Recombinant human insulin‐like growth factor‐1 therapy for 6 months improves growth but not motor function in boys with Duchenne muscular dystrophy
Introduction Recombinant human insulin‐like growth factor‐1 (rhIGF‐1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF‐1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD). Methods In this study...
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| Published in | Muscle & nerve Vol. 61; no. 5; pp. 623 - 631 |
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| Main Authors | , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Hoboken, USA
John Wiley & Sons, Inc
01.05.2020
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| Subjects | |
| Online Access | Get full text |
| ISSN | 0148-639X 1097-4598 1097-4598 |
| DOI | 10.1002/mus.26846 |
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| Abstract | Introduction
Recombinant human insulin‐like growth factor‐1 (rhIGF‐1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF‐1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).
Methods
In this study we compared prepubescent, ambulatory, glucocorticoid‐treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6‐month‐long, prospective, randomized, controlled trial of subcutaneous rhIGF‐1 therapy. The primary outcome was 6‐minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.
Results
Change in 6MWD was similar between groups (rhIGF‐1 vs controls [mean ± SD]: 3.4 ± 32.4 vs −5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6‐month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.
Discussion
In boys with DMD, 6 months of rhIGF‐1 therapy did not change motor function, but it improved linear growth. |
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| AbstractList | Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).
In this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.
Change in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD]: 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.
In boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth. Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).INTRODUCTIONRecombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).In this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.METHODSIn this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.Change in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD]: 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.RESULTSChange in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD]: 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.In boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth.DISCUSSIONIn boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth. Introduction Recombinant human insulin‐like growth factor‐1 (rhIGF‐1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF‐1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD). Methods In this study we compared prepubescent, ambulatory, glucocorticoid‐treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6‐month‐long, prospective, randomized, controlled trial of subcutaneous rhIGF‐1 therapy. The primary outcome was 6‐minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety. Results Change in 6MWD was similar between groups (rhIGF‐1 vs controls [mean ± SD]: 3.4 ± 32.4 vs −5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6‐month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects. Discussion In boys with DMD, 6 months of rhIGF‐1 therapy did not change motor function, but it improved linear growth. |
| Author | Wong, Brenda L. Taylor, Michael D. Horn, Paul S. Backeljauw, Philippe F. Collins, James J. Rutter, Meilan M. Sawnani, Hemant |
| Author_xml | – sequence: 1 givenname: Meilan M. surname: Rutter fullname: Rutter, Meilan M. email: meilan.rutter@cchmc.org organization: Cincinnati Children's Hospital Medical Center – sequence: 2 givenname: Brenda L. surname: Wong fullname: Wong, Brenda L. organization: University of Massachusetts Medical School – sequence: 3 givenname: James J. surname: Collins fullname: Collins, James J. organization: Mercy Clinic Pediatric Neurology – sequence: 4 givenname: Hemant surname: Sawnani fullname: Sawnani, Hemant organization: Cincinnati Children's Hospital Medical Center – sequence: 5 givenname: Michael D. surname: Taylor fullname: Taylor, Michael D. organization: The Heart Institute, Cincinnati Children's Hospital Medical Center – sequence: 6 givenname: Paul S. surname: Horn fullname: Horn, Paul S. organization: Cincinnati Children's Hospital Medical Center – sequence: 7 givenname: Philippe F. surname: Backeljauw fullname: Backeljauw, Philippe F. organization: Cincinnati Children's Hospital Medical Center |
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Recombinant human insulin‐like growth factor‐1 (rhIGF‐1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF‐1... Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1)... |
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| SubjectTerms | Duchenne muscular dystrophy glucocorticoid growth hormone IGF short stature steroid |
| Title | Recombinant human insulin‐like growth factor‐1 therapy for 6 months improves growth but not motor function in boys with Duchenne muscular dystrophy |
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