Endocrinologia Pediatra e Centro, Screening Neonatale, Ospedale Pediatrico Microcitemico "A. Cao", Cagliari, Italy.
University Hospitals Sussex NHS Trust, Royal Alexandra Children's Hospital, Brighton, UK.
Drug Des Devel Ther. 2024 Mar 2;18:667-684. doi: 10.2147/DDDT.S440009. eCollection 2024.
Omnitrope (somatropin) was approved as a biosimilar recombinant human growth hormone (rhGH) in 2006. Here, we report final data from the PAtients TReated with Omnitrope (PATRO) Children study, a post-marketing surveillance study designed to monitor the long-term safety and effectiveness of this treatment in pediatric patients.
The study population included all pediatric patients treated with Omnitrope (biosimilar rhGH), administered via daily injection, in routine clinical practice. The primary objective was to assess long-term safety, with effectiveness assessed as a secondary objective.
In total, 7359 patients were enrolled and treated in the PATRO Children study; 86.0% were treatment-naïve at baseline. Growth hormone deficiency was the most frequent indication (57.9%), followed by patients born small for gestational age (SGA; 26.6%). The mean (SD) duration of exposure to biosimilar rhGH was 3.66 years (2.39). A total of 16,628 adverse events (AEs) were reported in 3981 (54.1%) patients, most of which were mild/moderate. AEs suspected to be treatment related occurred in 8.3% of patients, most frequently headache (1.6%), injection-site pain (1.1%), or injection-site hematoma (1.1%). The incidence rate (IR) of type 2 diabetes mellitus was 0.11 per 1000 person-years (PY) across all patients, and 0.13 per 1000 PY in patients born SGA. The IR of newly diagnosed primary malignancies was 0.22 per 1000 PY across all patients. In the 6589 patients included in the effectiveness population, a sustained catch-up growth was observed across all indications. After 5 years of treatment, height SDS increased from baseline by a median (range) of +1.79 (-3.7 to 6.2) in treatment-naïve patients and +0.73 (-1.4 to 3.7) in pretreated patients.
This final analysis of the PATRO Children study indicates that biosimilar rhGH is well tolerated and effective in real-world clinical practice. These data are consistent with the well-characterized safety profile of rhGH treatment in pediatric patients.
奥米托肽（somatropin）于 2006 年被批准为生物类似重组人生长激素（rhGH）。在这里，我们报告了患者接受奥米托肽治疗（PATRO 儿童）研究的最终数据，这是一项上市后监测研究，旨在监测这种治疗在儿科患者中的长期安全性和有效性。
研究人群包括所有接受奥米托肽（生物类似 rhGH）治疗的儿科患者，每日注射。主要目的是评估长期安全性，次要目的是评估有效性。
共有 7359 名患者参加了 PATRO 儿童研究，并接受了治疗；86.0%的患者在基线时为治疗初治患者。生长激素缺乏症是最常见的适应证（57.9%），其次是出生体重小于胎龄（SGA；26.6%）的患者。接受生物类似 rhGH 治疗的平均（SD）暴露时间为 3.66 年（2.39 年）。3981 名（54.1%）患者共报告了 16628 例不良事件（AE），其中大多数为轻度/中度。8.3%的患者报告了疑似与治疗相关的 AE，最常见的是头痛（1.6%）、注射部位疼痛（1.1%）或注射部位血肿（1.1%）。所有患者的 2 型糖尿病发病率（IR）为 0.11/1000 人年（PY），SGA 出生的患者为 0.13/1000 PY。所有患者的新发原发性恶性肿瘤发病率（IR）为 0.22/1000 PY。在有效性人群中的 6589 名患者中，所有适应证均观察到持续追赶生长。治疗 5 年后，初治患者身高 SDS 从基线增加中位数（范围）+1.79（-3.7 至 6.2），预处理患者增加+0.73（-1.4 至 3.7）。
PATRO 儿童研究的最终分析表明，生物类似 rhGH 在真实世界的临床实践中具有良好的耐受性和有效性。这些数据与儿科患者 rhGH 治疗的良好特征安全性概况一致。
