Introduction/Aims: Glucocorticoid-induced osteoporosis with vertebral fractures is frequent in patients with Duchenne muscular dystrophy (DMD). In this study, we evaluated the effects of oral bisphosphonate (BP) therapy on the prevalence and severity of vertebral fractures by vertebral morphometry assessment. Methods: We reviewed the records and radiographs of patients with DMD who had been treated with oral BP (weekly alendronate) and had undergone routine spine radiographic monitoring for glucocorticoid-induced osteoporosis at Cincinnati Children's Hospital Medical Center between 2010 and 2017. Study outcomes were thoracic and lumbar vertebral fracture prevalence and severity, assessed by Genant semiquantitative grading of vertebral morphometry, for up to 5 years of treatment.Results: Fifty-two patients (median age, 11.8 years; 88% prepubertal; 31% nonambulatory) had been treated with long-term glucocorticoids (median duration, 4.7 years at BP start). Most patients (75%) had mild vertebral height loss or fractures

No abstract

Background and purpose The facial nerve is unique among cranial nerves in demonstrating normal enhancement of particular segments. The effect of varying T1 relaxivities of gadolinium-based contrast agents on facial nerve enhancement is unclear. In this study, we assess differences in normal facial nerve enhancement with two different gadolinium-based contrast agents, gadobutrol and gadopentetate dimeglumine. In addition, we evaluate differences in facial nerve enhancement with spin-echo (SE) T1 versus 3D inversion recovery prepared fast spoiled gradient-echo (FSPGR) post-contrast sequences. Methods A total of 140 facial nerves in 70 individuals were evaluated (70 with gadobutrol and 70 with gadopentetate dimeglumine) by two blinded reviewers. Differences in enhancement of facial nerve segments between the two agents were analyzed. Differences in enhancement between SE T1 and FSPGR imaging were also evaluated. Results There was no significant difference in facial nerve enhancement between gadobutrol and gadopentetate dimeglumine. Enhancement was commonly observed in the geniculate, tympanic and mastoid segments (98%-100%) with either contrast agent; enhancement was less common in the labyrinthine segments (9%-14%) and lateral canalicular segment (2%-5%). There was a smaller enhancing proportion of labyrinthine and tympanic segments with FSPGR as compared to SE T1 images with gadobutrol. Conclusion There is no significant difference in overall enhancement of the facial nerve between gadobutrol and gadopentetate dimeglumine. Mild enhancement of the lateral canalicular portion of the facial nerve may be a normal finding. With FSPGR sequence, there is lesser perceived enhancement of the labyrinthine and tympanic segments of the facial nerve with gadobutrol.

Regression analyses accounted for baseline characteristics, baseline medical care consumption, and follow-up duration in a 1:1 matched sample (283 for each group matched on age, disease progression stage, and medical events during baseline) and in a full sample analysis (314 treated and 648 control). Treated pts on average were 14.1 years old at initiation. Eteplirsen claims were observed for a median of 13.0 months. Median follow-up was 27.3 months. Pts without eteplirsen claims in later months may have continued to receive treatment via the manufacturer's patient support program. Significant treatment effects of eteplirsen were observed on yearly average rates of emergency room visits (3.05 vs 5.46, P = 0.031), pulmonary management visits (1.56 vs 2.44, P = 0.039), and tracheostomy (0.25 vs 1.13, P = 0.015). Consistent results were also found in the full sample analysis (2.61 vs 4.67, p = 0.010 for emergency room visits; 2.14 vs 3.00, P = 0.045 for pulmonary management visits; and 0.37 vs 2.17, P = 0.004 for tracheostomy). This study provides the first real-world evidence of eteplirsen treatment effects on key health outcomes in DMD pts.