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Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis

Stephanie D Davis1, Felix Ratjen2, Lyndia C Brumback3

  • 1Section of Pediatric Pulmonology, Allergy and Sleep Medicine, Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, IN, USA.

Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society
|November 9, 2015
PubMed

Insights

Infant pulmonary function tests (iPFTs) in cystic fibrosis (CF) trials are not ideal primary endpoints. While raised volume measures show potential, data collection challenges limit iPFT feasibility in multicenter studies.

Area of Science:

  • Pediatric Pulmonology
  • Clinical Trials in Cystic Fibrosis
  • Respiratory Medicine

Background:

  • The Infant Study of Inhaled Saline (ISIS) in CF was a pioneering multicenter clinical trial.
  • It was the first to incorporate infant pulmonary function tests (iPFTs) as a study endpoint.

Purpose of the Study:

  • To evaluate the feasibility of using iPFT measures in a multicenter clinical trial setting.
  • To assess the association between iPFT measures and respiratory symptoms in infants with CF.
  • To calculate standard deviations for power calculations in future CF clinical trials.

Main Methods:

  • Secondary analysis of data from the ISIS clinical trial.
  • Inclusion of 73 participants, with 70 completing the final visit.
  • Assessment of functional residual capacity (FRC) and raised volume measurements for acceptability.

Main Results:

  • 89% of participants had acceptable paired FRC measurements, while 64% had acceptable raised volume measurements.
  • Mean baseline z-scores for FEV0.5, FEF75, and FRC were 0.3, -0.2, and 1.8, respectively.
  • Calculated standard deviations were obtained for power calculations.

Conclusions:

  • iPFTs present challenges for data acquisition, making them unsuitable as primary endpoints in multicenter CF trials.
  • Raised volume measurements show promise as secondary endpoints in future clinical trials for CF.
  • Further refinement of iPFT methodologies is needed for their broader application in pediatric respiratory research.
Abstract

Keywords:
FEVForced expiratory flow ratesPulmonary function tests

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