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2 REAL-WORLD STUDIES.
2 YEARS+ PROLONGED AMBULATION vs PREDNISONE.1,2
In 2 separate real-world studies, adolescents treated with deflazacort showed prolonged ambulation compared to those treated with prednisone.1,2
Median age at loss of ambulation1,2
![2015 Study](/static/e68688e2d46f432895f1db70348e53cf/65073/efficacy-real-world-data-image-2.png)
A natural history observational prospective study evaluating age at loss of independent ambulation (LoA) and side-effect profiles associated with different corticosteroid regimens in DMD. The study included 340 DMD participants, aged 2-28 years, who were enrolled in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use, confirmed by inability to walk 10 minutes unaided. Effects of prednisone or prednisolone (PRED)/deflazacort (DFZ), administration frequency, and dose were analyzed by time-varying Cox regression. Average dose of daily PRED administered while ambulatory (n=94) was 75% ± 17% of recommended, which was lower than daily DFZ (83% ± 15%, n=80).1
![2020 Study](/static/57db6f085d8e720bc5fdb6c1097bdd84/65073/efficacy-real-world-data-image-3.png)
A single-center, retrospective cohort analysis of 435 boys with DMD at Cincinnati Children’s Hospital Medical Center (CCHMC) from 2004 to March 2017. Outcomes assessed included: age at clinical events, including loss of ambulation (defined as functional mobility scale >4), and scoliosis; ambulatory, pulmonary, and cardiac function; and weight, lean body mass, and bone health. Average age at steroid initiation was 6.4 years for patients taking prednisone and 5.7 years for those taking deflazacort.2
![](/static/9ffb752fa7a98da202c0e92b92294c12/dd511/efficacy-real-world-data-image-4.png)
Real-world outcomes. Real-life impact.2
2020
Long-Term
Real-World Study
Scoliosis developed later in patients taking deflazacort than those taking prednisone.2
7.9% of deflazacort-initiated patients and 17.9% of prednisone-initiated patients developed scoliosis by the end of the study.2
![2020 Study](/static/57db6f085d8e720bc5fdb6c1097bdd84/65073/efficacy-real-world-data-image-3.png)
A single-center, retrospective cohort analysis of 435 boys with DMD at Cincinnati Children’s Hospital Medical Center (CCHMC) from 2004 to March 2017. Outcomes assessed included: age at clinical events, including loss of ambulation (defined as functional mobility scale >4), and scoliosis; ambulatory, pulmonary, and cardiac function; and weight, lean body mass, and bone health. Average age at steroid initiation was 6.4 years for patients taking prednisone and 5.7 years for those taking deflazacort.2
![](/static/eb0465d448a593b07e201e94770891b5/0c3ad/efficacy-real-world-data-image-6.png)
Daily deflazacort delayed loss of ambulation longer vs daily, weekend, and intermittent prednisone1
Age at Loss of Ambulation with
deflazacort vs prednisone1
![2015 Study](/static/e68688e2d46f432895f1db70348e53cf/65073/efficacy-real-world-data-image-2.png)
A natural history observational prospective study evaluating age at loss of independent ambulation (LoA) and side-effect profiles associated with different corticosteroid regimens in DMD. The study included 340 DMD participants, aged 2-28 years, who were enrolled in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study (CINRG-DNHS). LoA was defined as continuous wheelchair use, confirmed by inability to walk 10 minutes unaided. Effects of prednisone or prednisolone (PRED)/deflazacort (DFZ), administration frequency, and dose were analyzed by time-varying Cox regression. Average dose of daily PRED administered while ambulatory (n=94) was 75% ± 17% of recommended, which was lower than daily DFZ (83% ± 15%, n=80).1
Study Limitations
Comparisons between deflazacort and prednisone are not included in the Prescribing Information for deflazacort because prednisone is not an approved treatment for DMD.