PMID- 32108355
OWN - NLM
STAT- MEDLINE
DCOM- 20200706
LR  - 20200706
IS  - 1097-4598 (Electronic)
IS  - 0148-639X (Linking)
VI  - 61
IP  - 5
DP  - 2020 May
TI  - Recombinant human insulin-like growth factor-1 therapy for 6 months improves 
      growth but not motor function in boys with Duchenne muscular dystrophy.
PG  - 623-631
LID - 10.1002/mus.26846 [doi]
AB  - 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 (DeltaHtSDS), 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 DeltaHtSDS: 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.
CI  - (c) 2020 Wiley Periodicals, Inc.
FAU - Rutter, Meilan M
AU  - Rutter MM
AD  - Division of Endocrinology, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio, USA.
FAU - Wong, Brenda L
AU  - Wong BL
AD  - Department of Pediatrics and Neurology, University of Massachusetts Medical 
      School, Worcester, Massachusetts, USA.
FAU - Collins, James J
AU  - Collins JJ
AD  - Mercy Clinic Pediatric Neurology, Springfield, Missouri, USA.
FAU - Sawnani, Hemant
AU  - Sawnani H
AD  - Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio, USA.
FAU - Taylor, Michael D
AU  - Taylor MD
AD  - The Heart Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, 
      Ohio, USA.
FAU - Horn, Paul S
AU  - Horn PS
AD  - Division of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio, USA.
FAU - Backeljauw, Philippe F
AU  - Backeljauw PF
AD  - Division of Endocrinology, Cincinnati Children's Hospital Medical Center, 
      Cincinnati, Ohio, USA.
LA  - eng
PT  - Journal Article
PT  - Randomized Controlled Trial
PT  - Research Support, Non-U.S. Gov't
DEP - 20200316
PL  - United States
TA  - Muscle Nerve
JT  - Muscle & nerve
JID - 7803146
RN  - 0 (Blood Glucose)
RN  - 0 (Glucocorticoids)
RN  - 0 (Growth Substances)
RN  - 0 (Recombinant Proteins)
RN  - 67763-96-6 (Insulin-Like Growth Factor I)
RN  - 7GR9I2683O (mecasermin)
SB  - IM
MH  - Absorptiometry, Photon
MH  - Blood Glucose/metabolism
MH  - Blood Glucose Self-Monitoring
MH  - Body Composition
MH  - *Body Height
MH  - Child
MH  - Drug Therapy, Combination
MH  - Glucocorticoids/therapeutic use
MH  - Growth Substances/*therapeutic use
MH  - Humans
MH  - *Insulin Resistance
MH  - Insulin-Like Growth Factor I/*therapeutic use
MH  - Magnetic Resonance Imaging
MH  - Male
MH  - *Muscle Strength
MH  - Muscular Dystrophy, Duchenne/*drug therapy/metabolism/physiopathology
MH  - Quality of Life
MH  - Recombinant Proteins/*therapeutic use
MH  - Treatment Outcome
MH  - Walk Test
OTO - NOTNLM
OT  - Duchenne muscular dystrophy
OT  - IGF
OT  - glucocorticoid
OT  - growth hormone
OT  - short stature
OT  - steroid
EDAT- 2020/02/29 06:00
MHDA- 2020/07/07 06:00
CRDT- 2020/02/29 06:00
PHST- 2019/07/29 00:00 [received]
PHST- 2020/01/15 00:00 [revised]
PHST- 2020/02/24 00:00 [accepted]
PHST- 2020/02/29 06:00 [pubmed]
PHST- 2020/07/07 06:00 [medline]
PHST- 2020/02/29 06:00 [entrez]
AID - 10.1002/mus.26846 [doi]
PST - ppublish
SO  - Muscle Nerve. 2020 May;61(5):623-631. doi: 10.1002/mus.26846. Epub 2020 Mar 16.