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<Emphasis Type="BoldItalic">What is known</Emphasis> <p><UnorderedList Mark="Bullet"> <ItemContent> <p>So far, observational studies have primarily examined adults and have used these findings to define endpoints for interventional studies, which also serve as a reference for interventional studies in children</p> </ItemContent> <ItemContent> <p>&#xa0;Most clinical trials currently use the measurement of definitely decreased autofluorescence</p> </ItemContent> </UnorderedList></p> <Emphasis Type="BoldItalic">What is new</Emphasis> <p><UnorderedList Mark="Bullet"> <ItemContent> <p>We have closely examined the variability of Stargardt disease in children</p> </ItemContent> <ItemContent> <p>&#xa0;Our results suggest that the same inclusion criteria and endpoints used for adults should not be applied to children, e.g. definitely decreased autofluorescence is not suitable in pediatric cohorts because it has only a very low prevalence</p> </ItemContent> </UnorderedList></p>

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ABCA4-associated disease in childhood and adolescence– a phenotype study

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年7月11日

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  • Jan-Philipp Bodenbender,
  • Annekatrin Rickmann,
  • Katarina Stingl,
  • Susanne Kohl,
  • Laura Kühlewein

摘要

What is known

So far, observational studies have primarily examined adults and have used these findings to define endpoints for interventional studies, which also serve as a reference for interventional studies in children

 Most clinical trials currently use the measurement of definitely decreased autofluorescence

What is new

We have closely examined the variability of Stargardt disease in children

 Our results suggest that the same inclusion criteria and endpoints used for adults should not be applied to children, e.g. definitely decreased autofluorescence is not suitable in pediatric cohorts because it has only a very low prevalence