小于胎龄儿出生后使用奥曲肽治疗终止后碳水化合物代谢及不良事件的长期随访
Long-term follow up of carbohydrate metabolism and adverse events after termination of Omnitrope® treatment in children born small for gestational age.
作者信息
Walczak Mieczyslaw, Szalecki Mieczyslaw, Horneff Gerd, Lebl Jan, Kalina-Faska Barbara, Giemza Tomasz, Moldovanu Florentina, Nanu Michaela, Zouater Hichem
机构信息
Department of Pediatrics, Endocrinology, Diabetology, Metabolic Diseases and Cardiology of the Developmental Age, Pomeranian Medical University, Szczecin, Zachodniopomorskie, Poland.
Collegium Medicum UJK, Kielce, Children's Memorial Health Institute, Warsaw, Poland.
出版信息
Ther Adv Endocrinol Metab. 2021 May 5;12:20420188211013121. doi: 10.1177/20420188211013121. eCollection 2021.
BACKGROUND
Recombinant human growth hormone (rhGH) therapy can affect carbohydrate metabolism and lead to impaired glucose tolerance during treatment. In addition, short children born small for gestational age (SGA) are predisposed to metabolic abnormalities. This study assessed the long-term safety of rhGH (Omnitrope®) use in short children born SGA.
METHODS
This was a follow-up observational study of patients from a phase IV study. The baseline visit was the final visit of the phase IV study. Further visits were planned after 6 months (F1), 1 year (F2), 5 years (F3), and 10 years (F4). The primary objective was to evaluate the long-term effect of rhGH treatment on the development of diabetes mellitus; secondary objectives included incidence/severity of adverse events (AEs).
RESULTS
In total, 130 subjects were enrolled in the follow-up study; 99 completed F1, 88 completed F2, and 13 completed F3 (no subject reached F4). The full analysis set for evaluation comprised 118 patients (64 female). Mean (standard deviation) duration of follow up was 39.6 (24.4) months. No subject was newly diagnosed with diabetes. The results for carbohydrate metabolism parameters were consistent with this finding. A total of 144 AEs were reported in 54 subjects; these were mostly of mild-to-moderate intensity (96.5%) and not suspected to be related to previous rhGH treatment (94.4%). Serious AEs ( = 18) were reported in eight patients; three (in one patient) were suspected as possibly related to previous rhGH treatment (anemia, menorrhagia, oligomenorrhoea). One fatal event occurred (sepsis), which was judged as not related to previous rhGH treatment.
CONCLUSIONS
None of the participating subjects, who had all been previously treated with Omnitrope® in a phase IV study, developed diabetes during this follow-up study. In addition, no other unexpected or concerning safety signals were observed.
背景
重组人生长激素（rhGH）治疗可能会影响碳水化合物代谢，并导致治疗期间葡萄糖耐量受损。此外，小于胎龄儿（SGA）出生的矮小儿童易患代谢异常。本研究评估了rhGH（奥曲肽®）用于SGA出生的矮小儿童的长期安全性。
方法
这是一项对IV期研究患者的随访观察性研究。基线访视是IV期研究的最后一次访视。计划在6个月（F1）、1年（F2）、5年（F3）和10年（F4）后进行进一步访视。主要目的是评估rhGH治疗对糖尿病发生的长期影响；次要目的包括不良事件（AE）的发生率/严重程度。
结果
共有130名受试者参加了随访研究；99人完成了F1，88人完成了F2，13人完成了F3（无受试者达到F4）。用于评估的完整分析集包括118名患者（64名女性）。平均（标准差）随访时间为39.6（24.4）个月。无受试者新诊断为糖尿病。碳水化合物代谢参数的结果与此发现一致。54名受试者共报告了144例AE；这些大多为轻度至中度强度（96.5%），且不怀疑与先前的rhGH治疗有关（94.4%）。8名患者报告了严重AE（=18）；3例（1名患者）怀疑可能与先前的rhGH治疗有关（贫血、月经过多、月经过少）。发生了1例致命事件（败血症），判定与先前的rhGH治疗无关。
结论
在这项随访研究中，所有在IV期研究中先前均接受过奥曲肽®治疗的参与受试者均未患糖尿病。此外，未观察到其他意外或令人担忧的安全信号。
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