Three years of growth hormone treatment in young adults with Prader-Willi syndrome : sustained positive effects on body composition
BACKGROUND: In children with Prader-Willi syndrome (PWS), the benefits of growth hormone treatment are well established. Several one-year studies have shown that growth hormone is also beneficial for adults with PWS, improving body composition. However, little is known about the longer-term effects. This study investigated the effects on body composition in adult patients with PWS during 3 years of growth hormone therapy in a dose of 0.33 mg/m2/day.
METHODS: Open-label, prospective study in 43 young adults with PWS with a median (IQR) age of 19.0 (17.5 to 20.7) years. Fat mass percentage SDS and lean body mass SDS were measured annually by DXA.
RESULTS: Estimated mean (95% CI) fat mass percentage SDS decreased during the three-year study from 2.1 (1.9 to 2.3) SDS at start to 1.9 (1.8 to 2.1) SDS, p = 0.012, while lean body mass SDS remained stable at - 2.1 (- 2.4 to - 1.8) SDS at start to - 1.9 (- 2.3 to - 1.6) after 3 years, p = 0.15. Fasting glucose and insulin remained similar during the three-year study, glucose being 4.6 (4.4 to 4.8) mmol/l at start and 4.6 (4.5 to 4.7) mmol/l after 3 years of growth hormone, p = 0.93 and insulin being 59.5 (42.2 to 81.5) pmol/l and 55.0 (42.4 to 69.2) pmol/l, resp., p = 0.54. There were no growth hormone-related adverse events during the study.
CONCLUSIONS: Three years of growth hormone treatment in young adults with PWS maintains the positive effects on body composition attained during childhood. Thus, adults with PWS benefit from longer-term growth hormone treatment.
TRIAL REGISTRATION: EudraCT, EudraCT number 2011-001313-14. Registered 17 October 2012.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2020 |
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| Erschienen: |
2020 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:15 |
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| Enthalten in: |
Orphanet journal of rare diseases - 15(2020), 1 vom: 24. Juni, Seite 163 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Damen, Layla [VerfasserIn] |
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| Links: |
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| Themen: |
12629-01-5 |
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| Anmerkungen: |
Date Completed 18.06.2021 Date Revised 18.06.2021 published: Electronic Citation Status MEDLINE |
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| doi: |
10.1186/s13023-020-01440-6 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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NLM311591671 |
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| 245 | 1 | 0 | |a Three years of growth hormone treatment in young adults with Prader-Willi syndrome |b sustained positive effects on body composition |
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| 500 | |a published: Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a BACKGROUND: In children with Prader-Willi syndrome (PWS), the benefits of growth hormone treatment are well established. Several one-year studies have shown that growth hormone is also beneficial for adults with PWS, improving body composition. However, little is known about the longer-term effects. This study investigated the effects on body composition in adult patients with PWS during 3 years of growth hormone therapy in a dose of 0.33 mg/m2/day | ||
| 520 | |a METHODS: Open-label, prospective study in 43 young adults with PWS with a median (IQR) age of 19.0 (17.5 to 20.7) years. Fat mass percentage SDS and lean body mass SDS were measured annually by DXA | ||
| 520 | |a RESULTS: Estimated mean (95% CI) fat mass percentage SDS decreased during the three-year study from 2.1 (1.9 to 2.3) SDS at start to 1.9 (1.8 to 2.1) SDS, p = 0.012, while lean body mass SDS remained stable at - 2.1 (- 2.4 to - 1.8) SDS at start to - 1.9 (- 2.3 to - 1.6) after 3 years, p = 0.15. Fasting glucose and insulin remained similar during the three-year study, glucose being 4.6 (4.4 to 4.8) mmol/l at start and 4.6 (4.5 to 4.7) mmol/l after 3 years of growth hormone, p = 0.93 and insulin being 59.5 (42.2 to 81.5) pmol/l and 55.0 (42.4 to 69.2) pmol/l, resp., p = 0.54. There were no growth hormone-related adverse events during the study | ||
| 520 | |a CONCLUSIONS: Three years of growth hormone treatment in young adults with PWS maintains the positive effects on body composition attained during childhood. Thus, adults with PWS benefit from longer-term growth hormone treatment | ||
| 520 | |a TRIAL REGISTRATION: EudraCT, EudraCT number 2011-001313-14. Registered 17 October 2012 | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a Adults | |
| 650 | 4 | |a Body composition | |
| 650 | 4 | |a Growth hormone | |
| 650 | 4 | |a Prader Willi syndrome | |
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| 650 | 7 | |a Growth Hormone |2 NLM | |
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| 700 | 1 | |a Donze, Stephany H |e verfasserin |4 aut | |
| 700 | 1 | |a Kuppens, Renske J |e verfasserin |4 aut | |
| 700 | 1 | |a Bakker, Nienke E |e verfasserin |4 aut | |
| 700 | 1 | |a de Graaff, Laura C G |e verfasserin |4 aut | |
| 700 | 1 | |a van der Velden, Janielle A E M |e verfasserin |4 aut | |
| 700 | 1 | |a Hokken-Koelega, Anita C S |e verfasserin |4 aut | |
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