Inhaled medications in cystic fibrosis beyond antibiotics

Sepe, Angela; Villella, Valeria Rachela; Cimbalo, Chiara; Castaldo, Alice; Nunziata, Francesco; Corcione, Adele; Bona, Gianni; Maiuri, Luigi; Raia, Valeria

doi:10.23736/s0026-4946.19.05509-9

Cited by 2 publications

(2 citation statements)

References 24 publications

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“…Furthermore, we recorded other complications, i.e., meconium ileus, metabolic alkalosis and distal intestine obstructive syndrome (DIOS) as described [ 2 ]. Finally, we recorded all biochemical parameters [ 19 ], the number of hospital access and therapies [ 20 ].…”

Section: Methodsmentioning

confidence: 99%

Cystic Fibrosis-Screening Positive Inconclusive Diagnosis: Newborn Screening and Long-Term Follow-Up Permits to Early Identify Patients with CFTR-Related Disorders

et al. 2020

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Background: Newborn screening (NBS) early-identifies cystic fibrosis (CF), but in CF-screening positive inconclusive diagnosis (CF-SPID) the results of immunoreactive trypsinogen (IRT), molecular analysis and sweat test (ST) are discordant. A percentage of CF-SPID evolves to CF, but data on long-term monitoring are lacking. We describe the follow-up of all CF and CF-SPID identified between 2008 and 2019. Methods: NBS was performed by IRT followed by molecular analysis and ST between 2008 and 2014; double IRT followed by molecular analysis and ST after 2014. Results: NBS revealed 47 CF and 99 CF-SPID newborn, a ratio 1:2.1—the highest reported so far. This depends on the identification by gene sequencing of the second variant with undefined effect in 40 CF-SPID that otherwise would have been defined as carriers. Clinical complications and pulmonary infections occurred more frequently among CF patients than among CF-SPID. Two CF-SPID cases evolved to CF (at two years), while eight evolved to CFTR-related disorders (CFTR-RD), between one and eight years, with bronchiectasis (two), recurrent pneumonia (four, two with sinonasal complications), recurrent pancreatitis (two). No clinical, biochemical or imaging data predicted the evolution. Conclusion: Gene sequencing within the NBS reveals a higher number of CF-SPID and we first describe an approach to early identify CFTR-RD, with relevant impact on their outcome.

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Section: Methodsmentioning

confidence: 99%

Cystic Fibrosis-Screening Positive Inconclusive Diagnosis: Newborn Screening and Long-Term Follow-Up Permits to Early Identify Patients with CFTR-Related Disorders

et al. 2020

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“…However, the CF Foundation has determined that there is not enough evidence to recommend for or against these drugs in patients with CF (Dasenbrook and Chmiel, 2017). Immunoglobulins and dornase alpha (DNase) (Sepe et al, 2019) are also used as antiinflammatory agents in addition to their mucolytic activity.…”

Section: Anti-inflammatory Drugsmentioning

confidence: 99%

Nanomedicine Approaches for the Pulmonary Treatment of Cystic Fibrosis

Velino

Carella

Adamiano

et al. 2019

Front. Bioeng. Biotechnol.

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Cystic fibrosis (CF) is a genetic disease affecting today nearly 70,000 patients worldwide and characterized by a hypersecretion of thick mucus difficult to clear arising from the defective CFTR protein. The overproduction of the mucus secreted in the lungs, along with its altered composition and consistency, results in airway obstruction that makes the lungs susceptible to recurrent and persistent bacterial infections and endobronchial chronic inflammation, which are considered the primary cause of bronchiectasis, respiratory failure, and consequent death of patients. Despite the difficulty of treating the continuous infections caused by pathogens in CF patients, various strategies focused on the symptomatic therapy have been developed during the last few decades, showing significant positive impact on prognosis. Moreover, nowadays, the discovery of CFTR modulators as well as the development of gene therapy have provided new opportunity to treat CF. However, the lack of effective methods for delivery and especially targeted delivery of therapeutics specifically to lung tissues and cells limits the efficiency of the treatments. Nanomedicine represents an extraordinary opportunity for the improvement of current therapies and for the development of innovative treatment options for CF previously considered hard or impossible to treat. Due to the peculiar environment in which the therapies have to operate characterized by several biological barriers (pulmonary tract, mucus, epithelia, bacterial biofilm) the use of nanotechnologies to improve and enhance drug delivery or gene therapies is an extremely promising way to be pursued. The aim of this review is to revise the currently used treatments and to outline the most recent progresses about the use of nanotechnology for the management of CF.

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Inhaled medications in cystic fibrosis beyond antibiotics

Cited by 2 publications

References 24 publications

Cystic Fibrosis-Screening Positive Inconclusive Diagnosis: Newborn Screening and Long-Term Follow-Up Permits to Early Identify Patients with CFTR-Related Disorders

Cystic Fibrosis-Screening Positive Inconclusive Diagnosis: Newborn Screening and Long-Term Follow-Up Permits to Early Identify Patients with CFTR-Related Disorders

Nanomedicine Approaches for the Pulmonary Treatment of Cystic Fibrosis

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