Riccardo Pascuzzo1, Vikram Venkatraghavan2, Marco Moscatelli1, Marina Grisoli1, Esther E. Bron2, Stefan Klein2, Janis Blevins3, Gianmarco Castelli1, Lawrence B. Schonberger4, Pierluigi Gambetti5, Brian S. Appleby3, and Alberto Bizzi1
1Neuroradiology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy, 2Biomedical Imaging Group Rotterdam, Departments of Medical Informatics & Radiology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, Netherlands, 3National Prion Disease Pathology Surveillance Center, Case Western Reserve University, School of Medicine, Cleveland, OH, United States, 4National Center for Emerging and Zoonotic Infectious Diseases, Centers for Disease Control and Prevention, Atlanta, GA, United States, 5Department of Pathology, Case Western Reserve University, School of Medicine, Cleveland, OH, United States
Synopsis
The subtypes of sporadic Creutzfeldt-Jakob
disease (sCJD), determined only at autopsy, may have different abnormality
patterns in diffusion-weighted magnetic resonance imaging (DW-MRI) according to
few reports. For the first time, we provide temporal cascades of the DW-MRI
abnormalities in seven distinct sCJD subtypes using a data-driven technique
named “discriminative event-based model”. Based on these cascades, we propose a
novel procedure to identify the subtype of a patient. We found that sCJD subtypes
have either initial cortical (MM/MV1, MM/MV2C, VV1 subtypes) or subcortical
involvement (MV2K and VV2) with specific orderings of DW-MRI abnormalities,
allowing a correct subtype prediction in most cases.
INTRODUCTION
Sporadic
Creutzfeldt-Jakob disease (sCJD) is the most common form of human prion
diseases, which are rare and fatal neurodegenerative pathologies with a rapid
course.1 sCJD has several subtypes (denoted as MM1, MV1, MM2C, MV2C,
MV2K, VV1, and VV2) that differ in brain lesion distribution, clinical signs
and survival times,2,3 and may respond differently to drug
treatment.4
To date, no
diagnostic test can reliably distinguish sCJD subtypes ante mortem. MRI has sensitivity and specificity >95% for the early diagnosis of
sCJD when diffusion-weighted imaging (DWI) is examined.5,6,7 It has
been argued that characteristic DWI abnormality patterns may occur for each
subtype,8 but these findings were not exploited for subtype
diagnosis.
We aim to
model DWI abnormality patterns in sCJD using a novel data-driven procedure and
use this model to identify the subtype of patients in two scenarios: (i) using
only MRI-derived data and (ii) including also the codon 129 genotype of the
prion protein gene (PRNP129), a major determinant of disease variability.METHODS
We collected the MRIs (DWI and ADC) of 453
sCJD patients with autopsy-confirmed subtype diagnosis. Brain MRIs were
visually examined by one neuroradiologist blind to the diagnosis, grading the DWI
signal hyperintensities on a 4-point ordinal scale, from “zero” (absence of
CJD-related DWI hyperintensities) to “three” (extensive CJD-related DWI
hyperintensities with low diffusivity on ADC maps). Twelve brain regions were
scored: 5 cortical regions (frontal, temporal, occipital, parietal and
separately precuneus), 3 limbic structures (cingulate, insula, hippocampus),
caudate, putamen, thalamus and cerebellum.
For each of the 7 sCJD subtypes, we applied
the discriminative event-based model (DEBM),9 a novel data-driven
technique that reconstructs the ordering of the 12
brain regions becoming abnormal in DWI.
First, each score was linearly converted into
the probability of the corresponding region becoming abnormal. The
subject-specific ordering of regions was established by sorting in a descending
way the corresponding probabilities of a patient. The (central) ordering of
each subtype was calculated as the ordering that minimized the sum of
probabilistic Kendall’s Tau (pKT) distances to all subject-wise orderings of
patients with the same subtype.
