Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status
Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2023 |
|---|---|
| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:12 |
|---|---|
| Enthalten in: |
Journal of comparative effectiveness research - 12(2023), 4 vom: 01. Apr., Seite e220190 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
McDonald, Craig M [VerfasserIn] |
|---|
| Links: |
|---|
| Anmerkungen: |
Date Completed 03.04.2023 Date Revised 12.08.2023 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.57264/cer-2022-0190 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM352592494 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM352592494 | ||
| 003 | DE-627 | ||
| 005 | 20231226054059.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2023 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.57264/cer-2022-0190 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1175.xml |
| 035 | |a (DE-627)NLM352592494 | ||
| 035 | |a (NLM)36749302 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a McDonald, Craig M |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status |
| 264 | 1 | |c 2023 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 03.04.2023 | ||
| 500 | |a Date Revised 12.08.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score. Results: Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years). Conclusion: Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients | ||
| 650 | 4 | |a Clinical Trial, Phase II | |
| 650 | 4 | |a Clinical Trial, Phase III | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a Duchenne muscular dystrophy | |
| 650 | 4 | |a clinical outcomes | |
| 650 | 4 | |a deflazacort | |
| 650 | 4 | |a disease milestones | |
| 650 | 4 | |a efficacy | |
| 650 | 4 | |a prednisone/prednisolone | |
| 650 | 7 | |a Adrenal Cortex Hormones |2 NLM | |
| 650 | 7 | |a Prednisone |2 NLM | |
| 650 | 7 | |a VB0R961HZT |2 NLM | |
| 700 | 1 | |a Marden, Jessica R |e verfasserin |4 aut | |
| 700 | 1 | |a Shieh, Perry B |e verfasserin |4 aut | |
| 700 | 1 | |a Wong, Brenda L |e verfasserin |4 aut | |
| 700 | 1 | |a Lane, Henry |e verfasserin |4 aut | |
| 700 | 1 | |a Zhang, Adina |e verfasserin |4 aut | |
| 700 | 1 | |a Nguyen, Ha |e verfasserin |4 aut | |
| 700 | 1 | |a Frean, Molly |e verfasserin |4 aut | |
| 700 | 1 | |a Trifillis, Panayiota |e verfasserin |4 aut | |
| 700 | 1 | |a Koladicz, Karyn |e verfasserin |4 aut | |
| 700 | 1 | |a Signorovitch, James |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Journal of comparative effectiveness research |d 2012 |g 12(2023), 4 vom: 01. Apr., Seite e220190 |w (DE-627)NLM218708033 |x 2042-6313 |7 nnns |
| 773 | 1 | 8 | |g volume:12 |g year:2023 |g number:4 |g day:01 |g month:04 |g pages:e220190 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.57264/cer-2022-0190 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 12 |j 2023 |e 4 |b 01 |c 04 |h e220190 | ||