Literature DB >> 26454351

Cystic fibrosis in young children: A review of disease manifestation, progression, and response to early treatment.

Donald R VanDevanter1, Jennifer S Kahle2, Amy K O'Sullivan3, Slaven Sikirica3, Paul S Hodgkins3.   

Abstract

BACKGROUND: Studies have described illness associated with cystic fibrosis (CF) early in life, but there is no comprehensive accounting of the prevalence and ages of disease manifestation and progression described in individual studies.
METHODS: We searched for peer-reviewed English-language studies of the health of children ≤6years old with CF (published 1990-2014). Structural abnormalities and dysfunction of the digestive and respiratory systems were summarized across relevant studies by system and age group.
RESULTS: Primary studies (125 total) from 22 countries described abnormalities, dysfunction, and disease progression in infancy and early childhood. Improved health was consistently observed in association with diagnosis via newborn screening compared with cohorts diagnosed later by symptomatic presentation.
CONCLUSIONS: The peer-reviewed literature is remarkably consistent: CF-associated growth impairment and airway abnormalities are reported at birth, and disease progression is reported in infancy and throughout childhood. Earlier access to routine CF management is associated with improved subsequent health status.
Copyright © 2015 The Authors. Published by Elsevier B.V. All rights reserved.

Entities:  

Keywords:  Child; Comprehensive survey; Data collection; Disease progression; Health status; Infant; Preschool

Mesh:

Year:  2015        PMID: 26454351     DOI: 10.1016/j.jcf.2015.09.008

Source DB:  PubMed          Journal:  J Cyst Fibros        ISSN: 1569-1993            Impact factor:   5.482


  31 in total

1.  Low linoleic and high docosahexaenoic acids in a severe phenotype of transgenic cystic fibrosis mice.

Authors:  Birgitta Strandvik; Wanda K O Neal; Mohamed A Ali; Ulf Hammar
Journal:  Exp Biol Med (Maywood)       Date:  2018-03

2.  Development of an airway mucus defect in the cystic fibrosis rat.

Authors:  Susan E Birket; Joy M Davis; Courtney M Fernandez; Katherine L Tuggle; Ashley M Oden; Kengyeh K Chu; Guillermo J Tearney; Michelle V Fanucchi; Eric J Sorscher; Steven M Rowe
Journal:  JCI Insight       Date:  2018-01-11

3.  Lumacaftor/Ivacaftor in Patients Aged 6-11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR.

Authors:  Carlos E Milla; Felix Ratjen; Gautham Marigowda; Fang Liu; David Waltz; Margaret Rosenfeld
Journal:  Am J Respir Crit Care Med       Date:  2017-04-01       Impact factor: 21.405

Review 4.  Early Lung Disease in Infants and Preschool Children with Cystic Fibrosis. What Have We Learned and What Should We Do about It?

Authors:  Sarath C Ranganathan; Graham L Hall; Peter D Sly; Stephen M Stick; Tonia A Douglas
Journal:  Am J Respir Crit Care Med       Date:  2017-06-15       Impact factor: 21.405

5.  Impact of guideline-recommended dietitian assessments on weight gain in infants with cystic fibrosis.

Authors:  Thida Ong; Frankline M Onchiri; Maria T Britto; Sonya L Heltshe; Larry G Kessler; Michael Seid; Bonnie W Ramsey
Journal:  J Cyst Fibros       Date:  2021-08-26       Impact factor: 5.482

6.  Elastase Exocytosis by Airway Neutrophils Is Associated with Early Lung Damage in Children with Cystic Fibrosis.

Authors:  Camilla Margaroli; Luke W Garratt; Hamed Horati; A Susanne Dittrich; Timothy Rosenow; Samuel T Montgomery; Dario L Frey; Milton R Brown; Carsten Schultz; Lokesh Guglani; Anthony Kicic; Limin Peng; Bob J Scholte; Marcus A Mall; Hettie M Janssens; Stephen M Stick; Rabindra Tirouvanziam
Journal:  Am J Respir Crit Care Med       Date:  2019-04-01       Impact factor: 21.405

7.  Refining the continuum of CFTR-associated disorders in the era of newborn screening.

Authors:  H Levy; M Nugent; K Schneck; D Stachiw-Hietpas; A Laxova; O Lakser; M Rock; M K Dahmer; J Biller; S Z Nasr; M Baker; S A McColley; P Simpson; P M Farrell
Journal:  Clin Genet       Date:  2016-01-20       Impact factor: 4.438

8.  Potential for Therapeutic Benefit among Cystic Fibrosis Populations Excluded from Clinical Trials or Labeling of Marketed Therapies.

Authors:  Donald R VanDevanter; Sonya L Heltshe; John J LiPuma
Journal:  Ann Am Thorac Soc       Date:  2016-11

9.  Long-Term Impact of Ivacaftor on Healthcare Resource Utilization Among People with Cystic Fibrosis in the United States.

Authors:  Teja Thorat; Lisa J McGarry; Krutika Jariwala-Parikh; Brendan Limone; Machaon Bonafede; Keval Chandarana; Michael W Konstan
Journal:  Pulm Ther       Date:  2021-04-28

Review 10.  Quantification of Phenotypic Variability of Lung Disease in Children with Cystic Fibrosis.

Authors:  Mirjam Stahl; Eva Steinke; Marcus A Mall
Journal:  Genes (Basel)       Date:  2021-05-25       Impact factor: 4.096

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