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重组人生长激素治疗儿科肠移植受者的疗效和安全性。

Efficacy and safety of recombinant human growth hormone therapy in pediatric intestinal transplant recipients.

机构信息

Pediatric Gastroenterology, Hepatology, and Nutrition, University of Nebraska Medical Center, Omaha, Nebraska, USA.

Pediatric Endocrinology, University of Nebraska Medical Center, Omaha, Nebraska, USA.

出版信息

Pediatr Transplant. 2022 Sep;26(6):e14321. doi: 10.1111/petr.14321. Epub 2022 May 19.

DOI:10.1111/petr.14321
PMID:35587547
Abstract

BACKGROUND

Recombinant human growth hormone (rhGH) is widely used to treat growth retardation in children. We aimed to examine the effect of rhGH therapy on growth and its impact on allograft function in children with growth retardation after intestinal transplant (IT).

METHODS

We retrospectively included children younger than 19 years who had received an IT with or without the liver, were diagnosed with growth retardation, and have received rhGH between January 2010 and January 2021. Changes in the patient's anthropometric parameters between baseline and various time points were compared using the paired t-test or Wilcoxon signed-rank test, as appropriate.

RESULTS

Seven patients (all males and prepubertal) received rhGH for the median duration of 2.3 years. The median age at rhGH start was 9.5 years. The median growth velocity z-score improved from -0.95 before treatment to 2.3 (p = .04) and 1.9 (p = .06) after 1 and 2 years of treatment, respectively. The median height-for-age z-score significantly improved from -3.4 at rhGH start to -1.3 (p = .005) at rhGH stop and remained above baseline at the last visit (-2.4, p = .002). The change in the first-year growth velocity was negatively correlated with the change in the second-year growth velocity (r = -.90, p = .04). No serious adverse effects or worsening allograft function were observed.

CONCLUSIONS

Severely growth retarded children after IT could benefit from rhGH treatment. A larger, longitudinal study would be warranted to monitor the long-term effect and safety of rhGH and examine predictors of growth response to rhGH therapy in these pediatric IT recipients.

摘要

背景

重组人生长激素(rhGH)被广泛用于治疗儿童生长迟缓。我们旨在研究 rhGH 治疗对生长的影响及其对肠移植(IT)后生长迟缓儿童同种异体移植物功能的影响。

方法

我们回顾性纳入了在 2010 年 1 月至 2021 年 1 月期间接受 IT 治疗且伴有或不伴有肝脏的年龄小于 19 岁的儿童患者,这些患者被诊断为生长迟缓,并接受了 rhGH 治疗。使用配对 t 检验或 Wilcoxon 符号秩检验比较基线和各时间点患者人体测量参数的变化,具体取决于数据分布。

结果

7 名患者(均为男性和青春期前)接受 rhGH 治疗,中位时间为 2.3 年。rhGH 起始治疗的中位年龄为 9.5 岁。rhGH 治疗 1 年和 2 年后,生长速度 z 评分的中位数分别从治疗前的-0.95 提高到 2.3(p =.04)和 1.9(p =.06)。rhGH 起始时身高年龄 z 评分中位数从-3.4 显著改善至 rhGH 停药时的-1.3(p =.005),最后一次随访时仍高于基线(-2.4,p =.002)。第一年生长速度的变化与第二年生长速度的变化呈负相关(r = -.90,p =.04)。未观察到严重不良事件或同种异体移植物功能恶化。

结论

IT 后严重生长迟缓的儿童可从 rhGH 治疗中获益。需要进行更大规模的纵向研究来监测 rhGH 的长期效果和安全性,并检查这些儿科 IT 受者对 rhGH 治疗生长反应的预测因素。

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