Reproducibility of cognitive endpoints in clinical trials: lessons from neurofibromatosis type 1.,Annals of Clinical and Translational Neurology

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Reproducibility of cognitive endpoints in clinical trials: lessons from neurofibromatosis type 1.
Annals of Clinical and Translational Neurology ( IF 5.3 ) Pub Date : 2019-12-03 , DOI: 10.1002/acn3.50952
Jonathan M Payne _{1,

2} , Stephen J C Hearps ₁ , Karin S Walsh ₃ , Iris Paltin ₄ , Belinda Barton _{5,

6,

7} , Nicole J Ullrich ₈ , Kristina M Haebich ₁ , David Coghill _{1,

2} , Gerard A Gioia ₃ , Alan Cantor ₉ , Gary Cutter ₁₀ , James H Tonsgard ₁₁ , David Viskochil ₁₂ , Celiane Rey-Casserly ₁₃ , Elizabeth K Schorry ₁₄ , Joseph D Ackerson ₁₅ , Laura Klesse ₁₆ , Michael J Fisher ₄ , David H Gutmann ₁₇ , Tena Rosser ₁₈ , Roger J Packer ₃ , Bruce Korf ₁₉ , Maria T Acosta _{3,

20} , Kathryn N North _{1,

2} ,

Affiliation

Murdoch Children's Research Institute, Royal Children's Hospital, Parkville, Victoria, Australia.
Department of Paediatrics, Faculty of Medicine, Dentistry and Health Sciences, University of Melbourne, Melbourne, Victoria, Australia.
Center for Neuroscience and Behavioral Medicine, Children's National Health System, Washington, DC.
Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Kids Neuroscience Centre, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.
Children's Hospital Education Research Institute, The Children's Hospital at Westmead, Westmead, New South Wales, Australia.
The University of Sydney Children's Hospital Westmead Clinical School, University of Sydney, Westmead, New South Wales, Australia.
Department of Neurology, Boston Children's Hospital, Boston, Massachusetts.
Department of Preventative Medicine, University of Alabama at Birmingham, Birmingham, Alabama.
School of Public Health, University of Alabama at Birmingham, Birmingham, Alabama.
Division of Neurology, The University of Chicago Medicine Comer Children's Hospital, Chicago, Illinois.
Department of Genetics, University of Utah, Salt Lake City, Utah.
Center for Neuropsychology, Boston Children's Hospital, Boston, Massachusetts.
Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
Department of Psychology, University of Alabama at Birmingham, Birmingham, Alabama.
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas.
Department of Neurology, Washington University School of Medicine, St Louis, Missouri.
Department of Neurology, Children's Hospital of Los Angeles, Los Angeles, California.
Department of Genetics, University of Alabama at Birmingham, Birmingham, Alabama.
National Institutes of Health, National Human Genome Research Institute, Bethesda, Maryland.

Rapid developments in understanding the molecular mechanisms underlying cognitive deficits in neurodevelopmental disorders have increased expectations for targeted, mechanism‐based treatments. However, translation from preclinical models to human clinical trials has proven challenging. Poor reproducibility of cognitive endpoints may provide one explanation for this finding. We examined the suitability of cognitive outcomes for clinical trials in children with neurofibromatosis type 1 (NF1) by examining test‐retest reliability of the measures and the application of data reduction techniques to improve reproducibility.

中文翻译：

临床试验中认知终点的可重复性：1型神经纤维瘤病的经验教训。

对神经发育障碍认知缺陷背后的分子机制的理解的迅速发展，提高了对针对性，基于机制的治疗的期望。但是，从临床前模型到人体临床试验的转换已被证明具有挑战性。认知终点的可重复性差可能为这一发现提供一种解释。我们通过检查测试的重测信度的可靠性以及应用数据缩减技术来提高可重复性，来检验认知结局在1型神经纤维瘤病（NF1）儿童临床试验中的适用性。

更新日期：2019-12-03

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