Abstract
Background and Objectives:
There are limited data on cystic fibrosis (CF) transmembrane conductance
regulator-related metabolic syndrome (CRMS) outcomes beyond infancy. The
goal of this study was to analyze outcomes of infants with CRMS up to
the age of 9-10 years using the CF Foundation Patient Registry (CFFPR).
Methods:
We analyzed data from the CFFPR for individuals with CF and CRMS born
between 2010-2020. We classified all patients based on the clinical
diagnosis reported by the CF care center and the diagnosis using CFF
guideline definitions for CF and CRMS, classifying children into groups
based on agreement between clinical report and guideline criteria.
Descriptive statistics for the cohort were calculated for demographics,
nutritional outcomes, and microbiology for the first year of life and
lung function and growth outcomes were summarized for ages 6-10 years.
Results:
From 2010-2020, there were 8,765 children with diagnosis of CF or CRMS
entered into the CFFPR with sufficient diagnostic data for
classification, of which 7,591 children had a clinical diagnosis of CF
and 1,174 had a clinical diagnosis of CRMS. CRMS patients exhibited
normal nutritional indices and pulmonary function up to age 9-10 years.
The presence of respiratory bacteria associated with CF, such asPseudomonas aeruginosa from CRMS patients ranged from 2.1-9.1%
after the first year of life.
Conclusions:
Children with CRMS demonstrate normal pulmonary and nutritional outcomes
into school age. However, a small percentage of children continue to
culture CF-associated respiratory pathogens after infancy.
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