There is a strong positive association between nutrition status and lung function in
cystic fibrosis (CF). Improvements in clinical care have increased longevity for individuals with CF, and it is unknown how
cystic fibrosis transmembrane regulator (CFTR) modulation
therapy affects nutrition status over time. The objective of this systematic review of the literature was to examine anthropometric (height, weight, and body mass index [BMI; calculated as kg/m2]) and body composition outcomes of CFTR modulation
therapy. A literature search of Medline (Ovid), Embase, and CINAHL (EBSCO) databases was conducted for randomized controlled trials examining the effect of CFTR modulation
therapy on anthropometric and body composition parameters, published in peer-reviewed journals from January 2002 until May 2018. Articles were screened, data were synthesized qualitatively, and evidence quality was graded by a team of content experts and systematic review methodologists. Significant
weight gain with
ivacaftor was noted in children and adults with at least 1 copy of G551D mutation. In adults with at least 1 copy of R117H the effect of
ivacaftor on BMI was not significant. Effects on BMI were mixed in adults with class II mutations taking
ivacaftor with
lumacaftor. There was no significant change in BMI in children homozygous for F508del who took
ivacaftor with
tezacaftor.
Elexacaftor-
tezacaftor-ivacaftor increased BMI and
body weight in individuals 12 years of age and older who were hetero- or homozygous for the F508del mutation. The effect of CFTR modulation
therapy on anthropometric parameters depends on the genetic mutation and the type of modulation
therapy used. More research is needed to understand the long-term clinical impact of these drugs on nutritional status, including body composition and the role of dietary intake.