Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center

Marden, Jessica R.; Freimark, Jonathan; Yao, Zhiwen; Signorovitch, James; Tian, Chengming; Wong, Brenda

doi:10.2217/cer-2019-0170

Cited by 36 publications

(27 citation statements)

References 43 publications

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“…Therefore, a plausible explanation for our findings is that the higher weight at 5 years in the deflazacort group might have been biased by the decision to start deflazacort in children who were already overweight. However, as previously reported [ 27 , 28 ], the prednisolone subgroup showed a higher yearly weight gain than the deflazacort subgroup (p < 0.01). Unlike previous studies [ 29 ], we did not find a significant difference in the yearly weight gain rate between the daily or intermittent subgroups.…”

Section: Discussionsupporting

confidence: 84%

“…Irrespective of the GC type, daily regimes showed the highest yearly stunting of height. As shown in previous studies [ 18 , 27 , 28 , 31 ] participants on daily deflazacort had the greatest stunting of height, showing a loss of 0.25 (95% CI 0.21, 0.3) SD every year compared to the UK 90. However, unlike weight, GC start age was not significantly associated with yearly height change.…”

Section: Discussionsupporting

confidence: 59%

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Growth pattern trajectories in boys with Duchenne muscular dystrophy

Stimpson

Raquq

Chesshyre

et al. 2022

Orphanet J Rare Dis

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Objectives The objective of this study is to analyse retrospective, observational, longitudinal growth (weight, height and BMI) data in ambulatory boys aged 5–12 years with Duchenne muscular dystrophy (DMD). Background We considered glucocorticoids (GC) use, dystrophin isoforms and amenability to exon 8, 44, 45, 51 and 53 skipping drug subgroups, and the impact of growth on loss of ambulation. We analysed 598 boys, with 2604 observations. This analysis considered patients from the UK NorthStar database (2003–2020) on one of five regimes: “GC naïve”, “deflazacort daily” (DD), “deflazacort intermittent” (DI), “prednisolone daily” (PD) and “prednisolone intermittent” (PI). A random slope model was used to model the weight, height and BMI SD scores (using the UK90). Results The daily regime subgroups had significant yearly height stunting compared to the GC naïve subgroup. Notably, the average height change for the DD subgroup was 0.25 SD (95% CI − 0.30, − 0.21) less than reference values. Those with affected expression of Dp427, Dp140 and Dp71 isoforms were 0.77 (95% CI 0.3, 1.24) and 0.82 (95% CI 1.28, 0.36) SD shorter than those with Dp427 and/or Dp140 expression affected respectively. Increased weight was not associated with earlier loss of ambulation, but taller boys still ambulant between the age of 10 and 11 years were more at risk of losing ambulation. Conclusion 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.

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Section: Discussionsupporting

confidence: 84%

Section: Discussionsupporting

confidence: 59%

Growth pattern trajectories in boys with Duchenne muscular dystrophy

Stimpson

Raquq

Chesshyre

et al. 2022

Orphanet J Rare Dis

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“…Height was considerably lower than expected for age in patients with DMD, with half having height‐for‐age Z‐scores below −2.0. Approximately 80% of patients were prescribed daily deflazacort 19 . Nineteen percent of our study sample had received growth hormone, and 14% had received testosterone therapies (Table 1).…”

Section: Resultsmentioning

confidence: 99%

Age‐related changes in appendicular lean mass in males with Duchenne muscular dystrophy: A retrospective review

et al. 2020

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Background Appendicular lean mass (ALM) trajectory in males with Duchenne muscular dystrophy (DMD) has potential applicability for treatment and research and has not been characterized. Methods This chart review included longitudinal data on 499 males with DMD receiving glucocorticoids and 693 controls, ages 5 to 22.9 y. ALM (kg) was measured by dual energy x‐ray absorptiometry (DXA). Appendicular lean mass index (ALMI, kg/m2) was calculated for height adjustment. Reference centiles were generated using data from healthy controls, and ALM and ALMI Z‐scores were calculated for patients with DMD. Generalized linear models were used to estimate median Z‐scores by age and functional mobility status (FMS) score. ALM velocity by age was modeled using superimposition, translation and rotation (SITAR). Results Compared to controls, males with DMD had lower ALM from an early age. ALMI Z‐scores dropped below 0 at age 8 y or FMS of 2, and below −2.0 at age 13 y or FMS of 3 (P < .05). Age at peak ALM velocity was similar in both groups, but the magnitude was higher in controls (3.5 vs. 0.7 kg/y, P < .0001). Patients with DMD had a transient loss of ALM around age 12 y, an increase at age 14 y, then a further decline at age 16 y, remaining low thereafter. Conclusions Males with DMD have progressive decline in lean mass with age and worsening functional mobility. DXA measurement of ALM may be useful for monitoring lean mass status in patients with DMD, providing valuable information for individual treatment plans and research endeavors.

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“…In the absence of head-to-head randomized trials between these treatments, the authors designed their study to indirectly compare the key safety and efficacy outcomes using a matching-adjusted indirect comparison [4]. In addition, both the articles of Mercuri et al and Marden et al [5,8], focus on Duchenne muscular dystrophy (DMD); Mecuri et al compared the results from the STRIDE Registry and CINRG DMD Natural History Study to assess the safety and effectiveness of ataluren treatment, while Marden et al looked at the experience of the Cincinnati Children's Hospital Medical Center to assess outcomes among patients with DMD receiving deflazacort or prednisone in real-world practice.…”

Section: Content Highlights Of 2020mentioning

confidence: 99%