Daily use of deflazacort (DD) may lead to greater stunting of height in patients with Duchenne muscular dystrophy (DMD) than those taking prednisolone or those naïve to glucocorticoids (GCs), according to results of a new study published in the Orphanet Journal of Rare Diseases.

Patients who received DD exhibited yearly growth stunting of -0.25 standard deviation (SD) scores (95% CI, -0.30-0.21) compared to the general pediatric population using the British 1990 growth reference. Daily use of prednisolone (PD) also caused significant stunting of height. Both DD and PD showed significantly greater stunting compared to patients naïve to GCs (P <.01 for both).

Patients receiving intermittent deflazacort and prednisolone did not show as large of stunting as those receiving daily dosing regimes. The age of GC initiation was not significantly associated with height trajectories over time.


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The authors also found that patients with mutations affecting the expression of dystrophin Dp71 were shorter at all time points compared to those with more proximal pathogenic variants. Participants who were missing Dp71, Dp140, and Dp427 were significantly shorter (P <.01) than patients missing only Dp 427 or both dp427 and Dp140 by 0.82 SD and 0.77 SD at all age points.

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It was also observed that older boys between the ages of 10 and 11 years had a higher risk of loss of ambulation depending on their height. Boys had a 6% increase in the chance of losing ambulation for every 0.25 SD taller they were. Height did not affect the risk of loss of ambulation in younger age groups, however.

Patients receiving prednisolone had a higher yearly weight gain compared to patients receiving deflazacort. No statistical difference was found between intermittent and daily regimes between the 2 GCs. The age that patients initiated GC use was found to be related to weight gain with those commencing treatment at a later age having a higher rate of weight gain.

“These findings may provide further guidance to clinicians when counselling and discussing GCs commencement with patients and their carers and may represent a benchmark set of data to evaluate the effects of new generations of GC with differential mechanisms of action designed to limit mineralocorticoid-related side effects,” the authors concluded.

A total of 598 patients with DMD from the NorthStar database (between 2003-2020) were included in the study and accounted for 2604 observations. The mean patient follow-up duration was 2.5 years with a median of 4 visits during the time period. Of the patients, 203 had experienced the loss of ambulation.

Reference

Stimpson G, Raquq S, Chesshyre M, et al. Growth pattern trajectories in boys with Duchenne muscular dystrophy. Orphanet J Rare Dis. 2022;17(1):20. doi:10.1186/s13023-021-02158-9