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慢性肾脏病儿童重组人生长激素治疗使用模式和生长反应。

Patterns of recombinant growth hormone therapy use and growth responses among children with chronic kidney disease.

机构信息

Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, 615 N. Wolfe Street Room E7642, Baltimore, MD, 21205, USA.

Department of Pediatrics, Division of Nephrology, Albert Einstein College of Medicine, New York, NY, USA.

出版信息

Pediatr Nephrol. 2021 Dec;36(12):3905-3913. doi: 10.1007/s00467-021-05122-8. Epub 2021 Jun 11.

Abstract

BACKGROUND

Recombinant growth hormone (rGH) is an efficacious therapy for growth failure in children with chronic kidney disease (CKD). We described rGH use and estimated its relationship with growth and kidney function in the Chronic Kidney Disease in Children (CKiD) cohort.

METHODS

Participants included those with growth failure, prevalent rGH users, and rGH initiators who did not meet growth failure criteria. Among those with growth failure, height z scores and GFR were compared between rGH initiators and non-initiators across 42 months. Inverse probability weights accounted for differences in baseline variables in weighted linear regressions.

RESULTS

Among 148 children with growth failure and no previous rGH therapy, 42 (28%) initiated rGH therapy. Of the initiators, average age was 8.9 years, height z score was 2.50 standard deviations (SDs) (0.6 percentile), and GFR was 44 ml/min/1.73m. They were compared to 106 children with growth failure who never initiated therapy (8.8 years, -2.33 SDs, and 51 ml/min/1.73m). At 30 and 42 months after rGH, height increased +0.26 (95%CI: -0.11, +0.62) and +0.35 (95%CI: -0.17, +0.87) SDs, respectively, relative to those who did not initiate rGH. rGH was not associated with GFR.

CONCLUSIONS

Participants with growth failure receiving rGH experienced significant growth, although this was attenuated relative to RCTs, and were more likely to have higher household income and lower GFR. A substantial number of participants, predominantly boys, without diagnosed growth failure received rGH and had the highest achieved height relative to mid-parental height. Since rGH was not associated with accelerated GFR decline, increasing rGH use in this population is warranted.

摘要

背景

重组人生长激素(rGH)是治疗慢性肾脏病(CKD)儿童生长障碍的有效疗法。我们描述了 rGH 的使用情况,并在慢性肾脏病儿童(CKiD)队列中评估了其与生长和肾功能的关系。

方法

纳入存在生长障碍的患者、rGH 现有使用者和未达到生长障碍标准的 rGH 起始使用者。在存在生长障碍的患者中,比较 rGH 起始使用者和未起始使用者在 42 个月内的身高 z 评分和 GFR。在加权线性回归中,逆概率权重考虑了基线变量的差异。

结果

在 148 名无 rGH 治疗史且存在生长障碍的儿童中,42 名(28%)起始 rGH 治疗。起始使用者的平均年龄为 8.9 岁,身高 z 评分 2.50 标准差(SD)(0.6%位),GFR 为 44ml/min/1.73m。与未起始治疗的 106 名生长障碍患儿(8.8 岁,-2.33SD,51ml/min/1.73m)相比。rGH 起始后 30 个月和 42 个月时,身高分别增加了+0.26(95%CI:-0.11,+0.62)和+0.35(95%CI:-0.17,+0.87)SD,与未起始 rGH 的患者相比。rGH 与 GFR 无关。

结论

接受 rGH 的生长障碍患者经历了显著的生长,但与 RCT 相比有所减弱,且更有可能来自高收入家庭,GFR 更低。相当数量的患儿,主要是男孩,尽管没有诊断出生长障碍,但仍接受了 rGH 治疗,且身高达到的最高值相对父母身高中位数较高。由于 rGH 与 GFR 下降速度加快无关,因此增加该人群中 rGH 的使用是合理的。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4106/8938997/d5dff41bace5/nihms-1785994-f0001.jpg

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