ReviewInternal medicine (Tokyo, Japan)2022
Predicting the CTG Repeat Size from a Single Spirometry Test Performed at Any Time during the Disease Course of Myotonic Dystrophy Type 1.
Review in Internal medicine (Tokyo, Japan), 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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7 authors.
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Abstract
Objective In myotonic dystrophy type 1 (DM1), the CTG repeat size in the dystrophia myotonica protein kinase gene has been shown to correlate with disease severity and is a potential predictive marker for respiratory decline. However, genetic testing can be challenging in some clinical situations. We developed a simple formula for estimating the CTG repeat size using a single spirometry test in patients with DM1. Methods In this single-center retrospective study, we reviewed 50 consecutive patients with genetically confirmed DM1 whose follow-up visits were at our hospital. The patients were randomly assigned to training and test analysis subsets. By applying a linear mixed model to the longitudinal spirometry results of the training set, we calculated the fixed effects on the annual respiratory decline. Subsequently, we derived a prediction formula to calculate the repeat size that incorporated %vital capacity (%VC) and the patient's age at the time of the spirometry evaluation; the results were validated by the test set. Results A total of 157 spirometry tests were recorded. The fixed effects on the annual %VC decline were <img align="middle" src="./Graphics/abst-61_2281_1.jpg"/>=-0.90. The derived formula [repeat size=-16.8× (age+%VC/0.90) +2663] had a moderate predictive performance with a mean coefficient of determination <img align="middle" src="./Graphics/abst-61_2281_2.jpg"/> of 0.41. Conclusion The CTG repeat size in patients with DM1 can be potentially predicted using a simple formula based on a single spirometry test conducted at any time over the disease course. It can be useful as a supportive tool for advance care planning when genetic testing is not available.
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