Effect of a short course of rhDNase on cough and mucociliary clearance in patients with cystic fibrosis

Hemming, A.L.; Moriarty, Carmel; Eberl, Stefan; Bye, Peter T. P.

doi:10.1002/1099-0496(200007)30:1<16::aid-ppul4>3.0.co;2-h

Cited by 31 publications

(8 citation statements)

References 28 publications

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“… 87 Finally, MCC is not sensitive to all therapeutic agents; while sensitive to agents that effectively change airway surface liquid hydration, it was not altered by dornase alfa in previous studies. 92 , 93 …”

Section: Introductionmentioning

confidence: 99%

Novel outcome measures for clinical trials in cystic fibrosis

Tiddens

Puderbach²,

Venegas

et al. 2014

Pediatric Pulmonology

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Cystic fibrosis (CF) is a common inherited condition caused by mutations in the gene encoding the CF transmembrane regulator protein. With increased understanding of the molecular mechanisms underlying CF and the development of new therapies there comes the need to develop new outcome measures to assess the disease, its progression and response to treatment. As there are limitations to the current endpoints accepted for regulatory purposes, a workshop to discuss novel endpoints for clinical trials in CF was held in Anaheim, California in November 2011. The pros and cons of novel outcome measures with potential utility for evaluation of novel treatments in CF were critically evaluated. The highlights of the 2011 workshop and subsequent advances in technologies and techniques that could be used to inform the development of clinical trial endpoints are summarized in this review. Pediatr Pulmonol. © 2014 The Authors. Pediatric Pulmonology published by Wiley Periodicals, Inc.

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Section: Introductionmentioning

confidence: 99%

Novel outcome measures for clinical trials in cystic fibrosis

Tiddens

Puderbach²,

Venegas

et al. 2014

Pediatric Pulmonology

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“…Both studies also recorded subjective measures of spontaneous cough. Neither study demonstrated a significant action of recombinant DNase over placebo, although Robinson et al 75 suggested that their study may have been underpowered given the large intersubject variability that they encountered.…”

Section: Pharmacologic Enhancement Of Cough Clearancementioning

confidence: 94%

“…The current guideline includes two new studies that investigated the effects of recombinant DNase on cough clearance 75 and mucociliary clearance 76 in subjects with cystic fibrosis (Table 5). Both studies also recorded subjective measures of spontaneous cough.…”

Section: Pharmacologic Enhancement Of Cough Clearancementioning

confidence: 99%

Cough Suppressant and Pharmacologic Protussive Therapy

Bolser¹

2006

Chest

126

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“…Furthermore, results from scintigraphic studies in using twice daily 2.5 mg of rhDNase I in CF patients suggested possible reductions in pulmonary obstruction and increased rates of mucociliary sputum clearance from the inner zone of the lung compared to controls (Laube et al 1996). This finding was not confirmed in a crossover design study using once-daily dosing, suggesting that improvement of mucociliary clearance may require higher doses (Robinson et al 2000). Epidemiologic evaluation of patients changing from once-daily dosing to twice-daily and vice versa has also shown improvement in lung function and fewer pulmonary exacerbations on higher doses, although the clinical indication for a dose change is not clear (VanDevanter et al 2018).…”

Section: ■ In Vivo Activity In Cf Sputummentioning

confidence: 99%