Iughetti Lorenzo, Antoniazzi Franco, Giavoli Claudia, Bellone Simonetta, Aversa Tommaso, Guazzarotti Laura, Street Maria Elisabeth, Miraglia Del Giudice Emanuele, Persani Luca, Pozzobon Gabriella, Ragusa Letizia, Stagi Stefano, Tornese Gianluca, Zecchino Clara, Mameli Chiara, Zecchi Emiliano, Fedeli Paolo, Zabransky Markus, Lucaccioni Laura, Zucchini Stefano
Pediatric Unit, Department of Medical and Surgical Sciences for Mother, Children and Adults, University of Modena and Reggio Emilia, Modena, Italy.
Pediatric Clinic C, Department of Surgical Sciences, Dentistry, Gynecology and Pediatrics, University of Verona, Verona, Italy.
Endocrine. 2025 Mar;87(3):933-942. doi: 10.1007/s12020-024-04090-x. Epub 2024 Nov 29.
Omnitrope (a somatropin biosimilar), used to treat growth disturbances, is considered to have a good safety profile in children. Here, we present the analysis of final data of the Italian cohort of the PAtients TReated with Omnitrope (PATRO) Children study.
This multicenter, open-label, longitudinal, post-marketing surveillance study enrolled eligible children during 2010-2018. The primary objective was to assess the long-term safety of Omnitrope by recording all adverse events (AEs), serious AEs, and adverse drug reactions (ADRs). A secondary objective was to evaluate the long-term effectiveness of Omnitrope using height measurements.
A total of 375 patients were included in the Italian cohort of the PATRO Children study. After a mean ± standard deviation (SD) follow-up duration of 40.9 ± 24.6 months, 607 AEs were reported in 58.4% of patients, mostly of mild (52.5%) or moderate (15.7%) severity. The most common AEs were headache (11.7%), elevated insulin-like growth factor (IGF)-1 (4.8%), abdominal pain (4.3%), and pyrexia (3.7%). Sixty-seven ADRs occurred in 52 patients (13.9%); the most common ADRs were elevated IGF-1 (3.5%) and insulin resistance (2.9%). Mean ± SD height standard deviation scores in treatment-naïve patients increased from -2.5 ± 0.7 at baseline (n = 318) to -1.3 ± 0.7 at 5 years (n = 56) and to -0.8 ± 0.7 at 7.5 years (n = 13).
This final analysis extends the interim analysis findings from the PATRO Children study and confirms the long-term safety and effectiveness of Omnitrope in Italian pediatric patients with growth disturbances.
Omnitrope（一种生长激素类似物）用于治疗生长障碍，被认为在儿童中具有良好的安全性。在此，我们展示了接受Omnitrope治疗的意大利儿童患者队列（PATRO）研究的最终数据分析。
这项多中心、开放标签、纵向、上市后监测研究在2010年至2018年期间招募了符合条件的儿童。主要目标是通过记录所有不良事件（AE）、严重不良事件和药物不良反应（ADR）来评估Omnitrope的长期安全性。次要目标是使用身高测量来评估Omnitrope的长期有效性。
PATRO儿童研究的意大利队列共纳入375例患者。在平均随访时间为40.9±24.6个月后，58.4%的患者报告了607例AE，大多为轻度（52.5%）或中度（15.7%）严重程度。最常见的AE是头痛（11.7%）、胰岛素样生长因子（IGF）-1升高（4.8%）、腹痛（4.3%）和发热（3.7%）。52例患者（13.9%）发生了67例ADR；最常见的ADR是IGF-1升高（3.5%）和胰岛素抵抗（2.9%）。初治患者的平均±标准差身高标准差分数从基线时的-2.5±0.7（n = 318）增加到5岁时的-1.3±0.7（n = 56），并在7.5岁时增加到-0.8±0.7（n = 13）。
这项最终分析扩展了PATRO儿童研究的中期分析结果，并证实了Omnitrope在意大利患有生长障碍的儿科患者中的长期安全性和有效性。
