% 2020 Journal of comparative effectiveness research
* Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophy: experience at a single, large care center.
- Aim: To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Methods: Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children's Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Results: Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. Conclusion: This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.
* Evolving Concepts in Ultrasonography of Pediatric Intussusceptions: Unequivocal Differentiation of Ileocolic, Obstructive and Transient Small-Bowel Intussusceptions.
- The main aim of the study was to elaborate on the various ultrasonographic parameters that can be used for definite differentiation of ileocolic, obstructive small-bowel and transient small-bowel intussusceptions (SBI). In this study, 90 children (63 boys and 27 girls) with suspected intussusception were evaluated. Of these, 54 cases were diagnosed as obstructive intussusceptions (33 ileocolic and 21 ileoileal), 15 cases were classified as transient SBIs and 21 cases were negative for intussusception. The mean fat core diameter was 1.45 ± 0.32 cm for ileocolic versus 0.37 ± 0.06 cm for obstructive SBI versus 0.29 ± 0.08 cm for transient SBI (p < 0.001). The mean lesion diameter was 3.23 ± 0.08 cm for ileocolic intussusceptions and 2.12 ± 0.038 cm for SBI (p < 0.001), and the ratio of inner fat core to outer wall thickness was greater than 1 for ileocolic intussusceptions and less than 1 for SBI. A statistically significant difference was found between segmental invagination of transient versus obstructive SBIs with mean values of 1.93 ± 0.39 cm and 3.17 ± 0.25 cm, respectively, and an "optimal" threshold at 2.5 cm.