Psychopharmacology Clinical Trial Recruitment Challenges in Neurogenetic Syndromes: Lessons from an Open-Label Trial of Fluoxetine in Down Syndrome.

IF 2.2 4区 医学 Q2 PEDIATRICS
Kimaya R Gracias, Laura Sarnie, Michelle Palumbo, Caitlin Ravichandran, Christopher J McDougle, Robyn P Thom
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Abstract

Background: Despite the increased risk for psychopathology in individuals with neurogenetic syndromes, very few psychopharmacology trials have been conducted in these populations. Objectives: The objective of this perspectives article is to describe recruitment challenges and potential solutions for psychopharmacology trials in neurogenetic syndromes. Methods: We describe recruitment challenges and lessons learned from an open-label trial of fluoxetine for the treatment of depression in adults with Down syndrome (DS). These challenges are contrasted with a successful open-label trial of buspirone for the treatment of anxiety in Williams syndrome. Results: Factors that contributed to recruitment challenges include limited research on the clinical presentation of depression in DS and the relatively small target population. This experience highlights the importance of foundational research studies on the phenomenology of target symptoms and burden of disease, as well as disseminating that information to the patient/family community. Partnership with a local family organization with close ties to the patient population can assist in overcoming recruitment barriers. Conclusion: The successes and challenges of early psychopharmacology clinical trials in neurogenetic syndromes should be considered for future trials.

神经遗传综合征的精神药理学临床试验招募挑战:来自氟西汀治疗唐氏综合征的开放标签试验的经验教训。
背景:尽管神经遗传综合征患者的精神病理风险增加,但在这些人群中进行的精神药理学试验很少。目的:这篇前瞻性文章的目的是描述神经遗传综合征的精神药理学试验的招募挑战和潜在的解决方案。方法:我们描述了氟西汀治疗成人唐氏综合征(DS)抑郁症的开放标签试验的招募挑战和经验教训。这些挑战与丁螺环酮治疗威廉姆斯综合征焦虑的成功开放标签试验形成对比。结果:导致招募挑战的因素包括对退行性痴呆患者抑郁症临床表现的研究有限以及相对较小的目标人群。这一经验突出了对目标症状和疾病负担的现象学进行基础研究的重要性,以及向患者/家庭社区传播这些信息的重要性。与与患者群体有密切联系的当地家庭组织建立伙伴关系有助于克服招募障碍。结论:神经遗传综合征早期精神药理学临床试验的成功和挑战应作为今后临床试验的参考。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
3.60
自引率
5.30%
发文量
61
审稿时长
>12 weeks
期刊介绍: Journal of Child and Adolescent Psychopharmacology (JCAP) is the premier peer-reviewed journal covering the clinical aspects of treating this patient population with psychotropic medications including side effects and interactions, standard doses, and research on new and existing medications. The Journal includes information on related areas of medical sciences such as advances in developmental pharmacokinetics, developmental neuroscience, metabolism, nutrition, molecular genetics, and more. Journal of Child and Adolescent Psychopharmacology coverage includes: New drugs and treatment strategies including the use of psycho-stimulants, selective serotonin reuptake inhibitors, mood stabilizers, and atypical antipsychotics New developments in the diagnosis and treatment of ADHD, anxiety disorders, schizophrenia, autism spectrum disorders, bipolar disorder, eating disorders, along with other disorders Reports of common and rare Treatment Emergent Adverse Events (TEAEs) including: hyperprolactinemia, galactorrhea, weight gain/loss, metabolic syndrome, dyslipidemia, switching phenomena, sudden death, and the potential increase of suicide. Outcomes research.
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