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生长激素对伴有生长激素缺乏的日本唐氏综合征身材矮小儿童的治疗效果及安全性

Therapeutic Efficacy and Safety of GH in Japanese Children with Down Syndrome Short Stature Accompanied by GH Deficiency.

作者信息

Meguri Kyoko, Inoue Masaru, Narahara Koji, Sato Takahiro, Takata Ami, Ohki Nobuhiko, Ozono Keiichi

机构信息

Department of Pediatrics, Okayama Red Cross General Hospital, Okayama, Japan.

出版信息

Clin Pediatr Endocrinol. 2013 Oct;22(4):65-72. doi: 10.1292/cpe.22.65. Epub 2013 Oct 26.

DOI:10.1292/cpe.22.65
PMID:24170963
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3809732/
Abstract

In this study, we investigated the effects of GH treatment in children with Down syndrome who had been diagnosed with GH deficiency (GHD). A total of 20 subjects were investigated in this study. Fourteen Down syndrome children (5 boys and 9 girls) with short stature due to GHD were treated with GH at Okayama Red Cross General Hospital, and 6 Down syndrome children (4 boys and 2 girls) with short stature due to GHD were registered in the Pfizer International Growth Database (KIGS). Height SD score (SDS) increased throughout the three-year GH treatment period. The overall mean height SDS increased from -3.5 at baseline to -2.5 after 3 yr of treatment. The mean change in height SDS during these 3 yr was 1.1. In addition, height assessment of SD score based on Down syndrome-specific growth data in the Japanese population revealed that the height SDS (Down syndrome) also increased across the 3-yr GH treatment period. The mean change in height SDS (Down syndrome) during these three years was 1.3. GH therapy was effective for Down syndrome short stature accompanied by GHD, and no new safety concerns were found in this study.

摘要

在本研究中,我们调查了生长激素(GH)治疗对已诊断为生长激素缺乏症(GHD)的唐氏综合征儿童的影响。本研究共纳入20名受试者。冈山红十字综合医院对14名因GHD导致身材矮小的唐氏综合征儿童(5名男孩和9名女孩)进行了GH治疗,6名因GHD导致身材矮小的唐氏综合征儿童(4名男孩和2名女孩)被纳入辉瑞国际生长数据库(KIGS)。在为期三年的GH治疗期间,身高标准差评分(SDS)持续增加。治疗3年后,总体平均身高SDS从基线时的-3.5增加到-2.5。这3年期间身高SDS的平均变化为1.1。此外,根据日本人群中唐氏综合征特异性生长数据进行的身高标准差评分评估显示,在为期3年的GH治疗期间,身高SDS(唐氏综合征)也有所增加。这三年期间身高SDS(唐氏综合征)的平均变化为1.3。GH治疗对伴有GHD的唐氏综合征身材矮小有效,且本研究未发现新的安全问题。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aaf8/3809732/3b54255482fe/cpe-22-065-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aaf8/3809732/9182b895f334/cpe-22-065-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aaf8/3809732/3b54255482fe/cpe-22-065-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aaf8/3809732/9182b895f334/cpe-22-065-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/aaf8/3809732/3b54255482fe/cpe-22-065-g002.jpg

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