Marcus J. Couch1,2, Robert Thomen3, Felix Ratjen1,4, Jason Woods5, and Giles Santyr1,2
1Translational Medicine Program, The Hospital for Sick Children, Toronto, ON, Canada, 2Department of Medical Biophysics, University of Toronto, Toronto, ON, Canada, 3School of Medicine, University of Missouri, Columbia, MO, United States, 4Division of Respiratory Medicine, The Hospital for Sick Children, Toronto, ON, Canada, 5Center for Pulmonary Imaging Research, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, United States
Synopsis
The ventilation defect percent (VDP), measured
from hyperpolarized 129Xe MRI, is sensitive to functional changes in
early cystic fibrosis (CF) lung disease; however, there is no consensus on
which VDP calculation method is most appropriate for future multi-center
clinical trials in CF. This study compared VDP analysis methods in
hyperpolarized 129Xe datasets acquired in stable pediatric CF subjects
at two institutions. In a combined dataset, a comparison of k-means,
mean-anchored linear binning, and 99th percentile-anchored linear
binning demonstrated that all three methods provide a good characterization of
the disease, but mean-anchored linear binning provided the strongest
correlation to pulmonary function tests.
Introduction
The spatial distribution of ventilation defects
in hyperpolarized (HP) 129Xe MRI provides insight into cystic
fibrosis (CF) lung disease1 and treatment.2 The ventilation defect percent (VDP)
measured from HP 129Xe images provides a sensitive measure of ventilation
heterogeneity in early cystic fibrosis (CF) lung disease.3,4 VDP is potentially
more sensitive for detecting early functional changes in CF lung disease
compared to pulmonary function test (PFT) indices, such as the forced
expiratory volume in one second (FEV1) and the lung clearance index
(LCI).3,5 VDP can be calculated
by segmenting the HP 129Xe image using k-means3,6 or linear binning4,7,8 to quantify the
fraction of the lung that corresponds to ventilation defect. A recent single-center
comparative study has found a good agreement between adaptive k-means and
linear binning VDP measurements in adults with asthma9; however, it is
unknown which method will be the most appropriate for future multi-center
prospective clinical trials in CF. The purpose of this study was to perform a
retrospective analysis of HP 129Xe images from stable pediatric CF
subjects obtained at two different institutions using slightly different MRI protocols
to assess the agreement between VDP calculation methods as well as the
correlation between VDP and PFT measurements (i.e., FEV1 and LCI). Methods
This retrospective analysis included 26 participants
from two institutions (18 CF, 8 healthy, age range 10–17). Pulmonary function
tests, N2 multiple breath washout, and HP 129Xe MRI were
performed using previously described methods.3,4 Table
1 shows the subject demographics for the two sites, and Table 2 shows the MR
acquisition parameters. VDP was calculated using (i) k-means clustering according
to Santyr et al.1, (ii)
mean-anchored linear binning according to Thomen et al.4, and (iii) 99th
percentile-anchored linear binning according to He et al.7 For each analysis
method, VDP was calculated as the total volume of unventilated lung obtained
from the respective segmented 129Xe images divided by the total lung
volume obtained from the 1H image masks. VDP was compared between
methods using a Bland-Altman analysis. VDP was compared with FEV1
and LCI using a linear regression analysis, where HP 129Xe images
with a center slice SNR below 8.5 were excluded.Results
Figure 1 shows a comparison of segmented
ventilation and defect maps obtained using all three VDP calculation methods in
two representative CF patients at Site #1 and Site #2, respectively. Figure 2
summarizes the VDP results in box-and-whisker plots for each calculation method,
where VDP results are shown separately for each site and also as a combined
dataset. Figure 3 shows Bland-Altman analyses comparing VDP calculation methods
for the combined data set from both sites. Generally, mean-anchored linear
binning provided the highest average VDP, with 99th
percentile-anchored linear binning providing the lowest average VDP. Figures 4(a)
to 4(c) show VDP calculated for each method and plotted against FEV1,
where only mean-anchored linear binning provides a weak but significant
correlation. Figures 4(d) to 4(f) show VDP calculated for each method and
plotted against LCI, and in all cases there was a moderate to strong
correlation.Discussion
This retrospective study is the first to compare
VDP measurements obtained from HP 129Xe MRI performed in stable
pediatric CF subjects at two institutions. Both CF populations were of a
similar age with a range of severities, as defined by LCI and VDP, providing a good
clinical characterization of the disease. For all three analysis methods, the VDP
values calculated for each subject were similar; however, mean-anchored linear
binning resulted in a systematically higher VDP value compared to k-means and
99th percentile-anchored linear binning. Mean-anchored linear
binning uses fixed segmentation thresholds that include poorly ventilated lung
regions (i.e. up to 60% of the mean signal value), in addition to defect, which
appears to provide robust VDP values despite minor acquisition differences between
the two sites. On the other hand, k-means uses variable thresholds that
consider defect only, and 99th percentile-anchored linear binning
used a low threshold (5.6%) that was determined from healthy reference data. Mean-anchored
linear binning was the only method that resulted in a significant correlation
to FEV1, and of all three methods it had the strongest correlation with LCI.Conclusions
VDP determined using mean-anchored linear
binning provides a robust measurement of ventilation heterogeneity in stable
pediatric CF subjects at two sites. Since measurements performed at two sites using
this analysis method yielded similar VDP values, implementation of the
technique in future multi-center trials in CF appears feasible. Acknowledgements
The authors gratefully acknowledge
helpful discussions with members of the 129Xe MRI Clinical Trials
Consortium. The authors would like to thank the following individuals at
SickKids for their help with data collection: Yonni Friedlander, Raymond Hu, Nikhil
Kanhere, Michelle Klingel, Krzysztof Kowalik, Andras Lindenmaier, Tammy Rayner,
Laura Seed, Elaine Stirrat, Ruth Weiss, David Wilson, and Brandon Zanette; in
addition to the following at Cincinnati Children’s: Laura Walkup, Zackary
Cleveland, Erin Watters, and Kelly Thornton. We would also like to thank the
following sources of funding: The Hospital for Sick Children (Catalyst Grant from
the Cystic Fibrosis Centre), Natural Sciences and Engineering Research Council
of Canada (NSERC)
Discovery grant (RGPIN 217015-2013), Canadian
Institutes of Health Research (CIHR) operating and project grants (MOP 123431, PJT
153099), the Cincinnati Children’s Research Foundation, and the National
Institutes of Health (T32 HL007752, R01 HL131012). MJC was funded by a Research Training
Competition (Restracomp) Fellowship from the Hospital for Sick Children and a
Mitacs Elevate Postdoctoral Fellowship.References
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