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Review
. 2016 Jan;15(1):10-20.
doi: 10.1016/j.jcf.2015.03.015. Epub 2015 Apr 15.

Is the raised volume rapid thoracic compression technique ready for use in clinical trials in infants with cystic fibrosis?

Stefan Matecki  1 , Lisa Kent  2 , Kris de Boeck  3 , Muriel Le Bourgeois  4 , Stefan Zielen  5 , Cesare Braggion  6 , H G M Arets  7 , Judy Bradley  2 , Stephanie Davis  8 , Isabelle Sermet  9 , Philippe Reix  10 ; respiratory function group of the European Cystic Fibrosis Society Clinical Trial Network
Affiliations
Free article
Review

Is the raised volume rapid thoracic compression technique ready for use in clinical trials in infants with cystic fibrosis?

Stefan Matecki et al. J Cyst Fibros. 2016 Jan.
Free article

Abstract

The European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) has established a Standardization Committee to undertake a rigorous evaluation of promising outcome measures with regard to use in multicentre clinical trials in cystic fibrosis (CF). The aim of this article is to present a review of literature on clinimetric properties of the infant raised-volume rapid thoracic compression (RVRTC) technique in the context of CF, to summarise the consensus amongst the group on feasibility and answer key questions regarding the promotion of this technique to surrogate endpoint status.

Methods: A literature search (from 1985 onwards) identified 20 papers that met inclusion criteria of RVRTC use in infants with CF. Data were extracted and tabulated regarding repeatability, validity, correlation with other outcome measures, responsiveness and reference values. A working group discussed the tables and answered 4 key questions.

Results: Overall, RVRTC in particular forced expiratory volume in 0.5s, showed good clinimetric properties despite presence of individual variability. Few studies showed a relationship between RVRTC and inflammation and infection, and to date, data remains limited regarding the responsiveness of RVRTC after an intervention. Concerns were raised regarding feasibility in multi-centre studies and availability of reference values.

Conclusion: The ECFS-CTN Working Group considers that RVRTC cannot be used as a primary outcome in clinical trials in infants with CF before universal standardization of this measurement is achieved and implementation of inter-institutional networking is in place. We advise its use currently in phase I/II trials and as a secondary endpoint in phase III studies. We emphasise the need for (1) more short-term variability and longitudinal 'natural history' studies, and (2) robust reference values for commercially available devices.

Keywords: Clinical trials; Clinimetric properties; Cystic fibrosis; Infant pulmonary function; Raised volume rapid thoracic compression; Surrogate outcome.

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