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Purpose <p>To investigate the morphological changes and long-term progression of optic disc peripapillary β-zone atrophy (β-PPA) in children with type 1 diabetes over a 3-year period, and to assess the independent predictive value of serum biochemical markers for β-PPA progression.</p> Methods <p>In this observational clinical cohort study, 24 children (24 eyes) with type 1 diabetes who met the inclusion criteria were followed for 3 years. At baseline and at follow-up, all subjects underwent comprehensive ophthalmic examinations (including uncorrected and best-corrected visual acuity, autorefraction, axial length measurement, and OCT imaging). Standardized methods were used on fundus images to identify and quantify the β-PPA area and the optic disc’s ellipticity index. Baseline demographic data, duration of diabetes, BMI, and laboratory parameters (including HbA1c, liver and renal function tests, bilirubin, etc.) were recorded. Paired t-tests (or Wilcoxon signed-rank tests when appropriate) were used to compare continuous variables between baseline and follow-up, and the percentage change in β-PPA area was calculated. Potential influencing factors were first screened by univariable linear regression; variables with significance or clinical relevance were then entered into a multivariate stepwise linear regression model to identify independent predictors. Identified predictors were dichotomized at their median values, and inter-group comparisons and risk stratification analyses were performed.</p> Results <p>After 3 years of follow-up, the mean axial length and β-PPA area significantly increased, and the spherical equivalent (SE) refraction shifted significantly toward myopia (<i>P</i> &lt; 0.001). Univariable analysis showed that the percentage change in axial length (AL_change%, which is positively correlated) and a more hyperopic (higher) baseline SE were both associated with a greater relative increase in β-PPA area, whereas higher baseline direct bilirubin (DBil) was associated with a smaller increase in β-PPA (all <i>P</i> &lt; 0.0001). Other factors negatively correlated with β-PPA increase included higher baseline BMI, total bilirubin (TBil), indirect bilirubin (UBil), and serum creatinine. In multivariate analysis, AL_change% was a positive independent predictor of β-PPA area growth, and baseline DBil was a negative independent predictor (adjusted R²=0.703; AL_change%: B = 13.55, <i>p</i> &lt; 0.001; DBil: B=–20.31, <i>p</i> = 0.002). When subjects were divided into four groups based on median baseline DBil and AL_change%, the group with low DBil and high AL_change% had the largest relative β-PPA increase, whereas the high-DBil/low-AL_change% group had the smallest (one-way ANOVA, <i>P</i> &lt; 0.001).</p> Conclusion <p>Serum direct bilirubin level, when considered together with axial elongation, may help identify children at higher risk for rapid β-PPA progression at an early stage, pending confirmation in larger and independent cohorts.</p>

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Morphological changes of β-Zone Peripapillary Atrophy (β-PPA) in children with type 1 Diabetes: A longitudinal study

  • 期刊论文

  • 发表日期：

    2026年2月24日

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  • Shuchang Zhang,
  • Chenhao Yang,
  • Qingyu An,
  • Lei Zhong,
  • Xixuan Zhao,
  • Xi Qu,
  • Yiwei Wu,
  • Haidong Zou

摘要

Purpose

To investigate the morphological changes and long-term progression of optic disc peripapillary β-zone atrophy (β-PPA) in children with type 1 diabetes over a 3-year period, and to assess the independent predictive value of serum biochemical markers for β-PPA progression.

Methods

In this observational clinical cohort study, 24 children (24 eyes) with type 1 diabetes who met the inclusion criteria were followed for 3 years. At baseline and at follow-up, all subjects underwent comprehensive ophthalmic examinations (including uncorrected and best-corrected visual acuity, autorefraction, axial length measurement, and OCT imaging). Standardized methods were used on fundus images to identify and quantify the β-PPA area and the optic disc’s ellipticity index. Baseline demographic data, duration of diabetes, BMI, and laboratory parameters (including HbA1c, liver and renal function tests, bilirubin, etc.) were recorded. Paired t-tests (or Wilcoxon signed-rank tests when appropriate) were used to compare continuous variables between baseline and follow-up, and the percentage change in β-PPA area was calculated. Potential influencing factors were first screened by univariable linear regression; variables with significance or clinical relevance were then entered into a multivariate stepwise linear regression model to identify independent predictors. Identified predictors were dichotomized at their median values, and inter-group comparisons and risk stratification analyses were performed.

Results

After 3 years of follow-up, the mean axial length and β-PPA area significantly increased, and the spherical equivalent (SE) refraction shifted significantly toward myopia (P < 0.001). Univariable analysis showed that the percentage change in axial length (AL_change%, which is positively correlated) and a more hyperopic (higher) baseline SE were both associated with a greater relative increase in β-PPA area, whereas higher baseline direct bilirubin (DBil) was associated with a smaller increase in β-PPA (all P < 0.0001). Other factors negatively correlated with β-PPA increase included higher baseline BMI, total bilirubin (TBil), indirect bilirubin (UBil), and serum creatinine. In multivariate analysis, AL_change% was a positive independent predictor of β-PPA area growth, and baseline DBil was a negative independent predictor (adjusted R²=0.703; AL_change%: B = 13.55, p < 0.001; DBil: B=–20.31, p = 0.002). When subjects were divided into four groups based on median baseline DBil and AL_change%, the group with low DBil and high AL_change% had the largest relative β-PPA increase, whereas the high-DBil/low-AL_change% group had the smallest (one-way ANOVA, P < 0.001).

Conclusion

Serum direct bilirubin level, when considered together with axial elongation, may help identify children at higher risk for rapid β-PPA progression at an early stage, pending confirmation in larger and independent cohorts.