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

Age at Loss of Ambulation1,2

2015 Study

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

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

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

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

Daily deflazacort delayed loss of ambulation longer vs daily, weekend, and intermittent prednisone1

Age at Loss of Ambulation with deflazacort vs prednisone1

2015 Study

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.

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Long-Term Data