical management of patients with CF.
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    Rates of adverse and serious adverse events in children with cystic fibrosis
    (Elsevier, 2021) Pittman, Jessica E.; Khan, Umer; Laguna, Theresa A.; Heltshe, Sonya; Goss, Christopher H.; Sanders, Don B.; Pediatrics, School of Medicine
    Background: Cystic fibrosis (CF) is an autosomal recessive disease characterized by chronic sinopulmonary symptoms and chronic gastrointestinal symptoms that begins in infancy. Children with CF are increasingly being included in clinical trials. In order to fully evaluate the impact of new therapies in future clinical trials, an understanding of baseline adverse event (AE) rates in children with CF is needed. To address this, we determined the rates of common AEs in pediatric patients with CF who participated in two clinical trials. Methods: We reviewed AEs for placebo recipients in the AZ0004 study and inhaled tobramycin recipients in the Early Pseudomonas Infection Control (EPIC) clinical trial. AEs were categorized based on Medical Dictionary for Regulatory Activities (MedDRA) coding classifications and pooled into common, batched AE descriptors. AE rates were estimated from negative binomial models according to age groups, severity of lung disease, and season. Results: A total of 433 children had 8,266 total AEs reported, or 18.1 (95% CI 17.0, 19.2) AEs per person per year. Respiratory AEs were the most commonly reported AEs, with a rate of 7.6 events per person-year. The total SAE rate was 0.33 per person per-year. Cough was the most commonly reported respiratory AE, with 61% of subjects reporting at least one episode of cough within 4 months. The rate ratio of any AE was higher in Spring, Fall, and Winter, compared with Summer. Conclusions: AEs occur commonly in pediatric CF clinical trial participants. Season of enrollment could affect AE rates.