Second,
we tested the model ability to predict
the sCJD subtype of a patient as follows: for each subtype $$$i$$$, we obtained the likelihood $$$L_{i,j}=e^{-d(s_j,S_i)}$$$ from the pKT
distances $$$d(s_j,S_i)$$$ between the
subject-specific ordering of a patient ($$$s_j$$$) and the central ordering ($$$S_i$$$). The subtype with highest posterior probability $$$L_{i,j}\times p_i$$$ was assigned to
patient $$$j$$$, where $$$p_i$$$ is the frequency
of subtype $$$i$$$ in the sCJD
population.
The balanced accuracy of the subtype
prediction was assessed by leave-one-out cross-validation. Misclassifications
between MM1 and MV1, or between MM2C and MV2C, were considered correct
diagnoses, because they appear phenotypically indistinguishable.10 When the PRNP129 information (MM, MV or VV)
was used, three separate classification
problems were solved: MM1 vs MM2; MV1 vs MV2C vs MV2K; VV1 vs VV2.
RESULTS
Figure 1 shows that the two most frequent
sCJD subtypes (MM1 and VV2) had opposite orderings: initially, cortical regions
were abnormal in MM1, followed by the striatum, and cerebellum and thalamus
appeared at the end. Striatum was first in VV2 ordering, followed by thalamus
and cerebellum, and cortical regions were the last. Misclassifications between
these two subtypes were rare (only 3%, 10/301 MM1 and VV2 cases combined)
(Table 1).
As expected, MM1 and MV1 orderings were
almost identical (Figure 2a), as well as those of MM2C and MV2C (Figure 2b).
MM/MV2C and VV1 orderings were similar to that of MM1 (Figure 3a), with the
main differences being the striatum affected earlier and the insula involved
later in MM1. Most of the misclassified MM1 were assigned to MM/MV2C
(21%,43/208) and VV1 (6%,13/208) (Table 1).
MV2K was similar to VV2, but with a later
involvement of the cerebellum and an earlier occurrence of the frontal cortex
(Figure 3b). These two subtypes were difficult to distinguish. On the contrary,
MM/MV2C and VV1 were rarely confused with MV2K or VV2 (2.5%,2/80) and vice-versa
(9.6%,12/125) (Table 1).
The balanced accuracy was 57% when using
only MRI-derived data. The subtypes with highest percentages of correct
diagnosis were MM/MV2C (82%,42/51) and MM/MV1 (67%,165/248) (Table 1). The
balanced accuracy increased to 76% if the PRNP129
information was considered (MM: 64%, MV: 72%, and VV: 94%) (Table 2),
and >90% of patients with MM1, MV2C, VV1 and VV2 subtypes were correctly
identified.DISCUSSION & CONCLUSION
We
estimated the temporal cascades of DWI abnormalities in different sCJD subtypes
and showed that subtype diagnosis is feasible with MRI.
Two main
groups of ordering were identified: (i) MM/MV1, MM/MV2C and VV1 showed an
initial cortical involvement in the parietal region; (ii) VV2 and MV2K had an initial subcortical
(striatum and thalamus) involvement, in keeping with
recent neuropathological reports indicating that VV2 and MV2K share the
same strain.10,11 Similar MRI progression patterns were previously found for MM1 and VV2,12,13
but our study is the first to use them for classification purposes.
Our
procedure reliably discriminated the sCJD subtypes between these two main
groups, but lower classification accuracies were obtained within each group.
This limitation was partially overcome by including the PRNP129 information,
that allowed to distinguish MV2C from MV2K and VV1 from VV2 with accuracies
>90%, but not MM1 from MM2.Acknowledgements
RP, VV, EEB, SK and AB are supported by the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 666992. PG is supported by NIH R01 NS083687 and P01 AI106705, and Charles S. Britton Fund. JB and BSA are supported by CDC NU38CK000480.
DISCLAIMER: The findings and conclusions in this
report are those of the authors and do not necessarily represent the official
position of CDC.
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