The final multivariate risk model was developed through a binary
logistic regression analysis of detailed phenotypic data from Cohort 1.
(B) The predictive efficacy of the TACScore. The X-axis shows
the spectrum of TACScore and Y-axis shows the predicted TACS frequency and the
percentage of TACS patients in all CS patients with each calculated score in
Cohort 1 and Cohort 2. The TACScore presented excellent predictive efficacy by
comparing the predicted TACS risk to the real TACS frequency. The cutoff point
was selected as 3 to achieve the highest accuracy. (C) ROC Curve for the
TACScore in Cohort 1 and Cohort 2. AUCs were 0.9
(P=1.6×10−15; 95% CI,
0.9–1.0) for the discovery cohort (Cohort 1) and 0.8
(P=1.5×10−4; 95% CI,
0.7–0.9) for the validation cohort (Cohort 2). (D) A proposed
guideline for predicting and evaluating TACS. The risk of TACS
evaluated by TACScore is suggested to perform prior to genetic testing. After
the detection of TACS, a systemic evaluation, with early interventions and
genetic consultation were recommended.
Abbreviation: CS, congenital scoliosis; TACS,
TBX6-associated CS; ES, exome sequencing; CMA, chromosomal
microarray analysis; GS, genome sequencing.
a
TBX6 compound variant contains a 16p11.2
deletion/TBX6 loss-of-function variant in the compound
heterozygous configuration with the risk haplotype providing a hypomorphic
variant.
b VUS, variants with unknown significance.
c Evidence from large-scale case-control studies, pedigree
analysis, and functional studies are needed.