Eran Lavi , Amitay Cohen , Abdulsalam Abu Libdeh , Reuven Tsabari , David Zangen , Talya Dor
{"title":"生长激素治疗Duchenne肌营养不良和糖皮质激素诱导的矮小儿童","authors":"Eran Lavi , Amitay Cohen , Abdulsalam Abu Libdeh , Reuven Tsabari , David Zangen , Talya Dor","doi":"10.1016/j.ghir.2023.101558","DOIUrl":null,"url":null,"abstract":"<div><h3>Objective</h3><p><span><span>To evaluate the outcome of recombinant human Growth Hormone (rhGH) therapy </span>in patients<span> with Duchene Muscular Dystrophy (DMD) and </span></span>glucocorticoid<span> treatment with compromised growth.</span></p></div><div><h3>Design</h3><p><span>Four DMD patients on Deflzacort 0.6–0.85 mg/kg/day or prednisolone 0.625 mg/kg/day recieved rhGH (0.24 mg/kg/week) for 6–18 months. Primary outcomes were Growth velocity and Height for age </span><em>Z</em>-scores (Height SD).</p></div><div><h3>Results</h3><p>Growth velocity increased from 0 to 3.25 cm/year prior to GH therapy to 3.3–7.8 cm/year over a period of 6–18 months. The typical Height SD decline in DMD was reversed in two patients and blunted in one. No adverse events or deterioration in cardiac or respiratory parameters were associated with the rhGH treatment.</p></div><div><h3>Conclusions</h3><p>rhGH appears to be safe and efficient in promoting growth of patients with glucocorticoid induced growth failure in DMD.</p></div>","PeriodicalId":12803,"journal":{"name":"Growth Hormone & Igf Research","volume":null,"pages":null},"PeriodicalIF":1.6000,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature\",\"authors\":\"Eran Lavi , Amitay Cohen , Abdulsalam Abu Libdeh , Reuven Tsabari , David Zangen , Talya Dor\",\"doi\":\"10.1016/j.ghir.2023.101558\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Objective</h3><p><span><span>To evaluate the outcome of recombinant human Growth Hormone (rhGH) therapy </span>in patients<span> with Duchene Muscular Dystrophy (DMD) and </span></span>glucocorticoid<span> treatment with compromised growth.</span></p></div><div><h3>Design</h3><p><span>Four DMD patients on Deflzacort 0.6–0.85 mg/kg/day or prednisolone 0.625 mg/kg/day recieved rhGH (0.24 mg/kg/week) for 6–18 months. Primary outcomes were Growth velocity and Height for age </span><em>Z</em>-scores (Height SD).</p></div><div><h3>Results</h3><p>Growth velocity increased from 0 to 3.25 cm/year prior to GH therapy to 3.3–7.8 cm/year over a period of 6–18 months. The typical Height SD decline in DMD was reversed in two patients and blunted in one. No adverse events or deterioration in cardiac or respiratory parameters were associated with the rhGH treatment.</p></div><div><h3>Conclusions</h3><p>rhGH appears to be safe and efficient in promoting growth of patients with glucocorticoid induced growth failure in DMD.</p></div>\",\"PeriodicalId\":12803,\"journal\":{\"name\":\"Growth Hormone & Igf Research\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2023-10-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Growth Hormone & Igf Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1096637423000369\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"CELL BIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Growth Hormone & Igf Research","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1096637423000369","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"CELL BIOLOGY","Score":null,"Total":0}
Growth hormone therapy for children with Duchenne muscular dystrophy and glucocorticoid induced short stature
Objective
To evaluate the outcome of recombinant human Growth Hormone (rhGH) therapy in patients with Duchene Muscular Dystrophy (DMD) and glucocorticoid treatment with compromised growth.
Design
Four DMD patients on Deflzacort 0.6–0.85 mg/kg/day or prednisolone 0.625 mg/kg/day recieved rhGH (0.24 mg/kg/week) for 6–18 months. Primary outcomes were Growth velocity and Height for age Z-scores (Height SD).
Results
Growth velocity increased from 0 to 3.25 cm/year prior to GH therapy to 3.3–7.8 cm/year over a period of 6–18 months. The typical Height SD decline in DMD was reversed in two patients and blunted in one. No adverse events or deterioration in cardiac or respiratory parameters were associated with the rhGH treatment.
Conclusions
rhGH appears to be safe and efficient in promoting growth of patients with glucocorticoid induced growth failure in DMD.
期刊介绍：
Growth Hormone & IGF Research is a forum for research on the regulation of growth and metabolism in humans, animals, tissues and cells. It publishes articles on all aspects of growth-promoting and growth-inhibiting hormones and factors, with particular emphasis on insulin-like growth factors (IGFs) and growth hormone. This reflects the increasing importance of growth hormone and IGFs in clinical medicine and in the treatment of diseases.
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