Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients

Sawicki, Gregory S.; Chou, Willis; Raimundo, Karina; Trzaskoma, Ben; Konstan, Michael W.

doi:10.1016/j.jcf.2015.04.003

Cited by 33 publications

(16 citation statements)

References 21 publications

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“…But the overall positive effect of DNase I on patients with cystic fibrosis was proven repeatedly. Positive outcomes from clinical studies led to Food and Drug Administration (FDA) approval and wide use of DNase I for this treatment [113][114][115]. [130] Acute kidney injury rats intraperitoneal 0.1 mg/kg positive [131] Acute liver injury rats intravenous 10 mg/kg positive [132] Ischemic-reperfusion syndrome mice intraperitoneal intravenous 50 µg, 2 times 10 µg, 1 time positive [133] Empyema thoracis humans intrapleural 2.5 mg (1-2 times) positive [134] A similar effect of DNase was also seen in other respiratory diseases.…”

Section: Application Of Dnase and Dnase Treatmentmentioning

confidence: 99%

Deoxyribonucleases and Their Applications in Biomedicine

et al. 2020

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Extracellular DNA, also called cell-free DNA, released from dying cells or activated immune cells can be recognized by the immune system as a danger signal causing or enhancing inflammation. The cleavage of extracellular DNA is crucial for limiting the inflammatory response and maintaining homeostasis. Deoxyribonucleases (DNases) as enzymes that degrade DNA are hypothesized to play a key role in this process as a determinant of the variable concentration of extracellular DNA. DNases are divided into two families—DNase I and DNase II, according to their biochemical and biological properties as well as the tissue-specific production. Studies have shown that low DNase activity is both, a biomarker and a pathogenic factor in systemic lupus erythematosus. Interventional experiments proved that administration of exogenous DNase has beneficial effects in inflammatory diseases. Recombinant human DNase reduces mucus viscosity in lungs and is used for the treatment of patients with cystic fibrosis. This review summarizes the currently available published data about DNases, their activity as a potential biomarker and methods used for their assessment. An overview of the experiments with systemic administration of DNase is also included. Whether low-plasma DNase activity is involved in the etiopathogenesis of diseases remains unknown and needs to be elucidated.

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Section: Application Of Dnase and Dnase Treatmentmentioning

confidence: 99%

Deoxyribonucleases and Their Applications in Biomedicine

et al. 2020

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“…This device is battery powered (thus is a mobile device) and delivers drug much faster than traditional nebulizers potentially leading to the high rate of adherence noted in an 18 month open label extension study . Recently, a similar device (e‐Rapid) has been studied for delivery of medications such as dornase alfa . Dry‐powder drug formulation may also decrease the patient burden for certain aerosolized medications.…”

Section: Tackling the Solutions: Integration Of Technologies To Cf Thmentioning

confidence: 99%

Tackling the increasing complexity of CF care

Sawicki

Goss

2015

Pediatric Pulmonology

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Summary Health outcomes for individuals with cystic fibrosis (CF) have dramatically improved in parallel with better organization of clinical care systems, evolution of novel therapeutics, and improvements in diagnosis and screening for CF and CF‐related complications. In parallel with these advances has come an increasing complexity and burden of care, leading to challenges with adherence to treatment regimens. As novel therapeutics continue to be developed and introduced to the CF care regimen, there are clear opportunities to refine and personalize care. This can be done by adding comparative effectiveness research to the CF clinical research paradigm and integrating novel technologies in drug delivery and remote monitoring that can facilitate adherence but also reduce the burden of treatment while maintaining efficacy. This review highlights both the challenges of the increasingly complex treatment regimens in CF and the opportunities to advance care by addressing adherence, implementation science, comparative effectiveness, and integration of novel technologies in CF care. Pediatr Pulmonol. 2015; 50:S74–S79. © 2015 Wiley Periodicals, Inc.

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“…Pulmozyme is a recombinant version of human Dnase1 marketed by Genentech for the treatment of CF. Pulmozyme is administered via nebulization and has been shown to reduce the incidence of CF infections [ 56 ].…”

Section: Reviewmentioning

confidence: 99%

Inhaled protein/peptide-based therapies for respiratory disease

2016

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Asthma, chronic obstructive pulmonary disease (COPD), and cystic fibrosis (CF) are all chronic pulmonary diseases, albeit with different etiologies, that are characterized by airflow limitation, chronic inflammation, and abnormal mucus production/rheology. Small synthetic molecule-based therapies are commonly prescribed for all three diseases. However, there has been increased interest in “biologicals” to treat these diseases. Biologicals typically constitute protein- or peptide-based therapies and are often more potent than small molecule-based drugs. In this review, we shall describe the pros and cons of several different biological-based therapies for respiratory disease, including dornase alfa, a recombinant DNAase that reduces mucus viscosity and short palate lung and nasal epithelial clone 1 (SPLUNC1)-derived peptides that treat Na+ hyperabsorption and rebalance CF airway surface liquid homeostasis.

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Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients

Abstract: Administration of dornase alfa via the eRapid nebulizer resulted in comparable efficacy and safety, shorter nebulization times, and higher patient preference.

Cited by 33 publications

References 21 publications

Deoxyribonucleases and Their Applications in Biomedicine

Deoxyribonucleases and Their Applications in Biomedicine

Tackling the increasing complexity of CF care

Inhaled protein/peptide-based therapies for respiratory disease

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