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

IF 1.6 4区医学 Q4 CELL BIOLOGY

Growth Hormone & Igf Research Pub Date : 2023-10-01 DOI:10.1016/j.ghir.2023.101558

Eran Lavi , Amitay Cohen , Abdulsalam Abu Libdeh , Reuven Tsabari , David Zangen , Talya Dor

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引用次数: 0

摘要

目的评价重组人生长激素（rhGH）治疗杜氏肌营养不良（DMD）和糖皮质激素治疗生长障碍的疗效。设计4名DMD患者接受0.6–0.85 mg/kg/天的替扎卡或0.625 mg/kg/天泼尼松治疗，接受rhGH（0.24 mg/kg/周）治疗6–18个月。主要结果是生长速度和身高Z评分（Height SD）。结果生长速度从生长激素治疗前的0至3.25 cm/年增加到6至18个月的3.3至7.8 cm/年。DMD的典型身高SD下降在两名患者中逆转，在一名患者中减弱。rhGH治疗无不良事件或心脏或呼吸参数恶化。结论srhGH对糖皮质激素诱导的DMD生长衰竭患者的生长具有安全有效的促进作用。

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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.

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来源期刊

Growth Hormone & Igf Research 医学-内分泌学与代谢

CiteScore

3.30

自引率

0.00%

发文量

审稿时长

57 days

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.