Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy
© 2022. The Author(s)..
We evaluated whether whole-body dual-energy X-ray absorptiometry (DXA) measures of lean body mass can be used as biomarkers for disease progression and treatment effects in patients with Duchenne muscular dystrophy. This post hoc analysis utilized data from a randomized, 2-period study of domagrozumab versus placebo in 120 ambulatory boys with DMD. DXA measures of lean body mass were obtained from the whole body (excluding head), arms, legs and appendicular skeleton at baseline and every 16 weeks. Treatment effects on DXA measures for domagrozumab versus placebo were assessed at Week 49. At Week 49, domagrozumab statistically significantly increased lean body mass versus placebo in the appendicular skeleton (p = 0.050) and arms (p < 0.001). The relationship between lean body mass at Week 49 and functional endpoints at Week 97 was evaluated. Changes in lean body mass at Week 49 in all regions except arms were significantly correlated with percent change from baseline in 4-stair climb (4SC) at Week 97. DXA-derived percent lean mass at Week 49 also correlated with 4SC and North Star Ambulatory Assessment at Week 97. These data indicate that whole-body DXA measures can be used as biomarkers for treatment effects and disease progression in patients with DMD, and warrant further investigation.Trial registration: ClinicalTrials.gov, NCT02310763; registered 8 December 2014.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2022 |
|---|---|
| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:12 |
|---|---|
| Enthalten in: |
Scientific reports - 12(2022), 1 vom: 05. Nov., Seite 18762 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Sherlock, Sarah P [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Biomarkers |
|---|
| Anmerkungen: |
Date Completed 08.11.2022 Date Revised 17.08.2023 published: Electronic ClinicalTrials.gov: NCT02310763 Citation Status MEDLINE |
|---|
| doi: |
10.1038/s41598-022-23072-5 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM348490615 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM348490615 | ||
| 003 | DE-627 | ||
| 005 | 20231226205517.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2022 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1038/s41598-022-23072-5 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1161.xml |
| 035 | |a (DE-627)NLM348490615 | ||
| 035 | |a (NLM)36335191 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Sherlock, Sarah P |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy |
| 264 | 1 | |c 2022 | |
| 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 08.11.2022 | ||
| 500 | |a Date Revised 17.08.2023 | ||
| 500 | |a published: Electronic | ||
| 500 | |a ClinicalTrials.gov: NCT02310763 | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a © 2022. The Author(s). | ||
| 520 | |a We evaluated whether whole-body dual-energy X-ray absorptiometry (DXA) measures of lean body mass can be used as biomarkers for disease progression and treatment effects in patients with Duchenne muscular dystrophy. This post hoc analysis utilized data from a randomized, 2-period study of domagrozumab versus placebo in 120 ambulatory boys with DMD. DXA measures of lean body mass were obtained from the whole body (excluding head), arms, legs and appendicular skeleton at baseline and every 16 weeks. Treatment effects on DXA measures for domagrozumab versus placebo were assessed at Week 49. At Week 49, domagrozumab statistically significantly increased lean body mass versus placebo in the appendicular skeleton (p = 0.050) and arms (p < 0.001). The relationship between lean body mass at Week 49 and functional endpoints at Week 97 was evaluated. Changes in lean body mass at Week 49 in all regions except arms were significantly correlated with percent change from baseline in 4-stair climb (4SC) at Week 97. DXA-derived percent lean mass at Week 49 also correlated with 4SC and North Star Ambulatory Assessment at Week 97. These data indicate that whole-body DXA measures can be used as biomarkers for treatment effects and disease progression in patients with DMD, and warrant further investigation.Trial registration: ClinicalTrials.gov, NCT02310763; registered 8 December 2014 | ||
| 650 | 4 | |a Randomized Controlled Trial | |
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 7 | |a Biomarkers |2 NLM | |
| 700 | 1 | |a Palmer, Jeffrey |e verfasserin |4 aut | |
| 700 | 1 | |a Wagner, Kathryn R |e verfasserin |4 aut | |
| 700 | 1 | |a Abdel-Hamid, Hoda Z |e verfasserin |4 aut | |
| 700 | 1 | |a Tian, Cuixia |e verfasserin |4 aut | |
| 700 | 1 | |a Mah, Jean K |e verfasserin |4 aut | |
| 700 | 1 | |a Muntoni, Francesco |e verfasserin |4 aut | |
| 700 | 1 | |a Guglieri, Michela |e verfasserin |4 aut | |
| 700 | 1 | |a Butterfield, Russell J |e verfasserin |4 aut | |
| 700 | 1 | |a Charnas, Lawrence |e verfasserin |4 aut | |
| 700 | 1 | |a Marraffino, Shannon |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Scientific reports |d 2011 |g 12(2022), 1 vom: 05. Nov., Seite 18762 |w (DE-627)NLM215703936 |x 2045-2322 |7 nnns |
| 773 | 1 | 8 | |g volume:12 |g year:2022 |g number:1 |g day:05 |g month:11 |g pages:18762 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1038/s41598-022-23072-5 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 12 |j 2022 |e 1 |b 05 |c 11 |h 18762 | ||