Supplementary Material for: Pituitary Dysfunction in Pediatric Patients with Optic Nerve Hypoplasia: A Retrospective Cohort Study (1975–2014)

<b><i>Background/Aims:</i></b> The risk factors for pituitary hormone dysfunction (PHD) in children with optic nerve hypoplasia (ONH) are not well understood. This study identified the type, timing, and predictors of PHD in children with ONH. <b><i>Methods:</i></b> ONH patient charts were reviewed retrospectively. The incidence rate of PHD was calculated assuming a Poisson distribution. Predictors of PHD were identified through a multivariable Cox proportional hazards model. <b><i>Results:</i></b> Among 144 subjects with ONH, 49.3% (<i>n</i> = 71) developed PHD over 614.7 person-years of follow-up. The incidence was 11.55 (95% confidence interval [CI]: 9.02–14.57/100 person-years). The median time to first PHD was 2.88 (interquartile range: 0.02–18.72) months. Eighty-two percent developed their first PHD by their 5th and 90% by their 10th birthday, and 89% within 5 years of ONH diagnosis. Prematurity (adjusted hazard ratio [aHR]: 0.33; 95% CI: 0.1–1.07), blindness (aHR: 1.72; 95% CI: 1.03–2.86), maternal substance abuse (aHR: 1.51; 95% CI: 0.91–2.48), abnormal posterior pituitary (aHR: 3.8; 95% CI: 2.01–7.18), and hypoplastic/absent anterior pituitary (aHR: 2.52; 95% CI: 1.29–4.91) were significant predictors of PHD. <b><i>Conclusions:</i></b> The clinical predictors of PHD included blindness, pituitary gland abnormalities, and maternal substance abuse. These predictors help clinical decision-making related to the need for and frequency of hormone testing in pediatric patients with ONH.

**背景/目的：** 儿童视神经发育不全（optic nerve hypoplasia, ONH）患者继发垂体激素功能障碍（pituitary hormone dysfunction, PHD）的危险因素目前尚未明确。本研究旨在明确ONH患儿发生PHD的类型、发病时间及相关预测因素。
**方法：** 本研究对ONH患者的病历资料进行回顾性分析。以泊松分布（Poisson distribution）为假设前提计算PHD的发病率。通过多变量Cox比例风险回归模型（multivariable Cox proportional hazards model）筛选PHD的独立预测因素。
**结果：** 本研究纳入144例ONH患儿，在614.7人-年的随访期间，共49.3%（n=71）的患儿发生PHD。PHD的发病率为11.55（95%置信区间[CI]：9.02~14.57/100人-年）。患儿首次发生PHD的中位时间为2.88个月（四分位间距：0.02~18.72个月）。82%的患儿在5岁前首次出现PHD，90%的患儿在10岁前发病，89%的患儿在ONH确诊后5年内发生PHD。早产（校正后风险比[aHR]：0.33；95%CI：0.1~1.07）、失明（aHR：1.72；95%CI：1.03~2.86）、母亲物质滥用史（aHR：1.51；95%CI：0.91~2.48）、垂体后叶异常（aHR：3.8；95%CI：2.01~7.18）以及垂体前叶发育不全或缺如（aHR：2.52；95%CI：1.29~4.91）均为PHD的独立显著预测因素。
**结论：** 本研究筛选出的PHD临床预测因素包括失明、垂体形态异常及母亲物质滥用史。上述预测因素可为ONH患儿是否需要进行激素检测及检测频率的临床决策提供参考依据。