Effect of CFTR Modulators on Anthropometric Parameters in Individuals with Cystic Fibrosis: An Evidence Analysis Center Systematic Review


      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.
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      J. Bailey is a clinical dietitian, University of Alabama at Birmingham.


      M. Rozga is a nutrition researcher, Academy of Nutrition and Dietetics, Evidence Analysis Center, Chicago, IL.


      C. M. McDonald is a clinical dietitian, Primary Children's Hospital, Salt Lake City, UT.


      E. K. Bowser is an associate in pediatrics and faculty nutritionist, Pediatric Pulmonary Division, University of Florida College of Medicine, Gainesville.


      K. Farnham is a clinical pediatric dietitian, Nemours Children's Specialty Care, Jacksonville, FL.


      M. Mangus is a patient advocate, San Antonio, TX.


      L. Padula is an advanced practice dietitian, Children's Hospital of Philadelphia, Philadelphia, PA.


      K. Porco is a patient advocate, Attain Health Foundation, Red Lodge, MT.


      J. A. Alvarez is an assistant professor, Emory University School of Medicine, Atlanta, GA.

      Linked Article

      • Dietary Macronutrient Distribution and Nutrition Outcomes in Persons with Cystic Fibrosis: An Evidence Analysis Center Systematic Review
        Journal of the Academy of Nutrition and DieteticsVol. 121Issue 8
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          Cystic fibrosis (CF) increases risk for undernutrition and malabsorption. Individuals with CF traditionally have been counseled to consume a high-fat diet. However, a new era of CF care has increased lifespan and decreased symptoms in many individuals with CF, necessitating a re-examination of the high-fat CF legacy diet. A literature search was conducted of Medline (Ovid), Embase, and CINAHL (EBSCO) databases to identify articles published from January 2002 to May 2018 in the English language examining the relationships between dietary macronutrient distribution and nutrition outcomes in individuals with CF.
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