Effect of Dornase Alfa on the Lung Clearance Index in Children with Cystic Fibrosis: A Lesson from a Case Series

Terlizzi, Vito; Parisi, Giuseppe Fabio; Ferrari, Béatrice; Castellani, Chiara; Manti, Sara; Leonardi, ﻿Salvatore; Taccetti, Giovanni

doi:10.3390/children9111625

Cited by 5 publications

(7 citation statements)

References 41 publications

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“…More recently, we have confirmed the sensitivity of LCI as a marker of disease in CF and in monitoring response to treatment, particularly with DNase (74).…”

Section: Our Experiencesupporting

confidence: 58%

New trends in pediatric pulmonology: our experience

Parisi¹,

Papale²,

Manti³

et al. 2023

Pediatr Respir J

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Until a few years ago, pediatric pulmonology was one of the most static branches of pediatrics. In recent years, the description of new pathological entities affecting the lung and airways, the identification of new disease biomarkers, the use of new respiratory function tests suitable not only for collaborating but also for non-collaborating children, the enhancement of respiratory endoscopy, the advent of new therapies such as biological drugs and genetic modulators have made pediatric pulmonology one of the most dynamic branches of pediatric medicine.The objective of this article will be to explore the new fields of pediatric pulmonology by making a parallelism with the evolutions that our center had to follow in order to adapt to the new healthcare and research standards. These same standards of care are needed by patients, their families, and need to be known by primary care pediatricians. In addition, health policy must take into account these upgrades in pediatric pulmonology to provide the best quality of care to all patients and uniformly throughout the country. CF is the most common life-shortening autosomal recessive hereditary disease in Caucasians with a prevalence of 1 case per 2500 live births. CF is a multisystem disease caused by mutations in the cystic fibrosis trans-membrane conductance regulator (CFTR) gene (11, 12). The lung is the primary site of CF, to which most of the CF-associated patient morbidity and mortality is linked. CF occurs very early in infancy and is defined by the presence of infections and chronic inflammation leading to a deterioration in lung function, respiratory exacerbations, and bronchiectasis (13,14). Thus, identifying a biomarker for disease progression might be crucial for improving these patients' outcomes. Moreover, with therapeutic introduction of potentiators and correctors of the CFTR protein, the study of biomarkers could be a valuable tool for monitoring their effectiveness over time (15). Globally, therefore, the study of biomarkers could have a strategic role in CF management and also in asthma. FRACTIONAL-EXHALED NITRIC OXIDE (FeNO)asthma treatment. Finally, CaNO is the least used in clinical practice because lack of standardization of measurement techniques. VOLATILE ORGANIC COMPOUNDS (VOCs) AND EXHALED BREATH CONDENSATE (EBC)

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“…More recently, we have confirmed the sensitivity of LCI as a marker of disease in CF and in monitoring response to treatment, particularly with DNase (74).…”

Section: Our Experiencesupporting

confidence: 58%

New trends in pediatric pulmonology: our experience

Parisi¹,

Papale²,

Manti³

et al. 2023

Pediatr Respir J

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“…In our center, LCI was performed every 6 months for stable children weighing more than 15 kg. Furthermore, LCI examinations were excluded from the current evaluation after the introduction of new drugs, such dornase alfa 22 . None of the included children took CFTR modulators.…”

Section: Methodsmentioning

confidence: 99%

“…Furthermore, LCI examinations were excluded from the current evaluation after the introduction of new drugs, such dornase alfa. 22 None of the included children took CFTR modulators. A stable condition was defined as having no symptoms or signs of pulmonary exacerbation according to CF Foundation criteria.…”

Section: Introduction Cystic Fibrosis (Cf) Is the Most Common Inherit...mentioning

confidence: 99%

“…Although the prognostic value of LCI is not yet that clear on parameters such as mortality, it is part of routine assessment of patients with CF (pwCF) in many centers and is useful to identify early lung disease in preschool children and monitor therapeutic interventions. [19][20][21][22] Here, the LCI was measured in: 9 Children were defined as CF if they were NBS positive with a raised SC (≥60 mmol/l) and/or had two CF-causing variants of the CFTR gene. 24 With the term "asymptomatic" we mean the absence of CF-related symptoms or chronic respiratory symptoms.…”

Section: Introduction Cystic Fibrosis (Cf) Is the Most Common Inherit...mentioning

confidence: 99%

“…To date there is an increasing acknowledgment of the LCI technique. Although the prognostic value of LCI is not yet that clear on parameters such as mortality, it is part of routine assessment of patients with CF (pwCF) in many centers and is useful to identify early lung disease in preschool children and monitor therapeutic interventions 19–22 …”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Lung clearance index in children with cystic fibrosis previously diagnosed with CRMS/CFSPID: A monocentric prospective experience

Terlizzi

Fevola

Ferrari

et al. 2023

Pediatric Pulmonology

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Introduction No data are available on the values and role of lung clearance index (LCI) in cystic fibrosis (CF) Screen Positive Inconclusive Diagnosis (CFSPID) progressed to CF diagnosis (CFSPID > CF). This study aimed to assess the value of the LCI in correctly predicting the progression of CFSPID to CF. Methods This is a prospective study carried out at the CF Regional Center of Florence, Italy from September 1, 2019. We compared LCI values in children with CF diagnosed for positive newborn screening (NBS), CFSPID or CFSPID > CF for pathological sweat chloride (SC). The Exhalyzer‐D (EcoMedics AG, Duernten, Switzerland, software version 3.3.1) was used to conduct the LCI tests, every 6 months on stable children. Results Forty‐two cooperating children were enrolled (mean age at LCI tests: 5.4 years, range: 2.7−8.7): 26 (62%) had CF, 8 (19%) were CFSPID > CF for positive SC, while 8 (19%) kept the CFSPID label at last LCI test. The mean LCI value for patients with CF (7.39; 5.98−10.24) was statistically higher compared to both the mean LCI in the CFSPID > CF (6.62; 5.69−7.58) and in CFSPID (6.56; 5.64−7.21). Conclusions Most of asymptomatic CFSPID or progressed to CF have normal LCI. Further data on the longitudinal course of LCI during follow up of CFSPID and on larger cohorts is needed.

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