Tags

Type your tag names separated by a space and hit enter

Assessing the usefulness of outcomes measured in a cystic fibrosis treatment trial.
Respir Med. 2007 Feb; 101(2):254-60.RM

Abstract

Forced expiratory volume in 1s (FEV(1)) is the usual primary outcome variable in clinical trials in cystic fibrosis (CF). Usually, several secondary outcomes are also measured. We assessed which secondary outcomes are likely to give additional clinically useful information about treatment effects, in order to inform the design of future studies. The study was performed as part of a trial comparing daily rhDNase with alternate day rhDNase and hypertonic saline in CF. The primary outcome was FEV(1). Secondary outcomes were forced vital capacity (FVC), forced expiratory flow at 25-75% of forced vital capacity (FEF(25-75)), number of pulmonary exacerbations, weight gain, quality of life (QOL), and exercise tolerance. The usefulness of each secondary outcome was investigated by assessing if the change in that outcome over the treatment period could be predicted from the primary outcome. Change in FEV(1) correlated with changes in FVC (r(2)=0.76, P=0.001), FEF(25-75) (r(2)=0.64, P=0.001), weight (r(2)=0.08, P=0.001), and change in oxygen saturation with exercise (r(2)=0.08, P=0.001). However, it did not correlate with changes in visual analogue score (VAS) with exercise, QOL, nor with the occurrence of pulmonary exacerbations. Only the outcomes QOL and VAS with exercise actually provided additional information to FEV(1) in this study.

Authors+Show Affiliations

Respiratory Unit, Great Ormond Street Hospital for Children NHS Trust, Great Ormond Street, London, UK. surir@gosh.nhs.ukNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

16806875

Citation

Suri, Ranjan, et al. "Assessing the Usefulness of Outcomes Measured in a Cystic Fibrosis Treatment Trial." Respiratory Medicine, vol. 101, no. 2, 2007, pp. 254-60.
Suri R, Metcalfe C, Wallis C, et al. Assessing the usefulness of outcomes measured in a cystic fibrosis treatment trial. Respir Med. 2007;101(2):254-60.
Suri, R., Metcalfe, C., Wallis, C., & Bush, A. (2007). Assessing the usefulness of outcomes measured in a cystic fibrosis treatment trial. Respiratory Medicine, 101(2), 254-60.
Suri R, et al. Assessing the Usefulness of Outcomes Measured in a Cystic Fibrosis Treatment Trial. Respir Med. 2007;101(2):254-60. PubMed PMID: 16806875.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Assessing the usefulness of outcomes measured in a cystic fibrosis treatment trial. AU - Suri,Ranjan, AU - Metcalfe,Chris, AU - Wallis,Colin, AU - Bush,Andrew, Y1 - 2006/06/27/ PY - 2005/10/13/received PY - 2006/05/05/revised PY - 2006/05/16/accepted PY - 2006/6/30/pubmed PY - 2007/4/3/medline PY - 2006/6/30/entrez SP - 254 EP - 60 JF - Respiratory medicine JO - Respir Med VL - 101 IS - 2 N2 - Forced expiratory volume in 1s (FEV(1)) is the usual primary outcome variable in clinical trials in cystic fibrosis (CF). Usually, several secondary outcomes are also measured. We assessed which secondary outcomes are likely to give additional clinically useful information about treatment effects, in order to inform the design of future studies. The study was performed as part of a trial comparing daily rhDNase with alternate day rhDNase and hypertonic saline in CF. The primary outcome was FEV(1). Secondary outcomes were forced vital capacity (FVC), forced expiratory flow at 25-75% of forced vital capacity (FEF(25-75)), number of pulmonary exacerbations, weight gain, quality of life (QOL), and exercise tolerance. The usefulness of each secondary outcome was investigated by assessing if the change in that outcome over the treatment period could be predicted from the primary outcome. Change in FEV(1) correlated with changes in FVC (r(2)=0.76, P=0.001), FEF(25-75) (r(2)=0.64, P=0.001), weight (r(2)=0.08, P=0.001), and change in oxygen saturation with exercise (r(2)=0.08, P=0.001). However, it did not correlate with changes in visual analogue score (VAS) with exercise, QOL, nor with the occurrence of pulmonary exacerbations. Only the outcomes QOL and VAS with exercise actually provided additional information to FEV(1) in this study. SN - 0954-6111 UR - https://www.unboundmedicine.com/medline/citation/16806875/Assessing_the_usefulness_of_outcomes_measured_in_a_cystic_fibrosis_treatment_trial_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0954-6111(06)00274-5 DB - PRIME DP - Unbound Medicine ER -